Myotonic Dystrophy Family Registry

Brief Title

Myotonic Dystrophy Family Registry

Official Title

Myotonic Dystrophy Family Registry

Brief Summary

      The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that
      collects information on myotonic dystrophy (DM) to aid researchers in developing new,
      effective treatments and help identify participants for research studies and clinical trials.
    

Detailed Description

      The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that
      collects information on myotonic dystrophy (DM) such as disease symptoms and demographic
      information to aid researchers in developing new, effective treatments and help identify
      participants for research studies and clinical trials.

      The Registry supports trials and studies, making it easier for researchers to explore data
      and identify possible trial and study participants. It is the first DM registry that gives
      community members the opportunity to explore anonymous Registry data, to see what the DM
      community looks like and what others with DM experience. It also provides information on the
      community of people living with DM, giving researchers and other medical professionals the
      opportunity to improve how they treat those affected with DM and learn more about how and why
      certain treatments work and don't work.
    


Study Type

Observational [Patient Registry]


Primary Outcome

Patient reported outcomes


Condition

Myotonic Dystrophy

Intervention

Patient-entered data


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

3000

Start Date

February 2013

Completion Date

February 2021

Primary Completion Date

February 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosed with congenital, juvenile-onset or adult onset DM1 or DM2 (confirmed by
             clinical exam or genetic test)

        Exclusion Criteria:

          -  Not diagnosed with DM, unaffected family members
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Tanya Stevenson, EdD, MPH, 415-800-7640, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02398786

Organization ID

MDF001


Responsible Party

Sponsor

Study Sponsor

Myotonic Dystrophy Foundation


Study Sponsor

Tanya Stevenson, EdD, MPH, Study Chair, Myotonic Dystrophy Foundation


Verification Date

June 2020