Ignite Creation Date:
2025-12-24 @ 6:44 PM
Ignite Modification Date:
2026-04-21 @ 11:46 AM
Study NCT ID:
NCT02819557
Status:
COMPLETED
Last Update Posted:
2020-08-28
First Post:
2016-06-16
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C515878', 'term': 'ataluren'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ptcbio.com', 'phone': '1-866-562-4620', 'title': 'Patient Advocacy', 'organization': 'PTC Therapeutics, Inc.'}, 'certainAgreement': {'otherDetails': 'The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the Sponsor for review. The Sponsor may consult with the PI to require changes to the communication or extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to Week 56', 'description': 'Adverse events were collected from all participants who received at least 1 dose of ataluren (Safety Population).', 'eventGroups': [{'id': 'EG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.', 'otherNumAtRisk': 14, 'deathsNumAtRisk': 14, 'otherNumAffected': 14, 'seriousNumAtRisk': 14, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Abdominal Pain Upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Stomatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Gait Disturbance', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Bronchitis Viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Croup Infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Ear Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Gastroenteritis Norovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Gingival Abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Pharyngitis Streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Upper Respiratory Tract Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Viral Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Arthropod Bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Tibia Fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hepatic Enzyme Increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Decreased Appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hyperlipidaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Metabolic Acidosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Pain In Extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Abnormal Behaviour', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Enuresis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Chromaturia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Rhinorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Tonsillar Hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Hypertrichosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 3}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Rash Generalised', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 19.0'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'At least 1 TEAE', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'Mild TEAE', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Moderate TEAE', 'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Severe TEAE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Related to Study Drug', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Leading to Participant Study Discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 56', 'description': 'A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity not related to dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}, {'type': 'PRIMARY', 'title': 'Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 56', 'description': 'Clinical laboratory results that were considered clinically meaningful were to be determined by the Investigator and Sponsor. Biochemistry parameters included sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters included white blood cell count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters included pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}, {'type': 'PRIMARY', 'title': 'Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 56', 'description': 'ECG results that were considered clinically meaningful were to be determined by the Investigator. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}, {'type': 'PRIMARY', 'title': 'Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Week 56', 'description': "Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following:\n\n* Hepatic: The participant's medical history, hepatitis screening results, all clinical blood values (particularly serum bilirubin, gamma-glutamyl transferase \\[GGT\\], aspartate aminotransferase \\[AST\\], and alanine aminotransferase \\[ALT\\] values), and all concomitant medications were reviewed.\n* Renal: The participant's medical history, all clinical blood and urine renal values, serum electrolytes, medications, and potential pre- or post-renal conditions were reviewed.", 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Day 1', 'categories': [{'measurements': [{'value': '15.95', 'spread': '9.51', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '12.54', 'spread': '4.43', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method.', 'unitOfMeasure': 'microgram/milliliter (µg/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Day 1', 'categories': [{'measurements': [{'value': '3.84', 'spread': '1.82', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '2.72', 'spread': '1.98', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method.', 'unitOfMeasure': 'hours', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).'}, {'type': 'SECONDARY', 'title': 'Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Day 1', 'categories': [{'measurements': [{'value': '101.64', 'spread': '58.52', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '82.13', 'spread': '27.43', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve.', 'unitOfMeasure': 'hour*μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).'}, {'type': 'SECONDARY', 'title': 'Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Day 1', 'categories': [{'measurements': [{'value': '9.12', 'spread': '8.88', 'groupId': 'OG000'}]}]}, {'title': 'Day 28', 'categories': [{'measurements': [{'value': '5.43', 'spread': '3.15', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method.', 'unitOfMeasure': 'microgram per milliliter (µg/ml)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population).'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Rise to Standing, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.2', 'spread': '7.21', 'groupId': 'OG000'}]}]}, {'title': 'Rise to Standing, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.1', 'spread': '6.47', 'groupId': 'OG000'}]}]}, {'title': 'Rise to Standing, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-3.1', 'spread': '6.50', 'groupId': 'OG000'}]}]}, {'title': 'Walk/Run 10 m, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.6', 'spread': '2.37', 'groupId': 'OG000'}]}]}, {'title': 'Walk/Run 10 m, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.8', 'spread': '1.54', 'groupId': 'OG000'}]}]}, {'title': 'Walk/Run 10 m, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.1', 'spread': '1.35', 'groupId': 'OG000'}]}]}, {'title': 'Ascend 4 Stairs, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.1', 'spread': '6.95', 'groupId': 'OG000'}]}]}, {'title': 'Ascend 4 Stairs, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.8', 'spread': '4.85', 'groupId': 'OG000'}]}]}, {'title': 'Ascend 4 Stairs, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.6', 'spread': '5.00', 'groupId': 'OG000'}]}]}, {'title': 'Descend 4 Stairs, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '7.5', 'spread': '3.95', 'groupId': 'OG000'}]}]}, {'title': 'Descend 4 Stairs, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.6', 'spread': '1.93', 'groupId': 'OG000'}]}]}, {'title': 'Descend 4 Stairs, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.2', 'spread': '2.58', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 28 and Week 52', 'description': 'TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable data for the applicable timed function test.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Physical Function as Measured by the NSAA', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': '16-Point Scale, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16.0', 'spread': '4.66', 'groupId': 'OG000'}]}]}, {'title': '16-Point Scale, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.5', 'spread': '3.43', 'groupId': 'OG000'}]}]}, {'title': '16-Point Scale, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.5', 'spread': '4.43', 'groupId': 'OG000'}]}]}, {'title': '8-Point Scale, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '10.5', 'spread': '2.56', 'groupId': 'OG000'}]}]}, {'title': '8-Point Scale, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.5', 'spread': '1.39', 'groupId': 'OG000'}]}]}, {'title': '8-Point Scale, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.3', 'spread': '2.13', 'groupId': 'OG000'}]}]}, {'title': '3-Point Scale, Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.4', 'spread': '0.63', 'groupId': 'OG000'}]}]}, {'title': '3-Point Scale, Change from Baseline at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.5', 'spread': '0.78', 'groupId': 'OG000'}]}]}, {'title': '3-Point Scale, Change from Baseline at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.3', 'spread': '0.73', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 28 and Week 52', 'description': "NSAA consists of 17 activities, including items assessing abilities necessary to remain functionally ambulant (that is, ability to rise from floor, to get from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (that is, assessing head raise and standing on heels); and a number of activities such as hopping, jumping, and running. Since the boys were \\<5 years old, revised 16 point, 8-point, and 3-point scales were used over the 17 point scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance), or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-point scale=32, 8-point scale=16, and 3-point scale=6. If an activity couldn't be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline calculated by subtracting Baseline value from value at Week 28 and Week 52.", 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable NSAA data.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '99.43', 'spread': '5.278', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.83', 'spread': '1.595', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.84', 'spread': '1.466', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.11', 'spread': '1.386', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.82', 'spread': '1.506', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.95', 'spread': '2.096', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.04', 'spread': '2.075', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56', 'unitOfMeasure': 'centimeters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population) and had evaluable height data.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '16.99', 'spread': '3.257', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.04', 'spread': '0.502', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.73', 'spread': '0.974', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.16', 'spread': '0.940', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.39', 'spread': '0.882', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '1.90', 'spread': '1.259', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.13', 'spread': '1.340', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56', 'unitOfMeasure': 'kg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population) and had evaluable weight data.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '17.094', 'spread': '2.2196', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.346', 'spread': '0.6804', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 16', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.026', 'spread': '0.3822', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 28', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.030', 'spread': '0.5567', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 40', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.008', 'spread': '0.6399', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.186', 'spread': '0.9237', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 56', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.015', 'spread': '1.2161', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56', 'description': 'Body mass index is an estimate of body fat based on body weight divided by height squared.', 'unitOfMeasure': 'kilograms per square meter (kg/m^2)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}, {'type': 'SECONDARY', 'title': 'Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'classes': [{'title': 'Question 1, Strongly disagree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 1, Disagree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 1, Neither Agree nor Disagree', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Question 1, Agree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 1, Strongly agree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 1, No Response', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, Strongly disagree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, Disagree', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, Neither Agree nor Disagree', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, Agree', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, Strongly Agree', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Question 2, No response', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, Strongly Disagree', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, Disagree', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, Neither agree or disagree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, Agree', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, Strongly Agree', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Question 3, No response', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 28', 'description': 'To assess palatability characteristics, participants/parents or guardians were asked to provide a response of "Strongly disagree", "Disagree", "Neither agree or disagree", "Agree", or "Strongly Agree" to the following 3 questions:\n\nQuestion 1. "Is the medicine palatable?"\n\nQuestion 2. "On the basis of reaction / facial expression of your child, do you think that the medication is pleasant?"\n\nQuestion 3."You sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up?"', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population).'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'Safety Population', 'comment': 'All participants who received at least 1 dose of ataluren.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'Pharmacokinetics (PK) Population', 'comment': 'All participants who received at least 1 dose of ataluren and had at least 1 PK concentration value.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'Evaluable Population', 'comment': 'Received ≥1 dose, baseline and a postbaseline TFT, NSAA, or answer to ataluren palatability question', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Participants aged ≥2 to \\<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene were recruited for this study.', 'preAssignmentDetails': 'Participants in the Evaluable Population included all participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for Timed Function Test (TFT), North Star Ambulatory Assessment (NSAA), or response to the palatability of ataluren questions.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose was provided based upon the weight of each participant, which was assessed every 12 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '3.4', 'spread': '0.76', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', '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 (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '3', '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': '11', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'All participants who received at least 1 dose of ataluren (Safety Population).'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2016-01-28', 'size': 737492, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-08-12T16:18', 'hasProtocol': True}, {'date': '2018-03-19', 'size': 872055, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-08-12T16:18', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-06-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-08', 'dispFirstSubmitDate': '2019-01-24', 'completionDateStruct': {'date': '2018-02-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-08-12', 'studyFirstSubmitDate': '2016-06-16', 'dispFirstSubmitQcDate': '2019-01-24', 'resultsFirstSubmitDate': '2020-08-12', 'studyFirstSubmitQcDate': '2016-06-27', 'dispFirstPostDateStruct': {'date': '2019-01-28', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-08-28', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-08-12', 'studyFirstPostDateStruct': {'date': '2016-06-30', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-08-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-02-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs)', 'timeFrame': 'Baseline up to Week 56', 'description': 'A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An SAE was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity not related to dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section.'}, {'measure': 'Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter', 'timeFrame': 'Baseline up to Week 56', 'description': 'Clinical laboratory results that were considered clinically meaningful were to be determined by the Investigator and Sponsor. Biochemistry parameters included sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters included white blood cell count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters included pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.'}, {'measure': 'Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results', 'timeFrame': 'Baseline up to Week 56', 'description': 'ECG results that were considered clinically meaningful were to be determined by the Investigator. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.'}, {'measure': 'Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity', 'timeFrame': 'Baseline up to Week 56', 'description': "Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following:\n\n* Hepatic: The participant's medical history, hepatitis screening results, all clinical blood values (particularly serum bilirubin, gamma-glutamyl transferase \\[GGT\\], aspartate aminotransferase \\[AST\\], and alanine aminotransferase \\[ALT\\] values), and all concomitant medications were reviewed.\n* Renal: The participant's medical history, all clinical blood and urine renal values, serum electrolytes, medications, and potential pre- or post-renal conditions were reviewed."}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr)', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method.'}, {'measure': 'Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr)', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method.'}, {'measure': 'Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr)', 'timeFrame': '0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve.'}, {'measure': 'Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr)', 'timeFrame': '0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28', 'description': 'Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method.'}, {'measure': 'Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs', 'timeFrame': 'Baseline, Week 28 and Week 52', 'description': 'TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used.'}, {'measure': 'Change From Baseline in Physical Function as Measured by the NSAA', 'timeFrame': 'Baseline, Week 28 and Week 52', 'description': "NSAA consists of 17 activities, including items assessing abilities necessary to remain functionally ambulant (that is, ability to rise from floor, to get from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (that is, assessing head raise and standing on heels); and a number of activities such as hopping, jumping, and running. Since the boys were \\<5 years old, revised 16 point, 8-point, and 3-point scales were used over the 17 point scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance), or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-point scale=32, 8-point scale=16, and 3-point scale=6. If an activity couldn't be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline calculated by subtracting Baseline value from value at Week 28 and Week 52."}, {'measure': 'Change From Baseline in Height of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56'}, {'measure': 'Change From Baseline in Weight of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56'}, {'measure': 'Change From Baseline in Body Mass Index of Participants at Weeks 4, 16, 28, 40, 52, and 56', 'timeFrame': 'Baseline, Weeks 4, 16, 28, 40, 52, and 56', 'description': 'Body mass index is an estimate of body fat based on body weight divided by height squared.'}, {'measure': 'Ataluren Palatability Characteristics as Determined by a Parent/Caregiver Questionnaire', 'timeFrame': 'Baseline up to Week 28', 'description': 'To assess palatability characteristics, participants/parents or guardians were asked to provide a response of "Strongly disagree", "Disagree", "Neither agree or disagree", "Agree", or "Strongly Agree" to the following 3 questions:\n\nQuestion 1. "Is the medicine palatable?"\n\nQuestion 2. "On the basis of reaction / facial expression of your child, do you think that the medication is pleasant?"\n\nQuestion 3."You sometimes have problems in giving the medication to your child because he/she refuses to take it or throws it up?"'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \\<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.', 'detailedDescription': 'In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males ≥2 to \\<5 years of age\n* Body weight ≥12 kg\n* Diagnosis of DMD\n* Nonsense mutation in at least 1 allele of the dystrophin gene\n\nExclusion Criteria:\n\n* Participation in any other drug or device clinical investigation\n* Ongoing use of prohibited concomitant medications'}, 'identificationModule': {'nctId': 'NCT02819557', 'briefTitle': 'Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy', 'orgStudyIdInfo': {'id': 'PTC124-GD-030-DMD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ataluren', 'description': 'Participants will be administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose will be provided based upon the weight of each participant, which will be assessed every 12 weeks.', 'interventionNames': ['Drug: Ataluren']}], 'interventions': [{'name': 'Ataluren', 'type': 'DRUG', 'otherNames': ['PTC124', 'Translarna'], 'description': 'White to off-white powder for oral suspension.', 'armGroupLabels': ['Ataluren']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32561', 'city': 'Gulf Breeze', 'state': 'Florida', 'country': 'United States', 'facility': 'Child Neuro NWF', 'geoPoint': {'lat': 30.35714, 'lon': -87.16386}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '75390-8843', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': "Children's Medical Center Dallas", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}], 'overallOfficials': [{'name': 'Francesco Bibbiani, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'PTC Therapeutics'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'PTC Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}