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

Launched by PEKING UNION MEDICAL COLLEGE HOSPITAL · Jul 30, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating a medication called Romiplostim to see how well it works and how safe it is for patients with a condition known as refractory transfusion-dependent Non-Severe Aplastic Anemia (NSAA). Aplastic Anemia is a serious blood disorder where the body doesn’t produce enough blood cells, and "refractory" means that previous treatments haven’t worked. The trial is looking for adults aged 18 and older who have been receiving blood transfusions regularly and have not responded to standard treatments for at least six months.

To participate, individuals must have specific low blood cell counts and agree to take part in the study. This means they will need to sign a consent form. However, certain health conditions, like other blood disorders or serious infections, may prevent someone from joining the trial. Participants will receive Romiplostim and will be monitored for its effects, helping researchers better understand this treatment for Aplastic Anemia. The trial is not yet recruiting, so interested individuals will need to wait for it to begin.

Gender

ALL

Eligibility criteria

• 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.