Eltrombopag and the Bcl-extra-large (xL) Pathway in Idiopathic Thrombocytopenic Purpura (ITP)

What Was Tested

This registered phase 2 trial tested Romiplostim.

The purpose of this study is to further evaluate the effects that eltrombopag (and romiplostim) have on platelets in subjects with chronic ITP.

Eltrombopag is approved by the Food and Drug Administration (FDA) for the treatment of low platelets in patients with chronic ITP.

It had three arms (groups): eltrombopag (experimental), romiplostim (experimental) and healthy controls (sham comparator).

No intervention single blood draw with complete studies.

The registry classes the interventions as drug and other.

More on the Plan

The B-cell lymphoma extra large (Bcl-xL/Bak) balance has been identified as an intrinsic mechanism that is critical in determining platelet lifespan (Mason, Cell 2007).

There is evidence that Bcl-xL protein expression in megakaryocytes is regulated by Thrombopoietin (TPO) mediated activation of Akt pathways mediated by Jak2 and Stat 5 (possibly by Stat 3 as well).

(e.g., Kozuma et. al., Journal of Thrombosis and Haemostasis).

Little is known about the Bcl-xL / Bak axis in patients with ITP, or the effect of TPO-R stimulation on platelet survival in patients with ITP.

Who Took Part

Enrollment: 20 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers could join, not only people with the condition.

Examples of who could join: Subject has signed and dated a written informed consent. Subjects must have responded with a platelet count \> 30,000/µL to a previous ITP therapy including thrombopoietic agents. Platelet count \< 30,000/µL. Female subjects of childbearing potential are practicing an acceptable method of contraception or are completely abstinent from intercourse.

The full record lists 5 inclusion and 11 exclusion criteria; among the exclusions: active infection; female subjects who are nursing or pregnant; thrombosis of any kind within past 6 months or on blood thinners because of thrombosis.

How the Study Was Run

It was run as a non-randomized, sequential and open-label study.

Being open-label, the Romiplostim study did not mask who received what.

It started in 2009-01 with completion listed for 2015-09-07.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 1 secondary outcome, for example How Many Patients Developed SAEs and/or Abnormal Liver Tests to a Level > 2 Times the Upper Limit of Normal.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of Romiplostim in 20 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.