Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 2
- 试验地点
- 3
- 主要终点
- Adverse Events as a Measure of Safety
研究概览
简要总结
The purpose of this study is to determine the safety and effectiveness of five infusions of characterized HLA-identical MSC in non immunosuppressed children with Osteogenesis Imperfecta (OI).
详细描述
The principal aim of this trial is to assess the safety of non-mutated HLA-identical Mesenchymal stem cell (MSC) transplantation for OI pediatric patients irrespective of treatment with biphosphonates. Since MSC are inherently non-immunogenic and do not elicit proliferation of allogeneic lymphocytes (in co-culture experiments), a cell therapy based on HLA-identical or histocompatible (at least 5 shared out of 6 HLA antigens) allogenic MSC may be accomplished without subjecting the patients to immunosuppressor treatment. Adverse secondary effects due to immunosuppressor treatment will be avoided using this strategy thus patients may benefit from two cellular infusions. The patients will be followed for 2 years post their fifth and last MSC infusion.
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
- 6 Months 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient age: older than 6 months and younger than 12 years old.
- •Patients with molecular confirmation of mutation in either COL1A1 or COL1A2 genes associated with OI (type III).
- •Patients with HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
- •All patients that fulfil the inclusion criteria regardless of whether or not they are undergoing biphosphonate treatment.
- •Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial.
排除标准
- •Patient age: older than 12 years old
- •Patients lacking confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
- •Other pathological subtypes of OI.
- •Patients lacking of HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
- •Immunodeficiencies and any other malignancies.
- •Participation in other clinical trial.
- •Any medical or psychiatric condition that in the researcher´s opinion could affect the patient´s ability to complete the trial or hamper the participation in the trial.
- •Patients whose parents or the legal guardians do not sign the consent forms
研究组 & 干预措施
Mesenchymal stem cells
Five Mesenchymal Stem Cell infusions
干预措施: Mesenchymal Stem Cells (Biological)
结局指标
主要结局
Adverse Events as a Measure of Safety
时间窗: up to 2 years post last MSCs infusion
次要结局
- change from baseline in degree of functionality(up to 2 years post last MSCs infusion)
- bone mineral density(up to 2 years post last MSCs infusion)
- fracture rate(up to 2 years post last MSCs infusion)
- growth velocity(up to 2 years post last MSCs infusion)
- change from baseline in well-being(up to 2 years post last MSCs infusion)
研究者
Clara I. Rodríguez
PI Stem Cell Laboratory
Hospital de Cruces