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New treatment option for Muscular Dystrophy, Facioscapulohumeral
Official title Clenbuterol to Target DUX4 in FSHD
ClinicalTrials.gov ID: NCT06721299
What this study is testing
What is Clenbuterol?
Clenbuterol is an investigational medicine, being studied as a potential treatment for muscular dystrophy, facioscapulohumeral.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility 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
- 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...
- Are taking any medications or therapies with a contraindication to Clenbuterol
The study team makes the final eligibility decision.
Where it's taking place
- Kansas City, Kansas, United States
- Rochester, New York, United States
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Kansas City, Kansas, United States; Rochester, New York, United States; Seattle, Washington, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.