Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
- 试验地点
- 14
- 主要终点
- Change in BCVA
研究概览
简要总结
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment
详细描述
The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment.
At study start subjects will be randomized to one of 3 treatment groups with either active study drug or sham treatment.
Sepofarsen (QR-110) will be administered via intravitreal (IVT) injection into the subject's treatment eye (the subject's worse eye).
Subjects in the sham-procedure group will undergo a procedure that will closely mimic the active injection.
After each dosing subjects will be assessed for safety and tolerability at follow up visits.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Group 1: Dose 1 sepofarsen (QR-110)
Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated
干预措施: sepofarsen (Drug)
Group 2: Dose 2 sepofarsen (QR-110)
Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated
干预措施: sepofarsen (Drug)
Group 3: Sham
Sham procedure (no experimental drug administered), Day 1, month 3 and every six months there after. After 12 months cross over to active study drug may be initiated
干预措施: Sham (Other)
结局指标
主要结局
Change in BCVA
时间窗: 12 months
Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
次要结局
- Change in FST light sensitivity(12 and 24 months)
- Change from baseline in BCVA ≤ -0.3 LogMAR(12 and 24 months)
- Change in oculomotor instability (OCI)(12 and 24 months)
- Change in LLVA(12 and 24 months)
- Change in FAF(12 and 24 months)
- Change in mobility course score(12 and 24 months)
- Change in BCVA based on FrACT(12 and 24 months)
- Ocular and non-ocular AEs(12 and 24 months)
- Clinical meaningful improvement in subjects with BCVA ≤ 1.7 LogMAR(12 and 24 months)
- Change in patient reported visual function via VFQ-25 (adults)(12 and 24 months)
- Systemic exposure to QR-110(12 and 24 months)
- Change in ellipsoid zone (EZ) width/area assessed by SD-OCT(12 and 24 months)
- Changes in microperimetry(12 and 24 months)
- Change in patient reported visual function via CVAQC (pediatrics)(12 and 24 months)
- Change in the Patient Global Impressions of Severity (PGI-S)(12 and 24 months)
- Change in the Patient Global Impressions of Change (PGI-C)(12 and 24 months)