Rho Kinase (ROCK) Inhibitor in Tauopathies - 1 (ROCKIT-1)
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|ClinicalTrials.gov Identifier: NCT04734379|
Recruitment Status : Active, not recruiting
First Posted : February 2, 2021
Last Update Posted : June 3, 2022
|Condition or disease||Intervention/treatment||Phase|
|Progressive Supranuclear Palsy Corticobasal Syndrome||Drug: Fasudil||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open label, single arm|
|Masking:||None (Open Label)|
|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.|
|Actual Study Start Date :||January 22, 2021|
|Estimated Primary Completion Date :||November 30, 2022|
|Estimated Study Completion Date :||November 30, 2023|
Oral fasudil 180 mg/day
Oral fasudil 180 mg/day
- 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.
- Phosphorylated tau [ Time Frame: 48 weeks ]Number of participants with changes in concentrations of cerebrospinal fluid (CSF) and plasma phosphorylated tau
- Biomarkers of neurodegeneration [ Time Frame: 48 weeks ]Number of participants with changes in biomarkers of neurodegeneration, including neurofilament light chain (NfL), and total tau fragment levels.
- Imaging biomarkers of neurodegeneration [ Time Frame: 48 weeks ]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)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04734379
|United States, California|
|University of California Weill Institute for Neurosciences|
|San Francisco, California, United States, 94158|
|Principal Investigator:||Peter Ljubenkov, MD||UCSF Weill Institute for Neurosciences|