UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000030314 |
|---|---|
| Receipt number | R000033840 |
| Scientific Title | Novel replacement therapy for inherited GPI deficiency (IGD) |
| Date of disclosure of the study information | 2018/01/15 |
| Last modified on | 2024/01/27 15:37:29 |
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Basic information
Public title
Novel replacement therapy for inherited GPI deficiency (IGD)
Acronym
Novel replacement therapy for inherited GPI deficiency (IGD)
Scientific Title
Novel replacement therapy for inherited GPI deficiency (IGD)
Scientific Title:Acronym
Novel replacement therapy for inherited GPI deficiency (IGD)
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 Folinic acid for IGD.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
The frequency of seizures
Key secondary outcomes
Neurological findings
Developmental profile
MRI findings
EEG finding
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Folinic acid 1mg/kg/day in three divided dose to start. If the 5-MTHF concentration in cerebrospinal fluid will be low after one month, folinic acid will be uo to 2-5mg/kg/day.The period of administration is six months in principle.
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.
4) If he/she is enrolled in "Interventional study of vitamin B6 for inherited GPI deficiency", he/she can enroll in this study after 3 months of end of previous study.
Key exclusion criteria
1) The patients with the past history of hypersensitivity for the agent of Folinic acid.
2) The patients whose doctor in charge considered that the participation was not recommended.
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | Shin |
| Middle name | |
| Last name | Nabatame |
Organization
Graduate School of Medicine, Osaka University
Division name
Department of Pediatircs
Zip code
5650871
Address
2-2 Yamadaoka, Suita, Osaka 565-0871, Japan
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
Graduate School of Medicine, Osaka University
Division name
Department of Pediatircs
Zip code
565-0871
Address
2-2 Yamadaoka, Suita, Osaka 565-0871, Japan
TEL
06-6879-3932
Homepage URL
nabatames@ped.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University
Institute
Department
Personal name
Funding Source
Organization
MEXT KAKENHI Grant number JP15K09618
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
Japan Research Foundation for Clinical Pharmacology
IRB Contact (For public release)
Organization
Osaka University Clinical Research Review Committee
Address
2-2, Yamadaoka, Suita City, Osaka Pref., Osaka
Tel
06-6210-8296
handai-nintei@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(大阪府)、名古屋大学医学部附属病院(愛知県)、三重大学医学部附属病院(三重県)
Other administrative information
Date of disclosure of the study information
| 2018 | Year | 01 | Month | 15 | 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
Open public recruiting
Date of protocol fixation
| 2017 | Year | 12 | Month | 08 | Day |
Date of IRB
| 2019 | Year | 02 | Month | 20 | Day |
Anticipated trial start date
| 2019 | Year | 04 | Month | 01 | Day |
Last follow-up date
| 2024 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2017 | Year | 12 | Month | 08 | Day |
Last modified on
| 2024 | Year | 01 | Month | 27 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000033840
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