Sulforaphane in Autism: A Treatment Trial to Confirm Phenotypic Improvement With Sulforaphane Treatment in a New Jersey (NJ) Population of Individuals With Autism
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Change in Aberrant Behavior Checklist (ABC) scores.
研究概览
简要总结
This study is a double blind treatment trial that will test if sulforaphane improves core symptoms in autism. The investigators expect to see clinical improvement in some of these areas. Sulforaphanes come from eating certain vegetables such as broccoli. The investigators will be using a preparation that gives specific and reproducible amounts. The investigators will also test specific chemicals and genes needed for sulforaphane usage to try to understand differences in response.
详细描述
This study is a double blind randomized treatment trial that will test if sulforaphane improves core symptoms in autism. It is designed to try to replicate a previous trial (ClinicalTrials.gov Identifier NCT01474993) which reported that the isothiocyanate, sulforaphane treatment led to improvement by multiple metrics. Significant improvement was seen in behavior as measured by the Aberrant Behavioral Checklist (ABC) and by the Social Responsiveness Scale (SRS). In addition a significantly greater number of participants receiving sulforaphane had improvement in social interaction, abnormal behavior, and verbal communication as per the Clinical Global Impression (CGI). In addition The investigators will attempt to account for some variability in response to sulforaphane treatment by testing alleles of genes that are relevant in sulforaphane metabolism. The investigators will also measure glutathione levels, which are also important in sulforaphane metabolism and are in part regulated by sulforaphane..
Sulforaphane is the most potent naturally occurring inducer of mammalian cytoprotective enzymes known. Therapeutic potential is based at least in part on their ability to up-regulate genes responsible for alleviation of oxidative stress and to regulate both the immune system and the inflammatory response
40 Males with autistic disorder will be randomly selected to receive either sulforaphane or placebo. Seven visits are required by the subjects including enrollment ,screening, baseline, weeks 4, 10 and 18 and a follow up visit at seek 22.
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
- 13 Years 至 30 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Autistic disorder diagnosis.
- •Age between 13-30 years.
- •Male gender.
排除标准
- •Absence of a parent or legal guardian and consent,
- •Those that can not or will not complete all visits and adherence to study regimen.
- •Seizure within 2 years of screening,
- •Impaired renal function (serum creatinine> 1.2 mg/dl).
- •Impaired hepatic function (> 2x upper limit of normal).
- •Impaired thyroid function (TSH outside normal limits).
- •Current infection or treatment with antibiotics.
- •Chronic medical disorder (e.g., cardiovascular disease, stroke or diabetes) or major surgery within 3 months prior to enrollment.
- •Less than 13 years or more than 30 years of age.
- •Female gender.
- •A diagnosis of autism spectrum disorder other than autistic disorder, for example, Asperger, Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS) etc.
研究组 & 干预措施
Placebo
About 15 subjects will be randomized into this arm, receiving pills with an inactive placebo.
干预措施: Placebo (Drug)
Sulforaphane
About 30 subjects will be randomized into this arm, receiving pills with glucoraphanin rich broccoli seed powder containing active myrosinase resulting in sulforaphane once ingested Doses will be weight dependent with each pill resulting in ~ 50 µmol sulforaphane.
Body weight Dose of sulforaphane 34 kg ~ 50 µmol 68 kg ~ 100 µmol 102 kg ~ 150 µmol
干预措施: Sulforaphane (Drug)
结局指标
主要结局
Change in Aberrant Behavior Checklist (ABC) scores.
时间窗: Baseline, week 4, week 10, week 18 and week 22.
Change in Social Responsiveness Scale (SRS) scores.
时间窗: Baseline, week 4, week 10, week 18 and week 22.
Clinical Global Impression Severity Scale (CGI-S).
时间窗: Baseline
Clinical Global Impression Improvement Scale (CGI-I) to measure change from baseline CGI-S scores.
时间窗: Week 4, week 10, week 18 and week 22.
次要结局
- Liver Function Tests as a screen for entry into the study and a monitor of potential change/adverse events due to treatment.(Baseline, week 4, week 18 and week 22.)
- Renal Function Tests as a screen for entry into the study and a monitor of potential change/adverse events due to treatment.(Baseline, week 4, week 18 and week 22.)
- Thyroid Stimulating Hormone (TSH) as a screen for entry into the study and a monitor of potential change/adverse events due to treatment.(Baseline, week 4, week 18 and week 22.)
研究者
Steven Buyske, Ph.D.
Associate Professor
Rutgers, The State University of New Jersey