An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 11
- 主要终点
- Safety as Assessed by Adverse Event Reporting
研究概览
简要总结
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
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
- •Have an area of the central 5 mm zone of cornea affected, presence of at least one clinical sign observed by slit-lamp examination with white light and/or cobalt blue light, fluorescein, and a Wratten filter, and at least one clinical sign observed with AS-OCT.
- •If dosing with Acthar at time of Screening Visit, must have a history of stable dosing for approximately 8 weeks, plan to maintain the same Acthar treatment dose and regimen, and have demonstrated little to no improvement in severity of LSCD.
- •Sponsor written confirmation of qualifying LSCD diagnosis.
- •Inflammation associated with LSCD or other non-infectious inflammation that will not interfere with subject's participation.
排除标准
- •Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye.
- •Ocular surgery within 30 days of Day 0 where investigator assesses that healing is not considered complete/stable.
- •Planned ocular surgery on or before Week 20 visit.
- •Presence or history of any ocular or systemic disorder, condition, or procedure that might hinder the efficacy of the study treatment or its evaluation, or could interfere with the interpretation of study results, in the opinion of the investigator.
- •Note: Other inclusion/exclusion criteria apply.
研究组 & 干预措施
Control
Period I: Subjects will be observed (non-interventional) for 20 weeks. Period II: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144.
干预措施: CSB-001 Ophthalmic Solution 0.1% (Biological)
CSB-001 QID
Period I: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 20. Period II: Subject will be observed (non-interventional) for 28 weeks to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144.
干预措施: CSB-001 Ophthalmic Solution 0.1% (Biological)
结局指标
主要结局
Safety as Assessed by Adverse Event Reporting
时间窗: Day 0 through Week 96
Incidence of ocular and systemic adverse events
Safety as Assessed by Slit-lamp Biomicroscopy
时间窗: Day 0 through Week 96
Change from baseline in slit-lamp parameters
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
时间窗: Day 0 through Week 96
Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
Safety as Assessed by Adverse Event Reporting
时间窗: Day 0 through Week 144
Incidence of ocular and systemic adverse events
Safety as Assessed by Slit-lamp Biomicroscopy
时间窗: Day 0 through Week 144
Change from baseline in slit-lamp parameters
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
时间窗: Day 0 through Week 144
Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
次要结局
- Efficacy as Assessed by Change in Stage of LSCD(Day 0 through Week 96)
- Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity(Day 0 through Week 96)
- Efficacy as Assessed by Change in Stage of LSCD(Day 0 through Week 144)
- Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity(Day 0 through Week 144)