Health TrialsbyHealthPanel

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

ConditionThalassemia
Trial statusRecruiting
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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Thalidomide

    Drug

    Tab Thalidomide 0.5 to 4 mg/kg orally every day for 12 months

  • Hydroxy Urea

    Drug

    Tab Hydroxyurea 10-20 mg/kg per day for 12 months

Treatment groups

240 Participants
are divided into 2 treatment groups
Group A: Blood transfusion GroupNo intervention 0 interventions
Group B: HbF inducersExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Improvement in the hemoglobin level

To evaluate the Improvement in Hemoglobin level, response criteria are defined as follows: Major response, an elevation in total Hb level of ≥2 g/dL Minor response, an elevation in total Hb level of 1 to 2 g/dL, or Hb not substantially increased (\<1 g/dL) but the patients achieve Hb\>7 g/L No response, an elevation in total Hb level of \<1 g/dL

Time frame
6 months
2

Decrease in transfusion requirement

To evaluate the decrease in transfusion requirement, response criteria are defined as follows: Major response: reduce transfusion requirements by ≥25% after 6 months of therapy compared to baseline. Minor response: reduce transfusion requirement by \<25 % after 6 months of therapy, No response: same blood transfusion requirement as baseline

Time frame
6 months

Secondary outcomes

1

Serum Bilirubin levels

0.2- 1.1 mg/dL Normal \>1.1- 3 mg/dL Mild derangement \>3 mg/dL Severe derangement

Time frame
1 year
2

Change in ALT levels

ALT levels range: 0- 42 U/L Normal \>42-126 U/L Mild severity \>126- 420 U/L Moderate severity \>420 U/L severe

Time frame
1 year
3

Change in serum creatinine levels

Serum creatinine 0.2 - 1.1 mg/dL Normal \>1.1- 1.5 mg/dL Mild severity \>1.5- 3.0 mg/dL Moderate severity \>3 mg/dL Severe

Time frame
1 year
4

Assess Quality of life by using EORTC QLQ-C30

EORTC QLQ C-30 URDU version, a self-administered questionnaire, will be filled by each participant at baseline and 6 months. Functional/Global: 100 = perfect function. 0 = no function. 10-point change = clinically meaningful. Symptoms: 0 = no symptoms. 100 = maximum symptoms 10-point increase = worse symptom burden.

Time frame
6 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Riphah International University

Lead sponsor

Source ClinicalTrials.gov
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