An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura

What Was Tested

The paper reports a study of Romiplostim, published as a phase 2 clinical trial.

Abstract The objective of this open label, phase 1-2, multicentre trial was to evaluate the safety of AMG 531, a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura.

Why It Was Studied

Platelet response was defined as a platelet count double the baseline value and between 50 and 450 x 10(9)/l.

The 500-microg cohort was discontinued because the first patient's platelet count became unacceptably high.

Who Took Part

The study group was 10 women.

By PubMed's age tags, the people studied were adults, middle-aged adults, older adults and people over 80, both men and women.

Sixteen patients (10 women) were enrolled.

How the Study Was Run

In design terms this was an open-label and multicenter study.

Nothing was hidden in this open-label Romiplostim study: the women and staff both knew the treatment.

More than one site took part, so the women came from several places.

Safety was assessed by adverse event (AE) monitoring, clinical laboratory studies and antibody assays.

What Was Measured

The subject headings attached to it are Carrier Proteins, Patient Selection, Platelet Count, Purpura, Thrombocytopenic, Idiopathic, Receptors, Fc, Recombinant Fusion Proteins and Thrombopoiesis.

What This Means in Plain Language

The paper is an open-label and multicenter study of Romiplostim in 10 women.

With fewer than 40 people, it is a small study, so it is better read as an early signal for further research than as a settled answer.

The publication is from 2006 (British journal of haematology), a journal based in England.

More context: the Romiplostim compound page, all human trials.