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

NCT04350606 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: B5411003ATGAM Japan local ph3 study
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 3

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.

Conditions

Aplastic Anemia

Keywords

Aplastic Anemia, globulin, ATG, Anti-human Thymocyte Immunogloblin

Outcome Measures

Primary Outcomes (1)

• 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.

  Week 12 Follow-up Visit

Secondary Outcomes (6)

• Number of Participants With Hematologic Response at Week 24

  Week 24 Follow-up Visit

• Absolute Neutrophil Count at Day 4, Weeks 1, 2, 4, 6, 8, 10, 12, 24

  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

  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

  Treatment: Day 4; Follow-up: Week 1, 2, 4, 6, 8, 10, 12, 24

• Number of Participants Who Survived During the Study

  Screening (up to 28 days prior to Day 1 of treatment) up to 24 weeks of follow-up (approximately up to 28 weeks)

Study Arms (1)

PF-006462700 group

EXPERIMENTAL

All enrolled participants will be administrated PF-006462700.

Biological: PF-06462700

Interventions

PF-06462700 BIOLOGICAL

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.

Also known as: Brand name in the US: ATGAM

PF-006462700 group

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female participants between the ages of 2 years and more, inclusive, at Visit
• (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.

You may not qualify if:

• 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
• (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).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (3)

Nagoya University Hospital

Nagoya, Aichi-ken, 466-8560, Japan

Location

Jichi Medical University Hospital

Shimotsuke, Tochigi, 329 0498, Japan

Location

Keio University Hospital

Shinjuku-ku, Tokyo, 160-8582, Japan

Location

Related Publications (1)

• Kanda Y, Mori T, Narita A, Wolter KD, Yoshimatsu H, Nishimura K. Efficacy and safety of equine anti-thymocyte immunoglobulin (eATG) in three Japanese patients with moderate to very severe aplastic anemia: a case series. Int J Hematol. 2023 Jan;117(1):37-43. doi: 10.1007/s12185-022-03496-5. Epub 2022 Nov 28.

  PMID: 36441357 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 1, 2020
• First Posted: April 17, 2020
• Study Start: July 25, 2020
• Primary Completion: January 22, 2021
• Study Completion: April 19, 2021
• Last Updated: April 27, 2022
• Results First Posted: April 27, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will share

Locations

JP (3)