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Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02909959
Recruitment Status : Completed
First Posted : September 21, 2016
Results First Posted : May 15, 2020
Last Update Posted : May 15, 2020
Sponsor:
Collaborator:
North Carolina Translational and Clinical Sciences Institute
Information provided by (Responsible Party):
University of North Carolina, Chapel Hill

Tracking Information
First Submitted Date  ICMJE September 7, 2016
First Posted Date  ICMJE September 21, 2016
Results First Submitted Date  ICMJE April 28, 2020
Results First Posted Date  ICMJE May 15, 2020
Last Update Posted Date May 15, 2020
Actual Study Start Date  ICMJE March 1, 2017
Actual Primary Completion Date May 30, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 28, 2020)
  • Social Responsiveness Scale-2 (SRS-2) Total Score at Baseline [ Time Frame: 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.
  • Social Responsiveness Scale-2 (SRS-2) Total Score at Week 4 [ Time Frame: 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.
  • Social Responsiveness Scale-2 (SRS-2) Total Score at Week 8 [ Time Frame: 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.
  • Social Responsiveness Scale-2 (SRS-2) Total Score at Week 12 [ Time Frame: 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.
  • Social Responsiveness Scale-2 (SRS-2) Total Score at Week 16 [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE
 (submitted: September 16, 2016)
Change in Social Responsiveness Scale-2 (SRS-2) total scores from baseline to weeks 4, 8, 12 and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
The 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).
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 28, 2020)
  • Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Awareness) [ Time Frame: Baseline, Week 4, Week 8, Week 12, 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-Social Awareness subscale measures a participant's ability to recognize social cues. This subscale comprises 8 items with scores ranging from 1 to 4 for a total range of scores from 8 to 32. A higher score represents a greater ability to recognize social cues. Subscales are not diagnostic in nature, but they provide some insight into individual behaviors. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Cognition) [ Time Frame: Baseline, Week 4, Week 8, Week 12, 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-Social Cognition subscale measures a participant's ability to interpret social behavior. This subscale comprises 12 items with scores ranging from 1 to 4 for a total range of scores from 12 to 48. A higher score represents a greater ability to interpret social behaviors. Subscales are not diagnostic in nature, but they provide some insight into individual behaviors. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Communication) [ Time Frame: Baseline, Week 4, Week 8, Week 12, 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-Social Communication subscale measures a participant's ability to assess reciprocal communication in social situations. This subscale comprises 22 items with scores ranging from 1 to 4 for a total range of scores from 22 to 88. A higher score represents a greater ability to assess reciprocal communication. Subscales are not diagnostic in nature, but they provide some insight into individual behaviors. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Motivation) [ Time Frame: Baseline, Week 4, Week 8, Week 12, 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-Social Motivation subscale measures the degree to which a participant is motivated to participate in social interactions. This subscale comprises 11 items with scores ranging from 1 to 4 for a total range of scores from 11 to 44. A higher score represents a greater motivation to participate in social interaction. Subscales are not diagnostic in nature, but they provide some insight into individual behaviors. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Social Responsiveness Scale-2 (SRS-2) Subscale Score (Restricted Interests/Repetitive Behaviors) [ Time Frame: Baseline, Week 4, Week 8, Week 12, 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-Restricted Interests/Repetitive Behaviors subscale assesses the participant's level of stereotypy and circumscribed interests. This subscale comprises 12 items with scores ranging from 1 to 4 for a total range of scores from 12 to 48. A higher score represents a greater level of stereotypy and circumscribed interests. Subscales are not diagnostic in nature, but they provide some insight into individual behaviors. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Aberrant Behavior Checklist (ABC) Subscale Score (Social Withdrawal) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms. The ABC-Social Withdrawal subscale assesses the participant's level social withdrawal. This subscale comprises 16 items with scores ranging from 0 to 3 for a total range of scores from 0 to 48. A higher score represents a greater level of social withdrawal. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Aberrant Behavior Checklist (ABC) Subscale Scores (Hyperactivity) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms. The ABC-Hyperactivity subscale assesses the participant's level hyperactivity and non-compliance. This subscale comprises 16 items with scores ranging from 0 to 3 for a total range of scores from 0 to 48. A higher score represents a greater level of hyperactivity/non-compliance. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Aberrant Behavior Checklist (ABC) Subscale Score (Inappropriate Speech) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms. The ABC-Inappropriate Speech subscale assesses the participant's use of inappropriate speech. This subscale comprises 4 items with scores ranging from 0 to 3 for a total range of scores from 0 to 12. A higher score represents a greater use of inappropriate speech. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Aberrant Behavior Checklist (ABC) Subscale Score (Stereotypy) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms. The ABC-Stereotypy subscale assesses the participant's level of stereotypic behavior. This subscale comprises 7 items with scores ranging from 0 to 3 for a total range of scores from 0 to 21. A higher score represents a greater level of stereotypic behavior. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Aberrant Behavior Checklist (ABC) Subscale Score (Irritability) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms. The ABC-Irritability subscale assesses the participant's level of irritability. This subscale comprises 15 items with scores ranging from 0 to 3 for a total range of scores from 0 to 45. A higher score represents a greater level of irritability. This subscale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Clinical Global Impression-Severity (CGI-S) Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The CGI-Severity (CGI-S) scale is a 7-point, clinician-rated scale providing an overall assessment of patient functioning relative to other patients with a similar diagnosis (1=not at all ill to 7=severely ill). A higher score indicates a higher level of severity of psychopathology. This scale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Number of Participants With Clinical Global Impression-Improvement (CGI-I) Score of Much Improved or Very Much Improved [ Time Frame: Week 4, Week 8, Week 12, Week 16 ]
    The clinician-rated CGI-Improvement (CGI-I) scale rates overall improvement or worsening of illness (ie, ASD) relative to baseline. The CGI-Improvement (CGI-I) scale rates overall improvement or worsening of illness (ie, ASD) relative to baseline. Possible scores on this scale range from 1-6 with the following assigned values: 1 = "Very Much Improved"; 2 = "Much Improved; 3 = "Minimally improved"; 4 = "No change"; 5 = "Minimally worse"; 6= "Much worse." A higher score indicates worsening of illness (ie, ASD), whereas a lower score indicates improvement of illness. This scale was measured at Week 4, Week 8, Week 12, and Week 16. In this measure, the number of participants in each treatment arm determined by the study physician to be "Much Improved" or "Very Much Improved" (indicative of meaningful clinical improvement) were counted.
  • Repetitive Behavior Scale-Revised (RBSR) Total Score [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    The RBS-R is a 43-item, informant-based questionnaire designed to quantify a range of restricted, repetitive behaviors (RRB) observed in ASD. Scores for each item range from 0 = "Behavior does not occur" to 3 = "Behavior occurs and is a severe problem". Thus the total range is 0 to 129. A higher score indicates a higher breadth of repetitive behaviors. This scale was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Mean Red Blood Cell Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the red blood cell value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean White Blood Cell Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the white blood cell value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Hemoglobin Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the hemoglobin value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Hematocrit Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the hematocrit value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Corpuscular Volume Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the mean corpuscular volume value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Corpuscular Hemoglobin Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the mean corpuscular hemoglobin value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Corpuscular Hemoglobin Concentration Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the mean corpuscular hemoglobin concentration value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Red Blood Cell Distribution Width Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the red blood cell distribution width value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Platelets Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the platelets value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Absolute Neutrophils Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the absolute neutrophils value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Absolute Lymphocytes Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the absolute lymphocytes value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Absolute Monocytes Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the absolute monocytes value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Absolute Eosinophils Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the absolute eosinophils value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Absolute Basophils Value [ Time Frame: Baseline, Week 12 ]
    This measure assesses the absolute basophils value as part of the complete blood count measurement. This blood count measure was assessed at Baseline and Week 12.
  • Mean Serum Chemistries (Sodium) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's sodium levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Potassium) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's potassium levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Chloride) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's chloride levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Bicarbonate) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's bicarbonate levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Blood Urea Nitrogen) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's blood urea nitrogen levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Creatinine) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's creatinine levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Serum Chemistries (Glucose) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's glucose levels. This serum chemistry level was measured at Baseline and Week 12.
  • Mean Liver Function Tests Values (Alanine Transaminase) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's level of liver function through measure of alanine transaminase (ALT). This liver function test value was measured at Baseline and Week 12.
  • Mean Liver Function Tests Values (Aspartate Transaminase) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's level of liver function through measure of aspartate transaminase. This liver function test value was measured at Baseline and Week 12.
  • Mean Liver Function Tests Values (Total Bilirubin) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's level of liver function through measure of total bilirubin. This liver function test value was measured at Baseline and Week 12.
  • Mean Value of Thyroid Stimulating Hormone (TSH) [ Time Frame: Baseline, Week 12 ]
    This measure assesses the change in the patient's value of thyroid stimulating hormone. This hormone value was measured at Baseline and Week 12.
  • Least Squares Mean of Vital Signs (Weight) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    This measure assesses the change in the patient's weight (lbs). This vital sign was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Least Squares Mean of Vital Signs (Height) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    This measure assesses the change in the patient's height (centimeters). This vital sign was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Least Squares Mean of Vital Signs (Blood Pressure) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    This measure assesses the change in the patient's systolic and diastolic blood pressure. This vital sign was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
  • Least Squares Mean of Vital Signs (Heart Rate) [ Time Frame: Baseline, Week 4, Week 8, Week 12, Week 16 ]
    This measure assesses the change in the patient's heart rate (beats per minute). This vital sign was measured at Baseline, Week 4, Week 8, Week 12, and Week 16.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 16, 2016)
  • Change in SRS-2 subscale scores (Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests/ Repetitive Behaviors) from baseline to weeks 4, 8, 12 and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
  • Change in Aberrant Behavior Checklist (ABC) subscale scores (Social Withdrawal, Hyperactivity, Inappropriate Speech, Stereotypy, and Irritability) from baseline to weeks 4, 8, 12, and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
    The ABC is a 58-item caregiver questionnaire developed to assess medication effects in individuals with developmental disorders and includes 5 distinct subscales of behavioral symptoms.
  • Change in Clinical Global Impression-Severity (CGI-S) scores from baseline to weeks 4, 8, 12, and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
    The CGI-Severity (CGI-S) scale is a 7-point, clinician-rated scale providing an overall assessment of patient functioning relative to other patients with a similar diagnosis (1=not at all ill to 7=severely ill).
  • Differences in Clinical Global Impression- Improvement (CGI-I) scores between treatment arms at weeks 4, 8, 12, and 16. [ Time Frame: 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
    The CGI-Improvement (CGI-I) scale rates overall improvement or worsening of illness (ie, ASD) relative to baseline. The proportion of subjects in each treatment arm with CGI-I scores of 1 ("very much improved") or 2 ("much improved") at weeks 4, 8, 12, and 16 will be calculated and compared for between-group differences.
  • Change in Repetitive Behavior Scale-Revised (RBSR) total scores from baseline to weeks 4, 8, 12, and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
    The RBS-R is a 43-item, informant-based questionnaire designed to quantify a range of restricted, repetitive behaviors (RRB) observed in ASD.
  • Complete Blood Count (CBC) with differential, change in values from baseline to 12 weeks [ Time Frame: Baseline, 12 weeks ]
  • Liver function tests (alanine transaminase, aspartate transaminase, total bilirubin), change values from baseline to 12 weeks [ Time Frame: Baseline, 12 weeks ]
  • Serum chemistries (sodium, potassium, chloride, bicarbonate, Blood Urea Nitrogen, creatinine, calcium, magnesium, phosphorous, glucose), change in values from baseline to 12 weeks. [ Time Frame: Baseline and 12 weeks ]
  • Thyroid Stimulating Hormone (TSH), change in values from baseline to 12 weeks. [ Time Frame: Baseline and 12 weeks ]
  • Vital signs (weight, height, blood pressure, and heart rate), actual values at each time point measured and change in values from Baseline to Weeks 4, 8, 12, and 16. [ Time Frame: Baseline, 4 weeks, 8 weeks, 12 weeks, 16 weeks ]
  • Change in the concentration of sulforaphane metabolites (dithiocarbamates) in overnight voided urine samples from baseline to week 12, the end of the active treatment phase. [ Time Frame: Baseline and 12 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder
Official Title  ICMJE A Phase II, Randomized, Double-blind, Placebo-controlled Study of Myrosinase-enriched Glucoraphanin, a Sulforaphane Precursor System, in Autism Spectrum Disorder
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.
Detailed Description

Autism spectrum disorder (ASD) is a neurodevelopmental disorder affecting 1 in 68 children, including 1 in 42 boys, characterized by marked social communication impairment and restricted, repetitive behaviors and interests. Evidence-based pharmacological treatments available for the treatment of the defining symptoms of ASD are currently lacking.

While the etiology of ASD is not fully understood, the pathogenesis is hypothesized to involve cellular dysfunction, including increased oxidative stress, aberrant neuroinflammation, and reduced mitochondrial capacity, leading to synaptic dysfunction in at least a subset of individuals. Sulforaphane is a powerful upregulator of antioxidant response elements and heat shock proteins, which may lead to improved redox capacity, decreased inflammation, and improved mitochondrial functioning in individuals with ASD. A trial by Singh and colleagues (2014) provided preliminary evidence suggesting that sulforaphane derived from broccoli sprout extract can have beneficial effects for improving symptoms of autism.

In this study, young men ages 13-30 years old with moderate to severe autism spectrum disorder will be randomly assigned to receive either a sulforaphane supplement or placebo for a 12 week treatment treatment period, followed by a 4 week blinded discontinuation phase. The uncoated tablets each contain 125 mg broccoli seed extract and 50 mg broccoli sprout extract, corresponding to approximately 15 µmol sulforaphane per tablet. The dose will vary from 3-8 tablets daily depending upon the participant's weight. Matched placebo tablets contain only inert ingredients

A serum sample will be collected prior to starting treatment and at the end of the treatment phase to quantify sulforaphane metabolites. Clinical response will be assessed through clinician- and caregiver-rated measures of autism symptoms (Social Responsiveness Scale-2; Repetitive Behavior Scale- Revised), challenging symptoms commonly observed in individuals with developmental disabilities (Aberrant Behavior Checklist), and global severity of symptoms and improvement (Clinical Global Impression Scale). A blood sample will be collected at baseline and at the end of the treatment phase to check safety labs, and a saliva sample will be collected at baseline for a future study of genetic biomarkers associated with treatment response.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Autism Spectrum Disorder
  • Autistic Disorder
  • Neurodevelopmental Disorder
  • Childhood Developmental Disorders, Pervasive
Intervention  ICMJE
  • Drug: Sulforaphane
    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.
    Other Names:
    • Avmacol
    • glucoraphanin
  • Drug: Placebo
    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.
    Other Name: Control
Study Arms  ICMJE
  • Active Comparator: Sulforaphane

    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

    Intervention: Drug: Sulforaphane
  • Placebo Comparator: Placebo
    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.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 21, 2019)
48
Original Estimated Enrollment  ICMJE
 (submitted: September 16, 2016)
54
Actual Study Completion Date  ICMJE May 30, 2019
Actual Primary Completion Date May 30, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 13 Years to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02909959
Other Study ID Numbers  ICMJE 16-2059
5KL2TR001109-05 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: The investigators plan to publish the results of this trial in a peer-reviewed journal and on ClinicalTrials.gov upon completion of the study. No personal identifiable participant data will be included in the reporting of results.
Responsible Party University of North Carolina, Chapel Hill
Study Sponsor  ICMJE University of North Carolina, Chapel Hill
Collaborators  ICMJE North Carolina Translational and Clinical Sciences Institute
Investigators  ICMJE
Principal Investigator: Laura Politte, M.D. University of North Carolina, Chapel Hill
PRS Account University of North Carolina, Chapel Hill
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP