Rho Kinase (ROCK) Inhibitor in Tauopathies - 1

NCT04734379 | Unknown Phase 2 FDA Drug

• 1 other identifier: WP-0512-002
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 2a Open-Label Preliminary Safety, Tolerability, and Biomarker Study of Oral Fasudil in Patients with the 4-Repeat Tauopathies of Progressive Supranuclear Palsy-Richardson Syndrome or Corticobasal Syndrome

Conditions

Progressive Supranuclear Palsy; Corticobasal Syndrome

Outcome Measures

Primary Outcomes (1)

• Adverse events

  Incidence of adverse events \[AEs\] and serious adverse events \[SAEs\] as assessed by clinically significant abnormal physical examination findings; changes in vital signs; 12-lead electrocardiogram \[ECG\]; magnetic resonance imaging \[MRI\]; and hematology, blood chemistry, liver function, and urine tests.

  48 weeks

Secondary Outcomes (3)

• Phosphorylated tau

  48 weeks

• Biomarkers of neurodegeneration

  48 weeks

• Imaging biomarkers of neurodegeneration

  48 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

Oral fasudil 180 mg/day

Drug: Fasudil

Interventions

Fasudil DRUG

Oral fasudil 180 mg/day

Treatment

Eligibility Criteria

• Age: 35 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Between 35 and 80 years of age (inclusive).
• Able to walk at least 10 steps with minimal assistance (stabilization of one arm or use of cane/walker).
• MRI at Screening is consistent with the underlying neurodegenerative disease of the respective diagnostic cohort (i.e. PSP-RS or CBS), with no large strokes or severe white matter disease.
• Mini-Mental State Exam (MMSE) at Screening is between 20 and 30 (inclusive).
• For CBS: Amyloid beta (Aβ) positron emission tomography (PET) scan (florbetapir or equivalent) at Screening is not consistent with underlying Alzheimer's disease (AD). Previous Aβ PET scan negativity (assessed by a certified neuroradiologist) or previous AD CSF biomarker (Aβ/tau level, P-tau181 or Aβ1-40 / Aβ1-42) or plasma AD biomarker (P-tau181 or P-tau217) negativity may be used instead of performing an Aβ PET scan at Screening at the Principal Investigator's (PI's) discretion.
• The following medications are allowed, but must be stable for 2 months prior to Baseline:
• FDA-approved AD medications
• FDA-approved Parkinson's Disease (PD) medications

You may not qualify if:

• Has a reliable study partner who agrees to accompany the participant to visits, and spends at least 5 hours per week with the participant.
• Signed and dated written informed consent obtained from the participant/legally authorized representative (LAR) and the participant's study partner in accordance with local Institutional Review Board (IRB) regulations.
• Women of childbearing potential (WCBP) must agree to abstain from sex or use an adequate method of contraception for the duration of the screening period, the study drug treatment period, and for 28 days after the last dose of study drug.
• Males must agree to abstain from sex with WCBP or use an adequate method of contraception for the duration of the study drug treatment period and for 75 days after.
• For PSP-RS Only
• Meets 2017 consensus criteria for possible or probable progressive supranuclear palsy-Richardson syndrome (PSP-RS).
• For CBS Only
• Meets 2013 consensus criteria for possible or probable corticobasal degeneration (CBD), CBS subtype.
• Meets criteria for probable AD established by the National Institute on Aging and the Alzheimer's Association (NIA-AA).
• Any other medical condition other than PSP-RS or CBS that could account for cognitive or motor deficits (e.g., active seizure disorder, stroke, vascular dementia, substance abuse or alcoholism).
• History of a prominent and sustained response to levodopa therapy in the opinion of the PI.
• Presence of significant cardiovascular, hematologic, renal, or hepatic disease.
• Suicidal ideation per the Columbia-Suicide Severity Rating Scale (C-SSRS) that in the opinion of the PI would pose a safety risk or interfere with the appropriate interpretation of study data
• History of major psychiatric illness or untreated depression that in the opinion of the PI would pose a safety risk or interfere with the appropriate interpretation of study data.
• Neutrophil count \<1,500/mm3, platelets \<100,000/mm3, total bilirubin ≥1.5 x Upper Limit of Normal (ULN), alanine aminotransferase (ALT) ≥3 x ULN, aspartate aminotransferase (AST) ≥3 x ULN, or International Normalized Ratio (INR) \>1.2.

Sponsors & Collaborators

• Woolsey Pharmaceuticals: lead

Study Sites (1)

University of California Weill Institute for Neurosciences

San Francisco, California, 94158, United States

Location

MeSH Terms

Conditions

Supranuclear Palsy, Progressive; Corticobasal Degeneration

Interventions

fasudil

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Peter Ljubenkov, MD

  UCSF Weill Institute for Neurosciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label, single arm

Study Record Dates

• First Submitted: January 18, 2021
• First Posted: February 2, 2021
• Study Start: January 22, 2021
• Primary Completion: November 30, 2022
• Study Completion: November 30, 2023
• Last Updated: June 3, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)