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

Nanoscope Therapeutics Inc.1 site in 1 country6 target enrollmentJuly 5, 2022

ConditionsStargardt Disease

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Stargardt Disease
Sponsor: Nanoscope Therapeutics Inc.
Enrollment: 6
Locations: 1
Primary Endpoint: Type, severity, and incidence of ocular and systemic adverse events (AEs)
Status: Completed
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials Related News

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

July 5, 2022

End Date

September 28, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Nanoscope Therapeutics Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•≥16 years of age
•Able to comprehend and give informed consent.
•Able to comply with testing and all protocol tests.
•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 PROM1
•In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.
•Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening

Exclusion Criteria

•Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
•Received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as ARGUS-II) or sub-retinal injections.
•Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
•Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
•Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
•Resting heart rate outside specified limits upon repeated measurement.
•History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
•Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
•Any major surgical procedure within one month of trial entry or anticipated during the trial.
•Clinically significant abnormal lab results at screening