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

Retinitis Pigmentosa Clinical Trial

— RESTORE  

Official title:

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]

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04945772
• Other study ID #: NTXMCO-002.
• Status: Completed
• Phase: Phase 2
• Start date: July 13, 2021
• Est. completion date: January 18, 2024

Study information

• Verified date: March 2024
• Source: Nanoscope Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 27
• Est. completion date: January 18, 2024
• Est. primary completion date: February 27, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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).

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
Puerto Rico	Nanoscope Clinical Site	Arecibo
United States	Nanoscope Clinical Site	Beverly Hills	California
United States	Nanoscope Clinical Site	Fargo	North Dakota
United States	Nanoscope Clinical Site	Houston	Texas
United States	Nanoscope Clinical Site	McAllen	Texas
United States	Nanoscope Clinical Site	Pensacola	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Nanoscope Therapeutics Inc.

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Efficacy of MCO-010 as assessed by a composite of functional assessments.	Proportion of subjects demonstrating a =2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52.	Week 52
Other	Safety of MCO-010.	Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs).	100 weeks
Primary	Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.	Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.	Week 52
Secondary	Efficacy of MCO-010 as assessed by best corrected visual acuity.	Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76.	Week 76
Secondary	Efficacy of MCO-010 as assessed by mobility testing.	Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome.	Weeks 16,24,32,52,76,100
Secondary	Efficacy of MCO-010 as assessed by mobility testing.	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.	Weeks 16,24,32,52,76,100
Secondary	Efficacy of MCO-010 as assessed by static shape recognition assay.	Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome.	Weeks 16,24,32,52,76,100
Secondary	Efficacy of MCO-010 as assessed by static shape recognition assay.	Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome.	Weeks 16,24,32,52,76,100
Secondary	Efficacy of MCO-010 as assessed on visual field.	Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry.	Weeks 16,24,32,52,76,100