Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE] (RESTORE)

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring Retinitis Pigmentosa, Eye Diseases Hereditary, Eye Diseases, Retinal Degeneration, Inherited Retinal Diseases, Rod & cone dystrophies, Optogenetics, Gene Therapy, AAV vectors, Intravitreal Injections, Low Vision, Multi-Characteristic Opsin, No Light Perception, Low-Vision Multi-Parameter Test (LVMPT) Read More

Inclusion Criteria:

The subject population includes subjects with advanced RP. Subjects are eligible to be included in the study only if all of the following criteria apply:

1. Age ≥ 18 years
2. Able to comprehend and give informed consent.
3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600, Count Fingers/ Hand Motion) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.
5. Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye during screening as determined by the Investigator and confirmed by the Sponsor or designee.

Exclusion Criteria:

Subjects are excluded from the study if any of the following criteria apply:

1. Prior participation in gene therapy program
2. Individuals who refuse or are incapable of performing mobility testing or pass the mobility testing at 0.3 or 1 lux as determined by the Investigator and confirmed by the Sponsor or designee during screening, will be excluded.
3. Presence of an active implantable medical device
4. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).
5. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
6. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded.
7. Subjects who have undergone ocular surgery in the study eye within three months prior to Day 0.
8. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
9. 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.
10. Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations and assessed by the investigator to significantly affect central vision.
11. Current evidence of retinal detachment in the study eye that significantly affects central vision.
12. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
13. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
14. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).