Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia (AMELI-01)
Primary Purpose
Relapsed/Refractory Acute Myeloid Leukemia
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
UCART123v1.2
Sponsored by
About this trial
This is an interventional treatment trial for Relapsed/Refractory Acute Myeloid Leukemia focused on measuring Acute Myeloid Leukemia, Relapsed/Refractory Acute Myeloid Leukemia, Chimeric Antigen Receptor T-Cell (CAR-T) therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN)
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
Sites / Locations
- University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer CenterRecruiting
- Sylvester Comprehensive Cancer CenterRecruiting
- H. Lee Moffitt Cancer Center & Research InstituteRecruiting
- Northwestern UniversityRecruiting
- Dana-Farber Cancer InstituteRecruiting
- Roswell Park Cancer InstituteRecruiting
- Weill Medical College of Cornell UniversityRecruiting
- University of Pennsylvania - Abramson Cancer CenterRecruiting
- MD Anderson Cancer CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Part 1: Dose Escalation
Arm Description
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
Outcomes
Primary Outcome Measures
Incidence of AE/SAE/DLT [Safety and Tolerability]
Safety of UCART123v1.2 - Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
Secondary Outcome Measures
Response Assessment
Duration of Response
Progression Free Survival
Overall Survival
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03190278
Brief Title
Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia
Acronym
AMELI-01
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2022
Overall Recruitment Status
Recruiting
Study Start Date
June 19, 2017 (Actual)
Primary Completion Date
March 2023 (Anticipated)
Study Completion Date
March 2023 (Anticipated)
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
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Relapsed/Refractory Acute Myeloid Leukemia
Keywords
Acute Myeloid Leukemia, Relapsed/Refractory Acute Myeloid Leukemia, 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
65 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Part 1: Dose Escalation
Arm Type
Experimental
Arm Description
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
Intervention Type
Biological
Intervention Name(s)
UCART123v1.2
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
Safety of UCART123v1.2 - Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study
Time Frame
24 Months
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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
Title
Overall Survival
Time Frame
From the first day of study treatment to the date of death from any cause, assessed up to Month 24
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 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Cellectis Central Contact
Phone
1-347-752-4044
Email
clinicaltrials@cellectis.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gail Roboz, Dr
Organizational Affiliation
Weill Medical College of Cornell University
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Individual Site Status
Recruiting
Facility Name
Sylvester Comprehensive Cancer Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Name
H. Lee Moffitt Cancer Center & Research Institute
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Individual Site Status
Recruiting
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60201
Country
United States
Individual Site Status
Recruiting
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Recruiting
Facility Name
Roswell Park Cancer Institute
City
Buffalo
State/Province
New York
ZIP/Postal Code
14263
Country
United States
Individual Site Status
Recruiting
Facility Name
Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Pennsylvania - Abramson Cancer Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia
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