Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial

Autor:	Brooke Curtis, Kefeng Li, Jane C. Naviaux, A. Taylor Bright, Marissa Westerfield, Cynthia Adams, Edmund V. Capparelli, Feng He, Jeanne Townsend, Sonia Jain, Suzanne Goh, Lisa E. Mash, Leanne Chukoskie, Robert K. Naviaux, Lin Wang, Alan J. Lincoln, William A. Alaynick, Ji Sun, Deyna A. Arellano, Gail E. Reiner
Rok vydání:	2017
Předmět:	0301 basic medicine medicine.medical_specialty Suramin Asymptomatic law.invention 03 medical and health sciences 0302 clinical medicine Randomized controlled trial law Internal medicine Medicine Psychiatry Adverse effect Research Articles business.industry General Neuroscience medicine.disease 030104 developmental biology Autism spectrum disorder Clinical Global Impression Autism Autism Treatment Evaluation Checklist Neurology (clinical) medicine.symptom business 030217 neurology & neurosurgery Research Article medicine.drug
Zdroj:	Annals of Clinical and Translational Neurology
ISSN:	2328-9503
DOI:	10.1002/acn3.424
Popis:	Objective No drug is yet approved to treat the core symptoms of autism spectrum disorder (ASD). Low-dose suramin was effective in the maternal immune activation and Fragile X mouse models of ASD. The Suramin Autism Treatment-1 (SAT-1) trial was a double-blind, placebo-controlled, translational pilot study to examine the safety and activity of low-dose suramin in children with ASD. Methods Ten male subjects with ASD, ages 5–14 years, were matched by age, IQ, and autism severity into five pairs, then randomized to receive a single, intravenous infusion of suramin (20 mg/kg) or saline. The primary outcomes were ADOS-2 comparison scores and Expressive One-Word Picture Vocabulary Test (EOWPVT). Secondary outcomes were the aberrant behavior checklist, autism treatment evaluation checklist, repetitive behavior questionnaire, and clinical global impression questionnaire. Results Blood levels of suramin were 12 ± 1.5 μmol/L (mean ± SD) at 2 days and 1.5 ± 0.5 μmol/L after 6 weeks. The terminal half-life was 14.7 ± 0.7 days. A self-limited, asymptomatic rash was seen, but there were no serious adverse events. ADOS-2 comparison scores improved by −1.6 ± 0.55 points (n = 5; 95% CI = −2.3 to −0.9; Cohen's d = 2.9; P = 0.0028) in the suramin group and did not change in the placebo group. EOWPVT scores did not change. Secondary outcomes also showed improvements in language, social interaction, and decreased restricted or repetitive behaviors. Interpretation The safety and activity of low-dose suramin showed promise as a novel approach to treatment of ASD in this small study.
Databáze:	OpenAIRE
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