Double Blind Trial in Children With Autism Spectrum Disorder

A Three-Arm, Prospective, Randomized, Double Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of 2 Doses of Suramin vs. Placebo in Male Children With ASD Receiving Standard Treatment

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

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: Suramin Sodium

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 participants who received treatment interventions for ASD; each treatment arm was assigned approximately 17 participants. The number of participants was increased to 52 participants (48 originally planned) due to early withdrawals related to the COVID-19 global pandemic lockdowns and other matters. Participants were stratified by age, ADOS-2, and Non-verbal Intelligence Quotient (NVIQ). Participants who met all the inclusion criteria and none of the exclusion criteria were randomized through an electronic system to either arm A (10 mg/kg suramin) or Arm B (20 mg/kg suramin) or Arm C (Placebo) in a targeted 1:1:1 ratio, as per the randomization schedule and stratified by age, ADOS-2, and NVIQ. All participants received a 50 mg test dose at their first administration (0.5 mL of freshly reconstituted 10% solution in 5 mL saline) to check for allergic reactions. The test dose was given by slow intravenous (IV) infusion for 30 minutes then flushed with 10 mL saline. Vitals were checked for 30 min and if there were no changes or evidence of allergic reaction, the rest of the study drug dose was administered (10 mg/kg or 20 mg/kg minus test dose [minus 50 mg] in a 50 mL saline up to a maximum of 1 g given over 30 minutes). Arm A - 50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.

Arm B - 50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4 and 5. The maximum dose administered was 1 g.

Arm C - Test dose of saline placebo, then saline placebo IV infusion of 50 mL administered over 30 minutes was given at Visits 2, 4 and 5

• Study Type: Interventional
• Enrollment (Actual): 52
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • Tarrytown, New York, United States, 10591
      • PaxMedica

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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 Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arm A; 50 mg test dose of suramin (upon first administration), then suramin 10 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5.	Drug: Suramin Sodium; Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.; Other Names: Placebo
Experimental: Arm B; 50 mg test dose of suramin (upon first administration), then suramin 20 mg/kg (minus 50 mg suramin test dose) in 50 mL of saline administered by IV infusion over 30 minutes was given at Visits 2, 4, and 5.	Drug: Suramin Sodium; Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.; Other Names: Placebo
Placebo Comparator: Arm C; Test dose of saline placebo, then saline placebo infusion of 50 mL administered over 30 minutes was given at Visits 2, 4, and 5.	Drug: Suramin Sodium; Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.; Other Names: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The primary objective was to evaluate the safety and efficacy of 2 dose levels of low dose suramin against placebo in children with ASD receiving standard treatment.	ABC-Core and CGI-I scales used to evaluate efficacy	98 Days

Collaborators and Investigators

• Sponsor: Paxmedica
• Investigators: Study Director: jennifer L bonfrisco, PaxMedica

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2019
• Primary Completion (Actual): January 15, 2021
• Study Completion (Actual): March 15, 2021

Study Registration Dates

• First Submitted: September 18, 2023
• First Submitted That Met QC Criteria: September 22, 2023
• First Posted (Actual): September 28, 2023

Study Record Updates

• Last Update Posted (Actual): September 28, 2023
• Last Update Submitted That Met QC Criteria: September 22, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autism Spectrum Disorder; Anti-Infective Agents; Antineoplastic Agents; Antiprotozoal Agents; Antiparasitic Agents; Antinematodal Agents; Anthelmintics; Trypanocidal Agents; Suramin
• Other Study ID Numbers: PUR-ONQ-ASD-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Abstract and CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No