Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Stopped early · Phase 2

Conditions studied: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

In brief

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 muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.

Key facts

Study ID

NCT00847379

Run by

PTC Therapeutics

People needed

173

Starts

2009-01-31

Expected to finish

2010-05-24

Last updated by the study team

2020-07-15

Who can join

Age: 5 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of California - Davis — Sacramento, California, United States
• Department of Rehabilitation, The Children's Hospital — Aurora, Colorado, United States
• Child Neurology Center of NW Florida — Gulf Breeze, Florida, United States
• University of Iowa Children's Hospital, Division of Child Neurology — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Children's Hospital of Boston — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University Medical School — St Louis, Missouri, United States
• Columbia University — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Shriners Hospital for Children-Portland — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Southwestern University — Dallas, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• The Royal Children's Hospital — Parkville, Victoria, Australia
• The Children's Hospital at Westmead — Westmead, Australia
• UZ Leuven — Leuven, Belgium
• Alberta Children's Hospital — London, Ontario, Canada
• Children's Hospital of Western Ontario — London, Ontario, Canada
• British Colombia Children's Hopsital — Vancouver, Canada
• Hopital d'Enfants CHU Timone — Marseille, France
• Laboratoire d'Exploration Fonctionnelles — Nantes, France
• Groupe Hospitalier Pitie-Salpetriere, Institut de Myologie — Paris, France
• University of Essen - Clinic for Children — Essen, Germany

Full record on ClinicalTrials.gov

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