About this trial
The purpose of this research study is to test the safety and efficacy of cytokine induced memory-like (CIML) natural killer (NK) cells expanded with Interleukin-2 (IL-2) at preventing relapse in acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or MDS and myeloproliferative neoplasm (MPN) overlap syndrome after a standard-of-care stem cell transplant.
Names of the study therapies involved in this study are:
* CIML NK cells intravenous infusion (cellular therapy) * Subcutaneous Interleukin-2 (recombinant, human glycoprotein)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
De novo AML diagnosed at or after age 60, except CBF AML
De novo AML in CR1 AND MRD+ by Hematologics Inc. flow cytometry pretransplant (this would be on the most recent pre-transplant bone marrow)
Secondary AML
AML with 5 - > 10% blasts at the time of transplant who are judged by their treating clinician and study PI to have no benefit from additional pre-transplant chemotherapy
Disqualifiers
Adult participants who are eligible for and who would be expected to have a greater benefit from myeloablative conditioning in their SOC allo HSCT as judged by their treating physician
Participants with mutations such as FLT3-ITD, IDH, or BCR-ABL mutations who are planned to receive targeted agent maintenance therapy to prevent relapse post-transplant are excluded.
Extramedullary leukemia involving sanctuary sites not readily accessible to immune surveillance, such as CNS or testis. Other sites of extramedullary relapse (e.g., leukemia cutis, granulocytic sarcoma) are acceptable.
The planned use of sirolimus for GVHD prophylaxis would result in exclusion of the patient from the study. Consideration of the addition of sirolimus to the GVHD prophylaxis regimen within the first 100 days after transplant must be reviewed with the study PI.
Trial design
Single group
Treatments tested in this trial
Cytokine Induced Memory-like Natural Killer Cells
Biological/VaccineAllogeneic, cytokine induced memory-like natural killer cells, via intravenous infusion per protocol.
Interleukin-2
Biological/VaccineRecombinant, human glycoprotein, single-use 22 MIU vials, via subcutaneous injection per protocol.
Treatment groups
Trial outcomes
Primary outcomes
Dose Limiting Toxicity (DLT) [Phase 1]
All DLT was defined as an adverse event (AE) that is related to the CIML NK cell infusion combined with IL-2 in adult patients undergoing RIC HLA-matched related or haploidentical donor stem cell transplantation using PTCY-based GVHD prophylaxis. Toxicities are to be assessed according to the CTCAEv5 (Appendix C). Management and dose modifications associated with the above adverse events are outlined in protocol 6.
Secondary outcomes
Maximum Tolerated Dose (MTD) [Phase 1]
The MTD in the CIML NK cell infusion combined with IL-2 in adult patients undergoing RIC HLA-matched related or haploidentical donor stem cell transplantation using PTCY-based GVHD prophylaxis is determined by the number pf patients who experience a DLT. See previous primary outcome measure for the DLT definition. The MTD is defined as the highest dose at which fewer than 2 out of 5 patient in each dose cohort experience a DLT. Dose-de-escalation will take place if MTD considered exceeded in each dose cohort.
Complete Remission (CR/CRi) Rate (CRR)
The CRR is defined as the proportion of participants achieving complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) based on criteria defined in protocol appendix H.
Measurable Residual Disease (MRD) Rate
MRD rate is defined as the proportion pf participants achieving MRD using the validated NGS-based assay.
1-year Progression-Free Survival (PFS) Rate
1-year PFS is a probability estimated using progression-free survival based on the Kaplan-Meier method is defined as the duration between randomization and documented disease progression (PD) or death, or is censored at time of last disease assessment.
Sponsors and contacts
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