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

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.

Study Overview

• Status: Completed
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Biological: WU-NK-101

Detailed Description

This is a first in human, multi-center Phase 1 single agent study in patients with R/R AML who have exhausted other treatment options. The study will consist of two phases, dose escalation and cohort expansion. During the Dose Escalation Phase, up to 18 patients will be treated with WU-NK-101 in up to 3 Dose Levels (DL) until maximum tolerated dose (MTD) or maximum administered dose (MAD) is determined.

Once the MTD/MAD is defined, 6 additional patients will be enrolled in the Cohort Expansion Phase to further characterize the safety, tolerability, as well as determining the recommended phase 2 dose (RP2D) of WU-NK-101. Patients in the Cohort Expansion Phase, who achieve a partial response (PR), may receive up to 2 further re-induction cycles contingent on safety in the Dose Escalation Phase; patients who achieve a complete remission with partial hematologic recovery (CRh) or complete remission with incomplete hematologic recovery (CRi) at any point during the course of treatment may receive a further consolidation cycle, for a total of up to 4 cycles per patient. During cohort expansion, dosing breaks of up to two weeks are allowed between cycles.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • Melbourne, Australia
    • Peter MacCallum Cancer Center
  • Perth, Australia
    • Royal Perth Hospital
  • Sydney, Australia
    • Royal Prince Alfred Hospital
• United States
  • California
    • Duarte, California, United States, 91010
      • City of Hope
    • Palo Alto, California, United States, 94304
      • Stanford Healthcare
  • Kentucky
    • Louisville, Kentucky, United States, 40299
      • Norton Health Care
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University
  • Oregon
    • Portland, Oregon, United States, 97213
      • Providence Portland Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Confirmed diagnosis of primary or secondary AML (any subtype except acute promyelocytic leukemia) according to World Health Organization (WHO) 2016 classification
2. Unlikely to benefit from standard of care therapy

3. 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
4. Adequate organ function as defined in the protocol
5. Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2 at screening

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: WU-NK-101; 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; WU-NK-101 administered on Day 1, Day 8, and Day 15.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	Time from study drug administration (Day 1) until death on study.	3 months
Duration of Response	Time of response to the time of disease relapse, progression, or death due to any cause.	24 months
Overall Response Rate (ORR)	ORR is defined as proportion of patients that achieve complete remission (CR) + complete remission with incomplete hematologic recover (CRi).	24 months

Collaborators and Investigators

• Sponsor: Wugen, Inc.
• Investigators: Study Director: Cherry Thomas, MD, Wugen, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2023
• Primary Completion (Actual): October 8, 2024
• Study Completion (Actual): October 8, 2024

Study Registration Dates

• First Submitted: July 20, 2022
• First Submitted That Met QC Criteria: July 20, 2022
• First Posted (Actual): July 22, 2022

Study Record Updates

• Last Update Posted (Estimated): September 30, 2025
• Last Update Submitted That Met QC Criteria: September 24, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: relapsed, refractory, leukemia, NK cells
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Recurrence; Leukemia; Leukemia, Myeloid, Acute
• Other Study ID Numbers: WUN101-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No