Clinical trial NCT06535685: A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA
Official title: A Single-centre, Prospective, Open-label, Single-arm Study of Romiplostim for the Treatment of Refractory Transfusion-dependent Non-severe Aplastic Anaemia (NSAA)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE4
Conditions: Aplastic Anemia
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2024-08-02
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Overall response rate (ORR)
timeFrame: 3 months
measure: Overall response rate (ORR)
timeFrame: 6 months
measure: CRR
timeFrame: 3 months
measure: CRR
timeFrame: 6 months
Secondary outcomes:
measure: safety events
timeFrame: 3 months
measure: safety events
timeFrame: 6months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
References:
citation: Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.
citation: Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.
citation: Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017 Mar 16;129(11):1428-1436. doi: 10.1182/blood-2016-08-693481. Epub 2017 Jan 17.
citation: Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.