Not Yet RecruitingPhase 3

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

210 participantsStarted 2026-01

Plain-language summary

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.

Who can participate

Age range15 Years

SexALL

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Inclusion criteria

✓. 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.
✓. Written informed consent

Exclusion criteria

✕. History and/or concomitant presence of other primary or secondary bone marrow failure (BMF) syndromes, such as:
✕. Evidence of clonal cytogenetic abnormalities at screening.
✕. Participation in another clinical trial with investigational drugs or medical devices within 30 days prior to the first dose or within 5 half-lives of the investigational product (whichever is longer).
✕. Previous use of any of the following agents prior to the first dose:
✕. Cumulative cyclosporine A (CsA) therapy exceeding 4 weeks prior to the first dose. If cumulative use is ≤4 weeks, a washout period of \>14 days prior to the first dose is required.
✕. Cumulative use of thrombopoietin receptor agonists (TPO-RAs) for \>14 days prior to the first dose, or cumulative use ≤14 days with a washout period of \<14 days, including:
✕. Previous history of hematopoietic stem cell transplantation.
✕. Uncontrolled bleeding and/or infection after standard treatment prior to the first dose \[defined as persistent signs/symptoms related to infection without improvement despite appropriate antibiotic and/or other therapy\], or requiring intravenous (IV) antibiotic administration.

What they're measuring

Complete Response (CR) rate at the 6-month time point of treatment.

Timeframe: During 6 months of therapy

Trial details

NCT IDNCT07345000

SponsorQilu Pharmaceutical Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-10

Contact for this trial

jun shi, Professor

022-23608326 shijun@ihcams.ac.cn

View on ClinicalTrials.gov More Treatment-naïve Severe Aplastic Anemia trials