Rho Kinase Inhibitor for ALS

(REAL Trial)

Recruiting at 11 trial locations

Overseen By Ann C Tunstall, Ph. D.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Woolsey Pharmaceuticals

Must be taking: Riluzole, Edaravone, Phenylbutyrate, TUDCA

Must not be taking: Clenbuterol, Anticoagulants, others

Disqualifiers: Neuromuscular diseases, Ventilation, 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: 12

Duration: 24 weeks

40 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new drug called WP-0512 to see if it can help people with ALS. It targets patients who have had ALS symptoms for a few years and meet specific health criteria. The drug aims to slow down the progression of ALS symptoms.

Do I need to stop my current medications to join the trial?

If you are taking riluzole, edaravone, or phenylbutyrate (PB) and/or tauroursodeoxycholic acid (TUDCA), you can continue them as long as your dose has been stable for a certain period before the trial. If you plan to stop these medications, you must do so at least 28 days before the trial starts. The protocol does not specify about other medications, so it's best to discuss with the trial team.

What data supports the effectiveness of the drug Fasudil for ALS?

Research shows that Fasudil, a drug already used for other conditions, improved motor function and survival in animal models of ALS. In a small compassionate use case, one ALS patient showed improved lung function, and the drug was well-tolerated by all patients. ^{1 2 3 4 5}

Is Fasudil safe for use in humans?

Fasudil has been used safely in Japan for many years to treat blood vessel spasms after brain bleeding, and in a small study with ALS patients, it was well-tolerated without obvious side effects. ^{1 2 3 4 5}

How is the drug Fasudil unique in treating ALS?

Fasudil is unique in treating ALS because it is a Rho kinase (ROCK) inhibitor that has shown potential to protect motor neurons and modulate immune cells, which may slow disease progression. Unlike existing treatments, it combines neuroprotection and immunomodulation, and is already used in Japan for other conditions, indicating a favorable safety profile. ^{1 2 3 4 5}

Eligibility Criteria

This trial is for adults aged 18-75 with ALS diagnosed within the last 4 years, showing a specific rate of disease progression. Participants must have been on stable doses of certain ALS medications if they're taking them and agree to use contraception. Those with severe blood pressure issues, kidney problems, other neuromuscular diseases, or who are pregnant can't join.

Inclusion Criteria

I have been on a stable dose of my ALS medication for the required time.

Your ALS disease is getting worse at a steady rate of 0.5 to 3 points per month.

I have been diagnosed with ALS and no other cause for my symptoms has been found.

Exclusion Criteria

My blood tests show low neutrophils or platelets, high INR, or I can't have a spinal tap.

I am on a breathing machine through a hole in my neck.

Known to be pregnant or lactating, or positive pregnancy test for WCBP

Timeline

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

2 visits (in-person)

Treatment

Participants receive open-label treatment with WP-0512, with regular ALS assessments and safety evaluations

24 weeks

6 visits (in-person), 1 visit (telephone)

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week

1 visit (in-person)

Extension

Participants may opt into a long-term extension phase with continued ALS assessments

42 months

Visits every 3 months

Treatment Details

Interventions

• Fasudil (WP-0512)

Trial Overview The REAL trial is testing Fasudil (WP-0512), a Rho Kinase Inhibitor, in an open-label Phase 2a study to evaluate its safety and effectiveness in slowing down the progression of ALS. It also looks at how the drug affects certain biomarkers related to the disease.

Participant Groups

Group I: Cohort 2 - Fasudil Experimental Treatment 1 Intervention

Oral fasudil at 300 mg/day

Group II: Cohort 1 - Fasudil Experimental Treatment 1 Intervention

Oral fasudil at 180 mg/day

Who Is Running the Clinical Trial?

Woolsey Pharmaceuticals

Lead Sponsor

Trials

3

Recruited

80+

Findings from Research

The ROCK inhibitor Fasudil, when administered orally to symptomatic SOD1.G93A transgenic ALS mice, significantly improved motor behavior in male mice, indicating potential therapeutic benefits even after the onset of symptoms.

Although Fasudil did not significantly alter survival or spinal cord pathology, its well-tolerated nature and existing clinical use for other conditions suggest it could be a promising candidate for further investigation in ALS treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rho Kinase Inhibition with Fasudil in the SOD1G93A Mouse Model of Amyotrophic Lateral Sclerosis-Symptomatic Treatment Potential after Disease Onset. Günther, R., Balck, A., Koch, JC., et al.[2020]

References

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

Rho kinase inhibition modulates microglia activation and improves survival in a model of amyotrophic lateral sclerosis. [2016]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Rho Kinase Inhibition with Fasudil in the SOD1G93A Mouse Model of Amyotrophic Lateral Sclerosis-Symptomatic Treatment Potential after Disease Onset. [2020]

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

Fasudil, a rho kinase inhibitor, limits motor neuron loss in experimental models of amyotrophic lateral sclerosis. [2022]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Compassionate Use of the ROCK Inhibitor Fasudil in Three Patients With Amyotrophic Lateral Sclerosis. [2020]

5. Switzerland pubmed.ncbi.nlm.nih.gov

ROCK-ALS: Protocol for a Randomized, Placebo-Controlled, Double-Blind Phase IIa Trial of Safety, Tolerability and Efficacy of the Rho Kinase (ROCK) Inhibitor Fasudil in Amyotrophic Lateral Sclerosis. [2020]