A Phase 2 interventional study of Crizanlizumab in RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy, sponsored by Washington University School of Medicine. Completed at 2 sites in United States. Open to participants aged 25 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-04-23.
Sponsored by Washington University School of Medicine · Phase 2, Interventional, and Treatment
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.
Retinal vasculopathy with cerebral leukoencephalopathy (RVCL) is a very rare and uniformly fatal genetic condition that affects the microvasculature, especially of the brain and eye. Symptoms begin in adulthood (usually in the mid-30s to early 40s) and include loss of vision, mini-strokes, and dementia. Other patients have suffered from microvascular disease involving the kidneys, osteonecrosis, and gut ischemia. Some of these features of microvascular occlusive disease resemble ischemic events that occur during sickle cell disease. Currently, there is no effective treatment for RVCL.
The goal of this study is to test the efficacy of RVCL patients treated with crizanlizumab, a humanized monoclonal anti-P-selectin antibody that prevents leukocyte adhesion to the vascular endothelium, thereby limiting risk of microvascular occlusion. P-selectin is mobilized to the surface of activated vascular endothelial cells and promotes leukocyte adhesion to the blood vessel wall. The Miner laboratory has preliminarily observed a correlation with levels of soluble P-selectin and the number of brain lesions in patients with RVCL. Since leukocyte adhesion to the vascular endothelium promotes microvascular occlusion, we will determine if crizanlizumab will help to limit ischemia and brain lesions in patients with RVCL. This may lead to the development of fewer ischemic brain and eye lesions.
Up to 20 RVCL patients will receive intravenous infusions of crizanlizumab 5 mg/kg at weeks 1 and 3. Thereafter, patients will receive crizanlizumab 5 mg/kg every 28 days for a total of 24 total months. Monitoring will include standard-of-care serial MRI as well as standard-of-care eye disease monitoring at pre-defined intervals. High-risk medication monitoring will include blood work monitoring (CBC/CMP) 1 month after initiation of treatment and every 3 months thereafter. Standard-of-care assessments will be performed including radiological and physical examinations as well as eye imaging and examinations. Patients will be followed for at least 2 years after completion of crizanlizumab administration.
Washington University School of Medicine is the lead sponsor of 1,765 studies on the registry; 271 are open to participants now.
Of its 324 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.
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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)
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Exclusion Criteria:
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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Single arm study: crizanlizumab will be supplied in single use vials containing 10 mL at a concentration of 10 mg/mL for administration by IV infusion. Each patient will receive one dose of crizanlizumab on day 1 of Week 1, day 1 of Week 3, day 1 of Week 7, and then day 1 of every 4-week cycle. On infusion day, the pharmacist or designated personnel will prepare individual doses of crizanlizumab for subjects on a milligram per kilogram basis (5 mg/kg) in a 100 mL infusion bag in accordance with the Pharmacy Manual. Crizanlizumab will be administered over 30 minutes by IV infusion
Drug: Crizanlizumab
Drug: crizanlizumab is a humanized monoclonal anti-P-selectin antibody that prevents leukocyte adhesion to the vascular endothelium, thereby limiting risk of microvascular occlusion. It is administered intravenously.
Also known as: ADAKVEO
Change in the Percentage of White Matter Hyperintensity (WMH) Lesion Volume on FLAIR MRI in RVCL Patients
At each study time-point, the white matter hyperintensity lesion volume (mL) was normalized to whole-brain volume (mL) and then log-transformed, from which the annualized percent change in the log-transformed normalized WMH volume was calculated.
Time frame: 1 year
| Milestone | Single Arm Study |
|---|---|
| Started | 18 |
| Completed | 11 |
| Not completed | 7 |
At each study time-point, the white matter hyperintensity lesion volume (mL) was normalized to whole-brain volume (mL) and then log-transformed, from which the annualized percent change in the log-transformed normalized WMH volume was calculated.
| Percent (%) | RVCL-S Cohort |
|---|---|
| Change in the Percentage of White Matter Hyperintensity (WMH) Lesion Volume on FLAIR MRI in RVCL Patients | 31.3 (20.8 to 42.3) |
Collected over From enrollment for up to 4 years.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Single Arm Study | 2/18 (11.1%) | 2/18 (11.1%) | 3/18 (16.7%) |
| Event | Single Arm Study |
|---|---|
| DeathGeneral disorders | 2/18 |
| Event | Single Arm Study |
|---|---|
| Right hemiparesisNervous system disorders | 1/18 |
| expressive aphasiaNervous system disorders | 1/18 |
| LeukopeniaBlood and lymphatic system disorders | 1/18 |
| Age, Categorical(Participants) | Single Arm Study |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 17 |
| >=65 years | 1 |
| Sex: Female, Male(Participants) | Single Arm Study |
|---|---|
| Female | 10 |
| Male | 8 |
| Race (NIH/OMB)(Participants) | Single Arm Study |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 17 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — Data will be shared among the principle investigator and the other investigators
Supporting information: Study protocol, Icf
This study is completed, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Washington University School of Medicine