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

Nanoscope Therapeutics Inc.27 enrolled

Overview

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).

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

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

Interventions

Gene Therapy Product-MCO-010BIOLOGICAL

The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Sham InjectionPROCEDURE

Sham Injection

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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).

Locations (6)

Nanoscope Clinical Site

Beverly Hills, California, United States

Nanoscope Clinical Site

Pensacola, Florida, United States

Nanoscope Clinical Site

Fargo, North Dakota, United States

Nanoscope Clinical Site

Houston, Texas, United States

Outcomes

Primary Outcomes

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.

Time frame: Week 52

Secondary Outcomes

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.

Time frame: Week 76

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.

Time frame: Weeks 16,24,32,52,76,100

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.

Time frame: Weeks 16,24,32,52,76,100

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.

Time frame: Weeks 16,24,32,52,76,100

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.

Time frame: Weeks 16,24,32,52,76,100

Data from ClinicalTrials.gov

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