Phase 2
N=48
Sulforaphane for the Treatment of Young Men With Autism Spectrum Disorder
Autism Spectrum Disorder · Autistic Disorder · Neurodevelopmental Disorder · Childhood Developmental Disorders, Pervasive
Bottom Line
View on ClinicalTrials.gov: NCT02909959 ↗Enrolled (actual)
48
Serious AEs
0.0%
Results posted
May 2020
Primary outcome: Primary: Social Responsiveness Scale-2 (SRS-2) Total Score at Baseline — 75.6; 72.6 T-score — p=0.331
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sulforaphane (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 13+ yrs
- Sex
- Male
- Sponsor
- University of North Carolina, Chapel Hill
- Primary completion
- May 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Social Responsiveness Scale-2 (SRS-2) Total Score at Baseline |
75.6; 72.6 | 0.331 |
| PRIMARY Social Responsiveness Scale-2 (SRS-2) Total Score at Week 4 |
74.1; 74.1 | 0.98 |
| PRIMARY Social Responsiveness Scale-2 (SRS-2) Total Score at Week 8 |
73.7; 74.5 | 0.797 |
| PRIMARY Social Responsiveness Scale-2 (SRS-2) Total Score at Week 12 |
75.3; 72.9 | 0.442 |
| PRIMARY Social Responsiveness Scale-2 (SRS-2) Total Score at Week 16 |
75.5; 72.7 | 0.373 |
| SECONDARY Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Awareness) |
10.9; 10.9; 10.5; 11.3; 10.6; 11.3 | — |
| SECONDARY Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Cognition) |
19.1; 17.4; 18.3; 18.2; 18.2; 18.4 | — |
| SECONDARY Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Communication) |
34.0; 32.0; 32.4; 33.6; 32.6; 33.5 | — |
| SECONDARY Social Responsiveness Scale-2 (SRS-2) Subscale Score (Social Motivation) |
15.5; 14.7; 14.8; 15.4; 14.6; 15.6 | — |
| SECONDARY Social Responsiveness Scale-2 (SRS-2) Subscale Score (Restricted Interests/Repetitive Behaviors) |
18.6; 17.6; 18.1; 18.2; 17.7; 18.5 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) Subscale Score (Social Withdrawal) |
8.7; 8.3; 8.2; 8.8; 8.2; 8.8 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) Subscale Scores (Hyperactivity) |
14.3; 10.8; 13.0; 12.1; 12.6; 12.5 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) Subscale Score (Inappropriate Speech) |
3.4; 2.8; 3.2; 2.9; 3.0; 3.1 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) Subscale Score (Stereotypy) |
1.3; 2.2; 1.1; 2.4; 1.0; 2.5 | — |
| SECONDARY Aberrant Behavior Checklist (ABC) Subscale Score (Irritability) |
9.6; 8.3; 9.4; 8.6; 9.7; 8.3 | — |
| SECONDARY Clinical Global Impression-Severity (CGI-S) Score |
4.74; 4.56; 4.70; 4.60; 4.71; 4.58 | — |
| SECONDARY Number of Participants With Clinical Global Impression-Improvement (CGI-I) Score of Much Improved or Very Much Improved |
3; 1; 21; 23; 4; 2 | — |
| SECONDARY Repetitive Behavior Scale-Revised (RBSR) Total Score |
23.0; 22.3; 21.7; 23.5; 23.3; 22.0 | — |
| SECONDARY Mean Red Blood Cell Value |
5.0; 5.1; 5.1; 5.0 | — |
| SECONDARY Mean White Blood Cell Value |
6.3; 6.5; 6.0; 6.9 | — |
| SECONDARY Mean Hemoglobin Value |
14.8; 15.0; 15.0; 14.5 | — |
| SECONDARY Mean Hematocrit Value |
43.8; 44.4; 44.4; 43.2 | — |
| SECONDARY Mean Corpuscular Volume Value |
87.3; 86.8; 87.8; 86.6 | — |
| SECONDARY Mean Corpuscular Hemoglobin Value |
29.4; 29.3; 29.7; 29.2 | — |
| SECONDARY Mean Corpuscular Hemoglobin Concentration Value |
33.8; 33.9; 33.8; 33.7 | — |
| SECONDARY Mean Red Blood Cell Distribution Width Value |
13.6; 13.7; 13.6; 13.6 | — |
| SECONDARY Mean Platelets Value |
253.8; 286.0; 246.0; 279.3 | — |
| SECONDARY Mean Absolute Neutrophils Value |
3.5; 3.7; 3.3; 6.3 | — |
| SECONDARY Mean Absolute Lymphocytes Value |
2.1; 2.1; 1.9; 3.5 | — |
| SECONDARY Mean Absolute Monocytes Value |
0.5; 0.6; 0.5; 0.9 | — |
| SECONDARY Mean Absolute Eosinophils Value |
0.2; 0.1; 0.2; 0.2 | — |
| SECONDARY Mean Absolute Basophils Value |
0.013; 0.013; 0.004; 0.009 | — |
| SECONDARY Mean Serum Chemistries (Sodium) |
140.6; 141.0; 141.8; 141.3 | — |
| SECONDARY Mean Serum Chemistries (Potassium) |
4.4; 4.5; 4.7; 4.5 | — |
| SECONDARY Mean Serum Chemistries (Chloride) |
100.1; 99.6; 100.1; 100.7 | — |
| SECONDARY Mean Serum Chemistries (Bicarbonate) |
22.8; 22.3; 23.1; 22.8 | — |
| SECONDARY Mean Serum Chemistries (Blood Urea Nitrogen) |
13.0; 11.4; 13.3; 11.6 | — |
| SECONDARY Mean Serum Chemistries (Creatinine) |
0.8; 0.8; 0.8; 0.8 | — |
| SECONDARY Mean Serum Chemistries (Glucose) |
90.9; 91.1; 87.0; 86.3 | — |
| SECONDARY Mean Liver Function Tests Values (Alanine Transaminase) |
22.1; 26.0; 29.6; 20.7 | — |
| SECONDARY Mean Liver Function Tests Values (Aspartate Transaminase) |
22.3; 21.9; 25.2; 19.8 | — |
| SECONDARY Mean Liver Function Tests Values (Total Bilirubin) |
0.5; 0.4; 0.5; 0.5 | — |
| SECONDARY Mean Value of Thyroid Stimulating Hormone (TSH) |
2.3; 2.2; 2.3; 1.8 | — |
| SECONDARY Least Squares Mean of Vital Signs (Weight) |
147.1; 148.3; 147.2; 148.2; 146.8; 148.6 | — |
| SECONDARY Least Squares Mean of Vital Signs (Height) |
173.9; 173.3; 173.9; 173.3; 173.9; 173.3 | — |
| SECONDARY Least Squares Mean of Vital Signs (Blood Pressure) |
115.2; 116.7; 69.8; 70.6; 116.3; 115.5 | — |
| SECONDARY Least Squares Mean of Vital Signs (Heart Rate) |
86.1; 87.5; 88.0; 85.6; 87.8; 85.7 | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT02909959). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.