We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04895215
Recruitment Status : Recruiting
First Posted : May 20, 2021
Last Update Posted : April 19, 2023
Information provided by (Responsible Party):
Axial Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder (ASD) Drug: AB-2004 Drug: Placebo Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Double Blind, Randomized, Placebo Controlled
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled Study of the Efficacy, Safety, and Tolerability of AB-2004 in an Autism Spectrum Disorder Population
Actual Study Start Date : August 2, 2021
Estimated Primary Completion Date : August 2023
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: AB-2004 Drug: AB-2004
Taken 3 times daily with food

Placebo Comparator: Placebo Drug: Placebo
Take 3 times daily with food

Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Number of participants who reported treatment emergent adverse events (TEAEs) [ Time Frame: From baseline to Week 8 visit ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04895215

Layout table for location contacts
Contact: Clinical Trial Team +1 781-701-8484 clinicaltrialteam@axialtx.com

Show Show 22 study locations
Sponsors and Collaborators
Axial Therapeutics, Inc.
Layout table for additonal information
Responsible Party: Axial Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04895215    
Other Study ID Numbers: AXL-2004-002
First Posted: May 20, 2021    Key Record Dates
Last Update Posted: April 19, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Axial Therapeutics, Inc.:
Additional relevant MeSH terms:
Layout table for MeSH terms
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders