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

NCT06535685 | Not Yet Recruiting Phase 4

• 1 other identifier: LRA-2024-002
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Aplastic Anemia

Outcome Measures

Primary Outcomes (4)

• Overall response rate (ORR)

  Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC \<0.5 × 10\^9/L increased by at least 0.5 × 10\^9/L after treatment, or initial PLT \<20 × 10\^9/L increased by at least 20 × 10\^9/L after treatment.

  3 months

• Overall response rate (ORR)

  Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC \<0.5 × 10\^9/L increased by at least 0.5 × 10\^9/L after treatment, or initial PLT \<20 × 10\^9/L increased by at least 20 × 10\^9/L after treatment.

  6 months

• CRR

  CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.

  3 months

• CRR

  CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.

  6 months

Secondary Outcomes (2)

• safety events

  3 months

• safety events

  6months

Study Arms (1)

Romiplostim group

EXPERIMENTAL

Romiplostim group

Drug: Romiplostim

Interventions

Romiplostim DRUG

Enrolled patients will be given Romiplostim (20 µg/kg subcutaneously once a week) for a minimum of 3 months.

Romiplostim group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

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%.
• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

Related Publications (4)

• Young NS. Aplastic anaemia. Lancet. 1995 Jul 22;346(8969):228-32. doi: 10.1016/s0140-6736(95)91273-8. No abstract available.

  PMID: 7616805 BACKGROUND

• Young NS. Aplastic Anemia. N Engl J Med. 2018 Oct 25;379(17):1643-1656. doi: 10.1056/NEJMra1413485. No abstract available.

  PMID: 30354958 BACKGROUND

• 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.

  PMID: 28096088 BACKGROUND

• Yang C, Zhang X. Incidence survey of aplastic anemia in China. Chin Med Sci J. 1991 Dec;6(4):203-7.

  PMID: 1813058 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 Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: chief physician

Study Record Dates

• First Submitted: July 24, 2024
• First Posted: August 2, 2024
• Study Start: August 2, 2024
• Primary Completion: March 30, 2025
• Study Completion: December 31, 2025
• Last Updated: August 2, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)