Trial · NCT02561481

Sulforaphane Treatment of Children With Autism Spectrum Disorder (ASD)

NCT02561481, Phase 1, Phase 2, Completed, University of Massachusetts, Worcester; results first posted 2020-11-25

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Sulforaphane Treatment of Children With Autism Spectrum Disorder (ASD). Trials, record NCT02561481. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02561481

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Sulforaphane Treatment of Children With Autism Spectrum Disorder (ASD)
NCT number: NCT02561481
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 60 participants, actual
Lead sponsor: University of Massachusetts, Worcester
Conditions: Autism Spectrum Disorder
Interventions: Sulforaphane, Placebo
First posted: 2015-09-28
Last updated: 2020-12-17
Results first posted: 2020-11-25
Limitations and caveats: The sample size was limited to 45 children with ASD and we could not impute missing data for those participants who were lost to follow up.

Source ClinicalTrials.gov

Registered outcomes17

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	The Ohio Autism Clinical Impressions Scale-Severity (OACIS-S) rates severity of symptoms in 10 categories: general level of autism, social interaction, aberrant and repetitive behavior, verbal and nonverbal communication, hyperactivity, anxiety, sensory sensitivities and restricted and narrow interests. Each category is rated from 1 (normal) to 7 (most severe). The OACIS-S was a reference at follow up visits when the OACIS-Improvement was compared to the OACIS-S, from 1 to 7: 4 was no change; 3 to 1 minimal to marked improvement and 5 to 7 minimal to marked worsening. The numerical score of the OACIS-S is independent and unrelated quantitatively to the OACIS-I. For analysis, the OACIS-I general score and subscale values were recoded, in which 4 (no change) was recoded as 0; 3 to 1 were recoded as +1 to +3 to denote improvement, and 5 to 7 were recoded as -1 to -3 for worsening.
Secondary	OACIS-I Response Rate on Aberrant Behaviors Subscale	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	See above in the primary outcome measure for a description of OACIS-I scale. This section describes the change from baseline of the OACIS-I subdomain of aberrant behaviors. This subdomain has a range of 1 to 7 - 1 is extremely improved from baseline, 7 is extremely worse from baseline, and 4 is no change. For analysis, the OACIS-I general score and subscale values were recoded, in which 4 (no change) was recoded as 0; 3 to 1 were recoded as +1 to +3 to denote improvement, and 5 to 7 were recoded as -1 to -3 for worsening.
Secondary	OACIS-I Response Rate on Social Communication Subscale	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	See above in the primary outcome measure for a description of OACIS-I scale. This section describes the change from baseline of the OACIS-I subdomain of social communication. This subdomain has a range of 1 to 7 - 1 is extremely improved from baseline, 7 is extremely worse from baseline, and 4 is no change. For analysis, the OACIS-I general score and subscale values were recoded, in which 4 (no change) was recoded as 0; 3 to 1 were recoded as +1 to +3 to denote improvement, and 5 to 7 were recoded as -1 to -3 for worsening.
Secondary	Change in Total Aberrant Behavior Checklist Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Aberrant Behavior Checklist (ABC) is a 58 item scale that primarily evaluates how aberrant or abnormal a patient's daily behaviors are. The items evaluate behaviors as they pertain to irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech. Each item is scored on a scale of 0 to 3, with 0 being better outcome and 3 being worse outcome. The score from each item is added up to calculate a total score. This outcome describes change in total ABC score from baseline at each follow up visit. The scores from all items are added to calculate a total score (0 to 174). This outcome describes change in total ABC score from baseline at each follow up visit.
Secondary	Change in Total SRS-2 Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	The Social Responsiveness Scale-2 (SRS-2) is a 65-item scale that measures total scores as well as subscales: four social behaviors (awareness, cognition, communication and motivation) and autistic mannerisms. Each item is rated from 1 to 4 (not true to almost always true) on worksheets that are blinded to the rater with respect to values. Total and subscale scores are calculated as raw scores and can be converted to T-scores. Raw scores (range 0-180) are reported here (unadjusted for general population, since all children had ASD). Higher or lower values at follow up visits compared to baseline indicated worsening or improvement, respectively.
Secondary	Dithiocarbamate Plasma Concentration Detected by Cyclocondensation at Each Visit	Week 0, Week 7, Week 15, Week 22, Week 30, Week 36	Sulforaphane (and other isothiocyanates, ITC) are conjugated by glutathione (GSH) which then undergoes further enzymatic modifications to give rise sequentially to the cysteinylglycine-, cysteine- and N-acetylcysteine-ITC conjugates, all of which are dithiocarbamates (DTC) and are detected in the cyclocondensation reaction-HPLC assay.
Secondary	Comparison of Free Reduced Glutathione (GSH), Total GSH, Oxidized Glutathione (GSSG) at Week 0 and 15	Week 0 and Week 15	F-statistic calculated by comparison of Free Reduced GSH, Total GSH and oxidized Glutathione (GSSG) of Week 15 to Week 0.
Secondary	Free GSH:GSSG and Total GSH:GSSG Ratios at Week 15	Week 15	Ratios of free GSH:GSSG and total GSH:GSSG were calculated by obtaining ratios of f-statistic scores from baseline to week 15 between free reduced GSH and GSSG and between total GSH and GSSG.
Other	NQO1: NAD(P)H:Quinone Oxidoreductase-1	Week 0, Week 15, Week 30	Cytoprotective enzyme regulated by nuclear factor erythroid 2-related factor 2 (Nrf2), the master regulator of cellular redox homeostasis and an inhibitor of a key pro-inflammatory pathway, of which both functions are critical factors in the neuropathology of ASD. Total cellular RNA was isolated from peripheral blood mononuclear cells (PBMCs) and complementary DNAs (cDNA) were synthesized. Quantitative real-time PCR analysis was performed using the Applied Biosystems QuantStudio™ 3 Real-Time PCR System (Thermo Fisher Scientiﬁc, Waltham, MA, USA). Relative mRNA expression was normalized to GAPDH. Gene expression was calculated using the comparative 2-ΔΔCT method.
Other	xCT	Week 0, Week 15, Week 30	xCT (SLC7A11): Cystine/glutamate antiporter encoded by the SLC7A11 gene.Total cellular RNA was isolated from peripheral blood mononuclear cells (PBMCs) and complementary DNAs (cDNA) were synthesized. Quantitative real-time PCR analysis was performed using the Applied Biosystems QuantStudio™ 3 Real-Time PCR System (Thermo Fisher Scientiﬁc, Waltham, MA, USA). Relative mRNA expression was normalized to GAPDH. Gene expression was calculated using the comparative 2-ΔΔCT method.

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes17

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Patients started dropping out at subsequent visits so the number analyzed at latter visits is less	score on a scale	MEAN
SECONDARY	OACIS-I Response Rate on Aberrant Behaviors Subscale	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Patients started dropping out at subsequent visits so the number analyzed at latter visits is less	score on a scale	MEAN
SECONDARY	OACIS-I Response Rate on Social Communication Subscale	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Patients started dropping out at subsequent visits so the number analyzed at latter visits is less	score on a scale	MEAN
SECONDARY	Change in Total Aberrant Behavior Checklist Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Patients started dropping out at subsequent visits so the number analyzed at later visits is less	score on a scale	MEAN
SECONDARY	Change in Total SRS-2 Score From Baseline	7 weeks, 15 weeks, 22 weeks, 30 weeks, 36 weeks	Patients dropped out (or families did not return forms) at subsequent visits so the number analyzed at later visits is less.	score on a scale	MEAN
SECONDARY	Dithiocarbamate Plasma Concentration Detected by Cyclocondensation at Each Visit	Week 0, Week 7, Week 15, Week 22, Week 30, Week 36	Number analyzed varied due to participants who dropped out, their blood samples could not be obtained, or data could not be obtained from the assay.	nmol DTC (Dithiocarbamates)/ml	MEAN
SECONDARY	Comparison of Free Reduced Glutathione (GSH), Total GSH, Oxidized Glutathione (GSSG) at Week 0 and 15	Week 0 and Week 15		F-statistic	NUMBER
SECONDARY	Free GSH:GSSG and Total GSH:GSSG Ratios at Week 15	Week 15		F-statistic	NUMBER
OTHER_PRE_SPECIFIED	NQO1: NAD(P)H:Quinone Oxidoreductase-1	Week 0, Week 15, Week 30	N varies due to missing data, variable clinic visits or assay failure.	log fold change	MEAN
OTHER_PRE_SPECIFIED	xCT	Week 0, Week 15, Week 30	N varies due to missing data, variable clinic visits or assay failure.	log fold change	MEAN

1 to 10 of 17Source ClinicalTrials.gov

See all 17 outcomes

Results by group126

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Sulforaphane	7 weeks	0.28	Standard Deviation 0.67	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Placebo	7 weeks	0.28	Standard Deviation 0.57	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Sulforaphane	15 weeks	0.28	Standard Deviation 0.57	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Placebo	15 weeks	0.33	Standard Deviation 0.69	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Sulforaphane	22 weeks	0.47	Standard Deviation 0.72	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Placebo	22 weeks	0.59	Standard Deviation 0.71	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Sulforaphane	30 weeks	0.47	Standard Deviation 0.67	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Placebo	30 weeks	0.69	Standard Deviation 0.77	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Sulforaphane	36 weeks	0.29	Standard Deviation 0.73	score on a scale
Change in Ohio Autism Clinical Impressions Scale - Improvement (OACIS-I) Average Score From Baseline	Placebo	36 weeks	0.29	Standard Deviation 0.61	score on a scale

1 to 10 of 126Source ClinicalTrials.gov

See all 126 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Pilot Trial	STARTED	Sulforaphane	10
Pilot Trial	STARTED	Placebo	0
Pilot Trial	COMPLETED	Sulforaphane	10
Pilot Trial	COMPLETED	Placebo	0
Pilot Trial	NOT COMPLETED	Sulforaphane	0
Pilot Trial	NOT COMPLETED	Placebo	0
Main Trial - Double Blind Phase	STARTED	Sulforaphane	25
Main Trial - Double Blind Phase	STARTED	Placebo	25
Main Trial - Double Blind Phase	COMPLETED	Sulforaphane	22
Main Trial - Double Blind Phase	COMPLETED	Placebo	23

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events10

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	insomnia	Nervous system disorders	Sulforaphane	2	32
other	insomnia	Nervous system disorders	Placebo		23
other	irritability	Nervous system disorders	Sulforaphane	2	32
other	irritability	Nervous system disorders	Placebo		23
other	taste/smell	General disorders	Sulforaphane	2	32
other	taste/smell	General disorders	Placebo		23
other	gastrointestinal upset	Gastrointestinal disorders	Sulforaphane	2	32
other	gastrointestinal upset	Gastrointestinal disorders	Placebo		23
serious	Hashimoto thyroiditis	Endocrine disorders	Sulforaphane	0	32	0
serious	Hashimoto thyroiditis	Endocrine disorders	Placebo	1	23	1

Source ClinicalTrials.gov

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Sulforaphane	0	32
Total, all-cause mortality	Placebo	0	23
Total, other adverse events	Sulforaphane	8	32
Total, other adverse events	Placebo	0	23
Total, serious adverse events	Sulforaphane	0	32
Total, serious adverse events	Placebo	1	23

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
25313065	RESULT	PubMed
32242086	RESULT	PubMed
34034808	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Sulforaphane vs Placebo: deaths from any cause	Trial comparisons
Sulforaphane vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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