Efficacy of Romiplostim in Treatment of Severe Aplastic Anemia in Non-Asian Adults Previously Untreated With or Refractory to Immunosuppressive Therapy

Phase 2

• Conditions: Severe Aplastic Anemia (SAA)

• Registration Number: JPRN-jRCT2031230178
• Lead Sponsor: Murakami Contact Naoko

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-06-25
• Last Update Posted: 1 April 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 0

Inclusion Criteria

1. Age >= 18 years at time of enrollment
2. Diagnosis of SAA/vSAA confirmed by blood, bone marrow, and cytogenetic studies
3. An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening
4. Arm 1 only: participant requires initial treatment for SAA/vSAA, no matched related donor is available for allogenic hematopoietic cell transplantation (HCT) and will begin IST with antithymocyte globulin and CsA
5. Arm 2 only: refractory to at least one course of immunosuppressive therapy including horse or rabbit ATG; or ineligible for ATG treatment and refractory to CsA

Exclusion Criteria

1. Diagnosed as having congenital aplastic anemia (AA) (Fanconi anemia, congenital dyskeratosis, etc)
2. History of other malignancy within the past 5 years, with exceptions.
3. Aplastic anemia with hemolytic paroxysmal nocturnal hemoglobinuria (PNH) (hemolytic predominant is defined as lactate dehydrogenase (LDH) > 1.5 x the upper limit of site normal
4. Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab
5. Previously treated with PEGylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin protein (TPO), romiplostim and other TPO-receptor
agonist (eltrombopag, etc)
6. Patients who are eligible for allogenic HCT and have an available matched related donor

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1. Arms 1 and 2: proportion of participants achieving any hematologic response at week 14[ Time Frame: Week 14 ]<br>Proportion of participants achieving any hematologic response at week 14 based on response criteria:<br> - Platelet response<br> - Erythroid response<br> - Red blood cell count<br> - Hemoglobin concentration<br> - Neutrophil response