A Study to Evaluate the Efficacy and Safety of AST-001 in ASD Children

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase III Clinical Trial to Evaluate the Efficacy and Safety of AST-001 Followed by an Open-Label Extension Treatment Period in Children With Autism Spectrum Disorder

1. Study purpose: To demonstrate the superiority of AST-001 compared to placebo in improving core symptoms of autism spectrum disorder (ASD) in children with ASD.
2. Background: ASD is a neurodevelopmental disorder characterized by deficits in social communication and social interaction as well as restricted, repetitive patterns of behavior, interests, or activities. There are no approved medicines to treat the core symptom of ASD. Although these drugs and other psychotropic medications are associated with side effects, the use of psychotropic drugs to treat associated psychiatric comorbidities is common. AST-001 is developed to treat the core symptom of ASD.
3. Design: Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 clinical trial followed by an Open-Label Extension Treatment Period

Study Overview

• Status: Completed
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: AST-001; Drug: Placebo of AST-001

• Study Type: Interventional
• Enrollment (Actual): 169
• Phase: Phase 3

Contacts and Locations

Study Locations

• South Korea
  • Daejeon, South Korea
    • Chungnam National University Hospital
  • Seoul, South Korea
    • Asan Medical Center
  • Seoul, South Korea
    • Samsung Medical Center
  • Seoul, South Korea
    • The Catholic University of Korea, Seoul ST. Mary's Hospital
  • Seoul, South Korea
    • Hanyang University Seoul Hospital
  • Seoul, South Korea
    • Koera University Guro Hospital
  • Chungcheongnam-do
    • Cheonan, Chungcheongnam-do, South Korea
      • Soon Chun Hyang University Hospital Cheonan
  • Gyeonggi-do
    • Anyang-si, Gyeonggi-do, South Korea
      • Hallym University Medical Center
  • Gyeongsangnam-do
    • Changwon, Gyeongsangnam-do, South Korea
      • Samsung Changwon Medical Center
    • Yangsan, Gyeongsangnam-do, South Korea
      • Pusan National University Yangsan Hospital
  • Jeollabuk-do
    • Jeonju, Jeollabuk-do, South Korea
      • Jeonbuk National University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Those who meet the criteria of diagnostic and statistical manual of mental disorder (DSM)-5 during screening
• During screening period, individuals who are diagnosed with ASD through ADI-R assessment
• Subject with a CGI-S score of 4 or higher as a result of Clinical Global Impression(CGI) evaluation during baseline visit(visit 2)
• Subject or/and legally authorized representative voluntarily agreed to participate in this clinical trial and provided their informed consent by signing the written consent form
• In case of subjects who were receiving non-pharmacological therapy at the time of screening, the non-pharmacological therapy lasted at least 3 months prior to participation in screening and is expected to be sustainable during this clinical trial
• Subject whose legally authorized representative is able to participate in the provision of reliable information about the subject's condition, execute all scheduled site visits, oversee IP administration, fully understand and speak Korean, and conduct survey evaluation regarding the subject

Exclusion Criteria:

• At the time of screening, subject has the medical history, concomitant condition, or surgical history
• During the screening period, uncontrolled medical conditions,
• During the screening period, display of severe self-harm or injury to others that requires medical treatment, determined by investigator
• At the time of screening, weight over 60kg
• Inappropriate to participate in the trial determined by investigator

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AST-001	Drug: AST-001; AST-001, PO bid for 24weeks (dosage according to weight range)
Placebo Comparator: Placebo of AST-001	Drug: Placebo of AST-001; Placebo of AST-001, PO bid for 12 weeks + AST-001, PO bid for 12 weeks (dosage according to weight range)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)	Baseline, 12week, 24week

Secondary Outcome Measures

Outcome Measure	Time Frame
CGI (Clinical Global Impression)	Baseline, 4week, 8week, 12week, 18week, 24week
SRS-2 (Social Responsiveness Scale-2)	Baseline, 4week, 12week, 24week
K-PSI-4-SF (Korean-Parenting Stress Index-4th Edition Short Form)	Baseline, 12week, 24week

Collaborators and Investigators

• Sponsor: Astrogen, Inc.
• Investigators: Principal Investigator: Yoo-Sook Joung, MD, Ph.D, Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): August 29, 2023
• Primary Completion (Actual): December 5, 2024
• Study Completion (Actual): December 5, 2024

Study Registration Dates

• First Submitted: March 21, 2024
• First Submitted That Met QC Criteria: March 21, 2024
• First Posted (Actual): March 27, 2024

Study Record Updates

• Last Update Posted (Actual): February 2, 2026
• Last Update Submitted That Met QC Criteria: January 29, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autism Spectrum Disorder
• Other Study ID Numbers: AST-001P_P301_ASD

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