Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AMELI-01)

Cellectis

Status and phase: Enrolling; Phase 1

Conditions: Relapsed/Refractory Acute Myeloid Leukemia

Treatments: Biological: UCART123v1.2

Study type: Interventional

Funder types: Industry

Identifiers

NCT03190278

UCART123_01

Details and patient eligibility

About

Phase I, open-label, dose-escalation and dose-expansion study evaluating the safety and efficacy of Universal Chimeric Antigen Receptor T-cell (UCART) targeting the Cluster of Differentiation 123 (CD123) in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of Universal Chimeric Antigen Receptor T-cells targeting CD123 (UCART123v1.2) and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).

Enrollment: 65 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Main Inclusion Criteria:

• Patients with relapsed or primary refractory AML (as defined in World Health Organization [WHO] criteria) with ≥5% bone marrow blasts
• Patients with CD123+ blast cells (verified by flow cytometry)
• Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of ≤1
• Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within screening period
• (Dose-escalation) Identified donor and transplant strategy prior to lymphodepletion (LD)
• Other criteria may apply

Main Exclusion Criteria:

• Patients with acute promyelocytic leukemia (APL) or central nervous system (CNS) Leukemia
• Previous investigation gene or cell therapy (including CAR)
• > 1 prior allogeneic stem cell transplantations (SCTs)
• Prior treatment with rituximab or other anti-cluster of differentiation 20 (anti-CD20) therapy within 3 months
• Any known active or uncontrolled infection
• Other criteria may apply

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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65 participants in 1 patient group

Dose Escalation

Experimental group

Description:

UCART123v1.2 tested at several dose levels with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the RP2D determined from the dose escalation phase

Treatment:

Biological: UCART123v1.2