An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 12
- 主要终点
- To assess the time from transplant to neutrophil engraftment
研究概览
简要总结
Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
详细描述
Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.
Omidubicel is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood consisting of mature myeloid and lymphoid cells as follows:
- Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)), containing a minimum of 8.0 × 10^8 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 × 10^7 CD34+ cells, and
- the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)), containing a minimum of 4.0 × 10^8 total viable cells with a minimum of 2.4 × 10^7 CD3+ cells
Omidubicel utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of hematopoietic progenitor cells (HPC) expanded in ex vivo cultures.
The overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must be at least 12 years of age
- •Applicable disease criteria
- •Patients must have one or two partially HLA-matched CBUs
- •Back-up stem cell source
- •Sufficient physiological reserves
- •Females of childbearing potential agree to use appropriate method of contraception
- •Signed written informed consent
排除标准
- •Extensive bone marrow fibrosis
- •Donor specific anti-HLA antibodies
- •Pregnancy
- •Medically unsuitable for transplant
研究组 & 干预措施
omidubicel
Received omidubicel
干预措施: omidubicel (Biological)
结局指标
主要结局
To assess the time from transplant to neutrophil engraftment
时间窗: by day 42 post-transplant inclusive
Time From Transplant to Neutrophil Engraftment
时间窗: by day 42 post-transplant inclusive
Neutrophil engraftment was defined as achieving an absolute neutrophil count (ANC) greater than or equal to 0.5 x 10\^9/L on 3 consecutive measurements by Day 42 post-transplant inclusive. The first day of the three measurements was designated the day of neutrophil engraftment.
Cumulative Incidence of Neutrophil Engraftment
时间窗: by day 42 post-transplant inclusive
Death, second transplant, and relapse were competing risks at the time they occur if they occur prior to neutrophil engraftment, and no transplant was a competing risk at Day 0. If the patient failed to achieve neutrophil engraftment, they were considered to have a competing risk at Day 43.
次要结局
- Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL(By Day 42 and Day 180 post-transplant)
- Time to Platelet Engraftment >20,000 Cells/uL(By Day 730 post-transplant)
- Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL(By Day 42 and Day 180 post-transplant)
- Time to Platelet Engraftment >50,000 Cells/uL(By Day 730 post-transplant)
- Non-relapse Mortality(By Day 180, Day 365 and Day 730 post-transplant)
- Overall Survival (OS)(By Day 180, Day 365 and Day 730 post-transplant)
- Disease Free Survival (DFS)(By Day 365 and Day 730 post-transplant)
- Donor Chimerism(By day 100 and Day 730 post-transplant)
- Secondary Graft Failure (SGF)(By Day 730 post-transplant)
- Disease Relapse(By Day 365 and Day 730 post-transplant)
- Cumulative Incidence of Acute GvHD Grade II-IV(By Day 100 post-transplant)
- Cumulative Incidence of aGvHD Grade III-IV(By Day 100 post-transplant)
- Cumulative Incidence of Chronic GvHD(By Day 180 and Day 730 post-transplant)
- Chronic GvHD-free Relapse-free Survival (cGRFS)(By Day 365 and Day 730 post-transplant)
- GvHD-free Relapse-free Survival (GRFS)(By Day 365 and Day 730 post-transplant)