Open Label of Clinical Trial of Sulforaphane in Children With Autism

• ClinicalTrials.gov Identifier: NCT02654743
• Recruitment Status: Completed
• First Posted: January 13, 2016
• Last Update Posted: March 6, 2019
• Sponsor: University of California, San Francisco
• Information provided by (Responsible Party): University of California, San Francisco

Tracking Information

• First Submitted Date: January 11, 2016
• First Posted Date: January 13, 2016
• Last Update Posted Date: March 6, 2019
• Study Start Date: January 2016
• Actual Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 12, 2016)

• Urinary metabolites [ Time Frame: 3 month ]
  Urine samples will be analyzed by lab and result will be provided
• Behavior Measures [ Time Frame: 3 months ]
  Measuring behavior using ABC (Aberrant Behavior Checklist)- self reported 58 questions from 0-3 scale (0-not a problem; 3=severe problem)

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Open Label of Clinical Trial of Sulforaphane in Children With Autism
• Official Title: An Examination of Changes in Urinary Metabolites With Use of an Antioxidant Supplement, Sulforaphane, in School-aged Children With Autism
• Brief Summary: This is an open-label, 4-month study examining the effects of Sulforaphane (SF) on behavior in children with ASD and the correlation between behavior change and urinary metabolites. The goal is to determine a potential mechanism of action of SF in this population.
• Detailed Description: Sulforaphane (SF) is an isothiocyanate found in high levels in crucifers belonging to the family Brassicaceae (including broccoli, cabbage, cauliflower, brussels sprouts, Chinese cabbage and turnips). Many previous studies have documented that consumption of these vegetables is associated with a reduced risk of cancer (lung, breast, colon, rectum, and prostate).(Juge et al. 2007) The mechanism of action of these beneficial effects is believed to be due to the ability of SF to up-regulate genes that improve cellular response to oxidative stress, inflammation, DNA-damaging electrophiles, and radiation.(Singh et al. 2014) In a recent small, randomized controlled trial in children with autism, SF was shown to have beneficial effects on aberrant and social behavior.(Singh et al. 2014) The mechanism of action of this beneficial effect has not been established in children with ASD. Our primary goal is to examine changes in urinary metabolites in children with autism who receive SF to determine if changes in behavior are associated with changes in urinary metabolites.
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Autism

Intervention

Dietary Supplement: Sulforaphane

Children with ASD will receive Sulforaphane in this study

Study Arms

Experimental: SF

Sulforaphane (SF) will be administered in an approximate dosage of 1 µmol SF/lb (2.2 kg µmol/kg) body weight. This dosage roughly approximates the dosage that was used in the Singh et al, (2014; PNAS) clinical trial of sulforaphane in male adolescents and adults with autism. The sulforaphane will be supplied as glucoraphanin (GR)-enriched broccoli seed extract tablets (manufacturing details follow). Each active tablet will contain 125 mg broccoli seed extract (containing 37 µmol GR, which is equivalent to about 15 µmol SF), 50 mg dried broccoli sprouts (a source of myrosinase, the enzyme that converts GR to SF), 15 mg ascorbic acid, 55.90 mg microcrystalline cellulose, and other minor GRAS excipients used for tablet forming.

The total dose per day will depend of study participants' body weight.

Intervention: Dietary Supplement: Sulforaphane

• Publications *: Bent S, Lawton B, Warren T, Widjaja F, Dang K, Fahey JW, Cornblatt B, Kinchen JM, Delucchi K, Hendren RL. Identification of urinary metabolites that correlate with clinical improvements in children with autism treated with sulforaphane from broccoli. Mol Autism. 2018 May 30;9:35. doi: 10.1186/s13229-018-0218-4. eCollection 2018.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 4, 2019): 21
• Original Estimated Enrollment (submitted: January 12, 2016): 40
• Actual Study Completion Date: September 2016
• Actual Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Male or female, enrolled at OHS, age 6-22 and with a diagnosis of ASD.
• ASD diagnosis will be established by standard criteria (DSM-IV criteria and expert clinician review of medical records and child observation).
• Written informed consent obtained from the subject's legal representative and ability for subject to comply with the requirements of the study.

Exclusion Criteria:

• Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years to 22 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT02654743
• Other Study ID Numbers: SF15-17002
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: University of California, San Francisco
• Original Responsible Party: Same as current
• Current Study Sponsor: University of California, San Francisco
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Robert Hendren, DO; University of California, San Francisco

• PRS Account: University of California, San Francisco
• Verification Date: March 2019