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+)
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Administered as a subcutaneous injection.
Horse or rabbit antithymocyte globulin administered as an intravenous infusion.
Administered orally.
Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
Proportion of participants achieving any hematologic response at week 14 based on response criteria: * Platelet response * Erythroid response * Red blood cell count * Hemoglobin concentration * Neutrophil response
Time frame: Week 14
Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14
Time frame: Week 14
Arms 1 and 2: number of participants who have a decrease in frequency of platelet and/or red blood cell (RBC) transfusions, or become platelet and/or RBC transfusion independent at week 14
Time frame: Week 14
Arms 1 and 2: number of participants with serious adverse events
Time frame: 24 Weeks
Arms 1 and 2: number of participants with clinically significant changes in laboratory values
Time frame: 24 Weeks
Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) bleeding scale at week 14
The Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto is as follows: 0: No bleeding 1. Petecjoae or mucosal or retinal bleeding that did not require red-cell transfusion 2. Melena, hematemesis, hematuria, or hemoptysis 3. Any bleeding that required red-cell transfusion 4. Retinal bleeding accompanied by visual impairment 5. Nonfatal cerebral bleeding 6. Fatal cerebral bleeding 7. Fatal noncerebral bleeding
Time frame: Baseline and Week 14
Arms 1 and 2: serum romiplostim trough concentrations
Time frame: Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24
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Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: area under the curve (AUC) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: half-life (t1/2) of romiplostim
Time frame: Weeks 1, 2, 4, 5, 9, 13, and 24
Arms 1 and 2: number of participant with anti-romiplostim antibodies
Time frame: Prior to romiplostim administration on Weeks 1 and 13
Arms 1 and 2: number of participants with antibodies to thrombopoietin
Time frame: Prior to romiplostim administration on Weeks 1 and 13