A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa

Study Overview
=================
Brief 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 one's 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. 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.

Detailed Description
-----------------
This study will involve about 18 visits over 2½ 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 visit for the surgical procedures. The primary effectiveness outcome for this study will be a visual field score one year after the implant surgery. There will be about 12 centers participating in this study, and up to 60 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 each eye, one of which will 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 retinitis pigmentosa (RP).

Official Title
-----------------
A Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Field Sensitivity as the Primary Outcome

Conditions
-----------------
Retinitis Pigmentosa

Intervention / Treatment
-----------------
* Drug: NT-501
* Drug: NT-501


Participation Criteria
=================
Eligibility Criteria
-----------------
Study inclusion / exclusion criteria: Criteria for patients to qualify for the study include, but are not limited to: Over 18 years of age, and less than 65 years of age Diagnosis of retinitis pigmentosa, Usher Syndrome Type 2 or 3 or Choroideremia Visual acuity no worse than 20/63 Experience with at least two full threshold Humphrey Visual Field 30-2 tests, one completed within the year prior to enrolling in this study 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. Retinitis pigmentosa caused by a classic syndrome, including Usher Type I Other eye diseases including advanced cataract. Chronic systemic disease requiring continuous treatment with systemic steroids, immunosuppressive medications or insulin.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 64 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1 NT-501 implant<br>High Dose | Drug: NT-501<br>* High Dose<br>* Other names: CNTF implant;|
| Experimental: 2 NT-501 implant<br>Low Dose | Drug: NT-501<br>* Low Dose<br>* Other names: CNTF implant;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Humphrey visual field sensitivity from baseline to month 12. |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in visual field sensitivity |  | 24 months | 
| Mean, median and distribution of change in Best Corrected Visual Acuity |  | 12 to 24 months | 
| Change in ERG |  | 12 to 24 months | 
| Change in OCT |  | 12 to 24 months | 
| Change in inflammation |  | 12 to 24 months | 
| Change in vision-related quality of life |  | 12 to 24 months | 

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