Crizanlizumab for RVCL
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests crizanlizumab, a medication that prevents blood cells from sticking to vessel walls, in patients with a rare and fatal condition called RVCL. The goal is to see if it can reduce brain and eye damage by preventing small blood vessel blockages. Crizanlizumab is a monoclonal antibody developed by Novartis Pharmaceuticals for the prevention of vaso-occlusive crises in patients with sickle cell disease.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications, such as other monoclonal antibody medications and anticoagulants, at least 30 days before starting the study drug. If you are on any investigational drugs, you must stop them 14 days before the trial begins.
How is the drug crizanlizumab unique for treating RVCL?
Crizanlizumab is unique because it is a monoclonal antibody that targets P-selectin, a molecule involved in blood vessel blockages, and is administered through an intravenous infusion. While it is primarily used to prevent pain crises in sickle cell disease, its mechanism of action may offer novel benefits for conditions like RVCL, where there are no standard treatments.12345
Who Is on the Research Team?
Andria Ford, MD
Principal Investigator
Washington University School of Medicine
Are You a Good Fit for This Trial?
This trial is for adults over 25 with a rare, fatal genetic condition called RVCL confirmed by genetic testing. Participants must not be pregnant or breastfeeding and agree to use contraception. They should have normal blood counts, no recent infections, HIV, hepatitis B/C, or TB, and haven't taken certain medications recently.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive intravenous infusions of crizanlizumab 5 mg/kg at weeks 1 and 3, and then every 4 weeks for a total of 24 months
Monitoring
Standard-of-care serial MRI and eye disease monitoring, along with blood work monitoring (CBC/CMP) 1 month after initiation and every 3 months thereafter
Follow-up
Participants are monitored for safety and effectiveness after treatment completion
What Are the Treatments Tested in This Trial?
Interventions
- Crizanlizumab
Crizanlizumab is already approved in United States for the following indications:
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Who Is Running the Clinical Trial?
Washington University School of Medicine
Lead Sponsor