UMIN-CTR Clinical Trial

Basic information
Public title	Analysis of allogenic stem cell transplantation for primary immunodeficiency using TRUMP data
Acronym	Analysis of allo-HCT for PIDs in Japan
Scientific Title	Analysis of allogenic stem cell transplantation for primary immunodeficiency using TRUMP data
Scientific Title:Acronym	Analysis of allo-HCT for PIDs in Japan
Region	Japan

Condition
Condition	Primary immunodeficiency
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	In order to improve hematopoietic cell transplantation (HCT) for primary immunodeficiencies (PIDs), we clarify the significance, efficacy and the risks of HCT, optimal donor selection, the choice of conditioning regimens, and management guidelines for adverse events by analyzing the data of patients with PIDs who have received HCT in Japan using PIDJ and TRUMP data.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	Overall survival/ event-free survival in patients with PID after HCT
Key secondary outcomes	We divide the patients into following subgroups; different periods of years, disease categories according to International Union of Immunological Societies; IUIS classification, pre-HCT status of complications; infection, autoimmune disease or autoinflammatory diseases, conditioning regimen; myeloablative, reduced intensity or nothing, and donor sources. We analyze survival, adverse events and long-term immunological reconstitution (i.e. post-transplant chimerism and secession of immunoglobulin supplementation) in each subgroup. In addition, we identify the factors contributing to survival by multivariate analysis, as well as the risks for post-HCT adverse events such as graft-versus host disease, infection or the late complications.

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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	The patients with PID in TRUMP registration who have received HCT from January 1974 to December 2016
Key exclusion criteria	The patients whose TRUMP data are insufficient for analysis.
Target sample size	800

Research contact person
Name of lead principal investigator	1st name Middle name Last name Tomohiro Morio
Organization	Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University
Division name	Department of Pediatrics and Developmental Biology
Zip code
Address	Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan
TEL	03-5803-5249
Email	tmorio.ped@tmd.ac.jp

Public contact
Name of contact person	1st name Middle name Last name Satoshi Miyamoto
Organization	Tokyo Medical and Dental University
Division name	Department of Pediatrics
Zip code
Address	Yushima 1-5-45, Bunkyo-ku, Tokyo, Japan
TEL	03-5803-5249
Homepage URL
Email	smiyped@tmd.ac.jp

Sponsor
Institute	Tokyo Medical and Dental University
Institute
Department

Funding Source
Organization	Ministry of Health, Labor and Welfare
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

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	2018 Year 12 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	Completed
Date of protocol fixation	2018 Year 07 Month 24 Day
Date of IRB	2018 Year 07 Month 24 Day
Anticipated trial start date	2018 Year 07 Month 24 Day
Last follow-up date	2018 Year 07 Month 24 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information	Observational study

Management information
Registered date	2018 Year 08 Month 27 Day
Last modified on	2022 Year 01 Month 19 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037635