Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS)

What Was Tested

The trial (phase 2) is built around Romiplostim.

The purpose of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with low or Intermediate-1 risk MDS.

In addition, the study will evaluate the platelet response to romiplostim.

It had seven arms (groups): one (experimental), one (experimental), one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 72 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: Diagnosis of MDS using the World Health Organization classification. Low or Intermediate-1 risk MDS using the International Prognostic Scoring System (IPSS). Must be ≥ 18 years of age at the time of obtaining informed consent. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 at the time of screening. Before any study-specific procedure, the appropriate written informed consent must be obtained (see Section 12.1).

The full record lists 8 inclusion and 22 exclusion criteria; among the exclusions: prior history of bone marrow transplantation; persistent peripheral blood monocytosis (≥ 3 months with an absolute monocyte count \> 1,000/µl); received anti-thymocyte globuline (atg) within 6 months of screening.

How the Study Was Run

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

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

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2006-02 with completion listed for 2008-05.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Part A: Number of Participants With Adverse Events (time frame: Treatment period (4 weeks) plus treatment extension (1 year)). Part B: Number of Participants With Adverse Events (time frame: Treatment period (8 weeks) plus treatment extension (1 year)).

It also tracked 15 secondary outcomes, for example Part A: Number of Participants With a Complete or Major Platelet Response, Part B: Number of Participants With a Complete or Major Platelet Response, Part A: Number of Participants With a Platelet Response Per IWG Criteria, Part B: Number of Participants With a Platelet Response Per IWG, Part B: Peak Platelet Count and Part B: Time to First Platelet Response.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of Romiplostim in 72 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.