A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa

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 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.

Detailed Description
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This study will involve about 16 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 visit for the surgical procedures. The primary effectiveness outcome for this study will be a visual acuity score one year after the implant surgery. There will be about 13 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 RP.

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

Conditions
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Retinitis Pigmentosa

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Criteria for patients to qualify for the study include, but are not limited to: Over 18 years of age, and less than 68 years of age Diagnosis of retinitis pigmentosa, Usher Syndrome Type 2 or 3 or Choroideremia Visual acuity no better than 20/63 and no worse than 20/320 Reduced electrical responses from the retina (ERG) and loss of peripheral vision Exclusion Criteria: 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
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Minimum Age: 18 Years
Maximum Age: 68 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 NT-501<br>High Dose Implant | Drug: NT-501<br>* High Dose<br>* Other names: CNTF implant;|
| Experimental: 2 NT-501<br>Low Dose Implant | Drug: NT-501<br>* Low Dose<br>* Other names: CNTF implant;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The primary outcome is the change in best-corrected visual acuity (BCVA) using the Electronic Visual Acuity (EVA) technology at month 12. |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Longer-term observations of change in visual acuity, disease modification, BCVA, ERG, optical coherence tomography, inflammation, and vision-related quality of life(NEI-VFQ25). |  | 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, Retinitis Pigmentosa