Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)

What Was Tested

In this not phased trial, researchers set out to test Romiplostim.

This is an open label extension study of romiplostim for treatment of thrombocytopenia (platelet count ≤ 50 x 10\^9/L) in MDS subjects.

The study is designed to assess the long-term safety of treatment with romiplostim, as measured by incidence of overall adverse events, the incidence of bleeding events, the utilization of platelet transfusions, and the duration of platelet response.

The study will further describe the time to disease progression to acute myeloid leukemia (AML) and survival.

It had 1 arm (groups): Open Label Romiplostim (formerly AMG 531) (experimental).

The registry classes the interventions as drug.

Who Took Part

72 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Subject completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS. Subject has an Eastern Cooperative Oncology (ECOG) performance status of 0 to 2. Subject or his/her legally acceptable representative provided written informed consent before any study-specific procedures were initiated.

The full record lists 4 inclusion and 15 exclusion criteria; among the exclusions: subject has a prior history of leukemia; subject has a prior history of bone marrow or stem cell transplantation; subject has active or uncontrolled infections.

How the Study Was Run

Design: non-randomized, single-group and open-label.

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

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

It started in 2007-04-01 with completion listed for 2011-07-18.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Overall Summary of Adverse Events. Incidence of Antibody (AB) Formation.

It also tracked 5 secondary outcomes, for example Weekly Bleeding Events Per 100 Subject Years, Platelet Transfusion Events Per 100 Subject Years, Weeks With Platelet Response Per Year, Time to First Platelet Response and Duration of Platelet Response.

What This Means in Plain Language

Put simply, this is a not phased, non-randomized trial of Romiplostim in 72 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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