UMIN-CTR Clinical Trial

Basic information
Public title	Inherited Bone Marrow Failure Syndrome Registry
Acronym	IBMFS Registry
Scientific Title	Inherited Bone Marrow Failure Syndrome Registry
Scientific Title:Acronym	IBMFS Registry
Region	Japan

Condition
Condition	Inherited bone marrow failure syndrome
Classification by specialty	Hematology and clinical oncology Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To study the clinical course and clinical feature of inherited bone marrow failure syndrome.
Basic objectives2	Others
Basic objectives -Others	To investigate the effect and safety profile of therapeutic agents administered for inherited bone marrow failure syndrome.
Trial characteristics_1	Exploratory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	none
Key secondary outcomes

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	Patients diagnosed with inherited bone marrow failure syndrome.
Key exclusion criteria	Those who judged to be ineligibility in the study by researchers.
Target sample size	9999

Research contact person
Name of lead principal investigator	1st name Etsuro Middle name Last name Ito
Organization	Hirosaki University Graduate School of Medicine
Division name	Department of Community Medicine
Zip code	036-8562
Address	5 Zaifu-cho, Hirosaki, Aomori, Japan
TEL	0172-39-5070
Email	eturou@hirosaki-u.ac.jp

Public contact
Name of contact person	1st name Kiminori Middle name Last name Terui
Organization	Hirosaki University Graduate School of Medicine
Division name	Department of Pediatrics
Zip code	036-8562
Address	5 Zaifu-cho, Hirosaki, Aomori, Japan
TEL	0172-39-5070
Homepage URL
Email	teru@hirosaki-u.ac.jp

Sponsor
Institute	Hirosaki University Graduate School of Medicine
Institute
Department

Funding Source
Organization	Ministry of Health, Labor and Welfare
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Kyoto University Graduate School and Faculty of Medicine, Ethics Committee
Address	Yoshida-Konoe-cho, Sakyo-ku, Kyoto, Japan
Tel	075-753-4680
Email	ethcom@kuhp.kyoto-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	弘前大学医学部附属病院（青森県）

Other administrative information
Date of disclosure of the study information	2021 Year 01 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	Enrolling by invitation
Date of protocol fixation	2021 Year 01 Month 01 Day
Date of IRB	2020 Year 10 Month 01 Day
Anticipated trial start date	2021 Year 01 Month 01 Day
Last follow-up date	2025 Year 12 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information	This study intends to collect medical information on inherited bone marrow failure syndrome patients and build a patient registry.

Management information
Registered date	2020 Year 10 Month 12 Day
Last modified on	2020 Year 10 Month 12 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000046157