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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Trial Status
Recruiting
Trial Sponsor
PTC Therapeutics
Age
Child, adolescent and adult
Mutation Specific
Mutation specific therapies, Nonsense mutation
Muscle Biopsy
No Muscle Biopsy Required
MRI
No
Phase
4
Length Of Participation
5 years
Recruitment Target
Approximately 200
Ambulatory
Ambulant and non-ambulant
Therapeutic Category
Ribosome targeting