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

Completed · Phase 2

Conditions studied: Stargardt Disease

In brief

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

Key facts

Study ID

NCT05417126

Run by

Nanoscope Therapeutics Inc.

People needed

6

Starts

2022-07-05

Expected to finish

2023-09-28

Last updated by the study team

2023-11-09

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

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..
• Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
• 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.
• Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
• Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
• Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
• Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

Where it is running

• Nanoscope Clinical Site — Miami, Florida, United States
• Nanoscope Clinical Site — McAllen, Texas, United States

Full record on ClinicalTrials.gov

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