An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients

NCT04662931 | Completed Phase 4 Results

• 1 other identifier: CSEG101A2403
• Study Type: interventional
• Target: 140
• Locations: 1 country
• Sites: 7

Brief Summary

Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older. The purpose of this local Phase IV study was to evaluate the safety of crizanlizumab specifically in Indian patients with SCD aged 16 years or older with a history of VOC leading to healthcare visit.

Conditions

Sickle Cell Disease (SCD)

Keywords

Sickle cell disease; Sickle cell disorder; SCD; Sickle cell anemia; Hemoglobin SC disease; Sickling disorder due to hemoglobin S; Vaso-occlusive crisis; VOC; P-selectin; Crizanlizumab; SEG101

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Emergent Serious Adverse Events (SAEs)

  Number of participants with treatment emergent SAEs and SAEs grade \>=3. Adverse events were assessed and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5. CTCAE ranges severity from Grade 1 through 5 being Grade 1 the lowest severity grade.

  Up to 15 months

Secondary Outcomes (2)

• Number of Participants With Treatment Emergent Adverse Events (AEs)

  Up to 15 months

• Number of Participants With Adverse Events of Special Interest (AESI)

  Up to 15 months

Study Arms (1)

Crizanlizumab

EXPERIMENTAL

Participants received Crizanlizumab at a dose of 5.0 mg/kg, and standard of care.

Drug: crizanlizumab

Interventions

crizanlizumab DRUG

Crizanlizumab 5.0 mg/kg i.v. initial dose on Week 1 Day 1, second dose on Week 3 Day 1. Subsequently, Day 1 of every 4 weeks until Week 51, in addition of standard of care.

Also known as: SEG101

Crizanlizumab

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent
• Male or female participant aged 16 years and older
• Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid chromatography (HPLC). All SCD genotypes are eligible.
• History of VOC leading to healthcare visit prior to screening visit
• Participants must meet the following central laboratory values at the screening visit:
• Absolute Neutrophil Count ≥1.0 x 109/L Platelet count ≥75 x 109/L Hemoglobin: for adults (Hb) ≥4.0 g/dL and for adolescents (Hb) ≥5.5 g/dL Glomerular filtration rate ≥ 45 mL/min/1.73 m2 using CKD-EPI formula Direct (conjugated) bilirubin \< 2.0 x ULN Alanine Aminotransferase (ALT) \< 3.0 x ULN
• ECOG performance status ≤2 for adults and Karnofsky Performance Scale ≥ 50% for adolescents.

You may not qualify if:

• Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug. History of severe hypersensitivity reaction to other monoclonal antibodies which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
• Participant has received crizanlizumab and/or other P-selectin inhibitor prior to the study or plans to receive it during the duration of the study.
• Concurrent severe and/or uncontrolled medical conditions which, in the opinion of the Investigator, could cause unacceptable safety risks or compromise participation in the study.
• Any condition which, in the opinion of the investigator, is likely to interfere with the successful collection of the measurements required for the study.
• Participant has documented immunogenicity to a prior biological drug.
• Participants who are on active treatment with Voxelotor, other investigational drug or other monoclonal antibody, or intend to initiate the same during the course of the trial.
• Pregnant females or females who have given birth within the past 90 days prior screening or who are breastfeeding.
• Women of childbearing potential unless using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment
• Significant bleeding disorder
• Active HIV infection
• Active Hepatitis B infection
• Positive test for Hepatitis C RNA
• Malignant disease
• Active infection or immune deficiency

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (7)

Novartis Investigative Site

Guwahati, Assam, 781003, India

Location

Novartis Investigative Site

Raipur, Chhattisgarh, 492099, India

Location

Novartis Investigative Site

Kozhikode, Kerala, 673008, India

Location

Novartis Investigative Site

Bhubaneswar, Odisha, 751003, India

Location

Novartis Investigative Site

Hyderabad, Telangana, 500082, India

Location

Novartis Investigative Site

Lucknow, Uttar Pradesh, 226014, India

Location

Novartis Investigative Site

Kolkata, West Bengal, 700014, India

Location

Related Links

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

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobin SC Disease; Vaso-Occlusive Crises

Interventions

crizanlizumab

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

novartis.email@novartis.com

+ 1 862 778 8300

Study Officials

• Novartis Pharmaceuticals Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2020
• First Posted: December 10, 2020
• Study Start: July 14, 2021
• Primary Completion: February 14, 2024
• Study Completion: February 14, 2024
• Last Updated: September 26, 2025
• Results First Posted: January 8, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

IN (7)