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

Amgen

Status and phase: Withdrawn; Phase 2

Conditions: Severe Aplastic Anemia (SAA)

Treatments: Drug: Romiplostim; Drug: Antithymocyte Globulin; Drug: Cyclosporine A

Study type: Interventional

Funder types: Industry

Identifiers

NCT05323617

20210112

Details and patient eligibility

About

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+)

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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0 participants in 2 patient groups

Arm 1: Previously Untreated IST

Experimental group

Description:

Participants with SAA/vSAA that are previously untreated with IST.

Treatment:

Drug: Antithymocyte Globulin

Drug: Cyclosporine A

Drug: Romiplostim

Arm 2: Refractory IST

Experimental group

Description:

Participants with SAA/vSAA that are refractory to IST.

Treatment:

Drug: Romiplostim