Open-label, Single-arm, Multicenter Study to Assess the Safety of Cystadrops® in Pediatric Cystinosis Patients From 6 Months to Less Than 2 Years Old
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 5
- 试验地点
- 6
- 主要终点
- All Adverse Events that required discontinuation/withdrawal of IMP
研究概览
简要总结
Cystadrops® is currently indicated in adults and children from 2 years of age diagnosed with cystinosis with corneal crystal accumulation observed.
However administration of Cystadrops® in patients below 2 years old could be of value for these patients and prevent the crystal deposit. It is the reason why as part of the Cystadrops® pediatric investigational plan (PIP), RECORDATI Rare Diseases committed to conduct a clinical study to assess Cystadrops® safety and efficacy in the pediatric population from 6 months to less than 2 years old.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 2 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient aged from 6 months to less than 2 years old
- •Cystinosis diagnosed patients confirmed by the physician and with presence of corneal cystine crystal deposits assessed during ophthalmic examination
- •Evidence of a signed and dated informed consent document indicating that parents/ legally acceptable representatives had been informed of all pertinent aspects of the study (if required by regulation)
- •Parents/ legally acceptable representatives who are willing to comply with regular visits and ophthalmic exams
排除标准
- •Contraindications to any of the Cystadrops® components
- •Participation in another ophthalmic investigational study or intent to participate during the course of the study
- •Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives
研究组 & 干预措施
Open-label arm
干预措施: Mercaptamine (Drug)
结局指标
主要结局
All Adverse Events that required discontinuation/withdrawal of IMP
时间窗: Over a 90-day period
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
时间窗: Over a 90-day period
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
时间窗: Over a 90-day period
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
次要结局
- Ophtalmologic assessments (Corneal Cystine Crystal Score)(90-day period)
- Ophtalmologic assessments (Best Corrected Visual Acuity)(90-day period)
- Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)(90-day period)