A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia
Condition: Aplastic Anemia
MeSH terms: Anemia; Anemia, Aplastic
Intervention: PF-06462700
Intervention description: 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.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 3.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Aplastic Anemia, globulin, ATG, Anti-human Thymocyte Immunogloblin

Summary:

      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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female participants between the ages of 2 years and more, inclusive, at Visit

             1 (Screening).

          -  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.

        Exclusion Criteria:

          -  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
             participation.

          -  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

             1 (HTLV-1).

          -  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
             this study.

          -  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
             the study.