Brief Title
A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia
Official Title
A MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY 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
Brief 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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of Participants With Hematologic Response at Week 12
Secondary Outcome
Number of Participants With Hematologic Response at Week 24
Condition
Aplastic Anemia
Intervention
PF-06462700
Study Arms / Comparison Groups
PF-006462700 group
Description: All enrolled participants will be administrated PF-006462700.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
3
Start Date
July 25, 2020
Completion Date
April 19, 2021
Primary Completion Date
January 22, 2021
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.
Gender
All
Ages
2 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Pfizer CT.gov Call Center, ,
Location Countries
Japan
Location Countries
Japan
Administrative Informations
NCT ID
NCT04350606
Organization ID
B5411003
Secondary IDs
ATGAM Japan local ph3 study
Responsible Party
Sponsor
Study Sponsor
Pfizer
Study Sponsor
Pfizer CT.gov Call Center, Study Director, Pfizer
Verification Date
March 2022