Phase 2
N=134
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
Retinitis Pigmentosa
Bottom Line
View on ClinicalTrials.gov: NCT00447993 ↗Enrolled (actual)
134
Serious AEs
2.3%
Results posted
Aug 2024
Primary outcome: Primary: The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12. — 1; 1; 3; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Low Dose NT-501 (Combination_product); High Dose NT-501 (Combination_product)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Neurotech Pharmaceuticals
- Primary completion
- May 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12. |
1; 1; 3; 1; 20; 20 | — |
| SECONDARY Number of Participants With and Without Best Corrected Visual Acuity Response Over the 18-month Follow-up Period (Months 1, 3, 6, 12 and 18) |
1; 1; 3; 1; 21; 21 | — |
Summary
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II & III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect our ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision.
The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. This study will look at the effect of the implant on vision loss by retinitis pigmentosa, Usher type II & III, and Choroideremia. In this study, two different CNTF dose levels will be used: a high dose and a low dose in one eye, as well as a sham (or placebo) surgery in the other eye.
Eligibility Criteria
Inclusion Criteria
- Participant was older than 18, but less than 68 years of age.
- Participant understood and signed the informed consent. If the participant's vision was impaired to the point where he/she could not read the informed consent document, the document would be read to the participant in its entirety.
- Females of childbearing potential (women with last menses standard 3, posterior subcapsular opacity > standard 3, or a nuclear opacity > standard 3 as measured on the AREDS clinical lens grading system.
- Participant had chronic requirement (e.g., > or =4 weeks at a time) for ocular medications or has disease(s) that in the judgment of the examining physician were vision threatening, toxic to the lens, retina, or optic nerve or might affect the primary outcome.
- Participant had a requirement of acyclovir and/or related products during study duration.
To be eligible for this study, the participant must have discontinued use of these products prior to enrollment and must not continue with the products until after they had completed the study.
- Participant had evidence of corneal opacification or lack of optical clarity.
- Participant had undergone lens removal in the last 3 months, with or without intra-ocular lens implantation, or had undergone intra-ocular lens replacement within 6 months prior to enrollment.
- Participant was receiving systemic steroids or other immunosuppressive medications.
- Participant had undergone LASIK surgery or other refractive surgery for either eye in less than 6 months prior to screening.
- Participant was currently participating in or had participated in any other clinical trial of a drug by ocular or systemic administration within the last 6 months.
- Participant had previous exposure to an intra-ocular device or implant into the eye (excluding intra-ocular lens).
- Participant had uveitis or other retinal inflammatory disease.
- Participant was receiving oral or other insulin treatment for diabetes.
- Participant who had a history of myocardial infarction within the last 12 months.
- Participant was pregnant or lactating.
- Participant was considered immunodeficient or had a known history of HIV. A laboratory test for HIV was performed, and a positive result was also an exclusion criterion.
- Participant with a history of ocular herpes zoster.
- Participant was on chemotherapy.
- Participant had a history of malignancy, except study participants having cancer treated successfully ≥5 years prior to inclusion in the trial.
- Participant with severe hearing disabilities in both ears.
- Participant with diabetic retinopathy in either eye.
- Participant had history of retinal detachment in either eye.
- Participant had been diagnosed and treated for amblyopia as an infant.
- Participant with a history of Pars Plana Vitrectomy.
Data sourced from ClinicalTrials.gov (NCT00447993). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.