Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis Pigmentosa
This clinical trial is a single-site, 30 patient study for participants who have early stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). Funding Source - FDA OOPD and Foundation Fighting Blindness.
Usher Syndrome Type 2
Usher Syndrome Type 3
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Photoreceptor Structure in A Phase 2 Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Rates of Change in Cone Spacing and Density|
- Cone photoreceptor preservation [ Time Frame: 6, 12, 18, 24 and 30 months post implant ] [ Designated as safety issue: No ]Evaluation of the changes (if present)in cone photoreceptor preservation in the CNTF-treated eye vs. the sham eye as measured by AOSLO.
- NT-501 device placement [ Time Frame: 6, 12, 18, 24 and 30 months post implant ] [ Designated as safety issue: Yes ]Safety will be measured,in part, by the presence or absence of rejection or extrusion of the implanted NT-501 device.
- Change(s) in ocular function [ Time Frame: 6, 12, 18, 24 and 30 months post implant ] [ Designated as safety issue: Yes ]
Change(s) in visual acuity and change in perimetry assessed by:
- Mean, median and distribution of change in best corrected visual acuity (BCVA)
- Changes in visual field using perimetry,
- Changes in the outer nuclear layer thickness as measure by sdOCT,
- Changes in full-field electroretinography (ERG) from Baseline through 24-months post implant
- The presence of peri-implant fibrosis that blocks the visual axis or affects the lens or retina
- Adverse events affecting ocular function which are thought to be potentially related to the implant
- Toxicity [ Time Frame: 6, 12, 18, 24 and 30 months post implant ] [ Designated as safety issue: Yes ]Safety will be evaluated by the presence or absence of local and/or systemic toxicities.
|Study Start Date:||January 2012|
|Estimated Study Completion Date:||September 2016|
|Estimated Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina
Study participants will undergo surgery to have an NT-501 Encapsulated Cell Therapy implant placed into the study eye.
Other Name: CNTF, Encapsulated Cell Therapy, ECT
This clinical trial is a prospective, randomized, double-masked, sham-controlled trial of 30 study participants who have early-stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). The trial will be conducted at the University of California, San Francisco. Individuals with these diseases experience gradually worsening vision that ultimately may lead to blindness due to a genetic condition in which specialized cells in the eye's retina called photoreceptor cells cease functioning and/or die. The study is intended to use a relatively new, non-invasive technology called AOSLO (adaptive optics scanning laser ophthalmoscopy) in combination with a routine standard of care measurement called sdOCT (Spectral Domain Optical Coherence Tomography) to demonstrate that when a device that secretes an investigational drug called CNTF (Ciliary Neurotrophic Factor) is surgically placed in the patient's eye, one type of photoreceptor called "cone photoreceptors" is preserved such that the gradual loss of vision is halted and to a lesser extent, reversed.
|Contact: Arshia Mian, BSemail@example.com|
|United States, California|
|University of California, San Francisco||Recruiting|
|San Francisco, California, United States, 94143|
|Contact: Arshia Mian, BS 415-476-0444 firstname.lastname@example.org|
|Principal Investigator: Jacque Duncan, MD|
|Principal Investigator:||Jacque Duncan, MD||University of California, San Francisco|