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Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

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ClinicalTrials.gov Identifier: NCT00847379
Recruitment Status : Terminated
First Posted : February 19, 2009
Last Update Posted : June 1, 2017
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
PTC Therapeutics

Tracking Information
First Submitted Date  ICMJE February 16, 2009
First Posted Date  ICMJE February 19, 2009
Last Update Posted Date June 1, 2017
Actual Study Start Date  ICMJE January 2009
Actual Primary Completion Date May 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 18, 2009)
Long-term safety of PTC124 in boys with nonsense-mutation mediated DMD/BMD, as determined by adverse events and laboratory abnormalities [ Time Frame: 2 years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00847379 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 30, 2017)
  • Ambulation [ Time Frame: 2 years ]
  • Proximal muscle function [ Time Frame: 2 years ]
  • Heart rate [ Time Frame: 2 years ]
  • Cognitive ability [ Time Frame: 2 years ]
  • Health Related Quality of life (HRQL) [ Time Frame: 2 years ]
  • Activities of daily living [ Time Frame: 2 years ]
  • Muscle fragility [ Time Frame: 2 years ]
  • Compliance with ataluren (PTC124) treatment [ Time Frame: 2 years ]
  • Ataluren (PTC124) pharmacokinetics [ Time Frame: 2 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 18, 2009)
  • Ambulation [ Time Frame: 2 years ]
  • Proximal muscle function [ Time Frame: 2 years ]
  • Heart rate [ Time Frame: 2 years ]
  • Cognitive ability [ Time Frame: 2 years ]
  • Quality of life [ Time Frame: 2 years ]
  • Activities of daily living [ Time Frame: 2 years ]
  • Muscle fragility [ Time Frame: 2 years ]
  • Compliance with ataluren (PTC124) treatment [ Time Frame: 2 years ]
  • Ataluren (PTC124) pharmacokinetics [ Time Frame: 2 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Official Title  ICMJE A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy
Brief Summary Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.
Detailed Description This Phase 2b, open-label, safety and efficacy study is anticipated to be performed at 37 sites in 11 countries. The study will enroll up to 174 boys with nonsense mutation DMD/BMD who participated in a previous Phase 2b study of ataluren (PTC124) (PTC124-GD-007-DMD, NCT00592553). Subjects will receive study drug 3 times per day (at breakfast, lunch, and dinner) for approximately 96 weeks (approximately 2 years). Study assessments will be performed at clinic visits during screening, every 6 weeks for 2 visits and then every 12 weeks until the end of the study. Additional safety laboratory testing, which may be performed at the investigational site or at an accredited local laboratory or clinic, is required 3 times during the course of the study.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
Intervention  ICMJE Drug: Ataluren (PTC124)
Oral powder for suspension taken 3 times per day (20 mg/kg with breakfast, 20 mg/kg with lunch, and 40 mg/kg with dinner) for up to 96 weeks.
Study Arms  ICMJE Experimental: Ataluren (PTC124)
Ataluren (PTC124)
Intervention: Drug: Ataluren (PTC124)
Publications *

*   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 Terminated
Actual Enrollment  ICMJE
 (submitted: August 28, 2013)
173
Original Estimated Enrollment  ICMJE
 (submitted: February 18, 2009)
174
Actual Study Completion Date  ICMJE May 2010
Actual Primary Completion Date May 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Completion of blinded study drug treatment in the previous Phase 2b study (PTC124-GD-007-DMD).
  • Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).
  • In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during PTC124 administration and the 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 (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], magnesium stearate).
  • Ongoing participation in any other therapeutic clinical trial.
  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 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,   Belgium,   Canada,   France,   Germany,   Israel,   Italy,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00847379
Other Study ID Numbers  ICMJE PTC124-GD-007e-DMD
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party PTC Therapeutics
Study Sponsor  ICMJE PTC Therapeutics
Collaborators  ICMJE Genzyme, a Sanofi Company
Investigators  ICMJE
Study Director: Leone Atkinson, M.D., Ph.D. PTC Therapeutics
PRS Account PTC Therapeutics
Verification Date May 2017

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