A Phase Ib, Open-Label, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Efmarodocokin Alfa in Combination With Standard of Care in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 8
- 主要终点
- Change from Baseline in Respiratory Rate Over Time
研究概览
简要总结
This is a Phase Ib, open-label, multicenter, dose-escalation study to evaluate the safety, tolerability, and pharmacokinetics of Efmarodocokin Alfa and to make a preliminary assessment of activity of Efmarodocokin Alfa in combination with standard-of-care (SOC) in the prevention of acute graft-versus-host disease (aGVHD) in participants undergoing allogeneic hematopoietic stem cell transplantation (HSCT).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Prevention
- 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
- •Eligible for hematopoietic stem cell transplantation (HSCT)
- •Donor meeting human leukocyte antigen (HLA) matching criteria of HLA-matched related or HLA-matched unrelated (HLA-A, HLA-B, HLA-C, and HLA-DRB1, eight out of eight) from either peripheral blood or bone marrow stem cells and meeting donor-eligibility criteria as outlined by the U.S. Food and Drug Administration (FDA) in 21 CFR 1271 (including screening for Zika and SARS-CoV-2 exposure or infection)
- •Planned HLA (HLA-A, HLA-B, HLA-C, and HLA-DRB1)-matched (eight out of eight) related or planned HLA-matched (eight out of eight) unrelated HSCT, from either peripheral blood or bone marrow stem cells, for patients with acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL) in first complete remission (per institutional criteria) or patients with intermediate or high-risk myelodysplastic syndrome (MDS)
- •Planned myeloablative conditioning regimen per institutional guidelines
- •Planned aGvHD prophylaxis consisting of tacrolimus and methotrexate; in cases of tacrolimus intolerance, cyclosporine or sirolimus may be used as a substitute
排除标准
- •Prior receipt of autologous or allogeneic HSCT
- •Diagnosis of myelofibrosis or myelodysplastic/myeloproliferative overlap syndrome
- •Treatment with investigational biologic or non-biologic therapy within 5 drug elimination half-lives (or within 90 days or 30 days, respectively, if half-life is unknown) prior to initiation of study drug
- •Positive hepatitis B virus (HBV) or hepatitis C virus (HCV) serologies
- •History of Grade >1 cervical intraepithelial neoplasia
- •A marked baseline prolongation of QT/QTc interval
- •Risk factors for torsades de pointes
- •Pregnant or breastfeeding
- •Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
研究组 & 干预措施
Cohort A: Efmarodocokin Alfa Dosage Level 1
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 1 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
干预措施: Efmarodocokin Alfa (Drug)
Cohort B: Efmarodocokin Alfa Dosage Level 2
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 2 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
干预措施: Efmarodocokin Alfa (Drug)
Cohort C: Efmarodocokin Alfa Dosage Level 3
Participants undergoing allogeneic hematopoietic stem cell transplantation will receive Efmarodocokin Alfa dosage level 3 in combination with standard of care prophylaxis treatment for acute graft versus-host disease (aGVHD), consisting of tacrolimus plus methotrexate per institutional practices.
干预措施: Efmarodocokin Alfa (Drug)
结局指标
主要结局
Change from Baseline in Respiratory Rate Over Time
时间窗: From Baseline up to 139 days
Number of Participants with Adverse Events by Severity, According to National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 (NCI-CTCAE v5.0)
时间窗: From Baseline up to 365 days
Change from Baseline in Diastolic Blood Pressure Over Time
时间窗: From Baseline up to 139 days
Change from Baseline in Body Temperature Over Time
时间窗: From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Hematology Tests
时间窗: From Baseline up to 139 days
Number of Participants with Laboratory Abnormalities in Blood Chemistry Tests
时间窗: From Baseline up to 139 days
Change from Baseline in Oxygen Saturation Over Time
时间窗: From Baseline up to 139 days
Change from Baseline in Pulse Rate Over Time
时间窗: From Baseline up to 139 days
Change from Baseline in Systolic Blood Pressure Over Time
时间窗: From Baseline up to 139 days
次要结局
- Serum Concentration of Efmarodocokin Alfa at Specified Timepoints(At predefined timepoints from Baseline until Day 139)
- Number of Participants with Anti-Drug Antibodies (ADAs) at Baseline and During the Study(At predefined timepoints from Baseline until Day 139)