Status:
TERMINATED
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Lead Sponsor:
PTC Therapeutics
Collaborating Sponsors:
Genzyme, a Sanofi Company
Conditions:
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Eligibility:
MALE
5+ years
Phase:
PHASE2
Brief Summary
Duchenne/Becker muscular dystrophy (DMD/BMD) 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 musc...
Detailed Description
This is a Phase 2b, international, multicenter, open-label extension study for participants who successfully completed blinded study drug in Study 007. This extension study will evaluate the long-term...
Eligibility Criteria
Inclusion
- Completion of blinded study drug treatment in the previous Phase 2b study (PTC124-GD-007-DMD).
- Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if less than \[\<\]18 years of age).
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during PTC124 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
- 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 \[colloidal silica\], magnesium stearate).
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, 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 :
January 31 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 24 2010
Estimated Enrollment :
173 Patients enrolled
Trial Details
Trial ID
NCT00847379
Start Date
January 31 2009
End Date
May 24 2010
Last Update
July 15 2020
Active Locations (37)
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1
University of California - Davis
Sacramento, California, United States, 95817
2
Department of Rehabilitation, The Children's Hospital
Aurora, Colorado, United States, 80045
3
Child Neurology Center of NW Florida
Gulf Breeze, Florida, United States, 32561
4
University of Iowa Children's Hospital, Division of Child Neurology
Iowa City, Iowa, United States, 52242