Clenbuterol treatment targeting DUX4 in FSHD

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a 6-month, open-label, multiple ascending dose Phase 1 program testing clenbuterol in adults with genetically or clinically confirmed FSHD. The primary goal is to determine safety and tolerability across three dose levels to identify the best dose for a future efficacy trial. Secondary measures collected at baseline and end of treatment include muscle function tests, MRI measures (lean muscle volume, fat infiltration, STIR), histopathology, and predetermined molecular baskets of DUX4-target, inflammation, and ECM genes. The approach is a repurposing of an EMA-approved COPD drug supported by patient-derived screens showing suppression of DUX4 in cultured FSHD muscle.

Why it matters

Who can join: the full rules

Inclusion 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 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 Criteria:

* 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's running

• University of Kansas Medical Center
  Kansas City, Kansas, United States Recruiting
• University of Rochester Medical Center
  Rochester, New York, United States Recruiting
• University of Washington
  Seattle, Washington, United States Recruiting

Study contacts

• Study coordinator: Rebecca Clay
• Email: rclay@kumc.edu
• Phone: 9139459936

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Apitegromab for FSHD in Adults (Phase 2)

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• Del-brax (AOC 1020) intravenous treatment for people with FSHD

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• Personalized exercise treatment for adults with rare neuromuscular disorders

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.