Clinical trial NCT06333964: A Study to Evaluate the Efficacy and Safety of AST-001 in ASD Children
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Autism Spectrum Disorder
Lead sponsor: Astrogen, Inc.
Sponsor class: INDUSTRY
First posted: 2024-03-27
Enrollment: 169
Sex: ALL
Minimum age (years): 2
Maximum age (years): 7
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Main study(0\~12 weeks) Experimental: AST-001 Comparator: Placebo of AST-001

Extension study(12\~24 weeks) Experimental: AST-001 Comparator: AST-001
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)
timeFrame: Baseline, 12week, 24week
Secondary outcomes:
measure: CGI (Clinical Global Impression)
timeFrame: Baseline, 4week, 8week, 12week, 18week, 24week
measure: SRS-2 (Social Responsiveness Scale-2)
timeFrame: Baseline, 4week, 12week, 24week
measure: K-PSI-4-SF (Korean-Parenting Stress Index-4th Edition Short Form)
timeFrame: Baseline, 12week, 24week
Other outcomes:
Locations:
city: Cheonan
country: Korea, Republic of
facility: Soon Chun Hyang University Hospital Cheonan
state: Chungcheongnam-do
city: Anyang
country: Korea, Republic of
facility: Hallym University Medical Center
state: Gyeonggi-do
city: Changwon
country: Korea, Republic of
facility: Samsung Changwon Medical Center
state: Gyeongsangnam-do
city: Yangsan
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
state: Gyeongsangnam-do
city: Jeonju
country: Korea, Republic of
facility: Jeonbuk National University Hospital
state: Jeollabuk-do
city: Daejeon
country: Korea, Republic of
facility: Chungnam National University Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Hanyang University Seoul Hospital
city: Seoul
country: Korea, Republic of
facility: Koera University Guro Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: The Catholic University of Korea, Seoul St. Mary's Hospital
Study officials:
affiliation: Samsung Medical Center
name: Yoo-Sook Joung, MD, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: