NCT05417126

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

A Phase 2a, Open Label Multicenter Clinical Trial to Evaluate the Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Nanoscope Therapeutics Inc. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease

Detailed description

This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010.

Conditions

• Stargardt Disease

Interventions

Type	Name	Description
BIOLOGICAL	Gene Therapy-vMCO-010	The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Timeline

• Start date: 2022-07-05
• Primary completion: 2023-08-02
• Completion: 2023-09-28
• First posted: 2022-06-14
• Last updated: 2023-11-09

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study