Von Hippel-Lindau SyndromePhase 1/2VHL3NCT00673816
Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome
A Phase 1/2 study testing Sunitinib Malate in people with Von Hippel-Lindau Syndrome, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
This open-label study will pilot the use of systemic sunitinib malate, a dual inhibitor of vascular endothelial growth factor (VEGF) and platelet derived growth factor (PDGF), in five participants with Von Hippel-Lindau (VHL) to investigate its potential efficacy as a treatment for retinal angiomas. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Hippel-Lindau Syndrome
- What is being tested?
- Sunitinib Malate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- May 2008to Feb 2011
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must understand and sign the informed consent.
- Participant must be at least 18 years of age.
- Participant must have genetically confirmed VHL disease.
- Participant must have an optic nerve angioma secondary to VHL in one or both eyes.
- Participant must have an optic nerve tumor that has caused any visual field depression on microperimetry-1 that correlates with the retinal angioma OR the participant clinically may have hard exudates correlating with the retinal angioma OR has best-corrected visual acuity of 20/40 or worse in the study eye.
- Participant must have clear ocular media and adequate pupillary dilation to permit good quality stereoscopic fundus photography.
Show all 14Show fewer
- All women of childbearing potential must have a negative urine pregnancy test at baseline, and have regular negative pregnancy testing while taking sunitinib malate. (Sunitinib malate has the potential for teratogenic or abortifacient effects, and no data regarding its safety in pregnant women are available).
- All women of childbearing potential who are sexually active and all men who are sexually active are required to use two forms of birth control during the course of the study.
- Participants must have normal organ and marrow function as defined below: WBC count ≥ 3,000/µL, absolute neutrophil count ≥ 1,500/µL, platelet count ≥ 100,000/µL, HGB> 10g/dl, serum creatinine ≤ 2.0 or measured 24 hr. creatinine clearance > 50 ml/min, AST and ALT < 2.5 x ULN, total bilirubin ≤ ULN (< 3 x NL in participants with Gilbert's disease).
- Participant must have a negative HbsAg and nonreactive HCV.
- Participant must have a negative HIV-1, as potential pharmacokinetic interactions of drugs used to treat HIV, such as anti-retroviral drugs, with sunitinib malate are unknown.
- Participant must be at least four weeks from completion of any investigational therapy for VHL.
- Participant must have an ECOG performance score of 0-2. (See Appendix 3 - ECOG Performance Criteria).
- Participant has recovered from the acute toxicities of prior treatment for VHL.
Who cannot join
- Participant has a history (within past five years) or evidence of severe cardiac disease including heart failure that meets New York Heart Association (NYHA) class III and IV definitions, uncontrolled dysrhythmias, dysrhythmias requiring anti-arhythmic drugs or has active ischemic heart disease including myocardial infarction and poorly controlled angina within 12 months of study entry.
- Participant has a history of serious ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation, ≥ three beats in a row) or left ventricular ejection fraction ≤ 40%.
- Participant has a history of serious intercurrent medical illness.
- Participant had transient ischemic attacks or cerebrovascular accident within 12 months of study entry.
- Participant has hypertension that cannot be controlled with medications (persistent elevation of systolic BP > 150 or diastolic BP > 100 mmHg despite optimal medical therapy).
- Participant is on therapeutic anticoagulation, including aspirin.
Show all 16Show fewer
- Participant who is breast-feeding, as there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with sunitinib malate.
- Participant has received any major surgical procedures within one month of study entry or has surgical scars that have not healed.
- Participant has a known serious allergy to fluorescein dye.
- Participant is currently taking drugs or ingesting food that affect sunitinib malate plasma concentrations: strong inhibitors of the CYP3A4 family (e.g., ketoconazole, itraconazole, clarithromycin, atazanavir, indinavir, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, voriconazole, grapefruit juice) and/or inducers of the CYP3A4 family (e.g., dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, rifapentin, phenobarbital, St. John's Wort).
- Participant has had a prior or concomitant non-VHL-associated malignancy with the exception of adequately treated basal or squamous cell carcinoma of the skin or any other malignancy from which the patient has remained disease free for more than five years.
- Participant has had chemotherapy or radiotherapy within four weeks (six weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events (to Grade 1 or less toxicity according to CTCAE 3.0) due to agents administered more than four weeks earlier.
- Participant is receiving other investigational agents.
- Participants with known brain metastases (except when adequately controlled, i.e., have not grown in size, for ≥ 6 months before enrollment), not including hemangioblastoma, a known VHL complication of the brain.
- Participant has a known bleeding disorder.
- Participant is currently taking sunitinib malate or has taken sunitinib malate in the past.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Pilot Study of Sunitinib Malate for Advanced Ocular Disease of Von Hippel-Lindau Syndrome
This open-label study will pilot the use of systemic sunitinib malate, a dual inhibitor of vascular endothelial growth factor (VEGF) and platelet derived growth factor (PDGF), in five participants with Von Hippel-Lindau (VHL) to investigate its potential efficacy as a treatment for retinal angiomas. Participants will have visual dysfunction with either visual acuity loss or visual field loss from retinal angiomas secondary to genetically confirmed VHL. This open-label study will pilot the use of systemic sunitinib malate in five participants to investigate its potential efficacy as a treatment for retinal angiomas associated with VHL. Participants will receive nine months of sunitinib malate therapy (six cycles total - one cycle consists of 50 mg oral dose once daily for four weeks followed by a two week rest period). The primary outcome will be a change in the best-corrected visual acuity of more than or equal to 15 letters from baseline to the Week 36 visit. The secondary ocular outcomes will focus on retinal thickness and leakage of the retinal angioma at the Week 36 visit. Optical coherence tomography will document changes in retinal thickening and fluorescein angiography will be used to determine leakage of the retinal angioma.
- Conditions
- Von Hippel-Lindau Syndrome
- Being tested
- Sunitinib Malate
- Keywords
- Von Hippel Lindau, Sunitinib Malate, Sutent, Von Hippel-Lindau Syndrome, VHL
- Medical subject headings
- von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sunitinib MalateDrug
Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period).
| Study group | Role | What they receive |
|---|---|---|
| Sunitinib Malate | Experimental | Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). Receives: Drug: Sunitinib Malate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
Measured: Baseline and 36 Weeks
Secondary outcomes (5)
Change in Retinal Thickness From Baseline to Week 36
Retinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
Measured: Baseline and 36 Weeks
Reduction in Leakage Intensity as Measured by Fluorescein Angiography From Baseline to Week 36
Number of participants with improvement in leakage intensity.
Measured: Baseline and 36 Weeks
Visual Field Changes as Documented by Microperimetry From Baseline to Week 36
Measured: Baseline and 36 Weeks
Changes in Non-ocular VHL Lesion Status
Count of participants with new non-ocular lesions present since baseline at any follow-up visit.
Measured: Duration of study
Reduction in Leakage Area as Measured by Fluorescein Angiography at the Week 36 Visit
Number of participants with improvement in leakage area.
Measured: Baseline and 36 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2008 (estimated)
- Study startMay 2008
- Primary completionFeb 2011
- Study completionFeb 2011
Why it stopped: Inability to recruit and adequate number of participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (45)Papers the sponsor has linked to this study.
- Other trials for Von Hippel-Lindau SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Catherine Meyerle, MD, Principal investigator, NEI/NIH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00673816
- Sponsor's protocol ID
- 080129
- Other IDs
- 08-EI-0129
- Acronym
- VHL3
- Status last verified
- Nov 2011
- First submitted
- 6 May 2008
- Last updated
- 5 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Siemeister G, Weindel K, Mohrs K, Barleon B, Martiny-Baron G, Marme D. Reversion of deregulated expression of vascular endothelial growth factor in human renal carcinoma cells by von Hippel-Lindau tumor suppressor protein. Cancer Res. 1996 May 15;56(10):2299-301.
PubMed 8625303Iliopoulos O, Levy AP, Jiang C, Kaelin WG Jr, Goldberg MA. Negative regulation of hypoxia-inducible genes by the von Hippel-Lindau protein. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):10595-9. doi: 10.1073/pnas.93.20.10595.
PubMed 8855223Gnarra JR, Zhou S, Merrill MJ, Wagner JR, Krumm A, Papavassiliou E, Oldfield EH, Klausner RD, Linehan WM. Post-transcriptional regulation of vascular endothelial growth factor mRNA by the product of the VHL tumor suppressor gene. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):10589-94. doi: 10.1073/pnas.93.20.10589.
PubMed 8855222Dahr SS, Cusick M, Rodriguez-Coleman H, Srivastava SK, Thompson DJ, Linehan WM, Ferris FL 3rd, Chew EY. Intravitreal anti-vascular endothelial growth factor therapy with pegaptanib for advanced von Hippel-Lindau disease of the retina. Retina. 2007 Feb;27(2):150-8. doi: 10.1097/IAE.0b013e318030a290.
PubMed 17290195Kurban G, Hudon V, Duplan E, Ohh M, Pause A. Characterization of a von Hippel Lindau pathway involved in extracellular matrix remodeling, cell invasion, and angiogenesis. Cancer Res. 2006 Feb 1;66(3):1313-9. doi: 10.1158/0008-5472.CAN-05-2560.
PubMed 16452184Aiello LP, George DJ, Cahill MT, Wong JS, Cavallerano J, Hannah AL, Kaelin WG Jr. Rapid and durable recovery of visual function in a patient with von hippel-lindau syndrome after systemic therapy with vascular endothelial growth factor receptor inhibitor su5416. Ophthalmology. 2002 Sep;109(9):1745-51. doi: 10.1016/s0161-6420(02)01159-4.
PubMed 12208726Girmens JF, Erginay A, Massin P, Scigalla P, Gaudric A, Richard S. Treatment of von Hippel-Lindau retinal hemangioblastoma by the vascular endothelial growth factor receptor inhibitor SU5416 is more effective for associated macular edema than for hemangioblastomas. Am J Ophthalmol. 2003 Jul;136(1):194-6. doi: 10.1016/s0002-9394(03)00101-6.
PubMed 12834696Seizinger BR, Rouleau GA, Ozelius LJ, Lane AH, Farmer GE, Lamiell JM, Haines J, Yuen JW, Collins D, Majoor-Krakauer D, et al. Von Hippel-Lindau disease maps to the region of chromosome 3 associated with renal cell carcinoma. Nature. 1988 Mar 17;332(6161):268-9. doi: 10.1038/332268a0.
PubMed 2894613Maher ER, Bentley E, Yates JR, Latif F, Lerman M, Zbar B, Affara NA, Ferguson-Smith MA. Mapping of the von Hippel-Lindau disease locus to a small region of chromosome 3p by genetic linkage analysis. Genomics. 1991 Aug;10(4):957-60. doi: 10.1016/0888-7543(91)90185-h.
PubMed 1680799MELMON KL, ROSEN SW. LINDAU'S DISEASE. REVIEW OF THE LITERATURE AND STUDY OF A LARGE KINDRED. Am J Med. 1964 Apr;36:595-617. doi: 10.1016/0002-9343(64)90107-x. No abstract available.
PubMed 14142412Horton WA, Wong V, Eldridge R. Von Hippel-Lindau disease: clinical and pathological manifestations in nine families with 50 affected members. Arch Intern Med. 1976 Jul;136(7):769-77. doi: 10.1001/archinte.136.7.769.
PubMed 945722Hardwig P, Robertson DM. von Hippel-Lindau disease: a familial, often lethal, multi-system phakomatosis. Ophthalmology. 1984 Mar;91(3):263-70. doi: 10.1016/s0161-6420(84)34304-4.
PubMed 6538954Green JS, Bowmer MI, Johnson GJ. Von Hippel-Lindau disease in a Newfoundland kindred. CMAJ. 1986 Jan 15;134(2):133-8, 146.
PubMed 3942913Ridley M, Green J, Johnson G. Retinal angiomatosis: the ocular manifestations of von Hippel-Lindau disease. Can J Ophthalmol. 1986 Dec;21(7):276-83.
PubMed 3801976Lamiell JM, Salazar FG, Hsia YE. von Hippel-Lindau disease affecting 43 members of a single kindred. Medicine (Baltimore). 1989 Jan;68(1):1-29. doi: 10.1097/00005792-198901000-00001.
PubMed 2642584
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Sunitinib Malate for Advanced Ocular Disease of Von Hippel-Lindau Syndrome
- ClinicalTrials.gov ID
- NCT00673816
- Acronym / sponsor ID
- VHL3 / 080129
- Conditions
- Von Hippel-Lindau Syndrome
- Keywords
- Von Hippel Lindau, Sunitinib Malate, Sutent, Von Hippel-Lindau Syndrome, VHL
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inability to recruit and adequate number of participants
- Enrolment
- 2 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- May 2008
- Primary completion
- Feb 2011 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 7 May 2008 (estimated)
- Results
- Posted 19 Dec 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This open-label study will pilot the use of systemic sunitinib malate, a dual inhibitor of vascular endothelial growth factor (VEGF) and platelet derived growth factor (PDGF), in five participants with Von Hippel-Lindau (VHL) to investigate its potential efficacy as a treatment for retinal angiomas. Participants will have visual dysfunction with either visual acuity loss or visual field loss from retinal angiomas secondary to genetically confirmed VHL. This open-label study will pilot the use of systemic sunitinib malate in five participants to investigate its potential efficacy as a treatment for retinal angiomas associated with VHL. Participants will receive nine months of sunitinib malate therapy (six cycles total - one cycle consists of 50 mg oral dose once daily for four weeks followed by a two week rest period). The primary outcome will be a change in the best-corrected visual acuity of more than or equal to 15 letters from baseline to the Week 36 visit. The secondary ocular outcomes will focus on retinal thickness and leakage of the retinal angioma at the Week 36 visit. Optical coherence tomography will document changes in retinal thickening and fluorescein angiography will be used to determine leakage of the retinal angioma.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sunitinib Malate | Experimental | Drug: Sunitinib MalateParticipants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Participant must understand and sign the informed consent.
- Participant must be at least 18 years of age.
- Participant must have genetically confirmed VHL disease.
- Participant must have an optic nerve angioma secondary to VHL in one or both eyes.
- Participant must have an optic nerve tumor that has caused any visual field depression on microperimetry-1 that correlates with the retinal angioma OR the participant clinically may have hard exudates correlating with the retinal angioma OR has best-corrected visual acuity of 20/40 or worse in the study eye.
Key exclusion criteria (16 listed)
- Participant has a history (within past five years) or evidence of severe cardiac disease including heart failure that meets New York Heart Association (NYHA) class III and IV definitions, uncontrolled dysrhythmias, dysrhythmias requiring anti-arhythmic drugs or has active ischemic heart disease including myocardial infarction and poorly controlled angina within 12 months of study entry.
- Participant has a history of serious ventricular arrhythmia (ventricular tachycardia or ventricular fibrillation, ≥ three beats in a row) or left ventricular ejection fraction ≤ 40%.
- Participant has a history of serious intercurrent medical illness.
- Participant had transient ischemic attacks or cerebrovascular accident within 12 months of study entry.
- Participant has hypertension that cannot be controlled with medications (persistent elevation of systolic BP > 150 or diastolic BP > 100 mmHg despite optimal medical therapy).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sunitinib MalateParticipants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period). | Drug | Sunitinib Malate |
Primary outcome
Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36
Time frame: Baseline and 36 Weeks
Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.
5 secondary outcomes
Change in Retinal Thickness From Baseline to Week 36
Time frame: Baseline and 36 Weeks
Reduction in Leakage Intensity as Measured by Fluorescein Angiography From Baseline to Week 36
Time frame: Baseline and 36 Weeks
Visual Field Changes as Documented by Microperimetry From Baseline to Week 36
Time frame: Baseline and 36 Weeks
Changes in Non-ocular VHL Lesion Status
Time frame: Duration of study
Reduction in Leakage Area as Measured by Fluorescein Angiography at the Week 36 Visit
Time frame: Baseline and 36 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 8625303Siemeister G, Weindel K, Mohrs K, Barleon B, Martiny-Baron G, Marme D. Reversion of deregulated expression of vascular endothelial growth factor in human renal carcinoma cells by von Hippel-Lindau tumor suppressor protein. Cancer Res. 1996 May 15;56(10):2299-301.
- Background: PubMed 8855223Iliopoulos O, Levy AP, Jiang C, Kaelin WG Jr, Goldberg MA. Negative regulation of hypoxia-inducible genes by the von Hippel-Lindau protein. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):10595-9. doi: 10.1073/pnas.93.20.10595.
- Background: PubMed 8855222Gnarra JR, Zhou S, Merrill MJ, Wagner JR, Krumm A, Papavassiliou E, Oldfield EH, Klausner RD, Linehan WM. Post-transcriptional regulation of vascular endothelial growth factor mRNA by the product of the VHL tumor suppressor gene. Proc Natl Acad Sci U S A. 1996 Oct 1;93(20):10589-94. doi: 10.1073/pnas.93.20.10589.
- Background: PubMed 17290195Dahr SS, Cusick M, Rodriguez-Coleman H, Srivastava SK, Thompson DJ, Linehan WM, Ferris FL 3rd, Chew EY. Intravitreal anti-vascular endothelial growth factor therapy with pegaptanib for advanced von Hippel-Lindau disease of the retina. Retina. 2007 Feb;27(2):150-8. doi: 10.1097/IAE.0b013e318030a290.
- Background: PubMed 16452184Kurban G, Hudon V, Duplan E, Ohh M, Pause A. Characterization of a von Hippel Lindau pathway involved in extracellular matrix remodeling, cell invasion, and angiogenesis. Cancer Res. 2006 Feb 1;66(3):1313-9. doi: 10.1158/0008-5472.CAN-05-2560.
- Background: PubMed 12208726Aiello LP, George DJ, Cahill MT, Wong JS, Cavallerano J, Hannah AL, Kaelin WG Jr. Rapid and durable recovery of visual function in a patient with von hippel-lindau syndrome after systemic therapy with vascular endothelial growth factor receptor inhibitor su5416. Ophthalmology. 2002 Sep;109(9):1745-51. doi: 10.1016/s0161-6420(02)01159-4.
- Background: PubMed 12834696Girmens JF, Erginay A, Massin P, Scigalla P, Gaudric A, Richard S. Treatment of von Hippel-Lindau retinal hemangioblastoma by the vascular endothelial growth factor receptor inhibitor SU5416 is more effective for associated macular edema than for hemangioblastomas. Am J Ophthalmol. 2003 Jul;136(1):194-6. doi: 10.1016/s0002-9394(03)00101-6.
- Background: PubMed 2894613Seizinger BR, Rouleau GA, Ozelius LJ, Lane AH, Farmer GE, Lamiell JM, Haines J, Yuen JW, Collins D, Majoor-Krakauer D, et al. Von Hippel-Lindau disease maps to the region of chromosome 3 associated with renal cell carcinoma. Nature. 1988 Mar 17;332(6161):268-9. doi: 10.1038/332268a0.
- Background: PubMed 1680799Maher ER, Bentley E, Yates JR, Latif F, Lerman M, Zbar B, Affara NA, Ferguson-Smith MA. Mapping of the von Hippel-Lindau disease locus to a small region of chromosome 3p by genetic linkage analysis. Genomics. 1991 Aug;10(4):957-60. doi: 10.1016/0888-7543(91)90185-h.
- Background: PubMed 14142412MELMON KL, ROSEN SW. LINDAU'S DISEASE. REVIEW OF THE LITERATURE AND STUDY OF A LARGE KINDRED. Am J Med. 1964 Apr;36:595-617. doi: 10.1016/0002-9343(64)90107-x. No abstract available.
- Background: PubMed 945722Horton WA, Wong V, Eldridge R. Von Hippel-Lindau disease: clinical and pathological manifestations in nine families with 50 affected members. Arch Intern Med. 1976 Jul;136(7):769-77. doi: 10.1001/archinte.136.7.769.
- Background: PubMed 6538954Hardwig P, Robertson DM. von Hippel-Lindau disease: a familial, often lethal, multi-system phakomatosis. Ophthalmology. 1984 Mar;91(3):263-70. doi: 10.1016/s0161-6420(84)34304-4.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Catherine Meyerle, MD principal investigator
NEI/NIH
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sunitinib Malate in people with Von Hippel-Lindau Syndrome, run by National Eye Institute (NEI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Dec 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36 (time frame: Baseline and 36 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00673816, which also lists 45 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2024.