A Study of an Encapsulated Cell Technology (ECT) Implant for Patients With Atrophic Macular Degeneration

Study Overview
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Brief Summary
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The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in participants with atrophic macular degeneration. This research is being done because there are no effective therapies for people with atrophic macular degeneration. Age-related macular degeneration (AMD) is a condition that affects the macula, the central part of the retina that we use for seeing details. There are two types of AMD, one is the wet type in which new blood vessels grow, also known as choroidal neovascularization (CNV), but the other is the dry type in which the healthy cells die, and that is the target of this study. This is called atrophic macular degeneration. 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. In this study, two different CNTF dose levels will be used: a high dose and a low dose, as well as a sham surgery (or placebo) group.

Detailed Description
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The study will involve about 18 visits over 1½ years for specific tests of the participant's vision and health. These visits may include visual exams, blood draw for laboratory testing, brief medical history and exam, and occasionally a questionnaire (survey), in addition to the visits for the surgical procedures. There will be about 8 centers participating in this study, and up to 48 people enrolled, across the US. Each participant joining the study who has completed initial screening will then be scheduled to have a brief surgical procedure performed on one eye, which may or may not include a very small cell-filled implant. Follow-up visits for repeat assessments will be required regularly to determine if the implant being tested is safe and effective for use to treat AMD.

Official Title
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A Phase II Study of Implants of Encapsulated Human NTC-201 Cells Releasing Ciliary Neurotrophic Factor (CNTF), in Participants With Visual Acuity Impairment Associated With Atrophic Macular Degeneration

Conditions
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Macular Degeneration

Intervention / Treatment
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* Drug: NT-501 implant
* Drug: NT-501 implant
* Other: Sham Procedure


Participation Criteria
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Eligibility Criteria
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Study inclusion / exclusion criteria: Criteria for patients to qualify for the study include, but are not limited to: Participants must be at least 50 years old Diagnosis of age-related macular degeneration with the presence of geographic atrophy Visual acuity no better than 20/63 and no worse than 20/160 History of recent visual acuity loss The following criteria will exclude patients from the study: Pregnant or lactating females, or females planning to become pregnant during the study or not using an acceptable method of contraception. Age-related macular degeneration with new blood vessel growth Other eye diseases including advanced cataract. Chronic systemic disease requiring continuous treatment with systemic steroids, immunosuppressive medications or insulin.

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1 high dose NT-501 implant<br> | Drug: NT-501 implant<br>* High Dose<br>|
| Experimental: 2 low dose NT-501 implant<br> | Drug: NT-501 implant<br>* Low Dose<br>|
| Sham Comparator: 3 sham procedure<br>No implant | Other: Sham Procedure<br>* Sham Procedure<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The increase in best-corrected visual acuity (BCVA) using the Electronic Visual Acuity (EVA) technology from baseline to 1 year. |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The mean, median, and distribution of change in BCVA over the 18-month follow-up period |  | 18 months | 
| change in ERG between baseline and months 12 and 18 |  | 12 and 18 months | 
| change in area of geographic atrophy from baseline to months 12 and 18 |  | 12 and 18 months | 
| change in area of drusen from baseline to months 12 and 18 |  | 12 and 18 months | 
| change in retinal thickness from baseline to months 12 and 18 measured by OCT |  | 12 and 18 months | 
| and change in quality of life between baseline and months 12 and 18 using NEI-VFQ25. |  | 12 and 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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eye disease, retinal disorder, CNTF