The Duchenne Registry (Previously DuchenneConnect)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02069756|
Recruitment Status : Recruiting
First Posted : February 24, 2014
Last Update Posted : July 10, 2018
|Condition or disease|
|Duchenne Muscular Dystrophy Becker Muscular Dystrophy|
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 firstname.lastname@example.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 [Patient Registry]|
|Estimated Enrollment :||10000 participants|
|Target Follow-Up Duration:||40 Years|
|Official Title:||The Duchenne Registry: An International, Patient-Report Registry for Individuals With Duchenne and Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Network)|
|Study Start Date :||October 2007|
|Estimated Primary Completion Date :||October 2027|
|Estimated Study Completion Date :||October 2047|
Duchenne and Becker Muscular Dystrophy
Patients with Duchenne or Becker Muscular Dystrophy, as well as carrier females.
- Genetic mutation [ Time Frame: Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime. ]Genetic mutation 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.
- 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.
- 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.
- 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.
- 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).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02069756
|Contact: Ann Martin, MS, CGC||888-520-8675 ext option email@example.com|
|United States, New Jersey|
|The Duchenne Registry / PPMD||Recruiting|
|Hackensack, New Jersey, United States, 07601|
|Contact: Ann Martin, MS, CGC 888-520-8675 ext option 2 firstname.lastname@example.org|