NCT06721299 · Jeffrey Statland
Clenbuterol to Target DUX4 in FSHD
(Target FSHD)
What this study is about
The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and how well patients handle the treatment of the medication at three different dose levels.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Kansas City, Kansas
- Rochester, New York
- Seattle, Washington
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 25, 2026 · Source of record for eligibility and locations