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

Phase 4

Not yet recruiting

• Conditions: Aplastic Anemia
• Interventions: Drug: Romiplostim

• Registration Number: NCT06516484
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-07
• Study First Submitted: 2024-07-18
• Study First Submitted QC: 2024-07-18
• Last Update Submitted: 2024-07-18
• Study First Posted: 2024-07-24
• Last Update Posted: 2024-07-24
• Study Start: 2024-07-30
• Primary Completion: 2025-07-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 40

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Romiplostim group	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.

Primary Outcome Measures

Name	Time	Method
ORR	3 months, 6 months	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.
CRR	3 months, 6months	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.

Secondary Outcome Measures

Name	Time	Method
safety events	3 months，6months	Proportion and severity of adverse events in patients during the study period

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China