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

NCT07214025 | Not Yet Recruiting FDA Drug

• 1 other identifier: Romiplostim In treatmentof ITP
• Study Type: observational
• Target: 100
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Romiplostim N01; ITP

Outcome Measures

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).

  Up to 48 weeks of treatment

Secondary Outcomes (3)

• Time to Initial Platelet Response

  Up to 48 weeks of treatment.

• Need for Rescue Medications

  Up to 48 weeks of treatment.

• Rate of Treatment-Free Remission

  Up to 48 weeks of treatment.

Interventions

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).

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study 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.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Assiut University: lead

Related Publications (6)

• 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. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

  PMID: 19005182 RESULT

• 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 RESULT

• 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 RESULT

• 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 RESULT

• 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 RESULT

• 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 RESULT

MeSH Terms

Interventions

Safety

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Central Study Contacts

Mostafa Refat Mostafa, Principal Investigator

CONTACT

+201140514880; moustafa.17298696@aun.edu.eg

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 26, 2025
• First Posted: October 9, 2025
• Study Start: November 1, 2025
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: October 9, 2025

Record last verified: 2025-09