Beta Thalassemia Clinical Trials

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Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia: a Prospective 12 Months Study

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Not Applicable
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
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Pakistan
Riphah International University
RECRUITING
Rawalpindi
Contact Information
Primary
Fariha Sardar, MBBS, FCPS
doc.fariha@yahoo.com
+923335517877
Backup
Fariha Sardar, MBBS, FCPS
afzaal2kmt20@gamil.com
+923350941494
Time Frame
Start Date: 2026-06-20
Estimated Completion Date: 2027-06-19
Participants
Target number of participants: 240
Treatments
No_intervention: Blood transfusion Group
Control group will be on regular blood transfusion and iron chelation therapy
Experimental: HbF inducers
All patients will receive thalidomide and hydroxyurea. Low dose Thalidomide will be administered to patients at a low dose of 0.5 to 4 mg/kg orally every day for 12 months until continuous transfusion-dependency or unacceptable toxicity occurred. The starting dose of hydroxyurea will be 10-20 mg/kg per day.
Sponsors
Leads: Riphah International University

This content was sourced from clinicaltrials.gov