Muscular Dystrophy, FacioscapulohumeralPhase 1Target FSHDNCT06721299
Clenbuterol to Target DUX4 in FSHD
A Phase 1 study testing Clenbuterol in people with Muscular Dystrophy, Facioscapulohumeral, run by Jeffrey Statland at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Muscular Dystrophy, Facioscapulohumeral
- What is being tested?
- Clenbuterol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 3 sitesUnited States
- Study dates
- 25 Jun 2025to Jul 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Rebecca Clay, BS, 913-945-9936, rclay@kumc.edu
Jeffrey Statland
- Recruiting
University of Rochester Medical Center
Rochester, New York, 14642
Leann Lewis, MSGC, 585-275-7680, Leann_Lewis@URMC.Rochester.edu
Brianna Brun
- Recruiting
University of Washington
Seattle, Washington, 98104
Lilly Young, 206-616-5957, eyoung8@uw.edu
Leo Wang
About this study
Official title: Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol
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.
Read the detailed description
Clenbuterol is an EMA approved drug for COPD that three independent patient derived screens identified as suppressing DUX4 expression in cultured FSHD muscle. Prior clinical studies with related beta2-agonists showed some activity in FSHD but did not meet their primary endpoint, although the prior studies would likely have been designed differently with current knowledge. Target FSHD is a 6-month open-label multiple ascending dose study of clenbuterol for safety and tolerability to determine the best dose for a future trial of efficacy. In addition, this study will collect secondary outcome data on muscle function, MRI changes (lean muscle volume, fat infiltration, STIR-rating) and molecular markers of disease activity (histopathology and pre-determined baskets of DUX4-target, inflammation, and ECM genes) at the beginning and end of the study to assess and power their utility as measures of drug activity in a future interventional study of efficacy.
- Conditions
- Muscular Dystrophy, Facioscapulohumeral
- Being tested
- Clenbuterol
- Keywords
- FSHD
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClenbuterolDrug
Beta-Agonist
| Study group | Role | What they receive |
|---|---|---|
| Clenbuterol Cohort 1 | Experimental | 20 mcg taken orally twice daily Receives: Drug: Clenbuterol |
| Clenbuterol Cohort 2 | Experimental | 40 mcg taken orally twice daily Receives: Drug: Clenbuterol |
| Clenbuterol Cohort 3 | Experimental | 60 mcg taken orally twice daily Receives: Drug: Clenbuterol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Clenbuterol-related adverse reactions
Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.
Measured: from baseline to 6 month visit
change in heart rate
Review of Vitals
Measured: from Month 1 to Month 6 visit
change in blood pressure
Review of Vitals
Measured: from Month 1 to Month 6 visit
Safety Lab Potassium (K)
patient safety measured by potassium for signs of hypokalemia
Measured: Baseline to month 6 visit
Safety Lab Glucose
Patient safety measured by blood glucose abnormalities
Measured: Baseline to month 6 visit
Tolerability of 3 doses of clenbuterol in sequential cohorts
Tolerability will be defined as completing an arm without any clenbuterol related adverse events causing someone to withdraw
Measured: from baseline to 6 month visit
Safety ECG
Looking for sympathomimetic side effects such as cardiac arrhythmias
Measured: from Month 1 to Month 6 visit
Safety Lab Creatine kinase (CK)
Patient Safety measured by creatine kinase abnormalities
Measured: Baseline to Month 6 visit
Secondary outcomes (9)
MRI
MRI measurements are considered gold standard for lean muscle volume measurement and disease biomarkers (fat volumes, fat faction, and STIR+ presence) that may be modulated by treatment. In addition, we will include analysis to help make needle biopsy based on STIR and quantitative fat fraction more efficient with higher yield using standard fiducial markings and body landmarks.
Measured: Screening to Month 6 Visit
Muscle Biopsy for RNA sequencing
change in a set of DUX4-regulated genes as measured in muscle using RNA sequencing. goal is to better understand the molecular changes in muscle tissue effected by the use of Clenbuterol.
Measured: Baseline and Month 6
FSHD-COM
An 18-item evaluator administered instrument comprised of individually validated functional motor task.the body regions assessed match areas of importance identified by patients and include leg function; shoulder and arm function; trunk function, hand function; and functional balance
Measured: Screening to Month 6 Visit
Manual Muscle Testing (MMT)
a modified Medical Research Council 13-point scale with standardized positions for each muscle will help look at subject's change in strength over time.
Measured: Screening to Month 6 Visit
Quantitative Muscle Testing (QMT)
Will test 5 bilateral muscles using a fixed myometry testing system with a force transducer attached by an inelastic strap to a metal frame, This will help us assess subjects change in strength
Measured: Screening to Month 6 Visit
Patient Reported Outcome Measurement Information System-57 (Promis57)
The PROMIS57 is an instrument developed by the NIH which generates scores for physical function, and the impact of physical limitations on daily life. 57 questions are summed into a total score, which is transformed into a normalized t-score with 50 representing normal, and lower scores representing increasing disability.
Measured: Screening to Month 6 Visit
FSHD Rasch-built overall disability scale (FSHD-RODS)
The FSHD-RODS is a questionnaire about the relationship between daily activities and health. 32 questions are are constructed using a modern clinimetric technique, the Rasch analysis, which by ordering the items in increasing difficulty, allows for the calculation of the sum of item scores to achieve a total score. Answers provide information about how FSHD affects daily and social activities and to what degree participants are able to perform usual activities.
Measured: Screening to Month 6 Visit
Upper Extremity Functional Index (UEFI)
This index measures upper extremity dysfunction. 20 questions are combined into a total score, the score is transformed into a normalized score with 80 representing normal, and lower scores representing increasing disability.
Measured: Screening to Month 6 visit
Clinical Global Impression of Severity and Change (CGI-S and CGI-C)
Is a rating scale that measures symptom severity and change in severity in response to treatment in clinical trials.
Measured: Screening to Month 6 visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2024
- Study start25 Jun 2025
- Primary completionFeb 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, FacioscapulohumeralStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Three sequential multiple ascending dose cohorts (of n=10 participants)
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jeffrey Statland
- Sponsor type
- University, hospital or other
- Collaborators
- University of Washington, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), University of Rochester
- Responsible party
- Sponsor-investigator, Jeffrey Statland, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06721299
- Sponsor's protocol ID
- STUDY00150777
- Other IDs
- P50AR065139
- Acronym
- Target FSHD
- Status last verified
- Feb 2026
- First submitted
- 5 Sep 2024
- Last updated
- 25 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol
- ClinicalTrials.gov ID
- NCT06721299
- Acronym / sponsor ID
- Target FSHD / STUDY00150777
- Conditions
- Muscular Dystrophy, Facioscapulohumeral
- Keywords
- FSHD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Jeffrey StatlandUniversity, hospital or other
- Collaborators
- University of Washington, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), University of Rochester
- Study start
- 25 Jun 2025 (actual)
- Primary completion
- Feb 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 6 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Clenbuterol is an EMA approved drug for COPD that three independent patient derived screens identified as suppressing DUX4 expression in cultured FSHD muscle. Prior clinical studies with related beta2-agonists showed some activity in FSHD but did not meet their primary endpoint, although the prior studies would likely have been designed differently with current knowledge. Target FSHD is a 6-month open-label multiple ascending dose study of clenbuterol for safety and tolerability to determine the best dose for a future trial of efficacy. In addition, this study will collect secondary outcome data on muscle function, MRI changes (lean muscle volume, fat infiltration, STIR-rating) and molecular markers of disease activity (histopathology and pre-determined baskets of DUX4-target, inflammation, and ECM genes) at the beginning and end of the study to assess and power their utility as measures of drug activity in a future interventional study of efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Three sequential multiple ascending dose cohorts (of n=10 participants)
| Arm | Type | Receives |
|---|---|---|
| Clenbuterol Cohort 1 | Experimental | Drug: Clenbuterol20 mcg taken orally twice daily |
| Clenbuterol Cohort 2 | Experimental | Drug: Clenbuterol40 mcg taken orally twice daily |
| Clenbuterol Cohort 3 | Experimental | Drug: Clenbuterol60 mcg taken orally twice daily |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ClenbuterolBeta-Agonist | Drug | Clenbuterol Cohort 1, Clenbuterol Cohort 2, Clenbuterol Cohort 3 |
Primary outcome
Frequency of Clenbuterol-related adverse reactions
Time frame: from baseline to 6 month visit
Patient report and medical records will be used to document adverse events and severe adverse events. Adverse events and severe adverse events will be assessed by the investigator and reported as needed for safety.
change in heart rate
Time frame: from Month 1 to Month 6 visit
Review of Vitals
change in blood pressure
Time frame: from Month 1 to Month 6 visit
Review of Vitals
Safety Lab Potassium (K)
Time frame: Baseline to month 6 visit
patient safety measured by potassium for signs of hypokalemia
Safety Lab Glucose
Time frame: Baseline to month 6 visit
Patient safety measured by blood glucose abnormalities
Tolerability of 3 doses of clenbuterol in sequential cohorts
Time frame: from baseline to 6 month visit
Tolerability will be defined as completing an arm without any clenbuterol related adverse events causing someone to withdraw
Safety ECG
Time frame: from Month 1 to Month 6 visit
Looking for sympathomimetic side effects such as cardiac arrhythmias
Safety Lab Creatine kinase (CK)
Time frame: Baseline to Month 6 visit
Patient Safety measured by creatine kinase abnormalities
9 secondary outcomes
MRI
Time frame: Screening to Month 6 Visit
Muscle Biopsy for RNA sequencing
Time frame: Baseline and Month 6
FSHD-COM
Time frame: Screening to Month 6 Visit
Manual Muscle Testing (MMT)
Time frame: Screening to Month 6 Visit
Quantitative Muscle Testing (QMT)
Time frame: Screening to Month 6 Visit
Patient Reported Outcome Measurement Information System-57 (Promis57)
Time frame: Screening to Month 6 Visit
FSHD Rasch-built overall disability scale (FSHD-RODS)
Time frame: Screening to Month 6 Visit
Upper Extremity Functional Index (UEFI)
Time frame: Screening to Month 6 visit
Clinical Global Impression of Severity and Change (CGI-S and CGI-C)
Time frame: Screening to Month 6 visit
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Clenbuterol in people with Muscular Dystrophy, Facioscapulohumeral, run by Jeffrey Statland at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rebecca Clay (9139459936, rclay@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of Clenbuterol-related adverse reactions (time frame: from baseline to 6 month visit). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2026.