Sulforaphane in a New Jersey (NJ) Population of Individuals With Autism

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Sulforaphane in Autism: A Treatment Trial to Confirm Phenotypic Improvement With Sulforaphane Treatment in a New Jersey (NJ) Population of Individuals With Autism

Conditions
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Autism, Autistic Disorder, Autism Spectrum Disorder, Autistic Behavior

Intervention / Treatment
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* Drug: Sulforaphane
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Autistic disorder diagnosis. Age between 13-30 years. Male gender. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 13 Years
Maximum Age: 30 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>About 15 subjects will be randomized into this arm, receiving pills with an inactive placebo. | Drug: Placebo<br> <br> |
| Experimental: Sulforaphane<br>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 | Drug: Sulforaphane<br>* Sulforaphane (1-isothiocyanato-4R- (methylsulfinyl)butane) is an isothiocyanate derived from the action of the plant enzyme myrosinase on glucosinolates including glucoraphanin and comes from consumption of many cruciferous vegetables.<br>* Other names: Avmacol;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Aberrant Behavior Checklist (ABC) scores. |  | Baseline, week 4, week 10, week 18 and week 22. | 
| Change in Social Responsiveness Scale (SRS) scores. |  | Baseline, week 4, week 10, week 18 and week 22. | 
| Clinical Global Impression Severity Scale (CGI-S). |  | Baseline | 
| Clinical Global Impression Improvement Scale (CGI-I) to measure change from baseline CGI-S scores. |  | Week 4, week 10, week 18 and week 22. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. |