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Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

This study is currently recruiting participants.
Verified November 2017 by PTC Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT03179631
First Posted: June 7, 2017
Last Update Posted: November 17, 2017
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.
Information provided by (Responsible Party):
PTC Therapeutics
  Purpose
This study is a long-term study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy.

Condition Intervention Phase
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 Drug: PLACEBO Phase 3

Study Type: Interventional
Study Design: 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
Official Title: A Phase 3 Randomized Double Blind Placebo Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) and Open Label Extension

Resource links provided by NLM:


Further study details as provided by PTC Therapeutics:

Primary Outcome Measures:
  • 6- Minute Walk Test [ Time Frame: 72 weeks ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 250
Actual Study Start Date: July 6, 2017
Estimated Study Completion Date: December 2021
Estimated Primary Completion Date: March 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ataluren
10, 10, 20 mg/kg
Drug: Ataluren
10, 10, 20 mg/kg
Other Name: PTC124
Placebo Comparator: Placebo
10, 10, 20 mg/kg
Drug: PLACEBO
10, 10, 20 mg/kg
Other Name: Matching Placebo

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. 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.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03179631


Contacts
Contact: Mary Frances Harmon 908-912-9256 mharmon@ptcbio.com
Contact: Amy Perniciaro 908-912-9264 aperniciaro@ptcbio.com

Locations
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Kiley Higgs    913-945-9922    ksims2@kumc.edu   
Principal Investigator: Jeffrey Statland, MD         
United States, Texas
University of Texas Health Science Center at San Antonio Recruiting
San Antonio, Texas, United States, 78229-3900
Contact: Floyd Jones    210-567-8222    JonesFA@uthscsa.edu   
Principal Investigator: Ratna Bhavaraju-Sanka, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Ana Christensen    206-987-5433    muscleresearch@seattlechildrens.org   
Principal Investigator: Susan Apkon, MD         
Sponsors and Collaborators
PTC Therapeutics
Investigators
Study Director: Joe McIntosh, MD PTC Therapeutics, Inc.
  More Information

Additional Information:
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT03179631     History of Changes
Other Study ID Numbers: PTC124-GD-041-DMD
First Submitted: June 1, 2017
First Posted: June 7, 2017
Last Update Posted: November 17, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by PTC Therapeutics:
Duchenne Muscular Dystrophy
Dystrophinopathy
Nonsense Mutation
Premature Stop Codon
Becker Muscular Dystrophy
DMD/BMD
PTC124
Ataluren

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Nervous System Diseases
Genetic Diseases, Inborn
Neuromuscular Diseases
Musculoskeletal Diseases
Muscular Diseases
Genetic Diseases, X-Linked
Muscular Disorders, Atrophic