A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism

Phase 2

Completed

• Conditions: Priapism
• Interventions: Drug: Crizanlizumab

• Registration Number: NCT03938454
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.

Detailed Description

Before participating in this study, information to determine key eligibility criteria was collected as a part of a 14-week Pre-Screening period. The study included a 12-week Screening period and a 52-week (1 year) Treatment period. Eligible participants received crizanlizumab 5 mg/kg by intravenous infusion (IV). Study treatment was received at clinic visits on Week 1 Day 1, Week 3 Day 1, and then on Day 1 of every 4-week cycle. Efficacy assessments included evaluation of priapic and vaso-occlusive (VOC) events. Safety assessments included laboratory tests, electrocardiograms (ECGs), vital signs and physical examinations. Participants had a safety follow-up for up to 15 weeks after the last dose.

Study Timeline

• Study First Submitted: 2019-05-02
• Study First Submitted QC: 2019-05-02
• Study First Posted: 2019-05-06
• Study Start: 2019-10-16
• Primary Completion: 2023-03-28
• Disposition First Submitted: 2023-10-31
• Study Completion: 2023-11-29
• Results First Submitted: 2024-11-26
• Results First Submitted QC: 2025-02-18
• Results First Posted: 2025-03-10
• Disposition First Posted: 2025-03-10
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-10
• Last Update Posted: 2025-04-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 36

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Crizanlizumab 5 mg/kg	Crizanlizumab	Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.

Primary Outcome Measures

Name	Time	Method
Percent Change in Priapic Events From Baseline to 26 Weeks	Baseline up to 26 weeks	A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. Number of priapic events was summarized at Baseline (adjusted for 26 weeks) and by 26 weeks, and percent reduction from adjusted Baseline by 26 weeks was summarized.

Secondary Outcome Measures

Name	Time	Method
Annualized Rate of Priapic Events	Baseline up to 26 and 52 weeks	A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period.; The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.
Number of Acute Priapic Events From Baseline to 26 and 52 Weeks	Baseline up to 26 and 52 weeks	An acute priapic event was defined as an unwanted, painful erection that lasted more than 4 hours and required a visit to the emergency room.
Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)	Baseline up to 26 and 52 weeks	An uncomplicated VOC event was defined as an acute event of pain with no known cause for pain other than a VOC event; and requiring treatment with a parenteral or oral opioids or other parenteral analgesic; but was NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism. Events included both healthcare and self-reported events.; The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.
Annualized Rate of Complicated VOCs	Baseline up to 26 and 52 weeks	Complicated VOCs were defined as acute chest syndrome, hepatic sequestration, splenic sequestration, and acute priapism recorded by healthcare visit.; The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.

Trial Locations

Locations (13)

University Of Alabama; 🇺🇸 Birmingham, Alabama, United States

University of Connecticut Health Center; 🇺🇸 Farmington, Connecticut, United States

Childrens National Hospital; 🇺🇸 Washington, District of Columbia, United States

Foundation for Sickle Cell Disease Research; 🇺🇸 Hollywood, Florida, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

LSU Medical Center; 🇺🇸 Shreveport, Louisiana, United States

Childrens Hosp Boston Dept of Hematology; 🇺🇸 Boston, Massachusetts, United States

Montefiore Medical Center; 🇺🇸 Bronx, New York, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Brody School of Medicine; 🇺🇸 Greenville, North Carolina, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Prisma Health Upstate; 🇺🇸 Greenville, South Carolina, United States

University of Texas Medical School; 🇺🇸 Houston, Texas, United States