Clinical Trial: Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis Pigmentosa

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Neurotech Pharmaceuticals

Current Primary Outcome: Cone photoreceptor preservation [ Time Frame: 6, 12, 18, 24 and 30 months post implant ]

Evaluation of the changes (if present)in cone photoreceptor preservation in the CNTF-treated eye vs. the sham eye as measured by AOSLO.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Safety of the implanted NT-501 investigational product [ Time Frame: 6, 12, 18, 24, 30 and 36 months post implant ]
    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, 30 and 36 months post implant ]

    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, 30 and 36 months post implant ]
    Safety will be evaluated by the presence or absence of local and/or systemic toxicities.


Original Secondary Outcome:

  • NT-503 device placement [ Time Frame: 6, 12, 18, 24 and 30 months post implant ]
    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 ]

    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 ]
    Saftey will be evaluated by the presence or absence of local and/or systemic toxicities.


Information By: Neurotech Pharmaceuticals

Dates:
Date Received: January 19, 2012
Date Started: January 2012
Date Completion: August 2019
Last Updated: October 26, 2016
Last Verified: October 2016