AUTOLOGOUS T-CELLS EX VIVO MODIFIED WITH A LENTIVIRAL VECTOR ENCODING A CHIMERIC ANTIGEN RECEPTOR SPECIFIC FOR CD1A for T-cell acute lymphoblastic leukaemia|T-cell lymphoblastic lymphoma

Inclusion criteria

• {"criterion_text":"- Children older than 2 years or adults, male and female in both groups."}
• {"criterion_text":"- Patients CD1a antigen blast expression ≥20% at inclusion, either immunophenotypically (flow cytometry) or histologically confirmed."}
• {"criterion_text":"- R/R CD1a-positive T-ALL/LL patients, including morphologic or MRD-detectable (≥1x10-4) bone marrow and/or extramedullary relapses after 2 therapy lines: ­\tRelapse after allogeneic haematopoietic stem cell transplantation (allo-HSCT) ­\tPrimary refractoriness, defined as either morphologic persistence or detectable MRD (≥1x10-4) after two standard therapy lines, making the patient not candidate for allo-HSCT. ­\tRefractory first relapse. ­\tSecond or further relapse."}
• {"criterion_text":"- Patient without reproductive capacity or else, commitment to the use of a highly effective method of contraception during the study."}

Exclusion criteria

• {"criterion_text":"- Limiting organ dysfunction, such as uncontrolled cardiac (e.g., depressed left ventricular ejection fraction (LVEF), <45%), pulmonary, liver, renal or CNS dysfunction."}
• {"criterion_text":"- Other non-controlled concomitant neoplasms."}
• {"criterion_text":"- Allo-HSCT within a time frame <3 months, or requiring continued immunosuppressive treatment for graft versus host disease (GvHD)."}
• {"criterion_text":"- Uncontrolled epilepsy or underlying central nervous system (CNS) severe disease."}
• {"criterion_text":"- Active bacterial, fungal or viral infection not controlled by adequate treatment."}
• {"criterion_text":"- Known HIV, active hepatitis B (HBV), or hepatitis C virus (HCV) infection."}
• {"criterion_text":"- Women who are pregnant (urine/blood pregnancy test positive) or lactating."}
• {"criterion_text":"- Severe illness or medical condition, which would not permit the patient to be managed according to the protocol."}
• {"criterion_text":"- Suffering from a serious autoimmune disease or immunodeficiency disease"}
• {"criterion_text":"- The patient participated in other experimental drug clinical trial within 6 weeks prior to OC-1 infusion."}

Primary endpoints

• {"endpoint_text":"- Number of adverse events grade III-IV using Common Toxicity Criteria for Adverse Events (CTCAE) version 5.","definition_or_measurement_approach":"AEs graded using Common Toxicity Criteria for Adverse Events (CTCAE) version 5."}
• {"endpoint_text":"- Incidence of severe Cytokine release syndrome (CRS) # grade III and Immune effector cell-associated neurotoxicity syndrome (ICANS) # grade II","definition_or_measurement_approach":"Incidence measured by reporting of CRS and ICANS events graded per established grading scales (grades specified in endpoint)."}
• {"endpoint_text":"- Proportion of patients with non-relapse, treatment-related mortality (NRM)","definition_or_measurement_approach":"Proportion of patients with treatment-related mortality not due to disease relapse (NRM)."}
• {"endpoint_text":"- Number of adverse events of special interest (AESI)","definition_or_measurement_approach":"Count of adverse events predefined as AESI (events of special interest per protocol)."}
• {"endpoint_text":"- Assessment of the immunological homeostasis, through the description of lymphocytes subpopulations at each study timepoint.","definition_or_measurement_approach":"Description/measurement of lymphocyte subpopulations at each study timepoint (immunophenotyping)."}
• {"endpoint_text":"- Incidence of severe (#3) treatment-related dermatological events.","definition_or_measurement_approach":"Incidence of grade 3 treatment-related dermatological events as recorded and graded per CTCAE."}
• {"endpoint_text":"- Number of patients developing dose limiting toxicity (DLT)","definition_or_measurement_approach":"Count of patients meeting protocol-defined dose limiting toxicity (DLT) criteria."}

Secondary endpoints

• {"endpoint_text":"- Remission rate: Percentage of patients presenting complete response (CR) or incomplete count recovery (CRi) at any point after treatment","definition_or_measurement_approach":"Percentage of patients achieving CR or CRi at any time post-treatment."}
• {"endpoint_text":"- Duration of remission: The duration of the remission will be assessed from the first documented date of remission status until progression (in days)","definition_or_measurement_approach":"Duration measured in days from first documented remission to progression."}
• {"endpoint_text":"- Minimal residual disease (MRD) response by flow cytometry: blast count among patients presenting bone marrow complete response (sensitivity 10-4).","definition_or_measurement_approach":"MRD measured by flow cytometry with sensitivity 10^-4 in bone marrow CR patients."}
• {"endpoint_text":"- Progression-free survival: time since the first infusion to the documented loss of response. In patients not presenting a CR or CRi progression free survival will be zero","definition_or_measurement_approach":"Time from first infusion to documented loss of response; PFS = 0 for patients without CR/CRi."}
• {"endpoint_text":"- Overall survival time since first infusion to date of death","definition_or_measurement_approach":"Time from first infusion until date of death."}
• {"endpoint_text":"- Persistence of OC-1, as determined by flow cytometry and quantitative analysis by qPCR","definition_or_measurement_approach":"Persistence measured by flow cytometry and quantitative qPCR analysis."}

Spain

Earliest CTIS Part Ii Submission Date

12-07-2024

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

642

Number Of Sites

2

Number Of Participants

20

Primary sponsor

Full Name

Onechain Immunotherapeutics S.L.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Spain