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

Leukemia Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05886491
• Other study ID #: TAK-012-1501
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 11, 2023
• Est. completion date: June 30, 2027

Study information

• Verified date: May 2024
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 53
• Est. completion date: June 30, 2027
• Est. primary completion date: April 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute

Intervention

Drug:
• GDX012
GDX012 suspension for IV infusion.
• Chemotherapy Agents
Chemotherapy agents (fludarabine/cyclophosphamide) as per standard of care.

Locations

Country	Name	City	State
United States	University of Alabama at Birmingham (UAB) Hospital	Birmingham	Alabama
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	Roswell Park Comprehensive Cancer Center	Buffalo	New York
United States	Comprehensive Cancer Center of Northwestern University	Chicago	Illinois
United States	Cleveland Clinic	Cleveland	Ohio
United States	Sarah Cannon/CBCI	Denver	Colorado
United States	City of Hope	Duarte	California
United States	MD Anderson Cancer Center	Houston	Texas
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Tri-Star BMT/Sarah Cannon Nashville	Nashville	Tennessee
United States	Thomas Jefferson University	New York	New York
United States	Stanford University	Palo Alto	California
United States	OHSU Knight Cancer Institute	Portland	Oregon
United States	Washington University	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Dose Limiting Toxicities (DLTs)		Up to 1 month
Primary	Maximum Tolerated Dose (MTD) of GDX012		Up to 1 month
Primary	Number of Participants With Adverse Events (AEs)	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	Number of Participants With Disease Response	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
Secondary	Number of Participants With Measurable Residual Disease (MRD) Negative Status as Determined by Flow Cytometry		Up to 14 months
Secondary	Duration of Response (DOR)	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
Secondary	Event-free Survival (EFS)	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
Secondary	Overall Survival (OS)	OS is defined as the time from the date of the first GDX012 administration to the date of death.	Up to 14 months