Health TrialsbyHealthPanel

The Duchenne Registry

ConditionsDuchenne Muscular DystrophyBecker Muscular DystrophyDystrophinopathyDystrophinopathy Symptomatic Female CarrierDystrophinopathy Female Carrier
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorThe Duchenne Registry

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial population

The Duchenne Registry is primarily a United States registry, but we welcome participants from other countries and we currently have over 125 countries represented in the registry.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

10,000 Participants
are grouped into 1 trial group
Group A: Duchenne and Becker Muscular Dystrophy

Trial outcomes

Primary outcomes

1

Genetic variant

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.

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

Secondary outcomes

1

Ambulation status

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.

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

Other outcomes

1

Corticosteroid use

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

Time frame
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
2

Cardiovascular status

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.

Time frame
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
3

Respiratory status

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).

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

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

The Duchenne Registry

Lead sponsor

Parent Project Muscular Dystrophy

Collaborator

Source ClinicalTrials.gov
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