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

Phase 1

• Conditions: Moderate and above aplastic anemia; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-002155-11-Outside-EU/EEA
• Lead Sponsor: Pfizer Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-09
• Last Update Posted: 5 July 2021

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

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

-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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Secondary end point(s): - Hematologic response at week 24<br>-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)<br>-Survival Status<br>-Transfusion independence at weeks 12 and 14;Timepoint(s) of evaluation of this end point: - week 24<br>- weeks 1, 2, 4, 6, 8, 10, 12 and 24<br>- up to week 24<br>-week 12<br>- week 24