Table 1.

Current clinical trials including agents used for patients with metastatic PPGL.

Agent	Eligible Patients	Phase	NCT#	Primary Outcome
I131-MIBG	Refractory neuroblastoma/metastatic PPGL	I	NCT03649438	Number of patients who receive MIBG
I131-MIBG	Refractory neuroblastoma or malignant PPGL	II	NCT00107289	Response rate
Lanreotide	Metastatic PPGL	II	NCT03946527	Tumor growth measurement
Sunitinib	Metastatic PPGL	II	NCT00843037	Clinical benefit rate1
Cabozantinib	Metastatic PPGL	II	NCT02302833	Response rate
Axitinib	Metastatic PPGL	II	NCT03839498	Response rate
Axitinib	Metastatic PPGL	II	NCT01967576	Response rate
Lenvatinib	Metastatic/advanced unresectable PPGL	II	NCT03008369	Response rate
ONC201	Neuroendocrine tumors	II	NCT03034200	Response rate
Temozolomide +/- Olaparib	Neuroendocrine tumor	II	NCT04394858	Progression Free Survival
Pembrolizumab	Rare unresectable or metastatic tumors	II	NCT02721732	Non-progression rate2

I¹³¹-MIBG, ¹³¹meta-iodo-benzyl-guanidine; PPGL, pheochromocytoma and paraganglioma. ¹Clinical benefit rate is defined as either a partial response, complete response or stable disease for more than 12 weeks using Response Evaluation Criteria in Solid Tumors. ²Defined as the proportion of subjects in the analysis population who have no progression of disease at 27 weeks.