Using retinal health to assess brain blood vessel conditions

Retinal Vasoreactivity is a Marker for Cerebral Small Vessel Disease Progression

Phase1; Phase2 Interventional Mayo Clinic · NCT04753970

This study is testing if changes in the eye can help track brain blood vessel problems and whether a medication called cilostazol can slow down these issues in people with certain types of brain diseases.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment100 (estimated)
Ages18 Years and up
SexAll
SponsorMayo Clinic Academic / other
Locations1 site (Jacksonville, Florida)
Trial IDNCT04753970 on ClinicalTrials.gov

What this trial studies

This study investigates whether retinal vasoreactivity can serve as a biomarker for the progression of cerebral small vessel disease (SVD) and evaluates the safety and efficacy of cilostazol, an antiplatelet agent, in slowing SVD progression. Participants will include individuals diagnosed with CADASIL, sporadic white matter disease, and lobar cerebral microbleeding, along with age-matched healthy controls. The study will involve retinal scans, brain imaging, cognitive assessments, and blood samples at baseline and after 12 months. Patients with sporadic white matter disease will be randomized to receive cilostazol or no treatment, with follow-up assessments to monitor disease progression and cognitive changes.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older diagnosed with CADASIL, sporadic white matter disease, or lobar cerebral microbleeding.

Not a fit: Patients under 18, pregnant or breastfeeding women, and those unable to tolerate MRI or follow commands may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for slowing the progression of cerebral small vessel disease.

How similar studies have performed: While the approach of using retinal biomarkers for cerebrovascular health is promising, it remains largely novel and untested in this specific context.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Age ≥18 yo.
* Diagnosis of CADASIL, sporadic WMD or lobar CMB and age-matched healthy controls (eg. patient's spouse or unrelated friends without SVD)

Exclusion Criteria:

* Age\<18yo
* Pregnant
* Breast feeding
* Unable to follow commands
* Unable to tolerate MRI

Where this trial is running

Jacksonville, Florida

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Cerebral Small Vessel DiseasesCerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and LeukoencephalopathyCerebral MicrobleedingSporadic White Matter Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.