AB-2004 for Autism Spectrum Disorder

No longer recruiting at 42 trial locations

Overseen By Clinical Trial Team

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Vertero Therapeutics

Must not be taking: Antibiotics, Antipsychotics

Disqualifiers: Schizophrenia, Bipolar, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AB-2004, a potential drug, to determine its effectiveness in reducing irritability in individuals with autism spectrum disorder (ASD). Researchers aim to assess the safety of AB-2004 and compare its efficacy to a placebo. The trial seeks participants diagnosed with ASD who frequently experience irritability. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

You may need to stop taking certain medications, like antibiotics or antipsychotics, before joining the trial. The trial does not allow the use of oral controlled or extended-release medications.

Is there any evidence suggesting that AB-2004 is likely to be safe for humans?

A previous study found AB-2004 to be safe and well-tolerated in teenagers with autism spectrum disorder (ASD). Participants did not experience major side effects, indicating that AB-2004 is generally easy to handle. This finding is encouraging for those considering joining a clinical trial, as it suggests the treatment is likely safe. ^{1 2 3 4 5}

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

Researchers are excited about AB-2004 for Autism Spectrum Disorder because it represents a novel approach to treatment. Unlike traditional options like behavioral therapies and medications that target symptoms, AB-2004 works by targeting the gut-brain axis. It is designed to bind and remove certain gut-derived metabolites that may exacerbate autism symptoms. This unique mechanism of action offers a fresh perspective and potential benefits that current therapies do not address, potentially leading to improved outcomes for individuals on the autism spectrum.

What evidence suggests that AB-2004 might be an effective treatment for irritability in autism spectrum disorder?

Research has shown that AB-2004, which participants in this trial may receive, might help reduce irritability in people with autism. An earlier study found AB-2004 to be safe and well-tolerated by teenagers with autism. The treatment also lowered certain blood markers linked to autism symptoms. Animal studies demonstrated that AB-2004 reduced harmful substances and improved gut health, potentially aiding symptoms. Early evidence suggests it targets specific gut-related issues connected to irritability in autism. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

Inclusion Criteria

Your condition is considered to be severe by the doctor conducting the screening.

Clinically diagnosed, documented ASD (Diagnostic and Statistical Manual of Mental Disorders [DSM-5] criteria)

Aberrant Behavior Checklist - Irritability (ABC-I) score ≥18 at the screening visit

Exclusion Criteria

Use of an oral, injected, or inhaled antibiotic within 30 days prior to screening. Prophylactic oral antibiotic use of no more than 1 dose will be permitted

Current use of an oral controlled or extended-release medication

Have a comorbid major psychiatric condition (eg, schizophrenia or bipolar disorder) at screening that in the opinion of the Investigator may interfere with the subject's ability to complete study procedures/comply with study requirements

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AB-2004 or placebo to assess efficacy, safety, and tolerability

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AB-2004
• Placebo

How Is the Trial Designed?

Group I: AB-2004 Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Vertero Therapeutics

Lead Sponsor

Axial Therapeutics, Inc.

Lead Sponsor

Trials

1

Recruited

160+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04895215

AB-2004 in Treatment of Irritability Associated With Autism ... The purpose of this study is to establish the potential benefits, safety, and tolerability of AB-2004 in participants with irritability associated with autism ...

2. axialtx.com axialtx.com/nature-medicine-publishes-full-results-from-axial-therapeutics-phase-1b-2a-clinical-trial-in-autism-spectrum-disorder-asd/

Nature Medicine Publishes Full Results from ... Open-label study showed Axial's Lead Candidate, AB-2004, is safe and well-tolerated in an adolescent ASD cohort. – AB-2004 lowered blood and ...

3. nature.com nature.com/articles/s41591-022-01683-9

Safety and target engagement of an oral small-molecule ... Results from this simplified mouse model indicate that AB-2004 was effective in reducing systemic levels of 4EPS and preventing 4EPS-induced ...

4. clinicaltrial.be clinicaltrial.be/en/details/43744?active_not_recruiting=1&completed=0&enrolling_by_invitation=1&only_active=0&only_eligible=0&only_recruiting=0&per_page=20

AB-2004 in Treatment of Irritability Associated With Auti... The purpose of this study is to establish the potential benefits, safety, and tolerability of AB-2004 in participants with irritability ...

5. axialtx.com axialtx.com/axial-biotherapeutics-announces-positive-topline-results-from-phase-1b-2a-clinical-trial-of-ab-2004-for-the-treatment-of-autism-spectrum-disorder/

Axial Biotherapeutics Announces Positive Topline Results ... In a preclinical mouse model of ASD, AB-2004 reduced urinary levels and neuroactive microbial metabolites (NMMs™), repaired leaky gut and ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35165451/

an open-label phase 1b/2a trial Accordingly, a pilot human study was designed and completed to evaluate the safety of AB-2004 in an open-label, single-cohort, multiple-ascending-dose clinical ...