Observational Study of Translarna in Usual Care
Research type
Research Study
Full title
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
IRAS ID
217989
Contact name
Mary Imelda Hughes
Contact email
Sponsor organisation
PTC Therapeutics International Limited
Duration of Study in the UK
6 years, 0 months, 1 days
Research summary
This study is a multi-centre, observational study being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of EMA, to gather data on safety, effectiveness, and prescription patterns of Ataluren in routine clinical practice.
Ataluren is being marketed as Translarna™ for treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.
Although clinical trials of ataluren conducted to date have suggested that ataluren has an acceptable benefit-risk profile, there is a need for real-world clinical data to extend the long-term safety profile of the product as well as to help characterize the effectiveness of ataluren, and patterns of use, in routine clinical practice.
The study intends to enroll up to 200 patients over a 2-year period (35 in the UK). Patients will be receiving Translarna from commercial supply (or named patient early access program) as part of their usual care, and will be able to provide written informed consent (either by the patient or through authorisation by a legal guardian). Enrolled patients will be followed for at least 5 years from their date of enrollment, or until patient withdrawal of consent, or death, whichever occurs first. Data will be collected from the patient's routine clinical practice visits. Patients who discontinue treatment with Translarna will continue to be followed for the duration of the study unless they withdraw consent to participate in the study.
Specific analyses on safety concerns being monitored for ataluren will be performed where data available.
REC name
West Midlands - Solihull Research Ethics Committee
REC reference
17/WM/0138
Date of REC Opinion
18 May 2017
REC opinion
Further Information Favourable Opinion