Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

• Register: ClinicalTrials.gov
• Last refreshed on: 20 February 2023
• Main ID: NCT01530659
• Date of registration: 19/01/2012
• Prospective Registration: No
• Primary sponsor: Neurotech Pharmaceuticals
• Public title: Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
• Scientific 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
• Date of first enrolment: January 2012
• Target sample size: 22
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01530659
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jacque Duncan, MD
• Affiliation: University of California, San Francisco

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Usher Syndrome Type 3

Retinitis Pigmentosa

Usher Syndrome Type 2

Intervention(s)

Procedure: Sham

Drug: NT-501

Primary Outcome(s)

Mean Change in Cone Spacing in Arcminutes (Z Score) of 2 Baseline Values Were Compared With Measurements Obtained at Post-op Month 36 [Time Frame: Post-op Month 36]

Secondary Outcome(s)

Difference in logMAR Visual Acuity Change Between CNTF- and Sham Treated Eyes [Time Frame: Post-op Month 36]

Secondary ID(s)

AOSLO-CNTF-FFB-01

FD-R-004100-01A1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

University of California, San Francisco

Results

• Results available: Yes
• Date Posted: 14/02/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01530659