Romiplostim N01NCT07214025
Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children
An observational study of Romiplostim N01 and ITP, run by Assiut University.
Trial summary
The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Romiplostim N01, ITP
- What is being tested?
- Safety and efficacy of Romiplostim in Treatment of chronic ITP in children
- Type of treatment
- Injection or infusion
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- 1 Nov 2025to 1 Jan 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
- Conditions
- Romiplostim N01, ITP
- Being tested
- Safety and efficacy of Romiplostim in Treatment of chronic ITP in children
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy of Romiplostim in Treatment of chronic ITP in childrenDrug
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).
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Up to 48 weeks of treatment
Secondary outcomes (3)
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.
Measured: Up to 48 weeks of treatment.
Need for Rescue Medications
Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy.
Measured: Up to 48 weeks of treatment.
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.
Measured: Up to 48 weeks of treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2025
- Study start1 Nov 2025 (estimated)
- Primary completion1 Nov 2026 (estimated)
- Study completion1 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Romiplostim N01Studies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mostafa Refat Mostafa Ali, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07214025
- Sponsor's protocol ID
- Romiplostim In treatmentof ITP
- Status last verified
- Sep 2025
- First submitted
- 26 Sep 2025
- Last updated
- 9 Oct 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
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. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.
PubMed 19005182Chen 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.
PubMed 34313512Vianelli 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.
PubMed 35201417Liu 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.
PubMed 36658588Singh 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.
PubMed 33669423Sandvad 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.
PubMed 34487679
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children
- ClinicalTrials.gov ID
- NCT07214025
- Acronym / sponsor ID
- Romiplostim In treatmentof ITP
- Conditions
- Romiplostim N01, ITP
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Nov 2025 (estimated)
- Primary completion
- 1 Nov 2026 (estimated)
- Study completion
- 1 Jan 2027 (estimated)
- First posted
- 9 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Safety and efficacy of Romiplostim in Treatment of chronic ITP in childrenChildren 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). | Drug | – |
Primary outcome
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).
3 secondary outcomes
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2025.
- Results publication: PubMed 19005182Rodeghiero 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.
- Results publication: PubMed 34313512Chen 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.
- Results publication: PubMed 35201417Vianelli 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.
- Results publication: PubMed 36658588Liu 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.
- Results publication: PubMed 33669423Singh 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.
- Results publication: PubMed 34487679Sandvad 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.
- Individual participant dataSharing planned: Undecided
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Sepsis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
An observational study of Romiplostim N01 and ITP, run by Assiut University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 9 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mostafa Refat Mostafa, Principal Investigator (+201140514880, moustafa.17298696@aun.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Durable Platelet Response in Patients Receiving Treatment for Chronic ITP (time frame: Up to 48 weeks of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07214025, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2025.