Ataluren for Duchenne Muscular Dystrophy

Recruiting at 39 trial locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: PTC Therapeutics

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial tests the safety and tolerance of ataluren in patients with a specific type of Duchenne Muscular Dystrophy who have been part of previous studies. Ataluren helps the body make important proteins that are usually missing due to genetic errors. The study includes these patients and their siblings who have completed earlier trial phases. Ataluren is the first approved drug for Duchenne muscular dystrophy (DMD) patients with premature stop codon mutations and has been conditionally approved in Europe.

Research Team

Vinay Penematsa, MD

Principal Investigator

PTC Therapeutics, Inc.

Eligibility Criteria

This trial is for individuals with a type of muscular dystrophy called nmDBMD who have previously taken Ataluren in a PTC sponsored clinical trial or treatment plan. It's also open to siblings of these participants if they've finished the placebo part of another study. Participants must understand and agree to the study requirements, be able to attend all visits, and men must use birth control during the study.

Inclusion Criteria

I agree to use contraception during and up to 50 days after the study if I am a fertile man not having had a vasectomy.

I have previously taken ataluren in a study or am a sibling of someone who has, after they completed the trial's initial phase.

I have previously taken ataluren in a study or have a sibling with nmDBMD who did.

Exclusion Criteria

I tested positive for Hepatitis B or C and have not used ataluren before or had a break from it for a year.

I am not allergic to any components of ataluren medication.

Exposure to another investigational drug within 1 month prior to start of study treatment.

Treatment Details

Interventions

• Ataluren (Nonsense Mutation Readthrough Agent)

Trial Overview The trial is testing the safety and tolerability of Ataluren at doses of 10 mg/kg (twice daily) and 20 mg/kg (once daily). The medication will be given until certain conditions are met such as consent withdrawal, worsening condition after starting treatment, eligibility for another Ataluren trial, or commercial availability.

Participant Groups

Group I: Ataluren Experimental Treatment 1 Intervention

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).

Who Is Running the Clinical Trial?

PTC Therapeutics

Lead Sponsor

Trials

75

Recruited

6,300+

Dr. Matthew B. Klein

PTC Therapeutics

Chief Executive Officer since 2023

BA from the University of Pennsylvania, MD from Yale University School of Medicine, MS in Epidemiology from the University of Washington School of Public Health

Dr. Stuart W. Peltz

PTC Therapeutics

Chief Medical Officer since 2023

MD from Harvard Medical School