Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

What Was Tested

The trial (phase 2) is built around Romiplostim.

The Data Monitoring Committee (DMC) for study 20060198 recommended that all subjects discontinue treatment of study drug and continue to be followed for long term follow-up.

It had two arms (groups): Romiplostim (experimental) and Placebo (placebo comparator).

The registry classes the interventions as biological and drug.

More on the Plan

The study is composed of a 26-week placebo controlled test treatment period (romiplostim versus Placebo), a 4 week interim wash-out period, a 24-week placebo controlled extended treatment period, and a 4-week follow-up period followed by an End of Study (EOS) visit.

During the interim wash-out period, a bone marrow biopsy will be performed in the absence of growth factor to assess changes in the marrow.

In the extended treatment period, safety assessments will continue and participants will be allowed to receive any standard of care treatments for MDS.

Patients will be followed for survival for an additional 60 months following the End of Study (EOS) visit.

Who Took Part

The registry lists 250 actual participants took part.

It was open to people of any sex aged from 18 years up to 90 years.

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

How the Study Was Run

It was run as a randomized, parallel-group and double-blind study.

Random assignment meant chance, not the researchers, decided who got Romiplostim and who got the comparison, which limits bias.

The Romiplostim group and the comparison group ran side by side rather than switching.

Neither the participants nor the staff measuring them knew who was on Romiplostim, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant and investigator.

It started in 2008-07-21 with completion listed for 2011-03-31.

Its stated primary purpose is supportive care.

What Was Measured

It also tracked 9 secondary outcomes, for example Annualized Rate of Platelet Transfusion Events, Annualized Rate of Overall Bleeding Events, Annualized Rate of Total Platelet Transfusion Units, Kaplan-Meier Estimate of Survival at Month 12 and Annualized Rate of Patient-reported Bleeding Events.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Romiplostim in 250 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.