Actively Recruiting

Phase 1
Phase 2
Age: 1Year - 24Years
All Genders
NCT05503134

Safety and Efficacy of Expanded, Universal Donor Natural Killer Cells for Relapsed/Refractory AML

Led by Nationwide Children's Hospital · Updated on 2024-11-04

20

Participants Needed

1

Research Sites

259 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a phase I/II dose escalation study designed to determine the safety and estimate the efficacy of UD-NK cells combined with FLA chemotherapy in patients age 1-24.99 with relapsed or refractory acute myeloid leukemia. PRIMARY OBJECTIVE: I. To determine the safety and recommended phase II dose of adoptive NK cell therapy using UD-NK cells in pediatric and young adult patients with relapsed/refractory AML. SECONDARY OBJECTIVES: I. To estimate the efficacy of UD- NK cells with FLA chemotherapy in pediatric and young adult patients with relapsed/refractory AML. EXPLORATORY OBJECTIVES: I. To determine the immunophenotype and function of UD-NK cells II. To characterize in vivo expansion of UD-NK cells III. To determine the persistence of UD-NK cells Six doses of universal donor mbIL-21 expanded NK cells (UD-NK) given thrice weekly for two weeks. Days may vary and NK cells can be given from days 0 to 21. Patients may receive up to 2 cycles of fludarabine/cytarabine (FLA) + NK cells (up to 12 NK cell infusions) if they do not achieve CR after cycle 1 or if necessary to bridge to transplant.

CONDITIONS

Official Title

Safety and Efficacy of Expanded, Universal Donor Natural Killer Cells for Relapsed/Refractory AML

Who Can Participate

Age: 1Year - 24Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with relapsed or primary refractory AML, including those in first or subsequent relapse or after stem cell transplant
  • Patients with primary refractory AML after 2 cycles of induction chemotherapy, including persistent minimal residual disease
  • Patients with isolated CNS or extramedullary AML disease
  • Age between 1 and 24.99 years
  • Negative pregnancy test within 2 weeks prior to enrollment for females of childbearing potential
  • Agreement to use effective contraception for 6 months after last chemotherapy or NK cell infusion for sexually active males and females of childbearing potential
  • Negative HIV serology
  • Adequate organ function: creatinine 64 2 mg/dl or creatinine clearance > 60 ml/min/1.73m2; total bilirubin 64 2 mg/dl (unless Gilbert's syndrome); AST and ALT 64 5x upper limit normal unless due to leukemia; cardiac ejection fraction 50% or shortening fraction 520%, with cardiology clearance if needed
  • Controlled seizure disorder if applicable
  • Prior treatment-related non-hematologic toxicities resolved to grade 2 or less
  • Ability to understand and willing to sign informed consent
Not Eligible

You will not qualify if you...

  • Receipt of AML-directed therapies within 2 weeks before starting study treatment (except hydroxyurea)
  • Current use of systemic immunosuppressive therapy within 2 weeks prior to enrollment
  • History of donor lymphocyte infusion or cellular therapy within 30 days
  • Allogeneic stem cell transplant less than 3 months before enrollment
  • Comorbidities that would prevent safe study treatment per investigator
  • Karnofsky or Lansky performance status less than 50
  • Uncontrolled infection not improving with treatment
  • Uncontrolled arrhythmias or symptomatic cardiac disease
  • History of autoimmune disease
  • Active graft-versus-host disease at enrollment
  • History of adoptive cell therapy within 30 days without hematologic recovery (ANC < 500/bcL or platelets < 50,000/bcL)

AI-Screening

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Trial Site Locations

Total: 1 location

1

Nationwide Children's Hospital

Columbus, Ohio, United States, 43205

Actively Recruiting

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Research Team

M

Melinda C Triplet

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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