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
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.
‣ \- Subject must meet all of the following criteria to be enrolled in this 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
• Leiter-3 non-verbal IQ \> 70
• Standard score \< 75 on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition
• Subjects who are sexually active or potentially sexually active agree to use condoms with a spermicidal as a barrier method of contraception during the treatment period and for at least 30 days after the last dose of study medication
• Subjects agree to wear sunscreen and to wear skin covering to the maximal degree tolerated by the child for the duration of the treatment period and for at least 30 days after the last dose of study medication
• Subjects must have a ≤ 90 minutes car ride from the study site
⁃ English-speaking child and parent/guardian or caregiver
⁃ Parent or their legal guardians must be willing to sign informed consent