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The eyes may hold a window to brain health: a trial tests a new marker and treatment

NCT ID NCT04753970

Disease control Sponsor: Mayo Clinic Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time

Summary

This study investigates whether changes in retinal blood vessels can serve as a marker for cerebral small vessel disease (SVD), a common condition in older adults that increases stroke and dementia risk. The trial includes people with SVD-related conditions like CADASIL, sporadic white matter disease, or lobar microbleeds, as well as healthy controls. Participants receive cilostazol, a drug with antiplatelet and vasodilatory effects, to see if it can slow disease progression. The study measures changes in white matter volume, cognition, and blood vessel reactivity in the brain and retina.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
cilostazol
What this could lead to
If successful, this could establish a simple eye test to track small vessel disease and point toward a treatment to slow its progression.
What could go wrong
This is an early-phase trial, so the drug may not prove effective or safe. The biomarker approach also needs validation before it can be used widely.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 100 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2021

Expected to finish

Jun 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

cerebral arteriopathy, autosomal dominant, with subcortical infarcts and leukoencephalopathy, type 1 Cerebral Small Vessel Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy Cerebral microbleeding Cerebral small vessel diseases Sporadic white matter disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Mayo Clinic Florida

    RECRUITING

    Jacksonville, Florida, 32224, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.