Safety and Efficacy of Romiplostim in T… | Clinical Trial

Key information

Conditions

Romiplostim N01 ITP

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Safety and efficacy of Romiplostim in Treatment of chronic ITP in children

Drug

Children diagnosed with chronic immune thrombocytopenia (ITP) will receive Romiplostim, a thrombopoietin receptor agonist administered as a once-weekly subcutaneous injection. The initial dose will be 1 µg/kg, with subsequent dose adjustments (up to a maximum of 10 µg/kg weekly) based on platelet response, in order to maintain platelet counts ≥50 × 10⁹/L while minimizing the risk of bleeding. Treatment duration will be determined according to study protocol, and patients will be monitored regularly for efficacy (platelet count response, bleeding events) and safety (adverse events, laboratory parameters).

Primary outcomes

Durable Platelet Response in Patients Receiving Treatment for Chronic ITP

Time frame: 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 outcomes

Time to Initial Platelet Response

Time frame: 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

Time frame: 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

Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Mostafa Refat Mostafa, Principal Investigator

CONTACT

[email protected]

+201140514880

Sponsors and collaborators

Lead sponsor

Assiut University

Other

Registry information

Important dates

Study start: 2025
Primary completion: 2026
Study completion: 2027
First posted: Oct 9, 2025
Registry last updated: Oct 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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