We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu

DuchenneConnect Registry

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 : September 26, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

September 23, 2013
February 24, 2014
September 26, 2017
October 2007
October 2027   (Final data collection date for primary outcome measure)
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.
Genetic mutation [ Time Frame: Registrants are requested to update their medical history every 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.
Complete list of historical versions of study NCT02069756 on ClinicalTrials.gov Archive Site
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 in home and away from home, ability to sit and stand, use of assistive devices, and age at full time wheelchair use.
Ambulation status [ Time Frame: Registrants are requested to update their medical history every 12 months, and they will be followed throughout their lifetime. ]
Ambulation status is assessed from several questions about mobility in home and away from home, 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).
  • Corticosteroid use [ Time Frame: Registrants are requested to update their medical history every 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, and dosing regimen (daily, weekends, etc).
  • Cardiovascular status [ Time Frame: Registrants are requested to update their medical history every 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 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).
 
DuchenneConnect Registry
DuchenneConnect: An International, Patient-Report Registry for Individuals With Duchenne/Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Alliance)
DuchenneConnect is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of DuchenneConnect 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, DuchenneConnect is a valuable resource for clinicians and researchers in academia and industry, allowing access to a de-identified, aggregate dataset provided by patients and their families—information that is vital to advances in the care and treatment of Duchenne. DuchenneConnect is a member of PCORnet, the National Patient-Centered Clinical Research Network.

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 DuchenneConnect and its material.

Questions may be addressed to the DuchenneConnect Coordinators at telephone 888-520-8675 or coordinator@duchenneconnect.org. The DuchenneConnect 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.

Observational [Patient Registry]
Observational Model: Cohort
Time Perspective: Prospective
40 Years
Not Provided
Probability Sample
DuchenneConnect is primarily a United States registry, but we do welcome participants from other countries and we currently have over 100 countries represented in the registry.
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
Not Provided
Duchenne and Becker Muscular Dystrophy
Patients with Duchenne or Becker Muscular Dystrophy, as well as carrier females.
 
Recruiting
10000
October 2047
October 2027   (Final data collection date for primary outcome measure)

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).
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact: Ann Martin, MS, CGC 888-520-8675 ext option 2 coordinator@duchenneconnect.org
United States
 
 
NCT02069756
DC-PPMD-2013
Yes
Not Provided
Plan to Share IPD: Yes
DuchenneConnect
DuchenneConnect
Parent Project Muscular Dystrophy
Not Provided
DuchenneConnect
September 2017