Clenbuterol for FSHD

(Target FSHD Trial)

Recruiting at 2 trial locations

Overseen By Rebecca Clay

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Jeffrey Statland

Must not be taking: Beta-blockers, Anabolic agents, Blood thinners

Disqualifiers: Pregnancy, Poorly controlled conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

Duration: 6 months

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Clenbuterol to determine its safety and tolerability for facioscapulohumeral muscular dystrophy (FSHD), a condition that causes muscle weakness, using three different doses. Participants will take Clenbuterol orally twice daily in varying amounts to identify the most effective dose without significant side effects. The trial seeks individuals with a genetically confirmed diagnosis of FSHD, who can walk 30 feet unassisted, and meet specific muscle condition criteria. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

You may need to stop taking certain medications, like beta-blockers, anabolic agents, potassium-wasting diuretics, blood thinners, or any medications that don't mix well with Clenbuterol. It's best to discuss your current medications with the study team to see if any changes are needed.

Is there any evidence suggesting that Clenbuterol is likely to be safe for humans?

Research shows that clenbuterol is under study as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD). Earlier studies have focused on determining the highest dose people can tolerate without major issues. The goal is to assess the safety of clenbuterol by monitoring for side effects.

Currently, the FDA has not approved clenbuterol for FSHD or any related condition in this trial. Ongoing research aims to evaluate how well participants tolerate doses ranging from 20 mcg to 60 mcg taken twice daily. As a phase 1 trial, the primary focus is on safety, and there is limited information on previous reactions.

Participants should be aware that clenbuterol is in the early testing stages for FSHD, with the main focus on ensuring its safety. Those considering joining the trial should consult their healthcare provider to understand the potential risks and benefits. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FSHD?

Clenbuterol is gaining attention for treating Facioscapulohumeral Muscular Dystrophy (FSHD) because it offers a fresh approach compared to current treatments, which primarily focus on managing symptoms. Unlike other treatments, Clenbuterol is a beta-2 adrenergic agonist, traditionally used in asthma, that may help increase muscle mass and strength by stimulating muscle growth and repair. Researchers are particularly interested in this mechanism because it directly targets muscle degeneration, the core issue in FSHD, rather than just alleviating symptoms. Additionally, Clenbuterol is administered orally, offering a convenient alternative to more invasive or complex treatment methods.

What evidence suggests that Clenbuterol might be an effective treatment for FSHD?

Research suggests that Clenbuterol, a type of medication, may help treat Facioscapulohumeral Muscular Dystrophy (FSHD) by blocking the activity of a protein called DUX4 in muscle cells. Since DUX4 is associated with muscle damage in FSHD, inhibiting it might protect muscles. Early lab studies have shown positive effects on weakened muscles, indicating potential for improvement. Although human studies provide limited information, these initial findings suggest Clenbuterol could be a promising treatment for FSHD. Participants in this trial will receive different dosages of Clenbuterol to evaluate its effectiveness and safety. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Facioscapulohumeral Muscular Dystrophy (FSHD). Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a confirmed diagnosis of FSHD.

Inclusion Criteria

I have FSHD type 1 or 2 confirmed by genetic tests or clinical diagnosis.

I can lift my foot against gravity or have a leg muscle eligible for biopsy.

Willing and able to provide informed consent

Exclusion Criteria

Pregnant or planning to become pregnant during the conduct of the study

Were involved in a study of an experimental agent within 3 months of enrollment

I am currently taking beta-blockers, anabolic agents, or diuretics that increase potassium loss.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive Clenbuterol at three different dose levels for safety and tolerability assessment

6 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Clenbuterol

Trial Overview The study is testing the safety and tolerability of Clenbuterol at three different doses to see if it can be a treatment option for FSHD. The goal is to find out which dose levels are safe for patients.

How Is the Trial Designed?

Group I: Clenbuterol Cohort 3 Experimental Treatment 1 Intervention

60 mcg taken orally twice daily

Group II: Clenbuterol Cohort 2 Experimental Treatment 1 Intervention

40 mcg taken orally twice daily

Group III: Clenbuterol Cohort 1 Experimental Treatment 1 Intervention

20 mcg taken orally twice daily

Who Is Running the Clinical Trial?

Jeffrey Statland

Lead Sponsor

Trials

2

Recruited

100+

University of Washington

Collaborator

Trials

1,858

Recruited

2,023,000+

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Collaborator

Trials

508

Recruited

1,090,000+

University of Rochester

Collaborator

Trials

883

Recruited

555,000+

Published Research Related to This Trial

The safety of marketed drugs is a significant concern, as some commonly prescribed medications can lead to serious or life-threatening side effects in patients.

The ChEMBL resource will provide a curated drug safety data set, including toxicity classifications and black box warnings, which will be freely available and regularly updated to aid in drug safety research and discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. Hunter, FMI., Bento, AP., Bosc, N., et al.[2023]

The study analyzed 345,662 spontaneous reports of adverse drug reactions (ADRs) submitted to the German Federal Institute for Drugs and Medical Devices, revealing a steady increase in reports since 1978, primarily due to stricter legal reporting requirements.

The most commonly reported ADRs were linked to drugs for nervous system disorders, with significant differences in reporting patterns between physicians and patients, suggesting a need for better patient education on potential ADRs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Frequent Adverse Drug Reactions, and Medication Groups under Suspicion. Dubrall, D., Schmid, M., Alešik, E., et al.[2023]

A total of 499 adverse drug reactions were reported under a voluntary reporting scheme, highlighting significant safety concerns such as life-threatening reactions to anti-inflammatory drugs and immediate hypersensitivity to anesthetics.

An additional 904 reactions were identified through an intensive survey in general practice, along with 342 from ongoing hospital monitoring, indicating a broad spectrum of drug reactions that require careful monitoring and analysis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Zealand Committee on Adverse Drug Reactions: eleventh annual report 1976. McQueen, EG.[2013]

The FDA Adverse Event Reporting System (FAERS) reported the highest number of nephrogenic systemic fibrosis (NSF) cases (1395), while the International Centre for Nephrogenic Systemic Fibrosis Registry (ICNSFR) provided the most complete histopathological data (335 cases).

The legal data set revealed that gadodiamide was the most commonly implicated gadolinium-based contrast agent (GBCA) in NSF cases, with 76.2% of cases linked to it, highlighting the importance of using diverse data sources for comprehensive safety evaluations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advancing pharmacovigilance through academic-legal collaboration: the case of gadolinium-based contrast agents and nephrogenic systemic fibrosis-a Research on Adverse Drug Events and Reports (RADAR) report. Edwards, BJ., Laumann, AE., Nardone, B., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06721299

NCT06721299 | Clenbuterol to Target DUX4 in FSHD In addition, this study will collect secondary outcome data on muscle function, MRI changes (lean muscle volume, fat infiltration, STIR-rating) and molecular ...

2. mdaconference.org mdaconference.org/abstract-library/dose-finding-safety-and-tolerability-study-of-clenbuterol-in-facioscapulohumeral-muscular-dystrophy-fshd/

Dose-Finding Safety and Tolerability Study of Clenbuterol ... Clenbuterol, a β2-adrenergic agonist, has shown promise in preclinical studies by inhibiting DUX4 activity in muscle cells derived from FSHD patients and ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12287641/

Current landscape for the management of ... The results showed no significant improvement in muscle strength or motor function with salbutamol compared to the placebo. They concluded that ...

4. kumc.trialstoday.org kumc.trialstoday.org/trial/NCT06721299

Clenbuterol to Target DUX4 in FSHD The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0022510X98000847

Examining potential drug therapies for muscular dystrophy ... The major finding of this study was that clenbuterol treatment has beneficial effects on 129ReJ dystrophic skeletal muscles, and has the potential to be a ...

6. trialx.com trialx.com/clinical-trials/listings/312423/clenbuterol-to-target-dux4-in-fshd/

Clenbuterol to Target DUX4 in FSHD 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 ...

7. synapse.patsnap.com synapse.patsnap.com/article/springbok-analytics-begins-phase-1-trial-for-clenbuterol-in-fshd-treatment

Springbok Analytics Begins Phase 1 Trial for Clenbuterol in ... Currently, there are no FDA-approved treatments for FSHD, highlighting a significant unmet medical need. Clenbuterol has shown promise in ...

8. reporter.nih.gov reporter.nih.gov/project-details/10922785

Facioscapulohumeral dystrophy clinical trial foundations The broad and long-term goals of this study are to further refine our understanding of MRI as a biomarker by applying artificial intelligence driven automated ...