NCT06942520招募中2 期
A Randomized, Open Label, Controlled, Phase 2 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 AAV Gene Therapy Administered Via Subretinal Delivery in Participants With Center Involved Diabetic Macular Edema
Sierra Eye Associates1 个研究点 分布在 1 个国家目标入组 18 人开始时间: 2025年3月18日最近更新:
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Mean change from baseline in BCVA in the study eye at Week 52
研究概览
简要总结
Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)
详细描述
This Phase 2 trial is an open label, randomized, active controlled, dose-ranging trial to allow for preliminary safety and efficacy data on subretinal (SR) delivery of RGX-314 in participants with CI - DME
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 25 Years 至 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Type I or Type II diabetes mellitus with center involving diabetic macular edema with previous treatment
- •HbA1c ≤12%
- •BCVA in study eye 78 to 25 ETDRS letters (~20/32 to 20/320) at screening
- •Decreased visual acuity attributable primarily due to CI - DME
- •Demonstrate clinical response to aflibercept injection in the study eye
- •Provide written informed consent
排除标准
- •Women of childbearing potential
- •Neovascularization in the study eye from a cause other than DR
- •Evidence in the study eye of optic nerve pallor on clinical examination
- •History of pan retinal photocoagulation in the study eye
- •Any concurrent ocular condition in the study eye other than CI-DME that could require surgical intervention within 6 months or any condition in the study eye that may increase the risk to the participant, require either medical or surgical intervention during the study to prevent or treat vision loss, or interfere with the study procedures or assessments
- •Presence of an implant in the study eye at screening (excluding intraocular lens)
- •Any condition in the investigator's opinion that could limit VA improvement in the study eye
- •Active or history of glaucoma, steroid response, or ocular hypertension
- •Any prior intravitreal steroid injection in the study eye within 6 months prior to screening, administration in the study eye of Ozudrex® within 12 months prior to screening, or administration in the study eye if Iluven® within 36 months prior to screening
- •Diabetic macular edema diagnosis ≥ 7 years
- •History of chronic renal failure requiring dialysis or kidney transplant
- •Participation in any other gene therapy study or receipt of any investigational product within 30 days prior to enrollment or 5 half-lives of the investigational product, whichever is longer, or any plans to use an investigational product within 6 months following enrollment
- •Note: Other inclusion/exclusion criteria apply
研究组 & 干预措施
RGX-314 Treatment Arm (Dose 1)
Experimental
Genetic: RGX-314 Dose 1
- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
干预措施: RGX-314 Dose 1 (Genetic)
Treatment Arm (Dose 2)
Experimental
Genetic: RGX-314 Dose 2
-AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
干预措施: RGX-314 Dose 2 (Genetic)
Aflibercept Treatment Arm
Active Comparator
Biological: Aflibercept IVT
- Commercially available Active Comparator
- Other Names: Eylea
干预措施: Aflibercept (2.0 mg) (Biological)
结局指标
主要结局
Mean change from baseline in BCVA in the study eye at Week 52
时间窗: 52 Weeks
To evaluate the effect of RGX-314 on BCVA at Week 52
次要结局
- Mean change from baseline in BCVA in the study eye over time(52 Weeks)
- Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time(52 Weeks)
- Mean change from baseline in central subfield thickness (CST) in the study at week 52(52 Weeks)
- Mean change from baseline in CST in the study eye on SD-OCT at Week 26 and Week 52(52 Weeks)
- Proportion of participants with an absence of CI-DME in the study eye at Week 26 and Week 52(52 Weeks)
- Aqueous RGX-314 transgene (TP) concentration at assessed time points(52 Weeks)
- Proportion of participants with visual stability in the study eye from baseline to Week 52(52 Weeks)
- Proportion of participants with vision gain or vision loss ≥ to 5, 10, or 15 ETDRS letters in the study eye from baseline to Week 52(52 Weeks)
- Proportion of participants achieving a 2-step or greater improvement or any improvement (a 1-step or greater improvement) in DR in the study eye by ETDRS DRSS on 4 widefield digital stereoscopic fundus photography at Week 52(52 Weeks)
- Incidences of overall and ocular adverse events (AEs)(52 Weeks)
- Proportion of participants requiring any additional intervention for ocular diabetic complications to Week 52(52 Weeks)
- Proportion of participants with any sight-threatening ocular diabetic complications to Week 52(52 Weeks)
- Proportion of participants developing ocular diabetic complications requiring treatment per standard of care (anti-VEGF treatment, steroid treatment, pan retinal photocoagulation, laser, or surgery) through Week 52(52 Weeks)
- Proportion of participants developing ocular diabetic complications (e.g., neovascularization due to DR) requiring treatment per standard of care (pan retinal photocoagulation or anti-VEGF treatment) through Week 52(52 Weeks)
- Proportion of participants developing ocular diabetic complications (e.g., retinal detachments) requiring surgical intervention per standard of care through Week 52(52 Weeks)
研究者
研究点 (1)
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