von Hippel-Lindau Disease clinical trials
The registry lists 55 studies for von Hippel-Lindau Disease across more than 10 countries. 23 are recruiting or about to start. 36 of them are mainly about von Hippel-Lindau Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-13.
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· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
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Who runs them
National Eye Institute (NEI) (7) · M.D. Anderson Cancer Center (5) · National Cancer Institute (NCI) (5) · Merck Sharp & Dohme LLC (3) · National Institute of Neurological Disorders and Stroke (NINDS) (3) · IRCCS San Raffaele (2) · IRCCS Ospedale San Raffaele (2) · Peking University First Hospital (2)
What is being tested here
Drugs under test in von Hippel-Lindau Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Belzutifan | 3 | 1 | 4 |
| Cabozantinib | 1 | 1 | 1 |
| HIF-2alpha Inhibitor HS-10516 | 1 | 0 | 1 |
| Propranolol | 1 | 0 | 1 |
| Sunitinib | 0 | 0 | 3 |
| Ranibizumab | 0 | 0 | 2 |
| Pazopanib | 0 | 0 | 1 |
| PT2385 | 0 | 0 | 1 |
| Pegpleranib sodium | 0 | 0 | 1 |
| Vorinostat | 0 | 0 | 1 |
Drug classes tested here Tyrosine kinase inhibitors (TKIs) 1 open · Angiogenesis inhibitors 6
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6
- Nov 2026Propranolol and Von Hippel-Lindau Disease · Assistance Publique - Hôpitaux de Paris
- Dec 2026Data Collection Protocol for Patients With Von Hippel Lindau Disease · M.D. Anderson Cancer Center
- Dec 2026CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau Disease · IRCCS Ospedale San Raffaele
- Apr 2027A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004) · Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
- May 2027Promoting Stress Management and Resilience Among Individuals With Von Hippel- Lindau Disease · Massachusetts General Hospital
- Jul 2027Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas · Assistance Publique - Hôpitaux de Paris
Who these studies take
Of the 20 von Hippel-Lindau Disease studies recruiting now, 16 (80%) set no upper age limit and 0 (0%) stop at 75 or younger. 9 (45%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 5 (25%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (25%) | 5 (25%) |
| 18 | 20 (100%) | 20 (100%) |
| 30 | 20 (100%) | 20 (100%) |
| 40 | 20 (100%) | 20 (100%) |
| 50 | 20 (100%) | 20 (100%) |
| 65 | 20 (100%) | 20 (100%) |
| 75 | 20 (100%) | 20 (100%) |
| 85 | 17 (85%) | 17 (85%) |
Across all 55 studies, recruiting or not: 5% stop at 75 or younger and 4% at 65; 36% take children; 0% are for women only and 0% for men only; 9% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 15, 2026.
Started recruiting1
- Belzutifan VHL RCC HK Prospective RegistryOct 22, 2026NCT07554508
Studies
Recruiting first, then most recently updated. Showing 50 of 55. Browse all 55 → or search them.
- Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)RecruitingNCT04924075Phase 222 countries
- Use of Tracking Devices to Locate Abnormalities During Invasive ProceduresEnrolling by invitationNCT00102544Not applicable1 country
- National Eye Institute Biorepository for Retinal DiseasesRecruitingNCT014966251 country
- Belzutifan VHL RCC HK Prospective RegistryRecruitingNCT075545081 country
- Extension Study for Participants in Studies That Include Belzutifan (MK-6482-043/LITESPARK-043)RecruitingNCT07405164Phase 322 countries
- Natural History and Management of Von Hippel-Lindau (VHL) Associated Pancreatic Neuroendocrine TumorsRecruitingNCT04074135Phase 21 country
- Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System HemangioblastomasNot yet recruitingNCT077055291 country
- Data Collection Protocol for Patients With Von Hippel Lindau DiseaseRecruitingNCT059550141 country
- Familial Investigations of Childhood Cancer PredispositionRecruitingNCT030502681 country
- 18F-T2 PET/CT Imaging for CAIX Positive Solid TumorsRecruitingNCT07557225Not applicable1 country
- CAT-VHL Exploring the Role of Carbonic Anhydrase IX as Diagnostic and Theranostic Target in Von-Hippel Lindau DiseaseRecruitingNCT07171905Phase 21 country
- Institutional Registry of Rare DiseasesRecruitingNCT065737231 country
- Real-World Effectiveness and Pharmacogenetics of Belzutifan in VHL Syndrome: The BELIEVE-VHL TrialRecruitingNCT07167329Phase 21 country
- A Study of HS-10516 in Patients With VHL Syndrome Associated TumorsRecruitingNCT06553339Phase 11 country
- Genetic Bases of Neuroendocrine Neoplasms in Mexican PatientsRecruitingNCT065235821 country
- Establishment of a Multidimensional Prediction Model for the Natural Course of VHL Disease-related Renal Cell CarcinomaRecruitingNCT063918791 country
- MyVHL: Patient Natural History StudyRecruitingNCT037499801 country
- Mechanisms of Somatic Mutation and Tumor Initiation in Pre-malignant Kidney Tubule CellsRecruitingNCT061946691 country
- Propranolol and Von Hippel-Lindau DiseaseRecruitingNCT05424016Not applicable1 country
- Frequency, Clinical Phenotype and Genetic Analysis of Heritable Kidney Cancer SyndromesRecruitingNCT055348541 country
- 68Ga-NY104 PET/CT in Von Hippel-Lindau DiseaseRecruitingNCT05810246Phase 21 country
- A Study of BPI-452080 in Subjects With Solid TumorsNot yet recruitingNCT05843305Phase 11 country
- Screening for Endolymphatic Sac Tumours (ELSTs) in Von Hippel-Lindau (vHL) PatientsRecruitingNCT024200671 country
- Overtaking Intra and Inter Tumoral Heterogeneity In Von Hippel-Lindau Related Renal CancerCompletedNCT061951501 country
- Study of Brain and Spinal Cord Tumor Growth and Cyst Development in Patients With Von Hippel Lindau DiseaseActive, not recruitingNCT000059021 country
- Promoting Stress Management and Resilience Among Individuals With Von Hippel- Lindau DiseaseActive, not recruitingNCT05737602Not applicable1 country
- Retrospective Case Series of Trans-scleral Cryotherapy for Retinal HemangioblastomaCompletedNCT044589351 country
- Quality of Life, Psychological Impact, and Care-related Challenges in Patients Affected by Von Hippel-Lindau Syndrome.Active, not recruitingNCT072750991 country
- A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)Active, not recruitingNCT03401788Phase 24 countries
- DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other MalignanciesTerminatedNCT04895748Phase 17 countries
- Precision Medicine for Neurocutaneous Syndromes in Western ChinaCompletedNCT073271641 country
- Pazopanib Hydrochloride in Treating Patients With Von Hippel-Lindau SyndromeCompletedNCT01436227Phase 21 country
- MSD Belzutifan PASWithdrawnNCT06554730
- MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)CompletedNCT03108066Phase 21 country
- Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to FollowTerminatedNCT00330564Phase 21 country
- Visualizing Vascular Endothelial Growth Factor (VEGF) Producing Lesions in Von Hippel-Lindau DiseaseCompletedNCT009709701 country
- Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau SyndromeTerminatedNCT00673816Phase 1/21 country
- A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist RanibizumabCompletedNCT02859441Phase 1/21 country
- 68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine TumorsTerminatedNCT03001349Early Phase 11 country
- Von Hippel-Lindau Disease Genetic Epidemiology StudyTerminatedNCT000018031 country
- Evaluation of 68Gallium-DOTATATE PET/CT for Detecting Neuroendocrine TumorsCompletedNCT01967537Phase 21 country
- Drivers of Hypoxia-induced Angiogenesis in Tumor DevelopmentStatus unknownNCT039798331 country
- Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau DiseaseCompletedNCT000621661 country
- Effect of Vorinostat on Nervous System Hemangioblastomas in Von Hippel-Lindau Disease (Missense Mutation Only)CompletedNCT02108002Phase 11 country
- Ranibizumab Injections to Treat Retinal Tumors in Patients With Von Hippel-Lindau SyndromeCompletedNCT00089765Phase 11 country
- Metabolic Mapping to Measure Retinal MetabolismCompletedNCT00385333Phase 21 country
- Treatment of Von Hippel-Lindau (VHL)-Related Hemangioblastoma With PTK787/ZK 222584CompletedNCT00052013Phase 21 country
- TKI 258 in Von Hippel-Lindau Syndrome (VHL)TerminatedNCT01266070Phase 21 country
- Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related MaculopathyCompletedNCT00470977Phase 1/21 country
- Study of Sunitinib in Patients With Von Hippel-Lindau (VHL) DiseaseCompletedNCT01168440Phase 21 country