A Phase 1/2 interventional study of Cilostazol in Cerebral Small Vessel Diseases, Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy and Cerebral Microbleeding, sponsored by Mayo Clinic. Recruiting at 1 site in United States. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-08-03.
Sponsored by Mayo Clinic · Phase 1/2, Interventional, and Prevention
Cerebral small vessel disease (SVD), present in 80-94% of adults over age 65 years, increases the risk of stroke by 2-fold, and dementia by 2.3-fold. There is currently no treatment to slow SVD progression. This study aims to test whether impaired cerebral and retinal vasoreactivity may serve as biomarker for SVD progression, and to evaluate the safety and efficacy of cilostazol (antiplatelet agent with vasodilatory and anti-inflammatory properties) for the treatment of SVD.
This is a prospective, observational nested pilot randomized controlled study to discover retinal biomarkers that would predict cerebral small vessel disease progression, and evaluate the safety/efficacy of cilostazols in slowing SVD progression. Twenty CADASIL, 40 sWMD, 20 lobar CMB, and 20 age-matched healthy controls from the Mayo Clinic Florida Familial Cerebrovascular Disease Registry and neurology clinic will be recruited. All participants will undergo OCTA retinal scan, MRI-BOLD brain scan, cognitive battery evaluation, and blood sample at baseline and a 12-month follow-up visit. Key outcome measures are: RVR, CVR, cognition, WMH volume, and CMB volume. The 40 patients diagnosed in the course of routine clinical care with sWMD will be randomized in 1:1 ratio to receive cilostazol 100mg bid (or 50 mg bid if taking medications known to affect metabolism of cilostazol) or no cilostazol, and followed for WMD progression, and secondarily for changes in cognition, RVR and CVR. Flow diagram below outlines the study design. Note that in addition to what is shown in the trial flow diagram, patients will have telephone visits between baseline and 12 month clinic visits biweekly for 3 months and then monthly thereafter. These visits will consist of a survey for adverse events and at the 1-, 3-, 6- and 9-month telephone visits patients will also get a modified Rankin scale assessment, a Six-item screener (cognitive assessment) and a PHQ-2 (depression screen).
33 studies on the registry are indexed under CADASIL; 18 are open to participants now.
This study's planned enrollment of 100 is above the median of 60 across 10 interventional studies indexed under CADASIL.
Browse CADASIL studies →Mayo Clinic is the lead sponsor of 3,269 studies on the registry; 672 are open to participants now.
Of its 452 completed or terminated interventional studies of FDA-regulated products, 321 (71%) have results posted.
Compared within CADASIL, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Cerebral Small Vessel Diseases. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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Exclusion Criteria:
Cilostazol 100mg BID
Drug: Cilostazol
Cilostazol 100mg BID
white matter disease volume
change in total white matter disease volume
Time frame: 1 year
cognition
global Z-score and by cognitive domain
Time frame: 1 year
stroke
ischemic stroke or hemorrhagic stroke
Time frame: 1 year
cerebrovasoreactivity
change in blood oxygen level dependence (BOLD) per unit of end tidal PCO2 mmHg
Time frame: 1 year
retinal vasoreactivity
change in retinal vessel density pre/post CO2 challenge
Time frame: 1 year
Plan to share: No
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Studies of the drug tested here: Cilostazol