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

NCT ID: NCT03938454

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-16
• Study Completion Date: 2023-11-29

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.

Conditions

Priapism

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Crizanlizumab 5 mg/kg

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.

Group Type: EXPERIMENTAL

Crizanlizumab

Intervention Type: DRUG

Crizanlizumab is a concentrate for solution for infusion, IV use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab.

Interventions

Crizanlizumab

Crizanlizumab is a concentrate for solution for infusion, IV use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab.

Intervention Type: DRUG

Other Intervention Names

SEG101

Eligibility Criteria

Inclusion Criteria

• Male patients aged 12 years and above
• Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
• Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
• Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
• If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
• If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
• Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures

Exclusion Criteria

• Had penile prosthetic implants or shunts or any other surgical procedure on the penis performed within 12 months prior to consenting was not allowed
• Took drugs/medications that may induce priapism over the 14 weeks preceding study entry
• Received leuprolide acetate (Lupron) or any other gonadotropin releasing hormone receptor agonist agent within 3 months before pre-screening
• Had an erection lasting more than 12 hours over the 14 weeks preceding study entry
• Had an erection lasting more than 12 hours during the 12 weeks of the Screening period

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University Of Alabama

Birmingham, Alabama, United States

University of Connecticut Health Center

Farmington, Connecticut, United States

Childrens National Hospital

Washington D.C., 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

The 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

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=2419

A Plain Language Trial Summary is available on www.novctrd.com

https://www.novctrd.com/ctrdweb/ppatientsummary/ppatientsummaries?periodicPatientSummaryId=677

A Pediatric Plain Language Trial Summary is available on www.novctrd.com

Other Identifiers

CSEG101AUS05

Identifier Type: -

Identifier Source: org_study_id