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Registered date:18/03/2020

The efficacy and safety of pyridoxamine in patients with autism spectrum disorder; Exploratory physician-led Phase 2 trial

Basic Information

Recruitment status Completed
Health condition(s) or Problem(s) studiedAutism Spectrum Disorder
Date of first enrollment13/08/2018
Target sample size78
Countries of recruitmentJapan
Intervention(s)Intervention type:DRUG. Intervention1:Oral dose of high dose of pyridoxiamine, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:Pyridoxamine + vitamin B1 20 mg. Intervention2:Oral dose of low dose of pyridoxiamine, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:Pyridoxamine + vitamin B1 20 mg. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:Placebo + vitamin B1 20 mg.


Primary OutcomeAmounts of change from excitatory subscale from baseline of Abnormal Behavior Checklist Japanese Version(ABC-J). Timepoint:4weeks and 8 weeks after beginning of administration..
Secondary OutcomeABC-J total score and other sub scale score change from baseline, Response rate, Sensory profile, repeat behavior scal, Hyperacusis Questionnaire, clinical global impression-improvement, sensory investigation and symptom change questionnaire.. Timepoint: 4weeks and 8 weeks after beginning of administration.

Key inclusion & exclusion criteria

Age minimum>=12 YEARS
Age maximumNo Limit
Include criteria1. Patients diagnosed with autism spectrum discrder by DSM-5 2. Patients aged 12 years or older at the time of acquisition 3. Patients who have consented to participate in clinical trials from principal and substitute in the document (If the age at the time of consent acquisition is under 20 years old, consent of participation in clinical trial is obtaind from the substitute,regardless of ages, if the patient does not have the ability of agreement, written consent is obtaind from the substitute), and the patient who can visit the outpatient clinic with guardian at the designated time, a designated number of times 4. Patients who scored 18 or more excitatory subscale scores of Abnormal Behavior Checklist Japanese Version (ABC-J)
Exclude criteria1.Patients with a history of hypersensitivity to vitamin B6 2.Patients diagnosed with schizophrenia 3.Patient who is diagnosed with bipolar I disorder, bipolar II disorder, depression / major depressive disorder, and undergoing oral treatment for treatment 4. Patients with severe liver disease or liver injury (AST or ALT> 3 times the upper limit of the facility reference value) at screening 5.Patients with severe heart disease 6.Patients with impaired renal function (creatinine value> 2.0 mg / dL) at screening 7.Patients with gastrointestinal disorders, respiratory disorders, hematological disorders, endocrine disorders, immune disorders or other systemic disorders 8.In case of epilepsy complications, patients who had a epileptic seizure during the last 6 months until subject screening 9. At the beginning of study medication administration, patients using either of the following; vitamin B6, typical antipsychotics, anxiolytics, antihistamine drugs, etc. 10.During the trial period, patients using two or more sleep inducer 11.Patients who can not agree that the dosage regimen of the drugs shown below is in principle unchangeable during the trial period. Patients who do not agree to discontinue taking as needed or to take at a fixed dose when using consent at the time of taking medicine; atypical antipsychotics, sleep inducer, antiepileptic drugs, ADHD therapeutic agent, etc 12.Patients with malignant tumor coexisting at the time of subject screening or undergoing surgery for malignant tumor within 5 years before subject screening 13.Patient who wishes to become pregnant during pregnancy or lactation when subject screening 14.Patients participating in other trials or clinical studies at registration 15.Because of self-injurious behavior, suicidal ideation or other symptoms, the investigator or clinical trial doctor consider as high risks such as poor treatment compliance, failure to complete the trial, obstructing participation in the trial, affecting safety, etc. 16. Patients judged by Principal Investigator or Sub-investigator to be inappropriate for other reasons

Related Information


public contact
Name C-55 Clinical trial coordinating office
Address Department of Development Promotion,1-1 Seiryo-cho, Aoba-ku, Sendai, Miyagi Japan
Telephone +81-22-717-7136
Affiliation Clinical research, Innovation and Education Center, Tohoku University Hospital
scientific contact
Name Mitugu Uematsu
Address Department of Pediatrics,1-1 Seiryo-cho, Aoba-ku, Sendai, Miyagi Japan
Telephone +81-22-717-7287
Affiliation Tohoku University Hospital