WithdrawnPhase 2

Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy

Stopped: Operational

0Started 2023-08-31

Plain-language summary

Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) 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 participants with SAA/vSAA. The primary objectives of this study are to: Arm 1: Evaluate the efficacy of romiplostim and IST in adult SAA/vSAA participants who are previously untreated with IST (1L) Arm 2: Evaluate the efficacy of romiplostim treatment in adult SAA/vSAA participants who are refractory to IST (2L+)

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Age ≥ 18 years at time of enrollment * Diagnosis of SAA/vSAA 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: 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 * 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: * Diagnosed as having congenital aplastic anemia (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) * Patients who are eligible for allogenic HCT and have an available matched related donor

What they're measuring

Arms 1 and 2: proportion of participants achieving any hematologic response at week 14

Timeframe: Week 14

Trial details

NCT IDNCT05323617

SponsorAmgen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-25