The purpose of this adaptive Phase 1/2 study is to evaluate the safety, tolerability, pharmacokinetics (PK), and antileukemic activity of CCTx-001 in adult patients with r/r Acute Myeloid Leukemia (AML). CCTx-001 targets IL-1RAP, which is specifically expressed in leukemic cells. In preclinical studies, IL-1RAP-targeted Chimeric antigen receptors (CARs) have demonstrated encouraging activity in both in vitro and in vivo experiments in AML models. Based on these promising preclinical results, it is expected that CCTx-001 could potentially alter the natural course of r/r AML and provide a potential novel treatment option.
Have a circulating blast count of less than 20,000/mm3 (control with hydroxyurea is allowed)
Absolute Lymphocyte count of >200/mm3
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
Life expectancy of more than 3 months
Patients with an acute promyelocytic leukaemia: t(15;17)(q22;q12); (promyelocytic leukaemia/retinoic acid receptor alpha) and variants
Patients with active central nervous system (CNS) leukaemia involvement. If the patient has prior history of CNS leukaemia, they must have a negative cerebrospinal fluid (CSF) assessment and magnetic resonance imaging (MRI) or computed tomography (if MRI is not feasible) of the brain demonstrating no evidence of CNS disease
Patients with isolated extramedullary AML disease
Patients who received previous treatment targeting IL-1RAP or previous gene therapy
Single group
CCTx-001
GeneticFrozen CAR T-cells suspensions in media containing dimethyl sulfoxide (DMSO)
Phase 1: To evaluate the safety, tolerability, and to define the recommended phase 2 dose (RP2D) of CCTx-001
Frequency, severity, relationship and persistence of adverse events (AEs) and dose-limiting toxicity (DLTs)
Phase 2: To evaluate the clinical activity, as assessed by the composite complete response rate, in patients treated with CCTx-001
Composite complete response rate (cCRR) defined as the proportion of patients with best overall response, as assessed by Independent Review Committee (IRC) based on European LeukemiaNet (ELN) 2022 criteria, at complete remission (CR), CR with partial haematologic recovery (CRh), or CR with incomplete haematologic recovery (CRi).
Phase 2: To evaluate the clinical activity, as assessed by the complete remission rate, in patients treated with CCTx-001
Complete remission rate (CRR) defined as the proportion of patients with best overall response, as assessed by IRC based on ELN 2022 criteria, at CR.
Phase 1: To evaluate the clinical activity, as assessed by the composite complete response rate, in patients treated with CCTx-001
cCRR
Phase 2: To assess the safety of CCTx-001
Frequency, severity, relationship and persistence of AEs
Phase 1 & 2: To assess additional parameters of clinical activity in patients treated with CCTx-001
Objective response rate (ORR)
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