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

• ClinicalTrials.gov Identifier: NCT02677051
• Recruitment Status: Active, not recruiting
• First Posted: February 9, 2016
• Last Update Posted: November 16, 2022
• Sponsor: Rutgers, The State University of New Jersey
• Collaborator: Rowan University
• Information provided by (Responsible Party): Steven Buyske, Ph.D., Rutgers, The State University of New Jersey

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Autism; Autistic Disorder; Autism Spectrum Disorder; Autistic Behavior	Drug: Sulforaphane; Drug: Placebo	Phase 2

Detailed Description:

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 48 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Sulforaphane in Autism: A Treatment Trial to Confirm Phenotypic Improvement With Sulforaphane Treatment in a New Jersey (NJ) Population of Individuals With Autism
• Study Start Date: February 2016
• Estimated Primary Completion Date: December 2022
• Estimated Study Completion Date: July 2023

Arms and Interventions

Arm	Intervention/treatment
Placebo Comparator: Placebo; About 15 subjects will be randomized into this arm, receiving pills with an inactive placebo.	Drug: Placebo
Experimental: 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	Drug: Sulforaphane; 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.; Other Name: Avmacol

Outcome Measures

Primary Outcome Measures :

1. Change in Aberrant Behavior Checklist (ABC) scores. [ Time Frame: Baseline, week 4, week 10, week 18 and week 22. ]
2. Change in Social Responsiveness Scale (SRS) scores. [ Time Frame: Baseline, week 4, week 10, week 18 and week 22. ]
3. Clinical Global Impression Severity Scale (CGI-S). [ Time Frame: Baseline ]
4. Clinical Global Impression Improvement Scale (CGI-I) to measure change from baseline CGI-S scores. [ Time Frame: Week 4, week 10, week 18 and week 22. ]

Secondary Outcome Measures :

1. Liver Function Tests as a screen for entry into the study and a monitor of potential change/adverse events due to treatment. [ Time Frame: Baseline, week 4, week 18 and week 22. ]
2. Renal Function Tests as a screen for entry into the study and a monitor of potential change/adverse events due to treatment. [ Time Frame: Baseline, week 4, week 18 and week 22. ]
3. Thyroid Stimulating Hormone (TSH) as a screen for entry into the study and a monitor of potential change/adverse events due to treatment. [ Time Frame: Baseline, week 4, week 18 and week 22. ]

Eligibility Criteria

• Ages Eligible for Study: 13 Years to 30 Years (Child, Adult)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

United States, New Jersey

Rutgers University - Staged Research Building

Piscataway, New Jersey, United States, 08854

Sponsors and Collaborators

Rutgers, The State University of New Jersey

Rowan University

Investigators

• Principal Investigator: Steven Buyske; Rutgers, The State University of NJ

  Study Documents (Full-Text)

Documents provided by Steven Buyske, Ph.D., Rutgers, The State University of New Jersey:

Informed Consent Form  [PDF] July 28, 2022

More Information

Publications:

Singh K, Connors SL, Macklin EA, Smith KD, Fahey JW, Talalay P, Zimmerman AW. Sulforaphane treatment of autism spectrum disorder (ASD). Proc Natl Acad Sci U S A. 2014 Oct 28;111(43):15550-5. doi: 10.1073/pnas.1416940111. Epub 2014 Oct 13.

• Responsible Party: Steven Buyske, Ph.D., Associate Professor, Rutgers, The State University of New Jersey
• ClinicalTrials.gov Identifier: NCT02677051
• Other Study ID Numbers: Pro20120001884; CAUT15APL013 ( Other Grant/Funding Number: NJ Governor's Council, Autism )
• First Posted: February 9, 2016
• Last Update Posted: November 16, 2022
• Last Verified: November 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Disease; Autistic Disorder; Autism Spectrum Disorder; Pathologic Processes; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Sulforaphane; Anticarcinogenic Agents; Protective Agents; Physiological Effects of Drugs; Antineoplastic Agents