Suramin for Autism

(STAT-2A Trial)

Recruiting at 1 trial location

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment called KZ101 (also known as Suramin) for boys aged 5-14 diagnosed with autism spectrum disorder (ASD). The goal is to determine if KZ101 can alleviate ASD symptoms when administered through an IV over several weeks. Participants will receive both the actual treatment and a placebo (inactive substance) at different times to compare effects. Boys diagnosed with ASD who can manage car rides up to 90 minutes to the study site might be suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that suramin, the treatment under study, holds promise in earlier trials for autism spectrum disorder (ASD). In one study, researchers tested a single low dose of suramin in children with ASD and found that the treatment was generally well-tolerated. Some children experienced mild side effects, such as a temporary rash or changes in blood tests, which were manageable.

Another study administered suramin through monthly infusions and suggested that this method was safe and might help with core ASD symptoms. As with any medical treatment, some side effects occurred, including mild skin reactions and changes in certain blood tests, but serious side effects were uncommon.

Overall, earlier studies suggest that suramin is fairly safe for children with ASD, although monitoring side effects remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Unlike the standard of care for autism, which often includes behavioral therapies and medications like antipsychotics and stimulants to manage symptoms, KZ101 is derived from Suramin, a drug with a unique mechanism of action. Researchers are excited about KZ101 because it targets purinergic signaling, a pathway involved in cellular communication that may play a role in autism. This innovative approach offers the potential to address the underlying biological factors of autism rather than just alleviating symptoms, which could lead to more effective and comprehensive treatment options.

What evidence suggests that suramin might be an effective treatment for autism?

Research has shown that suramin, which participants in this trial may receive, may help treat autism spectrum disorder (ASD). In earlier studies, children who received a single dose of suramin showed improvements in key autism symptoms. Suramin appears to help cells produce energy normally and reduce brain swelling. In animal studies, suramin improved symptoms and brain issues related to autism. Overall, these findings suggest that suramin could be an effective treatment for ASD. ^{1 2 4 5 6}

Who Is on the Research Team?

Aram Kim, MD

Principal Investigator

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

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5497533/

Low‐dose suramin in autism spectrum disorder Low‐dose suramin was effective in the maternal immune activation and Fragile X mouse models of ASD. The Suramin Autism Treatment‐1 (SAT‐1) trial was a double‐ ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02508259

University of California, San Diego (UCSD) Suramin ... Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism ( ...

3. brainfoundation.org brainfoundation.org/suramin-for-autism-update-2023/

Suramin for Autism Update: 2023 and Beyond The results demonstrated that a single intravenous dose of suramin improved core symptoms of autism in the five children who received the drug, ...

4. annals-general-psychiatry.biomedcentral.com annals-general-psychiatry.biomedcentral.com/articles/10.1186/s12991-023-00477-8

Randomized clinical trial of low dose suramin intravenous ... Monthly suramin intravenous infusions may be a safe and potentially efficacious treatment for the core symptoms of ASD. Availability of data and ...

5. pure.johnshopkins.edu pure.johnshopkins.edu/en/publications/randomized-clinical-trial-of-low-dose-suramin-intravenous-infusio/

Randomized clinical trial of low dose suramin intravenous ... The purinergic antagonist suramin may improve core symptoms through restoration of normal mitochondrial function and reduction of neuro-inflammation via its ...

6. trialx.com trialx.com/clinical-trials/listings/316363/suramin-for-the-treatment-of-autism-trial-kz101-in-a-male-pediatric-population-with-autism-spectrum-disorder-asd/

Suramin for the Treatment of Autism Trial: KZ101 in a Male ... 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 ...