Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

What Was Tested

This registered phase 2 trial tested Romiplostim.

The primary objective of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with ITP.

It had ten arms (groups): one (experimental), one (experimental), one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 45 actual participants took part.

It was open to people of any sex aged from 18 years up to 65 years.

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 at least 3 months before enrollment. Have completed at least 1 prior treatment for ITP. Two (including day -2) of the 3 platelet counts taken during the screening and pre-treatment periods must have fulfilled the following:. Less than 30 x 10\^9/L for those subjects not receiving any ITP therapy,. Less than 50 x 10\^9/L for those subjects receiving any ITP therapy.

The full record lists 10 inclusion and 17 exclusion criteria; among the exclusions: any known history of bone marrow stem cell disorder; known positive test for human immunodeficiency virus (hiv) infection or hepatitis c virus; received intravenous (iv) immunoglobulin (ig) or winrho within 2 weeks before the screening visit.

How the Study Was Run

Design: randomized, sequential and double-blind.

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

Blinding on both sides kept the participants and researchers unaware of who received Romiplostim.

Those kept unaware of assignments: participant and investigator.

It started in 2002-07-01 with completion listed for 2004-06-17.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Number of Participants With Adverse Events. Number of Participants With Anti-romiplostim or Anti-endogenous Thrombopoietin Neutralizing Antibodies (time frame: Assessed on day 29 (Part A only), day 43 (Part B only), and day 78).

It also tracked 17 secondary outcomes, for example Number of Participants Who Achieved Targeted Therapeutic Platelet Level in Part A, Number of Participants With a Peak Platelet Count ≥ 100 x 10⁹ Cells/L in Part A, Number of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part A, Duration Within the Targeted Therapeutic Platelet Range In Part A, Percentage of Participants Who Achieved Targeted Therapeutic Platelet Level In Part B and Percentage of Participants With a Peak Platelet Count of ≥ 100 x 10⁹ Cells/L in Part B.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Romiplostim in 45 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.