A Trial of TTA-121 on Autism Spectrum Disorder

Study Overview
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Brief Summary
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To test efficacy and safety of a novel nasal spray of oxytocin on social deifies in autism spectrum disorder, and To compare effect sizes of different doses

Official Title
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An Early Phase II Trial for Efficacy and Safety of TTA-121 on Autism Spectrum Disorder

Conditions
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Autism Spectrum Disorder

Intervention / Treatment
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* Drug: TTA-121


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of autism spectrum disorder based on Diagnostic and Statistical Manual of Mental Disorders-V with score exceeding the cut-off value of 10 for qualitative abnormalities in social reciprocity on Autism Diagnostic Interview Revised (ADIR) Full scale Intelligent quotient above 80 as measured using the Wechsler Adult Intelligent Scale-III Written informed consent for participating the trial Exclusion Criteria: Diagnosis of bipolar disorder or schizophrenia spectrum disorder Primary diagnosis of depressive disorders, obsessive-compulsive and related disorders, anxiety disorders, trauma- and stressor-related disorders, dissociative disorders, somatic symptom and related disorders, or neurodevelopmental disorders other than autism spectr um disorder Instability in symptoms of comorbid mental disorders such as depressive disorders or anxiety disorders History of changes in medication or doses of psychotropics within one month before registration Current treatment with more than one psychotropics History of hyper-sensitivity to oxytocin History of seizures or traumatic brain injury with loss of consciousness for longer than 5 minutes History of alcohol-related disorders, substance abuse, or addiction Family history of male breast cancer Subject who has severe complications Known hypersensitivity to some drugs and foods Subject who is not able to consent contraception during study period Participation in another registration clinical trial and administration of investigational drug during 120 days before informed consent Other Subjects whom a lead investigator or the patient's primary physician deems are not appropriate for this study

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 54 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Low dose once per day and placebo<br>Four weeks administrations of TTA-121 3U once per day in morning and placebo once per day in evening. After four weeks washout, four weeks administrations of placebo twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: Low dose twice per day and placebo<br>Four weeks administrations of TTA-121 3U twice per day in morning and evening. After four weeks washout, four weeks administrations of placebo twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: High dose once per day and placebo<br>Four weeks administrations of TTA-121 10U once per day in morning and placebo once per day in evening. After four weeks washout, four weeks administrations of placebo twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: High dose twice per day and placebo<br>Four weeks administrations of TTA-121 10U twice per day in morning and evening. After four weeks washout, four weeks administrations of placebo twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: Placebo and low dose once per day<br>Four weeks administrations of placebo twice per day in morning and evening. After four weeks washout, four weeks administrations of TTA-121 3U once per day in morning and placebo once per day in evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: Placebo and low dose twice per day<br>Four weeks administrations of placebo twice per day in morning and evening. After four weeks washout, four weeks administrations of TTA-121 3U twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: Placebo and high dose once per day<br>Four weeks administrations of placebo twice per day in morning and evening. After four weeks washout, four weeks administrations of TTA-121 10U once per day in morning and placebo once per day in evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|
| Other: Placebo and high dose twice per day<br>Four weeks administrations of placebo twice per day in morning and evening. After four weeks washout, four weeks administrations of TTA-121 10U twice per day in morning and evening. | Drug: TTA-121<br>* A nove intranasal spray of oxytocin and placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy on autism spectrum social core symptom assessed by social reciprocity score on the Autism Diagnostic Observation Schedule module 4 | Changes in social reciprocity score (range: 0-14, Higher value represent a worth outcome) on Autism Diagnostic Observation Schedule module 4 between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy on autism spectrum core symptom assessed by communication score on the Autism Diagnostic Observation Schedule module 4 | Changes in communication score (range: 0-8, Higher value represent a worth outcome) on Autism Diagnostic Observation Schedule module 4 between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration | 
| Efficacy on autism spectrum core symptom assessed by repetitive and restricted behavior score on the Autism Diagnostic Observation Schedule module 4 | Changes in repetitive and restricted behavior score (range: 0-10, Higher value represent a worth outcome) on Autism Diagnostic Observation Schedule module 4 between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration | 
| Efficacy on autism spectrum core symptom assessed by revised algorithm score of social affect on the Autism Diagnostic Observation Schedule module 4 | Changes in revised algorithm score of social affect (range: 0-20, Higher value represent a worth outcome) on Autism Diagnostic Observation Schedule module 4 between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration | 
| Efficacy on autism spectrum core symptom assessed by revised algorithm of repetitive and restricted behavior score on the Autism Diagnostic Observation Schedule module 4 | Changes in revised algorithm of repetitive and restricted behavior score (range: 0-10, Higher value represent a worth outcome) on Autism Diagnostic Observation Schedule module 4 between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration | 
| Efficacy assessed by Clinical Global Impression-Improvement | Changes in Clinical Global Impression-Improvement (range: 1-7, Higher value represent a worse outcome) between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was assessed within 100 min after the last drug administration | 
| Efficacy assessed by Clinical Global Impression-Severity | Changes in Clinical Global Impression-Severity (range: 1-7, Higher value represent a worse outcome) between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was assessed within 100 min after the last drug administration | 
| Efficacy assessed by Global Assessment of Functioning | Changes in Global Assessment of Functioning (range: 1-100, Higher value represent a better outcome) between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was assessed within 100 min after the last drug administration | 
| Efficacy assessed by gaze fixation time on social region | Changes in gaze fixation time on social region during being talked between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 60 min after the last drug administration | 
| Efficacy assessed by quantitative analysis of facial expression | Changes in quantitative measure of facial expression on videos recorded during ADOS administration between baseline and endpoint of each administration period | At baseline, which was before and on the same day as the first administration, and at endpoint, which was started approximately 15 min after the last drug administration |