NCT04945772
Completed
Phase 2
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]
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Retinitis Pigmentosa
- Sponsor
- Nanoscope Therapeutics Inc.
- Enrollment
- 27
- Locations
- 1
- Primary Endpoint
- Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
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
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.
Investigators
Eligibility Criteria
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).
Outcomes
Primary Outcomes
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
- Efficacy of MCO-010 as assessed by mobility testing.(Weeks 16,24,32,52,76,100)
- Efficacy of MCO-010 as assessed by static shape recognition assay.(Weeks 16,24,32,52,76,100)
- Efficacy of MCO-010 as assessed on visual field.(Weeks 16,24,32,52,76,100)
- Efficacy of MCO-010 as assessed by best corrected visual acuity.(Week 76)
Study Sites (1)
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Related News
Nanoscope's MCO-010 Receives FDA RMAT and Five EMA Orphan Designations for Retinal Dystrophies- The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to MCO-010 for Stargardt disease, building on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa and Stargardt disease.
- The European Medicines Agency awarded five Orphan designations to MCO-010 covering non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies.
- MCO-010 is a one-time intravitreal optogenetic therapy that activates bipolar retinal cells to restore vision without requiring genetic testing or surgical intervention.
- Nanoscope has initiated a rolling BLA submission following positive Phase 2b/3 RESTORE trial results in retinitis pigmentosa and plans Phase 3 trials for Stargardt disease in 2025.Nanoscope Therapeutics Showcases MCO-010 Optogenetic Therapy Data at ASGCT 2025- Nanoscope Therapeutics presented multiple research presentations at ASGCT 2025 highlighting the safety and efficacy of MCO-010 optogenetic therapy for retinal degenerative diseases.
- The company reported positive therapeutic benefits in Stargardt disease patients similar to durable vision gains previously observed in retinitis pigmentosa patients.
- MCO-010 represents the first mutation-agnostic therapy shown to restore vision in advanced RP patients via a one-time intravitreal injection.
- The company plans to initiate BLA submission for MCO-010 to treat RP in 2025 following positive Phase 2b RESTORE trial results.Gene and Cell Therapy Updates: FDA Actions, Clinical Advancements, and Strategic Acquisitions- Abeona Therapeutics has resubmitted its BLA to the FDA for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB) treatment.
- Nanoscope Therapeutics' MCO-010 gene therapy demonstrated improved visual acuity in retinitis pigmentosa (RP) patients in a Phase 2b trial compared to placebo.
- Lyell Immunopharma will acquire ImmPACT Bio, prioritizing development of CD19/CD20-directed CAR-T therapy IMPT-314 for large B-cell lymphoma.Nanoscope's MCO-010 Gene Therapy Shows Sustained Vision Improvement in Retinitis Pigmentosa Trial- Nanoscope Therapeutics' MCO-010 gene therapy significantly improved visual acuity in retinitis pigmentosa patients compared to the control group in a Phase 2b trial.
- The RESTORE trial demonstrated statistically significant and durable vision improvement over a two-year period, with some patients showing clinically meaningful gains.
- MCO-010 was generally well-tolerated, with the most common adverse events being anterior chamber cell and ocular hypertension, which were manageable.
- Nanoscope plans to submit a rolling Biologics License Application (BLA) to the FDA for MCO-010 in early 2025, aiming for a mutation-agnostic treatment for severe vision loss.Nanoscope Plans BLA Submission for MCO-010 Gene Therapy in Retinitis Pigmentosa- Nanoscope Therapeutics plans to submit a Biologics License Application (BLA) to the FDA in Q1 2025 for MCO-010 to treat retinitis pigmentosa.
- MCO-010, a gene-agnostic optogenetic therapy, has shown significant vision improvement in advanced RP patients in Phase 2b/3 RESTORE trial.
- New data shows MCO-010 arrests retinal degeneration in animal models, suggesting a disease-modifying effect in addition to vision restoration.
- The FDA has granted Fast Track designation to MCO-010 for both RP and Stargardt disease, and a Phase 3 trial for Stargardt is planned.