Clenbuterol to Target DUX4 in FSHD
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorJeffrey Statland
About this trial
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 criteria
Qualifiers
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Clenbuterol
Treatment groups
30 Participants
are divided into 3 treatment groupsSponsors and collaborators
Jeffrey Statland
Lead sponsor
University of Kansas Medical Center
Sponsor institution
University of Washington
Collaborator
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
University of Rochester
Collaborator
Source ClinicalTrials.gov