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

Trial Summary

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.

What data supports the effectiveness of the drug clenbuterol for FSHD?

Research suggests that clenbuterol, a drug similar to albuterol, may help increase muscle strength and volume in some types of muscular dystrophy, including FSHD, especially in the early stages of the disease. Additionally, clenbuterol has been shown to improve muscle mass in horses, indicating its potential to affect muscle growth. ^{1 2 3 4 5}

Is Clenbuterol generally safe for human use?

The available research does not provide specific safety data on Clenbuterol for human use, but it is important to note that drug safety is an ongoing concern, and adverse effects can occur with many medications. ^{6 7 8 9 10}

How does the drug clenbuterol differ from other treatments for FSHD?

Clenbuterol is unique because it is a beta2-adrenoceptor agonist, originally used as a bronchodilator for asthma and known for its anabolic effects on muscles, which may offer potential benefits for muscle-related conditions like FSHD. Unlike standard treatments, clenbuterol's mechanism of action involves stimulating muscle growth and fat loss, which is not typical for other FSHD therapies. ^{3 11 12 13 14}

What is the purpose of this trial?

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.

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Pilot trial of albuterol in facioscapulohumeral muscular dystrophy. FSH-DY Group. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Differential expression of skeletal muscle genes following administration of clenbuterol to exercised horses. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

Low Dose Clenbuterol Toxicity: Case Report and Review of Literature. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of clenbuterol HCl when administered to effect in horses with chronic obstructive pulmonary disease (COPD). [2019]

5. Japan pubmed.ncbi.nlm.nih.gov

[Therapeutic trial of beta 2-adrenergic agonist clenbuterol in muscular dystrophies]. [2013]

6. United States pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. [2023]

7. England pubmed.ncbi.nlm.nih.gov

A pilot study to build a database on seven anti-hypertensive drugs. [2006]

8. Germany pubmed.ncbi.nlm.nih.gov

Frequent Adverse Drug Reactions, and Medication Groups under Suspicion. [2023]

9. New Zealand pubmed.ncbi.nlm.nih.gov

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

10. England 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. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Comparison of serum and urinary concentrations of clenbuterol with and without concomitant administration of furosemide in horses. [2016]

12. England pubmed.ncbi.nlm.nih.gov

Role and possible mechanisms of clenbuterol in enhancing reverse remodelling during mechanical unloading in murine heart failure. [2022]

13. England pubmed.ncbi.nlm.nih.gov

Intratracheal clenbuterol in the horse: its pharmacological efficacy and analytical detection. [2013]

14. United States pubmed.ncbi.nlm.nih.gov

Acute clenbuterol induces hypotension, atrioventricular block and cardiac asystole in the rabbit. [2013]