Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

Description

The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD

Conditions

Muscular Dystrophy, Facioscapulohumeral

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Study Overview

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Study Details

Study overview

The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD

Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

Condition
Muscular Dystrophy, Facioscapulohumeral
Intervention / Treatment

-

Contacts and Locations

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Iowa City

University of Iowa, Iowa City, Iowa, United States, 52242

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Rochester

University of Rochester, Rochester, New York, United States, 14642

Durham

Duke University, Durham, North Carolina, United States, 27708

Salt Lake City

University of Utah, Salt Lake City, Utah, United States, 84112

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Age 5-17 years.
  • * Genetically confirmed FSHD (types 1 or 2).
  • * Symptomatic weakness (facial, shoulder, core, or limb weakness)
  • * Able to complete a 10-meter walk without the support of another person in less than 12 seconds (canes, walking sticks, and braces allowed; no walker). In order to include early onset participants up to 8 individuals will be entered with baseline 10MWR \> 12 seconds or who are no longer ambulatory (≤10%)
  • * Unwilling or unable to provide informed consent or assent. Any other medical condition which in the opinion of the investigator would interfere with study participation.
  • * Malignancy with ongoing treatment with chemotherapeutic agents or anabolic agents
  • * Use of immunosuppressants including prednisone or performance enhancing drugs including testosterone within 6 months
  • * Pregnancy
  • * Recent or ongoing infection
  • * Presence of contraindication to performance of MRI: pacemaker, metallic foreign body in eye, brain aneurysm clip (unless documented as MRI compatible)
  • * In the opinion of the investigator unable to follow directions for standardized testing
  • * Note: Not being able to complete MRI will not result in a screen failure. If subject is not able to complete the procedure due to fear or anxiety, they will have the opportunity to try again at later visits. However, Subject must be willing to attempt to perform the MRI to meet inclusion/exclusion criteria

Ages Eligible for Study

5 Years to 17 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Kansas Medical Center,

Jeffrey Statland, MD, PRINCIPAL_INVESTIGATOR, University of Kansas Medical Center

Study Record Dates

2028-05