UMIN-CTR Clinical Trial

Basic information
Public title	The efficacy and safety of pyridoxamine in patients with autism spectrum disorder; Exploratory physician-led Phase II trial
Acronym	Pyridoxamine for ASD trial
Scientific Title	The efficacy and safety of pyridoxamine in patients with autism spectrum disorder; Exploratory physician-led Phase II trial
Scientific Title:Acronym	Pyridoxamine for ASD trial
Region	Japan

Condition
Condition	Autism spectrum disorder
Classification by specialty	Pediatrics Psychiatry
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Exploritively evaluate the efficacy and safety of pyridoxamine for patients with autism spectrum disorder and determine the appropriate target patient population, dosage regimen, and evaluation index
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase II

Assessment
Primary outcomes	Amount of change from excitatory subscale score from baseline of Abnormal Behavior Checklist Japanese Version (ABC-J)
Key secondary outcomes	*ABC-J total score, other subscale score change from baseline *Sensory profile, repeat behavior scale, Hyperacusis Questionnaire, clinical global impression - improvement, sensory investigation and symptom change questionnaire

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Double blind -all involved are blinded
Control	Placebo
Stratification	YES
Dynamic allocation	YES
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment	Central registration

Intervention
No. of arms	3
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Placebo
Interventions/Control_2	Oral doses of low dose pyridoxamine divided into twice a day for 8 weeks 12-19 year;800mg 20 years or older;1200mg
Interventions/Control_3	Oral doses of high dose pyridoxamine divided into twice a day for 8 weeks 12-19 years;1200mg 20 years or older;2000mg
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	12 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1. 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)
Key exclusion criteria	1.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,ADHD therapeutic agent, 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, 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.
Target sample size	72

Research contact person
Name of lead principal investigator	1st name Shigeo Middle name Last name Kure
Organization	Tohoku University Hospital
Division name	Department of Pediatrics
Zip code	9808574
Address	1-1 Seiryo-cho Aoba-ku Sendai-shi Miyagi-ken Japan
TEL	022-717-7287
Email	kure@med.tohoku.ac.jp

Public contact
Name of contact person	1st name Hanai Middle name Last name Takuto
Organization	C-55 Clinical trial coordinating office
Division name	Clinical Research Innovation and Education Center
Zip code	9808574
Address	1-1 Seiryo-cho Aoba-ku Sendai-shi Miyagi-ken Japan
TEL	022-717-7136
Homepage URL
Email	c55chiken@crieto.hosp.tohoku.ac.jp

Sponsor
Institute	Department of Pediatrics,Tohoku University Hospital
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Non
Address	Non
Tel	Non
Email	Non

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	東北大学病院（宮城県）、自治医科大学附属病院（栃木県）、国立成育医療研究センター（東京都）、大阪市立総合医療センター（大阪府）、神戸大学医学部付属病院（兵庫県）、宮城県立こども病院（宮城県）、弘前大学病院精神科（青森県）、山形大学医学部付属病院（山形市）、国立精神・神経医療研究センター（東京都）

Other administrative information
Date of disclosure of the study information	2018 Year 12 Month 08 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	No longer recruiting
Date of protocol fixation	2018 Year 07 Month 31 Day
Date of IRB	2020 Year 09 Month 30 Day
Anticipated trial start date	2018 Year 08 Month 13 Day
Last follow-up date	2020 Year 11 Month 30 Day
Date of closure to data entry
Date trial data considered complete	2021 Year 03 Month 31 Day
Date analysis concluded	2021 Year 05 Month 31 Day

Other
Other related information

Management information
Registered date	2018 Year 12 Month 07 Day
Last modified on	2021 Year 06 Month 10 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000040082