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
Crossover
KZ101
DrugFor 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
DrugDosing in the placebo group will consist of a volume of normal saline equivalent to that given during the active treatment period for each participant.
Primary Efficacy Endpoint - Vineland-3 Socialization Domain
The primary efficacy endpoint will be the change in standard score on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition (Vineland-3), which includes subdomains for coping skills, play skills, and interpersonal relation skills.
Secondary Efficacy Endpoint - Vineland-3, Additional Scores
Vineland-3 Composite Score, Communication Domain Score, and Daily Living Skills Domain Score will also be assessed.
Secondary Efficacy Endpoint - Global Impresssion, Severity/Change
Clinician Global Impression of Severity and Change, and Caregiver/Parent Global Impression of Severity and Change, will be collected and assessed weighted for socialization.
Secondary Efficacy Endpoint - Social Responsiveness Scale-2
The Social Responsiveness Scale-2 (SRS-2) is a well-established tool to evaluate socialization that was also used in a prior pilot study.
Secondary Efficacy Endpoint - Aberrant Behavior Checklist, Second Edition (ABC-2)
The ABC-2 provides measures of repetitive behaviors and includes domains of irritability, social withdrawal, stereotypy, hyperactivity, and inappropriate speech.
Pharmacokinetic (PK) Endpoints: Cmax
Blood samples for measurement of KZ101 concentration in plasma will be collected and used to identify Peak Plasma Concentration (Cmax).
Pharmacokinetic (PK) Endpoints: Area Under the Curve (AUC)
Blood samples for measurement of KZ101 concentration in plasma will be collected, and area under the plasma concentration versus time curve (AUC) will be determined.
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Children's Hospital of Orange County
Lead sponsor
Kuzani Pharmaceuticals, Inc.
Collaborator
