Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)

Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism. A preliminary clinical trial (SAT-1) examined the safety and activity of a single low-dose of suramin in children with ASD and concluded suramin showed promise as a novel approach to treatment of ASD. The current study, STAT-2A, will be a randomized, double-blind, crossover, 30-week study to evaluate the preliminary proof of concept, safety, and PK of suramin sodium (KZ101) with repeat dosing by IV infusion in males 5-14 years of age who have been diagnosed with ASD. The study will be conducted at approximately 3 sites contributing approximately 15 subjects per site. Total enrollment of approximately 45 subjects is planned to achieve approximately 36 participants completing the study.

NCT Number	NCT06866275
Lead Sponsor	Children's Hospital of Orange County Collaborators: Kuzani Pharmaceuticals, Inc.
Conditions	Autism Spectrum Disorder (ASD)
Enrollment	45 participants
Start Date	2025-04-09
Primary Completion	2028-04 (estimated)
Study Completion	2028-04 (estimated)
Updated on ClinicalTrials.gov	2026-07-30