RECRUITING

Clenbuterol to Target DUX4 in FSHD

Description

The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.

Conditions

Muscular Dystrophy, Facioscapulohumeral
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Related Conditions:

Muscular Dystrophy, Facioscapulohumeral

Study Overview

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Study Details

Study overview

The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.

Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol

Clenbuterol to Target DUX4 in FSHD

Condition
Muscular Dystrophy, Facioscapulohumeral
Intervention / Treatment

-

Contacts and Locations

Kansas City

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

Rochester

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

Seattle

University of Washington, Seattle, Washington, United States, 98104

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

  • * Genetically confirmed diagnosis of FSHD type 1 or 2, or have a clinical diagnosis of FSHD type 1 with a first degree relative with confirmed mutation
  • * between 18 and 75 years of age
  • * with a clinical severity score between 1 and 4
  • * Able to walk 30ft without support of another person
  • * Showing anti-gravity strength on at least one of the tibialis anterior muscles or having an MRI eligible muscle in the leg for needle biopsy
  • * willing and able to provide informed consent
  • * Pregnant or planning to become pregnant during the conduct of the study
  • * have a poorly controlled medical condition
  • * Were involved in a study of an experimental agent within 3 months of enrollment
  • * Are taking beta-blockers or anabolic agent or potassium wasting diuretics
  • * have any condition or contraindication which would interfere with testing or preclude use of beta-agonist
  • * Are taking blood thinners or medications which make a needle muscle biopsy contra-indicated
  • * Are taking any medications or therapies with a contraindication to Clenbuterol

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Jeffrey Statland,

Study Record Dates

2028-07