Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

What Was Tested

In this phase 3 trial, researchers set out to test Romiplostim.

This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric participants with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10\^9/L in the absence of ITP rescue medications for the past 4 weeks.

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

The registry classes the interventions as drug.

Who Took Part

The registry lists 203 actual participants took part.

It was open to people of any sex aged from 1 Year up to 18 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 before screening, regardless of splenectomy status. Age ≥ 1 year and \< 18 years of age. Refractory to prior ITP therapy, relapsed after at prior ITP therapy, or be ineligible for other therapies. Examples of prior therapy include: corticosteroids, intravenous Immunoglobulin (IVIG), anti-D immunoglobulin, platelet transfusions. Platelet count ≤ 30 x10\^9/L or is experiencing uncontrolled bleeding. Has provided informed consent before any study-specific procedure;.

The full record lists 12 inclusion and 25 exclusion criteria; among the exclusions: prior bone marrow transplant or peripheral blood progenitor cell transplant; active or prior malignancy except non-melanoma skin cancers within the last 5 years; history of myelodysplastic syndrome.

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 2014-12-10 with completion listed for 2018-08-30.

It ran at 78 sites in United States (16), Israel (6), Russia (6), Belgium (5), Spain (5) and Brazil (4).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Percentage of Time With a Platelet Response During the First 6 Months of Treatment (time frame: Week 2 to Month 6, platelet response was assessed every week). Percentage of Participants Who Developed Collagen After Exposure to Romiplostim (time frame: Year 1 (Cohort 1) and year 2 (Cohort 2)). Percentage of Participants Who Developed Bone Marrow Abnormalities (time frame: Year 1 (Cohort 1) and year 2 (Cohort 2)).

It also tracked 6 secondary outcomes, for example Percentage of Time With a Platelet Response During the Overall Treatment Period, Number of Participants Reporting Use of Rescue Medications for ITP During the Treatment Period, Number of Participants Who Developed Anti-Romiplostim or Anti-Thrombopoietin Neutralizing Antibodies and Number of Participants With Adverse Events.

What This Means in Plain Language

Put simply, this is a phase 3 trial of Romiplostim in 203 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.