DuchenneConnect Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by DuchenneConnect
Sponsor:
Collaborator:
Parent Project Muscular Dystrophy
Information provided by (Responsible Party):
DuchenneConnect
ClinicalTrials.gov Identifier:
NCT02069756
First received: September 23, 2013
Last updated: February 20, 2014
Last verified: February 2014
  Purpose

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.


Condition
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 40 Years
Official Title: DuchenneConnect: An International, Patient-Report Registry for Individuals With Duchenne/Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Alliance)

Resource links provided by NLM:


Further study details as provided by DuchenneConnect:

Primary Outcome Measures:
  • Genetic mutation [ Time Frame: Registrants are requested to update their medical history every 12 months, and they will be followed throughout their lifetime. ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • Ambulation status [ Time Frame: Registrants are requested to update their medical history every 12 months, and they will be followed throughout their lifetime. ] [ Designated as safety issue: No ]
    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


Other Outcome Measures:
  • Corticosteroid use [ Time Frame: Registrants are requested to update their medical history every 12 months, and they will be followed throughout their lifetime. ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
    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).


Estimated Enrollment: 10000
Study Start Date: October 2007
Estimated Study Completion Date: October 2047
Estimated Primary Completion Date: October 2027 (Final data collection date for primary outcome measure)
Groups/Cohorts
Duchenne and Becker Muscular Dystrophy
Patients with Duchenne or Becker Muscular Dystrophy, as well as carrier females.

Detailed Description:

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 Coordinator at telephone 201.937.1408 or coordinator@duchenneconnect.org. The DuchenneConnect Coordinator is a certified genetic counselor who is available to answer questions regarding the registration process, genetic testing and counseling, and clinical trials and research studies.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

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.

Criteria

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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02069756

Locations
United States, New Jersey
DuchenneConnect / PPMD Recruiting
Hackensack, New Jersey, United States, 07601
Contact: Ann Martin, MS, CGC    201-937-1408    coordinator@duchenneconnect.org   
Sponsors and Collaborators
DuchenneConnect
Parent Project Muscular Dystrophy
  More Information

Additional Information:
Publications:
Responsible Party: DuchenneConnect
ClinicalTrials.gov Identifier: NCT02069756     History of Changes
Other Study ID Numbers: DC-PPMD-2013
Study First Received: September 23, 2013
Last Updated: February 20, 2014
Health Authority: European Union: TREAT-NMD Neuromuscular Alliance

Keywords provided by DuchenneConnect:
Duchenne
Becker
Muscular Dystrophy

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 24, 2014