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

Nanoscope Therapeutics

Status and phase: Completed; Phase 2

Conditions: Eye Diseases, Hereditary; Retinal Degeneration; Retinal Dystrophies; Retinitis Pigmentosa; Retinitis; Eye Diseases; Retinal Diseases

Treatments: Biological: Gene Therapy Product-MCO-010; Procedure: Sham Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT04945772

NTXMCO-002.

Details and patient eligibility

About

The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).

Full description

This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.

Enrollment: 27 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.

Exclusion criteria

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

1. Prior participation in gene therapy program
2. 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).
3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Triple Blind

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27 participants in 3 patient groups

MCO-010- High Dose

Experimental group

Description:

Participants receive 1.2E11gc/eye of MCO-010

Treatment:

Biological: Gene Therapy Product-MCO-010

MCO-010- Low Dose

Experimental group

Description:

Participants receive 0.9E11gc/eye of MCO-010

Treatment:

Biological: Gene Therapy Product-MCO-010

Sham Injection

Sham Comparator group

Description:

Participants receive sham injection

Treatment:

Procedure: Sham Injection