Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Safety of C2C_ASC (Adverse Events)
研究概览
简要总结
The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms.
详细描述
The ASC - Autism Pilot Study will be performed at the Child and Adolescent Psychiatry, Aalborg University Hospital, Denmark. 10 children aged 6-14 years with ASD and gastrointestinal symptoms will be enrolled through the Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark.
The participants will randomly be divied into two treatment groups:
- participant 1-5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
- participant 6-10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight
C2C_ASC treatment:
The C2C_ASCs product will be diluted in 50 ml isotonic saline and infused in an arm vein within 15 min. In both groups there will for each participant be a week observation for SAE's related to the cell treatment before the next participant will be treated, if no SAE's have been detected.
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
- 6 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of autism spectrum disorder.
- •Ongoing gastrointestinal symptoms or previous gastrointestinal symptoms which disappeared or were reduced after dietary changes.
- •Age 6 to 14 years.
排除标准
- •Known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder.
- •Known CNS-infection (now or previously) and/or HIV positivity.
- •Primary immunodeficiency disorder or autoimmune cytopenia.
- •Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other antiinflammatory medication (except nonsteroidal anti-inflammatory drugs).
- •Epilepsy or known seizure disorder (now or previously).
研究组 & 干预措施
1 x 1000000 C2C_ASCs/kg body weight
Trial participant 1 - 5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
干预措施: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) (Drug)
2 x 1000000 C2C_ASCs/kg body weight
Trial participant 6 - 10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight
干预措施: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) (Drug)
结局指标
主要结局
Safety of C2C_ASC (Adverse Events)
时间窗: 12 weeks
Safety of C2C_ASC within the 12 weeks follow-up period: Adverse event (AE), serious adverse event (SAE), suspected unexpected serious adverse reaction (SUSAR) and development safety update report (DSUR).
次要结局
- Changes in autism spectrum disorder(12 weeks)
- Changes in gastrointestinal symptoms(12 weeks)