A Phase IV, Single-site, Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.
研究概览
简要总结
If autism is the consequence of a chronic inflammatory process preventing the infant brain from forming the proper neural connections, then treatment that reduces inflammation might have a positive impact on autism. IGIV treatment has been known to suppress inflammation and has been used in the treatment of inflammatory conditions and autoimmune diseases.
详细描述
We have come to believe that an immature immune system with abnormal signaling and altered apoptotic pathways may result in a process of neuroinflammation with a clinical presentation of Autism Spectrum Disorder. If Autism Spectrum Disorders are the consequence of a chronic inflammatory process that prevents the infant brain from forming the proper neural connections, then treatment that reduces inflammation might have a positive impact on autism. We believe that there is a correlation between immunological abnormalities and the development of the autism.
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
- 4 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The subject is between the ages of or equal to 4 and 12 years of age, of either gender, and belonging to any ethnic group.
- •The subject has a diagnosis, for a minimum of 6 months prior to screening, of either autistic disorder, Asperger disorder, or pervasive developmental disorder (PDD).
- •Normal physical test results.
- •Immunological lab results showing reduced levels of activated CD40L or abnormal lymphocyte stimulation, or T or B cell dysfunction (hypgammaglobulinemia, primary immunodeficiency (PIDD), or common variable immune deficiency (CVID).
排除标准
- •A diagnosis of isolated IGA deficiency
- •Allergic reactions to blood products
研究组 & 干预措施
Gammaplex 5% IGIV
Gammaplex 5% IGIV administered intravenously
干预措施: Gammaplex 5% (Drug)
结局指标
主要结局
The primary objective is to assess the efficacy of Gammaplex as an immunomodulatory therapy in autistic children with evidence of immune dysfunction.
时间窗: 1 year
The primary endpoint is disease improvement, as evidenced by standardized test measures across 3 intervals: screening for baseline measurements, visit 5, and end of study visit measurements. Primary efficacy variables will be measured using the following standardized tests 1. Caregiver Scales (SRS, CCC-2, ABC) 2. CGI-S and CGI - I 3. PPVT 4. ADOS
次要结局
- The modulatory effect of IVIG on neuroinflammation will be assessed by laboratory measurements which we believe are indicative of immune dysregulation.(1 year)
研究者
Isaac Melamed
Principal Investigator
IMMUNOe Research Centers