A Phase 2, Open-Label Study to Evaluate the Efficacy and Safety of MGTA-145 in Combination With Plerixafor for the Mobilization of Hematopoietic Stem Cells in Patients With Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 试验地点
- 3
- 主要终点
- Apheresis Collection Yield
研究概览
简要总结
This research study is designed to investigate a new potential medicine for mobilizing stem cells and apheresis collection in patients with Sickle Cell Disease. MGTA-145, the new potential medicine, will be given with plerixafor.
详细描述
This Phase 2, multicenter, open-label study will be conducted in 2 parts (Parts A and B). Part A is intended to characterize the efficacy, safety, PK and PD of a single dose of MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD. Part B is designed to characterize the efficacy, safety, PK and PD of 2 consecutive days of dosing with MGTA-145 and plerixafor for HSC mobilization and apheresis collection in patients with SCD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 35 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must be ≥18 to ≤35 years of age.
- •Subject must weigh ≥30 kg.
- •Subject must have a diagnosis of Sickle Cell Disease.
排除标准
- •Subject must not have had a vaso-occlusive event (VOE) requiring a visit to a healthcare facility within 30 days of screening.
- •Subject must not have undergone or attempted and failed previous hematopoietic stem cell (HSC) collection.
- •Subject must not have had a prior autologous or allogeneic transplantation, inclusive of gene therapy.
- •Male subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
- •Female subject must not be pregnant or breastfeeding. If sexually active, female subject must be willing or able to use a highly effective method of contraception for 3 months during and after treatment.
研究组 & 干预措施
Part A: Single Day Dosing/Apheresis
Single dose of MGTA-145 in combination with plerixafor followed by apheresis
干预措施: MGTA-145 (Biological)
Part A: Single Day Dosing/Apheresis
Single dose of MGTA-145 in combination with plerixafor followed by apheresis
干预措施: Plerixafor (Drug)
Part B: 2-Day Dosing/Apheresis
MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
干预措施: MGTA-145 (Biological)
Part B: 2-Day Dosing/Apheresis
MGTA-145 in combination with plerixafor followed by apheresis on two consecutive days
干预措施: Plerixafor (Drug)
结局指标
主要结局
Apheresis Collection Yield
时间窗: Up to 2 days
Determination of the yield of CD34+ cells after either one or two consecutive days of MGTA-145 and plerixafor mobilization followed by apheresis.
Assess Number of Participants With Treatment Emergent Adverse Events Leading to Study Drug Discontinuation Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
时间窗: Up to 30 days
Assess number of participants with treatment emergent adverse events leading to study drug discontinuation based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Assess the Number of Participants With Treatment Emergent >/= Grade 3 Clinical Laboratory Abnormalities Based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
时间窗: Up to 11 days
Assess the number of participants with treatment emergent \>/= Grade 3 clinical laboratory abnormalities based on the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Vital Signs - Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
时间窗: Up to 11 days
Vital Signs - Number of participants with clinically significant changes from baseline in vital signs
Laboratory Assessment - Number of Participants With Clinically Significant Changes From Baseline in Hematology and Clinical Chemistry Laboratory Parameters.
时间窗: Up to 11 days
Laboratory Assessment - Number of participants with clinically significant changes from baseline in hematology and clinical chemistry laboratory parameters.
次要结局
- Mobilization Effects of Single-day and Two-day Dosing With MGTA-145 and Plerixafor in Peripheral Blood in Patients With SCD(Up to 2 days)
- Investigate Plasma Concentrations of MGTA-145 Per Timepoint of Collection (Pharmacokinetics)(Up to 2 days)
- Assess Presence of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])(Up to 11 days)
- Assess Titers of MGTA-145 Anti-Drug Antibodies (ADA) in Plasma Samples (Using Electrochemiluminescent Immunoassay [ECLIA])(Up to 11 days)