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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. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03179631
Recruitment Status : Active, not recruiting
First Posted : June 7, 2017
Last Update Posted : February 16, 2023
Information provided by (Responsible Party):
PTC Therapeutics

Tracking Information
First Submitted Date  ICMJE June 1, 2017
First Posted Date  ICMJE June 7, 2017
Last Update Posted Date February 16, 2023
Actual Study Start Date  ICMJE July 6, 2017
Actual Primary Completion Date March 5, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks [ Time Frame: 72 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 5, 2017)
6- Minute Walk Test [ Time Frame: 72 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
  • Change from Baseline to Week 72 in 6MWD [ Time Frame: Baseline, Week 72 ]
  • Change from Baseline to Week 72 in Time to Run/Walk 10 Meters [ Time Frame: Baseline, Week 72 ]
  • Change from Baseline to Week 72 in Time to Climb 4 Stairs [ Time Frame: Baseline, Week 72 ]
  • Change from Baseline to Week 72 in Time to Descend 4 Stairs [ Time Frame: Baseline, Week 72 ]
  • Change from Baseline to Week 72 in North Start Ambulatory Assessment (NSAA) Total Score [ Time Frame: Baseline, Week 72 ]
  • Time to Loss of Ambulation Over 72 Weeks [ Time Frame: 72 weeks ]
  • Time to Loss of Stair-Climbing Over 72 Weeks [ Time Frame: 72 Weeks ]
  • Time to Loss of Stair-Descending Over 72 Weeks [ Time Frame: 72 weeks ]
  • Risk of Loss of NSAA Items Over 72 weeks [ Time Frame: 72 weels ]
  • Number of Treatment-Emergent Adverse Events Considered Related to Study Drug [ Time Frame: 72 weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 5, 2017)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Official Title  ICMJE 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
Brief Summary This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Detailed Description 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Disease
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
Intervention  ICMJE
  • Drug: Ataluren
    10, 20 mg/kg
    Other Name: PTC124
  • Drug: PLACEBO
    10, 20 mg/kg
    Other Name: Matching Placebo
Study Arms  ICMJE
  • Experimental: Ataluren
    10, 20 milligrams per kilogram (mg/kg)
    Intervention: Drug: Ataluren
  • Placebo Comparator: Placebo
    10, 20 mg/kg
    Intervention: Drug: PLACEBO
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: February 4, 2022)
Original Estimated Enrollment  ICMJE
 (submitted: June 5, 2017)
Estimated Study Completion Date  ICMJE July 20, 2023
Actual Primary Completion Date March 5, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Brazil,   Bulgaria,   Canada,   China,   Hong Kong,   India,   Japan,   Korea, Republic of,   Malaysia,   Mexico,   Poland,   Puerto Rico,   Russian Federation,   Taiwan,   Thailand,   Turkey,   United States
Removed Location Countries Argentina,   Chile,   Jordan,   Sri Lanka
Administrative Information
NCT Number  ICMJE NCT03179631
Other Study ID Numbers  ICMJE PTC124-GD-041-DMD
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party PTC Therapeutics
Original Responsible Party Same as current
Current Study Sponsor  ICMJE PTC Therapeutics
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Vinay Penematsa, MD PTC Therapeutics, Inc.
PRS Account PTC Therapeutics
Verification Date February 2023

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