A Study to Evaluate the Efficacy and Safety of AST-001 in... | NCT06333964

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

NCT06333964

Sponsor

Astrogen, Inc.

Status

Completed

Last Update Posted

Feb 2, 2026Actual

Enrollment

169Actual

Phase

Phase 3

Conditions

Autism Spectrum Disorder

Interventions

AST-001
Placebo of AST-001

Countries

South Korea

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2026

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 29, 2023Actual

Primary Completion Date

Dec 5, 2024Actual

Completion Date

Dec 5, 2024Actual

First Submitted Date

Mar 21, 2024

First Submission Date that Met QC Criteria

Mar 21, 2024

First Posted Date

Mar 27, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 29, 2026

Last Update Posted Date

Feb 2, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

169Actual

Label	Type	Description	Intervention Names
AST-001	Experimental		Drug: AST-001
Placebo of AST-001	Placebo Comparator		Drug: Placebo of AST-001

Name	Type	Description	Arm Group Labels	Other Names
AST-001	Drug	AST-001, PO bid for 24weeks (dosage according to weight range)	AST-001

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	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)

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

7 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Soon Chun Hyang University Hospital Cheonan		Cheonan	Chungcheongnam-do		South Korea
Hallym University Medical Center

ID	Term
D002659	Child Development Disorders, Pervasive
D065886	Neurodevelopmental Disorders
D001523	Mental Disorders