Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia

ConditionThalassemia
Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorRiphah International University

About this trial

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

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

  • Thalidomide
  • Hydroxy Urea

Treatment groups

240 Participants
are divided into 2 treatment groups