A Phase 1, Prospective, Multi-Center, Open-Label, Dose-Escalation Study to Assess the Safety, and Tolerability of EO2002 with and Without Endothelial Brushing or Descemet Stripping in the Treatment of Corneal Edema (EMME-001)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 42
- 试验地点
- 8
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]
研究概览
简要总结
Corneal edema is the most common indication for corneal transplantation, accounting for approximately 70% of penetrating keratoplasty (PK), and 100% of endothelial keratoplasty (EK) procedures annually. There is currently no disease-modifying treatment for corneal edema. Topical treatments like hypertonic saline are not effective on a long-term basis. For those with advanced disease, where edema and vision loss are not controlled by topical treatment, the only option is a corneal transplant. A potential approach to avoidance of the risks of corneal transplantation is to inject cultured human corneal endothelial cells (HCECs) into the anterior chamber of the eye. This approach may avoid surgery by re-populating the inner most aspect of the cornea with functioning endothelial cells. Emmecell has developed a treatment based on technology integrating biocompatible magnetic nanoparticles with cultured HCECs to treat corneal edema in a minimally invasive way. The primary objective of this phase 1, prospective, multi-center, open-label, dose-escalation study is to evaluate the safety and tolerability of 3 doses of EO2002 with and without endothelial brushing (EB) or Descemet Stripping (DS) in eyes with corneal edema secondary to corneal endothelial dysfunction that qualify for surgery involving full-thickness corneal transplantation or EK.
详细描述
The study is designed to occur in 2 groups. The objective with the first group is to preliminarily evaluate the safety of 4 escalating doses. The purpose of the second group is to gather additional safety information on the 3 highest doses when administered according to a randomized schedule and evaluated by masked observers.
Group 1: Phase 1 prospective, multi-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of EO2002 with and without endothelial brushing (EB) or Descemet Stripping (DS) in eyes with corneal edema secondary to corneal endothelial dysfunction that qualify for surgery involving full-thickness corneal transplantation or EK. Four doses will be studied in approximately 21 study participants that meet the inclusion/exclusion criteria. Safety and tolerability will be the primary focus for 26 weeks following treatment with EO2002.
Group 2: The three highest doses will be administered to up to 21 subjects in a randomized, double-masked design.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 21 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All ocular criteria apply to study eye unless otherwise noted.
- •Age ≥ 21 years.
- •Phakic or Pseudophakic with a posterior chamber intraocular lens
- •Symptomatic corneal edema associated with endothelial dysfunction which may be secondary to Fuchs corneal dystrophy or pseudophakic bullous keratopathy.
排除标准
- •All ocular criteria apply to study eye unless otherwise noted.
- •Other corneal disease
- •Anterior chamber intraocular lens, Sutured or scleral-fixated intraocular lens.
- •Macular disease that in the investigator and/or sponsor's opinion would limit the ability of the subject to demonstrate improvement in BCVA.
- •History of refractive surgery.
- •Descemet membrane detachment.
- •History of uveitis or other ocular inflammatory disease.
- •History of incisional glaucoma surgery (e.g.,trabeculectomy, glaucoma drainage implant).
- •Intraocular pressure >21 or <7 mm Hg
- •Prior incisional eye surgery within 3 months prior to study treatment or penetrating or endothelial keratoplasty.
- •History of ocular neoplasm.
- •ETDRS BCVA in the fellow eye is worse than 35 letters (Snellen equivalent of 20/200).
- •Female who is pregnant, nursing, or planning to become pregnant, or who is of childbearing potential and not using a reliable means of contraception during the study.
- •Subject is currently participating in or has participated within the last 3 months in any other clinical trial of an investigational drug by ocular or systemic administration.
- •Any concomitant medical or psychological condition that could interfere with study participation or is otherwise not suitable for entry into the study in the opinion of the investigator.
研究组 & 干预措施
EO2002 treatment - Group 1
干预措施: EO2002 (Biological)
EO2002 treatment - Group 2 - low dose
干预措施: EO2002 low dose (Biological)
EO2002 treatment - Group 2 - mid dose
干预措施: EO2002 mid dose (Biological)
EO2002 treatment - Group 2 - high dose
干预措施: EO2002 high dose (Biological)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]
时间窗: 26 Weeks
Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability of EO2002 (+/- EB or Descemet Stripping)\] in subjects with corneal edema at week 26.
次要结局
- Effect of EO2002 (+/-EB or DS) on corneal thickness(26 Weeks)
- Effect of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA).(26 Weeks)