Global Registration Trial of Efficacy and Safety of CTL019 in Pediatric and Young Adult Patients with Relapsed/Refractory (R/R) Acute Lymphoblastic Leukemia (ALL): Update to the Interim Analysis

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Context

CTL019 is an investigational anti-CD19 chimeric antigen receptor (CAR) T-cell therapy that reprograms cytotoxic T cells to eliminate target cells. A single-center trial demonstrated high response rates and a manageable safety profile in pediatric/young adult patients with R/R B-ALL. We report an updated analysis from the first multicenter, global, pivotal registration trial of CAR T-cell therapy (ELIANA; NCT02435849).

Design

This is a single-arm, open-label, phase 2 study of CTL019 in children/young adults with CD19+ R/R B-ALL with ≥5% bone marrow lymphoblasts. Primary endpoint: overall remission rate (complete remission [CR]+CR with incomplete blood count recovery [CRi]) within 3 months.

Results

At data cutoff (November 2016), 88 patients were enrolled: there were 7 manufacturing failures (8%), 9 patients (10%) were not infused due to death or AEs, and 4 (5%) pending infusion. Following lymphodepleting chemotherapy (fludarabine/cyclophosphamide [n=64]), other [n=1]), 68 patients received a single infusion of CTL019 (median dose, 3.0×106 [range, 0.2-5.4×106] cells/kg); median 8.8 months from infusion to data cutoff. Median age, 12y (range, 3-23y); 59% had prior allogeneic stem cell

Conclusion

ELIANA confirmed efficacy of a single infusion of CTL019, without additional therapy. AEs were effectively and reproducibly managed.

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