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NCT02398786 Clinical Trial

Myotonic Dystrophy Family Registry

Myotonic Dystrophy 1 Clinical Trial

— MDFR  

Official title:
Myotonic Dystrophy Family Registry

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT02398786
Other study ID # MDF001
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date February 2013
Est. completion date February 2025

Study information
Verified date September 2023
Source Myotonic Dystrophy Foundation
Contact Sofia Olmos, PhD
Phone 415-800-7777
Email coordinator@myotonicregistry.org
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 3000
Est. completion date February 2025
Est. primary completion date February 2025
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility 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

Related Conditions & MeSH terms

Intervention

Other:
• Patient-entered data
This registry provides patient-entered data for future clinical trial and study use.

Locations
Country Name City State
United States Myotonic Dystrophy Foundation Oakland California

Sponsors (1)
Lead Sponsor Collaborator
Myotonic Dystrophy Foundation

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Patient reported outcomes 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. 36 months
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