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

NCT04895215 | Completed Phase 2 FDA Drug

• 1 other identifier: AXL-2004-002
• Study Type: interventional
• Target: 156
• Locations: 3 countries
• Sites: 24

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.

Conditions

Autism Spectrum Disorder (ASD)

Keywords

Irritability; Anxiety

Outcome Measures

Primary Outcomes (1)

• The mean change in the ABC-I score (Irritability) from Baseline to Week 8 for AB-2004

  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

  From baseline to Week 8 visit

• Number of participants who reported treatment emergent adverse events (TEAEs)

  From baseline to Week 8 visit

Study Arms (2)

AB-2004

EXPERIMENTAL

Drug: AB-2004

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

AB-2004 DRUG

Taken 3 times daily with food

AB-2004

Placebo DRUG

Take 3 times daily with food

Placebo

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Vertero Therapeutics: lead

Study Sites (24)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Southwestern Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

University of Arizona

Tucson, Arizona, 85724, United States

Location

Cortica Marin

San Rafael, California, 94903, United States

Location

Cortica Westlake

Westlake Village, California, 91361, United States

Location

IMMUNOe Research Centers

Centennial, Colorado, 80112, United States

Location

Yale University

New Haven, Connecticut, 06519, United States

Location

CNS Solutions

Jacksonville, Florida, 32256, United States

Location

CNS Solutions

Orlando, Florida, 32801, United States

Location

University of South Florida

Tampa, Florida, 33613, United States

Location

Baber Research

Naperville, Illinois, 60563, United States

Location

University of Missouri, Thompson Center for Autism and Neurodevelopmental Disorders

Columbia, Missouri, 65201, United States

Location

Clinical Research of Southern Nevada

Las Vegas, Nevada, 89128, United States

Location

Spectrum Neuroscience and Treatment Institute

New York, New York, 10021, United States

Location

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Relaro Medical Trials

Dallas, Texas, 75243, United States

Location

Brain Mind Centre

Sydney, New South Wales, 2050, Australia

Location

Westmead Children's Hospital

Sydney, New South Wales, 2145, Australia

Location

Children's Health Queensland Hospital

South Brisbane, Queensland, 4101, Australia

Location

Monash Kids Research

Clayton, Victoria, 3186, Australia

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

Optimal Clinical Trials

Grafton, Auckland, 1010, New Zealand

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Anxiety Disorders

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double Blind, Randomized, Placebo Controlled

Study Record Dates

• First Submitted: May 17, 2021
• First Posted: May 20, 2021
• Study Start: August 2, 2021
• Primary Completion: November 15, 2023
• Study Completion: December 15, 2023
• Last Updated: November 21, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (18); AU (5); NZ (1)