Long Term Beta Thalassemia Treatment: Findings From The Extension Period (Thalidomide)

National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Status and phase: Active, not recruiting; Phase 2

Conditions: Beta-Thalassemia; Fetal Hemoglobin; Hemoglobinopathies

Treatments: Drug: Thalidomide; Drug: thalidomide and hydroxyurea

Study type: Interventional

Funder types: Other

Identifiers

NCT06490601

NIBD/IRB-242/12-2022

Details and patient eligibility

About

The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. The study, lasting three years, is a Phase 2 single-center, open-label interventional study with a sample size of 30 participants aged 8-35 years. It includes specific inclusion and exclusion criteria for participant selection. Data will be collected through clinical interviews and medical records and analyzed using(Statistical Package for the Social Sciences. This project aims to enhance beta thalassemia treatment strategies, focusing on reducing transfusion dependency and improving patient quality of life.

Full description

Study titled Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial, conducted at the National Institute of Blood Disease & Bone Marrow Transplantation (NIBD & BMT).

This study focuses on the long-term comparison of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone in treating beta thalassemia. The objective is to evaluate the efficacy and safety of the combination therapy compared to thalidomide alone, with the hypothesis that the combination will be more effective. Beta thalassemia is defined as an autosomal recessive disorder affecting beta-globin production, influenced by genetic modifiers. Key variables include hemoglobin, red blood cells, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, nucleated red blood cells, ferritin, bilirubin, Serum Glutamate Pyruvate Transaminase, creatinine, transfusion frequency, spleen and liver size, hemoglobin subunit beta [ Homo sapiens (human) ] mutation, and certain polymorphism in gamma globin gene . The study took place at NIBD hospital over three years, designed as a Phase 2 single-center, two-arm open-label interventional study with a sample size of 30 participants using simple randomized sampling. Inclusion criteria are beta thalassemia major/intermediate patients aged 8-35 years, while exclusion criteria include patients with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis and fits. Data will be collected through clinical interviews and medical record reviews and analyzed using (Statistical Package for the Social Sciences.

Enrollment: 30 estimated patients

Sex: All

Ages: 8 to 35 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

• Known case of beta thalassemia major/intermediate (transfusion dependent)
• Willing to give informed consent

Exclusion Criteria

• Patients with comorbidities such as liver dysfunction
• Married patients
• Lactating mothers
• History of thrombosis and fits

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

combination therapy (thalidomide and hydroxyurea)

Experimental group

Description:

In this group, participants were treated with combination therapy that includes thalidomide and hydroxyurea at the dose of 100mg/day at night with aspirin and 500mg /day respectively.

Treatment:

Drug: thalidomide and hydroxyurea

thalidomide alone

Active Comparator group

Description:

In this group, participants were treated with thalidomide at the dose of 100mg/day at night with aspirin.

Treatment:

Drug: Thalidomide