Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

What Was Tested

This registered phase 3 trial tested Romiplostim.

It had two arms (groups): Romiplostim (experimental) and Placebo (placebo comparator).

Participants received weekly subcutaneous placebo for 24 weeks.

The registry classes the interventions as drug.

Who Took Part

62 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 primary ITP according to the American Society of Hematology (ASH) guidelines at least 6 months prior to screening, regardless of splenectomy status. Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies. Age ≥ 1 year and \< 18 years at the time of providing informed consent. The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10\^9/L with neither count \> 35 x 10\^9/L. A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period.

The full record lists 8 inclusion and 17 exclusion criteria; among the exclusions: known active or prior malignancy except adequately treated basal cell carcinoma; known history of congenital thrombocytopenia; known history of hepatitis b, hepatitis c, or human immunodeficiency virus (hiv).

How the Study Was Run

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

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.

The Romiplostim group and the comparison group ran side by side rather than switching.

Neither the participants nor the staff measuring them knew who was on Romiplostim, so expectations could not easily colour the readings.

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

It started in 2012-01 with completion listed for 2015-02.

It ran at 33 sites in United States (25), Canada (5) and Australia (3).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Percentage of Participants With a Durable Platelet Response (time frame: Week 18 to week 25).

It also tracked 5 secondary outcomes, for example Percentage of Participants With an Overall Platelet Response, Number of Weeks With Platelet Response, Percentage of Participants Who Received Rescue Medication During the Treatment Period, Total Number of Composite Bleeding Episodes and Number of Participants With Adverse Events.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Romiplostim in 62 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.