Cabozantinib in patients with unresectable and progressive metastatic phaeochromocytoma or paraganglioma (the Natalie Trial): a single-arm, phase 2 trial

• Published in: The lancet oncology Vol. 25; no. 5; pp. 658 - 667
• Main Authors: Jimenez, Camilo, Habra, Mouhammed Amir, Campbell, Matthew T, Tamsen, Gina, Cruz-Goldberg, Damaris, Long, James, Bassett, Roland, Dantzer, Robert, Balderrama-Brondani, Vania, Varghese, Jeena, Lu, Yang
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.05.2024; Elsevier Limited
• Subjects: Adrenal Gland Neoplasms - drug therapy; Adrenal Gland Neoplasms - secondary; Adult; Adverse events; Aged; Angiogenesis; Anilides - adverse effects; Anilides - therapeutic use; Antihypertensives; Blood pressure; Bones; Cancer therapies; Catecholamines; Chemotherapy; Clinical medicine; Clinical trials; Drug dosages; FDA approval; Female; Hematology, Oncology, and Palliative Medicine; Humans; Hypertension; Hypoxia; Inhibitor drugs; Kinases; Male; Metastases; Metastasis; Middle Aged; Paraganglioma; Paraganglioma - drug therapy; Paraganglioma - pathology; Patients; Phenotypes; Pheochromocytoma; Pheochromocytoma - drug therapy; Pheochromocytoma - genetics; Pheochromocytoma - pathology; Plasma; Protein Kinase Inhibitors - adverse effects; Protein Kinase Inhibitors - therapeutic use; Pyridines - adverse effects; Pyridines - therapeutic use; Radiation therapy; Response rates; Review boards; Solid tumors; Targeted cancer therapy; Toxicity; Tumors; United States > US

Metastatic phaeochromocytomas and paragangliomas (MPPGs) are orphan diseases. Up to 50% of MPPGs are associated with germline pathogenic variants of the SDHB gene. These tumours and many non-familial MPPGs exhibit a phenotype that is characterised by abnormal angiogenesis. We aimed to assess the act...