First in humans, exploratory, open-label, single-arm, multicentre, non-competitive, dose escalation study to assess the safety and efficacy of CD1a-CAR T therapy in patients with relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LL)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Autologous T-cells from peripheral blood, expanded and transduced with a lentivirus to express CD1a chimeric antigen receptor administered by intravenous infusion following a dose-escalation approach
Hospital Clínic
Barcelona, Spain
RECRUITINGHospital Sant Joan de Déu
Barcelona, Spain
RECRUITINGNumber of adverse events grade III-IV
Number of adverse events grade III-IV using common toxicity criteria (CTC)
Time frame: 1 year particularly the first 28 days after infusion
Incidence of severe Cytokine release syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS)
Incidence of severe Cytokine release syndrome (CRS) (≥ grade III) and Immune effector cell-associated neurotoxicity syndrome (ICANS) (≥ grade II)
Time frame: 1 year particularly the first 28 days after infusion
Non-relapse treatment-related mortality (NRM)
Non-relapse treatment-related mortality (NRM)
Time frame: 1 year
Number of adverse events of special interest (AESI)
Number of adverse events of special interest (AESI)
Time frame: 1 year
Assessment of the immunological homeostasis
Assessment of the immunological homeostasis, through the identification of lymphocytes subpopulations by flow cytometry at each study timepoint.
Time frame: 1 year
Incidence of the treatment-related dermatological events
Incidence of the treatment-related dermatological events
Time frame: 1 year
Number of patients developing dose limiting toxicity (DLT)
Number of patients developing dose limiting toxicity (DLT)
Time frame: first 28 days after infusion
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Remission rate
Percentage of patients presenting complete response (CR) or incomplete count recovery (CRi) at any point after treatment.
Time frame: 1 year
Response rates
Percentage of patients presenting CR, CRi, Complete remission duration (CRD), morphologic leukaemia-free status (MLFS), and no remission (NR).
Time frame: 1 year
Duration of remission
The duration of the remission will be assessed from the first documented date of remission status until progression (in days)
Time frame: 1 year
Minimal residual disease (MRD) response
Minimal residual disease (MRD) response by flow cytometry: blast count among patients presenting bone marrow complete response (sensitivity 10-4).
Time frame: 1 year
Progression-free survival (PFS)
time since the first OC-1 administration to the documented loss of response.
Time frame: 1 year
Overall survival
Overall survival time since first OC-1 administration to date of death.
Time frame: 1 year
Persistence of OC-1
• Persistence of OC-1, as determined by flow cytometry and quantitative analysis by qPCR. Genomic copy number integrations of the CAR in peripheral blood (PB) T cells and percentage of CD1a CAR-expressing T cells.
Time frame: 1 year