NCT03364153已完成2 期
A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 121
- 试验地点
- 41
- 主要终点
- Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).
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
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
- •Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive
排除标准
- •Macular atrophy secondary to any condition other than STGD1 in either eye
- •Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
- •Participation in an interventional study of a vitamin A derivative </= 3 months prior to screening
- •Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
- •Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
- •Diabetes mellitus
- •Hemoglobin A1c (HbA1c) value of >/=6.5%
- •Stroke within 12 months of trial entry
- •Any major surgical procedure within one month of trial entry or anticipated during the trial
- •Any treatment with an investigational agent in the past 60 days for any condition
- •Women who are pregnant or nursing
- •Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation
研究组 & 干预措施
avacincaptad pegol
Experimental
Participants will receive avacincaptad pegol monthly for up to 17 Months.
干预措施: avacincaptad pegol (Drug)
Sham
Sham Comparator
Participants will receive a matching sham monthly for up to 17 Months.
干预措施: Sham (Drug)
结局指标
主要结局
Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18
时间窗: Baseline to Month 18
The area of ellipsoid zone defect was measured by en face spectral domain-optical coherence tomography. Rate of change (slope) in the area of ellipsoid zone defect from Baseline through Month 18 was estimated using mixed model for repeated measures (MMRM).
次要结局
- Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18(Baseline and Month 18)
- Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18(Baseline and Month 18)
- Number of Participants With Adverse Events (AEs)(Up to 18 months)
研究者
研究点 (41)
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