Clinical trial NCT06516484: Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
Official title: Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE4
Conditions: Aplastic Anemia
Keywords: After radiotherapy and chemotherapy
Romiplostim
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
First posted: 2024-07-24
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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.

Primary outcomes:
measure: ORR
timeFrame: 3 months, 6 months
measure: CRR
timeFrame: 3 months, 6months
Secondary outcomes:
measure: safety events
timeFrame: 3 months，6months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Peking Union Medical College
name: Bing Han, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3.
citation: Lee JW, Lee SE, Jung CW, Park S, Keta H, Park SK, Kim JA, Oh IH, Jang JH. Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019 Nov;6(11):e562-e572. doi: 10.1016/S2352-3026(19)30153-X. Epub 2019 Aug 29.