What side effects are associated with this type of intervention?

The most common treatments for ALS include riluzole and edaravone. Riluzole is generally well-tolerated but can cause side effects such as nausea, weakness, decreased lung function, and liver enzyme abnormalities. Edaravone may lead to bruising, gait disturbances, and allergic reactions. Investigational treatments like levosimendan and reldesemtiv, which are being studied for their neuroprotective effects, have shown side effects including gastrointestinal disturbances and potential cardiovascular effects. Patients should discuss these potential side effects with their healthcare provider to make informed treatment decisions.

What prior FDA approvals exist?

The FDA has approved several treatments for Amyotrophic Lateral Sclerosis (ALS) that focus on neuroprotection and modulation of disease pathways. Riluzole, the first drug approved for ALS, is believed to reduce damage to motor neurons by decreasing the release of glutamate. Edaravone, another approved treatment, acts as a free radical scavenger, reducing oxidative stress in neurons. These treatments aim to slow disease progression and improve quality of life for ALS patients. Ongoing research, including trials like WP-0512, continues to explore new neuroprotective strategies and disease-modifying therapies.

What other types of research exist?

Promising alternatives to WP-0512 for ALS treatment include targeting the excitotoxicity pathway by reducing glutamate levels, as elevated glutamate is implicated in ALS progression. Additionally, reducing abnormal SOD1 from astrocytes or microglia has shown to slow disease progression. Anti-inflammatory approaches targeting microglia activation and the Wnt5a-mediated non-canonical Wnt/Ca2+ signaling pathway also offer potential neuroprotective benefits.

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Rho Kinase Inhibitor

Rho Kinase Inhibitor for ALS (REAL Trial)

Phase 2

Recruiting

Research Sponsored by Woolsey Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of probable laboratory-supported, probable, or definite ALS by Screening 1, with no other cause of neurological impairment identified by Screening 2

WCBP must agree to abstain from sex or use contraception, and males must agree to abstain from sex with WCBP or use contraception

Must not have

Neutrophil count < 1,500/mm3, platelets < 100,000/mm3, INR > 1.5, or any contraindication to or unable to tolerate lumbar puncture

Mechanical ventilation via tracheostomy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up monthly from screening to week 12; every six weeks to week 24; every 12 weeks to week 154

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for WP-0512 aren't listed here, similar drugs often cause headaches, dizziness, nausea, low blood pressure or changes in heart rate. Side effects may vary based on individual health conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I agree to use contraception or abstain from sex.

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My ALS symptoms started less than 4 years ago.

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I am between 18 and 75 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

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I have a condition that causes muscle weakness or a neurodegenerative disease.

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I have not taken clenbuterol in the last 28 days or between my first screening and enrollment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ monthly from screening to week 12; every six weeks to week 24; every 12 weeks to week 154

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~monthly from screening to week 12; every six weeks to week 24; every 12 weeks to week 154

This trial's timeline: 3 weeks for screening, Varies for treatment, and monthly from screening to week 12; every six weeks to week 24; every 12 weeks to week 154 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary study objectives

Change in the slope of the decline Revised ALS Functional Rating Scale (ALSFRS-R) during treatment vs pre-treatment lead-in

Change in the slope of the decline in muscle strength during treatment vs pre-treatment lead in

Change in the slope of the decline in percent predicted Slow Vital Capacity (SVC) during treatment vs pre-treatment lead-in

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2 - FasudilExperimental Treatment1 Intervention

Oral fasudil at 300 mg/day

Group II: Cohort 1 - FasudilExperimental Treatment1 Intervention

Oral fasudil at 180 mg/day

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Amyotrophic Lateral Sclerosis (ALS) focus on neuroprotection and modulation of disease pathways. Riluzole works by reducing glutamate excitotoxicity, which is believed to slow neuronal damage. Edaravone acts as an antioxidant, reducing oxidative stress and thereby protecting motor neurons from damage. Sodium phenylbutyrate-taurursodiol (PB-TURSO) combines two drugs that reduce neuronal cell death by targeting cellular stress pathways. These treatments are crucial for ALS patients as they aim to slow disease progression, preserve motor function, and improve quality of life by protecting neurons and modulating harmful cellular processes.

iPSC-derived LewisX+CXCR4+β1-integrin+ neural stem cells improve the amyotrophic lateral sclerosis phenotype by preserving motor neurons and muscle innervation in human and rodent models.