A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol A

Study Overview
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Brief Summary
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This study is a phase 3, randomized, multi-center study to evaluate the efficacy and safety of the NT-501 implants in participants with macular telangiectasia type 2.

Official Title
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A Phase III Multicenter Randomized, Sham Controlled, Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2

Conditions
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Macular Telangiectasia Type 2

Intervention / Treatment
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* Combination Product: NT-501
* Procedure: Sham


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Participant must have at least one study eye with a positive diagnosis of MacTel Type 2 Participant must have an Inner Segment - Outer Segment Junction Line (IS/OS) Photo Receptor (PR) break in the study eye(s) and en face EZ (area of IS/OS loss) as measured by spectral-domain optical coherence tomography (SDOCT) between 0.16 mm^2 and 2.00 mm^2 Participant's best corrected visual acuity is 54 letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart Key Exclusion Criteria: Participant received intravitreal steroid therapy for non-neovascular MacTel within the last 3 months Participant 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 in the fellow eye Participant 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 Participant was a study participant in any other clinical trial of an intervention (drug or device) within the last 6 months Participant is pregnant or breastfeeding Participant has a chronic requirement (eg ≥ 4 weeks at a time) for ocular medications

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NT-501<br> | Combination Product: NT-501<br>* Surgery to receive one NT-501 device implant.<br>|
| Sham Comparator: Sham<br> | Procedure: Sham<br>* Non-penetrating sham procedure to mimic implant procedure.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of Change in Ellipsoid Zone (EZ) Area Loss | 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. | Baseline through 24 months. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Change in Aggregate Sensitivity of Microperimetry | NT-501 compared to sham relative to mean change in aggregate sensitivity of microperimetry within the EZ line break area. | Baseline through 24 months. | 
| Mean Change in Reading Speed | NT-501 compared to sham relative to mean change in reading speed assessed using International Reading Speed Texts (IReST) cards. | Baseline through 24 months. | 
| National Eye Institute-Visual Function Questionnaire (NEI-VFQ) | NT-501 compared to sham relative to mean change in the NEI-VFQ near activities subscale score. | Baseline through 24 months. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Ciliary Neurotrophic Factor (CNTF), Macular Telangiectasia (MacTel)