The purpose of the study is to assess the efficacy and safety of PF-06462700 administered intravenously at 40 mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia for making an approval application in Japan.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
PF-06462700 is classified as an immunosuppressant/ immunosuppressive agent. It is the purified, concentrated, and sterile gamma globulin, primarily monomeric immunoglobulin G (IgG), from hyperimmune serum of horses that are immunized with human thymus lymphocytes.
Nagoya University Hospital
Nagoya, Aichi-ken, Japan
Jichi Medical University Hospital
Shimotsuke, Tochigi, Japan
Keio University Hospital
Shinjuku-ku, Tokyo, Japan
Number of Participants With Hematologic Response at Week 12
Hematologic response was considered to be "effective" when 2 or more of the following criteria were met: absolute neutrophil count greater than or equal to (\>=) 500 per microliters, platelet count \>=20,000 per microliters and reticulocyte count \>=60,000 per microliters was observed. In this outcome measure, number of participants with hematologic response classified as effective and not effective were reported. Improvement in counts that were dependent upon exogenously administered growth factors or transfusion, was not considered as fulfilling response criteria.
Time frame: Week 12 Follow-up Visit
Number of Participants With Hematologic Response at Week 24
Hematologic response was considered to be "effective" when 2 or more of the following criteria were met: absolute neutrophil count \>=500 per microliters, platelet count \>=20,000 per microliters and reticulocyte count \>=60,000 per microliters was observed. In this outcome measure, number of participants with hematologic response classified as effective and not effective were reported. Improvement in counts that were dependent upon exogenously administered growth factors or transfusion, was not been considered as fulfilling response criteria.
Time frame: Week 24 Follow-up Visit
Absolute Neutrophil Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24
Time frame: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
Platelet Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24
Time frame: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
Reticulocyte Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24
Time frame: Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24
Number of Participants Who Survived During the Study
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In this outcome measure, number of participants who survived during the study were observed.
Time frame: Screening (up to 28 days prior to Day 1 of treatment) up to 24 weeks of follow-up (approximately up to 28 weeks)
Number of Participants With Transfusion Independence at Weeks 12 and 24
Transfusion independence at Week 12 was defined as when participants did not have any transfusion records from the time of the first dose of the investigational product at Day 1 to the day of Week 12 follow-up visit (inclusive). Transfusion independence at Week 24 was defined as when participants did not have any transfusion records from the day after Week 12 follow-up visit to the day of Week 24 follow-up visit (inclusive).
Time frame: Week 12 Transfusion Independence: Day 1 of Treatment up to Week 12 Follow-up Visit (approximately 12 weeks); Week 24 Transfusion Independence: Day after Week 12 Follow-up visit to Week 24 Follow-up Visit (approximately 12 weeks)