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

University of California San Diego

Status and phase

Completed

Phase 2

Phase 1

Conditions

Autism Spectrum Disorders

Treatments

Drug: Saline

Drug: Suramin

Study type

Interventional

Funder types

Other

Identifiers

NCT02508259

15-0134

Details and patient eligibility

About

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

Full 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.

Enrollment

10 patients

Sex

Male

Ages

4 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Autism diagnostic observation schedule (ADOS) score of ≥ 7
Diagnosis of autism spectrum disorder by Diagnostic and Statistical Manual, 5th edition (DSM-V)
Stable treatment and diet regimen for ≥ 2 months
Resident of San Diego region

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 intravenous catheter 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