Rho Kinase Inhibitor for ALS

(REAL Trial)

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Fasudil, a Rho kinase inhibitor, for individuals with Amyotrophic Lateral Sclerosis (ALS), a condition affecting nerve cells in the brain and spinal cord. Researchers aim to determine if Fasudil is safe and effective in reducing symptoms. Participants will receive one of two doses of Fasudil. Those who have experienced ALS symptoms, such as weakness or speech problems, within the last four years might be suitable candidates. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that Fasudil (WP-0512) is likely to be safe for humans?

Research has shown that fasudil, the treatment being tested in this trial, is generally safe and well-tolerated by people with ALS (amyotrophic lateral sclerosis). Previous studies, carefully controlled and including a placebo (a harmless pill with no treatment effect), found that fasudil did not cause major side effects. However, experts indicate that more research is needed to fully understand its benefits. Overall, fasudil appears to be a safe option for those considering joining a trial like this one. ^{1 2 3 4 5}

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

Most treatments for ALS, like riluzole and edaravone, aim to slow disease progression by reducing neuron damage or oxidative stress. But fasudil works differently, targeting the Rho kinase enzyme that is involved in nerve cell degeneration. Researchers are excited about fasudil because it offers a new mechanism of action that could potentially protect nerve cells more effectively. Additionally, fasudil is administered orally, which is convenient compared to some existing treatments that require intravenous delivery. This novel approach could lead to better outcomes for people living with ALS.

What evidence suggests that Fasudil might be an effective treatment for ALS?

Research has shown that fasudil, a type of medication, is safe and well-tolerated in people with ALS. In this trial, participants will receive fasudil in different dosages: one group will receive 180 mg/day, and another will receive 300 mg/day. Studies suggest that its effects on ALS symptoms warrant further exploration in larger studies. While early results are promising, more research is needed to confirm its effectiveness in treating ALS. Fasudil targets pathways that may protect nerve cells, potentially slowing the disease's progression. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39424560/

a phase 2, randomised, double-blind, placebo-controlled trial Interpretation: Fasudil was well tolerated and safe in people with amyotrophic lateral sclerosis. The effect of fasudil on efficacy outcomes should be explored ...

2. thelancet.com thelancet.com/journals/laneur/article/PIIS1474-4422(24)00373-9/fulltext

Safety, tolerability, and efficacy of fasudil in amyotrophic ... Fasudil was well tolerated and safe in people with amyotrophic lateral sclerosis. The effect of fasudil on efficacy outcomes should be explored in larger ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05218668

Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL) This study will be composed of a Primary Phase, with 24 weeks of open-label treatment, and an optional 42-month Extension Phase.

4. jwatch.org jwatch.org/na58401/2025/02/13/safety-and-efficacy-rho-associated-kinase-inhibition-novel

Safety and Efficacy of Rho-Associated Kinase Inhibition, a ... A phase 2, double-blind, placebo-controlled trial showed that the inhibitor fasudil is safe and tolerable, but larger phase 3 trials are needed.

5. als.net als.net/als-trial-navigator/500014/

Trial: Rho Kinase Inhibitor in Amyotrophic Lateral Sclero... A Phase 2a Open-Label Preliminary Safety, Efficacy, and Biomarker Study of WP-0512 in Patients with Amyotrophic Lateral Sclerosis (ALS)