Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura

What Was Tested

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

This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety Follow-up.

Patients randomized to romiplostim must complete the taper or discontinuation of medical SOC for ITP as soon as medically feasible after the initiation of romiplostim.

After the completion or discontinuation of the study treatment period, any participant who does not transfer in to another romiplostim study will complete a 6-month Safety Follow-up period.

It had two arms (groups): Romiplostim (experimental) and Standard of Care (other).

Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.

The registry classes the interventions as biological and drug.

Who Took Part

The registry lists 234 actual participants took part.

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

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

Examples of who could join: Subject is ≥ 18 years of age. Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines. If subject is \> 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP. Subject has received at least 1 prior therapy for ITP. Subject has a platelet count \< 50,000 or their platelet count falls to \< 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy.

The full record lists 6 inclusion and 12 exclusion criteria; among the exclusions: subject has had a splenectomy for any reason; subject has an active malignancy; subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years.

How the Study Was Run

It was run as a randomized, parallel-group and open-label study.

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.

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

It started in 2006-12-01 with completion listed for 2008-11-07.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Number of Participants With Splenectomy During 52-Week Treatment Period (time frame: 52 weeks). Number of Participants With Treatment Failure During 52-Week Treatment Period (time frame: 52 weeks).

It also tracked 6 secondary outcomes, for example Time to Splenectomy, Percentage of Participants With Platelet Response, Change in ITP-PAQ Physical Health Domain of Symptoms, Change in ITP-PAQ Physical Health Domain of Fatigue, Change in ITP-PAQ Physical Health Domain of Bother and Change in ITP-PAQ Physical Health Domain of Activity.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Romiplostim in 234 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.