SC-4002: An Open Label, Expanded Access Protocol Using 1311-Metaiodobenzylguanidine (111_MIBG) Therapy in Patients with Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma.
SC-4002
What is the goal of the study?
Neuroblastoma, pheochromocytoma, and paraganglioma remain fatal diseases for a large percentage of patients, especially those with high-risk disease features who become resistant to conventional therapy. 131I-metaiodobenzylguanidine (131I-MIBG) is a norepinephrine analog that concentrates in adrenergic tissue and has been shown to be sensitive and specific for detecting localized and metastatic neuroblastoma, pheochromocytoma, and paraganglioma. More importantly, experience of many institutions has proven that this agent used as a targeted radiotherapeutic has significant anti-tumor activity against refractory neuroblastoma as well as pheochromocytoma and paraganglioma. Children's Hospital of Philadelphia, UCSF, and the University of Michigan have just completed a large Phase 2 study of 131I-MIBG given in doses of 10-18 mCi/kg with stem cell rescue, if necessary, and have shown that this agent is safe and effective palliative therapy for refractory or relapsed neuroblastoma patients. In addition, there is growing evidence that low-dose (5-10 mCi/kg) submyeloablative MIBG therapy is both safe and effective for disease palliation. This protocol therefore provides a mechanism to deliver this therapy when clinically indicated, but also to provide a mechanism to continue to collect safety data that will be provided to the Food and Drug Administration as described herein. Primary Objectives: ? Provide palliative therapy with MIBG for patients with advanced neuroblastoma, pheochromocytoma, or paraganglioma ? Gain more information about acute and late toxicity of 131I-MIBG therapy for patients with refractory neuroblastoma, pheochromocytoma, or paraganglioma
Who can participate in the study?
Please contact the study team listed below to learn more.