Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

Study Overview
=================
Brief Summary
-----------------
The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.

Detailed Description
-----------------
This study is designed to follow subjects with Stargardt Disease (SD) previously enrolled in study NTXMCO-004 (STARLIGHT, NCT05417126). In that study, all 6 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT). MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for STARLIGHT and are not repeated herein

Official Title
-----------------
Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study

Conditions
-----------------
Stargardt Disease

Intervention / Treatment
-----------------
* Biological: Gene Therapy Product-MCO-010


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Subjects are eligible to be included in the study only if all of the following criteria apply: Previously enrolled in study NTXMCO-004 study Able to comprehend and give informed consent. Able to comply with testing and all protocol tests. Agree to participate for the full 4-year study duration to the best of their ability and barring any unforeseen circumstances. Exclusion Criteria: Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at the Week 48 visit for the STARLIGHT study

Ages Eligible for Study
-----------------
Minimum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: Gene Therapy Product-MCO-010|The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of the long-term safety profile of a single intravitreal injection of MCO-010 | Assessment of incidences, nature, and severity of treatment emergent adverse events (TEAEs), serious adverse events (SAEs); intraocular inflammation graded through ocular exam; clinical examination, and intraocular pressure. | 204 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of the long-term effects on visual acuity of a single intravitreal injection of MCO-010; 1.2E11 gc/eye | Change from baseline in BCVA over time in the study eye and fellow eye | 204 weeks | 
| Exploration of the long-term impact of MCO-010 on retinal anatomy | Assessment of fundus photography and optical coherence tomography (OCT) outcomes over time | 204 weeks | 
| Assessment of the long-term durability of MCO-010 induced on gene reporter expression | Change from baseline of fundus fluorescence intensity of reporter (mCherry) over time in the study eye and fellow eye | 204 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Eye Diseases, Retinal Degeneration, Retinal Dystrophy, Eye Diseases, Hereditary