Evaluate the Efficacy and Safety of TTYP01 Tablets in Adolescents and Children With ASD

NCT06757504 | Recruiting Phase 2 DMC

• 1 other identifier: TTYP01-Ⅱ-ASD
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Autism Spectrum Disorder (ASD)

Outcome Measures

Primary Outcomes (3)

• 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.

  At baseline and week 12

• 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.

  Throughout the trial period

• 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.

  Visit 2 (Day 1): 30 minutes (±5 minutes) and 3 hours (±10 minutes) after the first dose; Visit 8 (Week 12 ±2 Days)or Early withdrawal: before the first dose (0 hour), and 30 minutes (±5 minutes) after the first dose on the day of the visit.

Secondary Outcomes (10)

• Changes from baseline in ABC-SW scores at Weeks 4 and 8.

  At baseline, Weeks 4 and 8

• Changes from baseline in other ABC subscale scores at Weeks 4, 8 and 12.

  At baseline, Weeks 4, 8 and 12

• Clinical Global Impressions-Improvement (CGI-I) scores at Weeks 4, 8, and 12.

  At Weeks 4, 8, and 12.

• Changes from baseline in Clinical Global Impressions-Severity (CGI-S) scores at Weeks 4, 8, and 12.

  At baseline, Weeks 4, 8 and 12

• Changes from baseline in Social Responsiveness Scale 2 (SRS-2) total scores at Weeks 4, 8, and 12.

  At baseline, Weeks 4, 8 and 12

Other Outcomes (5)

• 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.

  At baseline and week 12

• Changes from baseline in fNIRS examination at Week 12.

  At baseline and week 12

• 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.

  At baseline and week 12

Study Arms (3)

TTYP01 low-dose group

EXPERIMENTAL

TTYP01 Tablets (12mg or 18mg), Twice daily for 12 weeks. Dosage is determined based on body weight and group assignment, with the low-dose group receiving 12mg-30mg BID, the dosing regimen is detailed in the trial protocol.

Drug: TTYP01 Tablets (12mg or 18mg); Drug: Placebo (Simulant TTYP01 Tablets)

TTYP01 high-dose group

EXPERIMENTAL

TTYP01 Tablets (12mg or 18mg), Twice daily for 12 weeks. Dosage is determined based on body weight and group assignment, with the high-dose group receiving 24mg-60mg BID, the dosing regimen is detailed in the trial protocol.

Drug: TTYP01 Tablets (12mg or 18mg); Drug: Placebo (Simulant TTYP01 Tablets)

Placebo group

PLACEBO COMPARATOR

Simulant TTYP01 Tablets, Twice daily for 12 weeks.

Drug: Placebo (Simulant TTYP01 Tablets)

Interventions

TTYP01 Tablets (12mg or 18mg) DRUG

Taken 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.

TTYP01 high-dose group; TTYP01 low-dose group

Placebo (Simulant TTYP01 Tablets) DRUG

Taken 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 group; TTYP01 high-dose group; TTYP01 low-dose group

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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;
• CGI-S score ≥4;
• Participants and their legal guardians understand and are willing to participate in this trial, with ICF signed by the legal guardian and participants aged ≥ 8 years; for participants who, at the discretion of the investigator, have cognitive deficits that prevent them from signing the ICF, their legal guardians sign the ICF on behalf of them;
• Females of childbearing potential (as defined in Appendix 15.1) agree to remain abstinent or use a reliable method of contraception for the duration of the trial and until 3 months after the last dose of the investigational product.

You may not qualify if:

• Any participant who meets any of the following criteria will be excluded:
• Weight \<20.0 kg or \>70.0 kg, or BMI ≥35.0 kg/m\^2;
• 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;
• Participants with a history or symptoms of other mental illness that, at the discretion of the investigator, may affect the results of the study;
• Participants with a history of any unstable physical or neurological condition or currently suffering from a physical or neurological condition that, at the discretion of the investigator, may put them at risk of a significant adverse event or interfere with the assessment of safety and efficacy during the course of the trial;
• Pre-existing educational training and/or behavioral treatments fail to be stabilized prior to screening and consistent throughout the trial;
• Participants who are unable to discontinue or may use other treatments during the screening period and throughout the trial due to their condition, including: antipsychotic medications (except those that may be used in combination during the trial as specified in the protocol), medications that may have a therapeutic effect on ASD, nootropic drugs, medications for ADHD, intestinal flora modification, and other intestinal flora modification supplementation alternatives;
• Participants who have used treatment modalities such as transcranial magnetic stimulation therapy, direct current stimulation therapy, electroencephalographic biofeedback therapy, auditory integration training, executive function training, acupuncture therapy, etc., within 2 months prior to randomization, or may use such treatments during the treatment period;
• ECG and laboratory results as outlined below: (1)QTc is outside the normal range of the site; (2)Platelets are below the lower limit of normal for the site; (3)Haemoglobin is below the lower limit of normal for the site; (4)Neutrophil count is below the lower limit of normal for the site; (5)ALT or AST ≥ 2 × upper limit of normal, or total bilirubin \> upper limit of normal for the site; (6)Urea nitrogen or creatinine is above the upper limit of normal for the site; (7)Any other abnormal laboratory, vital sign, or 12-ECG findings that, at the discretion of the investigator, are abnormal and clinically significant and will affect the safety of the participant or the interpretation of the study results;
• Participation in any clinical trial of a drug or non-drug intervention within the last 1 month;
• Concomitant medications/therapies that are prohibited in the protocol may be required during the trial;
• Participants expected to undergo elective surgery during the trial;
• Participants whose parents/guardians are unable to understand and/or complete the scale assessments in this trial.

Sponsors & Collaborators

• Shanghai Auzone Biological Technology Co., Ltd.: lead

Study Sites (5)

Peking University Sixth Hospital

Beijing, China

RECRUITING

The Second Xiangya Hospital of Central South University

Changsha, China

NOT YET RECRUITING

West China Hospital of Sichuan University

Chengdu, China

NOT YET RECRUITING

Nanjing Brain Hospital, The Affiliated Brain Hospital of Nanjing Medical University

Nanjing, China

NOT YET RECRUITING

Tianjin Mental Health Center

Tianjin, China

NOT YET RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Jing Liu

  Peking University Sixth Hospital

  STUDY CHAIR

Central Study Contacts

Xiaolong Hou

CONTACT

+86-21-65039008; houxl@auzonebio.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 25, 2024
• First Posted: January 3, 2025
• Study Start: January 13, 2025
• Primary Completion: February 6, 2026
• Study Completion (Estimated): May 8, 2026
• Last Updated: May 14, 2025

Record last verified: 2025-05

Locations

CN (5)