Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• 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 1 and 4
• 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
Locations
United States
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
New York
University of Rochester Medical Center
RECRUITING
Rochester
Washington
University of Washington
RECRUITING
Seattle
Contact Information
Primary
Rebecca Clay
rclay@kumc.edu
9139459936
Time Frame
Start Date: 2025-06-25
Estimated Completion Date: 2028-07
Participants
Target number of participants: 30
Treatments
Experimental: Clenbuterol Cohort 1
20 mcg taken orally twice daily
Experimental: Clenbuterol Cohort 2
40 mcg taken orally twice daily
Experimental: Clenbuterol Cohort 3
60 mcg taken orally twice daily
Related Therapeutic Areas
Sponsors
Collaborators: University of Washington, University of Rochester, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Leads: Jeffrey Statland