What side effects are associated with this type of intervention?

Common treatments for Acute Myeloid Leukemia (AML) include chemotherapy, hypomethylating agents like decitabine and azacitidine, and targeted therapies such as venetoclax. Known side effects of these treatments can include myelosuppression, leading to increased risk of infection, anemia, and bleeding. Gastrointestinal symptoms like nausea, vomiting, and diarrhea are also common. Specific to CAR T cell therapy, which is similar to the PRGN-3006 trial, side effects can include cytokine release syndrome (CRS), neurotoxicity, and prolonged cytopenias. CRS can cause fever, hypotension, and respiratory distress, while neurotoxicity can lead to confusion, seizures, and encephalopathy.

What prior FDA approvals exist?

The FDA has approved several treatments for Acute Myeloid Leukemia (AML), including hypomethylating agents like azacitidine and decitabine, which are used for patients unfit for intensive chemotherapy. Targeted therapies such as enasidenib for IDH2 mutations and ivosidenib for IDH1 mutations have also been approved. While CAR T cell therapies are a promising area of research, specific CAR T cell treatments for AML, like the PRGN-3006 trial, have not yet received FDA approval. The focus remains on developing and evaluating these advanced therapies in clinical trials.

What other types of research exist?

Promising novel alternatives to PRGN-3006 for AML patients include: 1) Venetoclax in combination with hypomethylating agents or low-dose cytarabine, which targets BCL-2 and has shown efficacy in elderly or unfit patients. 2) FLT3 inhibitors like gilteritinib for patients with FLT3 mutations, which have demonstrated improved survival rates. 3) IDH1 and IDH2 inhibitors (ivosidenib and enasidenib) for patients with IDH1 or IDH2 mutations, respectively, offering targeted treatment options. 4) Bispecific T-cell engagers (BiTEs) such as AMG 330, which target CD33 and recruit T-cells to AML cells. These alternatives are in various stages of clinical development and have shown promising results in early trials.

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CAR T-cell Therapy

CAR T Cell Therapy for AML and MDS

Phase 1

Waitlist Available

Led By David A Sallman, MD

Research Sponsored by Precigen, Inc

Eligibility Criteria Checklist

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

Must have

Karnofsky performance status score ≥60%

Participants must be diagnosed with either relapsed or refractory AML (including extramedullary disease), MRD-positive AML, or higher risk MDS

Must not have

Participants requiring agents other than hydroxyurea to control blast counts within 14 days of study enrollment

Diagnosis of acute promyelocytic leukemia (APL M3): t(15;17)(q22;q12); (promyelocytic leukemia [PML]/retinoic acid receptor [RAR] alpha [a]) and variants excluded

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 12 months post treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment that uses modified immune cells to target and kill cancer cells in adults with difficult-to-treat or high-risk blood cancers.

Who is the study for?

Adults with relapsed or refractory acute myeloid leukemia (AML), MRD-positive AML, or high-risk MDS can join. They must have a certain level of physical fitness, organ function, and no need for oxygen support. Participants should not be pregnant and must agree to contraception. Those who've had bone marrow transplants are eligible if they meet specific conditions.

What is being tested?

The trial is testing PRGN-3006 T Cells, which are modified immune cells designed to target and kill cancer cells in patients with certain types of blood cancers like AML and MDS.

What are the potential side effects?

Potential side effects may include reactions related to the immune system's response to the modified T cells, such as fever, fatigue, inflammation in various organs, or symptoms similar to an infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can care for myself but may need occasional help.

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My condition is either a relapse or resistant AML, MRD-positive AML, or high-risk MDS.

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My kidney function, measured by creatinine clearance, is adequate.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need medication other than hydroxyurea to manage my blood cell counts before joining the study.

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My leukemia is specifically diagnosed as acute promyelocytic leukemia.

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I do not have any serious ongoing infections or heart, lung, mental health issues that would stop me from following the study's requirements.

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I have previously received CAR T therapy targeting CD33.

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I am taking high doses of immunosuppressants for an autoimmune disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 12 months post treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 12 months post treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 months post treatment 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 who Experience Dose Limiting Toxicities (DLTs)

Number of Participants who Experience Treatment Emergent Adverse Events (TEAEs)

Secondary study objectives

Absolute Lymphocyte Count (ALC)

Disease Progression in AML Participants

Disease Response in MDS Patients

+2 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Dose Escalation and Dose Expansion of PRGN-3006Experimental Treatment1 Intervention

Participants will be treated in dose expansion phase to evaluate the safety and efficacy of the identified dose of PRGN-3006.

0 / 8Eligibility criteria met

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 Acute Myeloid Leukemia (AML) include chemotherapy, targeted therapy, and immunotherapy. Chemotherapy, such as cytarabine and daunorubicin, works by killing rapidly dividing cells, including cancer cells. Targeted therapies, like FLT3 inhibitors, specifically target genetic mutations in cancer cells, thereby inhibiting their growth and survival. Immunotherapy, including CAR T-cell therapy like PRGN-3006, involves modifying a patient's own T cells to recognize and attack AML cells expressing specific proteins, such as CD33. These mechanisms are crucial for AML patients as they offer tailored approaches to eradicate cancer cells, potentially leading to better outcomes and fewer side effects compared to traditional treatments.

CAR T-cells targeting FLT3 have potent activity against FLT3-ITD+ AML and act synergistically with the FLT3-inhibitor crenolanib.