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

This study is currently recruiting participants.
Verified February 2017 by PTC Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT02369731
First Posted: February 24, 2015
Last Update Posted: February 14, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
TREAT-NMD
Cooperative International Neuromuscular Research Group (CINRG)
Information provided by (Responsible Party):
PTC Therapeutics
January 28, 2015
February 24, 2015
February 14, 2017
April 2015
April 2022   (Final data collection date for primary outcome measure)
Incidence of adverse events [ Time Frame: 5 years ]
Same as current
Complete list of historical versions of study NCT02369731 on ClinicalTrials.gov Archive Site
  • Changes in laboratory parameters [ Time Frame: 5 years ]
  • Changes in blood pressure [ Time Frame: 5 years ]
  • Prescriber and patient compliance with prescribing information according to the approved labelling [ Time Frame: 5 years ]
Same as current
Patient health management measures [ Time Frame: 5 years ]
Same as current
 
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual 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.
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.
Observational [Patient Registry]
Observational Model: Cohort
Time Perspective: Prospective
5 Years
Not Provided
Probability Sample
The target study population will include patients for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including patients in named patient early access programs. Patients within each prescriber's practice who are or will be receiving treatment with Translarna, and who meet the eligibility criteria and provide informed consent (either by the patient or through authorisation by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.
Muscular Dystrophy, Duchenne
Drug: Translarna
Other Names:
  • Ataluren
  • PTC124
Not Provided
Not Provided
 
Recruiting
200
April 2022
April 2022   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact: Abdallah Delage adelage@ptcbio.com
Austria,   France,   Germany,   Israel,   Sweden
 
 
NCT02369731
PTC124-GD-025o-DMD
No
Not Provided
Not Provided
PTC Therapeutics
PTC Therapeutics
  • TREAT-NMD
  • Cooperative International Neuromuscular Research Group (CINRG)
Study Director: Robert Spiegel, MD PTC Therapeutics
PTC Therapeutics
February 2017