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

NCT ID: NCT04945772

Last Updated: 2024-03-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-07-13

Study Completion Date

2024-01-18

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Retinitis Pigmentosa Retinitis Retinal Diseases Eye Diseases Eye Diseases, Hereditary Retinal Dystrophies Retinal Degeneration

Keywords

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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 Visual Acuity Multi-Luminance Y Mobility Test (MLYMT) Multi-Luminance Shape Discrimination Test (MLSDT)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Following a 1:1:1 block randomization schema, 9 subjects will be enrolled in each MCO-010 treatment group, and 9 subjects will be enrolled in the sham-controlled group.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.

Study Groups

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MCO-010- High Dose

Participants receive 1.2E11gc/eye of MCO-010

Group Type EXPERIMENTAL

Gene Therapy Product-MCO-010

Intervention Type BIOLOGICAL

The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

MCO-010- Low Dose

Participants receive 0.9E11gc/eye of MCO-010

Group Type EXPERIMENTAL

Gene Therapy Product-MCO-010

Intervention Type BIOLOGICAL

The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Sham Injection

Participants receive sham injection

Group Type SHAM_COMPARATOR

Sham Injection

Intervention Type PROCEDURE

Sham Injection

Interventions

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Gene Therapy Product-MCO-010

The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Intervention Type BIOLOGICAL

Sham Injection

Sham Injection

Intervention Type PROCEDURE

Eligibility Criteria

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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).
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Nanoscope Therapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Dr Samarendra Mohanty

Role: STUDY_CHAIR

Nanoscope Therapeutics Inc.

Locations

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Nanoscope Clinical Site

Beverly Hills, California, United States

Site Status

Nanoscope Clinical Site

Pensacola, Florida, United States

Site Status

Nanoscope Clinical Site

Fargo, North Dakota, United States

Site Status

Nanoscope Clinical Site

Houston, Texas, United States

Site Status

Nanoscope Clinical Site

McAllen, Texas, United States

Site Status

Nanoscope Clinical Site

Arecibo, , Puerto Rico

Site Status

Countries

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United States Puerto Rico

Other Identifiers

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NTXMCO-002.

Identifier Type: -

Identifier Source: org_study_id