Autism Spectrum Disorder (ASD)Phase 2NCT04895215
AB-2004 in Treatment of Irritability Associated With Autism Spectrum Disorder (ASD)
A Phase 2 study testing AB-2004 in people with Autism Spectrum Disorder (ASD), run by Vertero Therapeutics at 24 sites in 3 countries.
Trial summary
The purpose of this study is to establish the potential benefits, safety, and tolerability of AB-2004 in participants with irritability associated with autism spectrum disorder. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Autism Spectrum Disorder (ASD)
- What is being tested?
- AB-2004
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 156 participants
- Where
- 24 sitesin 3 countries
- Study dates
- 2 Aug 2021to 15 Dec 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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 Period
- Clinical Global Impression - Severity (CGI-S) scale score ≥4 at the Screening Period
Who cannot join
- 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
- Current use of antipsychotics (eg, aripiprazole or risperidone)
Trial locations and contacts
24 sites in 3 countries.
24 sites in 3 countries
United States18 sites
IMMUNOe Research Centers
Centennial, Colorado, 80112
University of Missouri, Thompson Center for Autism and Neurodevelopmental Disorders
Columbia, Missouri, 65201
Ohio State University
Columbus, Ohio, 43210
Relaro Medical Trials
Dallas, Texas, 75243
Harmonex Neuroscience Research
Dothan, Alabama, 36303
CNS Solutions
Jacksonville, Florida, 32256
Clinical Research of Southern Nevada
Las Vegas, Nevada, 89128
Baber Research
Naperville, Illinois, 60563
Vanderbilt University Medical Center
Nashville, Tennessee, 37212
Yale University
New Haven, Connecticut, 06519
Spectrum Neuroscience and Treatment Institute
New York, New York, 10021
Nathan S. Kline Institute for Psychiatric Research
Orangeburg, New York, 10962
CNS Solutions
Orlando, Florida, 32801
Southwestern Autism Research and Resource Center
Phoenix, Arizona, 85006
Cortica Marin
San Rafael, California, 94903
University of South Florida
Tampa, Florida, 33613
University of Arizona
Tucson, Arizona, 85724
Cortica Westlake
Westlake Village, California, 91361
Australia5 sites
Monash Kids Research
Clayton, Victoria, 3186
Murdoch Children's Research Institute
Parkville, Victoria, 3052
Children's Health Queensland Hospital
South Brisbane, Queensland, 4101
Brain Mind Centre
Sydney, New South Wales, 2050
Westmead Children's Hospital
Sydney, New South Wales, 2145
About this study
Official title: A Randomized, Double-blind, Placebo-controlled Study of the Efficacy, Safety, and Tolerability of AB-2004 in an Autism Spectrum Disorder Population
The purpose of this study is to establish the potential benefits, safety, and tolerability of AB-2004 in participants with irritability associated with autism spectrum disorder.
- Conditions
- Autism Spectrum Disorder (ASD)
- Being tested
- AB-2004, Placebo
- Keywords
- Irritability, Anxiety
- Medical subject headings
- Autism Spectrum Disorder, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AB-2004Drug
Taken 3 times daily with food
PlaceboDrug
Take 3 times daily with food
| Study group | Role | What they receive |
|---|---|---|
| AB-2004 | Experimental | Not described Receives: Drug: AB-2004 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The mean change in the ABC-I score (Irritability) from Baseline to Week 8 for AB-2004
Measured: From baseline to Week 8 visit
Secondary outcomes (2)
The mean change in the Clinical Global Impression-Severity (CGI-S) from Baseline to Week 8 for AB-2004 High Dose and AB-2004 Low Dose
Measured: From baseline to Week 8 visit
Number of participants who reported treatment emergent adverse events (TEAEs)
Measured: From baseline to Week 8 visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2021
- Study start2 Aug 2021
- Primary completion15 Nov 2023
- Study completion15 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum Disorder (ASD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Double Blind, Randomized, Placebo Controlled
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 156 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertero Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04895215
- Sponsor's protocol ID
- AXL-2004-002
- Status last verified
- Nov 2025
- First submitted
- 17 May 2021
- Last updated
- 21 Nov 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled Study of the Efficacy, Safety, and Tolerability of AB-2004 in an Autism Spectrum Disorder Population
- ClinicalTrials.gov ID
- NCT04895215
- Acronym / sponsor ID
- AXL-2004-002
- Conditions
- Autism Spectrum Disorder (ASD)
- Keywords
- Irritability, Anxiety
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 156 participants (actual)
- Lead sponsor
- Vertero TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 2 Aug 2021 (actual)
- Primary completion
- 15 Nov 2023 (actual)
- Study completion
- 15 Dec 2023 (actual)
- First posted
- 20 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to establish the potential benefits, safety, and tolerability of AB-2004 in participants with irritability associated with autism spectrum disorder.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Double Blind, Randomized, Placebo Controlled
| Arm | Type | Receives |
|---|---|---|
| AB-2004 | Experimental | Drug: AB-2004 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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 Period
- Clinical Global Impression - Severity (CGI-S) scale score ≥4 at the Screening Period
Key exclusion criteria (4 listed)
- 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
- Current use of antipsychotics (eg, aripiprazole or risperidone)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AB-2004Taken 3 times daily with food | Drug | AB-2004 |
| PlaceboTake 3 times daily with food | Drug | Placebo |
Primary outcome
The mean change in the ABC-I score (Irritability) from Baseline to Week 8 for AB-2004
Time frame: From baseline to Week 8 visit
2 secondary outcomes
The mean change in the Clinical Global Impression-Severity (CGI-S) from Baseline to Week 8 for AB-2004 High Dose and AB-2004 Low Dose
Time frame: From baseline to Week 8 visit
Number of participants who reported treatment emergent adverse events (TEAEs)
Time frame: From baseline to Week 8 visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AB-2004 in people with Autism Spectrum Disorder (ASD), run by Vertero Therapeutics at 24 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The mean change in the ABC-I score (Irritability) from Baseline to Week 8 for AB-2004 (time frame: From baseline to Week 8 visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2025.