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

Browse trials for

• Anemia 1,733
• Anemia, Aplastic 343

Keywords

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

Anemia

1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.

This study's enrollment of 3 is below the median of 94 across 1,291 interventional studies indexed under Anemia.

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Study arms

• Experimental
  PF-006462700 group

  All enrolled participants will be administrated PF-006462700.

  Biological: PF-06462700

Interventions

• BiologicalPF-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.

  Also known as: Brand name in the US: ATGAM

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.

   Time frame: Week 12 Follow-up Visit

Secondary outcomes

1. 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

2. 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

3. 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

4. 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

5. Number of Participants Who Survived During the Study

   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)

6. 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)

Baseline characteristics

Study documents

• Study protocol · Dec 24, 2019
• Statistical analysis plan · May 7, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — 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/data_requests.