NCT04802057终止1 期
An Open-label, Long-term, Safety and Tolerability Study of SAR445088 in Participants With Cold Agglutinin Disease Previously Treated With SAR445088 or Never Treated With SAR445088
Bioverativ, a Sanofi company5 个研究点 分布在 5 个国家目标入组 9 人开始时间: 2021年3月22日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 9
- 试验地点
- 5
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAE)
研究概览
简要总结
Primary Objective:
To assess the long-term safety and tolerability in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088
Secondary Objective:
To assess, in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088:
- The long-term effect of SAR445088 on complement mediated hemolysis
- The long-term pharmacodynamics (PD) effect of SAR445088 relating to complement inhibition
- The long-term pharmacokinetic (PK) profile of SAR445088
- The long-term immunogenicity of SAR445088
详细描述
The screening period for this study is up to 6 weeks. The treatment period for this study will continue for 2 years after last participant entered Part 2 (either having switched from Part 1 or as a SAR445088-naïve participant), or until SAR445088 development is discontinued, whichever comes first.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adult patients ≥18 years of age with CAD who were previously treated with SAR445088 and met criteria the below criteria for entry into Part 1:
- •met the eligibility criteria of a previous study evaluating SAR445088;
- •successfully enrolled and completed dosing in a previous study evaluating SAR445088;
- •successfully completed end of study procedures in a previous study evaluating SAR445088; and
- •per Investigator judgement, had a favorable benefit-to-risk profile after receiving SAR
- •OR were never treated with SAR445088 before entering Part 2, and met the below criteria to establish CAD diagnosis:
- •chronic hemolysis;
- •polyspecific direct antiglobulin test (DAT) positive status;
- •monospecific DAT strongly positive for C3d;
- •cold agglutinin [CAg] titer ≥64 at 4°C;
- •IgG DAT ≤1+;
- •hemoglobin level ≤10 g/dL;
- •elevated bilirubin not attributable to liver disease;
- •Documented vaccinations against encapsulated bacterial pathogens given within five years of enrollment and at least 14 days prior to dosing (vaccinations have to be initiated at least 14 days prior to dosing and completed before Week 5 of Part 2).
- •Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.
- •Having given written informed consent prior to undertaking any study-related procedure.
排除标准
- •Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high-grade hematologic malignancy, or known solid organ tumor.
- •Clinically relevant infection within 1 month of enrollment.
- •Clinical diagnosis of systemic lupus erythematosus (SLE).
- •Treatment with anti-CD20 monotherapy within 3 months or anti-CD20 combination therapies within 6 months prior to screening.
- •Concurrent treatment with systemic immunosuppressive agents targeting B- or T-cell function and/or cytotoxic agents within 3 months prior to screening. Concurrent treatment with other systemic immunosuppressants within 5.5 half-lives of the drug prior to screening.
- •Any specific complement system inhibitor other than SAR445088 (eg, eculizumab) within 3 months prior to screening.
- •Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within 3 months prior to screening.
- •History of hypersensitivity to SAR445088 or any of its components.
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
SAR445088
Experimental
Repeat dose of SAR445088
干预措施: SAR445088 (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAE)
时间窗: Day 1 or Day 1-IV to end of study, approximately 6 years
The number of participants experiencing TEAEs will be reported for the overall safety population.
次要结局
- Mean change from baseline in total bilirubin over time(Day 1 or Day 1-IV to end of study, approximately 6 years)
- Mean change from baseline in hemoglobin over time(Day 1 or Day 1-IV to end of study, approximately 6 years)
- Mean change from baseline in lactate dehydrogenase over time(Day 1 or Day 1-IV to end of study, approximately 6 years)
- Mean change from baseline in reticulocyte count over time(Day 1 or Day 1-IV to end of study, approximately 6 years)
- Total Complement Factor C4 Levels(Day 1 or Day 1-IV to end of study, approximately 6 years)
- PK parameter: Cmax(Day 1 or Day 1-IV to Week 48-IV)
- Complement System Classical Pathway Levels as Measured by WIESLAB Assay(Day 1 or Day 1-IV to Week 48-IV)
- Complement System Alternative Pathway Levels as Measured by WIESLAB Assay(Day 1 to Week 24)
- Number of participants with anti-SAR445088 antibodies(Day 1 or Day 1-IV to end of study, approximately 6 years)
- Mean change in CH50 over time(Day 1 or Day 1-IV to end of study, approximately 6 years)
- PK Parameter: AUC(Day 1 or Day 1-IV to Week 48-IV)
研究者
研究点 (5)
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