Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Part of paid clinical trials in La Jolla, California.

Sponsor
Virginia Commonwealth University
Study ID
NCT03981575
Status
Recruiting
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Conditions

  • DM1
  • Myotonic Dystrophy 1

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Study Details

Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward. Funding Source- FDA OOPD

Key Dates

Start date
Jan 1, 2019
Status verified
Jun 2026
Primary completion
Oct 1, 2026
Completion
Dec 1, 2026

Study Design

Enrollment
700 participants (estimated)

Arms

  • Arm: Study Visits
    Patients will receive standard of care as determined by their treating physician. Study visits occur at baseline/0 months, 12 months, and 24 months

Primary Outcome Measure

Change in ambulation over 24 months as measured by the 10 meter walk (m/s). [ Time Frame: 12 and 24 months ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
University of California, San DiegoLa JollaCalifornia92703
Elizabeth Moreno
[email protected]
Gabriella Penner
[email protected]
Chamindra Laverty, MD (PRINCIPAL_INVESTIGATOR)
University of California, Los AngelesLos AngelesCalifornia90095
Dennis Fernando
[email protected]
310-825-3264
Perry Shieh, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of Colorado - DenverDenverColorado80204
Alyssa Avilez
[email protected]
303-724-4644
Brianna Blume
[email protected]
303-724-6386
Matthew Wicklund, MD (PRINCIPAL_INVESTIGATOR)
University of FloridaGainesvilleFlorida32611
Whitney Miller
[email protected]
Donovan Lott
[email protected]
Sankarsubramoney Subramony, MD (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Maegan Tyrrell
[email protected]
319-356-8323
Loraine Brenner
[email protected]
Andrea Swenson, MD (PRINCIPAL_INVESTIGATOR)
Kansas University Medical CenterKansas CityKansas66160
Rebecca Clay
[email protected]
Michaela Walker
[email protected]
Jeffrey Statland, MD (PRINCIPAL_INVESTIGATOR)
University of RochesterRochesterNew York14642
Jim Hilbert
[email protected]
585-273-5590
Jeanne Dekdebrun
[email protected]
585-276-4611
Johanna Hamel, MD (PRINCIPAL_INVESTIGATOR)
Ohio State UniversityColumbusOhio43210
Kaneshia Hives
[email protected]
614-685-5661
Lischele Watkins
[email protected]
Bakri Elsheikh, MD (PRINCIPAL_INVESTIGATOR)
Houston Methodist Neurological InstituteHoustonTexas77030
Kadeesia Brown
[email protected]
346-238-2295
Erika P. Greene (PRINCIPAL_INVESTIGATOR)
Virginia Commonwealth UniversityRichmondVirginia23298
Carino Jennings
[email protected]
Jodie Howell
[email protected]
Nicholas Johnson, MD (PRINCIPAL_INVESTIGATOR)

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University of California, San Diego· La Jolla, CAUniversity of California, Los Angeles· Los Angeles, CAUniversity of Colorado - Denver· Denver, COUniversity of Florida· Gainesville, FLUniversity of Iowa· Iowa City, IAKansas University Medical Center· Kansas City, KS

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