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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Myotonic Dystrophy Congenital Myotonic Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dystrophia Myotonica Dystrophia Myotonica 1 Dystrophia Myotonica 2 Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonia Atrophica Myotonia Dystrophica Myotonic Disorders Myotonic Dystrophy 1 Myotonic Dystrophy 2 Myotonic Dystrophy, Congenital Myotonic Myopathy, Proximal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases PROMM (Proximal Myotonic Myopathy) Proximal Myotonic Myopathy Steinert Disease Steinert Myotonic Dystrophy Steinert's Disease

Sex eligibility

All sexes

Study type

Observational

Primary location

Myotonic Dystrophy Foundation

Oakland, California, 94612, United States

Location status: Recruiting

Location contact

Sofia Olmos, PhD

CONTACT

[email protected]

415-800-7777

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Patient-entered data

Other

This registry provides patient-entered data for future clinical trial and study use.

Primary outcomes

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.

Study contacts

Contact information is provided by the study sponsor or research team.

Sofia Olmos, PhD

CONTACT

[email protected]

415-800-7777

Sponsors and collaborators

Lead sponsor

Myotonic Dystrophy Foundation

Other

Registry information

Acronym: MDFR

Important dates

Study start: 2013
Primary completion: 2030
Study completion: 2030
First posted: Mar 26, 2015
Registry last updated: Nov 21, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Recruiting