CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA

Plain-language guide to NCT07523009: phase 2 interventional trial of Romiplostim, anemia. Who took part, design and what was measured.

Key Facts

Registry ID: NCT07523009
Phase: phase 2
Status: withdrawn
Start: 2026-06
Completion (planned or actual): 2028-12
Participants: n/a (actual)
Design: na, open label (everyone knows who gets what), treatment
Conditions studied: Severe Aplastic Anemia (SAA); Transfusion-dependent Non-severe Aplastic Anemia
Results posted: not yet

What This Study Did, in Plain Language

What Was Tested

This registered phase 2 trial tested Romiplostim for anemia.

It had 1 arm (groups): CsA+EPAG/HPAG+Romiplostim N01 (experimental).

The registry classes the interventions as drug.

More on the Plan

For SAA/TD-NSAA patients without HLA-matched donors and those over 40 years old, the first choice is immunosuppressive therapy (IST) + cyclosporine A (CsA) combined with thrombopoietin receptor agonists (TPO-RAs).

Therefore, in recent years, treatment regimens without ATG have also been gradually explored.

Although both romiplostim and eltrombopag/hyrtiopeg activate the TPO receptor (c-Mpl), there are differences and complementarities in their molecular mechanisms.

The binding sites of the two drugs are spatially separated and may produce a synergistic effect through different intensities and dynamics of downstream STAT, PI3K/AKT, and other signaling pathways.

Who 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: Age ≥ 18 years old;. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);. Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;. With baseline liver and kidney functions \<2 ULN;. ECOG score ≤ 2;.

The full record lists 7 inclusion and 12 exclusion criteria; among the exclusions: had other primary or secondary bone marrow failure (bmf) diseases, such as fanconi anemia, congenital keratinization disorder, etc.;; with evidence of clonal hematological bone marrow diseases (mds, aml) in cytogenetics;; received hsct before enrollment;.

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 2026-06 with completion listed for 2028-12.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Overall response rate (ORR) (time frame: 6-month).

It also tracked 3 secondary outcomes, for example ORR, red blood cell (RBC)/platelet (PLT) transfusion independent rate and AE rate.

What This Means in Plain Language

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

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, Anemia research.

Our Notes on the Compound

Romiplostim: A peptide-antibody fusion (peptibody) that activates the thrombopoietin receptor to raise platelet counts; sold as Nplate.

Source: ClinicalTrials.gov record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.