The Duchenne Registry

September 8, 2023 updated by: The Duchenne Registry

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

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.

Study Overview

Status

Recruiting

Conditions

Detailed Description

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 coordinator@duchenneregistry.org. The Duchenne Registry Coordinators are certified genetic counselors who are available to answer questions regarding the registration process, genetic testing and counseling, and clinical trials and research studies.

Study Type

Observational

Enrollment (Estimated)

10000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study 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.

Description

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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Duchenne and Becker Muscular Dystrophy
Patients with Duchenne or Becker Muscular Dystrophy, as well as carrier females.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
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.
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
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 and raw FVC in liters).
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Duchenne Registry

Collaborators

Parent Project Muscular Dystrophy

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2007

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

October 1, 2047

Study Registration Dates

First Submitted

September 23, 2013

First Submitted That Met QC Criteria

February 20, 2014

First Posted (Estimated)

February 24, 2014

Study Record Updates

Last Update Posted (Actual)

September 13, 2023

Last Update Submitted That Met QC Criteria

September 8, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DC-PPMD-2013

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

De-identified data is shared with researchers upon request and after careful review of application.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Duchenne Muscular Dystrophy

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Portugal

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe