Safety and efficacy of post-haematopoietic cell transplantation maintenance therapy with blinatumomab for relapsed/refractory CD19-positive B-cell acute lymphoblastic leukaemia: protocol for a phase I–II, multicentre, non-blinded, non-controlled trial (JPLSG SCT-ALL-BLIN21)
Yoshiyuki Takahashi,
Hisashi Noma,
Akiko Saito,
Keizo Horibe,
Hirotoshi Sakaguchi,
Katsustugu Umeda,
Itaru Kato,
Kimiyoshi Sakaguchi,
Hidefumi Hiramatsu,
Hiroyuki Ishida,
Hiromasa Yabe,
Hiroaki Goto,
Yuta Kawahara,
Yuka Iijima Yamashita,
Masashi Sanada,
Takao Deguchi,
Takashi Taga,
Souichi Adachi
Affiliations
Yoshiyuki Takahashi
Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
Hisashi Noma
3 Department of Data Science, The Institute of Statistical Mathematics, Tachikawa, Tokyo, Japan
Akiko Saito
Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan
Keizo Horibe
Clinical Research Center, NHO Nagoya Medical Center, Nagoya, Japan
Hirotoshi Sakaguchi
Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan
Katsustugu Umeda
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan
Itaru Kato
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan
Kimiyoshi Sakaguchi
Department of Pediatrics, Hamamatsu University School of Medicine, Hamamatsu, Japan
Hidefumi Hiramatsu
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan
Hiroyuki Ishida
Department of Pediatrics, Kyoto City Hospital, Kyoto, Japan
Hiromasa Yabe
Department of Innovative Medical Science, Tokai University School of Medicine, Isehara, Japan
Hiroaki Goto
Division of Hematology/Oncology, Kanagawa Children`s Medical Center, Yokohama, Japan
Yuta Kawahara
Department of Pediatrics, Jichi Medical University School of Medicine, Shimotsuke, Japan
Yuka Iijima Yamashita
Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan
Masashi Sanada
Clinical Research Center, National Hospital Organization Nagoya Medical Center, Nagoya, Japan
Takao Deguchi
Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan
Takashi Taga
Department of Pediatrics, Shiga University of Medical Science, Otsu, Japan
Souichi Adachi
Human Health Science, Kyoto University, Kyoto, Japan
Introduction Relapsed and refractory B-cell acute lymphoblastic leukaemia (R/R-B-ALL) is linked to a significant relapse rate after allogeneic haematopoietic cell transplantation (allo-HCT) in children, adolescents and young adults (CAYA). No standard treatment has been established to prevent relapse after allo-HCT for R/R-B-ALL, which is an unmet medical need. The administration of blinatumomab after allo-HCT is expected to enhance the antileukaemic effect on residual CD19-positive blasts by donor-derived CD3-positive T-cells.Methods and analysis The goal of this multicentre, open-label, uncontrolled, phase I–II clinical trial is to assess the safety and effectiveness of post-transplant maintenance therapy with blinatumomab for CAYA patients (25 years old or younger) with CD19-positive R/R-B-ALL who have received allo-HCT beyond first complete remission (CR) and have CR with haematological recovery between 30 and 100 days after allo-HCT. Eighty-five paediatric institutions in Japan are participating in this study. Forty-one patients will enrol within 2.25-year enrolment period and follow-up period is 1 year. The primary endpoints are the treatment completion rate for phase I study and the 1-year graft-versus-host disease-free/relapse-free survival rate for phase II study, respectively.Ethics and dissemination This research was approved by the Central Review Board at National Hospital Organization Nagoya Medical Center (Nagoya, Japan) on 21 January 2022 and was registered at the Japan Registry of Clinical Trials (jRCT) on 3 March 2022. Written informed consent is obtained from all patients and/or their guardians. The results of this study will be disseminated through peer-reviewed publications and conference presentations.Trial registration number jRCTs041210154.
