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

Phase 2

Completed

• Conditions: Eye Diseases, Hereditary; Retinal Degeneration; Retinal Diseases; Retinal Dystrophies; Retinitis Pigmentosa; Retinitis; Eye Diseases
• Interventions: Biological: Gene Therapy Product-MCO-010; Procedure: Sham Injection

• Registration Number: NCT04945772
• Lead Sponsor: Nanoscope Therapeutics Inc.

Brief 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.

Study Timeline

• Study First Submitted: 2021-06-15
• Study First Submitted QC: 2021-06-22
• Study First Posted: 2021-06-30
• Study Start: 2021-07-13
• Primary Completion: 2023-02-27
• Study Completion: 2024-01-18
• Disposition First Submitted: 2024-02-19
• Status Verified: 2024-03
• Disposition First Posted: 2024-03-12
• Last Update Submitted: 2024-03-20
• Last Update Posted: 2024-03-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

1. Age ≥ 18 years
2. Able to comprehend and give informed consent.
3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.

Exclusion Criteria

Subjects are excluded from the study if any of the following criteria apply:

1. Prior participation in gene therapy program
2. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).
3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
MCO-010- High Dose	Gene Therapy Product-MCO-010	Participants receive 1.2E11gc/eye of MCO-010
MCO-010- Low Dose	Gene Therapy Product-MCO-010	Participants receive 0.9E11gc/eye of MCO-010
Sham Injection	Sham Injection	Participants receive sham injection

Primary Outcome Measures

Name	Time	Method
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.	Week 52	Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.

Secondary Outcome Measures

Name	Time	Method
Efficacy of MCO-010 as assessed by mobility testing.	Weeks 16,24,32,52,76,100	Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome.
Efficacy of MCO-010 as assessed by static shape recognition assay.	Weeks 16,24,32,52,76,100	Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome.
Efficacy of MCO-010 as assessed on visual field.	Weeks 16,24,32,52,76,100	Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry.
Efficacy of MCO-010 as assessed by best corrected visual acuity.	Week 76	Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76.

Trial Locations

Locations (1)

Nanoscope Clinical Site; 🇵🇷 Arecibo, Puerto Rico