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Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia

NCT07572084 — Phase 2, Not yet recruiting, 43 participants, Peking Union Medical College Hospital
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Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study

RegistryNCT07572084
StatusNot yet recruiting
PhasePhase 2
Study typeInterventional; na, single group, masking: none, treatment
Enrollment43 (Estimated)
SponsorPeking Union Medical College Hospital (Other)
ConditionsAplastic Anemia
InterventionsRomiplostim N01 (Drug)
Datesstart 2026-04-01 (estimated), primary completion 2028-12-01 (estimated), completion 2029-12-01 (estimated), first posted 2026-05-07, last updated 2026-05-07

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-Summary

This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)

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+Arms and interventions

ArmTypeInterventionsDescription
Romiplostim N01ExperimentalDrug: Romiplostim N01Romiplostim N01 20 µg/kg
  • Romiplostim N01 (Drug): Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months

+Outcome measures

Primary outcomes

  • ORR (Overall Response Rate) [3-month, 6-month]: ORR=PRR (Partial Response Rate)+CRR (Complete Response Rate)

Secondary outcomes

  • CRR [3-month, 6-month]: Proportion of patients who achieved CR
  • Adverse event (AE) rate [through study completion, an average of 1 year]: Proportion of patients with AEs, according to CTCAE
  • Predictive factors for ORR or CRR [3-month]: Calculated by single-/multivariate regression
  • Clonal evolution rate [through study completion, an average of 1 year]: Proportion of patients progressed to myelodysplastic syndromes (MDS)/ acute myeloid leukemia (AML)

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Diagnosed with relapsed/refractory chemoradiotherapy-induced AA. Relapsed/ refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;
3. At least one of the following conditions must be met: hemoglobin < 90 g/ L, platelet count < 30×109/L, neutrophil count < 1.0×109/L;
4. With baseline liver and kidney functions <2 upper limit of normal (ULN);
5. Without active infection that cannot be controlled by standard treatment;
6. Signed the informed consent;
7. ECOG score ≤ 2;

Exclusion Criteria:

1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
4. Received hematopoietic stem cell transplantation before enrollment;
5. Received ATG treatment before enrollment;
6. Severe bleeding or infection that cannot be controlled by standard treatment;
7. Allergic or intolerant to romiplostim N01;
8. Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
9. History of malignant tumors within 5 years;
10. History of arterial or venous thrombosis;
11. Pregnant or lactating patients;
12. Participated in other clinical trials within 3 months.

My Notes