A Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Field Sensitivity as the Primary Outcome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 73
- 试验地点
- 12
- 主要终点
- Change in Humphrey Visual Fields - Total Sensitivity
研究概览
简要总结
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II & III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect one's ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision.
The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. In this study, two different CNTF dose levels will be used: a high dose and a low dose in one eye, as well as a sham (or placebo) surgery in the other eye.
详细描述
This study will involve about 18 visits over 2½ years for specific tests of the participant's vision and health. These visits may include visual exams, blood draw for laboratory testing, brief medical history and exam, and occasionally a questionnaire (survey), in addition to the visit for the surgical procedures. The primary effectiveness outcome for this study will be a visual field score one year after the implant surgery. There will be about 12 centers participating in this study, and up to 60 people enrolled, across the US. Each participant joining the study who has completed initial screening will then be scheduled to have a brief surgical procedure performed on each eye, one of which will include a very small cell-filled implant. Follow-up visits for repeat assessments will be required regularly to determine if the implant being tested is safe and effective for use to treat retinitis pigmentosa (RP).
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
- Double (Participant, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 64 Years（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
Low Dose
NT-501 Low Dose Implant: encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina
Sham Implant in Fellow Eye
干预措施: NT-501 (Drug)
High Dose
NT-501 High Dose Implant: encapsulated cell therapy that delivers ciliary neurotrophic factor to the retina
Sham Implant in Fellow Eye
干预措施: NT-501 (Drug)
结局指标
主要结局
Change in Humphrey Visual Fields - Total Sensitivity
时间窗: 12 months
The primary efficacy endpoint was the change in Humphrey VFS from baseline to month 12 (Visit 10) as determined by the HVF 30-2 test, comprised of 76 points. The measure was the sum of actual thresholds for all 76 locations.
次要结局
- Change in Mean Humphrey Visual Fields Sensitivity(Baseline compared to 6, 12, 18, 24 and 30 months)
- Mean Humphrey Visual Fields Sensitivity(Baseline compared to 6, 12, 18, 24 and 30 months)