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Terminated/Withdrawn
Sponsor ID
20210112

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

Sponsor ID
20210112
Clinicaltrials.gov ID
NCT05323617
EUCTIS ID
N/A
EudraCT ID
N/A
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Study Details

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+)

Additional Study Details

Phase
Phase 2
Product
romiplostim
Type
Interventional
Masking
None (Open Label)
Enrollment
0
Additional Study Details

Protocol Summary

Primary Outcome Measures

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

Timeframe: Week 14

Secondary Outcome Measures

Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14

Timeframe: 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

Timeframe: Week 14

Arms 1 and 2: number of participants with serious adverse events

Timeframe: 24 Weeks

Arms 1 and 2: number of participants with clinically significant changes in laboratory values

Timeframe: 24 Weeks

Arms 1 and 2: change from baseline in Gruppo Italiano Malattie Ematologiche Maligne dell’Adulto (GIMEMA) bleeding scale at week 14

Timeframe: Baseline and Week 14

Arms 1 and 2: serum romiplostim trough concentrations

Timeframe: Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24

Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim

Timeframe: Weeks 1, 2, 4, 5, 9, 13, and 24

Arms 1 and 2: area under the curve (AUC) of romiplostim

Timeframe: Weeks 1, 2, 4, 5, 9, 13, and 24

Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim

Timeframe: Weeks 1, 2, 4, 5, 9, 13, and 24

Arms 1 and 2: half-life (t1/2) of romiplostim

Timeframe: Weeks 1, 2, 4, 5, 9, 13, and 24

Arms 1 and 2: number of participant with anti-romiplostim antibodies

Timeframe: Prior to romiplostim administration on Weeks 1 and 13

Arms 1 and 2: number of participants with antibodies to thrombopoietin

Timeframe: Prior to romiplostim administration on Weeks 1 and 13

Locations

No locations found.
Study Info
Age
18+ years
Condition
  • Anemia
    • Sex
    All
    Trial Dates
    Aug 2023 - Feb 2025
    Inclusion and Exclusion Criteria
    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