NCT06866275: STAT-2A

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

Recruiting now Phase 2 Last updated 5 January 2026

What this trial tests

Phase 2 trial testing KZ101 in Autism Spectrum Disorder (ASD) in 45 participants. Currently enrolling.

Timeline

9 April 2025

Primary endpoint
1 April 2028

1 April 2028

Quick facts

Lead sponsor	Children's Hospital of Orange County
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	quadruple
Primary purpose	treatment
Enrollment	45
Start date	9 April 2025
Primary completion	1 April 2028
Estimated completion	1 April 2028
Sites	3 locations across United States

Drugs / interventions tested

• KZ101 — full drug profile →
• Placebo

Conditions studied

• Autism Spectrum Disorder (ASD) — all drugs for Autism Spectrum Disorder (ASD) →

Sponsor

Children's Hospital of Orange County

Who can join

Adults 5 to 14, male only, with Autism Spectrum Disorder (ASD). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Primary Efficacy Endpoint - Vineland-3 Socialization Domain
  Time frame: Change from Screening to Week 8 (before washout) will be compared to change from Week 16 to Week 24 (after washout).
  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.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Novel therapeutics in autism spectrum disorder.
   Sara Daniella Kevelson, Elmaghraby R, Patel F, Brown H, et al · · 2026 · cited 1× · PMID 41748402 · DOI 10.1016/j.neurot.2026.e00857
2. Suramin Interactions Across Biological Systems: From Molecular Targets to Therapeutic Implications.
   Catalano A, Scaglione V, Sgobba MN, Ferrone L, et al · · 2026 · PMID 42072648 · DOI 10.3390/biom16040527