UMIN-CTR Clinical Trial

Basic information
Public title	Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH
Acronym	Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH
Scientific Title	Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH
Scientific Title:Acronym	Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH
Region	Japan

Condition
Condition	BMF syndrome (AA, MDS, PNH)
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To determine the prevalence of patients bearing increased PNH-type granulocytes and erythrocytes in patients with BMF syndrome and in those suspected of having PNH, their peripheral blood samples are examined using high precision flow cytometry method.
Basic objectives2	Others
Basic objectives -Others	Observation study
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Proportion of patients showing PNH-type granulocytes and erythrocytes greater than or equal to 1% at the first FCM analysis
Key secondary outcomes	1.Proportion of patients showing PNH-type granulocytes greater than or equal to 0.003% and erythrocytes greater than or equal to 0.005% identified at the first FCM analysis 2.Changes in percentage of PNH-type cells in the patients who were found to be positive at the first FCM analysis (granulocytes greater than or equal to 0.003% and erythrocytes greater than or equal to 0.005%) 3.Relationship between response to IST made after the PNH-type cell analysis and increase or decrease of PNH-type cells

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	16 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1.BMF syndrome (AA, MDS, PNH) 2.Suspected of having PNH 3.Patients aged over 16 years and signed a written informed consents by themselves or legal guardians
Key exclusion criteria	1.Patients with malignancies requiring 2.Patients considered inappropriate to participate in the study 3.Patients who has been registered with this study 4.Patients who have used Eculizumab within at the informed consents
Target sample size	1000

Research contact person
Name of lead principal investigator	1st name Shinji Middle name Last name Nakao
Organization	Graduate school of Medical Sciences, Kanazawa University
Division name	Hematology/Respiratory medicine
Zip code	920-8641
Address	13-1 Takaramachi, Kanazawa-shi, Ishikawa
TEL	076-265-2274
Email	snakao8205@staff.kanazawa-u.ac.jp

Public contact
Name of contact person	1st name Takanobu Middle name Last name Kimura
Organization	Japan PNH Study Group
Division name	Research Support Office
Zip code	103-0025
Address	2-17-4 Nihonbashi Kayabacho, Chuo-ku, Tokyo
TEL	03-6810-9713
Homepage URL
Email	tkimura@jpsg.jp

Sponsor
Institute	Japan PNH Study Group
Institute
Department

Funding Source
Organization	Alexion Pharma Japan
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Independent Ethics Committee
Address	13-1 Takaramachi, Kanazawa-shi, Ishikawa
Tel	0776-265-2103
Email	t-isomu@adm.kanazawa-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

Other administrative information
Date of disclosure of the study information	2015 Year 10 Month 05 Day

Related information
URL releasing protocol
Publication of results	Partially published

Result
URL related to results and publications
Number of participants that the trial has enrolled	2956
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	2011 Year 02 Month 24 Day
Date of IRB	2011 Year 03 Month 23 Day
Anticipated trial start date	2011 Year 04 Month 01 Day
Last follow-up date	2019 Year 06 Month 30 Day
Date of closure to data entry	2019 Year 06 Month 30 Day
Date trial data considered complete	2019 Year 12 Month 31 Day
Date analysis concluded	2020 Year 03 Month 31 Day

Other
Other related information	Multicenter, prospective observation study. 3 year observation period if PNH clones are positive.

Management information
Registered date	2015 Year 10 Month 04 Day
Last modified on	2019 Year 07 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022206