Risk-Stratified Randomized Phase III Testing of Blinatumomab (IND#100135, NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)"

Sponsor: Children's Hospital of Philadelphia

Location(s): United States


A risk-stratified randomized phase III trial will test whether the incorporation of blinatumomab into a multi-agent chemotherapy regimen improves disease-free survival (DFS) for patients with a first relapse of B-lymphoblastic leukemia (B-ALL). Patients aged one to 30 years are risk-stratified based on the site and timing of relapse as well as response to a uniform first block of standard re-induction chemotherapy. Subjects are subsequently randomized 1:1 to receive a blinatumomab-containing multiagent chemotherapy regimen versus a control regimen consisting of a multiagent chemotherapy alone. Subjects with treatment failures are non-randomly assigned to receive blinatumomab, and eligible intermediate- and high-risk subjects proceed to hematopoietic stem cell transplantation (HSCT) following three treatment blocks (Figure). Secondary outcomes include rates of negative minimal residual disease (MRD), rates of second morphologic remission, proportion of patients who proceed to HSCT after treatment with blinatumomab, and safety and feasibility of rapid taper of immune suppression following HSCT in subjects with MRD ≥ 0.01 percent pre- and/or post-HSCT with no acute graft vs. host disease (GVHD).