Access Program Information
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