PRAISE-IR Versus CIBMTR Controls

N/ANot Yet RecruitingNCT07717164

Miltenyi Biomedicine GmbH51 enrolled

Overview

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.

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

Study Type

OBSERVATIONAL

Enrollment

Conditions

ALL (Acute B-Lymphoblastic Leukemia)AML (Acute Myeloid Leukemia)MDS (Myelodysplastic Syndrome)

Eligibility

Sex: ALLMin age: 1 YearMax age: 66 Years

Medical Language ↔ Plain English

Inclusion Criteria: 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) Exclusion Criteria: 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

Locations (1)

Memorial Sloan Kettering Cancer center

New York, New York, United States

Outcomes

Primary Outcomes

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.

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).

Time frame: two years

Secondary Outcomes

Acute GVHD, grades II-IV

Acute Graft-versus-Host Disease (GVHD), grades II-IV

Time frame: two years

Chronic GVHD

Chronic Graft-versus-Host Disease (GVHD)

Time frame: two years

Hematologic recovery (time to neutrophil engraftment and time to platelet engraftment)

Time frame: two years

Rate of primary graft failure

Time frame: two years

Non-relapse mortality

Time frame: two years

Central Contacts

Michaela Malchow

CONTACT

+49 15117151746 Michaela.Malchow@miltenyi.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.