Actively Recruiting
Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)
Led by Children's Hospital of Orange County · Updated on 2026-01-07
45
Participants Needed
3
Research Sites
155 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Orange County
Lead Sponsor
K
Kuzani Pharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 5 to 14 years
- Clinical diagnosis of Autism Spectrum Disorder by DSM-5 criteria
- ADOS-2 comparison score of 7 or higher on Modules 2-4 within the last 2 years
- Clinical Global Impression-Severity score of 4 or higher for socialization symptoms
- Non-verbal IQ above 70 on Leiter-3 test
- Vineland Adaptive Behavior Scale Third Edition socialization score below 75
- Agreement to use condoms with spermicidal during treatment and for 30 days after if sexually active or potentially sexually active
- Agreement to use sunscreen and skin covering during treatment and for 30 days after
- Ability to travel within 90 minutes to the study site
- English-speaking child and parent/guardian or caregiver
- Parent or legal guardian willing to provide informed consent
You will not qualify if you...
- Diagnosis of ASD with an underlying syndromic disorder such as Fragile X, Angelman, or Down's Syndrome
- Weight below the 5th percentile
- Inability to tolerate blood draws or urine collection
- Acute infections like cold, flu, strep, or COVID-19
- Severe co-existing conditions such as uncontrolled seizures, psychosis, or severe sensory impairments
- Organ dysfunction including liver, kidney, heart, or adrenal abnormalities
- Hospitalization within 2 months before screening
- Recent changes (within 2 months) in medication or psychosocial treatment
- Plans to start or change medication or psychosocial treatment during the study
- Use of medications that may interact with KZ101
- Participation in another clinical trial or investigational treatment within 30 days
- Taking more than 3 medications for behavioral or neuropsychiatric symptoms
- History of serious skin reactions or drug allergies including photosensitivity
- Inability or unwillingness to follow study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Southwest Autism Research and Resource Center
Phoenix, Arizona, United States, 85006
Not Yet Recruiting
2
Children's Hospital Orange County, Thompson Autism and Neurodevelopmental Center
Orange, California, United States, 92868
Actively Recruiting
3
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Actively Recruiting
Research Team
A
Adrienne Moore, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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