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

NCT03938454 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CSEG101AUS05
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 13

Brief Summary

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

Conditions

Priapism

Keywords

Priapism; sickle cell disease; SCD; crizanlizumab; P-selectin; SEG101; monoclonal antibody; prolonged erection; painful erection

Outcome Measures

Primary Outcomes (1)

• Percent Change in Priapic Events From Baseline 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.

  Baseline up to 26 weeks

Secondary Outcomes (4)

• Annualized Rate of Priapic Events

  Baseline up to 26 and 52 weeks

• Number of Acute Priapic Events From Baseline to 26 and 52 Weeks

  Baseline up to 26 and 52 weeks

• Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)

  Baseline up to 26 and 52 weeks

• Annualized Rate of Complicated VOCs

  Baseline up to 26 and 52 weeks

Study Arms (1)

Crizanlizumab 5 mg/kg

EXPERIMENTAL

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.

Drug: Crizanlizumab

Interventions

Crizanlizumab 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.

Also known as: SEG101

Crizanlizumab 5 mg/kg

Eligibility Criteria

• Age: 12 Years - 100 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (13)

University Of Alabama

Birmingham, Alabama, 35294, United States

Location

University of Connecticut Health Center

Farmington, Connecticut, 06030, United States

Location

Childrens National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33021, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30303, United States

Location

LSU Medical Center

Shreveport, Louisiana, 71130, United States

Location

Childrens Hosp Boston Dept of Hematology

Boston, Massachusetts, 02115, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Brody School of Medicine

Greenville, North Carolina, 27834, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213-2548, United States

Location

Prisma Health Upstate

Greenville, South Carolina, 29615, United States

Location

University of Texas Medical School

Houston, Texas, 77030, United States

Location

Related Links

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

MeSH Terms

Conditions

Priapism; Anemia, Sickle Cell

Interventions

crizanlizumab

Condition Hierarchy (Ancestors)

Penile Diseases; Genital Diseases, Male; Genital Diseases; Urogenital Diseases; Male Urogenital Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

This was a single-arm trial in a disease where clinical history has significant variability.

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

1 862 778 8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2019
• First Posted: May 6, 2019
• Study Start: October 16, 2019
• Primary Completion: March 28, 2023
• Study Completion: November 29, 2023
• Last Updated: January 13, 2026
• Results First Posted: March 10, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

US (13)