Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder
Condition: Autism Spectrum Disorder; Autistic Disorder; Neurodevelopmental Disorder; Childhood Developmental Disorders, Pervasive
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Developmental Disabilities
Study type: Interventional
Phase: Phase 2

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.
    

Intervention name(s): Sulforaphane; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): 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.; 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 intervention name(s): Avmacol; glucoraphanin; Control
Intervention MeSH terms: Sulforaphane
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: sulforaphane; glucoraphanin; physiologic effects of drugs