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

April 3, 2024 updated by: Takeda

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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:

  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.

Study Type

Interventional

Enrollment (Estimated)

53

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294-0004
        • Not yet recruiting
        • University of Alabama at Birmingham (UAB) Hospital
        • Contact:
        • Principal Investigator:
          • Pankit Vachhani
    • California
      • Duarte, California, United States, 91010-3012
        • Recruiting
        • City of Hope
        • Contact:
        • Principal Investigator:
          • MONZR AL MALKI
      • Palo Alto, California, United States, 94304-1812
        • Not yet recruiting
        • Stanford University
        • Contact:
        • Principal Investigator:
          • Alice Bertaina
    • Colorado
      • Denver, Colorado, United States, 80218-1258
        • Not yet recruiting
        • Sarah Cannon/CBCI
        • Principal Investigator:
          • Alireza Eghtedar
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611-3124
        • Not yet recruiting
        • Comprehensive Cancer Center of Northwestern University
        • Principal Investigator:
          • Jessica Altman
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Not yet recruiting
        • Dana-Farber Cancer Institute
        • Contact:
        • Principal Investigator:
          • Evan CHEN
    • Missouri
      • Saint Louis, Missouri, United States, 63110-1010
        • Not yet recruiting
        • Washington University
        • Contact:
        • Principal Investigator:
          • John DiPersio
    • New York
      • Buffalo, New York, United States, 14263-0001
        • Not yet recruiting
        • Roswell Park Comprehensive Cancer Center
        • Contact:
        • Principal Investigator:
          • Eunice Wang
      • New York, New York, United States, 10065-4870
        • Recruiting
        • Thomas Jefferson University
        • Contact:
        • Principal Investigator:
          • Usama Gergis
    • Ohio
      • Cleveland, Ohio, United States, 44195-0001
        • Recruiting
        • Cleveland Clinic
        • Contact:
        • Principal Investigator:
          • Moaath Mustafa Ali
    • Oregon
      • Portland, Oregon, United States, 97239-3011
        • Not yet recruiting
        • OHSU Knight Cancer Institute
        • Principal Investigator:
          • Jennifer Saultz
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37203-6521
        • Recruiting
        • Tri-Star BMT/Sarah Cannon Nashville
        • Contact:
        • Principal Investigator:
          • Stephen Strickland
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • MD Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Abhishek Maiti
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226-3522
        • Not yet recruiting
        • Medical College of Wisconsin
        • Contact:
        • Principal Investigator:
          • Karen Carlson

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: GDX012
GDX012 suspension for IV infusion.
Drug: Chemotherapy Agents
Chemotherapy agents (fludarabine/cyclophosphamide) as per standard of care.
Experimental: 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.
Drug: GDX012
GDX012 suspension for IV infusion.
Drug: Chemotherapy Agents
Chemotherapy agents (fludarabine/cyclophosphamide) as per standard of care.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Dose Limiting Toxicities (DLTs)
Time Frame: Up to 1 month
Up to 1 month
Maximum Tolerated Dose (MTD) of GDX012
Time Frame: Up to 1 month
Up to 1 month
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.
Up to 14 months

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Takeda

Investigators

  • Study Director: Study Director, Takeda

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 11, 2023

Primary Completion (Estimated)

April 1, 2026

Study Completion (Estimated)

June 30, 2027

Study Registration Dates

First Submitted

May 24, 2023

First Submitted That Met QC Criteria

May 24, 2023

First Posted (Actual)

June 2, 2023

Study Record Updates

Last Update Posted (Actual)

April 5, 2024

Last Update Submitted That Met QC Criteria

April 3, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TAK-012-1501

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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