A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late 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 Acuity as the Primary Outcome
1 other identifier
interventional
67
1 country
11
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 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Jan 2007
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 8, 2007
CompletedFirst Submitted
Initial submission to the registry
March 9, 2007
CompletedFirst Posted
Study publicly available on registry
March 15, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 26, 2009
CompletedStudy Completion
Last participant's last visit for all outcomes
May 26, 2009
CompletedResults Posted
Study results publicly available
August 13, 2024
CompletedAugust 13, 2024
July 1, 2024
2.4 years
March 9, 2007
April 28, 2022
July 23, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12.
The primary efficacy endpoint was the proportion of patients demonstrating an improvement in best-corrected visual acuity (BCVA), defined as an increase of 10 letters or more at the 1-Year post-implant visit (pre-explant, if explant is completed). No response is defined as an improvement of \<10 letters.
12 months post-implant
Secondary Outcomes (1)
Number of Participants With and Without Best Corrected Visual Acuity Response Over the 18-month Follow-up Period (Months 1, 3, 6, 12 and 18)
From initial implant to 18 months post-implant
Study Arms (2)
Low Dose NT-501
EXPERIMENTALNT-501 Low Dose Implant: encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina Sham Implant in Fellow Eye
High Dose NT-501
EXPERIMENTALNT-501 High Dose Implant: encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina Sham Implant in Fellow Eye
Interventions
Low Dose NT-501 Implanted in study eye and fellow eye received sham surgery
High Dose NT-501 Implanted in study eye and fellow eye received sham surgery
Eligibility Criteria
You may qualify if:
- 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.
- intraretinal bone-spicule'-like pigment observed in clinical examination (not necessarily applicable to choroideremia).
- peripheral visual field constriction documented on standard testing.
- symptomatic night blindness.
- Each eye had a visual acuity score of at least 24 (20/320) and no more than 63 (20/63) letters.
- Each eye had an ERG amplitude reduced below the 95% limit of normal (CI) per site by Full Field 28-32 Hz flicker.
You may not qualify if:
- 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.
- To be eligible for this study, the participant must have discontinued use of these products prior to enrollment and must not continue with the products until after they had completed the study.
- Participant had evidence of corneal opacification or lack of optical clarity.
- Participant had undergone lens removal in the last 3 months, with or without intra-ocular lens implantation, or had undergone intra-ocular lens replacement within 6 months prior to enrollment.
- Participant was receiving systemic steroids or other immunosuppressive medications.
- Participant had undergone LASIK surgery or other refractive surgery for either eye in less than 6 months prior to screening.
- Participant was currently participating in or had participated in any other clinical trial of a drug by ocular or systemic administration within the last 6 months.
- Participant had previous exposure to an intra-ocular device or implant into the eye (excluding intra-ocular lens).
- Participant had uveitis or other retinal inflammatory disease.
- +10 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Retina-Vitreous Associates Medical Group
Beverly Hills, California, 90211, United States
University of Califoria, Davis
Sacramento, California, 95817, United States
University of California, San Francisco
San Francisco, California, 94143-0730, United States
Retina Group of Florida
Hollywood, Florida, 33021-6746, United States
University of Florida
Jacksonville, Florida, 32216-1480, United States
Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
University of Minnesota
Minneapolis, Minnesota, 55455-0501, United States
NY University Medical Center
New York, New York, 10016, United States
Casey Eye Institue
Portland, Oregon, 97239-4197, United States
The Hamilton Eye Institute
Memphis, Tennessee, 38163, United States
Retina Foundation of Southwest
Dallas, Texas, 75231, United States
Related Publications (2)
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.
PMID: 23668681DERIVEDKauper 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.
PMID: 23049090DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- CMO
- Organization
- Neurotech Pharmaceuticals, LLC
Study Officials
- PRINCIPAL INVESTIGATOR
David Birch, MD
Retina Foundation of the Southwest
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 9, 2007
First Posted
March 15, 2007
Study Start
January 8, 2007
Primary Completion
May 26, 2009
Study Completion
May 26, 2009
Last Updated
August 13, 2024
Results First Posted
August 13, 2024
Record last verified: 2024-07
Data Sharing
- IPD Sharing
- Will not share