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
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)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06721299