Comparison of the PRAISE -IR ("Phase 2 Study of Personalized r-ATG (Rabbit Anti-thymocyte Globulin) Dosing to Improve Survival Through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex-vivo CD34-Selected Allogeneic-HCT (Hematopoietic Cell Transplantation) ", NCT04872595) Study Population With CIBMTR Controls
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 51
- 试验地点
- 1
- 主要终点
- To Compare overall survival (OS) between patients from PRAISE-IR study (Arm A) to CIBMTR control patients who have received a CD34 selected graft and standard dose ATG (Arm B).
研究概览
简要总结
The rationale to conduct this RWE study is to provide data to support the safety of CD34 selected grafts versus T replete PBSC transplants to address concerns about increased TRM in CD34 selected graft recipients.
详细描述
Comparison of the PRAISE -IR ("Phase 2 Study of Personalized r-ATG Dosing to Improve Survival Through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex-vivo CD34-Selected Allogeneic-HCT", NCT04872595) study population with CIBMTR controls
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 1 Year 至 66 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Arm A: Model-based ATG dosing CD34-selection (PRAISE-IR population)
- •Patients who participated in the PRAISE-IR single center phase II study
- •Patient age at transplant: ≥ 1 year and < 66 years
- •HLA 8/8 MRD or MUD
- •Conditioning intensity: myeloablative
- •Conditioning regimens: Model-based ATG with TBI/Thiotepa/Cyclophospamide (TBI/Thio/Cy) or Busulfan/Melphalan/Fludarabine (Bu/Mel/Flu).
- •Morphologic complete remission at the time of alloHCT Arm B: Standard ATG dosing CD34-selection (CIBMTR population)
- •First AlloHCT in the US between 2021-2023
- •Patient age at transplant: ≥ 1 year and < 66 years
- •Disease: Patients with AML, MDS, and ALL
- •HLA 8/8 MRD or MUD
- •Peripheral blood stem cell allograft
- •Conditioning intensity: myeloablative conditioning
- •Conditioning regimens: standard dose ATG (2.5 mg/kg/day given on Day -4 and Day -3)
- •GVHD prophylaxis: ex vivo CD34 selection
- •Morphologic complete remission at the time of alloHCT Arm B*:Standard ATG dosing CD34-selection (BMT CTN 1301 population)
- •Patients in BMT CTN 1301, who received the CD34-selected graft
- •Patient age at transplant: ≥ 1 year and < 66 years
- •Disease:Patients with AML, MDS, and ALL
- •HLA 8/8 MRD or MUD
- •Peripheral blood stem cell allograft
- •Conditioning intensity: myeloablative conditioning
- •Conditoning regmens: TBI/Thiotepa/Cyclophospamide (TBI/Thio/Cy) or Busulfan/Melphalan/Fludarabine (Bu/Mel/Flu) and standard dose ATG (2.5 mg/kg/day given on Day -4 and Day -3)
- •GVHD prophylaxis: ex vivo CD34 selection
- •Morphologic complete remission at the time of alloHCT Arm C: Control CIBMTR population
- •First AlloHCT in the US between 2021-2023
- •Patient age at transplant: ≥ 1 year and < 66 years
- •Disease:Patients with AML, MDS, and ALL
- •HLA 8/8 MRD or MUD
- •Peripheral blood stem cell
- •Conditioning intensity: myeloablative conditioning
- •Conditioning regimens: Busulfan/Cyclophosphamide (Bu/Cy), Busulfan/Fludaranbine (Bu/Flu), Cyclophosphamide/TBI (Cy/TBI), TBI/Etopsoside
- •GVHD prophyalxis: / CNI or PTCy-based
- •CNI-based: CNI (tacrolimus or ciclosporin) plus MTX
- •PTCy-based: Cyclophosphamide on day +3 and +4 (50 mg/kg/d) combined with CNI and mycophenolate mofetil (MMF)
排除标准
- •Patients will be entered into this trial only if they meet none of the following criteria:
- •Arm A: Model-based ATG dosing CD34-selection (PRAISE-IR population)
- •HLA <8/8 MRD or MUD Arm B: Standard ATG dosing CD34-selection (CIBMTR population)
- •Patients who participated in the PRAISE-IR study
- •Patients who did not sign consent for research
- •Patient transplanted at a center that does not meet CIBMTR data quality standards
- •Use of CNI-(Tac/MTX) or PTCy-based GVHD prophylaxis Arm B*:Standard ATG dosing CD34-selection (BMT CTN 1301 population)
- •Patients who did not sign consent for research
- •Patient transplanted at a center that does not meet CIBMTR data quality standards
- •Use of CNI- or PTCy-based GVHD prophylaxis Arm C: Control CIBMTR population
- •Patients who did not sign consent for research
- •Patient transplanted at a center that does not meet CIBMTR data quality standards
- •Use of ATG and/or alemtuzumab
- •Patients who received PTCy with sirolimus (and not a CNI)
- •Use of ex vivo CD34 selection
研究组 & 干预措施
Arm A: Model-based ATG dosing CD34-selection (PRAISE-IR population)
CliniMACS CD34 Reagent System
Arm B: Standard ATG dosing CD34-selection (CIBMTR population)
Intervention: CliniMACS CD34 Reagent System
Arm B*:Standard ATG dosing CD34-selection (BMT CTN 1301 population)
CliniMACS CD34 Reagent System
Arm C: Control CIBMTR population
结局指标
主要结局
To Compare overall survival (OS) between patients from PRAISE-IR study (Arm A) to CIBMTR control patients who have received a CD34 selected graft and standard dose ATG (Arm B).
时间窗: two years
To Compare overall survival (OS) between patients from PRAISE-IR study (Arm A) to CIBMTR control patients who have received a CD34 selected graft and standard dose ATG (Arm B). The endpoint should be to demonstrate that OS is better in Arm A compared to Arm B. To Compare OS between patients from PRAISE-IR study (Arm A) to CIBMTR control patients who have received standard of care (SoC) alloHCT without CD34 selection with PTCY or Tac/Methotrexate for GVHD prophylaxis (Arm C). The endpoint should be to demonstrate that OS in Arm A is comparable (not worse) than in Arm C
Comparison of OS between PRAISE-IR study participants (Arm A) with CIBMTR control participants who have received a CD34 selected graft and standard dose ATG (Arm B) with the goal of demonstrating superiority of Arm A.
时间窗: two years
Comparison of overall survival (OS) between PRAISE-IR study participants (Arm A) with CIBMTR control participants who have received a CD34 selected graft and standard dose ATG (Arm B). Note: If the number of participants meeting the criteria for Arm B is insufficient for 1:1 matching, Arm A could be compared with the CD34 selection group from the BMT CTN1301 study (Arm B\*). Comparison of OS between PRAISE-IR study participants (Arm A) with CIBMTR control patients who have received standard of care (SoC) allogeneic hematopoietic stem cell transplantation (alloHCT) without CD34 selection with post transplantation cyclophosphamide (PTCy) and Calcineurin inhibitor (CNI)/Methotrexate (MTX) for GVHD prophylaxis (Arm C).
次要结局
- Relapse-free survival will be evaluated at 2-years post-HCT.(two years)
- Acute GVHD, grades II-IV(two years)
- Chronic GVHD(two years)
- Hematologic recovery (time to neutrophil engraftment and time to platelet engraftment)(two years)
- Rate of primary graft failure(two years)
- Non-relapse mortality(two years)
- Relapse(two years)
- Relapse-free survival(two years)