Back to resultsPhase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia
Primary Purpose
Acute Myeloid Leukaemia
Status
Withdrawn
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
UCART123
Sponsored by
About this trial
This is an interventional treatment trial for Acute Myeloid Leukaemia focused on measuring Acute Myeloid Leukaemia, Newly diagnosed AML, ELN adverse genetic risk group, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
Eligibility Criteria
Main Inclusion Criteria:
- Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined as per ELN guidelines (Döhner et al., 2017)
- Eastern Cooperative Oncology Group performance status of 0 or 1
- No prior gene or experimental cellular therapy
- No organ dysfunction that in the opinion of the investigator precludes intensive induction chemotherapy or cellular therapy
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Dose escalation
Arm Description
Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified.
Outcomes
Primary Outcome Measures
Incidence of AE/SAE/DLT [Safety and Tolerability]
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04106076
Brief Title
Phase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia
Official Title
Phase I, Open Label Dose-escalation Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of Multiple Infusions of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor) in Patients With Adverse Genetic Risk Acute Myeloid Leukaemia
Study Type
Interventional
2. Study Status
Record Verification Date
July 2020
Overall Recruitment Status
Withdrawn
Why Stopped
The trial was discontinued due to sponsor's decision and not a consequence of any safety concern
Study Start Date
July 11, 2019 (Actual)
Primary Completion Date
December 5, 2019 (Actual)
Study Completion Date
December 5, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cellectis S.A.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a Phase I, open-label, dose escalation study of UCART123 administered intravenously to patients with newly diagnosed CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined in the ELN adverse genetic risk group (2017). The purpose of this study is to evaluate the safety and clinical activity of multiple infusions of UCART123 and to determine the Maximum Tolerated Dose (MTD).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Acute Myeloid Leukaemia
Keywords
Acute Myeloid Leukaemia, Newly diagnosed AML, ELN adverse genetic risk group, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dose escalation
Arm Type
Experimental
Arm Description
Several tested doses of UCART123 until the Maximum Tolerated Dose (MTD) is identified.
Intervention Type
Biological
Intervention Name(s)
UCART123
Intervention Description
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor
Primary Outcome Measure Information:
Title
Incidence of AE/SAE/DLT [Safety and Tolerability]
Description
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria:
Patients newly diagnosed with CD123 positive adverse genetic risk acute myeloid leukaemia (AML) defined as per ELN guidelines (Döhner et al., 2017)
Eastern Cooperative Oncology Group performance status of 0 or 1
No prior gene or experimental cellular therapy
No organ dysfunction that in the opinion of the investigator precludes intensive induction chemotherapy or cellular therapy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ghulam Mufti, Pr
Organizational Affiliation
Kings college London NHS Foundation Trust
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Phase I Study of UCART123 in Patient With Adverse Genetic Risk Acute Myeloid Leukemia
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