This is a multi-center, randomized, double-blind, multiple dose levels, parallal group, placebo-controlled study, to evaluate the safety, PK profiles and preliminary efficacy of TTYP01 tablets in adolescents and children with ASD.
Aged ≥ 6 to <16 years at the time of signing the ICF (age <16 years after 12-week treatment), including adolescents aged ≥ 12 to<16 years at the time of signing the ICF (age <16 years after 12-week treatment), and children aged ≥ 6 to<12 years at the time of signing the ICF;
Male or female;
Participants diagnosed with ASD according to the ASD diagnostic criteria in Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) and confirmed by the Schedule for Affective Disorders and Schizophrenia for School Aged Children Present and Lifetime version (K-SADS-PL), DSM-5 ASD module;
Child Autism Rating Scale 2-ST (CARS 2-ST) raw score ≥36;
Weight <20.0 kg or >70.0 kg, or BMI ≥35.0 kg/m²;
Pregnant or lactating women;
Presence of a serious mental disorder as assessed by the investigator, e.g., schizophrenia, bipolar disorder, depressive disorder, etc.; the presence of significant anxiety, tension, agitation, fear, depression, or the presence of suicidal risk, significant risk of self-injury, impulsivity, aggression, or behaviors based on the medical history and the routine mental status examination;
History of epileptic seizures within 3 months prior to screening, or presence of a history of severe physical or neurological disease, history of severe head trauma;
Parallel
TTYP01 Tablets (12mg or 18mg)
DrugTaken with a moderate amount of warm water or after disperse in a moderate amount of warm water, at least 60 minutes before breakfast and dinner.
Placebo (Simulant TTYP01 Tablets)
DrugTaken with a moderate amount of warm water or after disperse in a moderate amount of warm water, at least 60 minutes before breakfast and dinner.
Change from baseline in ABC-SW（Aberrant Behavioral Checklist - Social Withdrawal） scores at Week 12.
The ABC (Aberrant Behavioral Checklist) scale has 58 items. The scale is broken down into five subscales including ABC-SW（Aberrant Behavioral Checklist - Social Withdrawal）, ABC-I, A, C （Aberrant Behavioral Checklist - Irritability, Agitation, Crying）, ABC-SB （Aberrant Behavioral Checklist - Stereotypic Behavior）, ABC-H, N (Aberrant Behavioral Checklist - Hyperactivity, Noncompliance ), and ABC-IS (Aberrant Behavioral Checklist - Inappropriate Speech ). Scale ratings can be completed by parents, special educators, psychologists, direct caregivers, nurses, and others with knowledge of the person being assessed. The rater has to answer each of the 58 questions using a 4-point Likert scale. A score of a "0" means the behavior is not a problem, a score of a "1" means slight problem, a score of a "2" means a serious problem, and a "3" means a severe problem.
Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), serious adverse events (SAEs), and TEAEs that lead to treatment discontinuation.
To evaluate the safety and tolerability of TTYP01 tablets in adolescent and pediatric participants with ASD.
Plasma concentrations and population PK profiles of edaravone and its metabolites in adolescent and pediatric ASD participants.
Concentrations of edaravone and its metabolites are assayed in the plasma of adolescent and pediatric participants with ASD. They will be analyzed with descriptive statistics according to planned sampling points.
Changes from baseline in ABC-SW scores at Weeks 4 and 8.
The ABC (Aberrant Behavioral Checklist) scale has 58 items. The scale is broken down into five subscales including ABC-SW（Aberrant Behavioral Checklist - Social Withdrawal）, ABC-I, A, C （Aberrant Behavioral Checklist - Irritability, Agitation, Crying）, ABC-SB （Aberrant Behavioral Checklist - Stereotypic Behavior）, ABC-H, N (Aberrant Behavioral Checklist - Hyperactivity, Noncompliance ), and ABC-IS (Aberrant Behavioral Checklist - Inappropriate Speech ). Scale ratings can be completed by parents, special educators, psychologists, direct caregivers, nurses, and others with knowledge of the person being assessed. The rater has to answer each of the 58 questions using a 4-point Likert scale. A score of a "0" means the behavior is not a problem, a score of a "1" means slight problem, a score of a "2" means a serious problem, and a "3" means a severe problem.
Changes from baseline in other ABC subscale scores at Weeks 4, 8 and 12.
The ABC (Aberrant Behavioral Checklist) scale has 58 items. The scale is broken down into five subscales including ABC-SW（Aberrant Behavioral Checklist - Social Withdrawal）, ABC-I, A, C （Aberrant Behavioral Checklist - Irritability, Agitation, Crying）, ABC-SB （Aberrant Behavioral Checklist - Stereotypic Behavior）, ABC-H, N (Aberrant Behavioral Checklist - Hyperactivity, Noncompliance ), and ABC-IS (Aberrant Behavioral Checklist - Inappropriate Speech ). Scale ratings can be completed by parents, special educators, psychologists, direct caregivers, nurses, and others with knowledge of the person being assessed. The rater has to answer each of the 58 questions using a 4-point Likert scale. A score of a "0" means the behavior is not a problem, a score of a "1" means slight problem, a score of a "2" means a serious problem, and a "3" means a severe problem.
Clinical Global Impressions-Improvement (CGI-I) scores at Weeks 4, 8, and 12.
The CGI includes the CGI-S and CGI-I. The CGI rating scale allows for an overall assessment of participant improvement over time. At baseline, the CGI-S assessment is performed and the rating investigator rates the severity of the participant's condition based on a 7-point scale ranging from 1 (no symptoms) to 7 (very severe symptoms). At the follow-up visit, the scoring investigator assessed the degree of improvement in the participant's symptoms relative to baseline based on the CGI-I items on a 7-point scale ranging from 1 (very marked improvement) to 7 (very marked deterioration).
Changes from baseline in Clinical Global Impressions-Severity (CGI-S) scores at Weeks 4, 8, and 12.
The CGI includes the CGI-S and CGI-I. The CGI rating scale allows for an overall assessment of participant improvement over time. At baseline, the CGI-S assessment is performed and the rating investigator rates the severity of the participant's condition based on a 7-point scale ranging from 1 (no symptoms) to 7 (very severe symptoms). At the follow-up visit, the scoring investigator assessed the degree of improvement in the participant's symptoms relative to baseline based on the CGI-I items on a 7-point scale ranging from 1 (very marked improvement) to 7 (very marked deterioration).
Changes from baseline in the proportion of time spent looking at different facial areas and social situations in eye-tracking tasks by eye-tracking method at Week 12 of administration.
Exploratory objective metrics that may predict benefit from TTYP01 tablets
Changes from baseline in fNIRS examination at Week 12.
Exploratory Outcomes, Objectivity indicators that may predict benefit from TTYP01 tablets
Changes from baseline in the level of plasma cytokines (i.e., IL-1β, IL-6, TNF-α, INF-γ, IL-7, IL-12, and IL-17) at Week 12.
Exploratory Outcomes, Biomarker indicators that may predict benefit from TTYP01 tablets
Changes from baseline in the levels of plasma reduced glutathione (GSH), oxidized glutathione (GSSG), and GSH/GSSG at Week 12.
Exploratory Outcomes, Biomarker indicators that may predict benefit from TTYP01 tablets
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