Persistent or Chronic ITP Not Respnding to SteroidsNCT06568913
The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
A study testing CBC in people with Persistent or Chronic ITP Not Respnding to Steroids, run by Sohag University at 1 site in Egypt.
Trial summary
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Persistent or Chronic ITP Not Respnding to Steroids
- What is being tested?
- CBC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteEgypt
- Study dates
- 14 Jul 2024to 14 Jul 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- pediatric patients with persistent or chronic Itp on thrombopoietin receptor agonists unresponsive to steroid.
Who cannot join
- Any patient with other causes of thrombocytopenia. Any patient with chronic disease . Any patient with Acute ITP . Any newly diagnosed patient with ITP.
Trial locations and contacts
1 site in 1 country.
ALZahraa Elsayed Ahmed, Professor contact
Egypt1 site, 1 recruiting
- Recruiting
Sohag University hospitals
Sohag
Magdy M Ameen, Professor
About this study
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. It leads to the accelerated removal of these cells by antigen-presenting cells (macrophages) of the spleen and sometimes the liver or other components of the monocyte-macrophage system. The bone marrow compensates for platelet destruction by increasing platelet production. ITP most often occurs in healthy children and young adults within a few weeks following a viral infection .
New treatment guidelines have supported a shift from corticosteroids and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor agonists (TPO-RA), romiplostim, eltrombopag have markedly altered the treatment of ITP.
Read more
The thrombopoietin receptor agonists (TPO-Ras) romiplostim and eltrombopag have shown high therapeutic activity In primary ITP.
Romiplostim, a thrombopoiesis-stimulating peptibody, represents a new therapeutic option in adult refractory chronic immune thrombocytopenia (ITP). This study aimed to assess the short-term efficacy and safety of romiplostim in children withChronic ITP.
The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation.
TPO-RA do not need to be continued forever; about a third of patients In the first year and about another third after two years have a remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TPO-RA, their place in the treatment algorithm, unique aspects of their clinical use, adverse effects.
- Being tested
- CBC
- Keywords
- CBC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CBCDiagnostic test
Platelets counts in pediatric patient with perisistent or chronicITP unresponsive to steroids
| Study group | Role | What they receive |
|---|---|---|
| Persistent or chronic ITP not responsive to steroids | Experimental | The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids Receives: Diagnostic Test: CBC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists
The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2024
- Study start14 Jul 2024
- Primary completion14 Jul 2025 (estimated)
- Study completion14 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Persistent or Chronic ITP Not Respnding to SteroidsStudies of the same condition, open and under way.
- More trials run by Sohag 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Model description
- follow up of platelets count and The outcome of the thrombopoietin receptor agonists in pediatric patient with persistent or chronic ITP unresponsive to steroids
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sohag University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shimaa Abdelhameed Mohamed, Sohag University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06568913
- Sponsor's protocol ID
- Soh-Med-24-07-12MS
- Status last verified
- Aug 2024
- First submitted
- 21 Aug 2024
- Last updated
- 23 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Zaja F, Barcellini W, Cantoni S, Carpenedo M, Caparrotti G, Carrai V, Di Renzo N, Santoro C, Di Nicola M, Veneri D, Simonetti F, Liberati AM, Ferla V, Paoloni F, Crea E, Volpetti S, Tuniz E, Fanin R. Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study. Am J Hematol. 2016 May;91(5):E293-5. doi: 10.1002/ajh.24341. Epub 2016 Apr 4.
PubMed 26910388Mokhtar GM, Tantawy AA, El Sherif NH. Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia. Platelets. 2012;23(4):264-73. doi: 10.3109/09537104.2011.619601. Epub 2012 Apr 3.
PubMed 22471399Bussel JB, Soff G, Balduzzi A, Cooper N, Lawrence T, Semple JW. A Review of Romiplostim Mechanism of Action and Clinical Applicability. Drug Des Devel Ther. 2021 May 26;15:2243-2268. doi: 10.2147/DDDT.S299591. eCollection 2021.
PubMed 34079225Giordano P, Lassandro G, Barone A, Cesaro S, Fotzi I, Giona F, Ladogana S, Miano M, Marzollo A, Nardi M, Notarangelo LD, Pession A, Ruggiero A, Russo G, Saracco P, Spinelli M, Tolva A, Tornesello A, Palladino V, Del Vecchio GC. Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP). Front Med (Lausanne). 2020 Feb 28;7:66. doi: 10.3389/fmed.2020.00066. eCollection 2020.
PubMed 32181255
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
- ClinicalTrials.gov ID
- NCT06568913
- Acronym / sponsor ID
- Soh-Med-24-07-12MS
- Keywords
- CBC
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Sohag UniversityUniversity, hospital or other
- Study start
- 14 Jul 2024 (actual)
- Primary completion
- 14 Jul 2025 (estimated)
- Study completion
- 14 Jul 2025 (estimated)
- First posted
- 23 Aug 2024 (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.
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. It leads to the accelerated removal of these cells by antigen-presenting cells (macrophages) of the spleen and sometimes the liver or other components of the monocyte-macrophage system. The bone marrow compensates for platelet destruction by increasing platelet production. ITP most often occurs in healthy children and young adults within a few weeks following a viral infection .
New treatment guidelines have supported a shift from corticosteroids and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor agonists (TPO-RA), romiplostim, eltrombopag have markedly altered the treatment of ITP.
The thrombopoietin receptor agonists (TPO-Ras) romiplostim and eltrombopag have shown high therapeutic activity In primary ITP.
Romiplostim, a thrombopoiesis-stimulating peptibody, represents a new therapeutic option in adult refractory chronic immune thrombocytopenia (ITP). This study aimed to assess the short-term efficacy and safety of romiplostim in children withChronic ITP.
The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation.
TPO-RA do not need to be continued forever; about a third of patients In the first year and about another third after two years have a remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TPO-RA, their place in the treatment algorithm, unique aspects of their clinical use, adverse effects.
Study design
- Primary purpose
- Health services research
- Allocation
- Not applicable
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- follow up of platelets count and The outcome of the thrombopoietin receptor agonists in pediatric patient with persistent or chronic ITP unresponsive to steroids
| Arm | Type | Receives |
|---|---|---|
| Persistent or chronic ITP not responsive to steroids | Experimental | Diagnostic Test: CBCThe Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- pediatric patients with persistent or chronic Itp on thrombopoietin receptor agonists unresponsive to steroid.
Key exclusion criteria (1 listed)
- Any patient with other causes of thrombocytopenia. Any patient with chronic disease . Any patient with Acute ITP . Any newly diagnosed patient with ITP.
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 |
|---|---|---|
| CBCPlatelets counts in pediatric patient with perisistent or chronicITP unresponsive to steroids | Diagnostic test | Persistent or chronic ITP not responsive to steroids |
Primary outcome
Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists
Time frame: 12 months
The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2024.
- Background: PubMed 26910388Zaja F, Barcellini W, Cantoni S, Carpenedo M, Caparrotti G, Carrai V, Di Renzo N, Santoro C, Di Nicola M, Veneri D, Simonetti F, Liberati AM, Ferla V, Paoloni F, Crea E, Volpetti S, Tuniz E, Fanin R. Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study. Am J Hematol. 2016 May;91(5):E293-5. doi: 10.1002/ajh.24341. Epub 2016 Apr 4.
- Background: PubMed 22471399Mokhtar GM, Tantawy AA, El Sherif NH. Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia. Platelets. 2012;23(4):264-73. doi: 10.3109/09537104.2011.619601. Epub 2012 Apr 3.
- Background: PubMed 34079225Bussel JB, Soff G, Balduzzi A, Cooper N, Lawrence T, Semple JW. A Review of Romiplostim Mechanism of Action and Clinical Applicability. Drug Des Devel Ther. 2021 May 26;15:2243-2268. doi: 10.2147/DDDT.S299591. eCollection 2021.
- Background: PubMed 32181255Giordano P, Lassandro G, Barone A, Cesaro S, Fotzi I, Giona F, Ladogana S, Miano M, Marzollo A, Nardi M, Notarangelo LD, Pession A, Ruggiero A, Russo G, Saracco P, Spinelli M, Tolva A, Tornesello A, Palladino V, Del Vecchio GC. Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP). Front Med (Lausanne). 2020 Feb 28;7:66. doi: 10.3389/fmed.2020.00066. eCollection 2020.
- Individual participant dataSharing planned: Undecided
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. This study is not recruiting at the moment.
ALZahraa Elsayed Ahmed, Professor contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CBC in people with Persistent or Chronic ITP Not Respnding to Steroids, run by Sohag University at 1 site in Egypt.
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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 23 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact shaimaa abdel hameed, Resident (01154450851, shaymaa.abdelhameed@med.sohag.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists (time frame: 12 months). 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 NCT06568913, which also lists 4 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 23 Aug 2024.