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Hope for RVCL: experimental drug tested in tiny trial

NCT ID NCT04611880

Disease control Sponsor: Washington University School of Medicine Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tested the drug crizanlizumab in 18 people with RVCL, a rare and fatal genetic condition that damages small blood vessels in the brain and eyes. There is currently no treatment for RVCL. The study measured changes in brain lesions on MRI scans to see if the drug could slow the disease.

What this could mean

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

Active substance
crizanlizumab (Adakveo)
What this could lead to
If it works, this could point toward a treatment for RVCL, a rare and fatal disease with no current options.
What could go wrong
This is a very small early-phase trial (18 people) with no control group, so results may not be conclusive. The drug may not slow the disease or could have side effects.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

18 people

The number who actually took part.

Started

Jan 2021

Finished

Dec 2025

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

25 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: 1. A diagnosis of RVCL with confirmation by genetic test 2. At least 25 years of age with imaging evidence of brain or eye disease at the time of study registration 3. Normal hematologic function defined as: White blood cell count (WBC) \> 4x109/L, Absolute neutrophil count (ANC) \>1.5x109/L and Platelets \> 100x109/L 4. Females of childbearing potential (FCBP) must agree to refrain from becoming pregnant while on study drug and for 3 months after discontinuation from study drug, and must agree to use adequate contraception including hormonal contraception, (i.e. birth control pills, etc), barrier method contraception (i.e. condoms), or abstinence during that time-frame 5. Able to understand and willing to sign an Internal Review Board (IRB)-approved written informed consent document (or that of legally authorized representative, if applicable) \- Exclusion Criteria: 1. Acute bacterial, fungal, or viral infection 2. Known HIV, untreated latent tuberculosis (TB), or active hepatitis B or C infection or zoster 3. Pregnant and/or breastfeeding. Negative serum pregnancy test required prior to starting study treatment. For females of child-bearing potential (FCBP), a negative urine pregnancy test is required before each infusion. 4. Known hypersensitivity to one or more of the study agents 5. Currently receiving or has received any investigational drugs within the 14 days prior to the first dose of study drug 6. Liver function tests (LFTs) higher than 3x the upper limit of normal within the last 30 days 7. Treatment with other monoclonal antibody medications within the last 30 days 8. Treatment with various forms of anticoagulation within last 30 days, including but not limited to clopidogrel or coumadin or direct thrombin inhibitors \-

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Conditions

The condition(s) this trial relates to.

retinal vasculopathy with cerebral leukoencephalopathy and systemic manifestations

As listed by the trial registrant

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

RVCL - retinal vasculopathy cerebral leukoencephalopathy

Contacts and locations

Locations

  • Andria Ford

    St Louis, Missouri, 63110, United States

  • Perelman School of Medicine; University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States