Double Blind Trial in Children With Autism Spectrum Disorder
Condition: Autism Spectrum Disorder
MeSH terms: Autism Spectrum Disorder
Study type: Interventional
Phase: Phase 2

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 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
    

Intervention name(s): Suramin Sodium
Intervention type(s): Drug
Intervention description(s): Suramin sodium is a sodium salt form of suramin, an antitrypanosomal compound.
Other intervention name(s): Placebo
Intervention MeSH terms: Suramin
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: 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)
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment