| Principal Investigator . | Clinical Trial ID . | Year . | Patients . | Phase . | Agent . | Target . | PFS (Mos.) . | RR (PR+CR) (%) . |
|---|---|---|---|---|---|---|---|---|
| Emily Chew | NCT00056199 | 2003–2005 | 5 | 1 | Pegaptanib | VEGF | N/A | 20% (rHBL*) |
| Novartis | NCT00052013 | 2003–2006 | 11 | 2 | Vatalanib | RTK | N/A | 0% |
| Adrian L. Harris | N/A | 2004 | 6 | 1,2 | Semaxanib | VEGF | N/A | 0% |
| Emily Chew | NCT00089765 | 2004–2007 | 5 | 1 | Ranibizumab | VEGF | N/A | 20% (rHBL*) |
| William M. Linehan | NCT00088374 | 2004–2009 | 9 | 2 | Tanespimycin | HSP-90 | N/A | 0% |
| Eric Jonasch | NCT00330564 | 2006–2011 | 15 | 2 | Sunitinib | RTK | N/A | 33% (RCC), 0% (HBL) |
| Catherine Meyerle | NCT00673816 | 2008–2011 | 3 | 1,2 | Sunitinib | RTK | N/A | 0% |
| William M. Linehan | NCT00566995 | 2008–2015 | 37 | 2 | Vandetanib | RTK | (11.0 to 22.1) | 8% (RCC) |
| J. Marc Pipas | NCT01015300 | 2009–2012 | 1 | 1 | Bevacizumab | VEGF | N/A | 0% |
| Stephane Richard | NCT01168440 | 2010–2011 | 5 | 2 | Sunitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01266070 | 2012–2015 | 6 | 2 | Dovitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01436227 | 2012–2021 | 31 | 2 | Pazopanib | RTK | N/A | 42% (ORR), 52% (RCC), 53% (pNETs), 4% (HBL) |
| Kevin D. Courtney | NCT03108066 | 2014–2016 | 51 | 1 | PT2385 | HIF-2α | 53% at 52 Weeks | 14% (RCC) |
| Prashant Chittiboina | NCT02108002 | 2014–2018 | 7 | 0 | Vorinostat | HDAC | N/A | 0% |
| Henry E. Wiley | NCT02859441 | 2016–2020 | 3 | 1,2 | E10030 and Ranibizumab | PDGF, VEGF | N/A | 0% |
| Eric Jonasch | NCT03401788 | 2016–2020 | 95 | 1 | Belzutifan | HIF-2α | 14.5 Months | 25% (RCC) |
| Kevin D. Courtney | NCT03108066 | 2017–2022 | 4 | 2 | PT2385 | HIF-2α | N/A | N/A |
| Eric Jonasch | NCT03401788 | 2018–2026 | 61 | 2 | Belzutifan | HIF-2α | 98% at 52 Weeks | 47% (ORR), 80% (pNETs), 32% (HBL), 69% (rHBL*) |
| Principal Investigator . | Clinical Trial ID . | Year . | Patients . | Phase . | Agent . | Target . | PFS (Mos.) . | RR (PR+CR) (%) . |
|---|---|---|---|---|---|---|---|---|
| Emily Chew | NCT00056199 | 2003–2005 | 5 | 1 | Pegaptanib | VEGF | N/A | 20% (rHBL*) |
| Novartis | NCT00052013 | 2003–2006 | 11 | 2 | Vatalanib | RTK | N/A | 0% |
| Adrian L. Harris | N/A | 2004 | 6 | 1,2 | Semaxanib | VEGF | N/A | 0% |
| Emily Chew | NCT00089765 | 2004–2007 | 5 | 1 | Ranibizumab | VEGF | N/A | 20% (rHBL*) |
| William M. Linehan | NCT00088374 | 2004–2009 | 9 | 2 | Tanespimycin | HSP-90 | N/A | 0% |
| Eric Jonasch | NCT00330564 | 2006–2011 | 15 | 2 | Sunitinib | RTK | N/A | 33% (RCC), 0% (HBL) |
| Catherine Meyerle | NCT00673816 | 2008–2011 | 3 | 1,2 | Sunitinib | RTK | N/A | 0% |
| William M. Linehan | NCT00566995 | 2008–2015 | 37 | 2 | Vandetanib | RTK | (11.0 to 22.1) | 8% (RCC) |
| J. Marc Pipas | NCT01015300 | 2009–2012 | 1 | 1 | Bevacizumab | VEGF | N/A | 0% |
| Stephane Richard | NCT01168440 | 2010–2011 | 5 | 2 | Sunitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01266070 | 2012–2015 | 6 | 2 | Dovitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01436227 | 2012–2021 | 31 | 2 | Pazopanib | RTK | N/A | 42% (ORR), 52% (RCC), 53% (pNETs), 4% (HBL) |
| Kevin D. Courtney | NCT03108066 | 2014–2016 | 51 | 1 | PT2385 | HIF-2α | 53% at 52 Weeks | 14% (RCC) |
| Prashant Chittiboina | NCT02108002 | 2014–2018 | 7 | 0 | Vorinostat | HDAC | N/A | 0% |
| Henry E. Wiley | NCT02859441 | 2016–2020 | 3 | 1,2 | E10030 and Ranibizumab | PDGF, VEGF | N/A | 0% |
| Eric Jonasch | NCT03401788 | 2016–2020 | 95 | 1 | Belzutifan | HIF-2α | 14.5 Months | 25% (RCC) |
| Kevin D. Courtney | NCT03108066 | 2017–2022 | 4 | 2 | PT2385 | HIF-2α | N/A | N/A |
| Eric Jonasch | NCT03401788 | 2018–2026 | 61 | 2 | Belzutifan | HIF-2α | 98% at 52 Weeks | 47% (ORR), 80% (pNETs), 32% (HBL), 69% (rHBL*) |
The clinical trials with the “condition or disease” search terms “Von Hippel-Lindau” and “Hemangioblastoma” were searched on clinicaltrials.gov. This obtained 38 pan-VHL and 14 hemangioblastoma trials. Trials that included a pharmacologic agent were selected for the table. A PubMed search with the filter “Clinical Trials” using the terms “Von Hippel-Lindau” and “hemangioblastoma” returned 59 and 26 papers, respectively. Similarly, only pharmacologic agents were included in the table. The principal investigator was obtained from the corresponding clinicaltrials.gov entry or from the corresponding publication. The response rate was calculated as the sum of partial response and complete response. Retinal hemangioblastomas do not have formal RR; these represent the percentage of patients who experienced improvements in vision following treatment. These searches were performed on June 1, 2022. PFS: Progression-free survival. RR: Response rate. PR: Partial response. CR: Complete response. rHBL: Retinal hemangioblastoma. RCC: Renal cell carcinoma. HBL: Hemangioblastoma. pNETs: Pancreatic neuroendocrine tumors. ORR: Overall response rate. VEGF: vascular endothelial growth factor. RTK: receptor tyrosine kinase; can target multiple kinases including VEGF. HSP-90: heat-shock protein 90. HDAC: histone deacetylase. HIF-2α: hypoxia-inducible factor-2 alpha.
| Principal Investigator . | Clinical Trial ID . | Year . | Patients . | Phase . | Agent . | Target . | PFS (Mos.) . | RR (PR+CR) (%) . |
|---|---|---|---|---|---|---|---|---|
| Emily Chew | NCT00056199 | 2003–2005 | 5 | 1 | Pegaptanib | VEGF | N/A | 20% (rHBL*) |
| Novartis | NCT00052013 | 2003–2006 | 11 | 2 | Vatalanib | RTK | N/A | 0% |
| Adrian L. Harris | N/A | 2004 | 6 | 1,2 | Semaxanib | VEGF | N/A | 0% |
| Emily Chew | NCT00089765 | 2004–2007 | 5 | 1 | Ranibizumab | VEGF | N/A | 20% (rHBL*) |
| William M. Linehan | NCT00088374 | 2004–2009 | 9 | 2 | Tanespimycin | HSP-90 | N/A | 0% |
| Eric Jonasch | NCT00330564 | 2006–2011 | 15 | 2 | Sunitinib | RTK | N/A | 33% (RCC), 0% (HBL) |
| Catherine Meyerle | NCT00673816 | 2008–2011 | 3 | 1,2 | Sunitinib | RTK | N/A | 0% |
| William M. Linehan | NCT00566995 | 2008–2015 | 37 | 2 | Vandetanib | RTK | (11.0 to 22.1) | 8% (RCC) |
| J. Marc Pipas | NCT01015300 | 2009–2012 | 1 | 1 | Bevacizumab | VEGF | N/A | 0% |
| Stephane Richard | NCT01168440 | 2010–2011 | 5 | 2 | Sunitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01266070 | 2012–2015 | 6 | 2 | Dovitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01436227 | 2012–2021 | 31 | 2 | Pazopanib | RTK | N/A | 42% (ORR), 52% (RCC), 53% (pNETs), 4% (HBL) |
| Kevin D. Courtney | NCT03108066 | 2014–2016 | 51 | 1 | PT2385 | HIF-2α | 53% at 52 Weeks | 14% (RCC) |
| Prashant Chittiboina | NCT02108002 | 2014–2018 | 7 | 0 | Vorinostat | HDAC | N/A | 0% |
| Henry E. Wiley | NCT02859441 | 2016–2020 | 3 | 1,2 | E10030 and Ranibizumab | PDGF, VEGF | N/A | 0% |
| Eric Jonasch | NCT03401788 | 2016–2020 | 95 | 1 | Belzutifan | HIF-2α | 14.5 Months | 25% (RCC) |
| Kevin D. Courtney | NCT03108066 | 2017–2022 | 4 | 2 | PT2385 | HIF-2α | N/A | N/A |
| Eric Jonasch | NCT03401788 | 2018–2026 | 61 | 2 | Belzutifan | HIF-2α | 98% at 52 Weeks | 47% (ORR), 80% (pNETs), 32% (HBL), 69% (rHBL*) |
| Principal Investigator . | Clinical Trial ID . | Year . | Patients . | Phase . | Agent . | Target . | PFS (Mos.) . | RR (PR+CR) (%) . |
|---|---|---|---|---|---|---|---|---|
| Emily Chew | NCT00056199 | 2003–2005 | 5 | 1 | Pegaptanib | VEGF | N/A | 20% (rHBL*) |
| Novartis | NCT00052013 | 2003–2006 | 11 | 2 | Vatalanib | RTK | N/A | 0% |
| Adrian L. Harris | N/A | 2004 | 6 | 1,2 | Semaxanib | VEGF | N/A | 0% |
| Emily Chew | NCT00089765 | 2004–2007 | 5 | 1 | Ranibizumab | VEGF | N/A | 20% (rHBL*) |
| William M. Linehan | NCT00088374 | 2004–2009 | 9 | 2 | Tanespimycin | HSP-90 | N/A | 0% |
| Eric Jonasch | NCT00330564 | 2006–2011 | 15 | 2 | Sunitinib | RTK | N/A | 33% (RCC), 0% (HBL) |
| Catherine Meyerle | NCT00673816 | 2008–2011 | 3 | 1,2 | Sunitinib | RTK | N/A | 0% |
| William M. Linehan | NCT00566995 | 2008–2015 | 37 | 2 | Vandetanib | RTK | (11.0 to 22.1) | 8% (RCC) |
| J. Marc Pipas | NCT01015300 | 2009–2012 | 1 | 1 | Bevacizumab | VEGF | N/A | 0% |
| Stephane Richard | NCT01168440 | 2010–2011 | 5 | 2 | Sunitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01266070 | 2012–2015 | 6 | 2 | Dovitinib | RTK | N/A | 0% |
| Eric Jonasch | NCT01436227 | 2012–2021 | 31 | 2 | Pazopanib | RTK | N/A | 42% (ORR), 52% (RCC), 53% (pNETs), 4% (HBL) |
| Kevin D. Courtney | NCT03108066 | 2014–2016 | 51 | 1 | PT2385 | HIF-2α | 53% at 52 Weeks | 14% (RCC) |
| Prashant Chittiboina | NCT02108002 | 2014–2018 | 7 | 0 | Vorinostat | HDAC | N/A | 0% |
| Henry E. Wiley | NCT02859441 | 2016–2020 | 3 | 1,2 | E10030 and Ranibizumab | PDGF, VEGF | N/A | 0% |
| Eric Jonasch | NCT03401788 | 2016–2020 | 95 | 1 | Belzutifan | HIF-2α | 14.5 Months | 25% (RCC) |
| Kevin D. Courtney | NCT03108066 | 2017–2022 | 4 | 2 | PT2385 | HIF-2α | N/A | N/A |
| Eric Jonasch | NCT03401788 | 2018–2026 | 61 | 2 | Belzutifan | HIF-2α | 98% at 52 Weeks | 47% (ORR), 80% (pNETs), 32% (HBL), 69% (rHBL*) |
The clinical trials with the “condition or disease” search terms “Von Hippel-Lindau” and “Hemangioblastoma” were searched on clinicaltrials.gov. This obtained 38 pan-VHL and 14 hemangioblastoma trials. Trials that included a pharmacologic agent were selected for the table. A PubMed search with the filter “Clinical Trials” using the terms “Von Hippel-Lindau” and “hemangioblastoma” returned 59 and 26 papers, respectively. Similarly, only pharmacologic agents were included in the table. The principal investigator was obtained from the corresponding clinicaltrials.gov entry or from the corresponding publication. The response rate was calculated as the sum of partial response and complete response. Retinal hemangioblastomas do not have formal RR; these represent the percentage of patients who experienced improvements in vision following treatment. These searches were performed on June 1, 2022. PFS: Progression-free survival. RR: Response rate. PR: Partial response. CR: Complete response. rHBL: Retinal hemangioblastoma. RCC: Renal cell carcinoma. HBL: Hemangioblastoma. pNETs: Pancreatic neuroendocrine tumors. ORR: Overall response rate. VEGF: vascular endothelial growth factor. RTK: receptor tyrosine kinase; can target multiple kinases including VEGF. HSP-90: heat-shock protein 90. HDAC: histone deacetylase. HIF-2α: hypoxia-inducible factor-2 alpha.
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