Suramin for Autism

(STAT-2A Trial)

Overseen By Adrienne Moore, PhD

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: Children's Hospital of Orange County

Must not be taking: Sulfonamides, Chlorpromazine, Anticoagulants

Disqualifiers: Syndromic ASD, Severe comorbidities, Organ dysfunction, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

45 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you must stop all current medications, but you cannot start or change medications within two months before the trial or during the study. Also, you cannot take more than three medications for behavioral symptoms related to ASD or certain medications that might interact with the study drug.

What data supports the effectiveness of the drug Suramin for treating autism?

Research shows that low-dose Suramin improved symptoms in mouse models of autism, and a small clinical trial suggested it might help children with autism by improving mitochondrial function and reducing brain inflammation. ^{1 2 3 4 5}

How does the drug Suramin differ from other treatments for autism?

Suramin is unique because it was originally used to treat parasitic infections and has shown potential in treating autism by targeting specific biological pathways, unlike traditional autism treatments that focus on behavioral therapy or medications for symptoms. Its novel mechanism involves binding to proteins and affecting signaling pathways, which is different from other autism treatments. ^{6 7 8 9 10}

What is the purpose of this trial?

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.

Research Team

Aram Kim, MD

Principal Investigator

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

Eligibility Criteria

This trial is for boys aged 5-14 with Autism Spectrum Disorder (ASD), who meet specific diagnostic criteria and have certain socialization challenges. Participants must live within a 90-minute car ride from the study site, have an IQ over 70, and agree to use contraception if sexually active. They also need to commit to sun protection during the treatment period.

Inclusion Criteria

I have been diagnosed with autism spectrum disorder.

My social symptoms of autism are moderately severe.

My non-verbal IQ score is above 70.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment Period 1

Participants receive either active treatment with KZ101 or placebo for 8 weeks

8 weeks

2 visits (in-person) for IV infusions, 4 weeks apart

Washout

Participants undergo an 8-week washout period with no treatment

8 weeks

Treatment Period 2

Participants cross over to receive the opposite treatment (active or placebo) for 8 weeks

8 weeks

2 visits (in-person) for IV infusions, 4 weeks apart

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

Treatment Details

Interventions

• KZ101

Trial Overview The trial is testing Suramin (KZ101) given through IV infusion against a placebo in young males with ASD. It's designed as a double-blind, crossover study lasting 30 weeks at about three sites with around 45 participants in total.

Participant Groups

Group I: Drug followed by Placebo Experimental Treatment 2 Interventions

Group II: Placebo followed by Drug Placebo Group 2 Interventions

Who Is Running the Clinical Trial?

Children's Hospital of Orange County

Lead Sponsor

Trials

38

Recruited

5,700+

Kuzani Pharmaceuticals, Inc.

Industry Sponsor

Trials

1

Recruited

50+

Findings from Research

Cariprazine demonstrated dose-dependent efficacy in reversing core behavioral deficits associated with autism spectrum disorder (ASD) in a rat model, showing effectiveness in social play and hyperactivity.

Compared to risperidone and aripiprazole, cariprazine was uniquely effective in the social play test, suggesting it may offer a distinct therapeutic benefit for treating social communication deficits in ASD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cariprazine alleviates core behavioral deficits in the prenatal valproic acid exposure model of autism spectrum disorder. Román, V., Adham, N., Foley, AG., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Randomized clinical trial of low dose suramin intravenous infusions for treatment of autism spectrum disorder. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

The Multi-Targeting Ligand ST-2223 with Histamine H3 Receptor and Dopamine D2/D3 Receptor Antagonist Properties Mitigates Autism-Like Repetitive Behaviors and Brain Oxidative Stress in Mice. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy. [2022]

5. Germany pubmed.ncbi.nlm.nih.gov

Cariprazine alleviates core behavioral deficits in the prenatal valproic acid exposure model of autism spectrum disorder. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Renal clearance, tissue distribution, and CA-125 responses in a phase I trial of suramin. [2016]

7. Greece pubmed.ncbi.nlm.nih.gov

Stimulatory effect of suramin on the proliferation of human glioma cells. [2013]

8. United States pubmed.ncbi.nlm.nih.gov

The approved pediatric drug suramin identified as a clinical candidate for the treatment of EV71 infection-suramin inhibits EV71 infection in vitro and in vivo. [2018]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Suramin Targets the Conserved Ligand-Binding Pocket of Human Raf1 Kinase Inhibitory Protein. [2021]

10. Ireland pubmed.ncbi.nlm.nih.gov

Inhibition of insulin like growth factor II autocrine growth of Wilms' tumor by suramin in vitro and in vivo. [2019]