An Adaptive Open-label Multicentre Phase 1/2 Trial, to Determine the Recommended Phase 2 Dose of CCTx-001, and to Assess Safety, Tolerability, and Clinical Activity in Patients With Relapsed/Refractory Acute Myeloid Leukaemia

Advesya SAS6 sites in 4 countries143 target enrollmentDecember 1, 2025

ConditionsAcute Myeloid Leukemia, in Relapse Acute Myeloid Leukemia Refractory

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Acute Myeloid Leukemia, in Relapse
Sponsor: Advesya SAS
Enrollment: 143
Locations: 6
Primary Endpoint: Phase 1: To evaluate the safety, tolerability, and to define the recommended phase 2 dose (RP2D) of CCTx-001
Status: Not yet recruiting
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Registry

clinicaltrials.gov

Start Date

December 1, 2025

End Date

August 1, 2041

Last Updated

7 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Advesya SAS

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Patients with active (\> 5 % blasts in bone marrow) r/r AML (WHO 2022) who have exhausted their therapeutic alternatives or have contraindications to these alternatives as judged by the treating physician defined as either:
•a. Primary refractory: i. Patients who failed after two cycles of intensive induction including high-dose and/or standard dose cytarabine (including liposomal formulation), +/- anthracycline, +/- antimetabolite, +/- targeted therapy or ii. Older patients or patients unfit to receive intensive induction courses who failed after two cycles of venetoclax + azacitidine or 4 cycles of azacitidine b. Relapsing: i. Patients with early relapse after CR to first line therapy (within ≤ 6 months after CR1) or ii. Patients with relapse after later lines of therapy (Relapse after CR≥2) c. Patients relapsing after allogeneic hematopoietic stem cell transplant: i. Patients must be at least 3 months from hematopoietic stem cell transplant (HSCT) at the time of consent, and ii. Off immunosuppression for at least 1 month at the time of consent, and iii. Have no active graft versus host disease (GvHD)
•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
•Patient is ≥ 18 years of age at the time of informed consent
•Read, understood, and signed the informed consent form (ICF) prior to any study procedures
•Patient is willing and able to adhere to the study visit schedule and other protocol requirements
•Eligible for leukapheresis

Exclusion Criteria

•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
•Patients who underwent allo-HSCT within 90 days prior to leukapheresis
•Patients who received donor lymphocyte infusion within 60 days prior to leukapheresis
•Patients with active GvHD
•Patients with history of another primary malignancy other than disease under study unless the patient has been free of the disease for ≥ 2 years, except for the following non-invasive malignancies:
•Basal cell carcinoma of the skin
•Squamous cell carcinoma of the skin

Outcomes

Primary Outcomes

Phase 1: To evaluate the safety, tolerability, and to define the recommended phase 2 dose (RP2D) of CCTx-001

Time Frame: Up to 28 days

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

Time Frame: Up to 3 months

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).

Secondary Outcomes

Phase 2: To evaluate the clinical activity, as assessed by the complete remission rate, in patients treated with CCTx-001(Up to 3 months)
Phase 1: To evaluate the clinical activity, as assessed by the composite complete response rate, in patients treated with CCTx-001(Up to 3 months)
Phase 2: To assess the safety of CCTx-001(Up to 15 years)
Phase 1 & 2: To assess additional parameters of clinical activity in patients treated with CCTx-001(Up to 3 months)
Phase 1 & 2: To evaluate the overall safety and the tolerability of CCTx-001(Up to 15 years)