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

NCT06866275 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CHOC IRB# 2308114
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 3

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.

Conditions

Autism Spectrum Disorder (ASD)

Keywords

Suramin; suramin sodium

Outcome Measures

Primary Outcomes (1)

• Primary Efficacy Endpoint - Vineland-3 Socialization Domain

  The primary efficacy endpoint will be the change in standard score on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition (Vineland-3), which includes subdomains for coping skills, play skills, and interpersonal relation skills.

  Change from Screening to Week 8 (before washout) will be compared to change from Week 16 to Week 24 (after washout).

Secondary Outcomes (8)

• Secondary Efficacy Endpoint - Vineland-3, Additional Scores

  Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).

• Secondary Efficacy Endpoint - Global Impresssion, Severity/Change

  Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).

• Secondary Efficacy Endpoint - Social Responsiveness Scale-2

  Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).

• Secondary Efficacy Endpoint - Aberrant Behavior Checklist, Second Edition (ABC-2)

  Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).

• Secondary Efficacy Endpoint - Childhood Sleep Habits Questionnaire (CSHQ)

  Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).

Other Outcomes (2)

• Pharmacokinetic (PK) Endpoints: Cmax

  PK samples will be taken at week 0, 2, 4, 6, 8 (period 1); and 16, 18, 20, 22, 24, 26 (period 2 and follow up).

• Pharmacokinetic (PK) Endpoints: Area Under the Curve (AUC)

  PK samples will be taken at week 0, 2, 4, 6, 8 (period 1); and 16, 18, 20, 22, 24, 26 (period 2 and follow up).

Study Arms (2)

Drug followed by Placebo

EXPERIMENTAL

Drug: KZ101; Drug: Placebo

Placebo followed by Drug

PLACEBO COMPARATOR

Drug: KZ101; Drug: Placebo

Interventions

KZ101 DRUG

For active treatment with KZ101, a loading dose of 454 mg/m2 (salt-free) will be followed by a treatment dose of 363 mg/m2 (salt-free).

Also known as: Suramin, Suramin sodium

Drug followed by Placebo; Placebo followed by Drug

Placebo DRUG

Dosing in the placebo group will consist of a volume of normal saline equivalent to that given during the active treatment period for each participant.

Also known as: Saline

Drug followed by Placebo; Placebo followed by Drug

Eligibility Criteria

• Age: 5 Years - 14 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• \- Subject must meet all of the following criteria to be enrolled in this study.
• Male, aged 5-14 years
• Clinical diagnosis of ASD by DSM-5 criteria
• ADOS-2 ≥ 7 on the comparison score for Modules 2-4 (completed within the last 2 years).
• CGI-S ≥ 4 for socialization specific symptoms of ASD
• Leiter-3 non-verbal IQ \> 70
• Standard score \< 75 on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition
• 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
• 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
• Subjects must have a ≤ 90 minutes car ride from the study site
• English-speaking child and parent/guardian or caregiver
• Parent or their legal guardians must be willing to sign informed consent

You may not qualify if:

• Subjects who meet any of the following criteria will be excluded from the study.
• ASD diagnosis with underlying syndromic diagnosis (e.g., Fragile X, Angelman, Down's Syndrome, etc.)
• ≤ 5th percentile for weight
• Unable to tolerate venipuncture or urine collection
• Acute infection (e.g., upper respiratory tract infection, common cold, flu, strep, COVID-19)
• 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.
• 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
• Hospitalization within the previous 2 months from screening
• Initiation or change in pharmacotherapy within previous 2 months from screening
• Initiation or change in psychosocial interventions (formal behavioral, cognitive, or cognitive-behavior therapy) within previous 2 months from screening
• Plan to initiate or change pharmacotherapy or psychosocial interventions during the study
• 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
• Currently enrolled in another clinical study or has received any investigational treatment within 30 days of screening
• 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.
• History of serious dermatological reactions

Sponsors & Collaborators

• Children's Hospital of Orange County: lead
• Kuzani Pharmaceuticals, Inc.: collaborator

Study Sites (3)

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

NOT YET RECRUITING

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

Orange, California, 92868, United States

RECRUITING

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

RECRUITING

Related Publications (4)

• Naviaux RK, Zolkipli Z, Wang L, Nakayama T, Naviaux JC, Le TP, Schuchbauer MA, Rogac M, Tang Q, Dugan LL, Powell SB. Antipurinergic therapy corrects the autism-like features in the poly(IC) mouse model. PLoS One. 2013;8(3):e57380. doi: 10.1371/journal.pone.0057380. Epub 2013 Mar 13.

  PMID: 23516405 BACKGROUND

• Naviaux JC, Schuchbauer MA, Li K, Wang L, Risbrough VB, Powell SB, Naviaux RK. Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy. Transl Psychiatry. 2014 Jun 17;4(6):e400. doi: 10.1038/tp.2014.33.

  PMID: 24937094 BACKGROUND

• Naviaux JC, Wang L, Li K, Bright AT, Alaynick WA, Williams KR, Powell SB, Naviaux RK. Antipurinergic therapy corrects the autism-like features in the Fragile X (Fmr1 knockout) mouse model. Mol Autism. 2015 Jan 13;6:1. doi: 10.1186/2040-2392-6-1. eCollection 2015.

  PMID: 25705365 BACKGROUND

• Naviaux RK, Curtis B, Li K, Naviaux JC, Bright AT, Reiner GE, Westerfield M, Goh S, Alaynick WA, Wang L, Capparelli EV, Adams C, Sun J, Jain S, He F, Arellano DA, Mash LE, Chukoskie L, Lincoln A, Townsend J. Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial. Ann Clin Transl Neurol. 2017 May 26;4(7):491-505. doi: 10.1002/acn3.424. eCollection 2017 Jul.

  PMID: 28695149 BACKGROUND

Related Links

• Thompson Autism and Neurodevelopmental Center, Clinical Trials

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Suramin; Sodium Chloride

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Naphthalenesulfonates; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Arylsulfonates; Arylsulfonic Acids; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Polycyclic Compounds; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Aram Kim, MD

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Adrienne Moore, PhD

CONTACT

714-288-7456; adrienne.moore@choc.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 24, 2025
• First Posted: March 10, 2025
• Study Start: April 9, 2025
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2028
• Last Updated: January 7, 2026

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)