Autism Spectrum DisorderPhase 3NCT06333964
A Study to Evaluate the Efficacy and Safety of AST-001 in ASD Children
A Phase 3 study testing AST-001 in people with Autism Spectrum Disorder, run by Astrogen, Inc. at 11 sites in South Korea.
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… Read more about this study
- Who is it for?
- Ages 2 to 7 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- AST-001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 169 participants
- Where
- 11 sitesSouth Korea
- Study dates
- 29 Aug 2023to 5 Dec 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 7 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
South Korea11 sites
Hallym University Medical Center
Anyang-si, Gyeonggi-do
Samsung Changwon Medical Center
Changwon, Gyeongsangnam-do
Soon Chun Hyang University Hospital Cheonan
Cheonan, Chungcheongnam-do
Chungnam National University Hospital
Daejeon
Jeonbuk National University Hospital
Jeonju, Jeollabuk-do
Asan Medical Center
Seoul
Hanyang University Seoul Hospital
Seoul
Koera University Guro Hospital
Seoul
Samsung Medical Center
Seoul
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do
About this study
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 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
- Conditions
- Autism Spectrum Disorder
- Being tested
- AST-001, Placebo of AST-001
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AST-001Drug
AST-001, PO bid for 24weeks (dosage according to weight range)
Placebo of AST-001Drug
Placebo of AST-001, PO bid for 12 weeks + AST-001, PO bid for 12 weeks (dosage according to weight range)
| Study group | Role | What they receive |
|---|---|---|
| AST-001 | Experimental | Not described Receives: Drug: AST-001 |
| Placebo of AST-001 | Placebo comparator | Not described Receives: Drug: Placebo of AST-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)
Measured: Baseline, 12week, 24week
Secondary outcomes (3)
CGI (Clinical Global Impression)
Measured: Baseline, 4week, 8week, 12week, 18week, 24week
SRS-2 (Social Responsiveness Scale-2)
Measured: Baseline, 4week, 12week, 24week
K-PSI-4-SF (Korean-Parenting Stress Index-4th Edition Short Form)
Measured: Baseline, 12week, 24week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2024
- Study start29 Aug 2023
- Primary completion5 Dec 2024
- Study completion5 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 169 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astrogen, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Yoo-Sook Joung, MD, Ph.D, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06333964
- Sponsor's protocol ID
- AST-001P_P301_ASD
- Status last verified
- Jan 2026
- First submitted
- 21 Mar 2024
- Last updated
- 2 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06333964
- Acronym / sponsor ID
- AST-001P_P301_ASD
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 169 participants (actual)
- Lead sponsor
- Astrogen, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Aug 2023 (actual)
- Primary completion
- 5 Dec 2024 (actual)
- Study completion
- 5 Dec 2024 (actual)
- First posted
- 27 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- 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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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
| Arm | Type | Receives |
|---|---|---|
| AST-001 | Experimental | Drug: AST-001 |
| Placebo of AST-001 | Placebo comparator | Drug: Placebo of AST-001 |
Who may qualify
- Ages
- 2 to 7 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AST-001AST-001, PO bid for 24weeks (dosage according to weight range) | Drug | AST-001 |
| Placebo of AST-001Placebo of AST-001, PO bid for 12 weeks + AST-001, PO bid for 12 weeks (dosage according to weight range) | Drug | Placebo of AST-001 |
Primary outcome
K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II)
Time frame: Baseline, 12week, 24week
3 secondary outcomes
CGI (Clinical Global Impression)
Time frame: Baseline, 4week, 8week, 12week, 18week, 24week
SRS-2 (Social Responsiveness Scale-2)
Time frame: Baseline, 4week, 12week, 24week
K-PSI-4-SF (Korean-Parenting Stress Index-4th Edition Short Form)
Time frame: Baseline, 12week, 24week
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yoo-Sook Joung, MD, Ph.D principal investigator
Samsung Medical Center
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AST-001 in people with Autism Spectrum Disorder, run by Astrogen, Inc. at 11 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
K-VABS-II (Korean-Vineland Adaptive Behavior Scale-II) (time frame: Baseline, 12week, 24week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2026.