Open AccessLow‐dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial
Author(s) -
Naviaux Robert K.,
Curtis Brooke,
Li Kefeng,
Naviaux Jane C.,
Bright A. Taylor,
Reiner Gail E.,
Westerfield Marissa,
Goh Suzanne,
Alaynick William A.,
Wang Lin,
Capparelli Edmund V.,
Adams Cynthia,
Sun Ji,
Jain Sonia,
He Feng,
Arellano Deyna A.,
Mash Lisa E.,
Chukoskie Leanne,
Lincoln Alan,
Townsend Jeanne
Publication year - 2017
Publication title -
annals of clinical and translational neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.824
H-Index - 42
ISSN - 2328-9503
DOI - 10.1002/acn3.424
Subject(s) - medicine , suramin , autism spectrum disorder , randomized controlled trial , clinical trial , phase (matter) , autism , psychiatry , receptor , chemistry , organic chemistry
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 μ mol/L (mean ± SD) at 2 days and 1.5 ± 0.5 μ mol/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.