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.#
Anjali Sharathkumar
Lead sponsor
University of Iowa
Sponsor institution
Department of Health and Human Services
Collaborator
Food and Drug Administration (FDA)
Collaborator