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.
Male, aged 5-14 years
Clinical diagnosis of ASD by DSM-5 criteria
ADOS-2 ≥ 7 on the comparison score for Modules 2-4 (completed within the last 2 years).
CGI-S ≥ 4 for socialization specific symptoms of ASD
Subjects who meet any of the following criteria will be excluded from the study.
ASD diagnosis with underlying syndromic diagnosis (e.g., Fragile X, Angelman, Down's Syndrome, etc.)
≤ 5th percentile for weight
Unable to tolerate venipuncture or urine collection
Children's Hospital of Orange County
Lead sponsor
Kuzani Pharmaceuticals, Inc.
Collaborator