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

Recruiting in Palo Alto (17 mi)

+36 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: PTC Therapeutics

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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 13 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.

Research Team

Leone Atkinson, MD, PhD

Principal Investigator

PTC Therapeutics

Eligibility Criteria

Inclusion Criteria

Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).

Male sex.

Age ≥5 years.

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Treatment Details

Interventions

Ataluren (Nonsense Mutation Suppressor)
Placebo (Drug)

Participant Groups

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Low-Dose AtalurenExperimental Treatment1 Intervention

Participants will receive ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.

Group II: High-Dose AtalurenExperimental Treatment1 Intervention

Participants will receive ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for 48 weeks.

Group III: PlaceboPlacebo Group1 Intervention

Participants will receive placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.