UMIN-CTR Clinical Trial

Public title

Detection of markers for immune pathophysiology and analysis of their
prognostic value in patients with chronic thrombocytopenia without an increase in the number of bone marrow megakaryocytes: a prospective observation study.

Acronym

Detection of markers for immune pathophysiology and analysis of their
prognostic value in patients with chronic thrombocytopenia without an increase in the number of bone marrow megakaryocytes.

Scientific Title

Detection of markers for immune pathophysiology and analysis of their
prognostic value in patients with chronic thrombocytopenia without an increase in the number of bone marrow megakaryocytes: a prospective observation study.

Scientific Title:Acronym

Detection of markers for immune pathophysiology and analysis of their
prognostic value in patients with chronic thrombocytopenia without an increase in the number of bone marrow megakaryocytes.

Region

Japan

Condition

Pancytopenia, thrombocytopenia

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Others

Genomic information

NO

Narrative objectives1

We sometimes experience the patients, who show low platelets count but not reach a diagnosis as Idiopathic Thrombocytopenic Purpura (ITP) because of no increment of immature platelets fraction (IPF) in peripheral blood (PB) and megakaryocyte in bone marrow (BM).
Meanwhile, aplastic anemia (AA) is typified by pancytopenia, but some patients decrease in platelets prior to pancytopenia during the course.
Thus, we conduct an prospective study to investigate the frequency and the clinical course of the patients in 'thrombocytopenia with decreased megakaryocytes in BM (TDM)', frequency of PNH+ patients in TDM population, and revealing the response of TDM patients for IST.

Basic objectives2

Others

Basic objectives -Others

Observation study.

Trial characteristics_1

Confirmatory

Trial characteristics_2

Pragmatic

Developmental phase

Not applicable

Primary outcomes

Survival of the 'TDM' patients, maintaining the number of platelets greater than 5.0x10e4/mcl.

Key secondary outcomes

1) Changes of the severity of the diseases graded by the clinical stratification for aplastic anemia.
2) Frequency of patients, positive for PNH-type cells.
3) The choice of treatment selected by attending physician.

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

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

Age-lower limit

Not applicable

Age-upper limit

80

years-old

>=

Gender

Male and Female

Key inclusion criteria

No increment of the number of bone marrow megakaryocytes and peripheral blood examination meets the condition as follows; WBC >= 3000 /mcl
Hb >= 10 g/dl
Platelets < 10 x10e4/mcl

Key exclusion criteria

1) Patients treated by immunosuppressive therapy.
2) Patients with a history of hematologic malignancy.
3) Patients with malignancies requiring treatment.

Target sample size

50

Name of lead principal investigator

1st name
Middle name
Last name	Shinji Nakao

Organization

Kanazawa University Graduate School of Medical Science.

Division name

Cellular Transplantation Biology

Zip code

Address

13-1, Takaramachi, Kanazawa, Ishikawa 920-8641, Japan

TEL

Email

Name of contact person

1st name
Middle name
Last name	Ken Ishiyama

Organization

Kanazawa University Graduate School of Medical Science.

Division name

Cellular Transplantation Biology

Zip code

Address

13-1, Takaramachi, Kanazawa, Ishikawa 920-8641, Japan

TEL

81-76-265-2274

Homepage URL

Email

ishiyamak@med3.m.kanazawa-u.ac.jp

Institute

Cellular Transplantation Biology, Kanazawa University Graduate School of Medical Science.

Institute

Department

Personal name

Organization

Research Committee for the Idiopathic Hematopoietic Disorders, Ministry of Health, Labor, and Welfare, Japan.

Organization

Division

Category of Funding Organization

Nationality of Funding Organization

Co-sponsor

Name of secondary funder(s)

Organization

Address

Tel

Email

Secondary IDs

YES

Study ID_1

796

Org. issuing International ID_1

Medical Ethics Committee of Kanazawa University

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

金沢大学附属病院

Date of disclosure of the study information

2009

Year

11

Month

16

Day

URL releasing protocol

Publication of results

Unpublished

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

Recruitment status

Open public recruiting

Date of protocol fixation

2009

Year

10

Month

01

Day

Date of IRB

Anticipated trial start date

2009

Year

12

Month

01

Day

Last follow-up date

2011

Year

03

Month

01

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Multicenter, prospective observation study.
Register the patients who meet registration criteria, and follow their clinical course for 5 years.

Registered date

2009

Year

11

Month

09

Day

Last modified on

2010

Year

02

Month

16

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000003318