NCT04945772

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Nanoscope Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).

Detailed description

This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.

Conditions

• Retinitis Pigmentosa
• Retinitis
• Retinal Diseases
• Eye Diseases
• Eye Diseases, Hereditary
• Retinal Dystrophies
• Retinal Degeneration

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
PROCEDURE	Sham Injection	Sham Injection

Timeline

• Start date: 2021-07-13
• Primary completion: 2023-02-27
• Completion: 2024-01-18
• First posted: 2021-06-30
• Last updated: 2024-03-22

Locations

6 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study