Summary

for males ages 5 years and up (full criteria)
at Oakland, California and other locations
study started
estimated completion
Jonathan Strober

Description

Summary

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

Official Title

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Details

This study is a randomized, double-blind, placebo-controlled, 72-week study followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period. The total sample size of ~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary analysis population (age 7 to 16 years old, baseline six minute walk distance (6MWD) greater than or equal to (>=) 300 meters, supine to stand >= 5 seconds). The study will be conducted in the United States and other countries around the world.

Keywords

Muscular Dystrophy, DuchenneMuscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseaseNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornDuchenne Muscular DystrophyDystrophinopathyNonsense MutationPremature Stop CodonBecker Muscular DystrophyDMD/BMDPTC124AtalurenMusculoskeletal Diseases

Eligibility

You can join if…

Open to males ages 5 years and up

-

  • 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 CAN'T join 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.

Locations

  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oaklandnot yet accepting patients
    OaklandCalifornia94143United States
  • Stanford University Medical Centeraccepting new patients
    Palo AltoCalifornia94305United States

Lead Scientist

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
PTC Therapeutics
Links
References
ID
NCT03179631
Phase
Phase 3
Study Type
Interventional
Last Updated