TPO-Mimetic Use in Children for Hematopoietic Failure

What Was Tested

This registered early phase 1 trial tested Romiplostim for anemia.

It had two arms (groups): Arm A (experimental) and Arm B (experimental).

The registry classes the interventions as drug.

More on the Plan

Methods: This open label, prospective Pilot interventional study has two arms.

Arm B will include children with chemo and or radiotherapy induced thrombocytopenia/cytopenia and children undergoing stem cell transplantation (SCT).

Who Took Part

The registry lists 15 actual participants took part.

It was open to people of any sex aged from 0 years up to 21 years.

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

Examples of who could join: Age ≥ 0 to ≤ 21 years. Child should be receiving ongoing care with pediatric hematology/oncology providers. Those enrolled in Arm A of the study should have a confirmed diagnosis of any of the following. Those enrolled in Arm B of the study should have a confirmed diagnosis of any of the following:. Myelo-suppression specifically thrombocytopenia as defined by primary oncologist in children with solid tumors secondary to chemotherapy or radiation therapy contributing to delay in chemotherapy.

The full record lists 13 inclusion and 16 exclusion criteria; among the exclusions: gestational age \< 32 weeks or age \> 21 years at the time of study enrolment; preexisting condition with predisposition for thrombosis; presence of complex karyotype or monosomy 7 or 5q- or other cytogenetic abnormality with known predisposition to cancer.

How the Study Was Run

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

Nothing was hidden in this open-label Romiplostim study: the anemia participants and staff both knew the treatment.

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

It started in 2020-08-18 with completion listed for 2024-01-18.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Occurrence of treatment-related adverse events (AEs) according to NCI CTCAE v5.0 (time frame: During treatment and through 90 days following discontinuation of treatment).

It also tracked 7 secondary outcomes, for example To assess time to hematological response, To assess longitudinal changes in blood counts, To assess the incidence of bleeding (including muco-cutaneous bleeding), To assess the incidence of neutropenic fever, To assess the requirement of blood product support and To assess development of bone marrow myelofibrosis.

What This Means in Plain Language

Put simply, this is a early phase 1, non-randomized trial of Romiplostim in 15 people with anemia.

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, Anemia research.