- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02398786
Myotonic Dystrophy Family Registry (MDFR)
Study Overview
Status
Conditions
- Myotonic Dystrophy
- Myotonic Dystrophy 1
- Steinert's Disease
- Congenital Myotonic Dystrophy
- PROMM (Proximal Myotonic Myopathy)
- Steinert Disease
- Dystrophia Myotonica 1
- Myotonic Dystrophy 2
- Dystrophia Myotonica
- Dystrophia Myotonica 2
- Myotonia Dystrophica
- Myotonic Dystrophy, Congenital
- Myotonic Myopathy, Proximal
- Proximal Myotonic Myopathy
- Steinert Myotonic Dystrophy
- Myotonia Atrophica
Intervention / Treatment
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 Type
Enrollment (Estimated)
Contacts and Locations
Study Contact
- Name: Sofia Olmos, PhD
- Phone Number: 415-800-7777
- Email: coordinator@myotonicregistry.org
Study Locations
-
-
California
-
Oakland, California, United States, 94612
- Recruiting
- Myotonic Dystrophy Foundation
-
Contact:
- Sofia Olmos, PhD
- Phone Number: 415-800-7777
- Email: coordinator@myotonicregistry.org
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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 Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
36 months
|
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Tanya Stevenson, EdD, MPH, Myotonic Dystrophy Foundation
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neurologic Manifestations
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Muscular Disorders, Atrophic
- Heredodegenerative Disorders, Nervous System
- Muscular Dystrophies
- Muscular Diseases
- Myotonic Dystrophy
- Myotonia
- Myotonic Disorders
Other Study ID Numbers
- MDF001
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Myotonic Dystrophy
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University of RochesterNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMuscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular DystrophyUnited States
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Avidity Biosciences, Inc.CompletedMyotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert DiseaseUnited States
-
National Institute of Neurological Disorders and...TerminatedMyotonic Dystrophy Type-1 | Myotonic Dystrophy Type-2United States
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McMaster UniversityCompletedMuscular Dystrophies | Myotonic Dystrophy 1Canada
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PepGen IncRecruitingMyotonic Dystrophy 1United States, Canada
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Norwegian School of Sport SciencesUniversity of Oslo; Oslo University Hospital; University of Copenhagen; University... and other collaboratorsCompleted
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University Hospital, BonnForschungszentrum Juelich; The Marigold Foundation; Life and Brain Center BonnCompletedMyotonic Dystrophy 1 | Myotonic Dystrophy 2
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Avidity Biosciences, Inc.Active, not recruitingNervous System Diseases | Genetic Diseases, Inborn | Musculoskeletal Diseases | Muscular Diseases | Neuromuscular Diseases | Neurodegenerative Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Heredodegenerative Disorders, Nervous System | Myotonic Dystrophy | Myotonic Disorders | Myotonic Dystrophy... and other conditionsUnited States
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Université du Québec à ChicoutimiRecruitingMyotonic Dystrophy Type 1 (DM1)Canada
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Virginia Commonwealth UniversityRadboud University Medical Center; University of California, Los Angeles; University... and other collaboratorsRecruitingMyotonic Dystrophy 1United States, Netherlands, United Kingdom, New Zealand, Germany, Italy, Canada, France
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