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

Tracking Information
First Submitted Date	January 28, 2015
First Posted Date	February 24, 2015
Last Update Posted Date	February 14, 2017
Study Start Date	April 2015
Estimated Primary Completion Date	April 2022 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures; (submitted: February 17, 2015)	Incidence of adverse events [ Time Frame: 5 years ]
Original Primary Outcome Measures	Same as current
Change History	Complete list of historical versions of study NCT02369731 on ClinicalTrials.gov Archive Site
Current 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 ]
Original Secondary Outcome Measures	Same as current
Current Other Outcome Measures; (submitted: February 17, 2015)	Patient health management measures [ Time Frame: 5 years ]
Original Other Outcome Measures	Same as current
Descriptive Information
Brief Title	Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
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 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.
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 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.
Condition	Muscular Dystrophy, Duchenne
Intervention	Drug: Translarna; Other Names: Ataluren; PTC124
Study Groups/Cohorts	Not Provided
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: February 17, 2015)	200
Original Estimated Enrollment	Same as current
Estimated Study Completion Date	April 2022
Estimated Primary Completion Date	April 2022 (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 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
Sex/Gender	Sexes Eligible for Study: All
Ages	Child, Adult, Older Adult
Accepts Healthy Volunteers	No
Contacts	Contact: Abdallah Delage adelage@ptcbio.com
Listed Location Countries	Austria, France, Germany, Israel, Sweden
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	TREAT-NMD; Cooperative International Neuromuscular Research Group (CINRG)
Investigators	Study Director: Robert Spiegel, MD PTC Therapeutics
PRS Account	PTC Therapeutics
Verification Date	February 2017