Ignite Creation Date:
2024-05-05 @ 11:30 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00063765
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2003-07-03
Brief Title:
Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the Eye
Sponsor:
National Eye Institute NEI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase I Study of NT-501-10 and NT-501-6A02 Implants of Encapsulated Human NTC-210 Cells Releasing Ciliary Neurotrophic Factor CNTF in Patients With Retinitis Pigmentosa
Status:
COMPLETED
Status Verified Date:
2006-03
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 evaluate the safety of a ciliary neurotrophic factor CNTF implant placed in the eye to allow the release of CNTF directly on the retina The results of this study may lead to a larger investigation of CNTF implants to treat retinitis pigmentosa RP a progressive degenerative eye disease that begins with loss of peripheral vision and night blindness and often leads to blindness in later life
Currently there are no effective treatments for RP Researchers have found however that certain proteins called ciliary neurotrophic factor CNTF can partially protect cells in the eye if given directly inside the eye A major challenge in treating RP is to deliver medicine directly into the eye One way to ensure that CNTF gets into the eye is to surgically place an implant inside the eye to release the protein
Patients 18 years of age and older with retinitis pigmentosa whose visual acuity is 20100 or worse may be eligible for this study Candidates will be screened with a medical history physical examination eye examinations and eye photographs The eye examination includes measurement of visual acuity and eye pressure examination of the pupils and eye movements and examination of the lens and back of the eye In addition patients will have the following tests
Visual field test Patients look at a central spot on a white screen and tell the examiner whenever they see a small light appear at other places on the screen
Electroretinogram ERG Electrodes are taped to the patients forehead Special contact lenses are placed on the eyes similar to normal contact lenses after the eye has been numbed with drops The contact lenses sense small electrical signals generated by the retina The ERG measures the electrical activity of the retina when it is stimulated by light For the ERG recording the patient looks inside a large hollow dark sphere and sees flashes of light first in the dark and then with a light turned on in the sphere
Optical coherence tomography This test done with the machine used to examine the eye measures retinal thickness by producing cross-sectional pictures of the retina
Participants undergo surgery at the NIH Clinical Center in a 30-minute operation to place the implant in one eye The surgery is done under local anesthetic Before the procedure patients are given a steroid injection along side the eye to minimize inflammation after surgery Following the procedure patients return for follow-up visits once a month for 6 months At these visits several of the exams described above are repeated to evaluate treatment effects and check for adverse side effects After 6 months the implant is surgically removed Post-surgical care for both implant and explant surgeries include examinations the day and week after surgery to examine the wound a high dose of steroid immediately after surgery oral antibiotics for 7 days and eye drops for 1 week to prevent infection and inflammation
Currently there are no effective treatments for RP Researchers have found however that certain proteins called ciliary neurotrophic factor CNTF can partially protect cells in the eye if given directly inside the eye A major challenge in treating RP is to deliver medicine directly into the eye One way to ensure that CNTF gets into the eye is to surgically place an implant inside the eye to release the protein
Patients 18 years of age and older with retinitis pigmentosa whose visual acuity is 20100 or worse may be eligible for this study Candidates will be screened with a medical history physical examination eye examinations and eye photographs The eye examination includes measurement of visual acuity and eye pressure examination of the pupils and eye movements and examination of the lens and back of the eye In addition patients will have the following tests
Visual field test Patients look at a central spot on a white screen and tell the examiner whenever they see a small light appear at other places on the screen
Electroretinogram ERG Electrodes are taped to the patients forehead Special contact lenses are placed on the eyes similar to normal contact lenses after the eye has been numbed with drops The contact lenses sense small electrical signals generated by the retina The ERG measures the electrical activity of the retina when it is stimulated by light For the ERG recording the patient looks inside a large hollow dark sphere and sees flashes of light first in the dark and then with a light turned on in the sphere
Optical coherence tomography This test done with the machine used to examine the eye measures retinal thickness by producing cross-sectional pictures of the retina
Participants undergo surgery at the NIH Clinical Center in a 30-minute operation to place the implant in one eye The surgery is done under local anesthetic Before the procedure patients are given a steroid injection along side the eye to minimize inflammation after surgery Following the procedure patients return for follow-up visits once a month for 6 months At these visits several of the exams described above are repeated to evaluate treatment effects and check for adverse side effects After 6 months the implant is surgically removed Post-surgical care for both implant and explant surgeries include examinations the day and week after surgery to examine the wound a high dose of steroid immediately after surgery oral antibiotics for 7 days and eye drops for 1 week to prevent infection and inflammation
Detailed Description:
Retinitis Pigmentosa RP is a group of incurable degenerative diseases of the retina that have a complex molecular etiology Approximately 100000 Americans suffer from inherited retinal degenerative RP More than 100 RP-inducing mutations have been identified in several genes including rhodopsin the rod visual pigment peripherin a membrane structure protein and PDEB the beta subunit of rod cyclic GMP cGMP phosphodiesterase However the genotype is unknown for the majority of patients Despite this genetic heterogeneity there tends to be a common pattern of visual loss in patients with RP Typically patients experience disturbance in night vision early in life due to the degeneration of rod photoreceptors The remaining cone photoreceptors become their mainstay of vision but over the years and decades the cones slowly degenerate leading to blindness These two phases of degeneration in the visual life of an RP patient may involve different underlying pathogenic mechanisms Regardless of the initial causative defects the end results are photoreceptor degeneration This common pathogenesis pathway provides a target for therapeutic intervention
To date there are few available effective treatments for retinal degenerative disorders One major challenge is to deliver potential therapeutic agents to the back of the eye in particular to the retina The blood-eye barrier prevents the penetration of a variety of molecules to the neurosensory retina in a similar manner to the action of the blood-brain barrier which exists between the central nervous system and systemic circulation To overcome this challenge Neurotech USA Inc Neurotech developed encapsulated cell technology ECT specifically the NT-501-10 and NT-501-6A02 devices to enable controlled sustained delivery of therapeutic agents directly into the intra-ocular fluids and thus providing direct access to the retina ECT utilizes cells encapsulated within a semi-permeable polymer device that secretes therapeutic factors directly into the vitreous In addition ECT devices can be retrieved providing an added level of safety
Histopathologic studies have demonstrated the possibility of growth factors neurotrophic factors and cytokines as therapeutics for RP Specifically ciliary neurotrophic factor CNTF has proven to be the most effective in reducing retinal degeneration Therefore the use of implanted NT-501-10 and NT-501-6A02 devices which secrete CNTF into the retina may be beneficial in patients with RP and other retinal degenerative diseases
This pilot study will assess the ophthalmic and systemic safety and to some extent efficacy of the novel intra-ocular NT-501-10 and NT-501-6A02 implants in patients with RP and poor visual acuity in one eye The main purpose of the study is to assess the safety of the NT-501-10 and NT-501-6A02 implants Secondary outcomes will include the anterior chamber cell scale and vitreous haze grading to measure inflammation which may be caused by the implant Other secondary outcome measures related to potential product performance are visual acuity visual fields electroretinograms ERG and optical coherence tomography OCT3 to determine retinal thickness
To date there are few available effective treatments for retinal degenerative disorders One major challenge is to deliver potential therapeutic agents to the back of the eye in particular to the retina The blood-eye barrier prevents the penetration of a variety of molecules to the neurosensory retina in a similar manner to the action of the blood-brain barrier which exists between the central nervous system and systemic circulation To overcome this challenge Neurotech USA Inc Neurotech developed encapsulated cell technology ECT specifically the NT-501-10 and NT-501-6A02 devices to enable controlled sustained delivery of therapeutic agents directly into the intra-ocular fluids and thus providing direct access to the retina ECT utilizes cells encapsulated within a semi-permeable polymer device that secretes therapeutic factors directly into the vitreous In addition ECT devices can be retrieved providing an added level of safety
Histopathologic studies have demonstrated the possibility of growth factors neurotrophic factors and cytokines as therapeutics for RP Specifically ciliary neurotrophic factor CNTF has proven to be the most effective in reducing retinal degeneration Therefore the use of implanted NT-501-10 and NT-501-6A02 devices which secrete CNTF into the retina may be beneficial in patients with RP and other retinal degenerative diseases
This pilot study will assess the ophthalmic and systemic safety and to some extent efficacy of the novel intra-ocular NT-501-10 and NT-501-6A02 implants in patients with RP and poor visual acuity in one eye The main purpose of the study is to assess the safety of the NT-501-10 and NT-501-6A02 implants Secondary outcomes will include the anterior chamber cell scale and vitreous haze grading to measure inflammation which may be caused by the implant Other secondary outcome measures related to potential product performance are visual acuity visual fields electroretinograms ERG and optical coherence tomography OCT3 to determine retinal thickness
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-0234 | None | None | None |