The primary objective of the trial is to determine the maximum tolerated dose (MTD) of Azedra in children with neuroblastoma. Secondary goals are to determine tumor and dose responses, determine toxicity, estimate normal organ dosimetry and describe the effect of Azedra therapy on overall quality of life. The Molecular Insight trial is being coordinated by the New Approaches to Neuroblastoma Therapy (NANT) Consortium, a group of 14 universities and Children’s Hospitals with strong research and treatment programs for neuroblastoma.
Azedra is a targeted radiotherapeutic comprised of the known I-131 MIBG molecule radiolabeled using Molecular Insight’s proprietary Ultratrace technology, which removes unnecessary nonradioactive molecules, or cold contaminants, to maximize therapeutic potential.
During the trial, approximately 24 patients across NANT’s 14 member sites will be scanned using an imaging dose of Azedra to determine if the imaging dose demonstrates normal MIBG biodistribution and tumor uptake. Cohorts of three to six patients will then receive one therapeutic dose of Azedra within 28 days of the MIBG biodistribution and tumor uptake scan. Disease and final toxicity evaluation will be performed approximately 60 days post therapy, as will a quality of life assessment.