- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01530659
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
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
Study Overview
Status
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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California
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San Francisco, California, United States, 94143
- University of California, San Francisco
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Participant must be between 18 and 55 years of age.
- Participant must have a diagnosis of retinitis pigmentosa or Usher Syndrome type 2 or 3 (without profound deafness or cochlear implants).
- 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.
- Best-corrected visual acuity must be no worse than 20/63 (at least 59 letters).
- Participants must have clear natural lenses.
- Participants must have less than 6 diopters myopia.
- 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.
- 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.
- 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.
- 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.
- Participant's clinical diagnosis must be consistent with retinal degeneration in the set of retinitis pigmentosa (RP) dystrophies.
Exclusion Criteria:
- Participant is medically unable to comply with study procedures or follow-up visits.
- 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.
- Participant has history of corneal opacification or lack of optical clarity.
- Participant has undergone LASIK surgery or other refractive surgery for either eye.
- Participant has nystagmus.
- Participant has greater than 6 diopters myopia.
- 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.
- Participant has fewer than 7 regions of interest (ROIs) present on 2 baseline AOSLO image montages.
- Participant has retinal vascular disease such as diabetic retinopathy or prior retinal vascular occlusive disease.
- 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.
- 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.
- Participant is receiving systemic steroids or other immunosuppressive medications.
- 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.
- Participant has previous exposure to an intra-ocular device or implant into the eye (excluding intra-ocular lens).
- Participant has uveitis or other retinal inflammatory disease.
- Participant has a history of myocardial infarction within the last 12 months.
- Participant is pregnant or lactating.
- 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.
- Participant with a history of ocular herpes zoster.
- Participant is on chemotherapy.
- Participant has a history of malignancy, except study participant with cancer treated successfully ≥5 years prior to inclusion in the trial.
- Participant with severe hearing disabilities in both ears.
- Participant who has been diagnosed and treated for amblyopia as an infant.
- Participant who, in the opinion of the study doctor, will not be a good study subject.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: DOUBLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: NT-501
Encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina
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Study participants will undergo surgery to have an NT-501 Encapsulated Cell Therapy implant placed into the study eye.
Other Names:
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SHAM_COMPARATOR: Sham
Sham surgery
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Non-penetrating sham procedure to mimic implant procedure in the other eye.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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Average of cone spacing (nearest neighbor distance) at all regions of interest with at least 50 contiguous unambiguous cones identified over the central 5.7 degrees of the macula using confocal AOSLO at two baseline visits within each eye. Cone spacing measures were converted to Z scores based on normal mean values at similar distances from the fovea from a database of 27 age-similar normal eyes. The mean of 2 baseline cone spacing Z-score values were subtracted from the cone spacing Z score values obtained at post-op month 36 A Z-score of 0 represents the mean cone spacing value at the distance from the fovea measured from 27 healthy subjects. A Z-score greater than +2 represents an abnormally increased cone spacing value at the distance from the fovea where the measurement was performed. This suggests fewer cones are present than normal at that location. |
Post-op Month 36
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Difference in logMAR Visual Acuity Change Between CNTF- and Sham Treated Eyes
Time Frame: Post-op Month 36
|
Difference in change in logMAR visual acuity between NT-501 and contralateral sham-treated eyes. Change in visual acuity was measured based on the number of letters read on a vision chart using a standard protocol. The log of the mean angle of resolution (logMAR) was used to describe the size of the smallest letters that the patient could read. A logMAR value of 0.00 corresponds to visual acuity of 20/20, a logMAR value of 0.3 corresponds to visual acuity of 20/40, a logMAR value of 0.7 corresponds to visual acuity of 20/100, and a logMAR value of 1.00 corresponds to visual acuity of 20/200. |
Post-op Month 36
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Collaborators and Investigators
Sponsor
Collaborators
Publications and helpful links
General Publications
- Duncan JL, Zhang Y, Gandhi J, Nakanishi C, Othman M, Branham KE, Swaroop A, Roorda A. High-resolution imaging with adaptive optics in patients with inherited retinal degeneration. Invest Ophthalmol Vis Sci. 2007 Jul;48(7):3283-91. doi: 10.1167/iovs.06-1422.
- Sieving PA, Caruso RC, Tao W, Coleman HR, Thompson DJ, Fullmer KR, Bush RA. Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants. Proc Natl Acad Sci U S A. 2006 Mar 7;103(10):3896-901. doi: 10.1073/pnas.0600236103. Epub 2006 Feb 27.
- Talcott KE, Ratnam K, Sundquist SM, Lucero AS, Lujan BJ, Tao W, Porco TC, Roorda A, Duncan JL. Longitudinal study of cone photoreceptors during retinal degeneration and in response to ciliary neurotrophic factor treatment. Invest Ophthalmol Vis Sci. 2011 Apr 6;52(5):2219-26. doi: 10.1167/iovs.10-6479.
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Eye Diseases
- Neurologic Manifestations
- Disease
- Congenital Abnormalities
- Retinal Degeneration
- Retinal Diseases
- Genetic Diseases, Inborn
- Otorhinolaryngologic Diseases
- Ear Diseases
- Eye Diseases, Hereditary
- Retinal Dystrophies
- Sensation Disorders
- Abnormalities, Multiple
- Hearing Disorders
- Vision Disorders
- Deaf-Blind Disorders
- Hearing Loss, Sensorineural
- Blindness
- Hearing Loss
- Deafness
- Syndrome
- Retinitis
- Retinitis Pigmentosa
- Usher Syndromes
Other Study ID Numbers
- AOSLO-CNTF-FFB-01
- FD-R-004100-01A1 (OTHER_GRANT: FDA OOPD)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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