RECRUITING

Clenbuterol to Target DUX4 in FSHD

Conditions

Muscular Dystrophy, Facioscapulohumeral FSHD

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

Clenbuterol to Target DUX4 in FSHD (Target FSHD): Open Label Safety and Tolerability Study of 3 Doses of Clenbuterol

Quick Facts

Study Start:2025-06

Study Completion:2028-07

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06721299

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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 1 and 4 * 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	* 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 * 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 * Are taking any medications or therapies with a contraindication to Clenbuterol

Inclusion Criteria

Exclusion 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 1 and 4
* 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

* 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
* 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
* Are taking any medications or therapies with a contraindication to Clenbuterol

Contacts and Locations

Study Contact

Rebecca Clay

CONTACT

9139459936

rclay@kumc.edu

Study Locations (Sites)

University of Kansas Medical Center

Kansas City, Kansas, 66160

United States

University of Rochester Medical Center

Rochester, New York, 14642

United States

University of Washington

Seattle, Washington, 98104

United States

Collaborators and Investigators

Sponsor: Jeffrey Statland

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06

Study Completion Date2028-07

Study Record Updates

Study Start Date2025-06

Study Completion Date2028-07

Terms related to this study

Keywords Provided by Researchers

FSHD

Additional Relevant MeSH Terms

Muscular Dystrophy, Facioscapulohumeral