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 : September 3, 2018
|Condition or disease||Intervention/treatment|
|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|
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]|
|Estimated Enrollment :||3000 participants|
|Target Follow-Up Duration:||5 Years|
|Official Title:||Myotonic Dystrophy Family Registry|
|Study Start Date :||February 2013|
|Estimated Primary Completion Date :||February 2020|
|Estimated Study Completion Date :||February 2020|
- Other: Patient-entered data
This registry provides patient-entered data for future clinical trial and study use.
- 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.
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): NCT02398786
|Contact: Molly Whitefirstname.lastname@example.org|
|United States, California|
|Myotonic Dystrophy Foundation||Recruiting|
|San Francisco, California, United States, 94129|
|Contact: Molly White 415-800-7745 email@example.com|
|Study Chair:||Molly White||Myotonic Dystrophy Foundation|