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NCT Number: NCT02069756

The Duchenne Registry

The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, The Duchenne Registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to de-identified datasets provided by patients and their families-information that is vital to advances in the care and treatment of Duchenne. The Duchenne Registry is a member of the TREAT-NMD Neuromuscular Network.

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Key information

Conditions

Duchenne Muscular Dystrophy Becker Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dystrophinopathy Dystrophinopathy Female Carrier Dystrophinopathy Symptomatic Female Carrier Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

About this study

The Duchenne Registry (previously DuchenneConnect) was created in 2007 by Parent Project Muscular Dystrophy (PPMD), with assistance from the NIH, the CDC, and Emory Genetics. In early 2011, PPMD alone began financing the registry's operation and maintenance, and is the sole guardian of The Duchenne Registry and its material.

Questions may be addressed to the Duchenne Registry Coordinators at telephone 888-520-8675 or [email protected]. The Duchenne Registry Coordinators are certified genetic counselors who are available to answer questions regarding the registration process, genetic testing, and clinical trials and research studies.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of Duchenne or Becker muscular dystrophy; Manifesting female carriers and asymptomatic female carriers also included in registry.

Exclusion criteria

  • Diagnosis of any other type of muscular dystrophy (including limb-girdle muscular dystrophy).

Treatment and study plan

Primary outcomes

  1. Genetic variant

    Time frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

    Genetic variant data is collected by patient report and verified by curation/review of genetic test report when provided. Genetic test report is requested for each registrant and is required for participation in global DMD (TREAT-NMD) registry.

Secondary outcomes

  1. Ambulation status

    Time frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

    Ambulation status is assessed from several questions about mobility, ability to sit and stand, use of assistive devices, and age at full time wheelchair use.

Other outcomes

  1. Corticosteroid use

    Time frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

    Data collected includes whether or not corticosteroids are used, and if so, name of corticosteroid, age started/stopped, dose and dosing regimen.

  2. Cardiovascular status

    Time frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

    Multiple questions regarding cardiovascular symptoms, age at diagnosis of cardiomyopathy, use of cardiac medications, date of most recent echocardiogram and/or cardiac MRI, and the LVEF and/or LVSF value.

  3. Respiratory status

    Time frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

    Assessed from questions about use of breathing devices, age at which breathing devices were started, date of most recent pulmonary function test (spirometry), and the FVC value (% predicted).

Study contacts

Contact information is provided by the study sponsor or research team.

Ann Martin, MS, CGC

CONTACT

[email protected]

888-520-8675

Lauren Bogue, MS, CGC

CONTACT

[email protected]

888-520-8675

Sponsors and collaborators

Lead sponsor

The Duchenne Registry

Other

Collaborators

  • Parent Project Muscular Dystrophy

Registry information

Official study title

The Duchenne Registry: An International, Patient-Report Registry for Individuals With Duchenne and Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Network)

Important dates

Study start
2007
Primary completion
2027
Study completion
2047
First posted
Feb 24, 2014
Registry last updated
May 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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