Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Completed · Phase 3 · Has a placebo group

Conditions studied: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn

In brief

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Key facts

Study ID

NCT03179631

Run by

PTC Therapeutics

People needed

360

Starts

2017-07-06

Expected to finish

2023-07-25

Last updated by the study team

2026-03-10

Who can join

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

You may qualify if…

• Male sex
• Age ≥5 years
• Phenotypic evidence of Duchenne Muscular Dystrophy
• Nonsense point mutation in the dystrophin gene
• Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
• 6MWD ≥150 meters
• Ability to perform timed function tests within 30 seconds
• Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

You may not qualify if…

• Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
• Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
• Prior or ongoing therapy with ataluren.
• Known hypersensitivity to any of the ingredients or excipients of the study drug
• Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
• History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
• Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
• Uncontrolled clinical symptoms and signs of congestive heart failure
• Elevated serum creatinine or cystatin C at screening.

Where it is running

• Children's Hospital of Los Angeles — Los Angeles, California, United States
• University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland — Oakland, California, United States
• Stanford University Medical Center — Palo Alto, California, United States
• University of California (UC) Davis Medical Center — Sacramento, California, United States
• Loma Linda University Children's Hospital — San Bernardino, California, United States
• Northwest Florida Clinical Research Group, LLC — Gulf Breeze, Florida, United States
• Indiana University Health - Riley Child Neurology — Indianapolis, Indiana, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• University of Michigan - CS Mott Children's Hospital — Ann Arbor, Michigan, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Columbia University College of Physicians & Surgeons — New York, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Shriners Hospital for Children — Portland, Oregon, United States
• University of Pittsburgh School of Medicine — Pittsburgh, Pennsylvania, United States
• Cook Childrens Medical Center — Fort Worth, Texas, United States
• Texas Children's Hospital — Houston, Texas, United States
• University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States
• University Of Utah — Salt Lake City, Utah, United States
• Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• The Childrens Hospital at Westmead — Westmead, New South Wales, Australia
• The Royal Childrens Hospital — Parkville, Victoria, Australia
• Perth Children's Hospital — Nedlands, Western Australia, Australia
• Phoenix Childrens Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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