UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

What Was Tested

In this phase 2 trial, researchers set out to test Romiplostim for anemia.

It had two arms (groups): Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST) (experimental) and Relapsed or refractory SAA (Cohort B): Romiplostim (experimental).

Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).

Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.

The registry classes the interventions as drug.

More on the Plan

The study is designed as a Phase II, multicenter, investigator-initiated, open label, interventional study that will recruit children (age: \>2 to \<21 years) with SAA.

Hematologic complete response (HCR) will be used to assess the therapy response.

Who Took Part

It was open to people of any sex aged from 2 years up to 21 years.

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

Examples of who could join: Age ≥2 years to ≤21 years. Child should be receiving ongoing care with pediatric hematology/oncology provider. Confirmed Diagnosis of SAA and other related conditions based on following criteria. Patient with available allogenic stem cell donor chooses to defer HSCT option at least for 12-weeks. Parent, guardian, or patient (if \>18 years of old) must be able to provide written and voluntary informed consent and assent (if \>12 years of age).

The full record lists 6 inclusion and 22 exclusion criteria; among the exclusions: presence of complex karyotype or monosomy 7 or 5q- or other cytogenetic abnormality with known predisposition to cancer; diagnosis of mds; patients taking concurrent therapy with eltrombopag, avatrombopag, and lusutrombopag\*\*\*.

How the Study Was Run

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

Everyone knew when Romiplostim was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

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

It started in 2025-04-10 with completion listed for 2031-12-31.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 2 secondary outcomes, for example Incidence of adverse events as assessed by CTCAE v5.0 and Proportion of participants with a new cytogenetic abnormality.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized 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.