Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia

NCT07572084 | Not Yet Recruiting Phase 2

• 1 other identifier: K10498
• Study Type: interventional
• Target: 43
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• ORR (Overall Response Rate)

  ORR=PRR (Partial Response Rate)+CRR (Complete Response Rate)

  3-month, 6-month

Secondary Outcomes (4)

• CRR

  3-month, 6-month

• Adverse event (AE) rate

  through study completion, an average of 1 year

• Predictive factors for ORR or CRR

  3-month

• Clonal evolution rate

  through study completion, an average of 1 year

Study Arms (1)

Romiplostim N01

EXPERIMENTAL

Romiplostim N01 20 µg/kg

Drug: Romiplostim N01

Interventions

Romiplostim N01 DRUG

Romiplostim N01 20 µg/kg subcutaneously once a week for at least 3 months

Romiplostim N01

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years old;
• 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;
• 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;
• With baseline liver and kidney functions \<2 upper limit of normal (ULN);
• Without active infection that cannot be controlled by standard treatment;
• Signed the informed consent;
• ECOG score ≤ 2;

You may not qualify if:

• 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;
• Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;
• Received hematopoietic stem cell transplantation before enrollment;
• Received ATG treatment before enrollment;
• Severe bleeding or infection that cannot be controlled by standard treatment;
• Allergic or intolerant to romiplostim N01;
• Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension
• History of malignant tumors within 5 years;
• History of arterial or venous thrombosis;
• Pregnant or lactating patients;
• Participated in other clinical trials within 3 months.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Central Study Contacts

Bing Han

CONTACT

+86 13601059938; hanbing_li@sina.com.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 23, 2026
• First Posted: May 7, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: May 7, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share