Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

What Was Tested

The trial (phase 3) is built around Romiplostim.

The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim.

Participants must have previously completed a romiplostim ITP study.

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

The registry classes the interventions as biological.

Who Took Part

Enrollment: 313 actual participants took part.

It was open to people of any sex aged from 1 Year with no upper age limit.

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

Examples of who could join: Must have previously completed a romiplostim ITP study. Platelet count ≤ 50 x 10 \^9/L. Written informed consent.

The full record lists 3 inclusion and 6 exclusion criteria; among the exclusions: bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim itp study; received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study; not using adequate contraceptive precautions.

How the Study Was Run

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

Because people in this Romiplostim study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

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 2004-08 with completion listed for 2010-01.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Number of Participants With Adverse Events (time frame: Duration of treatment plus 8 weeks (up to 285 weeks)).

It also tracked 7 secondary outcomes, for example Number of Participants With a Platelet Response, Change From Baseline in ITP Patient Assessment Questionnaire, Change From Baseline in Short Form 36 (SF-36), Change From Baseline in Euroqol-5D (EQ-5D) Index Score, Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS) and Patient Global Assessment.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Romiplostim in 313 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.