Ignite Creation Date:
2024-05-06 @ 12:52 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03877809
Status:
COMPLETED
Last Update Posted:
2024-01-23
First Post:
2019-03-14
Brief Title:
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
Sponsor:
Rare Partners srl Impresa Sociale
Organization:
Rare Partners srl Impresa Sociale
Study Overview
Official Title:
A Personalized Medicine Approach for Beta-thalassemia Transfusion Dependent Patients Testing SIROLIMUS in a First Pilot Clinical Trial
Status:
COMPLETED
Status Verified Date:
2024-01
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:
SIRTHALACLIN
Brief Summary:
Beta-thalassemias are hereditary blood disorders caused by reduced or absent synthesis of hemoglobin beta chains with variable outcomes ranging from severe anemia to clinically asymptomatic individuals Treatment is symptomatic and thalassemia is a major unmet medical need Survival is increased even in patients needing transfusions in comparison with a few years ago but the quality of life is poor for many patients In some patients an anomalous expression of gamma-globin genes has been observed with a consequent rise in Fetal Hemoglobin levels The patients displaying a clinical phenotype known as Hereditary Persistence of Fetal Hemoglobin HPFH exhibit a positive clinical status To mimick HPFH several compounds able to induce expression of fetal hemoglobins HbF have been evaluated Within this framework sirolimus is particularly interesting as an inducer of HbF It has been used for many years for different indications and the available preclinical evidence warrant the start of a clinical development plan in thalassemia The investigators propose a clinical trial in beta-thalassemia patients designed to evaluate the effect of sirolimus on several parameters related to red blood cell status and to the level of HbF in particular as a first step for the full clinical development in this new indication
Detailed Description:
The general aim of the protocol is to demonstrate the applicability of a personalised and precision medicine approach in beta-thalassemia in a clinical trial setting for a repurposed drug namely sirolimus The presence of high level of Fetal Hemoglobin HbF is considered a condition predictive of a favourable outcome in thalassemia and its increase induced by pharmacological agents is considered a potential way to improve clinical status of the patients In the present trial in terms of efficacy analysis the investigators will focus their attention on HbF levels
Primary objective
To evaluate the suitability of sirolimus for the treatment of beta-thalassemia patients within the frame of a comprehensive project aimed to the reduction of their transfusions need consequently ameliorating their quality of life This goal can be obtained through a pharmacologically mediated increased level of HbF with a prerequisite to be verified namely the correlation between induction of HbF in vitro and in vivo in single patients
Secondary objectives
To assess safety of sirolimus and correlation between administered dose and blood levels in beta-thalassemia patients
To assess the influence of sirolimus on transfusion regimen
To assess the effect of sirolimus on hematopoietic and immune system of thalassemia patients
Primary objective
To evaluate the suitability of sirolimus for the treatment of beta-thalassemia patients within the frame of a comprehensive project aimed to the reduction of their transfusions need consequently ameliorating their quality of life This goal can be obtained through a pharmacologically mediated increased level of HbF with a prerequisite to be verified namely the correlation between induction of HbF in vitro and in vivo in single patients
Secondary objectives
To assess safety of sirolimus and correlation between administered dose and blood levels in beta-thalassemia patients
To assess the influence of sirolimus on transfusion regimen
To assess the effect of sirolimus on hematopoietic and immune system of thalassemia patients
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?:
False
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 208872Z17Z | OTHER_GRANT | Wellcome Trust Innovator Award | None |
| 2018-001942-33 | EUDRACT_NUMBER | None | None |