Evaluating TTYP01 Tablets for Autism in Children and Adolescents

A Multi-Center, Randomized, Double-Blind, Parallel Group, Placebo-controlled Phase II Trial of TTYP01 Tablets in Adolescents and Children With Autism Spectrum Disorders

This study is testing if TTYP01 tablets can be a safe and effective treatment for children and teens with Autism Spectrum Disorder.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 150 (estimated)
• Ages: 6 Years to 16 Years
• Sex: All
• Sponsor: Shanghai Auzone Biological Technology Co., Ltd. (Industry-sponsored)
• Locations: 5 sites (Beijing and 4 other locations)
• Trial ID: NCT06757504 on ClinicalTrials.gov

What this trial studies

This clinical trial is designed to assess the safety and preliminary efficacy of TTYP01 tablets in children and adolescents diagnosed with Autism Spectrum Disorder (ASD). It employs a multi-center, randomized, double-blind, placebo-controlled approach, with participants receiving either the active medication or a placebo. The trial will be conducted in three stages, starting with adolescents aged 12 to 16, followed by potential enrollment of younger children aged 6 to 12, based on safety evaluations by a Data and Safety Monitoring Board. The study aims to enroll a total of 150 participants across both age groups.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 6 to 16 years diagnosed with ASD as per DSM-5 criteria.

Not a fit: Patients outside the age range of 6 to 16 years or those not diagnosed with ASD will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for managing symptoms of Autism Spectrum Disorder in children and adolescents.

How similar studies have performed: While there have been various studies on treatments for ASD, the specific approach of using TTYP01 tablets is novel and has not been extensively tested in this population.

Eligibility criteria

Inclusion Criteria:

Participants are eligible for the study only if all of the following inclusion criteria are met:

1. 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;
2. Male or female;
3. 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;
4. Child Autism Rating Scale 2-ST (CARS 2-ST) raw score ≥36;
5. CGI-S score ≥4;
6. 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;
7. 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.

Exclusion Criteria:

Any participant who meets any of the following criteria will be excluded:

1. Weight \<20.0 kg or \>70.0 kg, or BMI ≥35.0 kg/m\^2;
2. Pregnant or lactating women;
3. 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;
4. 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;
5. Participants with a history or symptoms of other mental illness that, at the discretion of the investigator, may affect the results of the study;
6. 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;
7. Pre-existing educational training and/or behavioral treatments fail to be stabilized prior to screening and consistent throughout the trial;
8. 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;
9. 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;
10. 12-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;
11. Participation in any clinical trial of a drug or non-drug intervention within the last 1 month;
12. Concomitant medications/therapies that are prohibited in the protocol may be required during the trial;
13. Participants expected to undergo elective surgery during the trial;
14. Participants whose parents/guardians are unable to understand and/or complete the scale assessments in this trial.
15. Hypersensitivity to edaravone and excipients (Soluplus, sodium bisulphite, microcrystalline cellulose, magnesium stearate, polyvinylpolypyrrolidone) in TTYP01 tablets;
16. Participants who are judged by the investigator to be ineligible for other reasons.

Where this trial is running

Beijing and 4 other locations

• 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)

Study contacts

• Study coordinator: Xiaolong Hou

Conditions Autism Spectrum Disorder