UMIN-CTR Clinical Trial

Basic information
Public title	Prospective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiency
Acronym	Interventional study of vitamin B6 for inherited GPI deficiency
Scientific Title	Prospective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiency
Scientific Title:Acronym	Interventional study of vitamin B6 for inherited GPI deficiency
Region	Japan

Condition
Condition	Inherited GPI deficiency
Classification by specialty	Neurology Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To examine the safety and efficacy of pyridoxine for inherited GPI deficiency
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	The frequency of seizures during a year after the initiation of the administration Developmental profile during a year after the initiation of the administration
Key secondary outcomes	MRI finding during a year after the initiation of the administration EEG finding during a year after the initiation of the administration

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification	NO
Dynamic allocation	NO
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Initial dose: pyridoxine 10-15 mg/kg/day 1-2 weeks after the initiation, the dose is increased to maintenance dose, if there is no adverse effect. Maintenance dose: pyridoxine 20-30 mg/kg/day We prescribe pyridoxine for a year.
Interventions/Control_2
Interventions/Control_3
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	Not applicable
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) The patients of inherited GPI deficiency diagnosed by FACS analysis of granulocyte or genetic analysis. 2) The patient or his/her guardians approved with the participation in the study after the explanation with informed consent form. 3) There is no restriction about severity or age, if the patient is tolerable for evaluation.
Key exclusion criteria	1) The patients with the past history of hypersensitivity for the agent of vitamin B6. 2) The patients whose doctor in charge considered that the participation was not recommended.
Target sample size	15

Research contact person
Name of lead principal investigator	1st name Shin Middle name Last name Nabatame, M.D
Organization	Graduate School of Medicine, Osaka University
Division name	Department of Pediatrics
Zip code	565-0871
Address	2-2, Yamadaoka, Suita, Osaka, 565-0871
TEL	(06)6879-3932
Email	nabatames@ped.med.osaka-u.ac.jp

Public contact
Name of contact person	1st name Shin Middle name Last name Nabatame
Organization	Osaka University Hospital
Division name	Department of Pediatrics
Zip code	565-0871
Address	2-15, Yamadaoka, Suita, Osaka, 565-0871
TEL	(06)6879-3932
Homepage URL
Email	nabatames@ped.med.osaka-u.ac.jp

Sponsor
Institute	The Graduate School of Medicine, Osaka University
Institute
Department

Funding Source
Organization	Ministry of Education, Culture, Sports, Science and Technology - Japan
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor	Department of Child Neurology, Tottori University Hospital
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Osaka University Hospital
Address	2-15, Yamadaoka, Suita, Osaka, 565-0871
Tel	06-6879-5111
Email	rinri@hp-crc.med.osaka-u.ac.jp

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	大阪大学医学部附属病院

Other administrative information
Date of disclosure of the study information	2016 Year 09 Month 27 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	9
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	Completed
Date of protocol fixation	2014 Year 07 Month 31 Day
Date of IRB	2014 Year 10 Month 09 Day
Anticipated trial start date	2014 Year 07 Month 31 Day
Last follow-up date	2020 Year 04 Month 22 Day
Date of closure to data entry	2020 Year 04 Month 22 Day
Date trial data considered complete	2020 Year 04 Month 22 Day
Date analysis concluded	2020 Year 04 Month 22 Day

Other
Other related information

Management information
Registered date	2016 Year 09 Month 27 Day
Last modified on	2020 Year 04 Month 22 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000027842