Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients

What Was Tested

This registered phase 2 trial tested Romiplostim.

This is a single-arm study (no comparison group).

It had 1 arm (groups): Romiplostim N01 (experimental).

The registry classes the interventions as drug.

More on the Plan

Romiplostim N01 binds to the c-MPL receptor on megakaryocytes and their precursors, activating JAK-STAT and MAPK-ERK signaling pathways to stimulate platelet production.

The study involves 10 site visits over 36 days, including screening (≤14 days), baseline (Day 0), treatment (Days 1, 3, 5, 8, 15), and follow-up (Days 22, 29, 36).

Safety is rigorously monitored using NCI CTCAE v5.0 for adverse events (AEs), WHO bleeding scale for hemorrhage, and active surveillance for thrombosis.

Rescue therapies (e.g., platelet transfusions, rhTPO, rhIL-11) are permitted per protocol-specified thresholds.

Who Took Part

50 planned participants were the target.

It was open to people of any sex aged from 6 years up to 24 years.

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

Examples of who could join: Voluntary participation with signed informed consent. Aged 6-24 years, any gender. ECOG performance status 0-2. Histologically/cytologically confirmed non-myeloid malignancy requiring high-intensity chemotherapy with ≥1 myelosuppressive agent. Patients with potential curative opportunity eligible for standard therapy.

The full record lists 17 inclusion and 11 exclusion criteria; among the exclusions: hematologic disorders (non-cit etiology): aml, itp, mds, mpn, multiple myeloma, etc; non-cit thrombocytopenia within 6 months (e.g., chronic liver disease, hypersplenism, infection, hemorrhage); known hypersensitivity to romiplostim n01 or excipients (cellulose-lactose, l-hpc, magnesium stearate, film coating).

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label Romiplostim study: the 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 2025-12-25 with completion listed for 2026-09-01.

It ran at 1 site in China.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 11 secondary outcomes, for example Nadir platelet count, Median time to peak platelets, Median time to nadir platelets, Maximum platelet count, Frequency of platelet transfusions and Volume of platelet transfusions.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Romiplostim in 50 people.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

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