Clinical trial NCT00447980: A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinitis Pigmentosa
Keywords: Retinitis Pigmentosa
eye disease
retinal disorder
CNTF
Lead sponsor: Neurotech Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2007-03-15
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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.

Primary outcomes:
measure: Change in Humphrey Visual Fields - Total Sensitivity
timeFrame: 12 months
Secondary outcomes:
measure: Change in Mean Humphrey Visual Fields Sensitivity
timeFrame: Baseline compared to 6, 12, 18, 24 and 30 months
measure: Mean Humphrey Visual Fields Sensitivity
timeFrame: Baseline compared to 6, 12, 18, 24 and 30 months
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Retina-Vitreous Associates Medical Group
state: California
city: Sacramento
country: United States
facility: University of Califoria, Davis
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Miami
country: United States
facility: Bascom Palmer Eye Insitute
state: Florida
city: Ann Arbor
country: United States
facility: Kellogg Eye Center
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New York
country: United States
facility: NY University Medical Center
state: New York
city: Portland
country: United States
facility: Casey Eye Institue
state: Oregon
city: Memphis
country: United States
facility: The Hamilton Eye Institute
state: Tennessee
city: Dallas
country: United States
facility: Retina Foundation of Southwest
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Salt Lake City
country: United States
facility: The University of Utah - John A. Moran Eye Center
state: Utah
Study officials:
affiliation: Retina Foundation of the Southwest
name: David Birch, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.