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

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

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.

Study Overview

• Status: Completed
• Conditions: Retinitis Pigmentosa
• Intervention / Treatment: Drug: NT-501; Drug: NT-501

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).

• Study Type: Interventional
• Enrollment (Actual): 68
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Beverly Hills, California, United States, 90211
      • Retina-Vitreous Associates Medical Group
    • Sacramento, California, United States, 95817
      • University of Califoria, Davis
    • San Francisco, California, United States, 94143-0730
      • University of California, San Francisco
  • Florida
    • Miami, Florida, United States, 33101
      • Bascom Palmer Eye Insitute
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Ophthalmic Consultants of Boston
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Kellogg Eye Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455-0501
      • University of Minnesota
  • New York
    • New York, New York, United States, 10016
      • NY University Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239-4197
      • Casey Eye Institue
  • Tennessee
    • Memphis, Tennessee, United States, 38163
      • The Hamilton Eye Institute
  • Texas
    • Dallas, Texas, United States, 75231
      • Retina Foundation of Southwest
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • University of Utah

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 64 years (ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 1 NT-501 implant; High Dose	Drug: NT-501; High Dose; Other Names: CNTF implant; Drug: NT-501; Low Dose; Other Names: CNTF implant
EXPERIMENTAL: 2 NT-501 implant; Low Dose	Drug: NT-501; High Dose; Other Names: CNTF implant; Drug: NT-501; Low Dose; Other Names: CNTF implant

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Neurotech Pharmaceuticals
• Investigators: Study Director: Weng Tao, MD, PhD, Neurotech Pharmaceuticals

Publications and helpful links

General Publications

• Birch DG, Weleber RG, Duncan JL, Jaffe GJ, Tao W; Ciliary Neurotrophic Factor Retinitis Pigmentosa Study Groups. Randomized trial of ciliary neurotrophic factor delivered by encapsulated cell intraocular implants for retinitis pigmentosa. Am J Ophthalmol. 2013 Aug;156(2):283-292.e1. doi: 10.1016/j.ajo.2013.03.021. Epub 2013 May 10.
• Kauper K, McGovern C, Sherman S, Heatherton P, Rapoza R, Stabila P, Dean B, Lee A, Borges S, Bouchard B, Tao W. Two-year intraocular delivery of ciliary neurotrophic factor by encapsulated cell technology implants in patients with chronic retinal degenerative diseases. Invest Ophthalmol Vis Sci. 2012 Nov 1;53(12):7484-91. doi: 10.1167/iovs.12-9970.
• Talcott KE, Ratnam K, Sundquist SM, Lucero AS, Lujan BJ, Tao W, Porco TC, Roorda A, Duncan JL. Longitudinal study of cone photoreceptors during retinal degeneration and in response to ciliary neurotrophic factor treatment. Invest Ophthalmol Vis Sci. 2011 Apr 6;52(5):2219-26. doi: 10.1167/iovs.10-6479.

Study record dates

Study Major Dates

• Study Start: January 1, 2007
• Primary Completion (ACTUAL): July 1, 2010
• Study Completion (ACTUAL): July 1, 2010

Study Registration Dates

• First Submitted: March 9, 2007
• First Submitted That Met QC Criteria: March 14, 2007
• First Posted (ESTIMATE): March 15, 2007

Study Record Updates

• Last Update Posted (ESTIMATE): November 16, 2016
• Last Update Submitted That Met QC Criteria: November 15, 2016
• Last Verified: November 1, 2016

More Information

Terms related to this study

• Keywords: Retinitis Pigmentosa; eye disease; CNTF; retinal disorder
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinitis; Retinitis Pigmentosa
• Other Study ID Numbers: CNTF 4