Status:

COMPLETED

Double Blind Trial in Children With Autism Spectrum Disorder

Lead Sponsor:

Paxmedica

Conditions:

Autism Spectrum Disorder

Eligibility:

MALE

4-17 years

Phase:

PHASE2

Brief Summary

This study investigated the safe and efficacious use of suramin as a novel treatment for ASD by testing two suramin doses vs placebo

Detailed Description

PUR-ONQ-ASD-001 was a multicenter, 3-arm, prospective, randomized, double blind, placebo-controlled multiple dose trial involving 2 dose levels of suramin vs. placebo. The study randomized 52 male par...

Eligibility Criteria

Inclusion

  • Male children aged 4 - 17 years
  • Participants with or without treatment interventions for ASD
  • Participants must have been diagnosed with ASD by Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-V)
  • Autism diagnostic observation schedule, version 2 (ADOS-2) Comparison scores in the
  • moderate and high level as evaluated on the ADOS-2
  • Stable treatment intervention for ≥ 2 months
  • Participants agreed to not change their treatment interventions throughout the study
  • duration
  • Participants on Ritalin and Risperdal or similar medication agreed to not change their
  • dose during the study

Exclusion

  • Hospitalization within the previous 2 months
  • An acute medical problem, Rett syndrome, microcephaly, tuberous sclerosis, neurofibromatosis, epilepsy, or clinically significant liver, kidney, or adrenal disease or persons suffering from any serious acute condition where, in the investigator's opinion, the participant's well-being may have been compromised
  • 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 IV catheter for 3-4 hours
  • Plasma creatinine above normal for age and weight according to the laboratory reference ranges.
  • Liver function alanine aminotransferase (ALT) or aspartate aminotransferase (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
  • Children with known syndromic forms of ASD caused by DNA mutation or chromosomal copy number variation

Key Trial Info

Start Date :

June 1 2019

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

March 15 2021

Estimated Enrollment :

52 Patients enrolled

Trial Details

Trial ID

NCT06058962

Start Date

June 1 2019

End Date

March 15 2021

Last Update

November 13 2023

Active Locations (1)

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1

PaxMedica

Tarrytown, New York, United States, 10591