Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

What Was Tested

This registered phase 2, phase 3 trial tested Romiplostim for anemia.

To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.

It had 1 arm (groups): AMG531 (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 17 actual participants took part.

It was open to people of any sex aged from 20 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Voluntary signed informed consent to participate in the study;. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;. Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening.

The full record lists 4 inclusion and 20 exclusion criteria; among the exclusions: previously treated with atg, csa, or alemtuzumab;; diagnosed as having congenital aa (fanconi anemia, congenital dyskeratosis, etc.);; diagnosed as having aml or chronic myelomonocytic leukemia;.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the Romiplostim study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2019-04-25 with completion listed for 2020-11-05.

It ran at 1 site in Japan.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Achievement of complete response (CR) or partial response (PR).

It also tracked 8 secondary outcomes, for example Achievement of CR or PR, Achievement of CR, The time to CR or PR, Change from baseline in platelet count (/µL), Change from baseline in hemoglobin (Hb) concentration (g/dL) and Change from baseline in neutrophil count (/µL).

What This Means in Plain Language

Put simply, this is a phase 2, phase 3 trial of Romiplostim in 17 people with anemia.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, Anemia research.