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

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 Overview

• Status: Active, not recruiting
• Conditions: Autism Spectrum Disorder (ASD)
• Intervention / Treatment: Drug: AB-2004; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 156
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2145
      • Westmead Children's Hospital
    • Sydney, New South Wales, Australia, 2050
      • Brain Mind Centre
  • Queensland
    • South Brisbane, Queensland, Australia, 4101
      • Children's Health Queensland Hospital
  • Victoria
    • Clayton, Victoria, Australia, 3186
      • Monash Kids Research
    • Parkville, Victoria, Australia, 3052
      • Murdoch Children's Research Institute
• New Zealand
  • Auckland
    • Grafton, Auckland, New Zealand, 1010
      • Optimal Clinical Trials
• United States
  • Alabama
    • Dothan, Alabama, United States, 36303
      • Harmonex Neuroscience Research
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Southwestern Autism Research and Resource Center
    • Tucson, Arizona, United States, 85724
      • University of Arizona
  • California
    • San Rafael, California, United States, 94903
      • Cortica Marin
    • Westlake Village, California, United States, 91361
      • Cortica Westlake
  • Colorado
    • Centennial, Colorado, United States, 80112
      • IMMUNOe Research Centers
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University
  • Florida
    • Jacksonville, Florida, United States, 32256
      • CNS Solutions
    • Orlando, Florida, United States, 32801
      • CNS Solutions
    • Tampa, Florida, United States, 33613
      • University of South Florida
  • Illinois
    • Naperville, Illinois, United States, 60563
      • Baber Research
  • Missouri
    • Columbia, Missouri, United States, 65201
      • University of Missouri, Thompson Center for Autism and Neurodevelopmental Disorders
  • Nevada
    • Las Vegas, Nevada, United States, 89128
      • Clinical Research of Southern Nevada
  • New York
    • New York, New York, United States, 10021
      • Spectrum Neuroscience and Treatment Institute
    • Orangeburg, New York, United States, 10962
      • Nathan S. Kline Institute for Psychiatric Research
  • Ohio
    • Columbus, Ohio, United States, 43210
      • Ohio State University
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center
  • Texas
    • Dallas, Texas, United States, 75243
      • Relaro Medical Trials

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Take 3 times daily with food
Experimental: AB-2004	Drug: AB-2004; Taken 3 times daily with food

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Axial Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 2, 2021
• Primary Completion (Estimated): December 1, 2023
• Study Completion (Estimated): December 1, 2023

Study Registration Dates

• First Submitted: May 17, 2021
• First Submitted That Met QC Criteria: May 17, 2021
• First Posted (Actual): May 20, 2021

Study Record Updates

• Last Update Posted (Actual): September 29, 2023
• Last Update Submitted That Met QC Criteria: September 27, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Keywords: Anxiety; Irritability
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: AXL-2004-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No