Safety of Romiplostim (Nplate®) Following UCBT

What Was Tested

The trial (phase 1) is built around Romiplostim.

Engraftment is defined as a platelet count ≥ 20 x 109/L on 3 consecutive measurements without transfusion for 7 days.

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

The registry classes the interventions as drug.

Who Took Part

21 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: Any patient who has undergone a single or double umbilical cord blood transplant (UCBT) except those with primary myelofibrosis. Those with acute leukemia must be in remission at the time of transplant. Must have achieved neutrophil engraftment (defined as an ANC \>500 for three consecutive days) and be off daily G-CSF prior to starting romiplostim. Intermittent G-CSF is allowed. Failure to achieve platelet engraftment (defined as platelet count ≥20x10\^9/L on 3 consecutive measurements without transfusion for 7 days) by day +28 post UCBT. Between day +28 and day +42 status post myeloablative or nonmyeloablative UCBT (single or double cord blood transplant).

The full record lists 11 inclusion and 5 exclusion criteria; among the exclusions: known pregnancy - pregnancy category c: there are no adequate and well-controlled studies of romiplostim in pregnancy; recurrence of aml or myelodysplastic syndrome on bone marrow evaluation done within 21 days of enrollment; patients requiring more than one platelet transfusion per day.

How the Study Was Run

Design: single-group and open-label.

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 2015-04-10 with completion listed for 2020-12-12.

It ran at 1 site in United States.

Its stated primary purpose is supportive care.

What Was Measured

It also tracked 3 secondary outcomes, for example Platelet recovery, Thrombocytopenia and Bone marrow fibrosis.

What This Means in Plain Language

Put simply, this is a phase 1 trial of Romiplostim in 21 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, all human trials.