Disease · MONDO_0008667
von Hippel-Lindau disease
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. von Hippel-Lindau disease. Diseases, record MONDO_0008667. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008667
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
Lindau disease, VHL, VHL-related von Hippel-Lindau disease, Von Hippel-Lindau syndrome, Von Hippel-Lindau syndrome (VHL), cerebroretinal angiomatosis, familial cerebelloretinal angiomatosis, von Hippel-Lindau syndrome, modifier of
- Open Targets
- Highest stage of its drugs
- Approval, for 1 of its 11 drugs, from EMA Human Drugs, FDA and TTD
- Open Targets
Drugs11
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| BELZUTIFAN | CHEMBL4585668 | Small molecule | Approval | EMA Human Drugs, FDA and TTD | 4 | 1 | 1 | Drugs and targets |
| DOVITINIB | CHEMBL522892 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| PAZOPANIB HYDROCHLORIDE | CHEMBL1201733 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SUNITINIB | CHEMBL535 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SUNITINIB MALATE | CHEMBL1567 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TANESPIMYCIN | CHEMBL109480 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| VATALANIB | CHEMBL101253 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| 79A | CHEMBL4173075 | Small molecule | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
| EVEROLIMUS | CHEMBL1908360 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SPARTALIZUMAB | CHEMBL4297831 | Antibody | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets20
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| FLT1 | Vascular endothelial growth factor receptor | 5 | Vascular endothelial growth factor receptor 1 inhibitor; Vascular endothelial growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/2321 | Drugs and targets |
| FLT4 | Vascular endothelial growth factor receptor | 5 | Vascular endothelial growth factor receptor 3 inhibitor; Vascular endothelial growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/2324 | Drugs and targets |
| KDR | Vascular endothelial growth factor receptor | 5 | Vascular endothelial growth factor receptor 2 inhibitor; Vascular endothelial growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/3791 | Drugs and targets |
| KIT | Mast/stem cell growth factor receptor Kit | 5 | Stem cell growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/3815 | Drugs and targets |
| PDGFRA | Platelet-derived growth factor receptor | 5 | Platelet-derived growth factor receptor alpha inhibitor; Platelet-derived growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/5156 | Drugs and targets |
| PDGFRB | Platelet-derived growth factor receptor | 5 | Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/5159 | Drugs and targets |
| CSF1R | Macrophage colony-stimulating factor 1 receptor | 4 | Macrophage colony stimulating factor receptor inhibitor | Phase 2 | https://conductscience.org/library/genes/1436 | Drugs and targets |
| FLT3 | Receptor-type tyrosine-protein kinase FLT3 | 3 | Tyrosine-protein kinase receptor FLT3 inhibitor | Phase 2 | https://conductscience.org/library/genes/2322 | Drugs and targets |
| FGFR3 | Fibroblast growth factor receptor 3 | 2 | Fibroblast growth factor receptor 3 inhibitor | Phase 2 | https://conductscience.org/library/genes/2261 | Drugs and targets |
| RET | Proto-oncogene tyrosine-protein kinase receptor Ret | 2 | Tyrosine-protein kinase receptor RET inhibitor | Phase 2 | https://conductscience.org/library/genes/5979 | Drugs and targets |
1 to 10 of 20
See all 20 targetsTrials9
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01436227 | Pazopanib Hydrochloride in Treating Patients With Von Hippel-Lindau Syndrome | Phase 2 | 32 | 2025-05-02 | Von Hippel-Lindau Syndrome | PAZOPANIB HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT00330564 | Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow | Phase 2 | 15 | 2011-06-30 | Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Hemangioblastoma | SUNITINIB MALATE | Its record names the disease | ClinicalTrials.gov |
| NCT00088374 | 17AAG to Treat Kidney Tumors in Von Hippel-Lindau Disease | Phase 2 | 9 | 2011-09-29 | Hippel-Lindau Disease, Kidney Cancer | TANESPIMYCIN | Its record names the disease | ClinicalTrials.gov |
| NCT01266070 | TKI 258 in Von Hippel-Lindau Syndrome (VHL) | Phase 2 | 6 | 2017-02-13 | Von Hippel-Lindau Syndrome | DOVITINIB | Its record names the disease | ClinicalTrials.gov |
| NCT07167329 | Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL Trial | Phase 2 | 100 | Von Hippel Lindau, Von Hippel Lindau Disease, Von Hippel Lindau-Deficient Clear Cell Renal Cell Carcinoma, Hemangioblastoma (HB) of the Central Nervous System (CNS), PNET, Retinal Angiomatous Proliferation, Pheochromocytoma/Paraganglioma, Endolymphatic Sac Tumor | BELZUTIFAN | Its record names the disease | ClinicalTrials.gov | |
| NCT05424016 | Propranolol and Von Hippel-Lindau Disease | Not applicable | 85 | Hemangioblastoma of CNS, Von Hippel-Lindau Disease | PROPRANOLOL | Its record names the disease | ClinicalTrials.gov | |
| NCT04895748 | DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other Malignancies | Phase 1 | 40 | Carcinoma, Renal Cell | EVEROLIMUS, SPARTALIZUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT00052013 | Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584 | Phase 2 | 11 | Von Hippel-Lindau Disease, CNS Hemangioblastoma, Retinal Hemangioblastoma | VATALANIB | Its record names the disease | ClinicalTrials.gov | |
| NCT01168440 | Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) Disease | Phase 2 | 5 | Von Hippel-Lindau Disease | SUNITINIB | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials0
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
None in this release.
Papers from its trials3
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0008667
GET https://conductscience.org/api/diseases/v1/MONDO_0008667/drugs