Ignite Creation Date:
2025-12-25 @ 12:32 AM
Ignite Modification Date:
2026-02-20 @ 6:38 PM
Study NCT ID:
NCT02531867
Status:
COMPLETED
Last Update Posted:
2017-05-08
First Post:
2015-08-11
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007014', 'term': 'Hypophosphatasia'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D012279', 'term': 'Rickets'}, {'id': 'D010018', 'term': 'Osteomalacia'}], 'ancestors': [{'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': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D002128', 'term': 'Calcium 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': 'C570710', 'term': 'asfotase alfa'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alexion.com', 'phone': '(781) 357-9900', 'title': 'Medical Director', 'organization': 'Alexion'}, 'certainAgreement': {'otherDetails': 'The Investigators are subject to certain disclosure and publication limitations. In cases of publication of the study results at conferences or on academic journals, the post-approval clinical study coordinating investigator and the study sponsor should discuss about details of publication, publication methods and presenters before decision-making', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': "Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.", 'description': 'TEAEs were collected at every visit and follow-up.', 'eventGroups': [{'id': 'EG000', 'title': 'Overall Study', 'description': "Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.", 'otherNumAtRisk': 13, 'otherNumAffected': 9, 'seriousNumAtRisk': 13, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Enterocolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Conjunctivitis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 5, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Viral rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Feeding tube complicatino', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Intercostal neuralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}, {'term': 'Alopecia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.1'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Overall Study', 'description': "Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week."}], 'classes': [{'title': 'Number of Patients with AEs', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}]}]}, {'title': 'Number of Patients with ISRs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Number of Patients with IARs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': "Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.", 'description': "Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All patients who met all of the inclusion and none of the exclusion criteria were defined as the enrolled population, which was also the analysis population.The safety set comprised all patients who received at least 1 dose of the investigational drug.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Overall Study', 'description': "Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week."}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'There were 11 study sites, all in Japan; 13 patients were enrolled into this study. The first patient signed informed consent in June 2015; the study continued through November 2015 at multiple centers in Japan.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Overall Study', 'description': "Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week."}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '1.39', 'groupId': 'BG000', 'lowerLimit': '0.13', 'upperLimit': '35.04'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Japan', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All patients who met all of the inclusion and none of the exclusion criteria were considered the Baseline Analysis Population.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-03', 'completionDateStruct': {'date': '2015-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-03-28', 'studyFirstSubmitDate': '2015-08-11', 'resultsFirstSubmitDate': '2016-11-28', 'studyFirstSubmitQcDate': '2015-08-24', 'lastUpdatePostDateStruct': {'date': '2017-05-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2017-03-28', 'studyFirstPostDateStruct': {'date': '2015-08-25', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-05-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2015-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)', 'timeFrame': "Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.", 'description': "Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study."}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['HPP', 'Bone Disease', 'Soft Bones', 'Low Alkaline Phosphatase', 'Genetic Metabolic Disorder', 'Alkaline Phosphatase', 'Rickets', 'Osteomalacia'], 'conditions': ['Hypophosphatasia']}, 'descriptionModule': {'briefSummary': 'This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.', 'detailedDescription': 'The primary objective of this study was to collect data on the safety of repeated subcutaneous (SC) injections of asfotase alfa. There were no secondary objectives. Exploratory objectives related to the efficacy of asfotase alfa are not being reported.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.\n2. Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa\n\nExclusion Criteria:\n\n1. Patient has a documented form of rickets caused by a condition other than HPP, including, but not limited to, rickets caused by 25(OH) vitamin D deficiency\n2. Patient has serum calcium and/or phosphorus levels below the normal range\n3. Patient is pregnant or lactating\n4. Patient received treatment with bisphosphonates within 2 years prior to the Screening visit\n5. Patient has a documented sensitivity to any of the components of asfotase alfa\n6. Patient is currently enrolled in any other program or clinical study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation)\n7. Patient has clinically significant other disease in the opinion of the Investigator, defined as any other non HPP-related condition for which the patient is considered medically unstable.'}, 'identificationModule': {'nctId': 'NCT02531867', 'briefTitle': 'Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in Japan', 'orgStudyIdInfo': {'id': 'AA-HPP-409'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Asfotase Alfa', 'description': "Patients will receive asfotase alfa by subcutaneous injection. Asfotase alfa will be administered at either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week depending on investigator's discretion.", 'interventionNames': ['Biological: Asfotase Alfa']}], 'interventions': [{'name': 'Asfotase Alfa', 'type': 'BIOLOGICAL', 'otherNames': ['Strensiq'], 'armGroupLabels': ['Asfotase Alfa']}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Kenji Fujita, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Medical Monitor'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}