NCT07001254

UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

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

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Anjali Sharathkumar · Academic / Other
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Aplastic Anemia

Interventions

Type	Name	Description
DRUG	Romiplostim	The investigational drug, Romiplostim, is a thrombopoietin receptor agonist (TPO-RA) that has been granted orphan drug designation by the FDA.
DRUG	Immunosuppressive therapy (IST)	Standard of Care immunosuppressive therapy (IST) includes HORSE ANTI-THYMOCYTE GLOBULIN (H-ATG) and Cyclosporine (CSA)

Timeline

• Start date: 2026-08-01
• Primary completion: 2031-12-31
• Completion: 2031-12-31
• First posted: 2025-06-03
• Last updated: 2025-12-19

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study