Double Blind Trial in Children With Autism Spectrum Disorder

Autism Spectrum Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06058962
• Other study ID #: PUR-ONQ-ASD-001
• Status: Completed
• Phase: Phase 2
• Start date: June 1, 2019
• Est. completion date: March 15, 2021

Study information

• Verified date: November 2023
• Source: Paxmedica
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: March 15, 2021
• Est. primary completion date: January 15, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism Spectrum Disorder

Intervention

Drug:
• Suramin Sodium
Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.

Locations

Country	Name	City	State
United States	PaxMedica	Tarrytown	New York

Sponsors (1)

Lead Sponsor	Collaborator
Paxmedica

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The primary objective was to evaluate the safety of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.	Adverse Events and Laboratory Results	98 Days
Secondary	The primary objective was to evaluate the efficacy of 2 dose levels of low-dose suramin against placebo in children with ASD receiving standard treatment.	Aberrant Behavior Checklist (ABC) and (Clinical Global Impression) CGI scales used to evaluate efficacy.; ABC scoring is a 0 as improvement with a 3 as a problem being severe.; For each item, decide whether the behavior is a problem for your child/adolescent and select the appropriate number/response: "0 = not at all a problem; = the behavior is a problem but slight in degree; = the problem is moderately serious; = the problem is severe in degree	98 Days