Clenbuterol to Target DUX4 in FSHD

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-25

Study Completion Date

2028-07-31

Brief Summary

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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.

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Detailed Description

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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

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Muscular Dystrophy, Facioscapulohumeral

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Three sequential multiple ascending dose cohorts (of n=10 participants)

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Clenbuterol Cohort 1

20 mcg taken orally twice daily

Group Type EXPERIMENTAL

Clenbuterol

Intervention Type DRUG

Beta-Agonist

Clenbuterol Cohort 2

40 mcg taken orally twice daily

Group Type EXPERIMENTAL

Clenbuterol

Intervention Type DRUG

Beta-Agonist

Clenbuterol Cohort 3

60 mcg taken orally twice daily

Group Type EXPERIMENTAL

Clenbuterol

Intervention Type DRUG

Beta-Agonist

Interventions

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Clenbuterol

Beta-Agonist

Intervention Type DRUG

Eligibility Criteria

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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 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

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
* 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

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No