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

First listed
Jun 11, 2019
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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