TRANSPOCART19 for acute lymphoblastic leukemia (CD19+) | refractory or resistant acute lymphoblastic leukemia

Inclusion criteria

• {"criterion_text":"- Diagnosis of CD19+ acute lymphoblastic leukemia, with a prognosis of less than 2 years due to meeting one of the following conditions: ● Relapsed/refractory, not a transplant candidate (due to refractory disease or lack of a donor) ● Relapse after allogeneic transplant"}
• {"criterion_text":"- Measurable disease, understood as the presence of at least residual measurable disease by flow cytometry in bone marrow or peripheral blood at the time of inclusion."}
• {"criterion_text":"- Age over 4 years and under 70 years."}
• {"criterion_text":"- ECOG performance status 0-1. Patients with ECOG 2 may be included if due to hematologic disease (Appendix 1)."}
• {"criterion_text":"- Life expectancy greater than 3 months."}
• {"criterion_text":"- Adequate venous access for lymphapheresis. No contraindications."}
• {"criterion_text":"- Signing of informed consent (patient or legal guardian)."}

Exclusion criteria

• {"criterion_text":"- Patients who, in the opinion of their physician, may benefit from other approved, potentially curative therapeutic options, including commercial CAR-T cells."}
• {"criterion_text":"- HIV infecction"}
• {"criterion_text":"- Concurrent and uncontrolled medical conditions including cardiac, renal, hepatic, gastrointestinal, endocrine, pulmonary, neurological, or psychiatric conditions that, in the opinion of the investigator, pose a risk to the patient."}
• {"criterion_text":"- Positive serology for hepatitis B, defined as a positive test for HBsAg. Additionally, if the patient is HBsAg negative but has anti-HBc antibodies, a hepatitis B virus DNA test will be required. If the result is positive, the patient will be excluded."}
• {"criterion_text":"- Positive serology for hepatitis C, defined as a positive test for anti-HCV antibodies confirmed by RIBA."}
• {"criterion_text":"- Severe organ involvement, defined as cardiac ejection fraction <40%; DLCO <40%; estimated glomerular filtration rate <30 ml/min; bilirubin >3 times the upper limit of normal (unless due to Gilbert's syndrome)."}
• {"criterion_text":"- Pregnant or breastfeeding women. Women of childbearing age must have a negative pregnancy test during the screening phase."}
• {"criterion_text":"- Women of childbearing potential, including those whose last menstrual cycle was in the year prior to screening, who are unable or unwilling to use highly effective contraception* from baseline to completion."}
• {"criterion_text":"- Men who are unable or unwilling to use highly effective contraception* from the start of the study until its completion."}
• {"criterion_text":"- Treatment with any experimental or non-marketed substance within four weeks prior to enrollment, or actively participating in another therapeutic clinical trial."}
• {"criterion_text":"- Previous treatment with CART (commercial or experimental)."}
• {"criterion_text":"- Diagnosis of another malignancy, past or present. Patients who have been in complete remission for more than 3 years, or with a history of non-melanoma skin cancer or completely resected carcinoma in situ, may be included."}
• {"criterion_text":"- Overt central nervous system involvement (CNS-3) at the time of inclusion. Patients with a lesser degree (CNS-2) or CNS-3 who have responded to intrathecal chemotherapy will be eligible for inclusion."}
• {"criterion_text":"- Isolated extramedullary involvement (i.e., in the absence of residual measurable disease in peripheral blood or bone marrow)."}
• {"criterion_text":"- In the case of relapse after allogeneic transplantation, at least 3 months must have passed since the transplant for inclusion. Furthermore, the patient must have been off systemic immunosuppressants for at least 30 days to be eligible for inclusion."}
• {"criterion_text":"- Active immunosuppressive therapy for graft-versus-host disease and other conditions. The use of corticosteroids for leukemia control should be limited as much as possible at the time of enrollment and should be discontinued before the infusion of TranspoCART19 cells."}
• {"criterion_text":"- Active infection requiring systemic medical treatment."}

Primary endpoints

• {"endpoint_text":"- To determine the maximum tolerated dose and evaluate the safety of TranspoCART19 cell infusion.","definition_or_measurement_approach":""}
• {"endpoint_text":"- To determine the efficacy of TranspoCART19 cell infusion","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Toxicity assessment at 3 months and 1 year, defined as the number of grade II-IV adverse events using the CTC (Common Toxicity Criteria) version 5.0 (Annex 4) and the ASTCT classification.","definition_or_measurement_approach":"Number of grade II-IV adverse events using CTC v5.0 and ASTCT classification assessed at 3 months and 1 year."}
• {"endpoint_text":"- Treatment-related mortality (TRM) at 1, 3, and 1 year, defined as any death not directly caused by leukemia. For the purpose of estimating TRM, disease relapse or progression will be considered a competing event.","definition_or_measurement_approach":"TRM defined as any death not directly caused by leukemia; relapse/progression considered competing events; assessed at 1 month, 3 months, and 1 year."}
• {"endpoint_text":"- Progression-free survival (PFS) at six months and one year after the procedure, defined as the time between TranspoCART19 infusion and disease progression or death. Patients alive and in complete remission will be censored at the time of last follow-up.","definition_or_measurement_approach":"Time from infusion to disease progression or death; censoring at last follow-up for surviving patients in complete remission; assessed at 6 months and 1 year."}
• {"endpoint_text":"- Overall survival (OS) at one year after infusion, defined as the time between TranspoCART19 infusion and the patient's death from any cause. Surviving patients will be censored at the time of last follow-up.","definition_or_measurement_approach":"Time from infusion to death from any cause; censoring at last follow-up; assessed at 1 year."}
• {"endpoint_text":"- Response rate (overall and complete) at three months and one year.","definition_or_measurement_approach":"Overall and complete response rates assessed at 3 months and 1 year."}
• {"endpoint_text":"- Best response rate achieved during the first 3 months of follow-up after administration of the first fraction of TranspoCART19.","definition_or_measurement_approach":"Best response within first 3 months after first administration of TranspoCART19."}

Spain

Earliest CTIS Part Ii Submission Date

02-12-2025

Latest Decision Or Authorization Date

02-01-2026

Processing Time Days

31

Number Of Sites

1

Number Of Participants

24

Primary sponsor

Full Name

Clinica Universidad De Navarra

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Spain