Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01247207|
Recruitment Status : Enrolling by invitation
First Posted : November 24, 2010
Last Update Posted : December 5, 2019
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan.
The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Ataluren||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||160 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy|
|Actual Study Start Date :||November 30, 2010|
|Estimated Primary Completion Date :||December 31, 2020|
|Estimated Study Completion Date :||December 31, 2020|
Participants will receive 3 doses of ataluren oral suspension per day (10 mg/kg in the morning, 10 mg/kg at mid-day, and 20 mg/kg in the evening).
Ataluren will be administered as per the dose and schedule specified in the arm.
Other Name: PTC124
- Number of Participants With Adverse Events [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]
- Number of Participants With Laboratory Parameters Abnormalities [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.
- Number of Participants With Abnormal Physical Findings [ Time Frame: Baseline up to end of study (up to approximately 8 years) ]Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature.
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): NCT01247207
|Study Director:||Francesco Bibbiani, MD||PTC Therapeutics, Inc.|