Brief Report: An Open-Label Study of the Neurosteroid Pregnenolone in Adults with Autism Spectrum Disorder

• Published in: Journal of autism and developmental disorders Vol. 44; no. 11; pp. 2971 - 2977
• Main Authors: Fung, Lawrence K., Libove, Robin A., Phillips, Jennifer, Haddad, Francois, Hardan, Antonio Y.
• Format: Journal Article
• Language: English
• Published: Boston Springer US 01.11.2014; Springer; Springer Nature B.V
• Subjects: Aberrant Behavior Checklist; Adolescent; Adult; Adults; Anatomy; Autism; Autism Spectrum Disorders; Autistic adults; Behavior Change; Behavior Problems; Behavior Rating Scales; Behavioral Science and Psychology; Behavioral Sciences; Biological and medical sciences; Brief Report; Child and School Psychology; Child clinical studies; Child Development Disorders, Pervasive - drug therapy; Child Development Disorders, Pervasive - psychology; Complications and side effects; Control Groups; Criminality; Developmental disorders; Diarrhea; Dosage and administration; Drug Therapy; Efficacy; Evidence; Female; Humans; Infantile autism; Irritability; Irritable Mood - drug effects; Lethargy; Male; Medical sciences; Mental depression; Mental Disorders; Meta Analysis; Neurosciences; Outcomes of Treatment; Patients; Pediatrics; Pervasive Developmental Disorders; Pilot Projects; Pregnenolone - pharmacology; Pregnenolone - therapeutic use; Psychiatry; Psychology; Psychology. Psychoanalysis. Psychiatry; Psychopathology. Psychiatry; Psychotropic drugs; Public Health; Schizophrenia; Short Term Memory; Side effects; Sign changes; Statistical Significance; Symptoms (Individual Disorders); Treatment Outcome; Young Adult; United States; Irritability; Open-label trial; Neurosteroids; Autism spectrum disorder; Pregnenolone; Human; Corticosteroid; Antiinflammatory agent; Developmental disorder; Neurosteroid; Autism; Pervasive developmental disorder; Adrenal hormone; Adult
• ISSN: 0162-3257; 1573-3432; 1573-3432
• DOI: 10.1007/s10803-014-2144-4

The objective of this study was to assess the tolerability and efficacy of pregnenolone in reducing irritability in adults with autism spectrum disorder (ASD). This was a pilot, open-label, 12-week trial that included twelve subjects with a mean age of 22.5 ± 5.8 years. Two participants dropped out of the study due to reasons unrelated to adverse effects. Pregnenolone yielded a statistically significant improvement in the primary measure, Aberrant Behavior Checklist (ABC)-Irritability [from 17.4 ± 7.4 at baseline to 11.2 ± 7.0 at 12 weeks ( p  = 0.028)]. Secondary measures were not statistically significant with the exception of ABC-lethargy ( p  = 0.046) and total Short Sensory Profile score ( p  = 0.009). No significant vital sign changes occurred during this study. Pregnenolone was not associated with any severe side effects. Single episodes of tiredness, diarrhea and depressive affect that could be related to pregnenolone were reported. Overall, pregnenolone was modestly effective and well-tolerated in individuals with ASD.