Using Suramin to Treat Autism in Boys Aged 5-14

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

This study is testing if a drug called suramin can help boys aged 5 to 14 with autism feel better.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (estimated)
• Ages: 5 Years to 14 Years
• Sex: Male
• Sponsor: Children's Hospital of Orange County (Academic / other)
• Locations: 3 sites (Phoenix, Arizona and 2 other locations)
• Trial ID: NCT06866275 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and effectiveness of suramin sodium (KZ101) in treating Autism Spectrum Disorder (ASD) in male children aged 5 to 14. The study is designed as a randomized, double-blind, crossover trial, where participants will receive either the active treatment or a placebo in two separate 8-week periods, with a washout phase in between. The trial aims to assess the preliminary proof of concept, safety, and pharmacokinetics of suramin through intravenous infusions. Approximately 45 subjects will be enrolled across three sites, with the goal of completing the study with around 36 participants.

Who should consider this trial

Good fit: Ideal candidates for this study are male children aged 5-14 years who have a clinical diagnosis of Autism Spectrum Disorder.

Not a fit: Patients who do not meet the age or gender criteria, or those with a non-verbal IQ below 70, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for improving symptoms of autism in pediatric patients.

How similar studies have performed: Preliminary studies have shown promise for suramin in treating autism, indicating potential for success in this approach.

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

Where this trial is running

Phoenix, Arizona and 2 other locations

• Southwest Autism Research and Resource Center — Phoenix, Arizona, United States (Not_yet_recruiting)
• Children's Hospital Orange County, Thompson Autism and Neurodevelopmental Center — Orange, California, United States (Recruiting)
• Kennedy Krieger Institute — Baltimore, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Aram Kim, MD — Thompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County
• Study coordinator: Adrienne Moore, PhD

Conditions Autism Spectrum Disorder; Suramin; suramin sodium