A Phase 2 Multicenter Randomized Clinical Trial of CNTF for MacTel

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

Official Title
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A Phase 2 Multicenter Randomized Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel)

Conditions
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Macular Telangiectasia Type 2

Intervention / Treatment
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* Biological: Ciliary neurotrophic factor (CNTF)
* Procedure: Sham procedure
* Device: NT-501 Implant
* Procedure: NT-501 Implant procedure


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant must be offered sufficient opportunity to review and to understand the informed consent form, agree to the form's contents and sign the protocol's informed consent Participant must have at least one study eye with a positive diagnosis of MacTel Type 2 Participant must have an IS/OS PR break in the study eye(s) and en face ellipsoid zone (area of IS/OS loss) as measured by SDOCT between 0.16 mm2 and 4.00 mm2 If female, participant must be incapable of pregnancy If male, participant must agree to use an effective form of birth control during the study Exclusion Criteria: Participant is unable to provide informed consent Participant is less than 21 years of age or greater than 80 years of age Participant is medically unable to comply with study procedures or follow-up visits Participant was a study subject in any other clinical trial of an intervention (drug or device) within the last 6 months Participant is pregnant or breastfeeding

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: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NT-501 Implant procedure<br>The investigational product is the NT-501 encapsulated cell system which consists of cells encapsulated within a semi-permeable polymer membrane and supportive matrices. NT-501 contains NTC-201 cells that were derived from the NTC-200 cell line by genetic modification so as to secrete recombinant human ciliary neurotrophic factor (CNTF). | Biological: Ciliary neurotrophic factor (CNTF)<br>* Ciliary neurotrophic factor released from NT-501 encapsulated cell implant<br>Device: NT-501 Implant<br>* NT-501 encapsulated cell implant<br>Procedure: NT-501 Implant procedure<br>* Surgery to implant device for NT-501 encapsulated cell implant releasing human ciliary neurotrophic factor arm<br>|
| Sham Comparator: Sham procedure<br>Non-penetrating sham procedure to mimic implant procedure | Procedure: Sham procedure<br>* Sham surgery for Sham arm<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ellipsoid zone (area of IS/OS loss) as measured by en face imagining by SDOCT in study eye(s) | Change in the ellipsoid zone (area of IS/OS loss) from baseline to month 24 as measured by en face imaging by SDOCT in study eye(s) | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ellipsoid zone | Change in the ellipsoid zone from baseline to Month 12. | 12 months | 
| Retinal sensitivity (dB) as measured by microperimetry | Change in retinal sensitivity (dB) as measured by microperimetry from baseline to Months 12 and 24. | 12 and 24 months | 
| Increase in ellipsoid zone | Proportion of study eyes with a 35% or more increase from baseline in the ellipsoid zone at Months 12 and 24. | 12 and 24 months | 
| Visual Acuity | Change in best corrected visual acuity (BCVA) from baseline to Months 12 and 24. | 12 and 24 months | 
| Visual Acuity | Proportion of study eyes with 15 or more letter loss from baseline in BCVA at Months 12 and 24. | 12 and 24 Months | 
| Visual Acuity | Proportion of study eyes with 10 or more letter loss from baseline in BCVA at Months 12 and 24. | 12 and 24 Months | 
| Reading Speed | Change in reading speed as measured by the IReST from baseline to Months 12 and 24. | 12 and 24 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MacTel