Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia
Study ID: STU 052018-102
Before entering in the study protocol and according to their tumor burden and the investigator's judgment, patients could receive a cytoreductive treatment as part of the routine care, based on a dexamethasone-based debulking chemotherapy (if applicable). eligible patients following written informed consent signature will enter a Screening Period. Patients will then be included and will start the Lymphodepletion treatment period, which will take place from Day -7 to Day -2 during the week preceding uCaRT19 infusion at Day 0. The patients could be hospitalized, according to the local practices per country, from 7 days before the administration of uCaRT19 (D-7) or the day before the uCaRT19 administration (D-1). The lymphodepletion regimen combines fludarabine and cyclophosphamide. at the end of the lymphodepletion regimen, eligibility criteria allowing uCaRT19 administration should be assessed in order to ensure patients' safety. The Treatment Period (starting at the time of the investigational Medicinal Product (iMP) administration at D0) will end at Day 84 (D84) post uCaRT19 infusion. Patients will be closely monitored during the 9-month Follow-up Period until the end of Study (eoS) at Month 12 after uCaRT19 infusion. at the end of the 12-month follow up period post uCaRT19 infusion or at time of premature discontinuation, patients will participate in a separate long term follow-up (LTFu) study and will be followed for 15 years.
Patient with relapsed or refractory CD19-positive B-acute lymphoblastic leukaemia (B-ALL) who have exhausted alternative treatment options.
Estimated life expectancy ≥ 12 weeks
Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at time of assent/consent) performance status ≥ 50
CD19-negative B-cell leukemia
Active Central Nervous System (CNS) leukemia
Active acute or chronic Graft-versus-Host Disease (GvHD) requiring systemic use therapy
objective: This study aims to evaluate the safety and feasibility of uCaRT19 to induce molecular remission in paediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-aLL). Primary objective: To evaluate the safety of uCaRT19 in paediatric patients with relapsed or refractory B-aLL. Secondary objective: To determine the ability of uCaRT19 to achieve molecular remission at D28 after the first uCaRT19 infusion.