Expanded Access Protocol - Blinatumomab in Pediatric & Adolescent Subjects With Relapsed/Refractory B-precursor ALL

Summary

Primary Objective: To estimate the incidence of treatment-emergent and treatment-related adverse events during treatment with blinatumomab in pediatric and adolescent subjects with B-precursor ALL in second or later bone marrow relapse, in any marrow relapse after alloHSCT, or refractory to other treatments Secondary Objective(s): To describe key efficacy outcomes, including incidence of complete response (CR) within 2 cycles of blinatumomab, minimal residual disease (MRD) remission within 2 cycles of blinatumomab, relapse free survival (RFS), overall survival (OS), incidence of alloHSCT, and 100-day mortality after alloHSCT. Hypotheses: A formal statistical hypothesis will not be tested. The incidence of treatment-emergent and treatment-related adverse events will be estimated. Study Endpoints: * Incidence of treatment-emergent and treatment-related adverse events * Incidence of CR within 2 cycles of blinatumomab * MRD remission within 2 cycles of blinatumomab * RFS * OS * Incidence of alloHSCT * 100-day mortality after alloHSCT Study Design: Multi-center, open-label, single-arm expanded access protocol

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Publications

• Background Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Kormany WN, Viswagnachar P, Chen-Santel C. Blinatumomab in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia: RIALTO expanded access study final analysis. Blood Adv. 2022 Feb 8;6(3):1004-1014. doi: 10.1182/bloodadvances.2021005579.
• Background Locatelli F, Zugmaier G, Mergen N, Bader P, Jeha S, Schlegel PG, Bourquin JP, Handgretinger R, Brethon B, Rossig C, Chen-Santel C. Blinatumomab in pediatric patients with relapsed/refractory acute lymphoblastic leukemia: results of the RIALTO trial, an expanded access study. Blood Cancer J. 2020 Jul 24;10(7):77. doi: 10.1038/s41408-020-00342-x. No abstract available.
• Background Queudeville M, Stein AS, Locatelli F, Ebinger M, Handgretinger R, Gokbuget N, Gore L, Zeng Y, Gokani P, Zugmaier G, Kantarjian HM. Low leukemia burden improves blinatumomab efficacy in patients with relapsed/refractory B-cell acute lymphoblastic leukemia. Cancer. 2023 May 1;129(9):1384-1393. doi: 10.1002/cncr.34667. Epub 2023 Feb 24.

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