Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

What Was Tested

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

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

Weekly Subcutaneous (SC) administration.

The registry classes the interventions as biological.

Who Took Part

Enrollment: 46 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: Diagnosis of Aplastic Anemia (AA) confirmed by peripheral blood and bone-marrow examinations, etc. Refractory to at least one course of immunosuppressive therapy including horse or rabbit anti-human thymocyte immunoglobulin (ATG); or ineligible for ATG treatment and refractory to cyclosporin (CyA). Thrombocytopenia defined as a platelet count of ≤ 30 × 10\^9/L. Preserving main organ function as a result of screening as follows;. Total bilirubin: \< 1.5 times the upper limit of the laboratory normal range.

The full record lists 11 inclusion and 21 exclusion criteria; among the exclusions: concurrent active infection not adequately responding to appropriate therapy; bone marrow reticulin grade of ≥ 2 based on the grading scale for reticulin indicated in bone marrow pathology (2nd edition); arterial or venous thrombosis within one year before enrollment.

How the Study Was Run

The set-up was single-group and open-label.

Everyone knew when Romiplostim was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2016-05 with completion listed for 2017-11-14.

It ran at 3 sites in Japan (2) and South Korea (1).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27.

It also tracked 4 secondary outcomes, for example Proportion of subjects with a hematological response at the end-of- treatment examination, Time from the first romiplostim administration to hematological response and Proportion of subjects achieving platelet response, erythroid response, or neutrophil response at each of Week 27 and end of treatment.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3 trial of Romiplostim in 46 people with anemia.

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.