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

NCT ID: NCT06333964

Last Updated: 2025-02-11

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 169 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-29
• Study Completion Date: 2025-06-30

Brief Summary

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

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

AST-001

Group Type: EXPERIMENTAL

AST-001

Intervention Type: DRUG

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

Placebo of AST-001

Group Type: PLACEBO_COMPARATOR

Placebo of AST-001

Intervention Type: DRUG

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

Interventions

AST-001

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

Intervention Type: 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)

Intervention Type: DRUG

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 7 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Astrogen, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Yoo-Sook Joung, MD, Ph.D

Role: PRINCIPAL_INVESTIGATOR

Samsung Medical Center

Locations

Soon Chun Hyang University Hospital Cheonan

Cheonan, Chungcheongnam-do, South Korea

Hallym University Medical Center

Anyang-si, Gyeonggi-do, South Korea

Samsung Changwon Medical Center

Changwon, Gyeongsangnam-do, South Korea

Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, South Korea

Jeonbuk National University Hospital

Jeonju, Jeollabuk-do, South Korea

Chungnam National University Hospital

Daejeon, , South Korea

Asan Medical Center

Seoul, , South Korea

Hanyang University Seoul Hospital

Seoul, , South Korea

Koera University Guro Hospital

Seoul, , South Korea

Samsung Medical Center

Seoul, , South Korea

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

Seoul, , South Korea

Countries

South Korea

Other Identifiers

AST-001P_P301_ASD

Identifier Type: -

Identifier Source: org_study_id