This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage hematopoiesis in children and young adults.
Age ≥2 years to ≤21 years
Child should be receiving ongoing care with pediatric hematology/oncology provider.
Confirmed Diagnosis of SAA and other related conditions based on following criteria.
Females of childbearing potential agree to use effective contraception during the study period and for 4 months after completion of therapy.
Age < 2 years or >21 years *Availability of suitable HLA-matched related or HLA-matched unrelated (9/10 or 10/10) allogenic stem cell donor and the participant fulfills the requirement for HSCT and opts to undergo allogenic HSCT.**
Patients with Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones >50% of granulocytes or RBC at time of enrollment.
Preexisting condition with predisposition for thrombosis such as protein C, S, antithrombin deficiency, homozygous factor V Leiden or prothrombin 20210 polymorphism, history of idiopathic thromboembolism with patient or first degree relative.
Diagnosis of bone marrow failure syndrome with cancer predisposition, including chromosomal fragility disorders (Fanconi anemia, Bloom syndrome, Ataxia Telangiectasia) and other conditions with known association towards cancer predisposition.#
Single group
Romiplostim
DrugThe investigational drug, Romiplostim, is a thrombopoietin receptor agonist (TPO-RA) that has been granted orphan drug designation by the FDA.
Immunosuppressive therapy (IST)
DrugStandard of Care immunosuppressive therapy (IST) includes HORSE ANTI-THYMOCYTE GLOBULIN (H-ATG) and Cyclosporine (CSA)
To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24
Proportion of participants with hematopoietic complete response at 24 weeks defined as hemoglobin ≥10 g/dL, ANC ≥1 x 10\^9/L, and platelet count ≥100 x 10\^9/L without having received transfusion of packed red blood cells within the last 6 weeks or platelets or G-CSF/GM-CSF within the last 2 weeks.
Incidence of adverse events as assessed by CTCAE v5.0
To further characterize the safety profile
Proportion of participants with a new cytogenetic abnormality
For safety the proportion of participants with a new cytogenetic abnormality (e.g., paroxysmal nocturnal hemoglobinuria (PNH), acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS), thrombocytosis, and bone marrow fibrosis).
Click on the lead sponsor to view all of their trials.
Anjali Sharathkumar
Lead sponsor
University of Iowa
Sponsor institution
Department of Health and Human Services
Collaborator
Food and Drug Administration (FDA)
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
