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).
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
Drug | Synonyms | Arms |
---|
UCART123v1.2 | | Part 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
Name | Type | Description | Interventions |
---|
Part 1: Dose Escalation | Experimental | Several 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 | |
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
May 11, 2021