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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000008235
Receipt No. 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

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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
Email 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
Email onodera-m@ncchd.go.jp

Sponsor
Institute National Center for Child Heath and Development
Institute
Department

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
Email

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
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000009708

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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