Safety of Oral Thrombopoietin Receptor Agonist in Pediatric Chronic ITP

May 4, 2023 updated by: Mohamed Salama Sayed Hassan, Assiut University

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.

Study Overview

Status

Not yet recruiting

Conditions

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 Type

Observational

Enrollment (Anticipated)

100

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Infants and children between age of (2 year to <18 years old)

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
response to oral thrombopoietin receptor agonist
Time Frame: baseline
assess haematological response to oral thrombopoietin receptor agonist (Eltrombopag) therapy
baseline

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assiut University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

June 1, 2023

Primary Completion (Anticipated)

April 1, 2024

Study Completion (Anticipated)

June 1, 2024

Study Registration Dates

First Submitted

April 7, 2023

First Submitted That Met QC Criteria

May 4, 2023

First Posted (Estimate)

May 5, 2023

Study Record Updates

Last Update Posted (Estimate)

May 5, 2023

Last Update Submitted That Met QC Criteria

May 4, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Other Study ID Numbers

  • thrombopoietin agonist

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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