A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection

Study Overview
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Brief Summary
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This study A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection

Detailed Description
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The observational study population comprises of individuals who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection in a parent study. Enrolled subjects will undergo safety and efficacy assessments for up to 5 years from the vMCO-I administration. At the visits, subjects will have safety and efficacy evaluations. Subjects will have a final visit at 60 months post study drug administration.

Official Title
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A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection

Conditions
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Retinitis Pigmentosa, Retinal Disease, Retinal Degeneration

Intervention / Treatment
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* Biological: Gene Therapy product:vMCO-I


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The subject population includes subjects who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I), administered via intravitreal injection in a parent study. Exclusion Criteria: Subjects who will not consent for study.

Ages Eligible for Study
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Minimum Age: 18 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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Observation of Participants exposed to 1.2E11gc/eye of vMCO-I<br>This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of vMCO-I No investigational product will be administered in this study. | Biological: Gene Therapy product:vMCO-I<br>* Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients<br>|
| Observation of Participants exposed to 0.6E11gc/eye of vMCO-I<br>This is a long-term follow-up observational study of participants who previously received 0.6E11gc/eye of vMCO-I No investigational product will be administered in this study. | Biological: Gene Therapy product:vMCO-I<br>* Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I | Assessments of Incidences, nature, and severity of treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal anatomy | 20 Months | 
| Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I) | Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score) | 20 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of the effect of vMCO-I on functional vision outcomes | Assessment of the treatment effect on the quality of Life with changes from baseline in activities of daily living using the National Eye Institute (NEI) Visual Function Questionnaire-25 | 20 Months | 
| Assessment of the durability of vMCO-I induced gene reporter expression | Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry) | 20 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Optogenetics, Gene Therapy, AAV Vectors, Multicharacteristic Opsin (MCO)-I