An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFNγ) Monoclonal Antibody, and to Assess Its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients With Primary Hemophagocytic Lymphohistiocytosis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 35
- 试验地点
- 27
- 主要终点
- Overall Response at Week 8 or End of Treatment (if Earlier)
研究概览
简要总结
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term outcomes and quality of life assessments. Emapalumab can be administered as the first-line therapy to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the available standard of care.
Emapalumab is to be administered until the start of conditioning for hematopoietic stem cell transplantation (HSCT), with an anticipated duration ranging from a minimum of 4 weeks to approximately 12 weeks and not exceeding 6 months.
After treatment completion, patients will continue in the study for long-term follow-up until 1 year after either HSCT or last emapalumab infusion (if HSCT is not performed).
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Primary HLH patients with active disease.
- •Treatment naïve patients or patients having already received HLH conventional therapy, but having not responded, not achieved a satisfactory response or worsened, or reactivated, or are unable to tolerate current standard of care.
- •Informed consent signed by the patient or by the patient's legally authorized representative.
- •Received guidance on contraception.
排除标准
- •Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
- •Active mycobacteria, Histoplasma capsulatum, Shigella, Salmonella, Campylobacter or Leishmania infections.
- •Evidence of latent tuberculosis.
- •Presence of malignancy.
- •Concomitant disease or malformation severely affecting cardiovascular, pulmonary, central nervous system (CNS), liver, or renal function, that in the opinion of the Investigator may significantly affect the likelihood to respond to treatment and/or the assessment of emapalumab safety and/or efficacy.
- •History of hypersensitivity or allergy to any component of the study regimen.
- •Receipt of a BCG vaccine within 12 weeks prior to Screening.
- •Receipt of a live or attenuated-live (other than BCG) vaccine within 6 weeks prior to Screening.
- •Pregnant or lactating female patients.
研究组 & 干预措施
Emapalumab
干预措施: Emapalumab (Drug)
结局指标
主要结局
Overall Response at Week 8 or End of Treatment (if Earlier)
时间窗: Up to Week 8
The overall response rate (ORR) of patients achieving either Complete or Partial Response or HLH Improvement, at Week 8 or EOT (whichever occurs earlier).
次要结局
- Overall Survival to HSCT(From start of treatment to HSCT or from start of treatment until 1 year after EOT for patient who did not undergo HSCT)
- Incidence, Severity, Causality and Outcomes of AEs (Serious and Non-serious)(Up to 18 months)
- Quality of Life Assessed Through PedsQL™, Pediatric Quality of Life Inventory™(Up to 6 months)
- Evolution of Laboratory Parameters Change From Baseline to EOT/Week 8(To Week 8 or End of treatment if before Week 8.)
- Event-free Survival(Up to 1 year post HSCT)
- Time to Response(Up to 18 months)
- Number of Patients Reducing Glucocorticoids by 50% or More of the Baseline Dose During Emapalumab Treatment(Up to 6 months)
- Overall Survival at End of Study(Up to 18 months)
- Overall Survival for Patients Receiving HSCT(Up to 1 year post HSCT)
- Overall Response at Start of Conditioning(Up to 6 months)
- Duration of Response(Up to 18 months)
- Number of Patients Proceeding to HSCT(Up to 18 months)
- Quality of Life Assessed Through Behavioral, Affective and Somatic Experiences Scales (BASES)(Up to 6 months)
- Number of Patients Who Discontinued Emapalumab Treatment(Up to 6 months)