A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 58
- 试验地点
- 16
- 主要终点
- Number of Participants With Adverse Event (AE)
研究概览
简要总结
International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).
详细描述
The aim of this study is to monitor the long-term safety profile of emapalumab in participants who have previously received at least one dose of emapalumab, including survival time after the administration of emapalumab.
Moreover, the elimination profile of emapalumab and the immunogenicity will also be assessed.
Furthermore, safety, tolerability, efficacy, and pharmacokinetic (PK) profile of emapalumab will be closely monitored in the event that some participants, upon request of the treating physician, will receive emapalumab treatment in the follow-up study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Having received at least one dose of emapalumab.
- •Having signed the Informed Consent by the participant or the participant's legal representative(s), as applicable, with the assent of participant who are legally capable of providing it.
排除标准
- 未提供
研究组 & 干预措施
Enrolled-04 Cohort
Participants enrolled in Study NI-0501-04 (NCT01818492) will be invited to participate for long-term follow-up for 1 year either after haematopoietic stem cell transplantation (HSCT) or after the last administration of emapalumab.
In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT.
Participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT will be delayed for reasons unrelated to the administration of emapalumab, can continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05, NCT02069899) at the request of the investigator, providing a favorable benefit/risk assessment of treatment is established.
The dose and timing was either carried forward from the last administered emapalumab dose as part of the parent protocol or an adjusted dose was administered, if necessary.
干预措施: Emapalumab (Drug)
Enrolled-CU Cohort
In exceptional cases, at the spontaneous request of a treating physician, CU treatment will be granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who receive at least 1 dose of emapalumab under CU will be invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab.
Participants can continue treatment in the context of the current Study (NI-0501-05, NCT02069899) while stem cell donor search is ongoing, or if the investigator assesses that continuation of treatment is beneficial.
干预措施: Emapalumab (Drug)
结局指标
主要结局
Number of Participants With Adverse Event (AE)
时间窗: From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days)
Adverse events were defined as any undesirable experience occurring in a participant during the study, whether or not considered related to emapalumab.
次要结局
- Circulating Emapalumab Level (Enrolled-06 Cohort)(Baseline (first NI-0501-05 visit), Day 100, Month 6)
- Total Human Interferon Gamma Levels (Enrolled-04 Cohort)(First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, Day 100 post-transplant, 12 months post-transplant)
- Total Human Interferon Gamma Levels (Enrolled-06 Cohort)(Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study)
- Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort)(From HSCT up to 12 months)
- Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort)(From HSCT to 12 months)
- MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort)(Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study)
- Circulating Emapalumab Level (Enrolled-04 Cohort)(First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant)
- Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort)(From HSCT to 12 months)
- Number of Participants With Anti-drug Antibody(From enrolment up to 12 months post-transplant or last emapalumab infusion (maximum 639 days))
- Cumulative Duration of Response (Enrolled-04 Cohort)(From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days))
- Duration of First Response (Enrolled-06 Cohort)(From first date of response and first date of loss of response or death (maximum 416 days))
- Overall Survival (Enrolled-04 Cohort)(From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days))
- Overall Survival (Enrolled-06 Cohort)(From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days))