UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000024185 |
|---|---|
| Receipt number | 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 20:10:30 |
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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
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
nabatames@ped.med.osaka-u.ac.jp
Sponsor or person
Institute
The Graduate School of Medicine, Osaka University
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027842
| Research Plan | |
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| Registered date | File name |
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