Ignite Creation Date:
2025-12-25 @ 4:46 AM
Ignite Modification Date:
2026-02-23 @ 5:16 AM
Study NCT ID:
NCT02750618
Status:
COMPLETED
Last Update Posted:
2024-05-06
First Post:
2015-12-06
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D053098', 'term': 'Familial Hypophosphatemic Rickets'}], 'ancestors': [{'id': 'D063730', 'term': 'Rickets, Hypophosphatemic'}, {'id': 'D012279', 'term': 'Rickets'}, {'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D007015', 'term': 'Hypophosphatemia, Familial'}, {'id': 'D015499', 'term': 'Renal Tubular Transport, Inborn Errors'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D008664', 'term': 'Metal Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D002128', 'term': 'Calcium Metabolism Disorders'}, {'id': 'D017674', 'term': 'Hypophosphatemia'}, {'id': 'D010760', 'term': 'Phosphorus Metabolism Disorders'}, {'id': 'D014808', 'term': 'Vitamin D Deficiency'}, {'id': 'D001361', 'term': 'Avitaminosis'}, {'id': 'D003677', 'term': 'Deficiency Diseases'}, {'id': 'D044342', 'term': 'Malnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000601956', 'term': 'burosumab'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ultragenyx.com', 'phone': '1-888-756-8657', 'title': 'Medical Information', 'organization': 'Ultragenyx Pharmaceutical Inc'}, 'certainAgreement': {'otherDetails': 'The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose of study drug through the end of the study (at Week 160). Maximum duration of exposure to study drug was 160 weeks.', 'eventGroups': [{'id': 'EG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 13, 'seriousNumAtRisk': 13, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Skull Malformation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Syringomyelia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Deafness Unilateral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Ear Haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Ear Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Excessive Cerumen Production', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hypoacusis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Motion Sickness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Otorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Ocular Hyperaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Abdominal Discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Abdominal Pain Upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Aphthous Ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dental Caries', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Epigastric Discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gingival Blister', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gingival Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gingival Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Haematochezia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Loose Tooth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Oral Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Post-Tussive Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Stomatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Teething', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Vomiting Projectile', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Injection Site Bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Injection Site Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Injection Site Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Injection Site Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Injection Site Reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Non-Cardiac Chest Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 11}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Vaccination Site Reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Food Allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Atypical Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Croup Infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Ear Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Enterobiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Eye Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gastrointestinal Viral Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Genital Candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Gingival Abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hand-Foot-And-Mouth Disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Impetigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Molluscum Contagiosum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Otitis Media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pharyngitis Streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth Abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 10}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Urinary Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Viral Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Viral Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Arthropod Bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Lip Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Skin Abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth Fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Tooth Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Traumatic Tooth Displacement', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Amylase Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Blood 25-Hydroxycholecalciferol Decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Blood Parathyroid Hormone Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Blood Phosphorus Decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Heart Rate Abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Lipase Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Vitamin D Decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'White Blood Cell Count Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Decreased Appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Increased Appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Polydipsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Back Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Bone Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Foot Deformity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Knee Deformity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Myalgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pain In Extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 9}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dysarthria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Hypersomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Periodic Limb Movement Disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dysuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Polyuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Urinary Incontinence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Penile Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Penile Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Adenoidal Disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Chronic Throat Clearing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 11}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Nasal Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 8}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Nasal Discharge Discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Oropharyngeal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Productive Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Respiratory Tract Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 7}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Sinus Congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Sneezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Throat Irritation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Wheezing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Dermatitis Contact', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Rash Papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Skin Disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Swelling Face', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 4}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Urticaria Papular', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}], 'seriousEvents': [{'term': 'Tooth Abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 18.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline at Week 40 in Serum Phosphorus', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.96', 'spread': '0.117', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Least Squares Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.96', 'ciLowerLimit': '0.73', 'ciUpperLimit': '1.19', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Week 40', 'description': 'The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic/Pharmacodynamic (PK/PD) Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Adverse Event Starting during Screening Period', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'TEAEs', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Related TEAEs', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Serious Related TEAEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Grade 3 or 4 TEAE', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Leading to Study Discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Leading to Treatment Discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Leading to Death', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks.', 'description': 'An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Set: all participants who received at least one dose of study drug.'}, {'type': 'SECONDARY', 'title': 'Radiographic Global Impression of Change (RGI-C) Score at Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.21', 'spread': '0.071', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.21', 'ciLowerLimit': '2.07', 'ciUpperLimit': '2.35', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Week 40', 'description': 'Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'RGI-C Score at Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.23', 'spread': '0.111', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.23', 'ciLowerLimit': '2.01', 'ciUpperLimit': '2.45', 'groupDescription': 'The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. The least squares (LS) mean, standard error (SE), 95% confidence interval (CI) and 2-sided p-value are from the GEE model.', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Week 64', 'description': 'Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.75', 'spread': '0.116', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-1.75', 'ciLowerLimit': '-1.98', 'ciUpperLimit': '-1.53', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Week 40', 'description': 'The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-2.02', 'spread': '0.115', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-2.02', 'ciLowerLimit': '-2.25', 'ciUpperLimit': '-1.80', 'pValueComment': 'The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Week 64', 'description': 'The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'RGI-C Lower Limb Deformity Score at Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.21', 'spread': '0.155', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.21', 'ciLowerLimit': '0.90', 'ciUpperLimit': '1.51', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Week 40', 'description': 'Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'RGI-C Lower Limb Deformity Score at Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.51', 'spread': '0.123', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.51', 'ciLowerLimit': '1.27', 'ciUpperLimit': '1.76', 'pValueComment': 'The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. The LS Mean, SE, 95% CI and 2-sided p-value are from the GEE model.', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Week 64', 'description': 'Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in Recumbent Length/Standing Height', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.44', 'spread': '2.009', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.52', 'spread': '1.518', 'groupId': 'OG000'}]}]}, {'title': 'Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.29', 'spread': '2.451', 'groupId': 'OG000'}]}]}, {'title': 'Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.22', 'spread': '3.157', 'groupId': 'OG000'}]}]}, {'title': 'Week 88', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.30', 'spread': '3.400', 'groupId': 'OG000'}]}]}, {'title': 'Week 112', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '13.25', 'spread': '3.724', 'groupId': 'OG000'}]}]}, {'title': 'Week 136', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '15.41', 'spread': '3.699', 'groupId': 'OG000'}]}]}, {'title': 'Week 160', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '18.96', 'spread': '4.206', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160', 'unitOfMeasure': 'cm', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.082', 'spread': '0.4964', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.208', 'spread': '0.4540', 'groupId': 'OG000'}]}]}, {'title': 'Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.276', 'spread': '0.6647', 'groupId': 'OG000'}]}]}, {'title': 'Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.264', 'spread': '0.8755', 'groupId': 'OG000'}]}]}, {'title': 'Week 88', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.212', 'spread': '0.9115', 'groupId': 'OG000'}]}]}, {'title': 'Week 112', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.174', 'spread': '0.9273', 'groupId': 'OG000'}]}]}, {'title': 'Week 136', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.321', 'spread': '0.9123', 'groupId': 'OG000'}]}]}, {'title': 'Week 160', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.172', 'spread': '0.9048', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160', 'description': "Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.", 'unitOfMeasure': 'Z score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Percentiles', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.006', 'spread': '11.6149', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.940', 'spread': '13.1323', 'groupId': 'OG000'}]}]}, {'title': 'Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.283', 'spread': '20.1675', 'groupId': 'OG000'}]}]}, {'title': 'Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.980', 'spread': '23.4412', 'groupId': 'OG000'}]}]}, {'title': 'Week 88', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.155', 'spread': '23.9126', 'groupId': 'OG000'}]}]}, {'title': 'Week 112', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.000', 'spread': '24.7569', 'groupId': 'OG000'}]}]}, {'title': 'Week 136', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-7.026', 'spread': '24.8583', 'groupId': 'OG000'}]}]}, {'title': 'Week 160', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.628', 'spread': '25.5113', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160', 'unitOfMeasure': 'percentiles', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline Over Time in Serum Alkaline Phosphatase (ALP)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-82.91', 'spread': '23.348', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-83.84', 'spread': '45.263', 'groupId': 'OG000'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-161.38', 'spread': '12.924', 'groupId': 'OG000'}]}]}, {'title': 'Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-214.99', 'spread': '13.628', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-226.58', 'spread': '11.491', 'groupId': 'OG000'}]}]}, {'title': 'Week 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-216.45', 'spread': '16.165', 'groupId': 'OG000'}]}]}, {'title': 'Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-216.76', 'spread': '12.705', 'groupId': 'OG000'}]}]}, {'title': 'Week 76', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-231.22', 'spread': '15.964', 'groupId': 'OG000'}]}]}, {'title': 'Week 88', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-237.78', 'spread': '12.837', 'groupId': 'OG000'}]}]}, {'title': 'Week 100', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-218.14', 'spread': '15.340', 'groupId': 'OG000'}]}]}, {'title': 'Week 112', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-233.91', 'spread': '11.098', 'groupId': 'OG000'}]}]}, {'title': 'Week 124', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-252.22', 'spread': '8.312', 'groupId': 'OG000'}]}]}, {'title': 'Week 136', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-267.89', 'spread': '13.124', 'groupId': 'OG000'}]}]}, {'title': 'Week 148', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-248.05', 'spread': '12.653', 'groupId': 'OG000'}]}]}, {'title': 'Week 160', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-248.47', 'spread': '10.990', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.0004', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-82.91', 'ciLowerLimit': '-128.68', 'ciUpperLimit': '-37.15', 'groupDescription': 'Week 4', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '0.0640', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-83.84', 'ciLowerLimit': '-172.55', 'ciUpperLimit': '4.88', 'groupDescription': 'Week 12', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-161.38', 'ciLowerLimit': '-186.70', 'ciUpperLimit': '-136.05', 'groupDescription': 'Week 20', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-214.99', 'ciLowerLimit': '-241.70', 'ciUpperLimit': '-188.28', 'groupDescription': 'Week 40', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-226.58', 'ciLowerLimit': '-249.11', 'ciUpperLimit': '-204.06', 'groupDescription': 'Week 48', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-216.45', 'ciLowerLimit': '-248.13', 'ciUpperLimit': '-184.77', 'groupDescription': 'Week 56', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'Difference in LS Means', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-216.76', 'ciLowerLimit': '-241.66', 'ciUpperLimit': '-191.86', 'groupDescription': 'Week 64', 'statisticalMethod': 'GEE model', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-231.22', 'ciLowerLimit': '-262.51', 'ciUpperLimit': '-199.93', 'groupDescription': 'Week 76', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-237.78', 'ciLowerLimit': '-262.94', 'ciUpperLimit': '-212.62', 'groupDescription': 'Week 88', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-218.14', 'ciLowerLimit': '-248.21', 'ciUpperLimit': '-188.08', 'groupDescription': 'Week 100', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-233.91', 'ciLowerLimit': '-255.66', 'ciUpperLimit': '-212.16', 'groupDescription': 'Week 112', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-252.22', 'ciLowerLimit': '-268.51', 'ciUpperLimit': '-235.93', 'groupDescription': 'Week 124', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-267.89', 'ciLowerLimit': '-293.61', 'ciUpperLimit': '-242.16', 'groupDescription': 'Week 136', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-248.05', 'ciLowerLimit': '-272.85', 'ciUpperLimit': '-223.25', 'groupDescription': 'Week 148', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}, {'pValue': '< 0.0001', 'groupIds': ['OG000'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-248.47', 'ciLowerLimit': '-270.01', 'ciUpperLimit': '-226.93', 'groupDescription': 'Week 160', 'statisticalMethod': 'GEE', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160', 'description': 'The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.', 'unitOfMeasure': 'U/L', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'PK/PD Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples at given time point.'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline Over Time in Serum ALP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'classes': [{'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-12.47', 'spread': '16.620', 'groupId': 'OG000'}]}]}, {'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-5.00', 'spread': '60.124', 'groupId': 'OG000'}]}]}, {'title': 'Week 20', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-24.76', 'spread': '18.883', 'groupId': 'OG000'}]}]}, {'title': 'Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-36.25', 'spread': '12.787', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-37.29', 'spread': '13.936', 'groupId': 'OG000'}]}]}, {'title': 'Week 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-35.80', 'spread': '16.568', 'groupId': 'OG000'}]}]}, {'title': 'Week 64', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-35.95', 'spread': '14.851', 'groupId': 'OG000'}]}]}, {'title': 'Week 76', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-38.36', 'spread': '15.621', 'groupId': 'OG000'}]}]}, {'title': 'Week 88', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-39.08', 'spread': '12.987', 'groupId': 'OG000'}]}]}, {'title': 'Week 100', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-37.42', 'spread': '12.466', 'groupId': 'OG000'}]}]}, {'title': 'Week 112', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-39.05', 'spread': '13.808', 'groupId': 'OG000'}]}]}, {'title': 'Week 124', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-43.11', 'spread': '12.493', 'groupId': 'OG000'}]}]}, {'title': 'Week 136', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-47.01', 'spread': '11.752', 'groupId': 'OG000'}]}]}, {'title': 'Week 148', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-43.47', 'spread': '11.321', 'groupId': 'OG000'}]}]}, {'title': 'Week 160', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-42.35', 'spread': '13.574', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160', 'unitOfMeasure': 'percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK/PD Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples at given time point.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 160 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'Completed Week 40', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'Completed Week 64', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'preAssignmentDetails': 'The study enrolled pediatric participants between 1 and 4 years old, inclusive, with clinical findings consistent with XLH including hypophosphatemia and radiographic evidence of rickets, and a confirmed PHEX mutation or variant of uncertain significance.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Burosumab Q2W', 'description': 'Burosumab SC injections Q2W for a total of 160 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '2.94', 'spread': '1.146', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '11', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}, {'title': 'Black or African American', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Serum Phosphorus', 'classes': [{'categories': [{'measurements': [{'value': '2.51', 'spread': '0.284', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'mg/dL', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Rickets Severity Score (RSS) Total Score', 'classes': [{'categories': [{'measurements': [{'value': '2.92', 'spread': '1.367', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Recumbent length/Standing height', 'classes': [{'categories': [{'measurements': [{'value': '89.15', 'spread': '7.597', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Growth is measured by recumbent length for subjects \\< 2 years of age or those unable or unwilling to stand for the measurement.', 'unitOfMeasure': 'cm', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Recumbent length/Standing height (Z score)', 'classes': [{'categories': [{'measurements': [{'value': '-1.378', 'spread': '1.1947', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': "Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the Centers for Disease Control \\[CDC\\] growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome.", 'unitOfMeasure': 'Z score', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Recumbent length/Standing height (percentile)', 'classes': [{'categories': [{'measurements': [{'value': '18.044', 'spread': '25.2644', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'percentile', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Serum Alkaline Phosphatase (ALP)', 'classes': [{'categories': [{'measurements': [{'value': '548.5', 'spread': '193.80', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'U/L', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-06-12', 'size': 2470329, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_002.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-02-19T14:13', 'hasProtocol': True}, {'date': '2016-11-29', 'size': 1943267, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2018-04-19T14:24', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-05-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT03775187', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2024-05', 'completionDateStruct': {'date': '2019-09-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-05-02', 'studyFirstSubmitDate': '2015-12-06', 'resultsFirstSubmitDate': '2018-04-19', 'studyFirstSubmitQcDate': '2016-04-20', 'lastUpdatePostDateStruct': {'date': '2024-05-06', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2018-05-18', 'studyFirstPostDateStruct': {'date': '2016-04-25', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2018-06-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-04-20', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline at Week 40 in Serum Phosphorus', 'timeFrame': 'Baseline, Week 40', 'description': 'The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.'}, {'measure': 'Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation', 'timeFrame': 'From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks.', 'description': 'An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death).'}], 'secondaryOutcomes': [{'measure': 'Radiographic Global Impression of Change (RGI-C) Score at Week 40', 'timeFrame': 'Week 40', 'description': 'Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.'}, {'measure': 'RGI-C Score at Week 64', 'timeFrame': 'Week 64', 'description': 'Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.'}, {'measure': 'Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score', 'timeFrame': 'Baseline, Week 40', 'description': 'The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.'}, {'measure': 'Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score', 'timeFrame': 'Baseline, Week 64', 'description': 'The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.'}, {'measure': 'RGI-C Lower Limb Deformity Score at Week 40', 'timeFrame': 'Week 40', 'description': 'Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates.'}, {'measure': 'RGI-C Lower Limb Deformity Score at Week 64', 'timeFrame': 'Week 64', 'description': 'Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure.'}, {'measure': 'Change From Baseline Over Time in Recumbent Length/Standing Height', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160'}, {'measure': 'Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160', 'description': "Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome."}, {'measure': 'Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Percentiles', 'timeFrame': 'Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160'}, {'measure': 'Change From Baseline Over Time in Serum Alkaline Phosphatase (ALP)', 'timeFrame': 'Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160', 'description': 'The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure.'}, {'measure': 'Percent Change From Baseline Over Time in Serum ALP', 'timeFrame': 'Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True}, 'conditionsModule': {'conditions': ['X-Linked Hypophosphatemia']}, 'referencesModule': {'references': [{'pmid': '32721016', 'type': 'DERIVED', 'citation': 'Mao M, Carpenter TO, Whyte MP, Skrinar A, Chen CY, San Martin J, Rogol AD. Growth Curves for Children with X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2020 Oct 1;105(10):3243-9. doi: 10.1210/clinem/dgaa495.'}, {'pmid': '30638856', 'type': 'DERIVED', 'citation': 'Whyte MP, Carpenter TO, Gottesman GS, Mao M, Skrinar A, San Martin J, Imel EA. Efficacy and safety of burosumab in children aged 1-4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial. Lancet Diabetes Endocrinol. 2019 Mar;7(3):189-199. doi: 10.1016/S2213-8587(18)30338-3. Epub 2019 Jan 9.'}]}, 'descriptionModule': {'briefSummary': 'The primary objectives of the study are to:\n\n* Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old\n* Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers that reflect the status of phosphate homeostasis in children between 1 and 4 years old with XLH'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '4 Years', 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Male or female, aged ≥1 year and \\<5 years\n2. Diagnosis of XLH supported by ONE or more of the following\n\n * Confirmed phosphate regulating gene with homology to endopeptidases located on the X chromosome (PHEX) mutation in the patient or a directly related family member with appropriate X-linked inheritance\n * Serum fibroblast growth factor 23 (FGF23) level \\> 30 pg/mL by Kainos assay\n3. Biochemical findings associated with XLH including:\n\n * Serum phosphorus \\< 3.0 mg/dL (0.97 mmol/L)\n * Serum creatinine within age-adjusted normal range\n4. Radiographic evidence of rickets\n5. Willing to provide access to prior medical records for the collection of historical growth, biochemical, and radiographic data and disease history\n6. Provide written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures\n7. Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments\n\nExclusion Criteria:\n\n1. Unwilling to stop treatment with oral phosphate and/or pharmacologic vitamin D metabolite or analog (e.g. calcitriol, alfacalcidol) during the screening period and for the duration of the study\n2. Presence of nephrocalcinosis on renal ultrasound grade 4 based on the following scale: 0 = Normal, 1 = Faint hyperechogenic rim around the medullary pyramids, 2 = More intense echogenic rim with echoes faintly filling the entire pyramid, 3 = Uniformly intense echoes throughout the pyramid, 4 = Stone formation: solitary focus of echoes at the tip of the pyramid\n3. Planned or recommended orthopedic surgery including staples, 8-plates or osteotomy, within the clinical trial period\n4. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits\n5. Presence or history of any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study\n6. Presence of a concurrent disease or condition that would interfere with study participation or affect safety\n7. History of recurrent infection or predisposition to infection, or of known immunodeficiency\n8. Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments'}, 'identificationModule': {'nctId': 'NCT02750618', 'briefTitle': 'Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Kyowa Kirin Co., Ltd.'}, 'officialTitle': 'An Open-Label, Phase 2 Study to Assess the Safety, Pharmacodynamics, and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)', 'orgStudyIdInfo': {'id': 'UX023-CL205'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Burosumab Q2W', 'description': 'Burosumab subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 160 weeks.', 'interventionNames': ['Biological: Burosumab']}], 'interventions': [{'name': 'Burosumab', 'type': 'BIOLOGICAL', 'otherNames': ['KRN23', 'Crysvita®', 'UX023'], 'description': 'solution for subcutaneous injection', 'armGroupLabels': ['Burosumab Q2W']}]}, 'contactsLocationsModule': {'locations': [{'zip': '06510', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University School of Medicine', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University School of Medicine', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Shriners Hospital for Children', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Ultragenyx Pharmaceutical Inc'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kyowa Kirin, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}