AB-2004 in Treatment of Irritability Associated With Autism Spectrum Disorder (ASD)

A Randomized, Double-blind, Placebo-controlled Study of the Efficacy, Safety, and Tolerability of AB-2004 in an Autism Spectrum Disorder Population

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04895215

Enrollment

156

Registered

2021-05-20

Start date

2021-08-02

Completion date

2023-12-15

Last updated

2025-11-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Autism Spectrum Disorder (ASD)

Keywords

Irritability, Anxiety

Brief 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.

Interventions

DRUGAB-2004

Taken 3 times daily with food

DRUGPlacebo

Take 3 times daily with food

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Intervention model description

Double Blind, Randomized, Placebo Controlled

Eligibility

Sex/Gender

ALL

Age

5 Years to 17 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: * 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

* 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)

Design outcomes

Primary

Measure	Time frame
The mean change in the ABC-I score (Irritability) from Baseline to Week 8 for AB-2004	From baseline to Week 8 visit

Secondary

Measure	Time frame
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	From baseline to Week 8 visit
Number of participants who reported treatment emergent adverse events (TEAEs)	From baseline to Week 8 visit

Countries

Australia, New Zealand, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026