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NCT Number: NCT03190278

Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

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).

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Key information

Conditions

Relapsed/Refractory Acute Myeloid Leukemia Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

UCART123v1.2

Biological

Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor Biological/vaccine: CLLS52 A monoclonal antibody that recognizes the CD52 antigen Other Names: Alemtuzumab

Primary outcomes

  1. Incidence of adverse events (AE)/serious adverse events (SAE)/Dose Limiting Toxicities (DLT) [Safety and Tolerability]

    Time frame: 24 Months

    Safety of UCART123v1.2 - Incidence, nature, and severity of AE and SAEs throughout the study

  2. Dose escalation and expansion part: Occurrence of DLTs

    Time frame: Up to Day 28 post last UCART123v1.2 infusion

Secondary outcomes

  1. Investigators assessed overall response rate according to the European Leukemia Net (ELN) Response Criteria

    Time frame: At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24

  2. Duration of Response

    Time frame: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

  3. Progression Free Survival

    Time frame: From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24

  4. Overall Survival

    Time frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24

  5. Pharmacokinetic (PK) Analysis: Standard PK Analysis will be completed to obtain Maximum plasma concentration (Cmax)

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  6. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain time to reach Cmax (Tmax)

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  7. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain total area under curve from zero to infinity (AUC-infinity)

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  8. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Rate

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  9. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Terminal Half-life

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  10. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Clearance

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  11. Pharmacokinetic Analysis: Standard PK Analysis will be completed to obtain Volume of Distribution

    Time frame: alemtuzumab levels will be determined pre- and post-dose alemtuzumab and up to 48 hours after the last dose

  12. Pharmacodynamic Analysis: Pharmacodynamics Monitoring of the incidence of anti-cluster of differentiation 52 (anti-CD52; alemtuzumab) antibodies (ADA) in serum Pre-alemtuzumab administration and through Day 84

    Time frame: From screening through Day 84

  13. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of T cells in peripheral blood

    Time frame: From screening through Day 84

  14. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of B cells in peripheral blood

    Time frame: From screening through Day 84

  15. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of natural killer (NK) cells in peripheral blood

    Time frame: From screening through Day 84

  16. Pharmacodynamic Analysis: Pharmacodynamics Quantitation of total lymphocytes in peripheral blood

    Time frame: From screening through Day 84

Sponsors and collaborators

Lead sponsor

Cellectis S.A.

Industry

Registry information

Official study title

Phase I, Open Label Dose Escalation and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia

Acronym: AMELI-01

Important dates

Study start
2017
Primary completion
2024
Study completion
2025
First posted
Jun 16, 2017
Registry last updated
Aug 7, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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