Skip to main content
OpenTrials
Completed

NCT Number: NCT04945772

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

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Retinitis Pigmentosa Cone-Rod Dystrophies Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Sensation Disorders Signs and Symptoms Vision Disorders Vision, Low

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Nanoscope Clinical Site, Arecibo, Puerto Rico

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 years
  • Able to comprehend and give informed consent.
  • Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
  • 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:

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

Treatment and study plan

Gene Therapy Product-MCO-010

Biological

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

Sham injection

Procedure

Sham Injection

Primary outcomes

  1. Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.

    Time frame: Week 52

    Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.

Secondary outcomes

  1. Efficacy of MCO-010 as assessed by best corrected visual acuity.

    Time frame: Week 76

    Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76.

  2. Efficacy of MCO-010 as assessed by mobility testing.

    Time frame: Weeks 16,24,32,52,76,100

    Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome.

  3. Efficacy of MCO-010 as assessed by mobility testing.

    Time frame: Weeks 16,24,32,52,76,100

    Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome.

  4. Efficacy of MCO-010 as assessed by static shape recognition assay.

    Time frame: Weeks 16,24,32,52,76,100

    Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome.

  5. Efficacy of MCO-010 as assessed by static shape recognition assay.

    Time frame: Weeks 16,24,32,52,76,100

    Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome.

  6. Efficacy of MCO-010 as assessed on visual field.

    Time frame: Weeks 16,24,32,52,76,100

    Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry.

Other outcomes

  1. Efficacy of MCO-010 as assessed by a composite of functional assessments.

    Time frame: Week 52

    Proportion of subjects demonstrating a ≥2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52.

  2. Safety of MCO-010.

    Time frame: 100 weeks

    Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs).

Sponsors and collaborators

Lead sponsor

Nanoscope Therapeutics Inc.

Industry

Registry information

Official study title

A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]

Acronym: RESTORE

Important dates

Study start
2021
Primary completion
2023
Study completion
2024
First posted
Jun 30, 2021
Registry last updated
Mar 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.