NCT05744063已完成4 期
An Open Label, Single Arm, Multi-Centre, Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of Primary Hemophagocytic Lymphohistiocytosis in Treatment Experienced Chinese Patients
Swedish Orphan Biovitrum6 个研究点 分布在 1 个国家目标入组 13 人开始时间: 2023年2月3日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 13
- 试验地点
- 6
- 主要终点
- Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event
研究概览
简要总结
The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.
详细描述
This is an open-label, multi center, single arm, post-authorization study aiming to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with confirmed or suspected primary hemophagocytic lymphohistiocytosis (pHLH). The main objectives of the study are to collect safety and efficacy data on emapalumab in treatment experienced Chinese pHLH patients
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female HLH patients of any age.
- •Patients diagnosed with confirmed or suspected pHLH, based on; a molecular diagnosis or familial history consistent with pHLH or fulfilment of HLH-2004 diagnostic criteria, i.e., five out of eight of the criteria below:
- •Splenomegaly
- •Cytopenias affecting 2 of 3 lineages in the peripheral blood (hemoglobin <90 g/L; platelets <100 x 109/L; neutrophils <1 x 109/L)
- •Hypertriglyceridemia (fasting triglycerides ≥3 mmol/L or ≥265 mg/dL) and/or hypofibrinogenemia (≤1.5 g/L)
- •Hemophagocytosis in bone marrow, spleen, or lymph nodes, with no evidence of malignancy.
- •Low or absent NK-cell activity
- •Ferritin ≥500 μg/L
- •Soluble CD25 (sCD25; i.e., soluble IL-2 receptor) ≥2400 U/mL
- •Presence of active HLH disease as assessed by the investigator.
- •Patients must fulfil one of the following criteria as assessed by the investigator:
- •Having not responded to previous conventional treatment of HLH
- •Having not achieved a satisfactory response to previous conventional treatment of HLH or worsened
- •Having reactivated HLH
- •Showing intolerance to previous conventional treatment of HLH At the time of enrollment, eligible patients might still be receiving treatment (induction or maintenance) or might have already discontinued it.
- •Expectation of survival beyond 1 week as judged by the investigator.
- •Patient has expectation of proceeding to HSCT
- •Informed consent signed by the patient (as required by local law), or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.
- •Willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug, if female and of childbearing potential.
排除标准
- •Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
- •Active mycobacteria, Histoplasma capsulatum, Salmonella, or Leishmania infections.
- •Evidence of latent tuberculosis.
- •Presence of malignancy.
- •Existence of any severe co-morbidity or any other medical condition which, in the opinion of the investigator, makes the patient unsuitable for the treatment
- •History of hypersensitivity or allergy to any component of the study regimen (e.g., polysorbate).
- •Receipt of a Bacillus Calmette-Guérin (BCG) vaccine within 12 weeks prior to Screening.
- •Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to Screening.
- •Pregnant or lactating female patients.
- •Enrollment in another concurrent clinical interventional study, or intake of an IMP, within three months prior to inclusion in this study
- •Any condition or circumstance that in the opinion of the Investigator may make the patient unlikely to complete the study or comply with study procedures or requirements.
研究组 & 干预措施
emapalumab
Experimental
emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
干预措施: Emapalumab-Lzsg 5 MG/ML [Gamifant] (Drug)
结局指标
主要结局
Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event
时间窗: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Number of participants permanently discontinuation of study drug due to emapalumab-related adverse event as judged by Investigator, until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
次要结局
- Overall Response(End of treatment or week 8 (whichever occurs earlier))
- Time to First Overall Response(End of treatment, likely within 6 months from first dose)
- Cumulative Duration of Response(End of treatment, likely within 6 months from first dose)
- Ability to Reduce Glucocorticoids by 50% or More(End of treatment, likely within 6 months from first dose)
- Investigator Assessed Response(End of treatment)
- Survival(End of study (1 year))
研究者
研究点 (6)
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