A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia

What Was Tested

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

This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.

It had two arms (groups): Romiplostim N01+ IST (experimental) and Placebo+ IST (active comparator).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 210 planned participants were the target.

It was open to people of any sex aged from 15 years with no upper age limit.

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

Examples of who could join: Age ≥15 years, regardless of sex (subjects ≥18 years old will be enrolled first; enrollment of subjects aged 15-18 years will commence after sufficient PK/PD data are obtained). Diagnosis of SAA or VSAA according to the British Journal of Haematology (BJH) guidelines. The diagnostic criteria for SAA are as follows:. Absolute neutrophil count (ANC) \<0.5×10⁹/L. Platelet count (PLT) \<20×10⁹/L. Absolute reticulocyte count (RET) \<60×10⁹/L The diagnostic criterion for VSAA is: meeting the SAA criteria + ANC \<0.2×10⁹/L.

The full record lists 6 inclusion and 16 exclusion criteria; among the exclusions: history and/or concomitant presence of other primary or secondary bone marrow failure (bmf) syndromes, such as:; secondary: large granular lymphocyte (lgl) leukemia, infiltration of the bone marrow by other systemic malignancies, myelofibrosis, and acute hematopoietic arrest, etc; evidence of clonal cytogenetic abnormalities at screening.

How the Study Was Run

Design: randomized, parallel-group and double-blind.

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.

As a double-blind Romiplostim study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant and investigator.

It started in 2026-01 with completion listed for 2029-10.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Complete Response (CR) rate at the 6-month time point of treatment (time frame: During 6 months of therapy).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Romiplostim in 210 people with anemia.

Its status is not yet recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, Anemia research.