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Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02090959
First Posted: March 19, 2014
Last Update Posted: June 1, 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. Read our disclaimer for details.
Information provided by (Responsible Party):
PTC Therapeutics
March 17, 2014
March 19, 2014
June 1, 2017
March 2014
December 2017   (Final data collection date for primary outcome measure)
Long term safety of ataluren in boys with nonsense mutation dystrophinopathy, as determined by adverse events and laboratory abnormalities [ Time Frame: Baseline and 96 weeks ]
Same as current
Complete list of historical versions of study NCT02090959 on ClinicalTrials.gov Archive Site
  • Physical Function [ Time Frame: Baseline and 96 weeks ]
    North Star Ambulatory Assessment,Timed Function Testing, Upper Limb Function, 6 Minute Walk Test
  • Patient and/or parent-reported activities of daily living and disease symptoms [ Time Frame: Baseline and 96 weeks ]
  • Quality of Life [ Time Frame: Baseline and 96 weeks ]
  • Pulmonary function [ Time Frame: Baseline and 96 weeks ]
  • Ataluren blood levels [ Time Frame: Baseline and 96 weeks ]
Same as current
Not Provided
Not Provided
 
Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.
This Phase 3, open label safety and efficacy study will be performed at participating sites worldwide. The study will enroll ~ 220 boys with nonsense mutation dystrophinopathy who participated in a previous Phase 3 study of ataluren (Protocol # PTC124-GD-020-DMD). Patients will receive 10, 10, 20 mg/kg of ataluren TID at morning, midday, and evening for approximately 96 weeks. Study assessments will be performed at clinic visits every 12 weeks.
Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Muscular Dystrophy, Duchenne
  • Muscular Dystrophies
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
Drug: Ataluren
Other Name: PTC124
Experimental: Ataluren
10, 10, 20 mg/kg for 96 weeks.
Intervention: Drug: Ataluren
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
219
December 2017
December 2017   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Completion of study treatment in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-020-DMD).
  • Evidence of signed and dated informed consent/assent document(s) indicating that the patient (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
  • Willingness to abstain from sexual intercourse or employ an approved method of contraception during the period of study drug administration and 6-week follow-up period.
  • Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

  • Known hypersensitivity to any of the ingredients or excipients of the study drug
  • Ongoing participation in any clinical trial (except for studies specifically approved by PTC Therapeutics).
  • Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
Sexes Eligible for Study: Male
7 Years to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Belgium,   Brazil,   Canada,   Chile,   Czechia,   France,   Germany,   Israel,   Italy,   Korea, Republic of,   Poland,   Spain,   Sweden,   Switzerland,   Turkey,   United Kingdom,   United States
Czech Republic
 
NCT02090959
PTC124-GD-020e-DMD
Yes
Not Provided
Plan to Share IPD: No
PTC Therapeutics
PTC Therapeutics
Not Provided
Study Director: Robert Spiegel, M.D. PTC Therapeutics
PTC Therapeutics
May 2017

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