Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)
Suramin for the Treatment of Autism Trial (STAT): A Randomized, Double Blind, Crossover Trial of KZ101 in a Male Pediatric Population With Autism Spectrum Disorder
About This Trial
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.
Who May Be Eligible (Plain English)
Original Eligibility Criteria
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Treatments Being Tested
KZ101
For active treatment with KZ101, a loading dose of 454 mg/m2 (salt-free) will be followed by a treatment dose of 363 mg/m2 (salt-free).
Placebo
Dosing in the placebo group will consist of a volume of normal saline equivalent to that given during the active treatment period for each participant.