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

JRCT ID: jRCT2033240022

Registered date:10/04/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Leukemia
• Date of first enrollment: 11/07/2023
• Target sample size: 53
• Countries of recruitment: United States,Japan
• Study type: Interventional

Intervention(s)

Phase 1: Dose Escalation of GDX012
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 except in Japan.

Phase 2a: GDX012
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 except in Japan.

Outcome(s)

Primary Outcome

1.Number of Participants With Dose Limiting Toxicities (DLTs)
Time Frame: Up to 1 month

2.Maximum Tolerated Dose (MTD) of GDX012
Time Frame: Up to 1 month

3.Number of Participants With Adverse Events (AEs)
Time Frame: Up to 14 months
An adverse event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug.

Secondary Outcome

1.Number of Participants With Disease Response
Time Frame: Up to 14 months
Disease response includes participants achieving complete response [CR] complete response with incomplete hematologic recovery [CRi] complete response with partial hematologic recovery [CRh] morphological leukemia-free state [MLFS] or partial response [PR] based on 2022 European Leukemia Net [ELN] response criteria for AML after GDX012 administration.

2.Number of Participants With Measurable Residual Disease (MRD) Negative Status as Determined by Flow Cytometry
Time Frame: Up to 14 months

3.Duration of Response (DOR)
Time Frame: Up to 14 months
DOR is defined as the time from the date of first documented CR, CRh, or CRi to the date of relapse or death.

4.Event-free Survival (EFS)
Time Frame: Up to 14 months
EFS is defined as the time from the date of the first GDX012 administration to the date of treatment failure, relapse or death, whichever comes first.

5.Overall Survival (OS)
Time Frame: Up to 14 months
OS is defined as the time from the date of the first GDX012 administration to the date of death.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

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

Exclude 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.

Related Information

• Primary Sponsor: Sumi Eriko
• Secondary ID(s): NCT05886491