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

Disease Information

Descriptive Information
Brief Title † Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
Official Title † Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
Brief Summary 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.
Detailed Description This is a multicenter, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 200 patients across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include patients who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program) and who provide consent. Patients 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.
Study Phase N/A
Study Type † Observational [Patient Registry]
Study Design †
Primary Outcome Measure † Incidence of adverse events
Secondary Outcome Measure † Changes in laboratory parameters
Condition † Muscular Dystrophy, Duchenne
Intervention † DrugTranslarna
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 200
Start Date † April 2015
Completion Date April 2022
Primary Completion Date April 2022
Eligibility Criteria † Inclusion Criteria: - Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named patient early access program) - Willing to provide written informed consent to allow the study data collection procedures (either by the patient or through authorisation by a legal guardian) Exclusion Criteria: - Patients 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
Gender All
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts †† Abdallah Delage, adelage@ptcbio.com
Location Countries † Austria
Administrative Information
NCT ID † NCT02369731
Organization ID PTC124-GD-025o-DMD
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † PTC Therapeutics
Collaborators †† TREAT-NMD
Investigators † Study Director: Robert Spiegel, MD, PTC Therapeutics
Information Provided By
Verification Date February 2017
First Received Date † January 28, 2015
Last Updated Date February 13, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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