Status:
COMPLETED
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
Lead Sponsor:
Nanoscope Therapeutics Inc.
Conditions:
Stargardt Disease
Eligibility:
All Genders
16+ years
Phase:
PHASE2
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 fl...
Eligibility Criteria
Inclusion
- ≥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
- 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.
Key Trial Info
Start Date :
July 5 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 28 2023
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT05417126
Start Date
July 5 2022
End Date
September 28 2023
Last Update
November 9 2023
Active Locations (2)
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1
Nanoscope Clinical Site
Miami, Florida, United States, 33136
2
Nanoscope Clinical Site
McAllen, Texas, United States, 78503