GDX012 treatment for adults with relapsed or refractory acute myeloid leukemia

A Phase 1/2a, Open-Label, Dose Escalation, and Dose Expansion Study to Assess the Safety and Efficacy of GDX012 in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Phase1; Phase2 Interventional Takeda · NCT05886491

This study is testing a new cell therapy called GDX012 to see if it can help adults with relapsed or hard-to-treat acute myeloid leukemia feel better.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment53 (estimated)
Ages18 Years and up
SexAll
SponsorTakeda Industry-sponsored
Drugs / interventionschemotherapy
Locations14 sites (Birmingham, Alabama and 13 other locations)
Trial IDNCT05886491 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates GDX012, a novel cell therapy, for adults suffering from relapsed or refractory acute myeloid leukemia (AML). The study consists of two phases: a dose escalation phase to determine the safety and tolerability of GDX012 at different dose levels, followed by a dose expansion phase to identify the recommended phase 2 dose for future studies. Approximately 53 patients will be enrolled across multiple centers in the United States, with a total participation time of about 14 months.

Who should consider this trial

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

Not a fit: Patients with non-relapsed AML or those who do not meet the eligibility criteria will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat AML.

How similar studies have performed: While there have been studies on cell therapies for AML, the specific approach with GDX012 is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion criteria:

1. Total body weight of ≥40 kg.
2. Must have pathologically confirmed relapsed or refractory acute myeloid leukemia (R/R AML) including:

   1. Relapsed AML is defined as ≥5% blasts in the bone marrow (BM) or peripheral blood at any time after achieving a CR, CRh, Cri, or MLFS.
   2. Refractory AML is defined as failure to achieve a CR, CRh, Cri, or MLFS after 1 of the following regimens:

   i. Two courses of intensive induction chemotherapy. ii. At least 2 cycles of hypomethylating agent (HMA) or low-dose, cytarabine-based combination regimen.

   iii. At least 4 cycles of HMA monotherapy.
3. During dose escalation, participants must be ineligible for hematopoietic stem cell transplantation (HSCT).
4. Must have an anticipated life expectancy of \>3 months before lymphodepletion.
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
6. Participants must have adequate renal, cardiac, hepatic, pulmonary and bone marrow function as defined by the protocol.

Exclusion criteria:

1. Diagnosis of acute promyelocytic leukemia.
2. Has received or plans to receive any of the excluded therapy/treatment within the specified timeframe before lymphodepleting chemotherapy as defined by the protocol.
3. Prior allogeneic HSCT within 3 months of signing informed consent form (ICF) or with ongoing requirement for systemic graft-versus-host therapy.
4. Active central nervous system (CNS) involvement.
5. History of malignancy other than non-melanoma skin cancer or carcinoma in situ (eg. cervix, bladder, breast) low grade prostate cancer without treatment requirement unless in remission without treatment for ≥2 years.

Where this trial is running

Birmingham, Alabama and 13 other locations

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 LeukemiaDrug TherapyAMLacute myeloid leukemiacell therapyallogenicgamma delta T cellsrelapsed/ refractory
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.