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

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

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

Study Overview

• Status: Completed
• Conditions: Stargardt Disease
• Intervention / Treatment: Biological: Gene Therapy-vMCO-010

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.

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • Nanoscope Clinical Site
  • Texas
    • McAllen, Texas, United States, 78503
      • Nanoscope Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. ≥16 years of age
2. Able to comprehend and give informed consent.
3. Able to comply with testing and all protocol tests.
4. 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
5. 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.
6. Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening

Exclusion Criteria:

1. 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).
2. 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.
3. 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
4. Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
5. Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
6. Resting heart rate outside specified limits upon repeated measurement.
7. History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
8. Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
9. Any major surgical procedure within one month of trial entry or anticipated during the trial.
10. Clinically significant abnormal lab results at screening
11. 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
12. In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
13. 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).
14. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
15. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..
16. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
17. Presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including OCT, during the study period.
18. Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
19. Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
20. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
21. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental-vMCO-010; Participants receive 1.2E11gc/eye of vMCO-010	Biological: Gene Therapy-vMCO-010; The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of vMCO-010 as assessed by visual acuity	Change from baseline in BCVA at Weeks 12, 24, 48 in the study eye and the fellow eye	48 weeks
Effect of vMCO-010 on Light-guided Mobility	Change from baseline in Multi-Luminance Mobility Test at weeks 12, 24, 48 in the study eye and the fellow eye	48 Weeks
Effect of vMCO-010 on determination of shape	Change from baseline in accuracy in determination of shape using Low Vision Multi-Parameter Test (LVMPT) at weeks 12, 24, 48 in the study eye and the fellow eye	48 Weeks
Effect of vMCO-010 on determination of optical flow	Change from baseline in accuracy in determination of optical flow using the LVMPT at weeks 12, 24 and 48 in the study eye and the fellow eye	48 Weeks

Collaborators and Investigators

• Sponsor: Nanoscope Therapeutics Inc.
• Investigators: Study Director: Dr Samarendra Mohanty, Nanoscope Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 5, 2022
• Primary Completion (Actual): August 2, 2023
• Study Completion (Actual): September 28, 2023

Study Registration Dates

• First Submitted: June 9, 2022
• First Submitted That Met QC Criteria: June 9, 2022
• First Posted (Actual): June 14, 2022

Study Record Updates

• Last Update Posted (Estimated): November 9, 2023
• Last Update Submitted That Met QC Criteria: November 8, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophy; Retinal Degeneration
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Macular Degeneration; Stargardt Disease
• Other Study ID Numbers: NTXMCO-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.
• IPD Sharing Time Frame: Within 12 Months of study completion
• IPD Sharing Access Criteria: Safety and efficacy Results

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No