NCT04220684

Trial Testing Safety of IL-21 NK Cells for Induction of R/R AML

Completed Phase 1 Last updated 9 January 2026

What this trial tests

Phase 1 trial testing Cytarabine Hydrochloride in Allogeneic Stem Cell Transplant Recipient in 19 participants. Completed in 22 October 2024.

Timeline

11 June 2020

Primary endpoint
22 October 2024

22 October 2024

Quick facts

Lead sponsor	Sumithira Vasu
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	19
Start date	11 June 2020
Primary completion	22 October 2024
Estimated completion	22 October 2024
Sites	2 locations across United States

Drugs / interventions tested

• Cytarabine Hydrochloride — full drug profile →
• Fludarabine (FLUDARABINE) — full drug profile →
• Membrane-bound Interleukin-21-Expanded Haploidentical Natural Killer Cells — full drug profile →

Conditions studied

• Allogeneic Stem Cell Transplant Recipient — all drugs for Allogeneic Stem Cell Transplant Recipient →
• Blasts 10 Percent or More of Bone Marrow Nucleated Cells — all drugs for Blasts 10 Percent or More of Bone Marrow Nucleated Cells →
• Recurrent Acute Myeloid Leukemia — all drugs for Recurrent Acute Myeloid Leukemia →
• Refractory Acute Myeloid Leukemia — all drugs for Refractory Acute Myeloid Leukemia →

Sponsor

Sumithira Vasu — full company profile →

Who can join

18 and older, any sex, with Allogeneic Stem Cell Transplant Recipient or Blasts 10 Percent or More of Bone Marrow Nucleated Cells. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This phase I trial studies the side effects of donor natural killer (NK) cell therapy in treating patients with acute myeloid leukemia that has come back (recurrent) or has not responded to treatment (refractory). Natural killer cells are a type of immune cell. Immunotherapy with genetically modified NK cells from donors may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. NK cell-based cancer immunotherapy: from basic biology to clinical development.
   Liu S, Galat V, Galat Y, Lee YKA, et al · · 2021 · cited 504× · PMID 33407739 · DOI 10.1186/s13045-020-01014-w
2. Allogeneic natural killer cell therapy.
   Berrien-Elliott MM, Jacobs MT, Fehniger TA. · · 2023 · cited 153× · PMID 36416736 · DOI 10.1182/blood.2022016200
3. TP53-Mutated Myelodysplastic Syndrome and Acute Myeloid Leukemia: Biology, Current Therapy, and Future Directions.
   Daver NG, Maiti A, Kadia TM, Vyas P, et al · · 2022 · cited 126× · PMID 36218325 · DOI 10.1158/2159-8290.cd-22-0332
4. Natural killer cells in clinical development as non-engineered, engineered, and combination therapies.
   Lamers-Kok N, Panella D, Georgoudaki AM, Liu H, et al · · 2022 · cited 125× · PMID 36348457 · DOI 10.1186/s13045-022-01382-5
5. Natural killer cell-based immunotherapy for acute myeloid leukemia.
   Xu J, Niu T. · · 2020 · cited 83× · PMID 33287858 · DOI 10.1186/s13045-020-00996-x
6. Natural killer cell therapy for hematologic malignancies: successes, challenges, and the future.
   Lamb MG, Rangarajan HG, Tullius BP, Lee DA. · · 2021 · cited 55× · PMID 33766099 · DOI 10.1186/s13287-021-02277-x
7. Inflammation in cancer: therapeutic opportunities from new insights.
   Xie Y, Liu F, Wu Y, Zhu Y, et al · · 2025 · cited 53× · PMID 39994787 · DOI 10.1186/s12943-025-02243-8
8. TIGIT Expression on Activated NK Cells Correlates with Greater Anti-Tumor Activity but Promotes Functional Decline upon Lung Cancer Exposure: Implications for Adoptive Cell Therapy and TIGIT-Targeted Therapies.
   Hasan MF, Croom-Perez TJ, Oyer JL, Dieffenthaller TA, et al · · 2023 · cited 25× · PMID 37345049 · DOI 10.3390/cancers15102712