NCT06866275 Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)
| NCT ID | NCT06866275 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Children's Hospital of Orange County |
| Condition | Autism Spectrum Disorder (ASD) |
| Study Type | INTERVENTIONAL |
| Enrollment | 45 participants |
| Start Date | 2025-04-09 |
| Primary Completion | 2028-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 45 participants in total. It began in 2025-04-09 with a primary completion date of 2028-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: \- Subject must meet all of the following criteria to be enrolled in this study. 1. Male, aged 5-14 years 2. Clinical diagnosis of ASD by DSM-5 criteria 3. ADOS-2 ≥ 7 on the comparison score for Modules 2-4 (completed within the last 2 years). 4. CGI-S ≥ 4 for socialization specific symptoms of ASD 5. Leiter-3 non-verbal IQ \> 70 6. Standard score \< 75 on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition 7. 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 8. 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 9. Subjects must have a ≤ 90 minutes car ride from the study site 10. English-speaking child and parent/guardian or caregiver 11. Parent or their legal guardians must be willing to sign informed consent Exclusion Criteria: * Subjects who meet any of the following criteria will be excluded from the study. 1. ASD diagnosis with underlying syndromic diagnosis (e.g., Fragile X, Angelman, Down's Syndrome, etc.) 2. ≤ 5th percentile for weight 3. Unable to tolerate venipuncture or urine collection 4. Acute infection (e.g., upper respiratory tract infection, common cold, flu, strep, COVID-19) 5. Severe co-morbid conditions (e.g., psychosis, seizures/epilepsy uncontrolled by medication, presence of severe visual or hearing impairment) that may interact with study procedures. Controlled epilepsy is allowed providing there has not been a breakthrough seizure in the past year. 6. Any organ system dysfunction, especially liver (e.g., ALT or AST ≥ 1.5x the upper limit of normal), kidney (estimated glomerular filtration rate or eGFR \< 90 mL/min/1.73 m2; hematuria confirmed by urine microscopy \[ \> 5 red blood cells/high power field\]; proteinuria \[\> 1+ that does not resolve on repeat testing or urine protein to creatinine ratio \> 0.3\]; and/or presence of any granular, mixed cellular, red blood cell, white blood cell, or muddy brown casts on urine microscopy), or clinically relevant heart or adrenal abnormalities 7. Hospitalization within the previous 2 months from screening 8. Initiation or change in pharmacotherapy within previous 2 months from screening 9. Initiation or change in psychosocial interventions (formal behavioral, cognitive, or cognitive-behavior therapy) within previous 2 months from screening 10. Plan to initiate or change pharmacotherapy or psychosocial interventions during the study 11. Taking prescription medication that may interact adversely with KZ101 or expose the subject to increased risk of harm such as medications with plasma bound substances including sulfonamides, chlorpromazine, and anti-coagulants 12. Currently enrolled in another clinical study or has received any investigational treatment within 30 days of screening 13. Taking \> 3 medications addressing behavioral symptoms related to ASD (ie typical/atypical antipsychotics and alpha-adrenergic agonists) or comorbid medical conditions such as ADHD, anxiety, or depression. Anti-seizure medications and other medications not related to neurobehavioral symptoms do not count towards the total number of medications allowed. 14. History of serious dermatological reactions 15. History of allergy, intolerance, or photosensitivity to any drug 16. Unable or unwilling to adhere to study requirements
Contact & Investigator
Aram Kim, MD
PRINCIPAL INVESTIGATOR
Thompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County
Frequently Asked Questions
Who can join the NCT06866275 clinical trial?
This trial is open to male participants only, aged 5 Years or older, up to 14 Years, studying Autism Spectrum Disorder (ASD). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06866275 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06866275 currently recruiting?
Yes, NCT06866275 is actively recruiting participants. Contact the research team at adrienne.moore@choc.org for enrollment information.
Where is the NCT06866275 trial being conducted?
This trial is being conducted at Phoenix, United States, Orange, United States, Baltimore, United States.
Who is sponsoring the NCT06866275 clinical trial?
NCT06866275 is sponsored by Children's Hospital of Orange County. The principal investigator is Aram Kim, MD at Thompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County. The trial plans to enroll 45 participants.