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Not yet recruiting NCT07214025

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

— Observational Romiplostim N01 ITP

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Safety and efficacy of Romiplostim in Treatment of chronic ITP in children.
Who it may be relevant to
Registry conditions: Romiplostim N01, ITP. Basic parameters: up to 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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

Interventions

  • Drug Safety and efficacy of Romiplostim in Treatment of chronic ITP in children
    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 regula

Primary outcome measures

  • Durable Platelet Response in Patients Receiving Treatment for Chronic ITP [Time frame: Up to 48 weeks of treatment]
Secondary outcome measures (3)
  • Time to Initial Platelet Response [Time frame: Up to 48 weeks of treatment.]
  • Need for Rescue Medications [Time frame: Up to 48 weeks of treatment.]
  • Rate of Treatment-Free Remission [Time frame: Up to 48 weeks of treatment.]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

Center list to be confirmed — check the primary protocol.

Publications

  • Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 200 PMID 19005182
  • Chen F, McDonald V, Newland A. Experts' review: the emerging roles of romiplostim in immune thrombocytopenia (ITP). Expert Opin Biol Ther. 2021 Nov;21(11):1383-1393. doi: 10.1080/14712598.2021.1960979. Epub 2021 Aug 12. PMID 34313512
  • Vianelli N, Auteri G, Buccisano F, Carrai V, Baldacci E, Clissa C, Bartoletti D, Giuffrida G, Magro D, Rivolti E, Esposito D, Podda GM, Palandri F. Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives. Ann Hematol. 2022 May;101(5):963-978. doi: 10.1007/s00277-022-04786-y. Epub 2022 Feb 24. PMID 35201417
  • Liu XG, Hou Y, Hou M. How we treat primary immune thrombocytopenia in adults. J Hematol Oncol. 2023 Jan 19;16(1):4. doi: 10.1186/s13045-023-01401-z. PMID 36658588
  • Singh A, Uzun G, Bakchoul T. Primary Immune Thrombocytopenia: Novel Insights into Pathophysiology and Disease Management. J Clin Med. 2021 Feb 16;10(4):789. doi: 10.3390/jcm10040789. PMID 33669423
  • Sandvad M, Pedersen EA, Frederiksen H, Mannering N. Risk of infection in adult patients with primary immune thrombocytopenia (ITP): a systematic review. Expert Rev Hematol. 2021 Oct;14(10):961-974. doi: 10.1080/17474086.2021.1976635. Epub 2021 Sep 16. PMID 34487679

Identifiers

NCT: NCT07214025 · Romiplostim In treatmentof ITP

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗