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RecruitingPhase 1NCT06721299

Clenbuterol to Target DUX4 in FSHD

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

Sponsor

Jeffrey Statland

Enrollment

30 participants

Start Date

Jun 25, 2025

Study Type

INTERVENTIONAL

Conditions

Muscular Dystrophy, Facioscapulohumeral

Summary

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.

Eligibility

Min Age: 18 YearsMax Age: 75 Years

Inclusion Criteria7

  • 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 0 and 10
  • 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
  • agree to follow the contraceptive requirement for duration of the study

Exclusion Criteria9

  • 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
  • Are taking or are planning to take a GLP-1 Agonist during trial
  • 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
  • Has contraindication to lactose such as galactosmia, lactase deficiency and glucose-galactose malabsorption. For those who are lactose intolerant, the PI will determine acceptability based on tolerance reaction to lactose
  • Are taking any medications or therapies with a contraindication to Clenbuterol

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Interventions

DRUGClenbuterol

Beta-Agonist

Locations(3)

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Rochester Medical Center

Rochester, New York, United States

University of Washington

Seattle, Washington, United States

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NCT06721299

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