UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000008235 |
|---|---|
| Receipt number | R000009708 |
| Scientific Title | A gene therapy clinical trial for chronic granulomatous disease targeting the patient's hematopoietic stem cells |
| Date of disclosure of the study information | 2012/07/01 |
| Last modified on | 2014/01/22 13:36:30 |
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Basic information
Public title
A gene therapy clinical trial for chronic granulomatous disease targeting the patient's hematopoietic stem cells
Acronym
Gene therapy for chronic granulomatous disease
Scientific Title
A gene therapy clinical trial for chronic granulomatous disease targeting the patient's hematopoietic stem cells
Scientific Title:Acronym
Gene therapy for chronic granulomatous disease
Region
| Japan |
Condition
Condition
chronic granulomatous disease
Classification by specialty
| Medicine in general | Clinical immunology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
In this trial, patients with chronic granulomatous disease who have serious infections are treated with genetically collected their own hematopoietic stem cells using retroviral vector expressing the CYBB gene
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Pragmatic
Developmental phase
Phase I,II
Assessment
Primary outcomes
cure or mitigation of severe infections
Key secondary outcomes
a long-term protective effect on severe infections
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Gene |
Interventions/Control_1
infusion of the genetically collected cells
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
| 3 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male
Key inclusion criteria
chronic granulomatous disease
no choice for stem cell transplantation
severe infections
Key exclusion criteria
Severe cardiorespiratory failure
positive for HIV
severe allegy
no anticipation for long-term live survival
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | MASAFUMI ONODOERA |
Organization
National Center for Child Heath and Development
Division name
Department of Human Genetics
Zip code
Address
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL
03-5494-7295
onodera-m@ncchd.go.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | MASAFUMI ONODOERA |
Organization
National Center for Child Heath and Development
Division name
Department of Human Genetics
Zip code
Address
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL
03-5494-7295
Homepage URL
http://111.89.135.117/genetics/index.html
onodera-m@ncchd.go.jp
Sponsor or person
Institute
National Center for Child Heath and Development
Institute
Department
Personal name
Funding Source
Organization
Grant of MHLW
Organization
Division
Category of Funding Organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
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
Other administrative information
Date of disclosure of the study information
| 2012 | Year | 07 | Month | 01 | 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
| 2012 | Year | 07 | Month | 01 | Day |
Date of IRB
Anticipated trial start date
| 2012 | Year | 07 | Month | 01 | Day |
Last follow-up date
| 2022 | Year | 07 | Month | 01 | Day |
Date of closure to data entry
| 2022 | Year | 07 | Month | 01 | Day |
Date trial data considered complete
| 2022 | Year | 07 | Month | 01 | Day |
Date analysis concluded
| 2022 | Year | 07 | Month | 01 | Day |
Other
Other related information
Management information
Registered date
| 2012 | Year | 06 | Month | 22 | Day |
Last modified on
| 2014 | Year | 01 | Month | 22 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000009708
| Research Plan | |
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| Registered date | File name |
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| Registered date | File name |
| Research case data | |
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