Losmapimod for Facioscapulohumeral Muscular Dystrophy

Recruiting at 16 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Fulcrum Therapeutics

Disqualifiers: Cardiovascular, CNS disease, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 24

Duration: 48 weeks

76 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing if the medication Losmapimod is safe for long-term use in patients with FSHD1. Patients will take the medication twice daily and visit the clinic regularly to check for any side effects. Losmapimod has been previously tested in other conditions but did not show significant efficacy.

Will I have to stop taking my current medications?

You may need to stay on a stable dose of any medications or supplements that affect muscle function for at least 3 months before starting the study and continue on that dose during the study. Any changes to your medication can only be made for strict medical reasons.

Is losmapimod safe for humans?

Losmapimod has been evaluated for safety in a Phase 1 clinical trial for facioscapulohumeral muscular dystrophy, focusing on its safety and tolerability in humans. ^{1 2 3 4 5}

How is the drug losmapimod unique for treating facioscapulohumeral muscular dystrophy?

Losmapimod is unique because it targets the underlying genetic mechanism of facioscapulohumeral muscular dystrophy by engaging with specific proteins in the blood and muscle, which is different from other treatments that may not address the root cause of the disease. ^{1 3 6 7 8}

What data supports the effectiveness of the drug Losmapimod for treating facioscapulohumeral muscular dystrophy?

The 2021 FSHD International Research Congress discussed results from the ReDUX4 trial, a phase 2b clinical trial of losmapimod in FSHD, indicating ongoing research into its effectiveness. Additionally, advances in understanding the disease have led to the development of targeted therapies, including losmapimod, which are being explored in clinical trials. ^{1 3 9 10 11}

Who Is on the Research Team?

Marie-Helene Jouvin, MD

Principal Investigator

Fulcrum Therapeutics

Are You a Good Fit for This Trial?

This trial is for adults aged 18-65 with Facioscapulohumeral Muscular Dystrophy (FSHD) who were in the ReDux4 study. Participants must be able to follow the study plan and use approved birth control methods. Those with illnesses that could affect results or pose risks, allergies, heart or brain diseases, other muscle disorders, or significant mental illness cannot join.

Inclusion Criteria

Will practice an approved method of birth control

The patient must have consented to participate and must have provided signed, dated and witnessed an IRB-approved informed consent form that conforms to federal and institutional guidelines

I am willing and able to follow the study's schedule, treatments, tests, and guidelines.

Exclusion Criteria

I've been on a stable dose of medication or supplements that could affect muscle function for at least 3 months.

I do not have any health conditions that could interfere with the study or increase risk from the study drug.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 15 mg of losmapimod by mouth twice daily for a total of 30 mg daily

48 weeks

Clinic visits approximately every 12 weeks

Open-label extension

Participants continue to receive losmapimod until 90 days after it becomes commercially available or the study is closed

Up to 60 months

Clinic visits approximately every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Losmapimod

Trial Overview The trial tests Losmapimod's long-term safety and effectiveness in treating FSHD. It's an open-label extension meaning all participants know they are receiving Losmapimod and there is no placebo group involved.

How Is the Trial Designed?

Group I: Losmapimod Experimental Treatment 1 Intervention

FSHD1 patients with genetic confirmation will receive Losmapimod 15 mg by mouth twice daily for a total of 30 mg daily until 90 days after commercial drug is available post regulatory approval or until the study is discontinued by the Sponsor.

Who Is Running the Clinical Trial?

Fulcrum Therapeutics

Lead Sponsor

Trials

8

Recruited

680+

Headquarters

Cambridge, United States

Published Research Related to This Trial

Losmapimod was found to be well tolerated in both healthy volunteers and patients with facioscapulohumeral dystrophy (FSHD), with no serious adverse events reported, indicating a good safety profile for this medication.

The study demonstrated dose-dependent pharmacokinetics and target engagement in both blood and muscle, suggesting that losmapimod effectively reaches its intended sites of action, supporting its advancement to Phase 2 trials for FSHD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. Mellion, ML., Ronco, L., Berends, CL., et al.[2022]

The FSHD-COM, a new functional outcome measure for facioscapulohumeral muscular dystrophy (FSHD), showed excellent test-retest reliability with an intraclass correlation coefficient of 0.96, indicating it is a consistent tool for assessing patient function.

This composite measure demonstrated strong correlations with established disease metrics, suggesting it effectively reflects the severity and impact of FSHD, making it suitable for use in future clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Facioscapulohumeral muscular dystrophy functional composite outcome measure. Eichinger, K., Heatwole, C., Iyadurai, S., et al.[2020]

Citations

1. England pubmed.ncbi.nlm.nih.gov

Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement. [2022]

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

Meeting report: the 2021 FSHD International Research Congress. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Facioscapulohumeral muscular dystrophy: the road to targeted therapies. [2023]

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

Facioscapulohumeral muscular dystrophy functional composite outcome measure. [2020]

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

Muscle Quantitative MR Imaging and Clustering Analysis in Patients with Facioscapulohumeral Muscular Dystrophy Type 1. [2018]

6. Ukraine pubmed.ncbi.nlm.nih.gov

A rare coincidence: facioscapulohumeral muscular dystrophy and breast cancer. [2014]

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

Facioscapulohumeral dystrophy. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Updating the Clinical Picture of Facioscapulohumeral Muscular Dystrophy: Ramifications for Drug Development With Potential Solutions. [2021]

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

The boy who lost his smile: facioscapulohumeral dystrophy in the head and neck. [2015]

10. England pubmed.ncbi.nlm.nih.gov

Facioscapulohumeral muscular dystrophy. [2013]

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

Facioscapulohumeral muscular dystrophy. [2021]