Condition (MeSH)
von Hippel-Lindau Disease
ClinicalTrials.gov lists 55 trials for von Hippel-Lindau Disease or a narrower condition beneath it. National Eye Institute (NEI) has the most among industry and government lead sponsors, with 7.
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Trials per phase (log scale)
55 of the 55 trials are tagged with von Hippel-Lindau Disease itself; the rest are tagged with a narrower condition beneath it.
When did von Hippel-Lindau Disease trials start? (25)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Not applicable
- Observational
Top of scale: 7
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 6 |
| 2024 | 5 |
| 2023 | 7 |
| 2022 | 2 |
| 2021 | 2 |
| 2020 | 2 |
| 2019 | 1 |
| 2018 | 1 |
| 2017 | 4 |
| 2014 | 1 |
Showing 10 of 25 earlier years.
No forecast is made for this condition: it averages 4.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 7 trials still running at the 2026-10-09 snapshot; registered median of 45 months across 21 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are von Hippel-Lindau Disease trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 3 |
| Phase 2 | 15 |
| Phase 3 | 1 |
| Not applicable | 4 |
| Observational | 24 |
How many von Hippel-Lindau Disease trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 16 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 5 |
| Completed | 18 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most von Hippel-Lindau Disease trials? (8)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| National Eye Institute (NEI) | 7 |
| National Cancer Institute (NCI) | 5 |
| Merck Sharp & Dohme LLC | 3 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 3 |
| National Institutes of Health Clinical Center (CC) | 2 |
Showing 5 of 8 sponsors.
Interventions as named in the registry (15)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Bezlutifan | 5 |
| Sunitinib 4/2 | 3 |
| ranibizumab PRN | 3 |
| [15-O] H2O | 1 |
| BPI-452080 | 1 |
Showing 5 of 15 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do von Hippel-Lindau Disease trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Death | 22 | 1 |
| Death (unrelated to 68 Gallium DOTATATE) | 16 | 1 |
| Alanine aminotransferase increased | 4 | 1 |
| Aphasia | 3 | 1 |
| Thromboembolic event | 3 | 1 |
Showing 5 of 10 adverse events.
Which publications cover von Hippel-Lindau Disease? (73)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Belzutifan for von Hippel-Lindau disease-associated renal cell carcinoma and other neoplasms (LITESPARK-004): 50 months follow-up from a single-arm, phase 2 study. | The Lancet. Oncology | 2025 | Yes |
| Longitudinal Evaluation of Clear-cell Renal Cell Carcinoma in von Hippel-Lindau Disease. | European urology | 2025 | Yes |
| Belzutifan for von Hippel-Lindau Disease: Pancreatic Lesion Population of the Phase 2 LITESPARK-004 Study. | Clinical cancer research : an official journal of the American Association for Cancer Research | 2024 | Yes |
| Belzutifan for patients with von Hippel-Lindau disease-associated CNS haemangioblastomas (LITESPARK-004): a multicentre, single-arm, phase 2 study. | The Lancet. Oncology | 2024 | Yes |
| Comprehensive characterization and building of National Registry of von Hippel-Lindau disease in Brazil. | Molecular genetics & genomic medicine | 2023 | Yes |
Showing 5 of 73 publications.
Prevalence (15)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Von Hippel-Lindau disease | Prevalence at birth | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Von Hippel-Lindau disease | Prevalence at birth | 1-9 / 100 000 | United Kingdom | GB | Unspecified |
| Von Hippel-Lindau disease | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Unspecified |
| Von Hippel-Lindau disease | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Von Hippel-Lindau disease | Point prevalence | 1-9 / 100 000 | United Kingdom | GB | Exact |
Showing 5 of 15 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Von Hippel-Lindau disease | 892 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Von Hippel-Lindau disease | 892 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.