Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)

What Was Tested

In this phase 4 trial, researchers set out to test Romiplostim.

The purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.

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

The registry classes the interventions as biological.

More on the Plan

All participants received romiplostim for 3 years, unless withdrawn from the study early.

Who Took Part

Enrollment: 169 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: Diagnosis of ITP according to American Society of Hematology (ASH) guidelines. Subject must agree to a scheduled bone marrow biopsy at Year 1, Year 2, or Year 3 following romiplostim treatment and any unscheduled biopsies if clinically indicated. Subject ≥18 years of age. Baseline bone marrow reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scheme as assessed by central laboratory interpretation. Platelet count \< 50 x 10\^9/L.

The full record lists 8 inclusion and 12 exclusion criteria; among the exclusions: baseline bone marrow biopsy positive for collagen fibrosis; any known history of or currently active bone marrow stem cell disorder, hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome; any current active malignancy.

How the Study Was Run

It was run as a single-group and open-label study.

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 2009-08-11 with completion listed for 2014-01-09.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Percentage of Participants With Collagen Fibrosis (time frame: At Years 1, 2 or 3 after initial exposure of romiplostim).

It also tracked 7 secondary outcomes, for example Percentage of Participants With Clinically Relevant Changes in Total Cardiac Output Corrected (QTc) Intervals, Percentage of Participants With CTCAE Grade ≥ 2 Shift in Anemia or Neutropenia, Number of Participants With Adverse Events (AEs) and Number of Participants Who Developed Antibodies or Neutralizing Antibodies to Romiplostim or to Endogenous Thrombopoietin.

What This Means in Plain Language

Put simply, this is a phase 4 trial of Romiplostim in 169 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.