What side effects are associated with this type of intervention?

Common treatments for leukemia, particularly those involving cell therapy and immune system stimulation like GDX012, often include side effects such as cytokine release syndrome (CRS), which can cause fever, nausea, headache, rash, rapid heartbeat, low blood pressure, and trouble breathing. Other potential side effects include neurotoxicity, leading to confusion, difficulty speaking, and seizures. Additionally, patients may experience infections due to immunosuppression, as well as general side effects like fatigue, anemia, and thrombocytopenia.

What prior FDA approvals exist?

The FDA has approved several treatments for leukemia that involve cell therapy or immune system stimulation. One notable example is CAR-T cell therapy, such as tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (Yescarta), which are engineered to target specific antigens on leukemia cells. These therapies have shown significant efficacy in treating certain types of leukemia, particularly acute lymphoblastic leukemia (ALL) and diffuse large B-cell lymphoma (DLBCL). Additionally, immune checkpoint inhibitors like pembrolizumab (Keytruda) and nivolumab (Opdivo) have been approved for various cancers, including some forms of leukemia, by enhancing the immune system's ability to recognize and destroy cancer cells.

What other types of research exist?

<ol> <li>T22-DITOX-H6: A CXCR4-targeted nanocarrier that delivers toxins specifically to CXCR4+ lymphoma cells, showing antitumor effects without systemic toxicity.</li> </ol> This could be a promising option for certain types of leukemia expressing CXCR4. 2. Venetoclax: An orally available BCL2 inhibitor effective in combination with low-dose cytarabine (LoDAC) for initial treatment of AML, particularly for patients who are not candidates for intensive therapy. 3. Ivosidenib: A mutant-IDH1 inhibitor approved for advanced acute myeloid leukemia, showing good tolerance and potential efficacy in clinical trials. 4. Moxetumomab pasudotox: A treatment option for patients with hairy cell leukemia resistant to multiple therapies, offering a targeted approach with promising results.

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CAR T-cell Therapy

GDX012 for Acute Myeloid Leukemia

Phase 1 & 2

Recruiting

Research Sponsored by Takeda

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 14 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing GDX012, a new cell therapy, in adult patients with Acute Myeloid Leukemia (AML). The goal is to see how safe and tolerable the treatment is and to find the best dose. GDX012 works by using specially modified cells to attack cancer cells.

Who is the study for?

Adults with a body weight of at least 40 kg who have relapsed or refractory Acute Myeloid Leukemia (AML) and are not eligible for stem cell transplantation can join. They should be in relatively good health otherwise, with an expected lifespan over 3 months and able to perform daily activities with little or no assistance.

What is being tested?

The trial is testing GDX012, a new cell therapy for AML. It aims to find out how safe the treatment is, how well patients tolerate it, and determine the best dose when combined with chemotherapy agents.

What are the potential side effects?

While specific side effects of GDX012 aren't listed here, common side effects from similar treatments include immune system reactions, fatigue, fever, digestive issues and potential risks associated with chemotherapy such as hair loss and increased infection risk.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 14 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 14 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 14 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Participants With Adverse Events (AEs)

Secondary study objectives

Duration of Response (DOR)

Event-free Survival (EFS)

Number of Participants With Disease Response

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Phase 2a: GDX012Experimental Treatment2 Interventions

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.

Group II: Phase 1: Dose Escalation of GDX012Experimental Treatment2 Interventions

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

First Studied

Drug Approval Stage

How many patients have taken this drug

Chemotherapy Agents

2021

Completed Phase 2

~20

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for leukemia include chemotherapy, targeted therapy, and cell therapy. Chemotherapy uses drugs to kill rapidly dividing cells, including cancer cells, but can also affect healthy cells. Targeted therapy focuses on specific genetic mutations or proteins in cancer cells, minimizing damage to normal cells. Cell therapy, such as the investigational GDX012, involves modifying a patient's own immune cells to better recognize and attack cancer cells. This approach can provide a more precise and potent attack on leukemia cells while potentially reducing side effects. For leukemia patients, these treatments are crucial as they offer different strategies to combat the disease, improve survival rates, and enhance quality of life by targeting the cancer more effectively and sparing healthy tissues.

Epigenetic therapy: azacytidine and decitabine in acute myeloid leukemia.