Myotonic Dystrophy Family Registry

Recruiting

• Conditions: Myotonic Dystrophy 1; Steinert Myotonic Dystrophy; Myotonia Atrophica; Congenital Myotonic Dystrophy; Myotonia Dystrophica; Dystrophia Myotonica 1; Dystrophia Myotonica 2; Myotonic Dystrophy, Congenital; Steinert's Disease; Dystrophia Myotonica

• Registration Number: NCT02398786
• Lead Sponsor: Myotonic Dystrophy Foundation

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 Timeline

• Study Start: 2013-02
• Study First Submitted: 2015-03-06
• Study First Submitted QC: 2015-03-24
• Study First Posted: 2015-03-26
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-19
• Last Update Posted: 2024-11-21
• Primary Completion: 2030-02
• Study Completion: 2030-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 3500

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Patient reported outcomes	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.

Trial Locations

Locations (1)

Myotonic Dystrophy Foundation; 🇺🇸 Oakland, California, United States