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

• Status: Not yet recruiting
• Trial end date: 2024-01-26
• Target enrollment: 0
• Participant gender: All

Summary

Romiplostim has been used in clinical trials for the treatment of severe aplastic anemia (SAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to IST. This study will evaluate the efficacy of romiplostim in the treatment of non-Asian participants with SAA.

The primary objectives of this study are to:

Arm 1: evaluate the efficacy of romiplostim and immunosuppressive therapy (IST) in adult non-Asian severe aplastic anemia (SAA) participants who are previously untreated with IST (1L)

Arm 2: evaluate the efficacy of romiplostim treatment in adult non-Asian SAA participants who are refractory to IST (2L+)

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amgen
• Treatments: Antilymphocyte Serum; Cyclosporine; Cyclosporins

Criteria

Inclusion Criteria:

• Age ≥ 18 years at time of enrollment

• Diagnosis of severe aplastic anemia (AA) or very severe AA confirmed by blood, bone marrow, and cytogenetic studies

• An Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 1 at screening

• Arm 1 only: considered to require new treatment with anti-thymocyte globulin (ATG) and cyclosporine A (CsA)

• 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

• Arm 2 only: thrombocytopenia defined as a platelet count of ≤ 30 x 10⁹/L

Exclusion Criteria:

• Participants with Asian ethnicity

• Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc)

• History of other malignancy within the past 5 years, with exceptions.

• 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

• Arm 1 only: Previously treated with ATG, CsA, or Alemtuzumab

• 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)