Macular Telangiectasia Type 2
Conditions
Keywords
Ciliary Neurotrophic Factor, Macular Telangiectasia (MacTel) Type 2
Brief summary
This study is to determine the long-term levels of CNTF, and the clinical safety and efficacy of the NT-501 implants with the Medica Hollow Fiber Membrane (MHFM) in participants with macular telangiectasia type 2.
Detailed description
Two parallel randomized, controlled Phase 3 clinical trials showed that placing an NT-501 in the eyes of subjects with macular telangiectasia type 2 (MacTel) provides vision benefit with an acceptable risk profile. The NT-501 device used in these Phase 3 trials has a hollow fiber membrane (HFM) which houses the CNTF producing engineered NTC-201-6A cells. The produced CNTF passes through the membrane into the vitreous cavity of the affected eye and similarly required cell nutrients pass from the vitreous cavity through the membrane to the NTC-201-6A cells. NT-501 used in the above mentioned Phase 3 studies was manufactured with the clinical HFM (referred to as CHFM) from a single sourced supplier. Another supplier of this critical HFM component of the NT-501 has been identified. This membrane will be referred to as the Medica Hollow Fiber Membrane (MHFM). Though the NT-501 product manufactured with the MHFM has the same configuration, same RPE cells, and produces CNTF levels similar to the original NT-501 product manufactured with clinical membrane (CHFM) when tested in vivo (animal model), it has not been tested in humans. The purpose of this study is to characterize the clinical safety and efficacy of NT-501 with MHFM, consistent with the clinical safety and efficacy of NT-501 with CHFM.
Interventions
COMBINATION_PRODUCTNT-501 CNTF Implant
Single implantation of CNTF-secreting NT-501 hCNTF device into one eye.
Sponsors
Neurotech Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key Eligibility Criteria: 1. Subjects must have a positive diagnosis of MacTel with evidence of fluorescein leakage typical of MacTel and at least one of the other features that include hyperpigmentation that is outside of a 500 micron radius from the center of the fovea, retinal opacification, crystalline deposits, right-angle vessels, or inner/outer lamellar cavities seen clinically or on SD-OCT. 2. Subject must have a break in the ellipsoid zone (EZ) (area of IS/OS loss) as measured by SD-OCT between 0.16 and 2.00 millimeters squared. 3. Subject's best-corrected visual acuity (BCVA) is 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening. 4. Subject must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality retinal imaging. 5. Subject must be 21 years or older and less than 80 years of age at screening. 6. Subject or their legally authorized representative must be able to provide written informed consent to participate in the study, in accordance with the International Conference on Harmonisation Good Clinical Practices guidelines, and local regulations, before initiating any study-related procedures. Key
Exclusion criteria
1. Subject is medically unable to comply with study procedures or follow-up visits. 2. Subject received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months. 3. Subject has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye OR has, within the past 3 months, received intravitreal anti-VEGF therapy in the fellow eye at randomization. 4. Subject has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures, or outcome of the study (e.g., uncontrolled glaucoma, severe non-proliferative or proliferative diabetic retinopathy, uveitis). 5. Subject has a chronic requirement (e.g., ≥ 4 weeks at a time) for ocular medications and/or has a diagnosed disease that, in the judgment of the examining physician, may be vision threatening or may affect the primary outcome. The exceptions to this
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Rate of Change in Ellipsoid Zone (EZ) Area Loss | Baseline through 12 months. | Rate of change in EZ area loss as measured by en face imaging by Spectral-Domain Optical Coherence Tomography (SD-OCT) in the study eye. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety Outcomes | Baseline to 12 months | The number and severity of adverse events post-implantation. |
Countries
United States
Outcome results
None listed