Myotonic Dystrophy Family Registry

NCT ID: NCT02398786

Last Updated: 2024-11-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

3500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-02-28

Study Completion Date

2030-02-28

Brief Summary

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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

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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.

Conditions

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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

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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Patient-entered data

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

Intervention Type OTHER

Eligibility Criteria

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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
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Myotonic Dystrophy Foundation

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Tanya Stevenson, EdD, MPH

Role: STUDY_CHAIR

Myotonic Dystrophy Foundation

Locations

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Myotonic Dystrophy Foundation

Oakland, California, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Sofia Olmos, PhD

Role: CONTACT

415-800-7777

Facility Contacts

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Sofia Olmos, PhD

Role: primary

415-800-7777

Related Links

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https://myotonicregistry.patientcrossroads.org/

Myotonic Dystrophy Family Registry (MDFR) Web portal.

Other Identifiers

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MDF001

Identifier Type: -

Identifier Source: org_study_id

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