Clinical Trials /

Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia

NCT03190278

Description:

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

Related Conditions:
  • Acute Myeloid Leukemia
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

Title

  • Brief Title: Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia
  • Official 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

Clinical Trial IDs

  • ORG STUDY ID: UCART123_01
  • NCT ID: NCT03190278

Conditions

  • Relapsed/Refractory Acute Myeloid Leukemia

Interventions

DrugSynonymsArms
UCART123v1.2Part 1: Dose Escalation

Purpose

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

Trial Arms

NameTypeDescriptionInterventions
Part 1: Dose EscalationExperimentalSeveral tested doses of UCART123v1.2 with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the selected dose determined from the dose escalation phase
  • UCART123v1.2

Eligibility Criteria

        Main Inclusion Criteria:

          -  Patients with relapsed or primary refractory AML (as defined in 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 APL or CNS Leukemia

          -  Previous investigation gene or cell therapy (including CAR)

          -  ≥ 2 prior allogeneic SCTs

          -  Prior treatment with rituximab or other anti-CD20 therapy within 3 months

          -  Any known active or uncontrolled infection

          -  Other criteria may apply
      
Maximum Eligible Age:65 Years
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Incidence of AE/SAE/DLT [Safety and Tolerability]
Time Frame:24 Months
Safety Issue:
Description:Safety of UCART123v1.2 - Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study

Secondary Outcome Measures

Measure:Response Assessment
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
Safety Issue:
Description:
Measure: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
Safety Issue:
Description:
Measure: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
Safety Issue:
Description:
Measure:Overall Survival
Time Frame:From the first day of study treatment to the date of death from any cause, assessed up to Month 24
Safety Issue:
Description:

Details

Phase:Phase 1
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Cellectis S.A.

Trial Keywords

  • Acute Myeloid Leukemia
  • Relapsed/Refractory Acute Myeloid Leukemia
  • Chimeric Antigen Receptor T-Cell (CAR-T) therapy
  • Allogeneic
  • Transcription Activator-Like Effector Nuclease (TALEN)

Last Updated

February 5, 2021