A Study of GDX012 in Adults With Relapsed or Refractory Acute Myeloid Leukemia

Takeda

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Leukemia

Treatments: Drug: GDX012; Drug: Chemotherapy Agents

Study type: Interventional

Funder types: Industry

Identifiers

NCT05886491

TAK-012-1501

Details and patient eligibility

About

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.

Full 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:

1. GDX012 Dose 1
2. GDX012 Dose 2
3. 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. The overall time to participate in the study is approximately 14 months.

Enrollment: 53 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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53 participants in 2 patient groups

Phase 1: Dose Escalation of GDX012

Experimental group

Description:

Participants will receive GDX012 weight-based dose as intravenous (IV) infusion on Day 1 of Phase 1 after lymphodepleting chemotherapy. Three dose levels of GDX012 will be tested in Phase 1. Some participants may be eligible for a second dose.

Treatment:

Drug: GDX012

Drug: Chemotherapy Agents

Phase 2a: GDX012

Experimental group

Description:

Participants will receive GDX012 (weight-based) IV infusion at pre-selected one or two dose levels from Phase 1, on Day 1 after lymphodepleting chemotherapy. Some participants may be eligible for a second dose.

Treatment:

Drug: GDX012

Drug: Chemotherapy Agents