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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

Study Description
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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.

Condition or disease
Muscular Dystrophy, Duchenne

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 Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 270 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
Actual Study Start Date : April 30, 2015
Estimated Primary Completion Date : May 30, 2025
Estimated Study Completion Date : May 30, 2025

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Groups and Cohorts
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Group/Cohort
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.


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants With Adverse Events [ Time Frame: 5 years ]

Secondary Outcome Measures :
  1. Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling [ Time Frame: 5 years ]

Other Outcome Measures:
  1. Participant Health Management Measures [ Time Frame: 5 years ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02369731


Contacts
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Contact: Patient Advocacy Corporate Relations 1-866-562-4620 medinfo@ptcbio.com

Locations
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Sponsors and Collaborators
PTC Therapeutics
The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)
Cooperative International Neuromuscular Research Group
Investigators
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Study Director: Claudio Santos PTC Therapeutics
More Information
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Additional Information:

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02369731    
Other Study ID Numbers: PTC124-GD-025o-DMD
First Posted: February 24, 2015    Key Record Dates
Last Update Posted: June 9, 2020
Last Verified: May 2020
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked