This Phase I/IIa clinical study is testing an experimental treatment called GYA01 (CART84) for people with acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia (T-ALL) whose disease has come back after treatment (relapsed) or did not respond to treatment (refractory).
GYA01 (CART84) is a type of CAR T-cell therapy. In this approach, a participant's own T cells (a type of immune cell) are collected and changed in a laboratory to help them better recognize and attack leukemia cells. The modified cells GYA01 (CART84) are then given back to the participant through an infusion into a vein.
The study is being done to:
Find a dose that can be given safely (Phase I) by treating small groups of participants with increasing dose levels and carefully monitoring side effects.
Look for early signs that GYA01 (CART84) may help control AML or T-ALL (Phase IIa).
Participants will be closely monitored for side effects and for changes in their leukemia after the infusion, and followed over time to understand safety and possible benefit.
Age 18 years or older at the time of signing the informed consent.
Willing and able to give written, informed consent to the current study.
Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
Primary refractory disease (not achieving CR/CRi after more than two cycles of induction chemotherapy).
Isolated extramedullary (EM) disease.
Females who are pregnant or lactating.
For T-ALL patients: Patients with T-ALL exhibiting CD4+ and/or CD8+ immunophenotypes with detectable blasts in peripheral blood.
History or presence of clinically relevant CNS pathology, such as epilepsy, paresis, aphasia, stroke within 3 months prior to enrollment, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome, uncontrolled mental illness, or psychosis.
Single group
CART84
DrugGYA01 (CART84): autologous cell-based product containing CD3+ T cells transduced with a lentiviral vector expressing an anti-CD84 chimeric antigen receptor (CAR).
Number of Participants with Adverse Events.
Primary Outcome Measure Phase I and Secondary Outcome Measure Phase II: Frequency and severity of adverse events (AEs) and serious AEs (SAEs) occurring after CART84 infusion using the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 and American Society for Transplantation and Cellular Therapy (ASTCT) grading for CRS/ICANS.
Proportion of patients achieving ORR after first infusion of CART84
Primary Outcome Measure Phase II and Secondary Outcome Measure Phase I: Proportion of patients achieving ORR at day 30 after first infusion of CART84 as assessed by European Leukemia Net (ELN) 2022 guidelines
Proportion of patients achieving late ORR
Phases I \& II: Proportion of patients achieving late ORR, defined as a response occurring after day 30 and up to day 90, provided that no other therapy has been administered
Proportion of patients achieving measurable residual disease (MRD)-negative remission in bone marrow
Phase I: Proportion of patients achieving measurable residual disease (MRD)-negative remission in bone marrow (BM) by polymerase chain reaction (PCR) and/or flow cytometry at day 30.
Proportion of CART84-infused patients undergoing allo-HSCT
Phases I \& II: Proportion of CART84-infused patients undergoing allo-HSCT and achieving hematopoietic donor engraftment.
Proportion of patients with detectable CART84 cells
Phase I \& II: Proportion of patients with detectable CART84 cells by PCR in peripheral blood and/or bone marrow (BM) at day 30 and day 90 following CART84 infusion, comparing those who proceed to allo-HSCT versus those who do not at the last follow-up.
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Gyala Therapeutics
Lead sponsor
Astrum CRO, S.L.
Collaborator
