Clinical trial · Interventional
Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults With High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant
A Phase II Pilot Study of Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults With High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant: A Multicenter Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) Study
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is a Phase II pilot study to determine the efficacy of three fixed dose (1 x 108/kg) infusions of ex-vivo expanded human leukocyte antigen (HLA)-haploidentical donor natural killer (NK) cells (haploNK) in children and young adults with high risk acute myeloid leukemia (AML) undergoing HLA-haploidentical hematopoietic cell transplant (haploHCT) with a busulfan and cyclophosphamide-based myeloablative conditioning regimen and post-transplant cyclophosphamide (PTCy) for graft versus host disease (GVHD) prophylaxis. The investigators will also demonstrate the feasibility of performing this trial in a multi-center study. The investigators hypothesize that the infusion of haploNK in this setting will facilitate immune reconstitution and decrease relapse rates and infectious complications without increasing GVHD, resulting in improved survival as compared to recent historical cohorts of haploHCT without NK cell infusion.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment Arm
- description
- All subjects will receive NK infusions.
- interventionNames
- Drug: Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions
Primary outcomes (1)
- measure
- 1-year RFS
- timeFrame
- 1 year
- description
- The proportion and corresponding 95% exact binomial CI of patients who are relapse-free at 1-year from day of transplant (Day 0)
Secondary outcomes (6)
- measure
- Number of functional donor-derived NK cells generated from the device
- timeFrame
- 2 years
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 0 Years
- Maximum age
- 25 Years
Show eligibility criteria text
Inclusion Criteria: * Age ≤ 25 years at time of enrollment * High-risk AML, as defined by one of the following: * AML in CR1 (defined as \<5% blasts in BM by morphology and flow cytometry) having at least one of these high-risk features: * Mutations associated with high risk disease (Appendix A). Other high-risk features not explicitly stated in Appendix A can be considered after discussion/approval with the protocol chair/team * MRD-positive at the end of Induction I chemotherapy (defined as flow cytometry ≥ 0.1% blasts) * AML in ≥CR2 (defined by \<5% blasts in BM by morphology and flow cytometry) * Recovery from prior cycle of chemotherapy as defined by an absolute neutrophil count ≥ 500/mm3 * AML secondary to select germline marrow failure disorders (with exception of Fanconi Anemia) may be eligible but require approval from Protocol Chairs prior to enrollment. * Performance status ≥70% (Lansky for \<16 years; Karnofsky for ≥16 years) * Adequate major organ system function as demonstrated by: * Renal: Creatinine clearance (CrCl) ≥60 mL/min/1.73m2 by Cockcroft-Gault formula, Schwartz formula, or nuclear GFR study (Table 3) * Hepatic: Total bilirubin \<2 mg/dL (unless due to Gilbert syndrome) and ALT and AST \< 5x ULN * Cardiac: LVEF at rest ≥50% or SF ≥27% (by MUGA or ECHO) * Pulmonary: DLCO, FEV1, and FVC ≥ 50% of predicted corrected for hemoglobin. For patients \<7 years of age or those unable to perform PFTs: O2 Sat \>92% on room air by pulse oximetry and on no supplemental O2 at rest * The patient, patient's parent, guardian, or legal representative can provide written informed consent Exclusion Criteria: * Active extramedullary disease * Unresolved/ongoing and serious viral, bacterial, or fungal infection despite appropriate treatment * Positive pregnancy test in a female of child-bearing potential (FCBP) * Inability to comply with medical therapy or follow-up * Prior allogeneic transplant * Patients with Fanconi Anemia and Down syndrome
References
Publications (0)
Data not yet available