NCT06048185

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

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

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 6 (estimated)
• Sponsor: Nanoscope Therapeutics Inc. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

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

Conditions

• Stargardt Disease

Interventions

Type	Name	Description
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

Timeline

• Start date: 2023-10-19
• Primary completion: 2027-05-01
• Completion: 2027-07-01
• First posted: 2023-09-21
• Last updated: 2025-03-24

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study