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

NCT06333964 | Completed Phase 3

• 1 other identifier: AST-001P_P301_ASD
• Study Type: interventional
• Target: 169
• Locations: 1 country
• Sites: 11

Brief Summary

1. 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. 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. 3.Design: Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 clinical trial followed by an Open-Label Extension Treatment Period

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)

  Baseline, 12week, 24week

Secondary Outcomes (3)

• 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

Study Arms (2)

AST-001

EXPERIMENTAL

Drug: AST-001

Placebo of AST-001

PLACEBO COMPARATOR

Drug: Placebo of AST-001

Interventions

AST-001 DRUG

AST-001, PO bid for 24weeks (dosage according to weight range)

AST-001

Placebo of AST-001 DRUG

Placebo of AST-001, PO bid for 12 weeks + AST-001, PO bid for 12 weeks (dosage according to weight range)

Placebo of AST-001

Eligibility Criteria

• Age: 2 Years - 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Astrogen, Inc.: lead

Study Sites (11)

Soon Chun Hyang University Hospital Cheonan

Cheonan, Chungcheongnam-do, South Korea

Location

Hallym University Medical Center

Anyang-si, Gyeonggi-do, South Korea

Location

Samsung Changwon Medical Center

Changwon, Gyeongsangnam-do, South Korea

Location

Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, South Korea

Location

Jeonbuk National University Hospital

Jeonju, Jeollabuk-do, South Korea

Location

Chungnam National University Hospital

Daejeon, South Korea

Location

Asan Medical Center

Seoul, South Korea

Location

Hanyang University Seoul Hospital

Seoul, South Korea

Location

Koera University Guro Hospital

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Yoo-Sook Joung, MD, Ph.D

  Samsung Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Main study(0\~12 weeks) Experimental: AST-001 Comparator: Placebo of AST-001 Extension study(12\~24 weeks) Experimental: AST-001 Comparator: AST-001

Study Record Dates

• First Submitted: March 21, 2024
• First Posted: March 27, 2024
• Study Start: August 29, 2023
• Primary Completion: December 5, 2024
• Study Completion: December 5, 2024
• Last Updated: February 2, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

KR (11)