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

Plain-language summary

This study examines the safety and effects of vMCO-010, an optogenetic gene therapy given as a single injection into the eye (intravitreal), in people with the inherited eye disease Stargardt disease. It included people aged 16 and older with a clinical or genetic diagnosis (pathogenic variants in the ABCA4, ELOVL4 or PROM1 genes) whose vision fell within a defined range. The study was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Nanoscope Therapeutics Inc.

Intervention

Gene Therapy-vMCO-010

Start date

7/5/2022

Estimated completion

9/28/2023

Countries

United States

Related genes

Locations

• Miami, United States
• McAllen, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.