Ignite Creation Date:
2024-05-11 @ 8:30 AM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06408324
Status:
COMPLETED
Last Update Posted:
2024-05-16
First Post:
2023-12-18
Brief Title:
Evaluation of the Use of Thrombopoietin Receptor Agonists in Adults With Primary ITP in Europe
Sponsor:
European Research Consortium on ITP
Organization:
European Research Consortium on ITP
Study Overview
Official Title:
A Retrospective Study to Evaluate the Use of Thrombopoietin Receptor Agonists TPO-ra in Adults With Primary Immune Thrombocytopenia ITP in Europe
Status:
COMPLETED
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goal of this multicenter observational retrospective is to evaluate the standards of use of thrombopoietin receptor agonists TPO-RA in adult patients with primary immune thrombocytopenia ITP with a particular interest on phase of ITP defined according to time when diagnosis of ITP is established in the medical records newly diagnosed 0-3 months persistent 3-12 months and chronic 12 months tolerability safety posology and remissions outside controlled clinical trials
Secondary Objectives
To analyze the patient characteristics that could affect the choice of thrombopoietin receptor agonist for the treatment of ITP considering specific clinical aspects such as previous thromboembolic disease bleeding platelet count surgical procedures etc
To evaluate the degree of adherence to international guidelines related to the use of thrombopoietin receptor agonists in regular clinical practice
Data will be collected through a retrospective chart review of patients with ITP who started TPO-RA treatment between January 2014 and December 2018
Secondary Objectives
To analyze the patient characteristics that could affect the choice of thrombopoietin receptor agonist for the treatment of ITP considering specific clinical aspects such as previous thromboembolic disease bleeding platelet count surgical procedures etc
To evaluate the degree of adherence to international guidelines related to the use of thrombopoietin receptor agonists in regular clinical practice
Data will be collected through a retrospective chart review of patients with ITP who started TPO-RA treatment between January 2014 and December 2018
Detailed Description:
Objectives
Primary Objective
To evaluate the standards of use of TPO-RA in regular clinical practice in Europe with a particular interest on phase of ITP the phase of the disease will be defined according to time when diagnosis of ITP is established in the medical records newly diagnosed 0-3 months persistent 3-12 months and chronic 12 months tolerability safety posology and remissions outside controlled clinical trials
Secondary Objectives
To analyze the patient characteristics that could affect the choice of thrombopoietin receptor agonist for the treatment of ITP considering specific clinical aspects such as previous thromboembolic disease bleeding platelet count surgical procedures etc
To evaluate the degree of adherence to international guidelines related to the use of thrombopoietin receptor agonists in regular clinical practice
Description of study centers
The study will be conducted in medical departments of 18 European hospitals from 6 countries UK Spain Italy Norway France and Switzerland 3 sites per country
The founding members of the European Research Consortium on ITP ERCI will identify and select the Sites Investigators The sites that will participate in this project must ensure to provide all of the patients care for ITP and that all the information required to complete the CRF can be obtained from the sites medical records
Study population
Patients who started treatment for ITP with thrombopoietin receptor agonists romiplostimeltrombopag between January 2014 and December 2018
It is estimated that approximately 350 ITP TPO-RA treated patients will be included in the study
Observational study course
Screening
Each site will screen patients reviewing medical records of patients with ITP who started treatment with thrombopoietin analogues regardless of phase of the disease between January 2014 and December 2016 In order to prevent screening bias the medical records will be consecutively screened by the investigators starting with the oldest regarding start of ITP therapy with TPO-RA by selecting patients who started therapy with agonists on the date nearest to January 2014
To enroll a case in this study the patient must have initiated a TPO-RA at the specific site that recruits such patient If a sporadic patient with ITP has been referred from the participating site to a secondary center for specialized care the investigator should confirm that data can be fully extracted from centralized records
Information and consent Screened patients invited to take part in the study will be informed by the investigator or an authorized member of the team during a hospital visit or by phone No patient will be included in the study until having been duly informed by the investigator and having signed the informed consent
Data collection
Each site will collect retrospectively the data from all the patients with ITP who meet the inclusion criteria and no exclusion criteria and agree to participate in the study by signing the informed consent
The data will be obtained in a retrospective non-interventionist manner to meet this studys objective by reviewing the patients medical records The investigator will only collect the data available in the patients medical records patients will have been followed up according to regular clinical practice and received treatment according to the investigators clinical judgement In no case shall a patients inclusion in the study affect regular clinical practice
Collected data
The collected data will include variables at the time of diagnosis bleeding comorbidities biological studies treatments received before TPO-RA variables at time of TPO-RA initiation and following TPO-RA therapy Considering that patients initiated TPO-RA in 2014- 2018 data will include information before date of TPO-RA initiation diagnosis before or in 2014- 2018 and also all the information related to TPO-RA therapy and follow-up until the date of data collection planned in 2022-2023 independently of current or active treatment with TPO-RA agents The data will be collected into the eCRF electronic case report form by the designated person at each site
Sample size
The objective of the study is to evaluate the use of TPO-RA in adult patients with primary ITP in Europe The incidence of ITP in Europe ranges from 16 to 39 patients per 100000 inhabitants Considering a finite population in order to identify a pattern repeated in at least 35 of patients with 5 precision and a 95 CI a total of 350 patients have to be enrolled Considering a 5 loss rate patients that withdraw their consent or those with more than 20 missing information from CRF data from a total of at least 368 patients could finally be included
Primary Objective
To evaluate the standards of use of TPO-RA in regular clinical practice in Europe with a particular interest on phase of ITP the phase of the disease will be defined according to time when diagnosis of ITP is established in the medical records newly diagnosed 0-3 months persistent 3-12 months and chronic 12 months tolerability safety posology and remissions outside controlled clinical trials
Secondary Objectives
To analyze the patient characteristics that could affect the choice of thrombopoietin receptor agonist for the treatment of ITP considering specific clinical aspects such as previous thromboembolic disease bleeding platelet count surgical procedures etc
To evaluate the degree of adherence to international guidelines related to the use of thrombopoietin receptor agonists in regular clinical practice
Description of study centers
The study will be conducted in medical departments of 18 European hospitals from 6 countries UK Spain Italy Norway France and Switzerland 3 sites per country
The founding members of the European Research Consortium on ITP ERCI will identify and select the Sites Investigators The sites that will participate in this project must ensure to provide all of the patients care for ITP and that all the information required to complete the CRF can be obtained from the sites medical records
Study population
Patients who started treatment for ITP with thrombopoietin receptor agonists romiplostimeltrombopag between January 2014 and December 2018
It is estimated that approximately 350 ITP TPO-RA treated patients will be included in the study
Observational study course
Screening
Each site will screen patients reviewing medical records of patients with ITP who started treatment with thrombopoietin analogues regardless of phase of the disease between January 2014 and December 2016 In order to prevent screening bias the medical records will be consecutively screened by the investigators starting with the oldest regarding start of ITP therapy with TPO-RA by selecting patients who started therapy with agonists on the date nearest to January 2014
To enroll a case in this study the patient must have initiated a TPO-RA at the specific site that recruits such patient If a sporadic patient with ITP has been referred from the participating site to a secondary center for specialized care the investigator should confirm that data can be fully extracted from centralized records
Information and consent Screened patients invited to take part in the study will be informed by the investigator or an authorized member of the team during a hospital visit or by phone No patient will be included in the study until having been duly informed by the investigator and having signed the informed consent
Data collection
Each site will collect retrospectively the data from all the patients with ITP who meet the inclusion criteria and no exclusion criteria and agree to participate in the study by signing the informed consent
The data will be obtained in a retrospective non-interventionist manner to meet this studys objective by reviewing the patients medical records The investigator will only collect the data available in the patients medical records patients will have been followed up according to regular clinical practice and received treatment according to the investigators clinical judgement In no case shall a patients inclusion in the study affect regular clinical practice
Collected data
The collected data will include variables at the time of diagnosis bleeding comorbidities biological studies treatments received before TPO-RA variables at time of TPO-RA initiation and following TPO-RA therapy Considering that patients initiated TPO-RA in 2014- 2018 data will include information before date of TPO-RA initiation diagnosis before or in 2014- 2018 and also all the information related to TPO-RA therapy and follow-up until the date of data collection planned in 2022-2023 independently of current or active treatment with TPO-RA agents The data will be collected into the eCRF electronic case report form by the designated person at each site
Sample size
The objective of the study is to evaluate the use of TPO-RA in adult patients with primary ITP in Europe The incidence of ITP in Europe ranges from 16 to 39 patients per 100000 inhabitants Considering a finite population in order to identify a pattern repeated in at least 35 of patients with 5 precision and a 95 CI a total of 350 patients have to be enrolled Considering a 5 loss rate patients that withdraw their consent or those with more than 20 missing information from CRF data from a total of at least 368 patients could finally be included
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None