Status:
NOT_YET_RECRUITING
A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA
Lead Sponsor:
Peking Union Medical College Hospital
Conditions:
Aplastic Anemia
Eligibility:
All Genders
18+ years
Phase:
PHASE4
Brief Summary
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
Eligibility Criteria
Inclusion
- Age ≥18 years, male or female.
- 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.
- 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.
- Agree to sign the consent form.
- An Eastern Cooperative Oncology Group (ECOG) score of 0-2.
Exclusion
- Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
- Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
- PNH clones ≥50%.
- Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
- Prior treatment with ATG.
- Infection or haemorrhage uncontrolled by standard therapy.
- Allergy to roprostin.
- Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
- Liver and renal function at baseline that is more than two times normal.
- Active infection.
- Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
- Pregnant or lactating (breastfeeding) women.
- Participation in another clinical trial within 3 months.
Key Trial Info
Start Date :
August 2 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2025
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06535685
Start Date
August 2 2024
End Date
December 31 2025
Last Update
August 2 2024
Active Locations (1)
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1
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730