A PHASE I/II STUDY TO INVESTIGATE THE SAFETY AND FEASIBILITY OF POINT-OF-CARE HUMAN-CD19 TARGETING CAR T-CELLS IN PAEDIATRIC AND YOUNG ADULT PATIENTS WITH RELAPSED OR REFRACTORY B-CELL MALIGNANCIES (PACMAN)

Condition studied

B-cell precursor ALL, B-cell Non Hodgkin Lymphoma

Investigational medicinal product(s)

Eligibility

Primary endpoint

Dose at which ≤ 1 patients experience a Dose Limiting Toxicity (DLT) within 28 days after CAR T-cell infusion

Endpoint detail

For B-ALL: the MRD-negative CR rate at day 28, For B-NHL: the overall response rate (ORR, CR +PR according to Lugano criteria) at day 90, Surrogate endpoint for efficacy: B-cell aplasia (<5 B-cells/µl) at day 28 after infusion, Number of days until relapse, Number of days from CAR T-cell infusion until recovery of B-cells (B-cell recovery is defined as peripheral blood ≥10 CD19+ B-cells/mcL OR ≥1% CD19+ B-cells in the bone marrow. Results must be confirmed on a subsequent test ≥2 weeks apart with timing of B-cell recovery defined as the date of the initial sample, Event free survival (EFS) at 6 and 12 months, Overall Survival (OS) at 6 and 12 months, Cumulative Incidence of Relapse at 6 and 12 months, The percentage of products fulfilling the release criteria