Ataluren for Duchenne Muscular Dystrophy

Recruiting at 39 trial locations

Age: Any Age

Sex: Male

Trial Phase: Phase 3

Sponsor: PTC Therapeutics

Must be taking: Ataluren

Must not be taking: IV aminoglycosides, IV vancomycin

Disqualifiers: Hepatitis B, Hepatitis C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to approximately 8 years

270 Participants Needed

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

Vinay Penematsa, MD

Principal Investigator

PTC Therapeutics, Inc.

Are You a Good Fit for This Trial?

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants 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)

Up to approximately 8 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ataluren

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.

How Is the Trial Designed?

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