Recruiting
Phase 1

Clenbuterol

Sponsor:

Jeffrey Statland

Code:

NCT06721299

Conditions

Muscular Dystrophy, Facioscapulohumeral

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Clenbuterol

Study Details

Brief 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.

Conditions

Muscular Dystrophy, Facioscapulohumeral

Study ID

NCT06721299

Start date

Jun 25, 2025

Status verified date

Feb, 2026

Completion date

Jul, 2028

Anticipated

Primary completion date

Feb, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

30 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Clenbuterol Cohort 1

20 mcg taken orally twice daily

experimental: Clenbuterol Cohort 2

40 mcg taken orally twice daily

experimental: Clenbuterol Cohort 3

60 mcg taken orally twice daily

Interventions

Clenbuterol

Beta-Agonist

Primary outcome measure

  • Frequency of Clenbuterol-related adverse reactions [ Time Frame: from baseline to 6 month visit ]
  • change in heart rate [ Time Frame: from Month 1 to Month 6 visit ]
  • change in blood pressure [ Time Frame: from Month 1 to Month 6 visit ]
  • Safety Lab Potassium (K) [ Time Frame: Baseline to month 6 visit ]
  • Safety Lab Glucose [ Time Frame: Baseline to month 6 visit ]
  • Tolerability of 3 doses of clenbuterol in sequential cohorts [ Time Frame: from baseline to 6 month visit ]
  • Safety ECG [ Time Frame: from Month 1 to Month 6 visit ]
  • Safety Lab Creatine kinase (CK) [ Time Frame: Baseline to Month 6 visit ]

Central Contacts and Locations

Central contacts

Rebecca Clay

9139459936rclay@kumc.edu

Locations

University of Kansas Medical Center

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Rebecca Clay, BS

913-945-9936rclay@kumc.edu

Principal Investigator:

Jeffrey Statland

University of Rochester Medical Center

Recruiting

Rochester, New York, United States, 14642

Contacts

Leann Lewis, MSGC

585-275-7680Leann_Lewis@URMC.Rochester.edu

Principal Investigator:

Brianna Brun

University of Washington

Recruiting

Seattle, Washington, United States, 98104

Contacts

Lilly Young

206-616-5957eyoung8@uw.edu

More Information

Sponsor

Jeffrey Statland

Last update posted

Feb 25, 2026

Last verified

Feb, 2026

Keywords

  • FSHD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Jeffrey Statland on 2026-02-25.