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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000024185
Receipt No. R000027842
Scientific Title Prospective non- control non- randomized, open-label study about efficacy of vitamin B6 for inherited GPI deficiency
Date of disclosure of the study information 2016/09/27
Last modified on 2020/04/22

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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/ctr_e/ctr_view.cgi?recptno=R000027842

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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