Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children

Not yet recruiting

• Conditions: Romiplostim N01; ITP

• Registration Number: NCT07214025
• Lead Sponsor: Assiut University

Brief Summary

The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-09
• Study First Submitted: 2025-09-26
• Study First Submitted QC: 2025-10-02
• Last Update Submitted: 2025-10-02
• Study First Posted: 2025-10-09
• Last Update Posted: 2025-10-09
• Study Start: 2025-11-01
• Primary Completion: 2026-11-01
• Study Completion: 2027-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• Patients aged between 1 and 18 years
• Diagnosis of chronic ITP
• Refractory to or intolerant of first-line therapies (e.g.corticosteroids , intravenous immunoglobulin [ IVIG ] , and oral eltrombopag )
• Platelet count<30,000/µL, or evidence of bleeding or risk of bleeding with platelet count<50,000/µL.
• Written informed consent (or parental/guardian consent for minors)

Exclusion Criteria

• Evidence of secondary thrombocytopenia (e.g., HIV, HCV, systemic lupus erythematosus)
• congenital thrombocytopenia
• Age < 1year or more than 18 years
• Aplastic anemia
• Known hypersensitivity to romiplostim or any of its components

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Durable Platelet Response in Patients Receiving Treatment for Chronic ITP	Up to 48 weeks of treatment	The proportion of patients achieving a durable platelet response, defined as platelet counts ≥50 × 10⁹/L for at least 6 of the final 8 weeks of a 48-week treatment period, without the need for rescue therapy (e.g., IVIG, corticosteroids, platelet transfusion).

Secondary Outcome Measures

Name	Time	Method
Time to Initial Platelet Response	Up to 48 weeks of treatment.	Time (in days) from initiation of Romiplostim therapy to the first platelet count ≥50×10⁹/L without rescue medication in the preceding 7 days.
Need for Rescue Medications	Up to 48 weeks of treatment.	Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy.
Rate of Treatment-Free Remission	Up to 48 weeks of treatment.	Proportion of patients who maintain platelet counts ≥50×10⁹/L for at least 24 consecutive weeks after discontinuation of Romiplostim without rescue therapy.