A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia

NCT04350606

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Aplastic Anemia
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
2 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- 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.

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Advanced Information
Descriptive Information
Brief Title  ICMJE A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia
Official Title  ICMJE 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Aplastic Anemia
Intervention  ICMJE Biological: PF-06462700
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.
Other Name: Brand name in the US: ATGAM
Study Arms  ICMJE Experimental: PF-006462700 group
All enrolled participants will be administrated PF-006462700.
Intervention: Biological: PF-06462700
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: April 14, 2020)
3
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 30, 2021
Estimated Primary Completion Date January 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04350606
Other Study ID Numbers  ICMJE B5411003
ATGAM Japan local ph3 study ( Other Identifier: Alias Study Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP