NCT07402122NOT_YET_RECRUITING

Registry for Duchenne and Becker Muscular Dystrophy

Development and Implementation of a Transparent Registry Based on FAIR Principles for Duchenne and Becker Muscular Dystrophy (DBMD) Under the Auspices of the Society for Neuropediatrics (GNP) - FAIR-DMD

Sponsor: Dr. Andreas Ziegler

No open prediction endpoints

Endpoints are classified and published by ProgramSignal analysts.

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Key Facts

Study type

OBSERVATIONAL

Conditions

Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy Symptomatic Female Carrier

Interventions

—

Enrollment

1,500 participants

Primary completion

Dec 2040

Study completion

Jun 2041

First posted

Feb 2026

Last updated

Feb 2026

Primary Endpoints (CT.gov)

EQ-5D-5L

Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"

EQ VAS

Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"

DMD-QoL

Time frame: * "Baseline" * "every six months" * "through study completion, maximum 15 years"

Eligibility Criteria

Inclusion Criteria: * Genetically confirmed diagnosis of dystrophinopathy * Female carriers only if they show clinical symptoms of a dystrophinopathy * Treatment in one of the participating centers * No age restrictions * Ability to understand the patient information and sign the informed consent * Consent capability by the patient themselves and/or by the parents * Agreement to data exchange between the treating physicians, the telemedicine platform, and the registry * Possession of a tablet or a smartphone Exclusion Criteria: * Missing legally valid consent form from the patient and/or legal guardians * Project content is not understandable to the participant and/or legal guardians * Not in possession of a tablet or smartphone

✦ Analyst Commentary

Expert commentary on why this trial matters and what to watch for.

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Source

Open on ClinicalTrials.gov