Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06568926
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-21
Brief Title:
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The β-thalassemias are a group of inherited disorders of hemoglobin Hb synthesis characterized by chronic anemia of varying severity The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy It is now common practice to classify patients as having transfusion dependent β-thalassemia TDT or non-transfusion-dependent β-thalassemia NTDT Regarding geographical distribution of β-thalassemia it prevails more in countries on the Mediterranean South east of Asia and in the east of Europe In Egypt it is the most common cause of chronic blood loss One thousand cases are recorded annually for every 15 million live births the disease prevalence is equal to1000 cases per 15 million live births Ghazala et al 2021
The only curative treatment for thalassemia currently is a bone marrow transplant However it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost Thus most patients receive regular blood transfusions accompanied by iron chelation therapy ICT as the standard of care The ideal management of a patient with transfusion-dependent thalassemia TDT requires a multidisciplinary therapeutic approach The main iron chelating agents include deferoxamine DFO deferiprone DFP and deferasirox DFX Due to poor oral bioavailibility DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day DFP and DFX may be administered orally up to three times a day The known side effects associated with each chelator include infusion reactions in DFO gastrointestinal distress agranulocytosis in DFP and transaminitis in DFP and DFX
The only curative treatment for thalassemia currently is a bone marrow transplant However it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost Thus most patients receive regular blood transfusions accompanied by iron chelation therapy ICT as the standard of care The ideal management of a patient with transfusion-dependent thalassemia TDT requires a multidisciplinary therapeutic approach The main iron chelating agents include deferoxamine DFO deferiprone DFP and deferasirox DFX Due to poor oral bioavailibility DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day DFP and DFX may be administered orally up to three times a day The known side effects associated with each chelator include infusion reactions in DFO gastrointestinal distress agranulocytosis in DFP and transaminitis in DFP and DFX
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None