- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04662931
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients With Vaso-occlusive Crises.
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab has been approved in India and other countries 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 is 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.
Study Overview
Detailed Description
Sickle cell disease (SCD) is a genetic blood disorder, caused by a mutation in the β-globin gene, which early on progresses into a systemic disease. Vaso-occlusion is a hallmark of SCD and can lead to serious acute and chronic complications.
Crizanlizumab has been approved in US, India and other countries 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 is 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.
The study is open label and single armed. 140 patients will be treated with crizanlizumab for about one year at a dose of 5 mg/kg in addition to receiving standard of care.
The primary objective is to assess frequency, severity and causality of serious adverse events (SAEs) during the treatment period. Secondary objective is to assess overall safety and tolerability of crizanlizumab.
Study Type
Enrollment (Actual)
Phase
- Phase 4
Contacts and Locations
Study Locations
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Guwahati, India, 781003
- Novartis Investigative Site
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Chhattisgarh
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Raipur, Chhattisgarh, India, 492099
- Novartis Investigative Site
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Kerala
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Kozhikode, Kerala, India, 673008
- Novartis Investigative Site
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Odisha
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Bhubaneswar, Odisha, India, 751003
- Novartis Investigative Site
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Telangana
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Hyderabad, Telangana, India, 500082
- Novartis Investigative Site
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Uttar Pradesh
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Lucknow, Uttar Pradesh, India, 226014
- Novartis Investigative Site
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West Bengal
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Kolkata, West Bengal, India, 700014
- Novartis Investigative Site
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Crizanlizumab
Participants will receive Crizanlizumab at a dose of 5.0 mg/kg.
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Crizanlizumab will be taken by IV infusion, at Week 1 Day 1, Week 3 Day 1 and all 4 weeks thereafter.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percentage of patients with at least one serious adverse event (SAE) with 2-sided 95% confidence interval (CI)
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Incidence of serious adverse events (SAEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with SAEs of grades >=3
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Severity of serious adverse events (SAEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with treatment-related SAEs of all grades
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Causality of serious adverse events (SAEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with treatment-related SAEs of grades >=3
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Causality of severe serious adverse events (SAEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percentage of patients with at least one treatment-emergent adverse event (AE)(new or worsening from baseline)
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Incidence of treatment-emergent adverse events (AEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with AEs of grades >=3
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Severity of adverse events (AEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with treatment-related AEs of all grades
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Causality of adverse events (AEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Percentage of patients with treatment-related AEs of grades >=3
Time Frame: During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Causality of severe adverse events (AEs)
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During treatment period - from Week 1 Day 1 (first dose) to safety follow up visit (105 days after Week 51 Day 1, or 105 days after last dose in case of early discontinuation)
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Collaborators and Investigators
Sponsor
Investigators
- Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CSEG101A2403
- 2020-003625-31 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data is currently available according to the process described on www.clinicalstudydatarequest.com
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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