Phase IIa Multicenter Clinical Trial to Determine the Feasibility and Safety of the Use of Adipose-derived Mesenchymal Stem Cells (ASC) in the Treatment of Patients With Cicatricial Conjunctivitis Associated With Lyell's Syndrome, Stevens-Johnson Syndrome and Pemphigoid of the Mucous Membranes With Ocular Involvement.
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 20
- 试验地点
- 8
- 主要终点
- Percentage of complications
研究概览
简要总结
A phase IIa, open label, non controlled clinical trial to assess the feasibility and safety of allogeneic adipose-derived mesenchymal stem cells (ASC) in the treatment of cicatricial conjunctivitis associated with Lyell's syndrome, Stevens-Johnson's syndrome and mucous membrane pemphigoid with ocular involvement
详细描述
A multicentre, nationwide clinical trial will be performed. 20 patients fulfilling eligibility criteria will be included in two different dose level cohorts: the first 10 patients included will receive a single cell application and the remainder 10 will be administered 2 cell applications separated by 15 days.
The investigational drug consists of locally administered, expanded, allogeneic adipose-derived adult mesenchymal stem cells (ASCs), at a dose of 5 million cells per ml (0,5 ml to be infused per quadrant)
Study cronogram: recruitment is estimated to take 8 months, and follow-up period will be 12 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- •Men and women over 18 years of age.
- •Diagnosis of ocular pemphigoid in Foster stages I-IIcIIIb (2) or diagnosis of recurrent chronic or episodic inflammation accompanied by cicatricial conjunctivitis of the mucous membranes with ocular involvement after the acute phase of Stevens-Johnson Syndrome or Lyell Syndrome with insufficient disease control or inaceptable toxicity or impossibility to administer usual care treatments (according to physician or patient criteria)
- •In the case of women of childbearing age, who are willing to use an effective contraceptive method during the period of participation in the study
- •Consent to participate and signature of the informed consent
排除标准
- •Signs of active infection on the ocular surface.
- •History of neoplasms in the last 5 years. except for epithelial basal or squamous cell carcinoma
- •Allergy to local anesthetics
- •Patients who have participated in another clinical trial with medication during the 90 days prior to signing the IC
- •Medical or psychiatric illness of any kind that, in the opinion of the investigator, may be a reason for exclusion from the study.
- •Congenital or acquired immunodeficiencies.
- •Major surgery or serious trauma of the subject in the semester prior to signing the IC.
- •Pregnant or lactating women.
- •Impossibility or refusal to carry out the follow-up required in the study by the patient
研究组 & 干预措施
Single dose Allogenic Adiposse derived mesenchimal stem cells
Allogenic Adiposse derived mesenchimal stem cells (MSC) Dose: 12,5 million cells
干预措施: Single Dose (Drug)
Double dose Allogenic Adiposse derived mesenchimal stem cells
Allogenic Adiposse derived mesenchimal stem cells (MSC) Dose: 12,5 million cells at day 0 and 12,5 million cells at day 14
干预措施: Double Dose (Drug)
结局指标
主要结局
Percentage of complications
时间窗: Through study completion, an average of 3 years
Percentage of complications related to anesthesia, administration of investigational medicinal product and postoperative period.
次要结局
- changes in quality of life(At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks)
- Signs improvement(At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks)
- Symptom improvement(At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks)
- Visual acuity improvement(At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks)
- evolution of the conjunctival flora(At 7 days (and 21 days in the 2-dose group), 4 weeks, 12 weeks, 24 weeks and 52 weeks)