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.
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 140
- 试验地点
- 1
- 主要终点
- Number of Participants With Treatment Emergent Serious Adverse Events (SAEs)
研究概览
简要总结
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.
详细描述
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.
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.
The study was open label and single armed. 140 patients were treated with crizanlizumab for approximately one year at a dose of 5 mg/kg in addition to receiving standard of care.
The primary objective was to assess frequency, severity and causality of serious adverse events (SAEs) during the treatment period. Secondary objective was to assess overall safety and tolerability of crizanlizumab.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
Crizanlizumab
Participants received Crizanlizumab at a dose of 5.0 mg/kg, and standard of care.
干预措施: crizanlizumab (Drug)
结局指标
主要结局
Number of Participants With Treatment Emergent Serious Adverse Events (SAEs)
时间窗: Up to 15 months
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.
次要结局
- 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)