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

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.

Timeline

Start

2020-07-25

Primary completion

2021-01-22

Completion

2021-04-19

Outcome

Met primary endpoint

paper Two participants met the primary outcome of hematologic response at week 12 and improvements in disease severity were observed. PMID 36441357 ↗

Publications

• 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.

Drugs

Indications