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Completed

NCT Number: NCT02508259

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

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

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Key information

Conditions

Autism Spectrum Disorders Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

4 year–17 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of California, San Diego School of Medicine

La Jolla, California, 92093, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

suramin

Drug

20 mg/kg IV in 50 ml saline over 30 minutes

Other names: Germanin

Saline

Drug

50 ml IV over 30 minutes

Other names: Normal saline

Primary outcomes

  1. Autism Diagnostic Observation Schedule, 2nd Edition (ADOS2)

    Time frame: 6 weeks compared to baseline

    ADOS2 comparison scores are units on a scale of 0-10. A score of 7-10 was required for enrollment. A score of 7-10 is diagnostic for autism spectrum disorder (ASD). The higher the score, the more severe the core symptoms of autism spectrum disorder. Scores of 6 and below are considered off the ASD spectrum.

  2. Expressive Language

    Time frame: 6 weeks compared to baseline

    Expressive One Word Picture Vocabulary Test (EOWPVT) scores are normalized for age. Typical language development produces a mean score of 100 with a standard deviation of 15. Outcomes for EOWPVT were expressed as the mean of the child-specific difference before and 6-weeks after treatment. For example, if the 6-week standard EOWPVT score was 59.6 and the baseline score was 63.8, the difference is -4.2 (= 59.6 - 63.8). A decrease in score at 6 weeks would corresponds to a decrease in language performance, while an increase, a positive difference, would reflect an increase.

Secondary outcomes

  1. Aberrant Behavior Checklist (ABC)

    Time frame: 6 weeks compared to baseline

    The full ABC is a 58-item parent rating with five factors: Irritability, Social Withdrawal, Stereotypy, Hyperactivity and Inappropriate Speech. Stereotypy is reported, and scores range from 0 to 21, with higher scores indicating worse behavior. A negative difference corresponds to decreased symptoms after treatment. A positive difference corresponds to increased symptoms after treatment.

  2. Autism Treatment Evaluation Checklist (ATEC)

    Time frame: 6 weeks

    The reported value is the Language sub-score of the ATEC, and the range for the language sub-score is 0-20. The higher the score, the worse the disability. Outcomes were measured at 6 weeks after treatment compared to baseline. A negative difference corresponds to a decrease in language disability, i.e an improvement in speech and language. A positive difference reflects an increase in language disability, i.e. a decrease in speech and language.

  3. The Clinical Global Impression - Improvement Scale (CGI-I)

    Time frame: Overall ASD symptoms at 6 weeks

    The CGI-I is scale that ranges from 1-7, reflecting the change in core autism behaviors after treatment. 1 is much improved, 4 is unchanged, and 7 is much worse.

  4. Repetitive Behavior Questionnaire

    Time frame: 6 weeks compared to baseline

    Total repetitive behavior was assessed using the Repetitive behavior questionnaire (RBQ), which has a scale from 0-87. Higher scores correspond to more severe repetitive behavior. Outcomes were analyzed as the difference in the score 6 weeks after treatment compared to baseline. A negative difference corresponds to improved behavior compared to baseline. A positive difference corresponds to worse behavior.

Sponsors and collaborators

Lead sponsor

University of California, San Diego

Other

Registry information

Official study title

The UCSD Suramin Autism Treatment-1 (SAT1) Trial

Acronym: SAT1

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Jul 24, 2015
Registry last updated
Jul 16, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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