Phase III Clinical Trial to Evaluate the Efficacy and Safey of AST-001 in ASD Children

Autism Spectrum Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06333964
• Other study ID #: AST-001P_P301_ASD
• Status: Recruiting
• Phase: Phase 3
• Start date: August 29, 2023
• Est. completion date: December 31, 2024

Study information

• Verified date: March 2024
• Source: Astrogen, Inc.
• Contact: Hyun Kwon
• Phone: +82-10-4336-6061
• Email: imhyun[@]astrogen.co.kr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 160
• Est. completion date: December 31, 2024
• Est. primary completion date: September 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 7 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism Spectrum Disorder

Intervention

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

Locations

Country	Name	City	State
Korea, Republic of	Hallym University Medical Center	Anyang	Gyeonggi-do
Korea, Republic of	Samsung Changwon Medical Center	Changwon	Gyeongsangnam-do
Korea, Republic of	Soon Chun Hyang University Hospital Cheonan	Cheonan	Chungcheongnam-do
Korea, Republic of	Chungnam National University Hospital	Daejeon
Korea, Republic of	Jeonbuk National University Hospital	Jeonju	Jeollabuk-do
Korea, Republic of	Asan Medical Center	Seoul
Korea, Republic of	Hanyang University Seoul Hospital	Seoul
Korea, Republic of	Koera University Guro Hospital	Seoul
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	The Catholic University of Korea, Seoul St. Mary's Hospital	Seoul
Korea, Republic of	Pusan National University Yangsan Hospital	Yangsan	Gyeongsangnam-do

Sponsors (1)

Lead Sponsor	Collaborator
Astrogen, Inc.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)		Baseline, 12wk, 24wk
Secondary	CGI (Clinical Global Impression)		Baseline, 4wk, 8wk, 12wk, 18wk, 24wk
Secondary	SRS-2 (Social Responsiveness Scale-2)		Baseline, 4wk, 12wk, 24wk
Secondary	K-PSI-4-SF (Korean-Parenting Stress Index-4th Edition Short Form)		Baseline, 12wk, 24wk