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

Purpose

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.

Condition	Intervention	Phase
Retinitis Pigmentosa	Drug: NT-501	Phase 2 Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Outcomes Assessor) Primary Purpose: Treatment
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 Acuity as the Primary Outcome

Resource links provided by NLM:

Genetics Home Reference related topics: Lenz microphthalmia syndrome oculofaciocardiodental syndrome Peters plus syndrome retinitis pigmentosa X-linked juvenile retinoschisis

MedlinePlus related topics: Eye Diseases

U.S. FDA Resources

Further study details as provided by Neurotech Pharmaceuticals:

Primary Outcome Measures:

The primary outcome is the change in best-corrected visual acuity (BCVA) using the Electronic Visual Acuity (EVA) technology at month 12. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Longer-term observations of change in visual acuity, disease modification, BCVA, ERG, optical coherence tomography, inflammation, and vision-related quality of life(NEI-VFQ25). [ Time Frame: 18 months ] [ Designated as safety issue: No ]

Estimated Enrollment:	60
Study Start Date:	January 2007
Study Completion Date:	October 2009
Primary Completion Date:	October 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 1 NT-501 High Dose Implant	Drug: NT-501 High Dose
Experimental: 2 NT-501 Low Dose Implant	Drug: NT-501 Low Dose

Detailed Description:

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.

Eligibility

Ages Eligible for Study:	18 Years to 68 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00447993

Locations

United States, California
Retina-Vitreous Associates Medical Group
Beverly Hills, California, United States, 90211
University of Califoria, Davis
Sacramento, California, United States, 95817
University of California, San Francisco
San Francisco, California, United States, 94143-0730
United States, Florida
Retina Group of Florida
Hollywood, Florida, United States, 33021-6746
University of Florida
Jacksonville, Florida, United States, 32216-1480
United States, Massachusetts
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States, 02114
United States, Michigan
Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455-0501
United States, New York
NY University Medical Center
New York, New York, United States, 10016
United States, Oregon
Casey Eye Institue
Portland, Oregon, United States, 97239-4197
United States, Tennessee
The Hamilton Eye Institute
Memphis, Tennessee, United States, 38163
United States, Texas
Retina Foundation of Southwest
Dallas, Texas, United States, 75231
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84112

Sponsors and Collaborators

Neurotech Pharmaceuticals

Investigators

Study Director:

Weng Tao, M.D., PhD

Neurotech Pharmaceuticals

More Information

No publications provided by Neurotech Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

Responsible Party:	Weng Tao, MD, PhD, Neurotech USA
ClinicalTrials.gov Identifier:	NCT00447993 History of Changes
Other Study ID Numbers:	CNTF 3
Study First Received:	March 9, 2007
Last Updated:	April 12, 2011
Health Authority:	United States: Food and Drug Administration

Keywords provided by Neurotech Pharmaceuticals:

Retinitis Pigmentosa
eye disease
retinal disorder
CNTF

Additional relevant MeSH terms:

Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases

Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 18, 2013