A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA

Phase 4

Not yet recruiting

• Conditions: Aplastic Anemia
• Interventions: Drug: Romiplostim

• Registration Number: NCT06535685
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-07
• Study First Submitted: 2024-07-24
• Study First Submitted QC: 2024-07-30
• Last Update Submitted: 2024-07-30
• Study Start: 2024-08-02
• Study First Posted: 2024-08-02
• Last Update Posted: 2024-08-02
• Primary Completion: 2025-03-30
• 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 transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
3. Satisfy at least one of the following conditions at the time of enrolment: haemoglobin <90 g/L, platelets <30×10^9/L, neutrophils <1.0×10^9/L.
4. Agree to sign the consent form.
5. An 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. Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
5. Prior treatment with ATG.
6. Infection or haemorrhage 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. Liver and renal function at baseline that is more than two times normal.
11. Active infection.
12. Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
13. Pregnant or lactating (breastfeeding) women.
14. 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	Romiplostim group

Primary Outcome Measures

Name	Time	Method
CRR	6 months	CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.
Overall response rate (ORR)	6 months	Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC \<0.5 × 10\^9/L increased by at least 0.5 × 10\^9/L after treatment, or initial PLT \<20 × 10\^9/L increased by at least 20 × 10\^9/L after treatment.

Secondary Outcome Measures

Name	Time	Method
safety events	6months	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Trial Locations

Locations (1)

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