Actively Recruiting

Phase 1
Age: 0 - 30Years
All Genders
NCT05011422

Haploidentical Hematopoietic Stem Cell Transplantation With Ex Vivo TCR Alpha/Beta and CD19 Depletion in Pediatric Hematologic Malignancies

Led by Washington University School of Medicine · Updated on 2026-03-16

50

Participants Needed

1

Research Sites

343 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This single arm pilot phase I study with safety run-in is designed to estimate the safety and efficacy of a familial mismatched or haploidentical hematopoietic stem cell transplantation (haplo-HSCT) using a novel graft modification technique (selective αβ-TCR and CD19 depletion).

CONDITIONS

Official Title

Haploidentical Hematopoietic Stem Cell Transplantation With Ex Vivo TCR Alpha/Beta and CD19 Depletion in Pediatric Hematologic Malignancies

Who Can Participate

Age: 0 - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of high-risk pediatric hematologic malignancy including B cell ALL in first, second, or third remission with specific risk features
  • T cell ALL in first or subsequent remission with measurable residual disease
  • AML in first or subsequent remission with defined high-risk genetic or residual disease features
  • Mixed phenotype or undifferentiated leukemia, therapy-related leukemia, NK cell lineage leukemia, myelodysplastic syndrome, or juvenile myelomonocytic leukemia
  • Prior hematopoietic stem cell transplant allowed if no active graft-versus-host disease and off immunosuppression for at least 3 months
  • Availability of a familial haploidentical donor with at least one identical allele at HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1 loci (minimum 5/10 match)
  • Age 30 years or younger
  • Lansky or Karnofsky performance status greater than 50%
  • Adequate organ function including cardiac (LVEF  40%), hepatic (bilirubin and liver enzymes within limits), renal (GFR  60 mL/min/1.73m2), and pulmonary (oxygen saturation  92% and lung function tests  50% predicted)
  • Agreement to use effective contraception if of childbearing potential
  • Ability to understand and sign informed consent
Not Eligible

You will not qualify if you...

  • Presence of an available matched related donor
  • Active non-hematologic malignancy
  • Current treatment with other investigational agents at transplant
  • Active central nervous system or extramedullary disease
  • History of allergic reactions to conditioning agents
  • Uncontrolled illness including active infection, symptomatic heart failure, or unstable arrhythmia
  • Significant anti-donor HLA antibodies per institutional standards
  • Presence of a major disorder contraindicating hematopoietic stem cell transplantation
  • Pregnancy or breastfeeding; women of childbearing potential must have negative pregnancy test within 14 days before conditioning

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Washington University School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

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Research Team

T

Thomas Pfeiffer, M.D.

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Haploidentical Hematopoietic Stem Cell Transplantation With Ex Vivo TCR Alpha/Beta and CD19 Depletion in Pediatric Hematologic Malignancies | DecenTrialz