Myotonic Dystrophy Family Registry

ConditionsMyotonic 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

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorMyotonic Dystrophy Foundation

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosed with congenital, juvenile-onset or adult onset DM1 or DM2 (confirmed by clinical exam or genetic test)

Disqualifiers

Not diagnosed with DM, unaffected family members

Trial 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.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

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

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

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.

Time frame

36 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Myotonic Dystrophy Foundation

Lead sponsor

Source ClinicalTrials.gov

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