Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
研究概览
简要总结
This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events (safety).
详细描述
20 patients with severe to total Limbal Stem Cell Deficiency (LSCD) in one eye attributable to injury or ocular surgery. The first 5 eyes will receive the cLSCs to determine the feasibility and safety. Then the subsequent 15 eyes will be randomized into cLSC group and a scleral lens treatment (SCL) control group in a 2:1 ratio in blocks of 3 or 6 using a random number generator.
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
- 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
- •Main inclusion Criteria:
- •Male or female ≥18 years of age.
- •Best corrected visual acuity in the affected eye of 20/160 or less.
- •Documentation of a LSCD diagnosis and the central cornea is affected.
- •Absence of lagophthalmos and eyelid abnormality
- •Adequate forniceal depth is ≥ 5 mm.
- •LSCD fails to resolve by surgical treatments of the ocular surface during the previous 6 months of screening visit.
- •If the etiology of LSCD is due to chemical injury, a minimal interval of 1 year since the initial chemical injury is required.
- •A Schirmer test result at 5 minute of ≥5 mm of wetting.
- •Absence of active infectious keratitis in either eye at the Enrollment Visit.
- •Have a life expectancy ≥ 2 years after enrollment.
排除标准
- •Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
- •Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- •Exposure keratopathy or lagophthalmos of the study eye.
- •Persistent severe ocular surface inflammation and/or meibomian gland dysfunction
- •Chemical injury occurred less than 12 months ago.
- •Presence of ocular surface tumor.
- •Uncontrolled diabetes with last hemoglobin A1C (HgA1C) >8.
- •Presence of known allergies to any of the cLSC components.
- •Current participation in another simultaneous medical investigation or trial.
- •Unable to be compliant with or complete the requirements of the study.
研究组 & 干预措施
Cultivated Limbal Stem-Cells (cLSC)
One dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter.
干预措施: cLSC (Biological)
Scleral Contact Lens Device (SCL)
Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface.
干预措施: Scleral contact lens (SCL) (Device)
结局指标
主要结局
Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
时间窗: 12 months for all participants
Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.
Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation
时间窗: 12 months for all participants
Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.
Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination
时间窗: 12 months for all participants
Feasibility assessed by successful cLSC manufacturing without contamination.
次要结局
- Changes in the Clinical Score(At 6 months and 12 months in both cLSC and the control groups)
- Changes in the Area of Corneal Epithelial Defect(At 6 months and 12 months in both cLSC and the control groups)
研究者
Sophie Deng, MD, PhD
Professor
University of California, Los Angeles