Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder

NCT02909959 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 16-20595KL2TR001109-05
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this randomized controlled trial is to determine if a nutritional supplement containing broccoli sprout and seed extracts, a rich source of sulforaphane, is effective in reducing core symptoms of autism spectrum disorder (ASD). The study will also explore the safety and tolerability of a sulforaphane supplement in young men with ASD, as well as its effects on challenging neuropsychiatric symptoms that are commonly associated with ASD, such as hyperactivity, irritability, and repetitive movements.

Conditions

Autism Spectrum Disorder; Autistic Disorder; Neurodevelopmental Disorder; Childhood Developmental Disorders, Pervasive

Keywords

sulforaphane; glucoraphanin; physiologic effects of drugs

Outcome Measures

Primary Outcomes (5)

• Social Responsiveness Scale-2 (SRS-2) Total Score at Baseline

  The Social Responsiveness Scale-2 (SRS-2) is a 65-item caregiver report that includes 5 subscales covering core symptom domains of ASD (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors). The SRS-2 provides a T-score that is scaled such that the mean is 50 and the SD is 10. Higher scores indicate a higher presence and severity of autistic social impairment. A T-score of 76 or higher is considered "severe". T-scores between 66 and 75 are considered as "moderate". T-scores between 60 and 65 are considered "mild". A T-score below 60 is considered typical. The theoretical range of a T-score is 10 to 90. The actual range of the SRS-2 in this study is 45 to 90.

  Baseline

• Social Responsiveness Scale-2 (SRS-2) Total Score at Week 4

  The Social Responsiveness Scale-2 (SRS-2) is a 65-item caregiver report that includes 5 subscales covering core symptom domains of ASD (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors). The SRS-2 provides a T-score that is scaled such that the mean is 50 and the SD is 10. Higher scores indicate a higher presence and severity of autistic social impairment. A T-score of 76 or higher is considered "severe". T-scores between 66 and 75 are considered as "moderate". T-scores between 60 and 65 are considered "mild". A T-score below 60 is considered typical. The theoretical range of a T-score is 10 to 90. The actual range of the SRS-2 in this study is 45 to 90.

  Week 4

• Social Responsiveness Scale-2 (SRS-2) Total Score at Week 8

  The Social Responsiveness Scale-2 (SRS-2) is a 65-item caregiver report that includes 5 subscales covering core symptom domains of ASD (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors). The SRS-2 provides a T-score that is scaled such that the mean is 50 and the SD is 10. Higher scores indicate a higher presence and severity of autistic social impairment. A T-score of 76 or higher is considered "severe". T-scores between 66 and 75 are considered as "moderate". T-scores between 60 and 65 are considered "mild". A T-score below 60 is considered typical. The theoretical range of a T-score is 10 to 90. The actual range of the SRS-2 in this study is 45 to 90.

  Week 8

• Social Responsiveness Scale-2 (SRS-2) Total Score at Week 12

  The Social Responsiveness Scale-2 (SRS-2) is a 65-item caregiver report that includes 5 subscales covering core symptom domains of ASD (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors). The SRS-2 provides a T-score that is scaled such that the mean is 50 and the SD is 10. Higher scores indicate a higher presence and severity of autistic social impairment. A T-score of 76 or higher is considered "severe". T-scores between 66 and 75 are considered as "moderate". T-scores between 60 and 65 are considered "mild". A T-score below 60 is considered typical. The theoretical range of a T-score is 10 to 90. The actual range of the SRS-2 in this study is 45 to 90.

  Week 12

• Social Responsiveness Scale-2 (SRS-2) Total Score at Week 16

  The Social Responsiveness Scale-2 (SRS-2) is a 65-item caregiver report that includes 5 subscales covering core symptom domains of ASD (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors). The SRS-2 provides a T-score that is scaled such that the mean is 50 and the SD is 10. Higher scores indicate a higher presence and severity of autistic social impairment. A T-score of 76 or higher is considered "severe". T-scores between 66 and 75 are considered as "moderate". T-scores between 60 and 65 are considered "mild". A T-score below 60 is considered typical. The theoretical range of a T-score is 10 to 90. The actual range of the SRS-2 in this study is 45 to 90.

  Week 16

Secondary Outcomes (42)

• Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Awareness)

  Baseline, Week 4, Week 8, Week 12, Week 16

• Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Cognition)

  Baseline, Week 4, Week 8, Week 12, Week 16

• Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Communication)

  Baseline, Week 4, Week 8, Week 12, Week 16

• Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Motivation)

  Baseline, Week 4, Week 8, Week 12, Week 16

• Social Responsiveness Scale-2 (SRS-2) Subscale Score (Restricted Interests/Repetitive Behaviors)

  Baseline, Week 4, Week 8, Week 12, Week 16

Study Arms (2)

Sulforaphane

ACTIVE COMPARATOR

Participants will take a sulforaphane supplement 3-8 tablets daily, with dose depending upon body weight. Each tablet contains 125 mg broccoli seed powder and 50 mg broccoli sprout extract, providing approximately 15 µmol sulforaphane. The weight-based dosing schedule is as follows: 3 tablets (approx. 46.5 µmol SF) if \<100 lb; 5 tablets (approx. 77.5 µmol SF) if 100-125 lb; 6 tablets (approx. 93 µmol SF) if 126-175 lb; 7 tablets (approx. 108.5 µmol SF) if 176-199 lb; 8 tablets (approx. 124 µmol SF) if ≥ 200 lb

Drug: Sulforaphane

Placebo

PLACEBO COMPARATOR

Participants in this arm will take placebo tablets that are identical in shape, size, and color to the sulforaphane tablets. The number of tablets taken per day corresponds to the weight-based schedule described for the sulforaphane arm.

Drug: Placebo

Interventions

Sulforaphane DRUG

The investigational medicinal product is an uncoated tablet containing both glucoraphanin and myrosinase, the enzyme that converts glucoraphanin to sulforaphane in vivo. Participants in this arm will take 3-8 tablets by mouth once daily (dose depending upon weight) for 12 weeks.

Also known as: Avmacol, glucoraphanin

Sulforaphane

Placebo DRUG

Placebo tablets are uncoated and matched in appearance to the investigational medicinal product, containing inert components. Participants in this arm will take 3-8 tablets by mouth once daily (dose depending upon weight) for 12 weeks.

Also known as: Control

Placebo

Eligibility Criteria

• Age: 13 Years - 30 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males between ages 13-30 (inclusive) at the time of the consent
• Primary diagnosis of Autism Spectrum Disorder (ASD), confirmed by Diagnostic and Statistical Manual-5 (DSM-5) criteria and meeting the autism cut-off score of 9 or greater on the Autism Diagnostic Observation Schedule-2 (ADOS-2)
• Participant is capable of giving written informed consent or has a legally authorized representative (LAR) with sufficient capacity to provide written informed consent on the participant's behalf.
• Participant has a reliable informant (parent or caregiver) who has sufficient past and current knowledge of the subject and will oversee the administration of study medication and accompany the subject to each study visit.
• Participant and caregiver have reliable means of transportation to attend study visits.

You may not qualify if:

• Chronic medical illness that is not stable or would pose a risk to the participant if he participates in the trial
• History of clinical seizures within the 12 months preceding study enrollment
• Known genetic disorder that is presumed to be the cause of autism spectrum disorder (eg., Fragile x syndrome, tuberous sclerosis)
• Changes to psychopharmacological medications (e.g., stimulants, antidepressants, anxiolytics, antipsychotics) in the 4 weeks preceding study enrollment
• Significant changes to non-pharmacological treatments for ASD in the 4 weeks preceding study enrollment
• Chronic treatment with anti-inflammatory agents (e.g., ibuprofen, NSAIDs, corticosteroids)
• Clinically significant laboratory abnormalities at Screening visit (e.g., AST/ALT\> two times the upper normal limits; serum creatinine \> 1.2 mg/dl, TSH outside normal limits)
• Clinically significant findings on physical examination that investigator determines could increase risk of harm from participating in the study
• Participated in another clinical interventional trial or received an investigational product in the 30 days preceding study enrollment
• Previous therapeutic trial of sulforaphane or participation in a clinical trial in which sulforaphane was the investigational agent

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• North Carolina Translational and Clinical Sciences Institute: collaborator

Study Sites (1)

Carolina Institute for Developmental Disabilities, University of North Carolina School of Medicine

Carrboro, North Carolina, 27510, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; Neurodevelopmental Disorders

Interventions

sulforaphane; glucoraphanin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Mental Disorders

Results Point of Contact

• Title: Laura Politte, MD
• Organization: University of North Carolina at Chapel Hill

lpolitte@wakemed.org

919-350-1726

Study Officials

• Laura Politte, M.D.

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2016
• First Posted: September 21, 2016
• Study Start: March 1, 2017
• Primary Completion: May 30, 2019
• Study Completion: May 30, 2019
• Last Updated: June 4, 2020
• Results First Posted: May 15, 2020

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will share

Locations

US (1)