Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 18
SponsorAssiut University
About this trial
The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
Evidence of secondary thrombocytopenia (e.g., HIV, HCV, systemic lupus erythematosus)
congenital thrombocytopenia
Age < 1year or more than 18 years
Aplastic anemia
Trial population
The study will include pediatric patients aged 1 to 18 years with a diagnosis of chronic immune thrombocytopenia (ITP) who are refractory to or intolerant of first-line therapies (e.g., corticosteroids, intravenous immunoglobulin, or oral eltrombopag). Eligible patients must have a platelet count \<30,000/µL, or evidence of bleeding/risk of bleeding with platelet count \<50,000/µL. Patients with secondary or congenital thrombocytopenia, aplastic anemia, age \<1 year or \>18 years, or known hypersensitivity to romiplostim will be excluded. A total of 100 patients will be enrolled from the Pediatric Department at Assiut University Hospital.
Trial design
Design model
Other
Time perspective
Cross-sectional
Treatments tested in this trial
Safety and efficacy of Romiplostim in Treatment of chronic ITP in children
DrugChildren 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).
Treatment groups
No treatment groups listed
Trial outcomes
Primary outcomes
1
Durable Platelet Response in Patients Receiving Treatment for Chronic ITP
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).
Time frame
Up to 48 weeks of treatmentSecondary outcomes
1
Time to Initial Platelet Response
Time (in days) from initiation of Romiplostim therapy to the first platelet count ≥50×10⁹/L without rescue medication in the preceding 7 days.
Time frame
Up to 48 weeks of treatment.2
Need for Rescue Medications
Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy.
Time frame
Up to 48 weeks of treatment.3
Rate of Treatment-Free Remission
Proportion of patients who maintain platelet counts ≥50×10⁹/L for at least 24 consecutive weeks after discontinuation of Romiplostim without rescue therapy.
Time frame
Up to 48 weeks of treatment.Locations
Sponsors and contacts
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Source ClinicalTrials.gov
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