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Myotonic Dystrophy Family Registry (MDFR)

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: NCT02398786
Recruitment Status : Recruiting
First Posted : March 26, 2015
Last Update Posted : March 30, 2016
Sponsor:
Information provided by (Responsible Party):

March 6, 2015
March 26, 2015
March 30, 2016
February 2013
February 2020   (Final data collection date for primary outcome measure)
Patient reported outcomes [ Time Frame: 36 months ]
Number of patients reporting specific symptoms and symptom severity, as well as impacts to quality of life and overall burden of disease in order to inform clinical trial development, understanding of disease for academic, industry and federal agency stakeholders and overall policy decisions. Results will be analyzed in comparison to other registry data and surveys to characterize this disease population cohort and to further define the population.
Same as current
Complete list of historical versions of study NCT02398786 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Myotonic Dystrophy Family Registry
Myotonic Dystrophy Family Registry
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.

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.

Observational [Patient Registry]
Observational Model: Cohort
Time Perspective: Prospective
5 Years
Not Provided
Non-Probability Sample
Participants will volunteer to participate in this study and enter their own information. The study will be advertised through neuromuscular disease clinics and the Myotonic Dystrophy Foundation and Myotonic Dystrophy Family Registry websites.
  • Myotonic Dystrophy
  • Congenital Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy 2
  • Dystrophia Myotonica
  • Dystrophia Myotonica 1
  • Dystrophia Myotonica 2
  • Myotonia Dystrophica
  • Myotonic Dystrophy, Congenital
  • Myotonic Myopathy, Proximal
  • PROMM (Proximal Myotonic Myopathy)
  • Proximal Myotonic Myopathy
  • Steinert Disease
  • Steinert Myotonic Dystrophy
  • Steinert's Disease
  • Myotonia Atrophica
Other: Patient-entered data
This registry provides patient-entered data for future clinical trial and study use.
Not Provided
Not Provided
 
Recruiting
3000
February 2020
February 2020   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact: Molly White 415-800-7745 coordinator@myotonicregistry.org
United States
 
 
NCT02398786
MDF001
Yes
Not Provided
Not Provided
Myotonic Dystrophy Foundation
Myotonic Dystrophy Foundation
Not Provided
Study Chair: Molly White Myotonic Dystrophy Foundation
Myotonic Dystrophy Foundation
March 2016