A Phase 1 Study of WU-NK-101 in Patients With Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

NCT05470140 | Completed Phase 1 FDA Drug

• 1 other identifier: WUN101-01
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 9

Brief Summary

This study is a Phase 1, open-label, dose escalation, and cohort expansion study designed to characterize the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary anti-leukemic activity of WU-NK-101 in R/R AML.

Conditions

Acute Myeloid Leukemia

Keywords

relapsed, refractory, leukemia, NK cells

Outcome Measures

Primary Outcomes (2)

• Incidence of Adverse Events of WU-NK-101 as assessed by CTCAE v5

  Safety is based on evaluation of adverse events (AEs) and serious adverse events (SAEs) from the time of consent until End of Study (EOS) visit.

  24 months

• Maximum Tolerated Dose

  Maximum Tolerated or Administered Dose of WU-NK-101

  Up to 21 days from first dose

Secondary Outcomes (3)

• Overall Survival

  3 months

• Duration of Response

  24 months

• Overall Response Rate (ORR)

  24 months

Study Arms (1)

Experimental: WU-NK-101

EXPERIMENTAL

A non-engineered Natural Killer (NK) cell derived from peripheral blood mononuclear cells (PBMC) that is cytokine-reprogrammed, expanded, and cryopreserved to create an allogeneic enhanced Memory-like anti-tumor NK cell therapy product. Each 28-day cycle of treatment consists of 3 doses of WU-NK-101 administered on Day 1, Day 8, and Day 15.

Biological: WU-NK-101

Interventions

WU-NK-101 BIOLOGICAL

WU-NK-101 administered on Day 1, Day 8, and Day 15.

Experimental: WU-NK-101

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of primary or secondary AML (any subtype except acute promyelocytic leukemia) according to World Health Organization (WHO) 2016 classification
• Unlikely to benefit from standard of care therapy
• Patients with AML post hematopoietic stem cell transplant (HSCT) \[permitted in Cohort Expansion Phase only\] must meet the following criteria:
• There must be histological confirmation of AML relapse after HSCT
• Undergone allogeneic HSCT (alloSCT) \> 90 days prior to enrollment from a match related donor, matched unrelated donor, cord blood donor, or haplo- identical donor
• Off all immunosuppressive medications for a minimum of 2 weeks
• Adequate organ function as defined in the protocol
• Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2 at screening

You may not qualify if:

• Circulating blast count \>30,000/µL by morphology or flow cytometry (cytoreductive therapies such as leukapheresis or hydroxyurea are allowed)
• Uncontrolled or untreated bacterial, fungal, or viral infections, including HIV, Hepatitis B or C infection, or uncontrolled infection of any etiology
• Uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiogram (ECG) suggestive of acute ischemia or active conduction system abnormalities
• Severe renal impairment, defined as creatinine clearance \<40 mL/min

Sponsors & Collaborators

• Wugen, Inc.: lead

Study Sites (9)

City of Hope

Duarte, California, 91010, United States

Location

Stanford Healthcare

Palo Alto, California, 94304, United States

Location

Norton Health Care

Louisville, Kentucky, 40299, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Providence Portland Medical Center

Portland, Oregon, 97213, United States

Location

Peter MacCallum Cancer Center

Melbourne, Australia

Location

Royal Perth Hospital

Perth, Australia

Location

Royal Prince Alfred Hospital

Sydney, Australia

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence; Leukemia

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Cherry Thomas, MD

  Wugen, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This study will determine the safety and tolerability of WU-NK-101 and define the RP2D.

Study Record Dates

• First Submitted: July 20, 2022
• First Posted: July 22, 2022
• Study Start: July 1, 2023
• Primary Completion: October 8, 2024
• Study Completion: October 8, 2024
• Last Updated: September 30, 2025

Record last verified: 2025-09

Locations

AU (3); US (6)