Phase 2
N=18
Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL)
RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy
Bottom Line
View on ClinicalTrials.gov: NCT04611880 ↗Enrolled (actual)
18
Serious AEs
11.1%
Results posted
Apr 2026
Primary outcome: Primary: Change in the Percentage of White Matter Hyperintensity (WMH) Lesion Volume on FLAIR MRI in RVCL Patients — 31.3 Percent (%)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Crizanlizumab (Drug)
- Age
- Adult, Older Adult · 25+ yrs
- Sex
- All
- Sponsor
- Washington University School of Medicine
- Primary completion
- Apr 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in the Percentage of White Matter Hyperintensity (WMH) Lesion Volume on FLAIR MRI in RVCL Patients |
31.3 | — |
Summary
This is a Phase 2 trial that will test the efficacy and safety of crizanlizumab for the treatment of retinal vasculopathy with cerebral leukoencephalopathy (RVCL), a very rare and uniformly fatal genetic condition that affects the microvasculature, especially of the brain and eye. There currently is no treatment for RVCL. A maximum of 20 patients will be enrolled.
Eligibility Criteria
Inclusion Criteria
- A diagnosis of RVCL with confirmation by genetic test
- At least 25 years of age with imaging evidence of brain or eye disease at the time of study registration
- Normal hematologic function defined as: White blood cell count (WBC) > 4x109/L, Absolute neutrophil count (ANC) >1.5x109/L and Platelets > 100x109/L
- 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
- 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
- Acute bacterial, fungal, or viral infection
- Known HIV, untreated latent tuberculosis (TB), or active hepatitis B or C infection or zoster
- 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.
- Known hypersensitivity to one or more of the study agents
- Currently receiving or has received any investigational drugs within the 14 days prior to the first dose of study drug
- Liver function tests (LFTs) higher than 3x the upper limit of normal within the last 30 days
- Treatment with other monoclonal antibody medications within the last 30 days
- 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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Data sourced from ClinicalTrials.gov (NCT04611880). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.