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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: MDS (Myelodysplastic Syndrome); ALL (Acute B-Lymphoblastic Leukemia); AML (Acute Myeloid Leukemia)

Detailed Description

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 (Estimated): 51

Contacts and Locations

• Study Contact: Name: Michaela Malchow; Phone Number: +49 15117151746; Email: Michaela.Malchow@miltenyi.com

Study Locations

• United States
  • New York
    • New York, New York, United States, 10021
      • Memorial Sloan Kettering Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Disease: Patients with AML, MDS, and ALL

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	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	two years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Relapse-free survival will be evaluated at 2-years post-HCT.	Acute GVHD, grades II-IV Chronic GVHD Hematologic recovery (time to neutrophil engraftment and time to platelet engraftment) Rate of primary graft failure Non-relapse mortality Relapse Relapse-free survival	two years

Collaborators and Investigators

• Sponsor: Miltenyi Biomedicine GmbH

Study record dates

Study Major Dates

• Study Start (Estimated): July 31, 2026
• Primary Completion (Estimated): September 20, 2026
• Study Completion (Estimated): September 30, 2026

Study Registration Dates

• First Submitted: July 16, 2026
• First Submitted That Met QC Criteria: July 16, 2026
• First Posted (Actual): July 21, 2026

Study Record Updates

• Last Update Posted (Actual): July 21, 2026
• Last Update Submitted That Met QC Criteria: July 16, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Anemia; Leukemia; Myelodysplastic Syndromes; Anemia, Refractory; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts
• Other Study ID Numbers: PROJXXXX

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No