Progressive Supranuclear PalsyPhase 2ROCKIT-1NCT04734379
Rho Kinase (ROCK) Inhibitor in Tauopathies - 1
A Phase 2 study testing Fasudil in people with Progressive Supranuclear Palsy and Corticobasal Syndrome, run by Woolsey Pharmaceuticals at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 35 to 80 years
- Conditions the study is for
- Progressive Supranuclear Palsy, Corticobasal Syndrome
- What is being tested?
- Fasudil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jan 2021to 30 Nov 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
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- FDA-approved AD medications
- FDA-approved Parkinson's Disease (PD) medications
- Other prescription medications are allowed as long as the dose is stable for 30 days prior to Baseline. (Note Exclusion Criteria 17 and 18.)
- 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.
Who cannot join
- 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.
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- 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.
- Serum creatinine >1.3 mg/dL.
- Evidence of any clinically significant findings on screening or baseline evaluations which, in the opinion of the PI would pose a safety risk or interfere with appropriate interpretation of study data.
- Current or recent history (within four weeks prior to Screening) of a clinically significant bacterial, fungal, or mycobacterial infection.
- Current clinically significant viral infection.
- Major surgery within four weeks prior to Screening.
- Any contraindication for MRI or unable to tolerate MRI scan at Screening.
- Any contraindication to or unable to tolerate lumbar puncture at Screening, including use of anticoagulant medications such as warfarin. Daily administration of aspirin up to 81mg is not a contraindication, as long as the dose is stable for 30 days prior to Screening.
- Participants who, in the opinion of the PI, are unable or unlikely to comply with the dosing schedule or study evaluations.
- Treatment with another investigational drug within 30 days or 5 half-lives of drug before Baseline, whichever is longer. Treatment with investigational drugs other than fasudil while on study will not be allowed.
- Treatment with systemic corticosteroids within 30 days or 5 half-lives of drug before Baseline, whichever is longer.
- On more than one of the following drug classes: long-acting nitrates, beta-blockers, or calcium channel blockers.
- Known hypersensitivity to the inactive ingredients in the study drug (fasudil).
- Known to be pregnant or lactating; or positive pregnancy test at Screening or Baseline (Day 1) for WCBP.
- Cancer within 5 years of Screening, except for basal cell carcinoma.
- History of serum or plasma progranulin level less than one standard deviation below the normal participant mean for the laboratory performing the assay.
- History or evidence at Screening of known disease-associated mutations in GRN, TBK1, C9ORF72, TARBP, CHMPB2, or VCP genes (FTLD causative gene mutations not associated with underlying tau pathology).
- Blood pressure < 90/60.
- Evidence of orthostatic hypotension.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California Weill Institute for Neurosciences
San Francisco, California, 94158
About this study
Official title: 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.
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
Read the detailed description
After consent, participants will undergo screening evaluations, which may occur over the course of up to 6 weeks. Subjects who meet inclusion/exclusion criteria will be enrolled into the study and complete baseline evaluations. Dosing with study drug will begin on Day 1 and continue for 48 weeks. Participants will return to the clinic at Week 1 (7 ± 2 days after the first study drug administration) and at Weeks 12, 24, 36, and 48 for study evaluations, and at Week 52 for post-treatment follow-up evaluations. Plasma biomarker collection will occur at baseline, and Weeks 12, 24, 36, and 48. Cerebrospinal fluid (CSF) Biomarker collection will occur at screening, Week 24 and Week 48. Brain magnetic resonance imaging (MRI) will occur at screening, and Weeks 24 and 48. Safety labs will be collected at each study visit as well as during Week 4.
Adverse events (AEs) will be assessed at all visits and subjects will be contacted one day after the start of treatment (that is, one day after Visit 1), and monthly thereafter including at each visit. Subject will also be contacted one day after Visit 6/last day of dosing with study drug for subjects who discontinue early.
Subjects/caregivers will be queried for study drug compliance one day after the start of treatment (that is, one day after Visit 1), and monthly thereafter.
After completion of the 48-week primary portion of the study, subjects will be given the option to continue open-label treatment for up to an additional 12 months.
- Being tested
- Fasudil
- Medical subject headings
- Supranuclear Palsy, Progressive, Corticobasal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FasudilDrug
Oral fasudil 180 mg/day
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Oral fasudil 180 mg/day Receives: Drug: Fasudil |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 48 weeks
Secondary outcomes (3)
Phosphorylated tau
Number of participants with changes in concentrations of cerebrospinal fluid (CSF) and plasma phosphorylated tau
Measured: 48 weeks
Biomarkers of neurodegeneration
Number of participants with changes in biomarkers of neurodegeneration, including neurofilament light chain (NfL), and total tau fragment levels.
Measured: 48 weeks
Imaging biomarkers of neurodegeneration
Number of participants with changes in imaging biomarkers of neurodegeneration, including changes in brain volume (whole brain, ventricles, hippocampus, frontal operculum, pre-central gyri, midbrain, pons and superior cerebellar peduncle) and white matter tract integrity (aslant tract, superior longitudinal fasciculus, and superior cerebellar peduncle) as determined by T1-weighted volumetric magnetic resonance imaging (MRI), diffusion tensor imaging (DTI)
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2021
- Study start22 Jan 2021
- Primary completion30 Nov 2022 (estimated)
- Study completion30 Nov 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Supranuclear PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label, single arm
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Woolsey Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter Ljubenkov, MD, Principal investigator, UCSF Weill Institute for Neurosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04734379
- Sponsor's protocol ID
- WP-0512-002
- Acronym
- ROCKIT-1
- Status last verified
- May 2022
- First submitted
- 18 Jan 2021
- Last updated
- 3 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT04734379
- Acronym / sponsor ID
- ROCKIT-1 / WP-0512-002
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Woolsey PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 22 Jan 2021 (actual)
- Primary completion
- 30 Nov 2022 (estimated)
- Study completion
- 30 Nov 2023 (estimated)
- First posted
- 2 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
After consent, participants will undergo screening evaluations, which may occur over the course of up to 6 weeks. Subjects who meet inclusion/exclusion criteria will be enrolled into the study and complete baseline evaluations. Dosing with study drug will begin on Day 1 and continue for 48 weeks. Participants will return to the clinic at Week 1 (7 ± 2 days after the first study drug administration) and at Weeks 12, 24, 36, and 48 for study evaluations, and at Week 52 for post-treatment follow-up evaluations. Plasma biomarker collection will occur at baseline, and Weeks 12, 24, 36, and 48. Cerebrospinal fluid (CSF) Biomarker collection will occur at screening, Week 24 and Week 48. Brain magnetic resonance imaging (MRI) will occur at screening, and Weeks 24 and 48. Safety labs will be collected at each study visit as well as during Week 4.
Adverse events (AEs) will be assessed at all visits and subjects will be contacted one day after the start of treatment (that is, one day after Visit 1), and monthly thereafter including at each visit. Subject will also be contacted one day after Visit 6/last day of dosing with study drug for subjects who discontinue early.
Subjects/caregivers will be queried for study drug compliance one day after the start of treatment (that is, one day after Visit 1), and monthly thereafter.
After completion of the 48-week primary portion of the study, subjects will be given the option to continue open-label treatment for up to an additional 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label, single arm
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: FasudilOral fasudil 180 mg/day |
Who may qualify
- Ages
- 35 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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.
Key exclusion criteria (25 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FasudilOral fasudil 180 mg/day | Drug | Treatment |
Primary outcome
Adverse events
Time frame: 48 weeks
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.
3 secondary outcomes
Phosphorylated tau
Time frame: 48 weeks
Biomarkers of neurodegeneration
Time frame: 48 weeks
Imaging biomarkers of neurodegeneration
Time frame: 48 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Ljubenkov, MD principal investigator
UCSF Weill Institute for Neurosciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fasudil in people with Progressive Supranuclear Palsy and Corticobasal Syndrome, run by Woolsey Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2022.