Sulforaphane Treatment of Children With Autism Spectrum Disorder (ASD)
Status:
Completed
Trial end date:
2020-01-01
Target enrollment:
Participant gender:
Summary
ASD is a diverse disorder starting in early childhood and characterized by social
communication impairment as well as restricted interests and repetitive behaviors. It affects
1:68 children and is an enormous medical and economic problem for which there is no
established, mechanism-based treatment. Sulforaphane is an isothiocyanate derived from
broccoli, and has potent activity in transcriptionally up-regulating genes that control
mechanisms whereby aerobic cells protect themselves against oxidative stress, mitochondrial
dysfunction, and inflammation.
This study is a clinical trial of oral sulforaphane (as broccoli seed powder) in 50 boys and
girls (3-12 years) with ASD in 3 phases over 36 weeks. In Phase 1, 25 children will receive
active drug and 25 will receive placebo for 15 weeks; in Phase 2, all children will receive
sulforaphane from 15-30 weeks; in Phase 3, children will receive no treatment for 6 weeks.
Study visits will take place at screening, 7, 15, 22, 30 and 36 weeks, when the Ohio Autism
Clinical Clinical Impressions Scale - Severity and Improvement (OACIS-S and OACIS-I),
Aberrant Behavior Checklist (ABC) and Social Responsiveness Scale (SRS) will be recorded.
Children will be monitored with physical examinations and for toxicity with clinical
laboratory studies and examine possible biomarkers: Nuclear factor-erythroid factor 2 (Nrf2),
oxidative stress and mitochondrial function, the mechanistic target of rapamycin (mTOR)
pathway and cytokine expression. In addition, prior to the main clinical trial, a pilot study
will be carried out in 10 children with ASD, 6-12 years of age, who will receive
sulforaphane, 2.2 micromoles/kg daily for 14 days. Blood and urine samples before and at the
end of treatment will be collected, in order to measure several parameters that are likely to
demonstrate expected effects of sulforaphane, to standardize the assays and procedures, and
to determine the most effective measures.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
University of Massachusetts, Worcester
Collaborators:
Congressionally Directed Medical Research Programs Johns Hopkins University