NCT02508259

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

The UCSD Suramin Autism Treatment-1 (SAT1) Trial

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: University of California, San Diego · Academic / Other
• Sex: Male
• Age: 4 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Autism Spectrum Disorders

Interventions

Type	Name	Description
DRUG	Suramin	20 mg/kg IV in 50 ml saline over 30 minutes
DRUG	Saline	50 ml IV over 30 minutes

Timeline

• Start date: 2015-05-01
• Primary completion: 2016-03-01
• Completion: 2016-04-01
• First posted: 2015-07-24
• Last updated: 2019-07-16
• Results posted: 2019-07-16

Locations

1 site across 1 country: United States