Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

 Duchenne Muscular Dystrophy / Posted 4 weeks ago

Detailed Description
This is a multicenter, observational study of participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 270 participants across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants will be followed for at least 5 years from their date of enrollment. Safety and efficacy data will be collected in conjunction with routine visits conducted as per usual care. Although there are no protocol-mandated procedures, it is expected that physicians and other caregivers will follow published treatment guidelines and standards of care.
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety, effectiveness, and prescription patterns in routine clinical practice.
  • Inclusion Criteria :
    • Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program)
    • Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian)
  • Exclusion Criteria :
    • Participants who are receiving ataluren or placebo in a blinded, randomized clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study
  • Study start date : April 30, 2015
  • Study end date : May 30, 2025
  • Wales-Based Study Contact : Please speak to your clinician
  • Principal Investigator : Adnan Manzur
Contact details

UCL Great Ormond Street Institute of Child Health, 30 Guidlford St, LondonLondon,England,WC1N 1EH  Show Phone Number medinfo@ptcbio.com www.ucl.ac.uk

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