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

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000020930

Enrollment

2400

Registered

2016-02-09

Start date

2016-04-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

BMF syndrome (PNH, AA, MDS) and suspected of PNH

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 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.

Exclusion criteria

Exclusion criteria: 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)

Design outcomes

Primary

Measure	Time frame
Changes over time in PNH-type blood cells by blood cell lineage, in patients with PNH-type cells.	—

Secondary

Measure	Time frame
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.	—

Measure

Time frame

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.

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Countries

Japan

Contacts

Public ContactTakanobu Kimura

Japan PNH Study Group Research Support Office

supremacy@jpsg.jp03-6810-9713

Outcome results