Adherence of Beta Thalssemia Patients to Oral Chelation T... | NCT06568926

Studies
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy

NCT06568926

Sponsor

Sohag University

Status

Recruiting

Last Update Posted

Aug 23, 2024Actual

Enrollment

200Estimated

Phase

Not Applicable

Conditions

Beta-Thalassemia

Interventions

Serum Ferritin level

Countries

Egypt

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

Annotation Module

Unposted Annotation

Unposted Responsible Party

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2024

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 14, 2024Actual

Primary Completion Date

Jul 14, 2025Estimated

Completion Date

Jul 14, 2025Estimated

First Submitted Date

Aug 21, 2024

First Submission Date that Met QC Criteria

Aug 21, 2024

First Posted Date

Aug 23, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 21, 2024

Last Update Posted Date

Aug 23, 2024Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Marwa Aly Mousa, Resident-pediatric department-sohag hospital university, Sohag UniversityPrincipal Investigator

Lead Sponsor

Sohag UniversityOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 1.5 million live births the disease prevalence is equal to1000 cases per 1.5 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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

200Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
patient group	Experimental	Beta thalassemia patients on oral chelation therapy	Diagnostic Test: Serum Ferritin level

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Serum Ferritin level	Diagnostic Test	follow up to the ferritin level in beta thalaseemia patients on oral chelation therapy	patient group

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
serum ferritin level in beta thalassemia patients adherent to oral chelation therapy	Adherence of Beta Thalssemia Patients to Oral Chelation Therapy	1 year

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

confirmed diagnosis of beta thalassemia major or intermedia,
Age between 2-18 years,
Receiving regular blood transfusions every 2-5 weeks
Prescribed oral iron chelation therapy with deferasirox for at least 1 year prior to enrollment.

Exclusion Criteria:

Age less than 2 years and more than 18 years
Any cause of blood transfusion other than beta Thalassemia
Patients on deferoxamine

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Marwa Ali Mousa, resident	Contact	01111815617		marwa.mousaa@med.sohag.edu.eg
ALzahraa Elsayad Ahmed, professor	Contact	01224340998

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Sohag university Hospital	Recruiting	Sohag			Egypt

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
22281991	Background	Belhoul KM, Bakir ML, Saned MS, Kadhim AM, Musallam KM, Taher AT. Serum ferritin levels and endocrinopathy in medically treated patients with beta thalassemia major. Ann Hematol. 2012 Jul;91(7):1107-14. doi: 10.1007/s00277-012-1412-7. Epub 2012 Jan 28.
17880620	Background	Delea TE, Edelsberg J, Sofrygin O, Thomas SK, Baladi JF, Phatak PD, Coates TD. Consequences and costs of noncompliance with iron chelation therapy in patients with transfusion-dependent thalassemia: a literature review. Transfusion. 2007 Oct;47(10):1919-29. doi: 10.1111/j.1537-2995.2007.01416.x.

Type	Date	Date Unknown
Release	Jan 14, 2026
Reset	Jan 29, 2026

ID	Term
D013789	Thalassemia
D000745	Anemia, Hemolytic, Congenital
D000743	Anemia, Hemolytic
D000740	Anemia