NCT04623606Unknown1 期
Exploratory, Open Label, Multiple Dose, Phase I/II Trial Evaluating Safety, Efficacy of Intravenous and Intraosseous Infusion of Allogeneic Fetal Mesenchymal Stem In Treatment of Severe Osteogenesis Imperfecta Compared With Historical and Untreated Prospective Controls
Christian Medical College, Vellore, India2 个研究点 分布在 1 个国家目标入组 15 人开始时间: 2019年5月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)
研究概览
简要总结
An exploratory, open label, multiple dose, phase I/II trial (n=15) evaluating safety and efficacy of intravenous and intraosseous infusion of allogeneic expanded fetal mesenchymal stem cells (MSC) for the treatment of severe Osteogenesis Imperfecta (OI) compared with historical and untreated prospective controls.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •(i)Inclusion Criteria Treatment group
- •Parent's/legal guardian's signed informed-consent form
- •Clinical diagnosis of OI type III or IV AND
- •Molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
- •Age between 1 to 4 years
- •BP treatment initiated before inclusion
- •Parent/legal guardian over 18 years of age
- •(ii)Inclusion Criteria Prospective Untreated Control Group and Historical Control Group:
- •Parent's/legal guardian's signed informed-consent form
- •Clinical and molecular diagnosis of OI (Glycine substitution in the collagen triple-helix encoding region of either the COL1A1 or COL1A2 gene)
- •Age between 4 to 8 years
- •Parent/legal guardian over 18 years of age
排除标准
- •Treatment group Prospective and historical control group:
- •Existence of other known disorder that might interfere with the treatment (such as severe malformations, congenital heart defect, hypoxic encephalopathy (l-lll), neurological problems, immune deficiencies, muscle diseases, syndromes) diagnosed by clinical examination
- •Any contraindication for invasive procedures such as a moderate/severe bleeding tendency or contagious infections
- •Abnormal karyotype or other confirmed genetic syndromes
- •Oncologic disease
- •Inability to comply with the trial protocol and evaluation and follow-up schedule
- •Inability to understand the information and to provide informed consent
结局指标
主要结局
Safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)
时间窗: From baseline to 16 months follow up
The primary endpoint is safety and tolerability measured as seriousness, severity and frequency of treatment related adverse events (AEs)/Serious AE (SAE)/Suspected Unexpected Serious Adverse Reaction (SUSAR)with specific focus on the following: 1. Vital signs in conjunction with the MSC infusion 2. Transfusion reactions (infusion toxicity, embolism, allergy, infections) 3. Immune reaction towards the cells, donor-specific antibodies, graft rejection, Graft versus Host Disease, autoimmunity) 4. Tumourigenicity 5. Mortality/morbidity
次要结局
- Weight (kg). [ Time Frame: From baseline to 16 months follow up](From baseline to 16 months follow up)
- Number of fractures [ Time Frame: From baseline to 16 months follow-up ](From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers fP-PTH (pg/mL)in blood samples.(From baseline to 16 months follow up)
- Growth (cm). [ Time Frame: From baseline to16 months follow up](From baseline to 16 months follow up)
- Change in clinical status of OI. [ Time Frame: From baseline to 16 months follow up](From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers P-Calcium (mg %) in blood samples.(From at baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers S-ALP (IU/L) in blood samples.(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers S-CTx (mg %) in blood samples.(From baseline to 16 months follow up)
- Time (days) to first fracture after each stem cell administration. [ Time Frame: From each dose of stem cells to the time point of the first fracture.(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers P-Phosphate (mg %) in blood samples.(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers Bone specific S-ALP (μg/L) in blood samples.(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers S-Osteocalcin (ng/mL) in blood samples.(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers U-DPD/Krea and U-NTx/Krea (mg %) in blood samples.(From baseline to 16 months follow up)
- Change in bone-marrow density (g/cm2). [ Time Frame: From baseline to the primary follow-up (From baseline to 16 months follow up)(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers P-Albumin (g/dL)(From baseline to 16 months follow up)
- Assessment of biochemical bone turnover by analysis of the markers Vitamin D (nmol/L) in blood samples.(From baseline to 16 months follow up)
研究者
Vrisha Madhuri
Professor
Christian Medical College, Vellore, India
研究点 (2)
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