Clinical trial NCT05323617: Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
Official title: Two Arm Bridging Study to Evaluate the Efficacy of Romiplostim in the Treatment of Adult Severe Aplastic Anemia Participants Who Are Either Previously Untreated With IST or Refractory to IST
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Severe Aplastic Anemia (SAA)
Keywords: Severe Aplastic Anemia
SAA
Very Severe Aplastic Anemia
vSAA
Romiplostim
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2022-04-12
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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+)


Eligibility 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

Primary outcomes:
measure: Arms 1 and 2: proportion of participants achieving any hematologic response at week 14
timeFrame: Week 14
Secondary outcomes:
measure: Arm 1: number of participants who achieve a complete response (CR) or partial response (PR) at week 14
timeFrame: Week 14
measure: 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
measure: Arms 1 and 2: number of participants with serious adverse events
timeFrame: 24 Weeks
measure: Arms 1 and 2: number of participants with clinically significant changes in laboratory values
timeFrame: 24 Weeks
measure: 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
measure: Arms 1 and 2: serum romiplostim trough concentrations
timeFrame: Prior to romiplostim administration on Weeks 1, 2, 4, 5, 9, 13, and 24
measure: Arms 1 and 2: maximum serum concentration (Cmax) of romiplostim
timeFrame: Weeks 1, 2, 4, 5, 9, 13, and 24
measure: Arms 1 and 2: area under the curve (AUC) of romiplostim
timeFrame: Weeks 1, 2, 4, 5, 9, 13, and 24
measure: Arms 1 and 2: time to reach maximum concentration (tmax) of romiplostim
timeFrame: Weeks 1, 2, 4, 5, 9, 13, and 24
measure: Arms 1 and 2: half-life (t1/2) of romiplostim
timeFrame: Weeks 1, 2, 4, 5, 9, 13, and 24
measure: Arms 1 and 2: number of participant with anti-romiplostim antibodies
timeFrame: Prior to romiplostim administration on Weeks 1 and 13
measure: Arms 1 and 2: number of participants with antibodies to thrombopoietin
timeFrame: Prior to romiplostim administration on Weeks 1 and 13
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: