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

NCT ID: NCT06866275

Last Updated: 2025-11-06

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-04-09
• Study Completion Date: 2028-04-30

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.

Detailed Description

After up to a 4-week screening period, participants will undergo 8 weeks of active or placebo treatment (Period 1), followed by an 8-week washout period, and then cross over to 8 weeks of placebo or active treatment (Period 2). Patients will be followed for 2 weeks after completion of Period 2. Two dosing groups are designated as Group A, who are randomly assigned to active treatment with KZ101 in Period 1 and saline in Period 2, and Group B, who are randomly assigned to saline infusion in Period 1 and active treatment with KZ101 in Period 2. Dosing in both periods will consist of 2 IV infusions of either saline (placebo) or KZ101 (active treatment), given 4 weeks apart.

Conditions

Autism Spectrum Disorder (ASD)

Keywords

Suramin; suramin sodium

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Drug followed by Placebo

Group Type: EXPERIMENTAL

KZ101

Intervention Type: 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).

Placebo

Intervention Type: 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.

Placebo followed by Drug

Group Type: PLACEBO_COMPARATOR

KZ101

Intervention Type: 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).

Placebo

Intervention Type: 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.

Interventions

KZ101

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).

Intervention Type: DRUG

Placebo

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.

Intervention Type: DRUG

Other Intervention Names

Suramin; Suramin sodium; Saline

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 14 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Kuzani Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: collaborator

Children's Hospital of Orange County

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aram Kim, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Orange, California, United States

Site Status: RECRUITING

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status: NOT_YET_RECRUITING

Countries

United States

Central Contacts

Adrienne Moore, PhD

Role: CONTACT

Phone: 714-288-7456

Email: adrienne.moore@choc.org

Facility Contacts

Adrienne R Moore, PhD

Role: primary

Katie Kay

Role: primary

Karen Chen

Role: backup

References

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.

Reference Type: BACKGROUND

PMID: 24937094 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 25705365 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 28695149 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 23516405 (View on PubMed)

Related Links

https://choc.org/research/autism-research/

Thompson Autism and Neurodevelopmental Center, Clinical Trials

Other Identifiers

CHOC IRB# 2308114

Identifier Type: -

Identifier Source: org_study_id