Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT06408324 |
| Other study ID # |
VERTEX 3.0 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
November 24, 2022 |
| Est. completion date |
April 5, 2024 |
Study information
| Verified date |
May 2024 |
| Source |
European Research Consortium on ITP |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial 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.
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 patient´s care for ITP and that all the information required to
complete the CRF can be obtained from the site´s medical records.
Study population:
Patients who started treatment for ITP with thrombopoietin receptor agonists
(romiplostim/eltrombopag) 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 study's
objective, by reviewing the patient's 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 investigator's
clinical judgement. In no case shall a patient's 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 1.6 to 3.9 patients per 100,000
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.
