Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA

Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study

This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia

Study Overview

• Status: Not yet recruiting
• Conditions: Aplastic Anemia
• Intervention / Treatment: Drug: Romiplostim N01

Detailed Description

Currently, there are few studies on the use of TPO-RAs after tumor radiotherapy and chemotherapy. Our center has published a study on the efficacy and safety of avatrombopag (AVA) in AA secondary to radiotherapy and chemotherapy. The study demonstrated that the ORR at 1, 3, and 6 months were 32.4%, 55.9%, and 58.8%, respectively. In a phase II/III study for refractory AA, romiplostim monotherapy achieved an ORR of 84% at week 27, which showed that romiplostim seemed to be effective and safe in patients with refractory aplastic anemia, with a recommended starting dose of 20 μg/kg once a week.

• Study Type: Interventional
• Enrollment (Estimated): 43
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Bing Han, Doctor; Phone Number: +86 13601059938; Email: hanbing_li@sina.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Diagnosed with relapsed/refractory chemoradiotherapy-induced aplastic anemia (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 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. PNH clone ≥ 50%;
4. Received HSCT 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Romiplostim N01; Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months	Drug: Romiplostim N01; Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR	Overall response rate (ORR) = PRR+CRR	3-month, 6-month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
CRR	Complete response rate	3-month, 6-month, 12-month
AE rate	Proportion of patients with adverse events, according to CTCAE	through study completion, an average of 1 year
Predictive factors of efficacy	Predictive factors of ORR or CRR	3-month
Clonal evolution rate	Proportion of patients progressed to MDS/ AML	through study completion, an average of 1 year

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: April 8, 2026
• First Submitted That Met QC Criteria: April 8, 2026
• First Posted (Actual): April 15, 2026

Study Record Updates

• Last Update Posted (Actual): April 15, 2026
• Last Update Submitted That Met QC Criteria: April 8, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Hemic and Lymphatic Diseases; Anemia, Aplastic
• Other Study ID Numbers: LP-N01-CR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No