Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies

NCT02323867 | Active Not Recruiting Phase 2 DMC

• 2 other identifiers: 13-010495BT13BTθ51
• Study Type: interventional
• Target: 140
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Leukemia; Lymphoma; Myelodysplasia

Keywords

Transplant; Alternative Donor

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Severe acute and chronic graft versus host disease (GVHD)

  1 year

Study Arms (3)

Alpha Beta Total Body Irradiation - total body irradiation (TBI) first

EXPERIMENTAL

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

Alpha Beta Total Body Irradiation - TBI last

EXPERIMENTAL

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

Alpha Beta Non-irradiation regimen

EXPERIMENTAL

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

Interventions

Alpha Beta T cell depletion DEVICE

Alpha Beta Non-irradiation regimen

Eligibility Criteria

• Age: Up to 23 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• Acceptable organ status clearance
• No active untreated infection. Patients with possible fungal infections must have had at least 2 weeks of appropriate anti-fungal antibiotics and be asymptomatic
• Signed informed consent.
• Negative pregnancy test for females of childbearing potential.

You may not qualify if:

• Patients who do not meet disease, organ or infectious criteria.
• No suitable donor
• 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.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19103, United States

Location

Related Publications (1)

• 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.

  PMID: 34872106 DERIVED

MeSH Terms

Conditions

Leukemia; Lymphoma; Anemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Timothy Olson, MD, PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Hematopoietic Stem Cell Transplantation (HSCT) Program

Model Details: intervention with device: Alpha Beta T cell depletion

Study Record Dates

• First Submitted: October 22, 2014
• First Posted: December 24, 2014
• Study Start: October 1, 2014
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2027
• Last Updated: October 31, 2025

Record last verified: 2025-10

Locations

US (1)