Multicenter Study of Cultured Autologous Oral Mucosal Epithelial Cell-Sheet (CAOMECS) Transplantation to Patients With Total Limbal Stem Cell Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 试验地点
- 2
- 主要终点
- Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
研究概览
简要总结
This clinical study is a scientific study on patients who do not have any limbal stem cells. In this clinical study tissue is taken from the inside of the mouth, and cells from that tissue (epithelial cells) will be grown to form a multilayered cell-sheet, called CAOMECS, which is then transplanted onto the cornea. This transplantation method should repair the damage of the cornea.
The aim of this study is to see if the transplantation of CAOMECS renews the surface of the eye, by preventing the growth of the conjunctiva over the cornea and stopping new small blood vessels forming.
详细描述
This is an open clinical study without a control group, which means that all patients will be given the study treatment (CAOMECS).
Adults as well as minor patients can participate in this clinical trial.
CAOMECS is undergoing a clinical trial, which means that it has not yet been officially approved for the treatment of this disease. Up to now it was clinically tested in France, on 26 people with limbal stem cell loss.
Study objective
The objective of this study is to restore the ocular surface epithelium of patients with total LSCD by preventing or reducing recurrent conjunctivalization and neovascularization up to 12 months post-transplantation.
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
- 2 Years 至 74 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥2 years to 74 years
- •Males or females with bilateral or unilateral total LSCD due to one of the following causes:
- •Chemical burns
- •Thermal burns
- •Contact lens wear
- •Surgery of the ocular surface
- •Stevens-Johnson syndrome and other inflammatory disease under stable condition
- •Documented conjunctivalization of the corneal surface, measured by fluorescein staining
- •Stable disease, i.e. history of LSCD for at least 6 months
- •Clinical signs indicative of conjunctivalisation:
- •Superficial blood vessels on the corneal surface
- •Loss of epithelial transparency or persistent epithelial defect
- •Healthy oral mucosa
- •Absence from tobacco and alcohol (7 days before the biopsy)
- •Regular tooth brushing (at least twice daily)
- •Ability to comply with the protocol
- •Covered by a social security system
- •Signed informed consent form, ability to understand the study procedures, and contractual capability. Applicable to patient or legal carer (including parent)
- •Special inclusion criteria for patients between 18 and 74 years of age (adults):
- •Multiple surgeries in the limbal region
排除标准
- •Acute systemic infection
- •Acute ocular inflammation in the previous 6 months
- •Previous neoplastic/cancer disease
- •Severe dry eye confirmed by a Schirmer test
- •Lyell-Syndrome, epidermolysis bullosa
- •Total symblepharon
- •Medical history of hypersensitivity or allergy to bovine or murine derived materials
- •Pregnant and lactating patients, positive urine pregnancy test in women of childbearing potential
- •Any systemic infectious disease as diagnosed by serology tests such as syphilis, HIV-1, HIV-2, hepatitis B or C, or HTLV-1 infection at screening and at the day of the biopsy
- •Current or previous (within 30 days of enrolment) treatment with another investigational drug or participation in another clinical study
- •Previous participation of the patient in this study
- •Evidence of any other medical conditions (such as psychiatric illness, physical examination or laboratory findings) that may interfere with the planned treatment, affect patient compliance or place the patient at high risk of treatment-related complications
- •Employees of the sponsor or patients who are employees or relatives of the investigator
- •Genetic conditions such as ectodermal dysplasia or multiple endocrine neoplasia
- •Special exclusion criteria for patients ≥2 and <18 years of age (children):
- •Multiple surgeries in the limbal region
结局指标
主要结局
Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis
时间窗: month 12
This is assessed by delayed fluorescein staining and impression cytology.
Extent of neovascularization
时间窗: month 12
Measurement of the area of corneal neovascularisation as documented by photographs.
次要结局
- Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis(month 24 and 36)
- Extent of neovascularization(month 24 and 36)