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Trial record 40 of 657 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Diseases"

Myotonic Dystrophy Family Registry (MDFR)

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ClinicalTrials.gov Identifier: NCT02398786
Recruitment Status : Recruiting
First Posted : March 26, 2015
Last Update Posted : September 3, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
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 Other: Patient-entered data

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 : Observational [Patient Registry]
Estimated Enrollment : 3000 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Myotonic Dystrophy Family Registry
Study Start Date : February 2013
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020



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


Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants will volunteer to participate in this study and enter their own information. The study will be advertised through neuromuscular disease clinics and the Myotonic Dystrophy Foundation and Myotonic Dystrophy Family Registry websites.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02398786


Contacts
Contact: Molly White 415-800-7745 coordinator@myotonicregistry.org

Locations
United States, California
Myotonic Dystrophy Foundation Recruiting
San Francisco, California, United States, 94129
Contact: Molly White    415-800-7745    coordinator@myotonicregistry.org   
Sponsors and Collaborators
Myotonic Dystrophy Foundation
Investigators
Study Chair: Molly White Myotonic Dystrophy Foundation

Responsible Party: Myotonic Dystrophy Foundation
ClinicalTrials.gov Identifier: NCT02398786     History of Changes
Other Study ID Numbers: MDF001
First Posted: March 26, 2015    Key Record Dates
Last Update Posted: September 3, 2018
Last Verified: August 2018

Additional relevant MeSH terms:
Muscular Diseases
Muscular Disorders, Atrophic
Myotonic Dystrophy
Myotonia
Myotonic Disorders
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Muscular Dystrophies
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms