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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	29 April 2024
Main ID:	NCT02369731
Date of registration:	28/01/2015
Prospective Registration:	Yes
Primary sponsor:	PTC Therapeutics
Public title:	Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Scientific title:	Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
Date of first enrolment:	April 30, 2015
Target sample size:	316
Recruitment status:	Active, not recruiting
URL:	https://clinicaltrials.gov/ct2/show/NCT02369731
Study type:	Observational [Patient Registry]
Study design:
Phase:

Countries of recruitment
Austria	Brazil	Czechia	France	Germany	Greece	Hungary	Israel
Italy	Latvia	Norway	Portugal	Romania	Slovenia	Sweden	United Kingdom

Contacts

Name:	Shelley Johnson
Address:
Telephone:
Email:
Affiliation:	PTC Therapeutics

Key inclusion & exclusion 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

Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Primary Outcome(s)

Percentage of Participants With Adverse Events [Time Frame: 5 years]

Secondary Outcome(s)

Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling [Time Frame: 5 years]

Secondary ID(s)

PTC124-GD-025o-DMD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The John Walton Muscular Dystrophy Research Centre (TREAT-NMD)

Cooperative International Neuromuscular Research Group

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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