Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies (AB-CliniMACs)

UNRELATED AND PARTIALLY MATCHED RELATED DONOR PERIPHERAL STEM CELL TRANSPLANTATION WITH Alpha/Beta T CELL AND B CELL DEPLETION FOR PATIENTS WITH HEMATOLOGIC MALIGNANCIES

This is a single arm pilot study for patients using α/β T cell-depleted peripheral Stem Cell Transplantation (PSCT) in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and graft versus host disease (GVHD), and one year leukemia free survival.

Study Overview

• Status: Active, not recruiting
• Conditions: Lymphoma; Leukemia; Myelodysplasia
• Intervention / Treatment: Device: Alpha Beta T cell depletion

• Study Type: Interventional
• Enrollment (Estimated): 140
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19103
      • The Children's Hospital of Philadelphia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 23 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Leukemias/lymphomas

   • Acute myeloid leukemia, primary or secondary Disease status: remission or <10% bone marrow blasts
   • Myelodysplasia
   • Acute lymphoblastic leukemia Disease status: in hematologic remission
   • Chronic myelogenous leukemia Disease status: chronic phase, accelerated phase or blast crisis now in second chronic phase.
   • Mixed lineage or biphenotypic acute leukemia
   • Lymphoblastic lymphoma Disease status: remission
   • Burkitt's lymphoma/leukemia: Disease status- in remission
2. Acceptable organ status clearance
3. No active untreated infection. Patients with possible fungal infections must have had at least 2 weeks of appropriate anti-fungal antibiotics and be asymptomatic
4. Signed informed consent.
5. Negative pregnancy test for females of childbearing potential.

Exclusion Criteria:

1. Patients who do not meet disease, organ or infectious criteria.
2. No suitable donor
3. Received previous allogeneic transplant

Donor must be either unrelated, mismatched up to one antigen/one antigen and one allele or a related, HLA match other than a sibling.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Alpha Beta Total Body Irradiation - total body irradiation (TBI) first; Alpha Beta Total Body Irradiation - TBI first Day Treatment; 11 Anti-thymocyte globulin (ATG); 10 ATG; 9 ATG; 8 TBI; 7 TBI; 6 TBI; 5 Thiotepa; 4 Thiotepa; 3 Cyclophosphamide; 2 Cyclophosphamide; 1 Rest 0 Transplant with alpha beta T cell depleted stem cells	Device: Alpha Beta T cell depletion
Experimental: Alpha Beta Total Body Irradiation - TBI last; Alpha Beta Total Body Irradiation - TBI last Day Treatment; 9 ATG; 8 ATG; 7 Thiotepa + ATG; 6 Thiotepa; 5 Cyclophosphamide; 4 Cyclophosphamide; 3 TBI; 2 TBI; 1 TBI 0 Transplant with alpha beta T cell depleted stem cells	Device: Alpha Beta T cell depletion
Experimental: Alpha Beta Non-irradiation regimen; Alpha Beta Non-irradiation regimen Day Treatment; 9 Busulfan + ATG; 8 Busulfan + ATG; 7 Busulfan +ATG; 6 Busulfan; 5 Thiotepa; 4 Thiotepa; 3 Cyclophosphamide; 2 Cyclophosphamide; 1 0 Transplant with alpha beta T cell depleted stem cells	Device: Alpha Beta T cell depletion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Engraftment: rates of engraftment, defined as the first day of Absolute Neutrophil Count (ANC) greater than 500 for the first of three consecutive days	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Severe acute and chronic graft versus host disease (GVHD)	Estimation of the incidence and extent of acute and chronic GVHD. The overall incidence of acute GVHD, the incidence of Grade 4 acute GVHD and the incidence of chronic GVHD.	1 year

Collaborators and Investigators

• Sponsor: Children's Hospital of Philadelphia
• Investigators: Principal Investigator: Timothy Olson, MD, PhD, Children's Hospital of Philadelphia

Publications and helpful links

General Publications

• Leahy AB, Li Y, Talano JA, Elgarten CW, Seif AE, Wang Y, Johnson B, Monos DS, Kadauke S, Olson TS, Freedman J, Wray L, Grupp SA, Bunin N. Unrelated donor alpha/beta T cell- and B cell-depleted HSCT for the treatment of pediatric acute leukemia. Blood Adv. 2022 Feb 22;6(4):1175-1185. doi: 10.1182/bloodadvances.2021005492.

Study record dates

Study Major Dates

• Study Start: October 1, 2014
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2027

Study Registration Dates

• First Submitted: October 22, 2014
• First Submitted That Met QC Criteria: December 23, 2014
• First Posted (Estimated): December 24, 2014

Study Record Updates

• Last Update Posted (Estimated): October 31, 2025
• Last Update Submitted That Met QC Criteria: October 29, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Transplant; Alternative Donor
• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Bone Marrow Diseases; Anemia; Myelodysplastic Syndromes; Anemia, Refractory; Hemic and Lymphatic Diseases; Leukemia; Lymphoma; Anemia, Refractory, with Excess of Blasts
• Other Study ID Numbers: 13-010495; BT13BTθ51 (The Children's Hospital of Philadelphia)