ABOUT THIS STUDY
- Male or female participants between the ages of 2 years and more, inclusive, at Visit
- Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
- Have a clinical diagnosis of aplastic anemia by bone marrow aspiration/biopsy findings and/or magnetic resonance imaging (MRI) etc.
- Must meet the following criteria of moderate and above aplastic anemia
- Capable of giving signed informed consent/assent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the informed consent document (ICD)/assent document and in this protocol.
- Eligible and willing to have a sibling allogeneic stem cell transplantation.
- Evidence of a myelodysplastic syndrome (except for refractory cytopenia in children),
as well as other primitive marrow disease.
- History or clinical suspicion of congenital aplastic anemia (Fanconi anemia,
Congenital keratosis, etc).
- History of malignant tumors with active disease within 5 years from study
- Participants who are clearly infected with hepatitis B virus (HBV), hepatitis C virus
(HCV), human immunodeficiency virus (HIV), and human T-cell leukemia virus type
- Pregnant or breast-feeding participants.
- Participants with severe hepatic, renal or cardiac failure, or any other
life-threatening concurrent [aspartate aminotransferase (AST) or alanine
aminotransferase (ALT) or total bilirubin values >5 × upper limit of normal (ULN),
and/or creatinine value >2 × ULN].
- Participants with hypersensitivity such as shock after skin test of this study drug.
- Participants with uncontrolled severe infection (pneumonia, sepsis, etc).
- Participants who received live vaccine or live attenuated vaccine within 6 weeks prior
to the first dose of study drug.
- Other acute or chronic medical or psychiatric condition including recent (within the
past year) or active suicidal ideation or behavior or laboratory abnormality that may
increase the risk associated with study participation or investigational product
administration or may interfere with the interpretation of study results and, in the
judgment of the investigator, would make the participant inappropriate for entry into
- Prior immunosuppressive therapy with lymphocyte-depleting agents/therapies, including
both non-B-cell selective and B-cell-depleting agents (e.g., alefacept, alemtuzumab,
rituximab). However, participants previously treated with rATG may enroll.
- Previous history of stem cell transplantation.
- Previous administration with an investigational drug within 30 days (or as determined
by the local requirement) or 5 half-lives preceding the first dose of investigational
product used in this study (whichever is longer).
- Baseline 12-lead electrocardiogram (ECG) that demonstrates clinically relevant
abnormalities that may affect participant safety or interpretation of study results
(e.g., baseline corrected QT [QTc] interval >450 msec, complete left bundle branch
block [LBBB], signs of an acute or indeterminate-age myocardial infarction, ST-T
interval changes suggestive of myocardial ischemia, second- or third-degree
atrioventricular [AV] block, or serious bradyarrhythmias or tachyarrhythmias). If the
baseline uncorrected QT interval is >450 msec, this interval should be rate-corrected
using the Fridericia method and the resulting QTcF should be used for decision making
and reporting. If QTc exceeds 450 msec, or QRS exceeds 120 msec, the ECG should be
repeated 2 more times and the average of the 3 QTc or QRS values should be used to
determine the participant's eligibility. Computer-interpreted ECGs should be overread
by a physician experienced in reading ECGs before excluding participants.
- Investigator site staff members directly involved in the conduct of the study and
their family members, site staff members otherwise supervised by the investigator, or
Pfizer employees, including their family members, directly involved in the conduct of
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