= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
Patient Registry Translarna (Ataluren)
Long-term observational study of Translarna safety and effectiveness in usual care
Hub Summary
This phase 4 clinical study is designed to assess the safety of Translarna, also known at Ataluren. This study will follow patients who are receiving Translarna as part of their usual care for 5 years. At the patients usual visits, data will be collected to determine the safety and effectiveness of Translarna.
Study Number: NCT02369731
Description by PTC Therapeutics
This is a multicentre, observational study of patients receiving Translarna based on inclusion of their data in a registry. This study is intended to enrol approximately 200 patients across ~60 care centres 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 enrolment. 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.
Primary Outcome Measures
- Incidence of adverse events.
Other Outcome Measures
- Changes in laboratory parameters.
- Changes in blood pressure.
- Prescriber and patient compliance with prescribing information according to the approved labelling.
Can I take part?
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, randomised clinical trial, or ataluren in any other ataluren clinical trial or cohort early access program that prevents participation in this study
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Fully recruited
PTC Therapeutics