Unique ID issued by UMIN | UMIN000020930 |
---|---|
Receipt number | R000024147 |
Scientific Title | Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations by High Precision Flow Cytometry |
Date of disclosure of the study information | 2016/02/09 |
Last modified on | 2023/03/02 10:41:52 |
Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations
by High Precision Flow Cytometry
Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations
by High Precision Flow Cytometry
Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations
by High Precision Flow Cytometry
Search for Unidentified Links between PNH Clone Size and the Related Clinical Manifestations
by High Precision Flow Cytometry
Japan |
BMF syndrome (PNH, AA, MDS) and suspected of PNH
Hematology and clinical oncology |
Others
NO
1. Describe the change in PNH-type cells, by type of peripheral PNH-type blood cells, in all enrolled patients over a 3-year period.
2. Identify predictive factors associated with changes in size of PNH-type cells and progression to clinical PNH, and factors associated with occurrence of PNH-type cells, in Japanese patients suspected of PNH or with bone marrow failure over a 3-year period, and categorize patients with PNH-type cells into patterns of clinical progression in PNH based on these results.
3. Determine the utility of monocyte PNH-type cells for characterization of PNH patients.
To achieve these objectives, the presence and proportion (clone size) of peripheral PNH-type blood cells (granulocytes, erythrocyte, and monocytes) will be determined by HPFC. These cytological changes will be analyzed together with other types of patient data (demography, medical history, concomitant medications and QoL) to identify and correlate "prognostic factors" for disease progression, and factors associated with disease occurrence.
Others
Observation study
Changes over time in PNH-type blood cells by blood cell lineage, in patients with PNH-type cells.
1. Frequency of patients in the study population with PNH-type cells and with clinical PNH (as defined by PNH-type cells <=1%) by type of peripheral PNH-type blood cell, over time.
2. Assessment of demographic characteristics, QoL, clinical signs and symptoms, immunosuppressive therapy (IST) use, IST efficacy, and aplastic anemia by severity, over time.
3. Assessment of PNH monocyte positivity in patients with PNH erythrocyte-positive and/or PNH granulocyte-positive status.
Observational
16 | years-old | <= |
Not applicable |
Male and Female
atients will fulfill either 1) or 2), and 3) below to be selected for the study:
1) Patients suspected of PNH as defined by patients presenting with PNH (diagnosed by non-High Precision Flow Cytometry methods), aplastic anemia, myelodysplastic syndrome, or other bone marrow failure syndrome. Undiagnosed bone marrow failure will be included.
2) Patients suspected of PNH as defined by patients presenting with signs such as Coombs test negative hemolytic anemia, hemoglobinuria, unexplained cytopenia, undiagnosed bone marrow failure, or unexplained thrombosis.
3) Patients aged 16 or older whose consent is obtained, including pediatric assent (verbal), and written consent of the patient and a legal guardian must be obtained.
A patient who fulfills any of the following is excluded:
1) Patients who developed acute leukemia, malignant lymphoma and/or other hematologic malignancies
2) Patients who are considered clinically ineligible for the study by the investigator
3) Patients who previously enrolled in this study
4) Patients who were registered for another clinical study and have not completed the observation period
5) Patients who are receiving medications for PNH treatment, including eculizumab, other complement-related inhibitors or other agents thought to modify the disease of PNH at the time of obtaining consent. Agents used for symptomatic therapy only (eg,steroids, anabolic hormones, iron, Vitamin B12), are allowed to be used during the study.
6) Patients currently treated with IST (eg, anti-thymocyte globulin (ATG), cyclosporine A)
2400
1st name | Shinji |
Middle name | |
Last name | Nakao |
Graduate school of Medical Sciences, Kanazawa University
Hematology
920-8641
13-1 Takaramachi, Kanazawa-shi, Ishikawa
076-265-2274
snakao8205@staff.kanazawa-u.ac.jp
1st name | Takanobu |
Middle name | |
Last name | Kimura |
Japan PNH Study Group
Research Support Office
103-0025
2-17-4 Nihonbashi Kayabacho, Chuo-ku, Tokyo
03-6810-9713
supremacy@jpsg.jp
Alexion Pharma Japan
Alexion Pharma Japan
Profit organization
Independent Ethics Committee
13-1 Takaramachi, Kanazawa-shi, Ishikawa
0776-265-2103
t-isomu@adm.kanazawa-u.ac.jp
NO
2016 | Year | 02 | Month | 09 | Day |
Unpublished
1985
Completed
2016 | Year | 01 | Month | 05 | Day |
2016 | Year | 02 | Month | 17 | Day |
2016 | Year | 04 | Month | 01 | Day |
2023 | Year | 02 | Month | 28 | Day |
Multicenter, prospective observation study. 3 year observation period if PNH clones.
2016 | Year | 02 | Month | 08 | Day |
2023 | Year | 03 | Month | 02 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000024147
Research Plan | |
---|---|
Registered date | File name |
Research case data specifications | |
---|---|
Registered date | File name |
Research case data | |
---|---|
Registered date | File name |