Ignite Creation Date:
2024-05-05 @ 11:30 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00056199
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2003-03-07
Brief Title:
EYE001 to Treat Retinal Tumors in Patients With Von Hippel-Lindau Syndrome
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pilot Study of Intravitreal Injection of EYE001 Anti-VEGF Pegylated Aptamer for Advanced Ocular Disease of Von Hippel-Lindau VHL Disease
Status:
COMPLETED
Status Verified Date:
2005-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will test the ability of the experimental drug EYE001 to reduce retinal thickening and improve vision in patients with Von Hippel-Lindau syndrome VHL Angiomas blood vessel tumors commonly develop in the back of the eye on the retina and the optic nerve in patients with VHL Although the tumors are not cancerous they may cause significant vision loss Current treatments including laser therapy cryotherapy and vitrectomy may not be successful or possible for all patients EYE001 decreases production of VEGF a growth factor that is important for the formation of new blood vessels and that is elevated in VHL Preliminary findings from studies of other retinal diseases suggest that EYE001 can reduce retinal thickening and improve vision
Patients 18 years of age and older with retinal angiomas due to VHL in one or both eyes and central vision loss of 2040 or worse may be eligible for this study Participants will undergo the following tests and procedures
Medical history physical examination electrocardiogram EKG and blood tests
Eye examination including eye pressure measurement and dilation of the pupils to examine the retina
Fluorescein angiography to evaluate the eyes blood vessels For this test a yellow dye is injected into an arm vein and travels to the blood vessels in the eyes Pictures of the retina are taken using a camera that flashes a blue light into the eye The pictures will reveal if any dye has leaked from the vessels into the retina indicating possible blood vessel abnormality
Optical coherence tomography to measure retinal thickness The eyes are examined through a machine that produces cross-sectional pictures of the retina These measures are repeated during the study to determine changes if any in retinal thickening
Electroretinogram ERG to measure electrical responses generated from within the retina For this test the patient sits in a dark room for 30 minutes with his or her eyes patched Then a small silver disk electrode is taped to the forehead the eye patches are removed the surface of the eye is numbed with eye drops and contact lenses are placed on the eyes The patient looks inside an open white globe that emits a series of light flashes for about 20 minutes The contact lenses sense small electrical signals generated by the retina when the light flashes
Stereoscopic color fundus photography to examine the back of the eye The pupils are dilated with eye drops to examine and photograph the back of the eye
EYE001 injections to treat ocular angiomas Patients receive EYE001 injections through a needle into the eyes vitreous gel-like substance that fills the inside of the eye Six injections are given over a 30-week period Before each injection the surface of the eye is numbed with anesthetic eye drops This is followed by injection of another anesthetic into the lower portion of they eye in the clear tissue surrounding the white of the eye After a few minutes the EYE001 is injected into the vitreous Patients receive EYE001 injections at the first visit during enrollment and again at 6 12 18 24 and 30 weeks after the first injection
At each injection visit participants repeat most of the tests described above to evaluate the response to treatment and return a week later for another eye examination After the last injection patients whose vision has improved may receive three more treatments at visits 36 42 and 48 All participants will return for examinations at week 54 and at 2 months after their final injection
Patients 18 years of age and older with retinal angiomas due to VHL in one or both eyes and central vision loss of 2040 or worse may be eligible for this study Participants will undergo the following tests and procedures
Medical history physical examination electrocardiogram EKG and blood tests
Eye examination including eye pressure measurement and dilation of the pupils to examine the retina
Fluorescein angiography to evaluate the eyes blood vessels For this test a yellow dye is injected into an arm vein and travels to the blood vessels in the eyes Pictures of the retina are taken using a camera that flashes a blue light into the eye The pictures will reveal if any dye has leaked from the vessels into the retina indicating possible blood vessel abnormality
Optical coherence tomography to measure retinal thickness The eyes are examined through a machine that produces cross-sectional pictures of the retina These measures are repeated during the study to determine changes if any in retinal thickening
Electroretinogram ERG to measure electrical responses generated from within the retina For this test the patient sits in a dark room for 30 minutes with his or her eyes patched Then a small silver disk electrode is taped to the forehead the eye patches are removed the surface of the eye is numbed with eye drops and contact lenses are placed on the eyes The patient looks inside an open white globe that emits a series of light flashes for about 20 minutes The contact lenses sense small electrical signals generated by the retina when the light flashes
Stereoscopic color fundus photography to examine the back of the eye The pupils are dilated with eye drops to examine and photograph the back of the eye
EYE001 injections to treat ocular angiomas Patients receive EYE001 injections through a needle into the eyes vitreous gel-like substance that fills the inside of the eye Six injections are given over a 30-week period Before each injection the surface of the eye is numbed with anesthetic eye drops This is followed by injection of another anesthetic into the lower portion of they eye in the clear tissue surrounding the white of the eye After a few minutes the EYE001 is injected into the vitreous Patients receive EYE001 injections at the first visit during enrollment and again at 6 12 18 24 and 30 weeks after the first injection
At each injection visit participants repeat most of the tests described above to evaluate the response to treatment and return a week later for another eye examination After the last injection patients whose vision has improved may receive three more treatments at visits 36 42 and 48 All participants will return for examinations at week 54 and at 2 months after their final injection
Detailed Description:
Von Hippel-Lindau VHL is an autosomal dominant heritable disorder in which multiple benign and malignant neoplasms and cysts of specific histopathologies develop in the kidney adrenal gland pancreas brain spinal cord eye inner ear epididymis and broad ligament Retinal angioma may be one of the earliest manifestations of VHL disease and may lead to a significant decrease in visual acuity of the affected individual These tumors rarely regress spontaneously The main cause of vision loss is retinal edema specifically macular edema secondary to enlargement of peripheral retinal angiomas or angiomas found on or around the optic disk Treatment of retinal angiomas depends on the location and size of the lesions but typically consists of photocoagulation or cryotherapy However there is no proven effective therapy for the treatment of VHL ocular lesions on or surrounding the optic nerve or lesions in the peripheral retina too large to respond to the traditional therapies The genetic mutation found in VHL disease up-regulates the production of vascular endothelial growth factor VEGF Immunochemical studies of the VHL ocular lesions as well as others found elsewhere in the body show marked increase in VEGF This open-label study will pilot the use of anti-VEGF therapy EYE001 in 5 patients to investigate the potential efficacy as a treatment for retinal angiomas associated with VHL Patients will receive 6 intravitreal injections of study drug over a 30-week period then return for a follow-up visit 1 year after initiating injections The primary outcomes will be improvement in best corrected visual acuity of 15 letters or more at 1 year reduction in retinal thickening and leakage at one year changes in ERG amplitude and implicit time and adverse events including local and systemic toxicities
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 03-EI-0101 | None | None | None |