Clenbuterol to Target DUX4 in FSHD

Recruiting now · Phase 1

Conditions studied: Muscular Dystrophy, Facioscapulohumeral

In brief

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.

Key facts

Study ID

NCT06721299

Run by

Jeffrey Statland

People needed

30

Starts

2025-06-25

Expected to finish

2028-07-01

Last updated by the study team

2026-02-25

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
• University of Rochester Medical Center — Rochester, New York, United States (enrolling)
• University of Washington — Seattle, Washington, United States (enrolling)

Full record on ClinicalTrials.gov

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