Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study

To investigate the efficacy and safety of roprostin in the treatment of refractory AA after radiotherapy.

Study Overview

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

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Bing Han, PhD; Phone Number: +8613601059938; Email: Hanbing_li@sina.com.cn
• Study Contact Backup: Name: Leyu Wang; Phone Number: 18239490957; Email: wangleyu_ys@163.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100730
      • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Age ≥18 years, male or female.
2. Diagnosis consistent with refractory AA after radiotherapy. refractory is defined as patients who have failed to respond to at least an adequate amount of supportive therapy, cyclosporine or povidone TPO-RA for 3 months .
3. At least one of the following conditions was met at enrolment: haemoglobin <90 g/L. Platelets <30 x 10^9/L, neutrophils <1.0 x 10^9/L.
4. Baseline liver and renal function is less than two times the normal value.
5. No active infection.
6. Agreed to sign the consent form.
7. Eastern Cooperative Oncology Group (ECOG) score of 0-2.

Exclusion Criteria:

1. Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
2. Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
3. PNH clones ≥50%.
4. Hematopoietic stem cell transplantation (HSCT) prior to enrolment.
5. Prior treatment with ATG.
6. Infection or bleeding uncontrolled by standard therapy.
7. Allergy to roprostin.
8. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
9. Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
10. Previous history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
11. Pregnant or lactating (breastfeeding) women.
12. Participation in another clinical trial 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 group; Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.	Drug: Romiplostim; Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10^9/L and continuation of roprostin for platelet counts <50 x 10^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR	Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.	3 months, 6 months
CRR	Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count >1.5 × 10^9/L and platelet count >150 × 10^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC < 0.5 x 10^9/L increased by at least 0.5 x 10^9/L after treatment, or initial PLT < 20 x 10^9/L increased by at least 20 x 10^9/L after treatment.	3 months, 6months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
safety events	Proportion and severity of adverse events in patients during the study period	3 months，6months

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Investigators: Principal Investigator: Bing Han, PhD, Peking Union Medical College

Publications and helpful links

General Publications

• Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
• Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.

Study record dates

Study Major Dates

• Study Start (Estimated): July 30, 2024
• Primary Completion (Estimated): July 31, 2025
• Study Completion (Estimated): December 31, 2025

Study Registration Dates

• First Submitted: July 18, 2024
• First Submitted That Met QC Criteria: July 18, 2024
• First Posted (Actual): July 24, 2024

Study Record Updates

• Last Update Posted (Actual): July 24, 2024
• Last Update Submitted That Met QC Criteria: July 18, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Romiplostim; After radiotherapy and chemotherapy
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: LRA-2024-001

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