Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim

What Was Tested

The trial (phase 2) is built around Romiplostim.

The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.

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

The registry classes the interventions as biological.

More on the Plan

If a participant maintains a platelet count of ≥ 50 x 10\^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue), the participant will be followed for at least 6 months to confirm the incidence of ITP remission.

If a participant's platelet count falls below 50 x 10\^9/L and the participant has tapered off treatment with romiplostim, the participant will enter the stabilization period and reinitiate romiplostim for up to 8 weeks.

Who Took Part

The registry lists 75 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: Initial diagnosis of primary ITP within 6 months of enrollment. Age ≥ 18 years at screening. A single platelet count ≤ 30 x 10⁹/L at any time during the screening period. Subject or subject's legally acceptable representative has provided informed consent.

The full record lists 4 inclusion and 22 exclusion criteria; among the exclusions: known history of a bone marrow stem cell disorder; surgical resection of the spleen; known history of congenital thrombocytopenia.

How the Study Was Run

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

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 2010-11-30 with completion listed for 2013-09-20.

It ran at 48 sites in United States (8), Italy (8), Czechia (5), France (5), Poland (5) and Spain (5).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Number of Months With Platelet Response During the 12-Month Treatment Period (time frame: 12 months).

It also tracked 4 secondary outcomes, for example Percentage of Participants With ITP Remission, Percentage of Participants With Splenectomy During the 12-month Treatment Period, Number of Participants With Adverse Events and Number of Participants Who Developed Antibodies to Romiplostim.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Romiplostim in 75 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.