RECRUITING

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

Conditions

Autism Spectrum Disorder (ASD)Suramin suramin sodium

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2025-04-09

Study Completion:2028-04

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06866275

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:5 Years to 14 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
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	* 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

Inclusion Criteria

Exclusion Criteria

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

* 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

Contacts and Locations

Study Contact

Adrienne Moore, PhD

CONTACT

714-288-7456

adrienne.moore@choc.org

Principal Investigator

Aram Kim, MD

PRINCIPAL_INVESTIGATOR

Thompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County

Study Locations (Sites)

Children's Hospital Orange County, Thompson Autism and Neurodevelopmental Center

Orange, California, 92868

United States

Collaborators and Investigators

Sponsor: Children's Hospital of Orange County

Aram Kim, MD, PRINCIPAL_INVESTIGATOR, Thompson Autism and Neurodevelopmental Center, Children's Hospital of Orange County

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04-09

Study Completion Date2028-04

Study Record Updates

Study Start Date2025-04-09

Study Completion Date2028-04

Terms related to this study

Keywords Provided by Researchers

Suramin
suramin sodium

Additional Relevant MeSH Terms

Autism Spectrum Disorder (ASD)