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

Who is this study for? Patients with Retinal Vasculopathy Cerebral Leukoencephalopathy

What treatments are being studied? Crizanlizumab

Status: Active_not_recruiting

Location: See all (2) locations...

Intervention Type: Drug

Study Type: Interventional

Study Phase: Phase 2

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

Participation Requirements

Sex: All

Minimum Age: 25

Maximum Age: 75

Healthy Volunteers: f

View:

• 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)

Locations

United States

Missouri

Andria Ford

Saint Louis

Pennsylvania

Perelman School of Medicine; University of Pennsylvania

Philadelphia

Time Frame

Start Date: 2021-01-25

Completion Date: 2027-08-25

Participants

Target number of participants: 20

Treatments

Other: Single Arm Study

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

Authors

Andria Ford, Jonathan Miner

Related Therapeutic Areas

CACH Syndrome

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

Similar Clinical Trials

An Open-Label, Proof of Concept Study of Vorinostat for the Treatment of Moderate-to-Severe Crohn's Disease and Maintenance Therapy With Ustekinumab

An Open-Label, Proof of Concept Study of Vorinostat for the Treatment of Moderate-to-Severe Crohn's Disease and Maintenance Therapy With Ustekinumab

A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Darvadstrocel in the Treatment of Complex Perianal Fistula in Pediatric Subjects With Crohn's Disease Over a Period of 24 Weeks and an Extended Follow-up Period for a Total of up to 52 Weeks

A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Darvadstrocel in the Treatment of Complex Perianal Fistula in Pediatric Subjects With Crohn's Disease Over a Period of 24 Weeks and an Extended Follow-up Period for a Total of up to 52 Weeks

Phase II Trial of Enzalutamide and M9241 in PET Positive Recurrent Prostate Cancer (pprPC) Without Testosterone Lowering Therapy

Phase II Trial of Enzalutamide and M9241 in PET Positive Recurrent Prostate Cancer (pprPC) Without Testosterone Lowering Therapy