A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigm… (NCT00447993) | Clinical Trial Compass
CompletedPhase 2
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
United States67 participantsStarted 2007-01-08
Plain-language summary
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect our ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision.
The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. This study will look at the effect of the implant on vision loss by retinitis pigmentosa, Usher type II \& III, and Choroideremia. In this study, two different CNTF dose levels will be used: a high dose and a low dose in one eye, as well as a sham (or placebo) surgery in the other eye.
Who can participate
Age range
18 Years – 68 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participant was older than 18, but less than 68 years of age.
. Participant understood and signed the informed consent. If the participant's vision was impaired to the point where he/she could not read the informed consent document, the document would be read to the participant in its entirety.
. Females of childbearing potential (women with last menses \<1 year prior to screening) agreed to use an effective form of birth control from study onset until they completed the 18-month study visit.
. Participant was medically able to undergo ophthalmic surgery for the NT-501 device.
. Participant's clinical diagnosis was consistent with retinal degeneration in the set of retinitis pigmentosa (RP) dystrophies characterized by the following features:
. clinical evidence of progressive photoreceptor cell dysfunction and degeneration of the outer retina.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12.
. intraretinal bone-spicule'-like pigment observed in clinical examination (not necessarily applicable to choroideremia).
. peripheral visual field constriction documented on standard testing.
Exclusion criteria
. Participant was medically unable to comply with study procedures or follow-up visits.
. Participant had glaucoma (defined as independent optic atrophy causing vision loss), irrespective of whether it was currently treated or untreated.
. Participant had classic syndromic RP.
. Participant had optic nerve atrophy beyond modest pallor, primary cone-rod dystrophy, unilateral bulls-eye maculopathy, cystoid maculopathy as judged by OCT reading center, or other retinal dystrophy.
. Participant who had any of the following lens opacities: cortical opacity \> standard 3, posterior subcapsular opacity \> standard 3, or a nuclear opacity \> standard 3 as measured on the AREDS clinical lens grading system.
. Participant had chronic requirement (e.g., \> or =4 weeks at a time) for ocular medications or has disease(s) that in the judgment of the examining physician were vision threatening, toxic to the lens, retina, or optic nerve or might affect the primary outcome.
. Participant had a requirement of acyclovir and/or related products during study duration.
. Participant had evidence of corneal opacification or lack of optical clarity.