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Open-label, single-arm, multicenter study in Japanese patients with aplastic anemia

A MULTICENTER, OPEN-LABEL, SINGLE-ARM STUDY TO ACCESS 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

Status
Unknown
Phases
Phase 3
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2021-002155-11-Outside-EU/EEA
Enrollment
Unknown
Registered
2021-06-09
Start date
Unknown
Completion date
Unknown
Last updated
2021-07-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Moderate and above aplastic anemia

Interventions

Trade Name: Atgam Product Name: Atgam Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Anti-human T-lymphocyte immunoglobulin from horse Other descriptive name: anti-hum

Sponsors

Pfizer Inc.
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: -Male or female participants between the ages of 2 years and more inclusive at visit 1 (screening) -Have a clinical diagnosis of aplastic anemia by bone marrow aspiration/biopsy findings and/or magnetic resonance imaging (MRI) etc. -Must meet the criteria of moderate and above aplastic anemia (stage 2b and above) Are the trial subjects under 18? yes Number of subjects for this age range: 1 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 2 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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 of clinical suspicion of congenital aplastic anemia (Fanconi anemia, Congenital keratosis, etc). -Previous history of stem cell transplantation - Prior immunosuppressive therapy with lymphocyte-depleting agents/therapies, including both non-B-cell selective and B-cell-depleting agents. However, participants previously treated with rATG may enroll.

Design outcomes

Primary

MeasureTime frame
Main Objective: To investigate the efficacy of PF-06462700 administered intravenously at 40mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia;Secondary Objective: To investigate the efficacy of PF-06462700 administered intravenously at 40mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia;Primary end point(s): Hematologic response at week 12;Timepoint(s) of evaluation of this end point: week 12—

Secondary

MeasureTime frame
Secondary end point(s): - Hematologic response at week 24 -Hematological test values at day 4 and at weeks 1, 2, 4, 6, 8, 10, 12 and 24 (Absolute neutrophil count, Platelet count, Reticulocyte count) -Survival Status -Transfusion independence at weeks 12 and 14;Timepoint(s) of evaluation of this end point: - week 24 - weeks 1, 2, 4, 6, 8, 10, 12 and 24 - up to week 24 -week 12 - week 24—

Countries

Japan

Contacts

Public ContactClinical Trials.gov Call Center

Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com8001718-1021

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026