Clinical trial NCT02879110: A 12-weeks Study to Evaluate Sulforaphane in Treatment of Autism Spectrum Disorder
Official title: A 12-weeks, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety and Related Mechanism of Sulforaphane in Treatment of Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Autism Spectrum Disorder
Keywords: Sulforaphane
clinic trial
Autism Spectrum Disorder
Efficacy
Safety
Mechanism
Lead sponsor: Central South University
Sponsor class: OTHER
Collaborators: name: Davis family funding
name: University of California
name: University of Illinois at Chicago
First posted: 2016-08-25
Enrollment: 110
Sex: ALL
Minimum age (years): 3
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: In this proposed study, the investigators will evaluate the the efficacy, safety and related mechanism of sulforaphane in treatment of autism spectrum disorder (ASD). The study will recruit 120 ASD patients, then these patients will be randomized to sulforaphane group or placebo group (60 patients per arm) for 12 weeks clinic trial. Clinical efficacy and safety assessment will be done at screen/baseline, 4 week, 8 week and 12 week. The specific aims are to compare sulforaphane versus placebo on: 1) clinical core symptoms; 2) other behavioral problems and adaptive behaviors. Biological samples also will be collected, and stored to research related mechanisms.

Detailed description: In this proposed study, the investigators will evaluate the the efficacy, safety and related mechanism of sulforaphane in treatment of autism spectrum disorder (ASD). The study will recruit 120 ASD patients, then these patients will be randomized to sulforaphane group or placebo group (60 patients per arm) for 12 weeks clinic trial. Clinical efficacy and safety assessment will be done at screen/baseline, 4 week, 8 week and 12 week. The specific aims are to compare sulforaphane versus placebo on: 1) clinical core symptoms; 2) other behavioral problems and adaptive behaviors. The investigators hypothesize that (1) sulforaphane is superior to placebo in the treatment of clinical symptoms in patients with ASD, measured by the Social Responsiveness Scale, Aberrant Behavior Checklist, Repetitive Behavior Scale - Revised and Ohio State University Autism Clinical Global Impression; (2) sulforaphane is superior to placebo in the treatment of other behavioral problems and adaptive behaviors patients with ASD, measured by Achenbach's Child Behavior Checklist and Adaptive Behavior Assessment System, Second Edition; and (3) Biological samples will be collected, and stored so that the hypothesis sulforaphane may alter oxidative stress indexes or inflammatory biomarkers, and influence histone deacetylase inhibitor mechanism or inflammatory mechanism et al that may be significantly correlated with clinical improvement.

Eligibility criteria: Inclusion Criteria:

1. Aged 3 to 15 years.
2. Meet DSM-V diagnostic criteria for autism spectrum disorder, and been checked with Autism Diagnostic Interview-Revised (ADI-R) and Autism Diagnostic Observation Schedule (ADOS).

Exclusion Criteria:

1. With severe physical disease (i.e. congenital heart disease, thyroid disease, diseases with severe abnormality of liver or kidney function, diseases with abnormality vision or hearing et al.)
2. With severe central nervous system disease (i.e. epilepsy et al).
3. With other specific genetic syndromes (i.e. Fragile-X syndrome, Down's syndrome et al.)

Primary outcomes:
measure: The change of social impairments of children with autism spectrum disorder
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
Secondary outcomes:
measure: The change of rigid interests and behaviors of children with autism spectrum disorder
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
measure: The change of clinical symptoms of children with autism spectrum
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
measure: The change of other behavioral problems of children with autism spectrum
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
measure: The change of adaptive behaviors of children with autism spectrum
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
measure: The change of clinical general impression of children with autism spectrum
timeFrame: At baseline, 4 week, 8 week and 12 week/endpoint
measure: The change of heart rate as measured by stopwatch
timeFrame: At baseline and 12 week/endpoint
measure: The change of weight as measured by weighing-machine
timeFrame: At baseline and 12 week/endpoint
measure: The change of height as measured by Height measurement tools
timeFrame: At baseline and 12 week/endpoint
measure: The change of blood routine test as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of fasting blood-glucose as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of blood lipid as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of liver function as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of kidney function as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of thyroid function as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of HBV test as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of helicobacter pylori test as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: The change of urine routine test as tested by clinical laboratory
timeFrame: At baseline and 12 week/endpoint
measure: Number of participants with treatment-related adverse events as assessed by Systematic Assessment for Treatment Emergent Effects
timeFrame: At 4 week, 8 week and 12 week/endpoint
Other outcomes:
measure: The change of Oxidative stress indexes as tested by Oxidative stress indexes detection kit
timeFrame: At baseline and 12 week/endpoint
measure: The change of Epigenetics indicators as tested by Epigenetics indicators
timeFrame: At baseline and 12 week/endpoint
measure: The change of Cytokines & Chemokines as tested by Cytokines & Chemokines detection kit
timeFrame: At baseline and 12 week/endpoint
measure: The change of Metabolites as tested by Metabolites detection kit
timeFrame: At baseline and 12 week/endpoint
measure: The change of RNA expression as tested by RNA expression detection kit
timeFrame: At baseline and 12 week/endpoint
measure: The change of intestinal microflora as tested by Metagenomic technique
timeFrame: At baseline and 12 week/endpoint
Locations:
city: Guangzhou
country: China
facility: Guangzhou Huiai Hospital
state: Guangdong
city: Changsha
country: China
facility: The second Xiangya hospital of central south university
state: Hunan
Study officials:
affiliation: Central South University
name: Jingping Zhao, M.D., Ph. D.
role: STUDY_CHAIR
affiliation: Central South University
name: Jianjun Ou, M.D., Ph. D.
role: STUDY_DIRECTOR
affiliation: Department of Psychiatry, University of California
name: Hua Jin, M.D., Ph. D.
role: STUDY_DIRECTOR
affiliation: Global Clinical and Translational Research Institute
name: Fengyu Zhang, Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Guangzhou Huiai Hospital
name: Daomeng Cheng, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Central South University
name: Renrong Wu, M.D.,Ph.D
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Psychiatry, University of Illinoisat at Chicago
name: John M Davis, M.D.,Ph.D
role: STUDY_DIRECTOR
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