Study Evaluating Safety and Efficacy of UCART123v1.2 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AMELI-01)
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ClinicalTrials.gov Identifier: NCT03190278 |
Recruitment Status :
Recruiting
First Posted : June 16, 2017
Last Update Posted : February 5, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Relapsed/Refractory Acute Myeloid Leukemia | Biological: UCART123v1.2 | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 65 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | June 19, 2017 |
Estimated Primary Completion Date : | December 2024 |
Estimated Study Completion Date : | December 2024 |
Arm | Intervention/treatment |
---|---|
Experimental: Dose Escalation
UCART123v1.2 tested at several dose levels with different lymphodepletion regimens to establish Maximum Tolerated Dose (MTD) and identify Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART123v1.2 administered at the RP2D determined from the dose escalation phase |
Biological: UCART123v1.2
Allogeneic engineered T-cells expressing anti-CD123 Chimeric Antigen Receptor Biological/vaccine: CLLS52 A monoclonal antibody that recognizes the CD52 antigen Other Names: Alemtuzumab |
- 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
- Dose escalation and expansion part: Occurrence of DLTs [ Time Frame: Up to Day 28 post last UCART123v1.2 infusion ]
- 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 ]
- 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 ]
- 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 ]
- Overall Survival [ Time Frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- Pharmacodynamic Analysis: Pharmacodynamics Quantitation of T cells in peripheral blood [ Time Frame: From screening through Day 84 ]
- Pharmacodynamic Analysis: Pharmacodynamics Quantitation of B cells in peripheral blood [ Time Frame: From screening through Day 84 ]
- Pharmacodynamic Analysis: Pharmacodynamics Quantitation of natural killer (NK) cells in peripheral blood [ Time Frame: From screening through Day 84 ]
- Pharmacodynamic Analysis: Pharmacodynamics Quantitation of total lymphocytes in peripheral blood [ Time Frame: From screening through Day 84 ]
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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03190278
Contact: Cellectis Central Contact | 1-347-752-4044 | clinicaltrials@cellectis.com |
United States, California | |
University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center | Recruiting |
San Francisco, California, United States, 94143 | |
United States, Florida | |
Sylvester Comprehensive Cancer Center | Withdrawn |
Miami, Florida, United States, 33136 | |
H. Lee Moffitt Cancer Center & Research Institute | Recruiting |
Tampa, Florida, United States, 33612 | |
United States, Illinois | |
Northwestern University | Recruiting |
Chicago, Illinois, United States, 60201 | |
United States, Massachusetts | |
Dana-Farber Cancer Institute | Recruiting |
Boston, Massachusetts, United States, 02215 | |
United States, New York | |
Roswell Park Cancer Institute | Recruiting |
Buffalo, New York, United States, 14263 | |
Weill Medical College of Cornell University | Recruiting |
New York, New York, United States, 10021 | |
United States, Pennsylvania | |
University of Pennsylvania - Abramson Cancer Center | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Texas | |
MD Anderson Cancer Center | Recruiting |
Houston, Texas, United States, 77030 |
Principal Investigator: | Gail Roboz, Dr | Weill Medical College of Cornell University |
Responsible Party: | Cellectis S.A. |
ClinicalTrials.gov Identifier: | NCT03190278 |
Other Study ID Numbers: |
UCART123_01 |
First Posted: | June 16, 2017 Key Record Dates |
Last Update Posted: | February 5, 2024 |
Last Verified: | January 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Acute Myeloid Leukemia Relapsed/Refractory Acute Myeloid Leukemia Chimeric Antigen Receptor T-Cell (CAR-T) therapy Allogeneic Transcription Activator-Like Effector Nuclease (TALEN) |
Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute |
Neoplasms by Histologic Type Neoplasms Hematologic Diseases |