Crizanlizumab for RVCL

Recruiting in Palo Alto (17 mi)

+1 other location

Overseen byAndria Ford, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Washington University School of Medicine

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

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.

Eligibility Criteria

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

I have been diagnosed with RVCL through a genetic test.

I am 25 or older and have brain or eye disease confirmed by imaging.

Your blood counts for white cells, neutrophils, and platelets are within normal range.

+2 more

Exclusion Criteria

You are allergic to any of the medications being used in the study.

Your liver function tests showed results more than three times the normal range in the last 30 days.

I have taken blood thinners like clopidogrel or coumadin in the last 30 days.

+5 more

Participant Groups

The study is evaluating the effectiveness and safety of Crizanlizumab in treating RVCL. This Phase 2 trial aims to enroll up to 20 patients who suffer from this condition that severely affects brain and eye blood vessels.

1Treatment groups

Experimental Treatment

Group I: Single Arm StudyExperimental Treatment1 Intervention

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

Crizanlizumab is already approved in United States for the following indications:

🇺🇸 Approved in United States as Adakveo for: