ANBL1531: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)
Despite recent improvements in outcome for children with newly diagnosed high-risk neuroblastoma, cure rates remain unsatisfactory. Further, these gains have been the result of interventions during the Consolidation (tandem autologous stem cell transplant) and Post-Consolidation (dinutuximab immunotherapy) phases of treatment, while rates of disease control during Induction have not improved in recent COG trials. The current phase 3 trial seeks to improve the event-free survival (EFS) for children with high-risk neuroblastoma through early integration of promising novel targeted therapies: targeted radiopharmaceutical therapy with 131I-MIBG or the ALK inhibitor, crizotinib. After enrollment, patients will receive one cycle of Induction chemotherapy. Subsequent therapy will be based upon MIBG avidity and ALK status. Patients with MIBG-avid, ALK wild type (or ALK unknown) disease will be randomized to one of three arms: A) current COG recommended high-risk therapy including four more cycles of Induction chemotherapy and surgical resection of the primary tumor, Consolidation with tandem transplant and focal external beam radiation, and dinutuximab immunotherapy with isotretinoin; B) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle; or C) current COG recommended high-risk therapy with the addition of a block of 131I-MIBG after the third Induction cycle and substitution of busulfan / melphalan (BuMel) single autologous stem cell transplant in place of tandem transplant. Patients with MIBG non-avid, ALK wild type (or ALK unknown) disease will be non-randomly assigned to receive current COG recommended high-risk therapy without the addition of 131I-MIBG. Patients with ALK aberrant tumors (ALK tyrosine kinase mutation or ALK amplification) will be non-randomly assigned to receive crizotinib added to current COG recommended high-risk therapy. The primary endpoint is EFS and 774 eligible and evaluable patients are anticipated to enroll over approximately 5 years. Key secondary endpoints are toxicity, end-Induction response, and overall survival. Late effects of therapy including targeted therapies will be compared with late effects of current COG recommended treatment. Embedded correlative studies seek to understand predictors of benefit and resistance to 131I-MIBG and crizotinib.
Andrew W. Brotman, MD | NYU Langone Health
Andrew W. Brotman is senior advisor to CEO and Dean Alec C Kimmelman, MD, PhD.
Anesthesiology Doctors | NYU Langone Health
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Angelman Syndrome Clinic | NYU Langone Health
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Angina with No Blockage | NYU Langone Health
NYU Langone doctors diagnose and treat chest discomfort (angina) with no blockage, also known as ischemia with no obstructive coronary artery disease (INOCA).
Anita Saltz Institute for Anxiety & Mood Disorders | NYU Langone Health
The Anita Saltz Institute for Anxiety and Mood Disorders at NYU Langone’s Child Study Center treats depression in children and adolescents.
Annette Johnson, JD, PhD | NYU Langone Health
Annette Johnson, JD, PhD, is executive vice president and vice dean, general counsel, at NYU Langone.
Anomalous Aortic Origin of a Coronary Artery (AAOCA): Establishing a Multi-Institutional Registry
This is a retrospective and longitudinal cohort study. Subjects will be enrolled either when identified from medical records or when diagnosed with AAOCA. Baseline demographics, diagnoses, and test results will be obtained through retrospective chart review. Follow-up health-related information will be obtained through annual questionnaire. The project will be carried out at several participating CHSS member institutions, with the data stored and analyzed at the CHSS Data Center.
Anosmia Center | NYU Langone Health
NYU Langone’s Anosmia Center are experts at diagnosing and treating anosmia, or loss of smell.
Anosmia Center Doctors | NYU Langone Health
Find a doctor at the Anosmia Center at NYU Langone.