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NCT07673302
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
Conditions: Thalassemia
Sex: All
Healthy volunteers: No
Phase: NA
Enrollment: 240
Sponsor: Riphah International University
Location: Riphah International University Rawalpindi Punjab Province
Summary
The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients. The main objectives of this study are:
* To determine the therapeutic efficacy of HbF inducers (combination therapy: thalidomide and hydroxyurea) on hemoglobin level and blood transfusion in beta thalassemia patients.
* To determine the safety of HbF inducers (combination therapy: thalidomide and hydroxyurea) in beta thalassemia patients
* To determine effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on quality of life of beta thalassemia patients
Eligibility Criteria
Inclusion criteria:
* Confirmed diagnosis of Beta thalassemia Major (BTM) ascertained by Hemoglobin Electrophoresis or HPLC report performed pre-transfusion or genetic testing profile (comprising PCR or HBB gene sequencing) suggestive of β-thalassemia syndrome.
* All ages and both genders will be included
* Written informed consent
Exclusion criteria:
* Pregnancy or unwilling to follow contraception or planning conception (Enrolled female patients will be strictly advised to avoid pregnancy during the study period and until 6 months after thalidomide withdrawal.
* Hemoglobinopathies other than beta thalassemia
* History of neurological problems
* Inability to regularly follow up
Source: ClinicalTrials.gov (NCT07673302). StuddyBuddy aggregates publicly available trial information.