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

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.
 
ClinicalTrials.gov Identifier: NCT02369731
Recruitment Status : Recruiting
First Posted : February 24, 2015
Last Update Posted : June 9, 2020
Sponsor:
Collaborators:
The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)
Cooperative International Neuromuscular Research Group
Information provided by (Responsible Party):
PTC Therapeutics

Tracking Information
First Submitted Date January 28, 2015
First Posted Date February 24, 2015
Last Update Posted Date June 9, 2020
Actual Study Start Date April 30, 2015
Estimated Primary Completion Date May 30, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 6, 2020)
Percentage of Participants With Adverse Events [ Time Frame: 5 years ]
Original Primary Outcome Measures
 (submitted: February 17, 2015)
Incidence of adverse events [ Time Frame: 5 years ]
Change History
Current Secondary Outcome Measures
 (submitted: January 6, 2020)
Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling [ Time Frame: 5 years ]
Original Secondary Outcome Measures
 (submitted: February 17, 2015)
  • 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 ]
Current Other Pre-specified Outcome Measures
 (submitted: January 6, 2020)
Participant Health Management Measures [ Time Frame: 5 years ]
Original Other Pre-specified Outcome Measures
 (submitted: February 17, 2015)
Patient health management measures [ Time Frame: 5 years ]
 
Descriptive Information
Brief Title Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
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 participants receiving Translarna based on inclusion of their data in a registry. This study is intended to enroll 270 participants across ~50 care centers in Europe and other regions over a period of ~ 2 years. The study population will include participants who are receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program) and who provide consent. Participants 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 Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population The target study population will include participants for whom the decision to prescribe Translarna (outside of a clinical trial) has already been made, including participants in named participant early access programs. Participants 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 participant or through authorisation by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.
Condition Muscular Dystrophy, Duchenne
Intervention Not Provided
Study Groups/Cohorts Translarna
Participants with nmDMD receiving Translarna will be followed for at least 5 years from their date of enrollment, or until participant withdrawal of consent or death, whichever occurs first.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: January 6, 2020)
270
Original Estimated Enrollment
 (submitted: February 17, 2015)
200
Estimated Study Completion Date May 30, 2025
Estimated Primary Completion Date May 30, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Receiving or will be receiving usual care treatment with commercial supply of Translarna (or receiving care within a named participant early access program)
  • Willing to provide written informed consent to allow the study data collection procedures (either by the participant or through authorisation by a legal guardian)

Exclusion Criteria:

  • Participants 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
Sex/Gender
Sexes Eligible for Study: All
Ages 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Patient Advocacy Corporate Relations 1-866-562-4620 medinfo@ptcbio.com
Listed Location Countries Austria,   Czechia,   France,   Germany,   Greece,   Hungary,   Israel,   Italy,   Latvia,   Norway,   Portugal,   Romania,   Slovenia,   Sweden,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number NCT02369731
Other Study ID Numbers PTC124-GD-025o-DMD
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party PTC Therapeutics
Study Sponsor PTC Therapeutics
Collaborators
  • The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)
  • Cooperative International Neuromuscular Research Group
Investigators
Study Director: Claudio Santos PTC Therapeutics
PRS Account PTC Therapeutics
Verification Date May 2020