Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects

What Was Tested

This registered phase 1, phase 2 trial tested Romiplostim.

The purpose of this study is to evaluate the safety and tolerability of romiplostim (AMG 531) in the treatment of thrombocytopenia in pediatric subjects with chronic ITP.

It had two arms (groups): II. (placebo comparator) and I. (experimental).

5 thrombocytopenic (as defined per protocol) subjects.

15 thrombocytopenic (as defined per protocol) subjects.

The registry classes the interventions as drug.

Who Took Part

The registry lists 22 actual participants took part.

It was open to people of any sex aged from 1 Year up to 17 years.

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

Examples of who could join: Diagnosis of ITP according to The American Society of Hematology (ASH) Guidelines at least six months prior to screening. Age ≥ 12 months and \< 18 years at enrollment. The mean of two platelet counts taken during the screening period must be ≤ 30 x 10\^9/L with no single count \>35 x 10\^9/L. A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category). Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range.

The full record lists 7 inclusion and 20 exclusion criteria; among the exclusions: known history of venous or arterial thrombotic or thromboembolic event; known history of congenital thrombocytopenia; known history of malignancy except basal cell carcinoma.

How the Study Was Run

Design: randomized, parallel-group and triple-blind.

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.

As a double-blind Romiplostim study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, investigator and outcomes assessor.

It started in 2007-07 with completion listed for 2009-03.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Adverse Events (time frame: 12 weeks).

It also tracked 5 secondary outcomes, for example Weeks With Platelet Count ≥ 50 x 10^9/L, Bleeding Events (Grade 2 or Higher), Platelet Count ≥ 50 x 10^9/L for Two Consecutive Weeks and Requirement for Rescue Therapy (as Defined Per Protocol).

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of Romiplostim in 22 people.

Its size is small, so on its own it can only give an early look.

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.