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

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.

PTC Identifier:
PTC124-GD-025o-DMD
ClinicalTrials.gov Identifier:
NCT02369731
EudraCT Identifier:
N/A
Not Recruiting

Trial Purpose

Age
2+ years
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

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

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
316

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
Apr 2015 - May 2025

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
N/A

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
N/A

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Observational

Observational

Participants in these studies are not assigned to a treatment by researchers, but prescribed a treatment as they normally would be.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
N/A

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
N/A

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
N/A

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Percentage of Participants With Adverse Events

Timeframe: 5 years

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Prescriber and Participant Compliance With Prescribing Information According to the Approved Labelling

Timeframe: 5 years

Contact Information

Location
Status
Location
Universitaetsklinikum Freiburg
Freiburg, Germany, 79106
Status
Recruiting
Location
Universitaetsklinikum Essen (AoR)
Essen, Germany, 45122
Status
Recruiting
Location
SPZ Frankfurt Mitte
Frankfurt am Main, Germany, 60316
Status
Recruiting
Location
Klinikum Bayreuth GmbH
Bayreuth, Germany, 95445
Status
Recruiting
Location
Klinik und Poliklinik f. Kinder- und Jugendmedizin
Hamburg, Germany, 20246
Status
Recruiting
Location
Sozialpädiatrisches Zentrum (SPZ) Charité Berlin
Berlin, Germany, 13353
Status
Recruiting