Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH

Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH - Observation of GPI-anchored protein-deficient (PNH-Type) cells In Japanese patients with bone MArrow failure syndrome and in those suspected of having PNH

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000019227

Enrollment

1000

Registered

2015-10-05

Start date

2011-04-01

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 (AA, MDS, PNH)

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Proportion of patients showing PNH-type granulocytes and erythrocytes greater than or equal to 1% at the first FCM analysis	—

Secondary

Measure	Time frame
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	—

Measure

Time frame

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

—

Countries

Japan

Contacts

Public ContactTakanobu Kimura

Japan PNH Study Group Research Support Office

tkimura@jpsg.jp03-6810-9713

Outcome results