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

Phase 2

Not yet recruiting

• Conditions: Aplastic Anemia
• Interventions: Drug: Romiplostim; Drug: Immunosuppressive therapy (IST)

• Registration Number: NCT07001254
• Lead Sponsor: Anjali Sharathkumar

Brief 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.

Study Timeline

• Status Verified: 2025-05
• Study First Submitted: 2025-05-23
• Study First Submitted QC: 2025-05-23
• Last Update Submitted: 2025-05-23
• Study First Posted: 2025-06-03
• Last Update Posted: 2025-06-03
• Study Start: 2025-07-31
• Primary Completion: 2031-12-31
• Study Completion: 2031-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)	Romiplostim	Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).
Treatment naïve SAA (Cohort A): Romiplostim and immunosuppressive therapy (IST)	Immunosuppressive therapy (IST)	Treatment naïve SAA (Cohort A) will be treated with romiplostim and immunosuppressive therapy (IST).
Relapsed or refractory SAA (Cohort B): Romiplostim	Romiplostim	Relapsed or refractory SAA (Cohort B) will be treated with romiplostim alone.

Primary Outcome Measures

Name	Time	Method
To evaluate the efficacy of romiplostim added to Immunosuppressive therapy (IST) as measured by the hematologic complete response rate (HCRR) at Week 24	During 24 weeks of therapy	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.

Secondary Outcome Measures

Name	Time	Method
Incidence of adverse events as assessed by CTCAE v5.0	One year following completion of treatment	To further characterize the safety profile
Proportion of participants with a new cytogenetic abnormality	One year following completion of treatment	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).

Trial Locations

Locations (1)

University of Iowa Health Care; 🇺🇸 Iowa City, Iowa, United States