Double Blind Trial in Children With Autism Spectrum Disorder

NCT06058962 | Completed Phase 2 DMC

• 1 other identifier: PUR-ONQ-ASD-001
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 1

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

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• 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.

  98 Days

Study Arms (3)

Suramin 10 mg/kg

EXPERIMENTAL

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 20 mg/kg

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

Suramin Sodium DRUG

Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.

Also known as: Placebo

Placebo; Suramin 10 mg/kg; Suramin 20 mg/kg

Eligibility Criteria

• Age: 4 Years - 17 Years
• Gender Eligibility Details: Males only
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Paxmedica: lead

Study Sites (1)

PaxMedica

Tarrytown, New York, 10591, United States

Location

Related Publications (1)

• Hough D, Mao AR, Aman M, Lozano R, Smith-Hicks C, Martinez-Cerdeno V, Derby M, Rome Z, Malan N, Findling RL. Randomized clinical trial of low dose suramin intravenous infusions for treatment of autism spectrum disorder. Ann Gen Psychiatry. 2023 Nov 6;22(1):45. doi: 10.1186/s12991-023-00477-8.

  PMID: 37932739 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Suramin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Naphthalenesulfonates; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Arylsulfonates; Arylsulfonic Acids; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Polycyclic Compounds

Study Officials

• jennifer L bonfrisco

  Paxmedica

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The study was double-blind. Suramin and placebo are both clear solutions when prepared. Suramin was prepared by an unblinded pharmacist by reconstituting 1 g vial of suramin in 10 mL of sterile water for infusion to prepare a 10% (100 mg/mL) solution.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The participants were randomized to one of three double-blind treatment groups, i.e., 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 stratification plan. The stratification plan was to match patients by age (\< 7 vs ≥ 7), ADOS-2 comparison scores (≤ 8.5 vs \> 8.5) and NVIQ (≤ 80 vs \> 80)

Study Record Dates

• First Submitted: September 18, 2023
• First Posted: September 28, 2023
• Study Start: June 1, 2019
• Primary Completion: January 15, 2021
• Study Completion: March 15, 2021
• Last Updated: November 13, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)