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Recruiting NCT06721299

Clenbuterol to Target DUX4 in FSHD

Phase I Interventional Muscular Dystrophy, Facioscapulohumeral

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Clenbuterol.
Who it may be relevant to
Registry conditions: Muscular Dystrophy, Facioscapulohumeral. Basic parameters: 18 years — 75 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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.

Interventions

  • Drug Clenbuterol
    Beta-Agonist

Primary outcome measures

  • 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]
Secondary outcome measures (9)
  • 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]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 3 centers
  • University of Kansas Medical Center — Kansas City
  • University of Rochester Medical Center — Rochester
  • University of Washington — Seattle

Identifiers

NCT: NCT06721299 · STUDY00150777 · P50AR065139

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗