CER-1236 in Patients With Acute Myeloid Leukemia (AML)

Phase 1

Recruiting

• Conditions: AML; Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia
• Interventions: Drug: CER-1236; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Mesna

• Registration Number: NCT06834282
• Lead Sponsor: CERo Therapeutics Holdings, Inc.

Brief Summary

This is a first in human, multi center, open label, phase 1/1b study to evaluate the safety and preliminary efficacy of CER-1236 in patients with relapsed/refractory (R/R), measurable residual disease (MRD) positive acute myeloid leukemia (AML), or TP53mut disease.

Detailed Description

CER-1236 is a first in class chimeric engulfment receptor T-cell therapy candidate that targets the Tim4 ligand.

This is a first in human, multi center, open label, phase 1/1b study to evaluate the safety and preliminary efficacy of CER-1236 in patients with relapsed/refractory (R/R), measurable residual disease (MRD) positive acute myeloid leukemia (AML), or TP53mut disease.

The study is divided into Part 1 (escalation phase) and Part 2 (expansion phase).

Part 1 (Escalation Phase): The primary objectives of Part 1 are to define the safety of different doses of CER-1236 and to define the recommended dose for Part 2 (RP2D) of CER-1236.

Part 2 (Expansion Phase): The objective of the Part 2 expansion cohort is to evaluate the safety and efficacy of CER-1236 in patients with acute myeloid leukemia.

Study Timeline

• Status Verified: 2025-02
• Study First Submitted: 2025-02-14
• Study First Submitted QC: 2025-02-14
• Study First Posted: 2025-02-19
• Study Start: 2025-04-07
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-04-30
• Primary Completion: 2026-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

• Patients need to have a confirmed diagnosis of de novo or secondary AML, or myelodysplastic syndrome (MDS)/AML with 10% to 19% blasts, per the International Consensus Classification 2022 or the WHO 2022 classification.
• Absolute lymphocyte count >0.3 x 109/L prior to apheresis.
• Eastern cooperative oncology group (ECOG) performance status 0 to 1.

Exclusion Criteria

• Prior therapy with a permanently integrated, genetically modified cell product.
• No measurable leukemia on the screening bone marrow evaluation prior to any bridging therapy.
• Active autoimmune disease or history of autoimmune disease requiring treatment within the prior 2 years. Patients with history of autoimmune thyroiditis or type 1 diabetes well controlled on replacement regimen are eligible.
• A known hypersensitivity or severe allergy to fludarabine, cyclophosphamide, or study drug components or diluents.
• Any other medical, psychological, or social condition that may interfere with study participation or compliance, or compromise patient safety in the opinion of the physician.
• Primary immunodeficiency disorder.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Part 1: Single Agent CER-1236	CER-1236	AML patient treated with a single dose of CER-1236 monotherapy
Part 1: Single Agent CER-1236	Cyclophosphamide	AML patient treated with a single dose of CER-1236 monotherapy
Part 1: Single Agent CER-1236	Fludarabine	AML patient treated with a single dose of CER-1236 monotherapy
Part 1: Single Agent CER-1236	Mesna	AML patient treated with a single dose of CER-1236 monotherapy
Part 2: Single Agent CER-1236	Cyclophosphamide	AML patient treated with a single dose of CER-1236 monotherapy
Part 2: Single Agent CER-1236	Fludarabine	AML patient treated with a single dose of CER-1236 monotherapy
Part 2: Single Agent CER-1236	Mesna	AML patient treated with a single dose of CER-1236 monotherapy
Part 2: Single Agent CER-1236	CER-1236	AML patient treated with a single dose of CER-1236 monotherapy

Primary Outcome Measures

Name	Time	Method
Incidence of adverse events (AEs) and serious adverse events (SAEs) - (Part 1)	2 year	Escalation Period
Incidence of dose-limiting toxicities (DLTs) of CER-1236 Monotherapy - (Part 1)	28 days	Escalation Period
Estimation of the objective response rate (ORR), complete response (CR), composite complete response (cCR), and measurable residual disease (MRD) negativity rates - (Part 2)	2 years	Expansion Period

Secondary Outcome Measures

Name	Time	Method
PK (AUC) of CER-1236 - (Part 1)	2 year	Escalation Period
Estimation of the objective response rate (ORR), complete response (CR), composite complete response (cCR), and measurable residual disease (MRD) negativity rates - (Part 1)	2 years	Escalation Period
Incidence of adverse events (AEs) and serious adverse events (SAEs) - (Part 2)	2 years	Expansion Period
PK (Cmax) of CER-1236 - (Part 1)	2 years	Escalation Period

Trial Locations

Locations (2)

Colorado Blood Cancer Institute; 🇺🇸 Denver, Colorado, United States

The University of Texas MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States