Trial Outcomes & Findings for Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL) (NCT NCT04611880)

Last Updated: 2026-04-23

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

1 year

Results posted on

2026-04-23

Participant Flow

Participant milestones

Participant milestones
Measure	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
Overall Study STARTED	18
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

number is not different

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm Study n=18 Participants 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
Age, Categorical <=18 years	0 Participants n=60 Participants • number is not different
Age, Categorical Between 18 and 65 years	17 Participants n=60 Participants • number is not different
Age, Categorical >=65 years	1 Participants n=60 Participants • number is not different
Sex: Female, Male Female	10 Participants n=60 Participants • no discrepancies
Sex: Female, Male Male	8 Participants n=60 Participants • no discrepancies
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=60 Participants
Race (NIH/OMB) Asian	1 Participants n=60 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=60 Participants
Race (NIH/OMB) Black or African American	0 Participants n=60 Participants
Race (NIH/OMB) White	17 Participants n=60 Participants
Race (NIH/OMB) More than one race	0 Participants n=60 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=60 Participants

PRIMARY outcome

Timeframe: 1 year

Population: The white matter hyperintensity lesion volume (mL) was normalized to whole-brain volume (mL) and then log-transformed. The annualized percent change was calculated using a linear mixed-model.

Outcome measures

Outcome measures
Measure	RVCL-S Cohort n=11 Participants Study was a single arm of RVCL-S patients who had genetic confirmation of TREX1 mutation.
Change in the Percentage of White Matter Hyperintensity (WMH) Lesion Volume on FLAIR MRI in RVCL Patients	31.3 Percent (%) Interval 20.8 to 42.3

Adverse Events

Single Arm Study

Serious events: 2 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm Study n=18 participants at risk 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
General disorders Death	11.1% 2/18 • From enrollment for up to 4 years.

Other adverse events

Other adverse events
Measure	Single Arm Study n=18 participants at risk 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
Nervous system disorders Right hemiparesis	5.6% 1/18 • From enrollment for up to 4 years.
Nervous system disorders expressive aphasia	5.6% 1/18 • From enrollment for up to 4 years.
Blood and lymphatic system disorders Leukopenia	5.6% 1/18 • From enrollment for up to 4 years.

Additional Information

Andria Ford, MD

Washington University School of Medicine

Phone: 314-362-7382

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place