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

Brief Title

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

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

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 Description

      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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Cone photoreceptor preservation

Secondary Outcome

 Safety of the implanted NT-501 investigational product

Condition

Retinitis Pigmentosa

Intervention

NT-501

Study Arms / Comparison Groups

 NT-501
Description:  Encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

30

Start Date

January 2012

Completion Date

August 2019

Primary Completion Date

August 2019

Eligibility Criteria

        Inclusion Criteria:

          1. Participant must be between 18 and 55 years of age.

          2. Participant must have a diagnosis of retinitis pigmentosa or Usher Syndrome type 2 or
             3 (without profound deafness or cochlear implants).

          3. Participant must understand and sign the protocol informed consent. If the
             participant's vision is impaired to the point where he/she cannot read the informed
             consent document, the document will be read to the participant in its entirety.

          4. Best-corrected visual acuity must be no worse than 20/63 (at least 59 letters).

          5. Participants must have clear natural lenses.

          6. Participants must have less than 6 diopters myopia.

          7. Participants must be medically able to undergo ophthalmic surgery for the NT-501
             device insertion and able to undergo all assessments and tests associated with the
             protocol.

          8. Females of childbearing potential (women with last menses <1 year prior to screening)
             must agree to use an effective form of birth control from study onset until they
             complete the study.

          9. Participants must have reproducible baseline AOSLO image at 2 baseline imaging
             sessions with quality suitable to identify a minimum of 7 regions of interest (ROIs)
             at which reliable cone spacing and/or density measures can be made over the central
             5.7 degrees.

         10. Participants must have interocular symmetry of disease severity as measured by cone
             spacing, with a difference of less than 2 standard deviations in average cone spacing
             z-scores at the selected ROIs between the 2 eyes.

         11. Participant's clinical diagnosis must be consistent with retinal degeneration in the
             set of retinitis pigmentosa (RP) dystrophies.

        Exclusion Criteria:

          1. Participant is medically unable to comply with study procedures or follow-up visits.

          2. Participant who has 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; or participant is pseudophakic or
             aphakic.

          3. Participant has history of corneal opacification or lack of optical clarity.

          4. Participant has undergone LASIK surgery or other refractive surgery for either eye.

          5. Participant has nystagmus.

          6. Participant has greater than 6 diopters myopia.

          7. Participant has cystoid macular edema with cysts present within 4 degrees of the
             foveal center that prevent acquisition of at least 7 regions of interest with clear
             images of cone photoreceptors.

          8. Participant has fewer than 7 regions of interest (ROIs) present on 2 baseline AOSLO
             image montages.

          9. Participant has retinal vascular disease such as diabetic retinopathy or prior retinal
             vascular occlusive disease.

         10. Participant has chronic requirement (e.g., ≥4 weeks at a time) for ocular medications
             or has disease(s) that in the judgment of the examining physician are vision
             threatening, toxic to the lens, retina, or optic nerve or may affect the primary
             outcome.

         11. Participant has a requirement of acyclovir and/or related products during study
             duration. To be eligible for this study, the participant must discontinue use of these
             products prior to enrollment and must not continue with the products until after they
             have completed the study.

         12. Participant is receiving systemic steroids or other immunosuppressive medications.

         13. Participant is currently participating in or has participated in any other clinical
             trial of a drug by ocular or systemic administration within the last 6 months.

         14. Participant has previous exposure to an intra-ocular device or implant into the eye
             (excluding intra-ocular lens).

         15. Participant has uveitis or other retinal inflammatory disease.

         16. Participant has a history of myocardial infarction within the last 12 months.

         17. Participant is pregnant or lactating.

         18. Participant is considered immunodeficient or has a known history of HIV. A laboratory
             test for HIV will be performed, and a positive result is also an exclusion criterion.

         19. Participant with a history of ocular herpes zoster.

         20. Participant is on chemotherapy.

         21. Participant has a history of malignancy, except study participant with cancer treated
             successfully ≥5 years prior to inclusion in the trial.

         22. Participant with severe hearing disabilities in both ears.

         23. Participant who has been diagnosed and treated for amblyopia as an infant.

         24. Participant who, in the opinion of the study doctor, will not be a good study subject.
      

Gender

All

Ages

18 Years - 55 Years

Accepts Healthy Volunteers

No

Contacts

Jacque Duncan, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01530659

Organization ID

AOSLO-CNTF-FFB-01

Secondary IDs

FD-R-004100-01A1

Responsible Party

Sponsor

Study Sponsor

Neurotech Pharmaceuticals

Collaborators

 University of California, San Francisco

Study Sponsor

Jacque Duncan, MD, Principal Investigator, University of California, San Francisco


Verification Date

November 2018