Could a common asthma drug help with a rare muscle disease? new study launches.
NCT ID NCT06721299
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study is testing whether clenbuterol, a drug used for breathing problems, is safe for people with facioscapulohumeral muscular dystrophy (FSHD). About 30 adults with FSHD will receive one of three doses of the drug. Researchers will monitor side effects, heart rate, blood pressure, and lab values to find a dose that people can tolerate.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2025
- Expected to finish
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Jul 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
3 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Rochester Medical Center
RECRUITINGRochester, New York, 14642, United States
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University of Washington
RECRUITINGSeattle, Washington, 98104, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New hope for FSHD: Long-Term drug safety trial underway
- New hope for FSHD: experimental drug AOC 1020 completes early testing
- New study aims to sharpen tools for testing kids with rare muscle disease
- FSHD patients unite: new registry aims to accelerate research and care
- New hope for rare muscle disease: experimental drug enters human trials