Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study

NCT06516484 | Not Yet Recruiting Phase 4

• 1 other identifier: LRA-2024-001
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Aplastic Anemia

Keywords

After radiotherapy and chemotherapy; Romiplostim

Outcome Measures

Primary Outcomes (2)

• ORR

  Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment.

  3 months, 6 months

• CRR

  Overall response rate (ORR) is defined as the ratio of complete response (CR) + partial response (PR).CR is defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients not receiving transfusion.PR is defined as not transfusion dependent (if previously dependent), or at least one cell lineage doubled or normalised or increased at baseline, or initial ANC \< 0.5 x 10\^9/L increased by at least 0.5 x 10\^9/L after treatment, or initial PLT \< 20 x 10\^9/L increased by at least 20 x 10\^9/L after treatment.

  3 months, 6months

Secondary Outcomes (1)

• safety events

  3 months，6months

Study Arms (1)

Romiplostim group

EXPERIMENTAL

Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10\^9/L and continuation of roprostin for platelet counts \<50 x 10\^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.

Drug: Romiplostim

Interventions

Romiplostim DRUG

Enrolled patients were given roprostin (20 µg/kg subcutaneously once weekly) for at least 3 months, with discontinuation of roprostin for platelet counts ≥50 x 10\^9/L and continuation of roprostin for platelet counts \<50 x 10\^9/L. Responders were continued to 6 months. Responders continue to use the drug until 6 months.

Romiplostim group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years, male or female.
• 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 .
• 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.
• Baseline liver and renal function is less than two times the normal value.
• No active infection.
• Agreed to sign the consent form.
• Eastern Cooperative Oncology Group (ECOG) score of 0-2.

You may not qualify if:

• 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%.
• Hematopoietic stem cell transplantation (HSCT) prior to enrolment.
• Prior treatment with ATG.
• Infection or bleeding 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.
• Previous 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (2)

• 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.

  PMID: 23821332 BACKGROUND

• 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.

  PMID: 31474546 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Bing Han, PhD

  Peking Union Medical College

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Bing Han, PhD

CONTACT

+8613601059938; Hanbing_li@sina.com.cn

Leyu Wang

CONTACT

18239490957; wangleyu_ys@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: July 24, 2024
• Study Start: July 30, 2024
• Primary Completion: July 31, 2025
• Study Completion: December 31, 2025
• Last Updated: July 24, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)