University of California, San Diego (UCSD) Suramin Autism Treatment-1 (SAT1) Trial

Last updated: June 21, 2019
Sponsor: University of California, San Diego
Overall Status: Completed

Phase

1/2

Condition

Asperger's Disorder

Autism

Williams Syndrome

Treatment

N/A

Clinical Study ID

NCT02508259
15-0134
  • Ages 4-17
  • Male

Study Summary

This study is designed to test the safety and efficacy of a single, intravenous dose of suramin in autism spectrum disorders (ASD).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Autism diagnostic observation schedule (ADOS) score of ≥ 7

  • Diagnosis of autism spectrum disorder by Diagnostic and Statistical Manual, 5thedition (DSM-V)

  • Stable treatment and diet regimen for ≥ 2 months

  • Resident of San Diego region

Exclusion

Exclusion Criteria:

  • Any prescription medications

  • Hospitalization within the previous 2 months

  • Active medical problem such as seizures, heart, liver, kidney, or adrenal disease

  • Planning to start a new drug, diet, or behavioral intervention during the study

  • Weight under the 5th percentile for age

  • Unable to tolerate venipuncture, urine collection, or an indwelling intravenouscatheter for 3-4 hours

  • Plasma creatinine ≥ 1.4 mg/dl

  • Liver function alanine amino transferase (ALT) or aspartate amino transferase (AST) ≥ 1.5-fold above the upper limit of normal

  • Known intolerance to suramin or other antipurinergic drugs

  • Unable to perform or cooperate with study requirements

Study Design

Total Participants: 10
Study Start date:
May 01, 2015
Estimated Completion Date:
April 30, 2016

Study Description

This study is designed to test a new theory of the origin and treatment of ASD. In this theory, ASD is caused by both genes and environment interacting to produce a persistent cell danger response (CDR; Naviaux RK, 2014) that interferes with and alters normal child brain development. Gut microbiome and immune systems are also affected. In this theory, the pathological persistence of the cell danger response is traceable to mitochondria, and maintained by purinergic signaling mediated by the release of extracellular nucleotides like adenosine triphosphate (ATP), adenosine diphosphate (ADP), uridine triphosphate (UTP), and uridine diphosphate (UDP). Suramin inhibits excess purinergic signaling by acting as a competitive inhibitor of nucleotide signaling at both ionotropic purinergic (P2X) receptors, and G-protein coupled, metabotropic purinergic (P2Y) receptors. Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism (Naviaux JC, et al. 2015; Naviaux JC, et al. 2014; Naviaux RK, et al. 2013). This study will test the safety and efficacy of a single dose of suramin in children with ASD. While it is not anticipated that a single dose will produce benefits for more than a few weeks, if successful, this study may lead to the development of newer and safer drugs for autism treatment.

Connect with a study center

  • University of California, San Diego School of Medicine

    La Jolla, California 92093
    United States

    Site Not Available

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