Phase II Open-label Clinical Trial Evaluating Efficacy of Romiplostim Added to Standard of Care for Children and Young Adults With Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
Anjali Sharathkumar
Summary
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.
Description
The study is designed as a Phase II, multicenter, investigator-initiated, open label, interventional study that will recruit children (age: \>2 to \<21 years) with SAA. The primary objective of the study is to evaluate the efficacy of romiplostim (a TPO-RA with an orphan drug designation) for the treatment of SAA in children and young adults with newly diagnosed and relapsed or refractory SAA. Hematologic complete response (HCR) will be used to assess the therapy response.
Eligibility
- Age range
- 2–21 years
- Sex
- All
- Healthy volunteers
- No
INCLUSION CRITERIA In order to be eligible to participate in this study, an individual must meet all of the following criteria: * 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. Diagnosis of severe Aplastic anemia (newly diagnosed or refractory based on history of prior treatments) is established if bone marrow cellularity \<25% to 30% and at least two of the following criteria are met: (a) absolute neutrophil count \<0.5 × 10\^9 /L, (b) pla…
Interventions
- DrugRomiplostim
The investigational drug, Romiplostim, is a thrombopoietin receptor agonist (TPO-RA) that has been granted orphan drug designation by the FDA.
- DrugImmunosuppressive therapy (IST)
Standard of Care immunosuppressive therapy (IST) includes HORSE ANTI-THYMOCYTE GLOBULIN (H-ATG) and Cyclosporine (CSA)
Location
- University of Iowa Health CareIowa City, Iowa