Status:
TERMINATED
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
Lead Sponsor:
PTC Therapeutics
Collaborating Sponsors:
Genzyme, a Sanofi Company
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
Phase:
PHASE2
Brief Summary
Duchenne muscular dystrophy (DMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structur...
Detailed Description
This Phase 2a, multicenter, open-label safety and efficacy study will be performed at 3 sites in the United States. The study will enroll up to 38 participants with nonsense mutation Duchenne muscular...
Eligibility Criteria
Inclusion
- Completion of ataluren treatment in the previous Phase 2a study (Protocol PTC124-GD-004-DMD).
- Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if \<18 years of age).
- Confirmed screening laboratory values within the central laboratory ranges.
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 6-week follow up period.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion
- Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of study treatment.
- Treatment with warfarin within 1 month prior to start of study treatment.
- Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM \[refined polydextrose\], polyethylene glycol 3350, Lutrol® micro F127 \[poloxamer 407\], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P \[colloidal silica\], and magnesium stearate).
- Exposure to another investigational drug within 2 months prior to start of study treatment.
- History of major surgical procedure within 1 month prior to start of study treatment.
- Ongoing immunosuppressive therapy (other than corticosteroids).
- Ongoing participation in any other clinical trial (except for sub-studies specifically approved by PTC Therapeutics).
- Clinically significant symptoms and signs of congestive heart failure (CHF) (American College of Cardiology/American Heart Association Stage C or Stage D).
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
Key Trial Info
Start Date :
August 13 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 17 2010
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT00759876
Start Date
August 13 2008
End Date
May 17 2010
Last Update
October 29 2020
Active Locations (3)
Enter a location and click search to find clinical trials sorted by distance.
1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
3
University of Utah
Salt Lake City, Utah, United States, 84112