NCT04945772已完成2 期
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]
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 7
- 主要终点
- Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
盲法说明
Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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).
结局指标
主要结局
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
时间窗: Week 52
Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.
次要结局
- 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)
- Efficacy of MCO-010 as assessed by mobility testing.(Weeks 16,24,32,52,76,100)
研究者
研究点 (7)
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相关资讯
FDA Accepts Nanoscope's BLA for MOGENRY, an Optogenetic Gene Therapy for Severe Vision Loss in Retinitis Pigmentosa- The FDA has accepted and filed Nanoscope Therapeutics' Biologics License Application for MOGENRY (sonpiretigene isteparvovec, MCO-010) in retinitis pigmentosa with severe vision loss.
- The filing is supported by the Phase 1/2a trial and the RESTORE Phase 2b/3 sham-controlled trial, which met its primary and key secondary endpoints at weeks 52 and 76.
- MOGENRY is a one-time, in-office intravitreal optogenetic gene therapy that requires no genetic testing or surgical suite and showed no treatment-related serious adverse events.
- If approved, MOGENRY would become the first gene-agnostic therapy to improve vision in RP patients with severe vision loss, who currently have no approved therapy.8 days agoNanoscope'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.last yearNanoscope 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.last yearGene 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.last yearNanoscope'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.last year
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