NCT01838187

Expanded Access Protocol Using 131I-MIBG Therapy +/- Vorinostat for Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma

An Open Label, Expanded Access Protocol Using 131I-METAIODOBENZYLGUANIDINE (131I-MIBG) Therapy +/- Vorinostat in Patients With Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 12 Months
• Healthy volunteers: —

Summary

Currently there is no known effective treatment for patients with advanced stage neuroblastoma who have relapsed or not responded to standard therapy. There is also no known effective treatment for patients with pheochromocytoma or paraganglioma who are less than 12 years of age. In previous studies that used 131I-MIBG as a potential anti-cancer therapy, a decrease in the size of tumors was seen in some of the children and adults. This research study will continue to evaluate the side effects of 131I-MIBG +/- Vorinostat when treating children and adults with neuroblastoma, pheochromocytoma, or paraganglioma. The 131I-MIBG compound is intended to work by selectively delivering the radioactive iodine to the tumor cells, which is then intended to result in their destruction. The purpose of this research study is to: * Make 131I-MIBG therapy available to patients with advanced neuroblastoma, pheochromocytoma, or paraganglioma * Further assess the side effects of 131I-MIBG therapy

Detailed description

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 1-7 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. In addition, a recently published randomized phase 2 trial in patients with relapsed/refractory neuroblastoma showed that the combination of vorinostat/MIBG had a higher response rate than 131I-MIBG alone, with manageable toxicity. This protocol therefore provides a mechanism to deliver this therapy when clinically indicated. Primary Objectives: * Provide palliative therapy with MIBG + vorinostat for patients with advanced neuroblastoma who can take vorinostat * Provide palliative therapy with MIBG without vorinostat for patients with advanced neuroblastoma who cannot take vorinostat, and those with pheochromocytoma, or paraganglioma * Gain more information about acute and late toxicity of 131I-MIBG therapy +/- vorinostat for patients with refractory neuroblastoma, and 131I-MIBG therapy alone for those with pheochromocytoma, or paraganglioma

Conditions

• Neuroblastoma
• Pheochromocytoma
• Paraganglioma

Interventions

Type	Name	Description
RADIATION	I-131 MIBG	The therapeutic dose (5-18 mCi/kg at investigator's discretion; any dose ≥12 mCi/kg requires stored stem cells) will be diluted in normal saline, and will be infused intravenously over 90-120 minutes.
DRUG	Vorinostat	180 mg/m2 orally once daily on days 0 to +13

Timeline

• First posted: 2013-04-23
• Last updated: 2023-08-18

Locations

1 site across 1 country: United States