The UCSD Suramin Autism Treatment-1 (SAT1) Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)
研究概览
简要总结
This study is designed to test the safety and efficacy of a single, intravenous dose of suramin in autism spectrum disorders (ASD).
详细描述
This study is designed to test a new theory of the origin and treatment of ASD. In this theory, ASD is caused by both genes and environment interacting to produce a persistent cell danger response (CDR; Naviaux RK, 2014) that interferes with and alters normal child brain development. Gut microbiome and immune systems are also affected. In this theory, the pathological persistence of the cell danger response is traceable to mitochondria, and maintained by purinergic signaling mediated by the release of extracellular nucleotides like adenosine triphosphate (ATP), adenosine diphosphate (ADP), uridine triphosphate (UTP), and uridine diphosphate (UDP). Suramin inhibits excess purinergic signaling by acting as a competitive inhibitor of nucleotide signaling at both ionotropic purinergic (P2X) receptors, and G-protein coupled, metabotropic purinergic (P2Y) receptors. Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism (Naviaux JC, et al. 2015; Naviaux JC, et al. 2014; Naviaux RK, et al. 2013). This study will test the safety and efficacy of a single dose of suramin in children with ASD. While it is not anticipated that a single dose will produce benefits for more than a few weeks, if successful, this study may lead to the development of newer and safer drugs for autism treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Autism diagnostic observation schedule (ADOS) score of ≥ 7
- •Diagnosis of autism spectrum disorder by Diagnostic and Statistical Manual, 5th edition (DSM-V)
- •Stable treatment and diet regimen for ≥ 2 months
- •Resident of San Diego region
排除标准
- •Any prescription medications
- •Hospitalization within the previous 2 months
- •Active medical problem such as seizures, heart, liver, kidney, or adrenal disease
- •Planning to start a new drug, diet, or behavioral intervention during the study
- •Weight under the 5th percentile for age
- •Unable to tolerate venipuncture, urine collection, or an indwelling intravenous catheter for 3-4 hours
- •Plasma creatinine ≥ 1.4 mg/dl
- •Liver function alanine amino transferase (ALT) or aspartate amino transferase (AST) ≥ 1.5-fold above the upper limit of normal
- •Known intolerance to suramin or other antipurinergic drugs
- •Unable to perform or cooperate with study requirements
研究组 & 干预措施
Suramin
20 mg/kg suramin in 50 ml of saline by intravenous infusion over 30 minutes
干预措施: Suramin (Drug)
Saline
50 ml of saline by intravenous infusion over 30 minutes
干预措施: Saline (Drug)
结局指标
主要结局
Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)
时间窗: 6 weeks compared to baseline
ADOS2 comparison scores are units on a scale of 0-10. A score of 7-10 was required for enrollment. A score of 7-10 is diagnostic for autism spectrum disorder (ASD). The higher the score, the more severe the core symptoms of autism spectrum disorder. Scores of 6 and below are considered off the ASD spectrum.
Expressive Language
时间窗: 6 weeks compared to baseline
Expressive One Word Picture Vocabulary Test (EOWPVT) scores are normalized for age. Typical language development produces a mean score of 100 with a standard deviation of 15. Outcomes for EOWPVT were expressed as the mean of the child-specific difference before and 6-weeks after treatment. For example, if the 6-week standard EOWPVT score was 59.6 and the baseline score was 63.8, the difference is -4.2 (= 59.6 - 63.8). A decrease in score at 6 weeks would corresponds to a decrease in language performance, while an increase, a positive difference, would reflect an increase.
次要结局
- Aberrant Behavior Checklist (ABC)(6 weeks compared to baseline)
- The Clinical Global Impression - Improvement Scale (CGI-I)(Overall ASD symptoms at 6 weeks)
- Autism Treatment Evaluation Checklist (ATEC)(6 weeks)
- Repetitive Behavior Questionnaire(6 weeks compared to baseline)
研究者
Robert K. Naviaux
Principal Investigator
University of California, San Diego