Thrombopoietin Receptor AgonistNCT05846529
Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP
An observational study of Thrombopoietin Receptor Agonist, run by Assiut University.
Trial summary
This study aimed to evaluate the response rate and safety of oral thrombopoietin receptor agonist (Eltrombopag drug) used for children with chronic immune thrombocytopenia in patients attending Assiut university children's hospital. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Thrombopoietin Receptor Agonist
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Jun 2023to Jun 2024
- Phase
- Observational
- 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 2 to 18 years
- All sexes
- Child, Adult
Study population: Infants and children between age of (2 year to <18 years old)
Who may be able to join
- Subjects between 2 year and <18 years of age in both sex
- Confirmed diagnosis of chronic ITP
- all patient received oral thrombopoietin receptor agonist (Eltrombopag) for 6 months or more
Who cannot join
- age of the patient < 2years and >18 years
- secondary thrombocytopenia
- Concurrent or past malignant disease, including myeloproliferative disorder
- patient with platelet agglutination abnormality that prevents reliable measurement of platelet counts
- patient with bone marrow aplasia
About this study
Official title: Assessment of Response Rate and Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP in Assiut.
This study aimed to evaluate the response rate and safety of oral thrombopoietin receptor agonist (Eltrombopag drug) used for children with chronic immune thrombocytopenia in patients attending Assiut university children's hospital.
Read the detailed description
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by decrease number of the platelets due to destruction of the platelets by antiplatelet antibodies. Also, platelet production can be reduced because the antiplatelet antibodies can also damage megakaryocytes . Its prevalence being approximately 8 per 100,000 children It is characterized by temporary or persistent reduction of the platelet number to less than 100 × 109/liter.
All new cases at diagnosis are defined as acute ITP. While, Persistent ITP is defined as ITP lasting between 3 and 12 months from diagnosis while chronic ITP is defined as the presence of ITP for more than one-year ITP Patients have an increased hazard of bleeding that may have no effect on patient's life or have life-threatening danger the goal of treatment in chronic ITP is to increase and then keep platelets in a sufficient number to prevent bleeding
. Meanwhile most life-threatening bleeding occurs with platelet number less than 30 × 109/liter.
New guideline recommend that treatment constricted on symptomatic patients with number lower than 30 × 109/liter (3,6) new Guidelines recommended that corticosteroids as first-line therapy and splenectomy considered as one of several second-line therapies. Other choices exist for ITP patients with insufficient response or who do not undergo to splenectomy including IVIg, IV anti-D, Rituximab, danazol, azathioprine, Vinca alkaloids and cyclophosphamide New consensus statements and guidelines recommend thrombopoietin-receptor (TPO-R) agonists as second and third line of treatment Eltrombopag consider the first oral, nonpeptide TPO-R agonist accepted for the treatment of chronic ITP in patients with insufficient response to at least one other therapy It rises platelet number by increase platelet production by binding to the transmembrane domain of the TPO-R. It does not compete with endogenous TPO in vitro and it enhance production and differentiation of BM progenitor cells in the megakaryocytic lineage
- Conditions
- Thrombopoietin Receptor Agonist
What is being measured
The primary outcome is the main question the study is designed to answer.
response to oral thrombopoietin receptor agonist
assess haematological response to oral thrombopoietin receptor agonist (Eltrombopag) therapy
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2023
- Study startJun 2023 (estimated)
- Primary completionApr 2024 (estimated)
- Study completionJun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Thrombopoietin Receptor AgonistStudies 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
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohamed Salama Sayed Hassan, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05846529
- Sponsor's protocol ID
- thrombopoietin agonist
- Status last verified
- Apr 2023
- First submitted
- 7 Apr 2023
- Last updated
- 6 May 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Kuter DJ. The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Rev. 2022 May;53:100909. doi: 10.1016/j.blre.2021.100909. Epub 2021 Nov 16.
PubMed 34815110Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. doi: 10.1111/bjh.15573. Epub 2018 Sep 7.
PubMed 30191972
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Response Rate and Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP in Assiut.
- ClinicalTrials.gov ID
- NCT05846529
- Acronym / sponsor ID
- thrombopoietin agonist
- Conditions
- Thrombopoietin Receptor Agonist
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- Jun 2023 (estimated)
- Primary completion
- Apr 2024 (estimated)
- Study completion
- Jun 2024 (estimated)
- First posted
- 6 May 2023 (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.
This study aimed to evaluate the response rate and safety of oral thrombopoietin receptor agonist (Eltrombopag drug) used for children with chronic immune thrombocytopenia in patients attending Assiut university children's hospital.
Detailed description
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by decrease number of the platelets due to destruction of the platelets by antiplatelet antibodies. Also, platelet production can be reduced because the antiplatelet antibodies can also damage megakaryocytes . Its prevalence being approximately 8 per 100,000 children It is characterized by temporary or persistent reduction of the platelet number to less than 100 × 109/liter.
All new cases at diagnosis are defined as acute ITP. While, Persistent ITP is defined as ITP lasting between 3 and 12 months from diagnosis while chronic ITP is defined as the presence of ITP for more than one-year ITP Patients have an increased hazard of bleeding that may have no effect on patient's life or have life-threatening danger the goal of treatment in chronic ITP is to increase and then keep platelets in a sufficient number to prevent bleeding
. Meanwhile most life-threatening bleeding occurs with platelet number less than 30 × 109/liter.
New guideline recommend that treatment constricted on symptomatic patients with number lower than 30 × 109/liter (3,6) new Guidelines recommended that corticosteroids as first-line therapy and splenectomy considered as one of several second-line therapies. Other choices exist for ITP patients with insufficient response or who do not undergo to splenectomy including IVIg, IV anti-D, Rituximab, danazol, azathioprine, Vinca alkaloids and cyclophosphamide New consensus statements and guidelines recommend thrombopoietin-receptor (TPO-R) agonists as second and third line of treatment Eltrombopag consider the first oral, nonpeptide TPO-R agonist accepted for the treatment of chronic ITP in patients with insufficient response to at least one other therapy It rises platelet number by increase platelet production by binding to the transmembrane domain of the TPO-R. It does not compete with endogenous TPO in vitro and it enhance production and differentiation of BM progenitor cells in the megakaryocytic lineage
Study design
- Observational model
- Case-control
- Time perspective
- Retrospective
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Infants and children between age of (2 year to <18 years old)
Key inclusion criteria (3 listed)
- Subjects between 2 year and <18 years of age in both sex
- Confirmed diagnosis of chronic ITP
- all patient received oral thrombopoietin receptor agonist (Eltrombopag) for 6 months or more
Key exclusion criteria (5 listed)
- age of the patient < 2years and >18 years
- secondary thrombocytopenia
- Concurrent or past malignant disease, including myeloproliferative disorder
- patient with platelet agglutination abnormality that prevents reliable measurement of platelet counts
- patient with bone marrow aplasia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
response to oral thrombopoietin receptor agonist
Time frame: baseline
assess haematological response to oral thrombopoietin receptor agonist (Eltrombopag) therapy
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2023.
- Background: PubMed 34815110Kuter DJ. The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Rev. 2022 May;53:100909. doi: 10.1016/j.blre.2021.100909. Epub 2021 Nov 16.
- Background: PubMed 30191972Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. doi: 10.1111/bjh.15573. Epub 2018 Sep 7.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Thrombopoietin Receptor Agonist, 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 3 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: status not confirmed, as of the sponsor's last update on 6 May 2023. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
response to oral thrombopoietin receptor agonist (time frame: baseline). 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 NCT05846529, which also lists 2 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 6 May 2023.