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NCT04611880

Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL)

Active, enrolled Phase 2 Last updated 26 June 2025
What this trial tests

Phase 2 trial testing Crizanlizumab in RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy in 20 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
25 January 2021
Primary endpoint
2 April 2025
31 December 2025

Quick facts

Lead sponsorWashington University School of Medicine
PhasePhase 2
StatusActive, enrolled
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment20
Start date25 January 2021
Primary completion2 April 2025
Estimated completion31 December 2025
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Washington University School of Medicine

Who can join

Adults 25 to 75, any sex, with RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. P- and E- selectin in venous thrombosis and non-venous pathologies.
    Purdy M, Obi A, Myers D, Wakefield T. · · 2022 · cited 60× · PMID 35243742 · DOI 10.1111/jth.15689
  2. Retinal Vasculopathy with Cerebral Leukoencephalopathy and Systemic manifestations (RVCL-S): An update on basic science and clinical perspectives.
    Wilms AE, de Boer I, Terwindt GM. · · 2022 · cited 15× · PMID 36324396 · DOI 10.1016/j.cccb.2022.100046
  3. Targeting the P-selectin/PSGL-1 pathway: discovery of disease-modifying therapeutics for disorders of thromboinflammation.
    Escopy S, Chaikof EL. · · 2024 · cited 8× · PMID 40766814 · DOI 10.1016/j.bvth.2024.100015
  4. Crizanlizumab for retinal vasculopathy with cerebral leukoencephalopathy in a phase II clinical study.
    Wang WX, Spiegelman D, Rao PK, Rhee RL, et al · · 2024 · cited 6× · PMID 38950286 · DOI 10.1172/jci180916
  5. Retinal Vasculopathy With Cerebral Leukoencephalopathy and Systemic Manifestations: Critical Role of Retina Specialists.
    Houghton OM, Carter J, Dhamija R. · · 2023 · cited 2× · PMID 37006667 · DOI 10.1177/24741264221129095
  6. Mechanistic and Therapeutic Implications of Protein and Lipid Sialylation in Human Diseases.
    Zhong X, D'Antona AM, Rouse JC. · · 2024 · PMID 39596031 · DOI 10.3390/ijms252211962

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Other trials of Crizanlizumab

Trials testing the same drug.

Other Washington University School of Medicine trials

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Data sources for this page

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