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

• ClinicalTrials.gov Identifier: NCT02369731
• Recruitment Status: Recruiting
• First Posted: February 24, 2015
• Last Update Posted: June 27, 2022
• Sponsor: PTC Therapeutics
• 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 27, 2022
• 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 ]
• 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 *: Mercuri E, Muntoni F, Osorio AN, Tulinius M, Buccella F, Morgenroth LP, Gordish-Dressman H, Jiang J, Trifillis P, Zhu J, Kristensen A, Santos CL, Henricson EK, McDonald CM, Desguerre I; STRIDE; CINRG Duchenne Natural History Investigators. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res. 2020 Apr;9(5):341-360. doi: 10.2217/cer-2019-0171. Epub 2020 Jan 30.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: February 24, 2021): 360
• 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, Brazil, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Latvia, Norway, Portugal, Romania, Slovenia, Sweden, United Kingdom

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
• Current Responsible Party: PTC Therapeutics
• Original Responsible Party: Same as current
• Current Study Sponsor: PTC Therapeutics
• Original Study Sponsor: Same as current

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: June 2022