Registry for Duchenne and Becker Muscular Dystrophy

Starting soon

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

In brief

Duchenne muscular dystrophy (DMD) is an X-linked, recessive, progressive, and degenerative neuromuscular disorder that affects approximately one in 5,000 newborn boys. The established "standard of care" has improved prognosis; however, a causal therapy is not yet available. In 2024 and 2025, the first disease-modifying therapies were approved. These include Vamorolone (Agamree®) as a corticosteroid replacement with a more favorable side-effect profile for children aged four and older, and Givinostat (Duvyzat®) as a combination therapy with corticosteroids for ambulatory boys aged six and older. In this context, the FAIR-DMD Registry was initiated. The registry is based on the so-called FAIR principles. The acronym FAIR stands for the data principles Findable, Accessible, Interoperable and Reusable. The international FAIR principles are guidelines for the description, storage, and publication of scientific or administrative data. The FAIR-DMD registry is a disease-specific, academically managed registry for patients with Duchenne and Becker muscular dystrophy (DMD/BMD). Its goal is to systematically collect clinical data, scientifically monitor new disease-modifying therapies in routine care, and create an evidence-based foundation for the further development of diagnostics, therapy, and care structures. Furthermore, the registry collects data on patients' health related quality of live using an app for data entry. The FAIR-DMD Registry is being established under the auspices of the Society for Neuropediatrics (GNP) and operated in close coordination with Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD). The GNP is a non-profit professional society that covers the entire spectrum of neuropediatric topics in clinical and cross-sector care. In the planned pilot phase, the GNP will act as trustee for financing. This model creates the opportunity to structurally address central challenges in health services research and establish a high-quality, internationally compatible registry structure. In the long term, the FAIR-DMD Registry aims to significantly improve care for DMD and BMD patients in German-speaking countries, evaluate the effectiveness of new therapies in clinical practice, and establish binding frameworks for quality-assured care.

Key facts

Study ID: NCT07402122
Run by: Dr. Andreas Ziegler
People needed: 1500
Starts: 2026-04-01
Expected to finish: 2041-06-01
Last updated by the study team: 2026-02-11

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

Universitätsklinikum Essen Klinik für Kinderheilkunde I — Essen, Germany
Universitätsklinik Heidelberg, Zentrum für Kinder- und Jugendmedizin — Heidelberg, Germany

Full record on ClinicalTrials.gov

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