Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada
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|ClinicalTrials.gov Identifier: NCT01557400|
Recruitment Status : Completed
First Posted : March 19, 2012
Last Update Posted : April 12, 2019
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy Becker Muscular Dystrophy Dystrophinopathy||Drug: Ataluren||Phase 3|
All participating sites must have had at least 1 patient that received ataluren treatment in a prior PTC-sponsored clinical study in DBMD. It is planned that up to ~96 patients will be enrolled.
Subjects will receive ataluren 3 times per day (TID) at respective morning, midday, and evening doses of 10 mg/kg, 10 mg/kg, and 20 mg/kg, for approximately 336 weeks. Study assessments will be performed at clinic visits during screening, on the first day of ataluren dosing, and then every48 weeks during the ataluren treatment period, except for weight, which will be measured every 24 weeks at a primary care physician (PCP). No measures of efficacy will be captured.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||95 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy|
|Actual Study Start Date :||May 31, 2012|
|Actual Primary Completion Date :||January 31, 2018|
|Actual Study Completion Date :||January 31, 2018|
Oral powder for suspension taken 3 times per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20mg/kg in the evening).
Other Name: PTC124
- Long term Safety and Tolerability of Ataluren. [ Time Frame: 336 weeks ]The primary objective of this study is to assess the long-term safety and tolerability of 10, 10, 20 mg/kg ataluren in patients with nmDBMD who had prior exposure to ataluren in a PTC-sponsored clinical trial.Safety profile characterized by type, frequency, severity, timing, and relationship to Ataluren of any adverse events or laboratory abnormalities.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01557400
|Study Director:||Edward O'Mara, MD||PTC Therapeutics|