Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCT05417126 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: NTXMCO-004
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

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

Conditions

Stargardt Disease

Keywords

Eye Diseases; Retinal Degeneration; Retinal Dystrophy; Eye Diseases, Hereditary

Outcome Measures

Primary Outcomes (1)

• Type, severity, and incidence of ocular and systemic adverse events (AEs)

  Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010

  48 weeks

Secondary Outcomes (4)

• Effect of vMCO-010 as assessed by visual acuity

  48 weeks

• Effect of vMCO-010 on Light-guided Mobility

  48 Weeks

• Effect of vMCO-010 on determination of shape

  48 Weeks

• Effect of vMCO-010 on determination of optical flow

  48 Weeks

Study Arms (1)

Experimental-vMCO-010

EXPERIMENTAL

Participants receive 1.2E11gc/eye of vMCO-010

Biological: Gene Therapy-vMCO-010

Interventions

Gene Therapy-vMCO-010 BIOLOGICAL

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

Experimental-vMCO-010

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥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

You may not qualify if:

• 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
• Known serious allergies to the fluorescein dye used in angiography or intraocular pressure measurement, povidone iodine, or to the components of the vMCO-010 formulation
• In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
• Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, history of uveitis, corneal or lenticular opacities).
• Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
• Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..

Sponsors & Collaborators

• Nanoscope Therapeutics Inc.: lead

Study Sites (2)

Nanoscope Clinical Site

Miami, Florida, 33136, United States

Location

Nanoscope Clinical Site

McAllen, Texas, 78503, United States

Location

MeSH Terms

Conditions

Stargardt Disease; Eye Diseases; Retinal Degeneration; Retinal Dystrophies; Eye Diseases, Hereditary

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Dr Samarendra Mohanty

  Nanoscope Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 6 subjects will be enrolled for vMCO-010 treatment

Study Record Dates

• First Submitted: June 9, 2022
• First Posted: June 14, 2022
• Study Start: July 5, 2022
• Primary Completion: August 2, 2023
• Study Completion: September 28, 2023
• Last Updated: November 9, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will share
• Time Frame: Within 12 Months of study completion
• Access Criteria: Safety and efficacy Results

Locations

US (2)