Actively Recruiting
Clenbuterol to Target DUX4 in FSHD: Open Label Safety and Tolerability Study of Three Doses of Clenbuterol
Led by Jeffrey Statland · Updated on 2026-02-25
30
Participants Needed
3
Research Sites
21 weeks
Total Duration
On this page
Sponsors
J
Jeffrey Statland
Lead Sponsor
U
University of Washington
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating Clenbuterol as a possible treatment for Facioscapulohumeral Muscular Dystrophy (FSHD). This study aims to determine the safety and tolerability of three different doses of Clenbuterol in people with FSHD. Prior studies with related medications showed some effects but did not meet their main goals, so this 6-month open-label study will help identify the best dose for future research. Participants will receive one of three doses of Clenbuterol taken orally twice daily: 20 mcg, 40 mcg, or 60 mcg. The study begins with screening, followed by treatment over six months with dose escalation in sequential groups. This design allows researchers to monitor safety and tolerability at each dose level before moving to the next. During the study, participants will undergo assessments including MRI scans to evaluate muscle changes, muscle biopsies for RNA analysis, muscle strength and function tests, and patient-reported outcome measures. Researchers will track adverse reactions, heart rate, blood pressure, and lab safety markers such as potassium, glucose, and creatine kinase from baseline to month 6. The total study duration for participants is six months, with careful safety monitoring throughout.
CONDITIONS
Brief Title
Clenbuterol to Target DUX4 in FSHD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of FSHD type 1 or 2, or a clinical diagnosis of FSHD type 1 with a first degree relative with confirmed mutation
- Age between 18 and 75 years
- Clinical severity score between 0 and 10
- Able to walk 30 feet without support from another person
- Anti-gravity strength on at least one tibialis anterior muscle or having an MRI-eligible muscle in the leg for needle biopsy
- Willing and able to provide informed consent
- Agree to follow contraceptive requirements during the study
You will not qualify if you...
- Pregnant or planning to become pregnant during the study
- Poorly controlled medical condition
- Participation in a study of an experimental agent within 3 months prior to enrollment
- Taking beta-blockers, anabolic agents, potassium wasting diuretics, GLP-1 agonists, blood thinners, or therapies contraindicated with Clenbuterol
- Any condition or contraindication interfering with testing or use of beta-agonists
- Contraindication to lactose such as galactosemia or lactase deficiency; lactose intolerant participants may be evaluated for tolerance
- Taking medications or therapies with contraindications to Clenbuterol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 months
Participants receive one of three doses of clenbuterol taken orally twice daily to assess safety and tolerability over 6 months. Muscle function, MRI changes, and molecular markers of disease activity are assessed at the beginning and end of treatment.
Monthly visits for 6 months
Trial Site Locations
Total: 3 locations
1
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
2
University of Rochester Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
3
University of Washington
Seattle, Washington, United States, 98104
Actively Recruiting
Research Team
R
Rebecca Clay
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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