LeukemiaPhase 1/2NCT05886491
A Study of GDX012 in Adults With Relapsed or Refractory Acute Myeloid Leukemia
A Phase 1/2 study testing GDX012 and Chemotherapy Agents in people with Leukemia, run by Takeda at 11 sites in 2 countries.
Trial summary
GDX012 is a novel cell therapy developed for the treatment of certain types of cancer, including Acute Myeloid Leukemia (AML). The main aims of the study are to learn how safe GDX012 is, how treatment with GDX012 is tolerated and to determine the best dose of GDX012. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- GDX012 and Chemotherapy Agents
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 11 Jul 2023to 30 Nov 2025
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Total body weight of ≥40 kg.
- Must have pathologically confirmed relapsed or refractory acute myeloid leukemia (R/R AML) including:
- 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.
- Refractory AML is defined as failure to achieve a CR, CRh, Cri, or MLFS after 1 of the following regimens:
- Two courses of intensive induction chemotherapy. ii. At least 2 cycles of hypomethylating agent (HMA) or low-dose, cytarabine-based combination regimen.
- At least 4 cycles of HMA monotherapy.
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- During dose escalation, participants must be ineligible for hematopoietic stem cell transplantation (HSCT).
- Must have an anticipated life expectancy of >3 months before lymphodepletion.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
- Participants must have adequate renal, cardiac, hepatic, pulmonary and bone marrow function as defined by the protocol.
Who cannot join
- Diagnosis of acute promyelocytic leukemia.
- Has received or plans to receive any of the excluded therapy/treatment within the specified timeframe before lymphodepleting chemotherapy as defined by the protocol.
- Prior allogeneic HSCT within 3 months of signing informed consent form (ICF) or with ongoing requirement for systemic graft-versus-host therapy.
- Active central nervous system (CNS) involvement.
- 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.
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States10 sites
University of Alabama at Birmingham (UAB) Hospital
Birmingham, Alabama, 35205
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Comprehensive Cancer Center of Northwestern University
Chicago, Illinois, 60611
Cleveland Clinic
Cleveland, Ohio, 44195
Sarah Cannon/CBCI
Denver, Colorado, 80218
City of Hope
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77030
Tri-Star BMT/Sarah Cannon Nashville
Nashville, Tennessee, 37203
Thomas Jefferson University
New York, New York, 10065
Washington University
St Louis, Missouri, 63110
About this study
Official title: 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
GDX012 is a novel cell therapy developed for the treatment of certain types of cancer, including Acute Myeloid Leukemia (AML). The main aims of the study are to learn how safe GDX012 is, how treatment with GDX012 is tolerated and to determine the best dose of GDX012.
Read the detailed description
The drug being tested in this study is called GDX012. GDX012 is being tested to evaluate the safety and tolerability in adult participants with AML.
The study will enroll approximately 53 patients in two phases, dose escalation and dose expansion.
During Phase 1 (sequential dose escalation), participants will be assigned to one of the following treatment groups each consisting of 3 to 6 participants to receive GDX012 at one of the three dose levels:
- GDX012 Dose 1
- GDX012 Dose 2
- GDX012 Dose 3
Upon completion of Phase 1, 1 to 2 dose levels will be selected for Phase 2a of the study. At the completion of Phase 2a of the study a single dose may be selected by the sponsor and investigators as the recommended phase 2 dose (RP2D) for future study.
This multi-center trial will be conducted in the United States and Japan. The overall time to participate in the study is approximately 14 months.
- Conditions
- Leukemia
- Being tested
- GDX012, Chemotherapy Agents
- Keywords
- Drug Therapy, AML, acute myeloid leukemia, cell therapy, allogenic, gamma delta T cells, relapsed/ refractory
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GDX012Drug
Also called TAK-012
GDX012 suspension for IV infusion.
Chemotherapy AgentsDrug
Chemotherapy agents (fludarabine/cyclophosphamide) as per standard of care.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: Dose Escalation of GDX012 (Dose Level 1) | Experimental | Participants received GDX012 weight-based dose level 1 as an intravenous (IV) infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 1 was the lowest dose level. Receives: Drug: GDX012; Drug: Chemotherapy Agents |
| Phase 1: Dose Escalation of GDX012 (Dose Level 2) | Experimental | Participants received GDX012 weight-based dose level 2 as an IV infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 2 was higher than Dose Level 1. Receives: Drug: GDX012; Drug: Chemotherapy Agents |
| Phase 1: Dose Escalation of GDX012 (Dose Level 3) | Experimental | Participants received GDX012 weight-based dose level 3 as an IV infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 3 was higher than Dose Level 2. Receives: Drug: GDX012; Drug: Chemotherapy Agents |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Dose Limiting Toxicities (DLTs) to Determine the Maximum Tolerated Dose (MTD) of GDX012
DLTs were defined as events occurring by Day 30 after the first GDX012 infusion considered related to GDX012 if they could not be explained by underlying disease, prior therapy, intercurrent illness, or lymphodepleting chemotherapy. Events were graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. DLTs included severe non-hematologic toxicities (Grade 3-4, unresolved or involving vital organs), Grade ≥3 graft versus host disease (GvHD) graded per the Mount Sinai Acute GvHD International Consortium recommendations and 2014 National Institutes of Health (NIH) Consensus, Grade 3-4 cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) per American Society for Transplantation and Cellular Therapy (ASTCT) criteria, Grade ≥3 infusion reactions or tumor lysis syndrome (TLS), persistent cytopenia or marrow hypocellularity, and any Grade 5 toxicity attributable to GDX012.
Measured: Up to 30 days post infusion
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. This includes any newly occurring event or a previous condition that has increased in severity or frequency since the administration of study drug. TEAEs are defined as any AE that begins on or after the initiation of treatment with a study intervention or any existing event that worsens following exposure to the study intervention.
Measured: Up to 14 months
Secondary outcomes (5)
Percentage of Participants With Disease Response
Percentage of participants with disease response included participants who achieved complete response (CR), complete response with partial hematologic recovery (CRh), or complete response with incomplete hematologic recovery (CRi), based on responses assessed by the investigator according to the 2022 European Leukemia Net [ELN] response criteria for acute myeloid leukemia (AML). CR was defined as the sum of complete remission and complete remission without measurable residual disease (MRD). CRh was defined as the sum of CRh and CRhMRD-. CRi was defined as the sum of CRi and CRiMRD-.
Measured: Up to 14 months
Percentage of Participants With Measurable Residual Disease (MRD) Negative Status as Determined by Flow Cytometry
The MRD negative status was defined as (less than) <0.1% detectable disease based on local assessment of bone marrow and/or blood.
Measured: Up to 14 months
Duration of Response (DOR)
DOR was planned to be assessed for participants with CR, CRh, or CRi and was defined as the time from the date of first documented CR, CRh, or CRi to the date of first documented relapse or death from any cause, whichever comes first. CR was defined as the sum of Complete Remission and Complete Remission without MRD. CRh was defined as the sum of CRh and CRhMRD-. CRi was defined as the sum of CRi and CRiMRD- .
Measured: Up to 14 months
Event-free Survival (EFS)
EFS was defined as the time from the date of the first GDX012 administration to the date of treatment failure, relapse for those who achieved CR, CRh, or CRi, or death from any cause, whichever came first. CR was defined as the sum of Complete Remission and Complete Remission without MRD (CRMRD-). CRh was defined as the sum of CRh and CRhMRD-. CRi was defined as the sum of CRi and CRiMRD-. EFS was analyzed using the Kaplan-Meier method.
Measured: Up to 14 months
Overall Survival (OS)
OS was defined as the time from the date of the first GDX012 administration to the date of death from any cause. Participants without documentation of death at the time of analysis were censored at the last contact date. OS was analyzed using the Kaplan-Meier method.
Measured: Up to 14 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2023
- Study start11 Jul 2023
- Primary completion20 Aug 2025
- Study completion30 Nov 2025
Why it stopped: Sponsor decision (No safety concern)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05886491
- Sponsor's protocol ID
- TAK-012-1501
- Other IDs
- jRCT2033240022
- Status last verified
- Sep 2026
- First submitted
- 24 May 2023
- Last updated
- 25 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05886491
- Acronym / sponsor ID
- TAK-012-1501
- Conditions
- Leukemia
- Keywords
- Drug Therapy, AML, acute myeloid leukemia, cell therapy, allogenic, gamma delta T cells, relapsed/ refractory
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision (No safety concern)
- Enrolment
- 16 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 11 Jul 2023 (actual)
- Primary completion
- 20 Aug 2025 (actual)
- Study completion
- 30 Nov 2025 (actual)
- First posted
- 2 Jun 2023 (actual)
- Results
- Posted 25 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
GDX012 is a novel cell therapy developed for the treatment of certain types of cancer, including Acute Myeloid Leukemia (AML). The main aims of the study are to learn how safe GDX012 is, how treatment with GDX012 is tolerated and to determine the best dose of GDX012.
Detailed description
The drug being tested in this study is called GDX012. GDX012 is being tested to evaluate the safety and tolerability in adult participants with AML.
The study will enroll approximately 53 patients in two phases, dose escalation and dose expansion.
During Phase 1 (sequential dose escalation), participants will be assigned to one of the following treatment groups each consisting of 3 to 6 participants to receive GDX012 at one of the three dose levels:
- GDX012 Dose 1
- GDX012 Dose 2
- GDX012 Dose 3
Upon completion of Phase 1, 1 to 2 dose levels will be selected for Phase 2a of the study. At the completion of Phase 2a of the study a single dose may be selected by the sponsor and investigators as the recommended phase 2 dose (RP2D) for future study.
This multi-center trial will be conducted in the United States and Japan. The overall time to participate in the study is approximately 14 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Phase 1: Dose Escalation of GDX012 (Dose Level 1) | Experimental | Drug: GDX012, Drug: Chemotherapy AgentsParticipants received GDX012 weight-based dose level 1 as an intravenous (IV) infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 1 was the lowest dose level. |
| Phase 1: Dose Escalation of GDX012 (Dose Level 2) | Experimental | Drug: GDX012, Drug: Chemotherapy AgentsParticipants received GDX012 weight-based dose level 2 as an IV infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 2 was higher than Dose Level 1. |
| Phase 1: Dose Escalation of GDX012 (Dose Level 3) | Experimental | Drug: GDX012, Drug: Chemotherapy AgentsParticipants received GDX012 weight-based dose level 3 as an IV infusion on Day 1 of Phase 1, following lymphodepleting chemotherapy. Dose Level 3 was higher than Dose Level 2. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Total body weight of ≥40 kg.
- Must have pathologically confirmed relapsed or refractory acute myeloid leukemia (R/R AML) including:
- During dose escalation, participants must be ineligible for hematopoietic stem cell transplantation (HSCT).
- Must have an anticipated life expectancy of >3 months before lymphodepletion.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
Key exclusion criteria (5 listed)
- Diagnosis of acute promyelocytic leukemia.
- Has received or plans to receive any of the excluded therapy/treatment within the specified timeframe before lymphodepleting chemotherapy as defined by the protocol.
- Prior allogeneic HSCT within 3 months of signing informed consent form (ICF) or with ongoing requirement for systemic graft-versus-host therapy.
- Active central nervous system (CNS) involvement.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GDX012Also: TAK-012GDX012 suspension for IV infusion. | Drug | Phase 1: Dose Escalation of GDX012 (Dose Level 1), Phase 1: Dose Escalation of GDX012 (Dose Level 2), Phase 1: Dose Escalation of GDX012 (Dose Level 3) |
| Chemotherapy AgentsChemotherapy agents (fludarabine/cyclophosphamide) as per standard of care. | Drug | Phase 1: Dose Escalation of GDX012 (Dose Level 1), Phase 1: Dose Escalation of GDX012 (Dose Level 2), Phase 1: Dose Escalation of GDX012 (Dose Level 3) |
Primary outcome
Number of Participants With Dose Limiting Toxicities (DLTs) to Determine the Maximum Tolerated Dose (MTD) of GDX012
Time frame: Up to 30 days post infusion
DLTs were defined as events occurring by Day 30 after the first GDX012 infusion considered related to GDX012 if they could not be explained by underlying disease, prior therapy, intercurrent illness, or lymphodepleting chemotherapy. Events were graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. DLTs included severe non-hematologic toxicities (Grade 3-4, unresolved or involving vital organs), Grade ≥3 graft versus host disease (GvHD) graded per the Mount Sinai Acute GvHD International Consortium recommendations and 2014 National Institutes of Health (NIH) Consensus, Grade 3-4 cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) per American Society for Transplantation and Cellular Therapy (ASTCT) criteria, Grade ≥3 infusion reactions or tumor lysis syndrome (TLS), persistent cytopenia or marrow hypocellularity, and any Grade 5 toxicity attributable to GDX012.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to 14 months
An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. This includes any newly occurring event or a previous condition that has increased in severity or frequency since the administration of study drug. TEAEs are defined as any AE that begins on or after the initiation of treatment with a study intervention or any existing event that worsens following exposure to the study intervention.
5 secondary outcomes
Percentage of Participants With Disease Response
Time frame: Up to 14 months
Percentage of Participants With Measurable Residual Disease (MRD) Negative Status as Determined by Flow Cytometry
Time frame: Up to 14 months
Duration of Response (DOR)
Time frame: Up to 14 months
Event-free Survival (EFS)
Time frame: Up to 14 months
Overall Survival (OS)
Time frame: Up to 14 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.Link provided by the sponsor.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Takeda
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing GDX012 and Chemotherapy Agents in people with Leukemia, run by Takeda at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Dose Limiting Toxicities (DLTs) to Determine the Maximum Tolerated Dose (MTD) of GDX012 (time frame: Up to 30 days post infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026.