Suramin for the Treatment of Autism Trial: KZ101 in a Male Pediatric Population With Autism Spectrum Disorder (ASD)

Launched by CHILDREN'S HOSPITAL OF ORANGE COUNTY · Mar 5, 2025

Keywords

ClinConnect Summary

The Suramin for the Treatment of Autism Trial (KZ101) is a research study looking at a new treatment for children with Autism Spectrum Disorder (ASD). The researchers want to see if a medication called suramin can help improve symptoms of ASD in boys ages 5 to 14. Previous studies in animals showed that suramin might help with symptoms related to autism, and this trial will test its safety and effectiveness in children. The study will last for 30 weeks and will involve about 45 boys, who will receive suramin through an intravenous (IV) infusion.

To participate, boys must be between 5 and 14 years old and have a confirmed diagnosis of ASD. They should also meet specific test scores related to social skills and cognitive abilities. Parents or guardians will need to provide consent for their child to join the study. Participants will need to follow some guidelines, like using sunscreen during treatment and agreeing to certain safety measures. The trial is not yet recruiting participants, but it aims to gather important information about how well suramin works for children with ASD.

Gender

MALE

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