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

• Conditions: Retinal Disease; Retinitis Pigmentosa; Retinal Degeneration

• Registration Number: NCT05921162
• Lead Sponsor: Nanoscope Therapeutics Inc.

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2023-06-19
• Study First Submitted QC: 2023-06-19
• Study First Posted: 2023-06-27
• Study Start: 2023-07-14
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-21
• Last Update Posted: 2025-03-25
• Primary Completion: 2025-05
• Study Completion: 2025-05

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 11

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I	20 Months	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
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I)	20 Months	Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)

Secondary Outcome Measures

Name	Time	Method
Assessment of the effect of vMCO-I on functional vision outcomes	20 Months	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
Assessment of the durability of vMCO-I induced gene reporter expression	20 Months	Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry)

Trial Locations

Locations (1)

JPM Rotary Club of Cuttack Eye Hospital and Research Institute; 🇮🇳 Cuttack, Odisha, India