Disease Detail


Missing content? – Request curation!

Request curation for specific Genes, Variants, or PubMed publications.

Have questions, comments, or suggestions? - Let us know!

Email us at : ckbsupport@jax.org


  • Case insensitive filtering will display rows if any text in any cell matches the filter term
  • Use simple literal full or partial string matches
  • Separate multiple filter terms with a space. Any order may be used (i. e. a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page. Filtering has no impact on query parameters.
  • Use quotes to match on a longer phrase with spaces (i.e. "mtor c1483f")


  • Generally, the default sort order for tables is set to be first column ascending; however, specific tables may set a different default sort order.
  • Click on any column header arrows to sort by that column
  • Hold down the Shift key and click multiple columns to sort by more than one column. Be sure to set ascending or descending order for a given column before moving on to the next column.

Molecular Profile Therapy Indication/Tumor Type Response Type Profile Response Detail
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT00843037 Phase II Sunitinib Study Of Sunitinib In Patients With Recurrent Paraganglioma/Pheochromocytoma Completed NLD | CAN 0
NCT01371201 Phase II Sunitinib First International Randomized Study in Malignant Progressive Pheochromocytoma and Paraganglioma (FIRSTMAPPP) Completed NLD | ITA | FRA | DEU 0
NCT01396408 Phase II Temsirolimus Sunitinib A Phase II Study of Sunitinib or Temsirolimus in Patients With Advanced Rare Tumours Active, not recruiting CAN 0
NCT01838187 Expanded access Vorinostat Expanded Access Protocol Using 131I-MIBG Therapy +/- Vorinostat for Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma No longer available USA 0
NCT01967576 Phase II Axitinib Phase II Study of Axitinib (AG-013736) With Evaluation of the VEGF-pathway in Metastatic, Recurrent or Primary Unresectable Pheochromocytoma/Paraganglioma Completed USA 0
NCT02302833 Phase I Cabozantinib Cabozantinib for Malignant Pheochromocytoma Active, not recruiting USA 0
NCT02721732 Phase II Pembrolizumab Study for the Evaluation of Efficacy of Pembrolizumab (MK-3475) in Patients With Rare Tumors Active, not recruiting USA 0
NCT02834013 Phase 0 Ipilimumab + Nivolumab Nivolumab and Ipilimumab in Treating Patients With Rare Tumors Active, not recruiting USA 1
NCT02936323 Phase Ib/II PEN-221 PEN-221 in Somatostatin Receptor 2 Expressing Advanced Cancers Including Neuroendocrine and Small Cell Lung Cancers Completed USA | GBR 0
NCT03008369 Phase II Lenvatinib Lenvatinib in Treating Patients With Metastatic or Advanced Pheochromocytoma or Paraganglioma That Cannot Be Removed by Surgery Completed USA 0
NCT03165721 Phase II Guadecitabine A Phase II Trial of the DNA Methyl Transferase Inhibitor, Guadecitabine (SGI-110), in Children and Adults With Wild Type GIST,Pheochromocytoma and Paraganglioma Associated With Succinate Dehydrogenase Deficiency and HLRCC-associated Kidney Cancer Terminated USA 0
NCT03839498 Phase II Axitinib Study of Axitinib (AG-013736) With Evaluation of the VEGF-pathway in Pheochromocytoma/Paraganglioma Recruiting USA 0
NCT04106843 Phase II lutetium Lu 177 dotatate Radioactive Drug (177Lu-DOTATATE) for the Treatment of Locally Advanced, Metastatic, or Unresectable Rare Endocrine Cancers Withdrawn USA 0
NCT04187404 Phase Ib/II EO2401 + Nivolumab A Novel Therapeutic Vaccine (EO2401) in Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/Paraganglioma (Spencer) Active, not recruiting USA | NLD | ITA | FRA | ESP | DEU 2
NCT04394858 Phase II Olaparib + Temozolomide Temozolomide Testing the Addition of an Anticancer Drug, Olaparib, to the Usual Chemotherapy (Temozolomide) for Advanced Neuroendocrine Cancer Recruiting USA 0
NCT04400474 Phase II Atezolizumab + Cabozantinib Trial of Cabozantinib Plus Atezolizumab in Advanced and Progressive Neoplasms of the Endocrine System. The CABATEN Study (CABATEN) Active, not recruiting ESP 0
NCT04614766 Phase Ib/II 131I-MIBG + lutetium Lu 177 dotatate lutetium Lu 177 dotatate A Clinical Trial Evaluating the Safety of Combining Lutathera(R) and Azedra(R) to Treat Mid-gut Neuroendocrine Tumors (SPORE-3) Withdrawn USA 0
NCT04924075 Phase II Belzutifan Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL) or Pancreatic Neuroendocrine Tumor (pNET) (MK-6482-015) Recruiting USA | NLD | ITA | GBR | FRA | ESP | DEU | CAN | AUS 9
NCT05142241 Phase II Talazoparib + Temozolomide Testing the Combination of Anti-Cancer Drugs Talazoparib and Temozolomide in Patients >= 18 Years Old With Advanced Stage Rare Cancers, RARE 2 Study Recruiting USA 0
NCT06427798 Phase Ib/II 212Pb-VMT-alpha-NET Somatostatin-Receptors (SSTR)-Agonist [212Pb]VMT-alpha-NET in Metastatic or Inoperable SSTR+ Gastrointestinal Neuroendocrine Tumor and Pheochromocytoma/Paraganglioma Previously Treated With Systemic Targeted Radioligand Therapy Not yet recruiting USA 0
NCT06479811 Phase I 212Pb-VMT-alpha-NET [212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers Not yet recruiting USA 0