Natural Killer-cell Therapy for Acute Myeloid Leukemia

Infusion of ex Vivo-generated Allogeneic Natural Killer Cells in Combination With Subcutaneous IL-2 in Patients With Acute Myeloid Leukemia: a Phase I/IIa Study

Phase1; Phase2 Interventional Radboud University Medical Center · NCT04347616

This study is testing a new treatment using special immune cells from umbilical cord blood to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment23 (estimated)
Ages18 Years and up
SexAll
SponsorRadboud University Medical Center Academic / other
Drugs / interventionschemotherapy, cyclophosphamide, fludarabine
Locations1 site (Nijmegen)
Trial IDNCT04347616 on ClinicalTrials.gov

What this trial studies

This study investigates a novel treatment for relapsed or refractory acute myeloid leukemia (AML) using ex vivo-generated allogeneic natural killer (NK) cells. Patients will first receive non-myeloablative conditioning chemotherapy, followed by the infusion of NK cells derived from umbilical cord blood. The study consists of two phases: a safety assessment of different doses of IL-2 in phase I, followed by an efficacy evaluation in phase IIa. The goal is to determine the safety and effectiveness of this cellular immunotherapy approach.

Who should consider this trial

Good fit: Ideal candidates include adults with relapsed or refractory AML who are ineligible for allogeneic stem cell transplantation.

Not a fit: Patients with progressive disease or those on immunosuppressive drugs may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with difficult-to-treat acute myeloid leukemia.

How similar studies have performed: Other studies utilizing natural killer cell therapies have shown promise, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* AML patients (de novo and secondary) or patients with MDS excess blasts-2 according to WHO criteria 2016, who have stable disease or non-rapidly progressive disease with or without disease controlling medication who are (at time of inclusion) ineligible for allo-SCT.
* Patients may belong to any of the following categories:

  * Relapsed/refractory disease after treatment with intensive chemotherapy, hypomethylating agents, targeted agents, autologous or allo-SCT (at least 6 months ago) and DLI
  * Newly diagnosed, untreated patients ineligible for allo-SCT

Other inclusion criteria:

* Age ≥ 18 years
* WHO performance 0-2
* Life expectancy of \> 4 months
* Written informed consent
* Hydrea is allowed as pre-treatment to control blast count until day -3
* Other disease controlling medication is allowed until day -7

Exclusion Criteria:

* Progressive disease according to ELN criteria in case of previous therapy
* Patients on immunosuppressive drugs or active GvHD
* Patients with active infections (viral, bacterial or fungal); acute anti-infectious therapy must have been completed within 14 days prior to study treatment
* Severe cardiovascular disease (CTCAE III-IV)
* Severe pulmonary dysfunction (CTCAE III-IV)
* Severe renal dysfunction (CTCAE III-IV)
* Severe hepatic dysfunction (CTCAE III-IV)
* Severe neurological or psychiatric dysfunction (CTCAE III-IV)
* Patients on concurrent chemotherapy or interferon-alpha treatment
* Pregnancy or breastfeeding

Where this trial is running

Nijmegen

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Acute Myeloid Leukemia RefractoryAcute Myeloid Leukemia, Relapsed, AdultNatural Killer cellsAcute Myeloid LeukemiaCellular Immunotherapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.