International Phase II Studies of I-mIBG in Combination With Topotecan and Peripheral Blood Stem Cell Rescue for (A) Primary Resistant High Risk Neuroblastoma and (B) Relapsed Stage 4 Neuroblastoma
- Determine response (partial and complete response at metastatic sites) in children with
relapsed stage 4 neuroblastoma or primary resistant high-risk neuroblastoma treated
with high-dose iodine I 131 metaiodobenzylguanidine, topotecan hydrochloride, and
peripheral blood stem cell transplantation.
- Determine the proportion of patients who, as a result of this treatment, are able to
progress to potentially curative surgery and further systemic treatment.
- Correlate tumor dosimetry (to determine whether the tumor absorbed the radiation dose)
with response in patients treated with this regimen.
- Determine the time to tumor progression.
OUTLINE: This is an open-label, multicenter study. Patients are stratified according to
disease type (relapsed stage 4 vs primary resistant high-risk neuroblastoma).
Patients receive topotecan hydrochloride IV over 30 minutes on days 1-5 and 15-19 and
high-dose iodine I 131 metaiodobenzylguanidine (^131I-MIBG) IV over 30 minutes on days 1 and
15. Patients receive autologous CD 34+ peripheral blood stem cells when ^131I-MIBG dosimetry
levels reach an acceptable low on days 25-29.
Total whole-body absorbed dose is measured periodically after the first ^131I-MIBG dose is
administered and periodically thereafter.
After completion of study treatment, patients are followed periodically for up to 10 years.
PROJECTED ACCRUAL: A total of 67 patients will be accrued for this study.
Masking: Open Label, Primary Purpose: Treatment
Proportion of patients who respond to treatment (partial response and complete response at metastatic sites) as measured by metaiodobenzylguanidine scintigraphy and positron emission tomography and CT imaging
Mark N. Gaze, MD
University College London Hospitals