Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
NCT ID: NCT03458832
Last Updated: 2025-04-23
Study Results
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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RECRUITING
320 participants
OBSERVATIONAL
2018-03-05
2027-12-31
Brief Summary
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Detailed Description
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To this end, the researchers propose to develop two novel clinical outcome assessments (COA), a composite functional outcome measure (FSHD-COM) and skeletal muscle biomarker, electrical impedance myography (EIM). In addition, there is broad consensus a better understanding of the relationship of genetic and demographic features to disease progression will be necessary for enumerating eligibility criteria.
The specific aims are to: 1. Determine the multi-site validity of the COAs, 2. Compare the responsiveness of new COAs to other FSHD outcomes and determine the minimal clinically meaningful changes, and 3. establish FSHD cohort characteristics useful for determining clinical trial eligibility criteria. To achieve these aims, the researchers are conducting a multicenter, prospective, 24 months study of 320 subjects.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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FSHD-COM
All participants will be asked to undergo FSHD-specific functional rating scale tests and procedures and Electrical Impedance Myography.
FSHD-specific functional rating scale
The FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance.
Electrical Impedance Myography
EIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs.
Interventions
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FSHD-specific functional rating scale
The FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance.
Electrical Impedance Myography
EIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patients with symptomatic limb weakness
* Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
* If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
Exclusion Criteria
* Patients with orthopedic conditions that preclude safe testing of muscle function
* Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
* Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
* Patients that are pregnant
18 Years
75 Years
ALL
No
Sponsors
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National Institute of Neurological Disorders and Stroke (NINDS)
NIH
FSHD Society
OTHER
Friends Research Institute, Inc.
OTHER
Muscular Dystrophy Association
OTHER
AFM Telethon
UNKNOWN
University of Rochester
OTHER
Leiden University Medical Center
OTHER
Dyne Therapeutics
INDUSTRY
University of Kansas Medical Center
OTHER
Responsible Party
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Principal Investigators
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Jeffrey Statland, MD
Role: PRINCIPAL_INVESTIGATOR
University of Kansas Medical Center
Rabi Tawil, MD
Role: PRINCIPAL_INVESTIGATOR
University of Rochester
Locations
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University of California Los Angeles
Los Angeles, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Rochester Medical Center
Rochester, New York, United States
The Ohio State University
Columbus, Ohio, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
University of Washington
Seattle, Washington, United States
Chu De Nice
Nice, , France
Institut de Myologie
Paris, , France
Ludwig-Maximilians-Universität München
München, , Germany
Centro Clinico NeMO
Milan, , Italy
Radboud Unviersity
Nijmegen, , Netherlands
University of College London - Queens Square
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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References
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LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x.
Other Identifiers
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STUDY00140842
Identifier Type: -
Identifier Source: org_study_id
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