Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03179631
Recruitment Status : Recruiting
First Posted : June 7, 2017
Last Update Posted : August 22, 2018
Information provided by (Responsible Party):
PTC Therapeutics

June 1, 2017
June 7, 2017
August 22, 2018
July 6, 2017
March 2020   (Final data collection date for primary outcome measure)
6- Minute Walk Test [ Time Frame: 72 weeks ]
Same as current
Complete list of historical versions of study NCT03179631 on Archive Site
  • Timed Function Tests [ Time Frame: 72 weeks ]
  • North Star Ambulatory Assessment [ Time Frame: 72 weeks ]
  • Performance of Upper Limb (in patients >=7 years old at baseline) [ Time Frame: 72 weeks ]
  • Myometry (in patients <7 years old at baseline) [ Time Frame: 72 weeks ]
  • Magnetic Resonance Imaging (MRI) (at pre-qualified sites) [ Time Frame: 72 weeks ]
  • EQ-5D [ Time Frame: 72 weels ]
  • Ataluren safety profile characterized by adverse events and abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms [ Time Frame: 72 weeks ]
Same as current
Not Provided
Not Provided
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
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
This study is a long-term study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy.
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. Patients will be randomized in a 1:1 ratio to ataluren or placebo. Patients will receive blinded study drug TID at morning, midday, and evening for 72 weeks, after which all patients 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 6MWD >=300 meters, supine to stand >=5 seconds). The study will be conducted in the United States and other countries around the world.
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
This study describes the randomized, double-blind, placebo-controlled, 72-week study and its 72-week open-label extension
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
A randomized, double-blind, placebo-controlled,72-week study and its 72-week open-label extension
Primary Purpose: Treatment
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Disease
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
  • Drug: Ataluren
    10, 10, 20 mg/kg
    Other Name: PTC124
  • Drug: PLACEBO
    10, 10, 20 mg/kg
    Other Name: Matching Placebo
  • Experimental: Ataluren
    10, 10, 20 mg/kg
    Intervention: Drug: Ataluren
  • Placebo Comparator: Placebo
    10, 10, 20 mg/kg
    Intervention: Drug: PLACEBO
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
December 2021
March 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria -

  • Male sex.
  • Age ≥5 years.
  • Phenotypic evidence of DMD
  • 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.

Exclusion Criteria:

  • Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
  • Prior or 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.
Sexes Eligible for Study: Male
5 Years and older   (Child, Adult, Older Adult)
Contact: Mary Frances Harmon 908-912-9256
Contact: PTC Medical Information
Argentina,   Australia,   Brazil,   Bulgaria,   Canada,   Chile,   China,   Hong Kong,   India,   Japan,   Korea, Republic of,   Malaysia,   Mexico,   Poland,   Puerto Rico,   Russian Federation,   Taiwan,   Turkey,   United States
Not Provided
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
PTC Therapeutics
PTC Therapeutics
Not Provided
Study Director: Francesco Bibbiani, MD PTC Therapeutics, Inc.
PTC Therapeutics
August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP