Clinical trial NCT06490601: Long Term Beta Thalassemia Treatment: Findings From The Extension Period
Official title: Long Term Comparison of Combination Therapy (Thalidomide and Hydroxyurea) vs Thalidomide Alone in Beta Thalassemia.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Fetal Hemoglobin
Beta-Thalassemia
Hemoglobinopathies
Keywords: thalidomide
safety and efficacy
thalassemia
fetal hemoglobin inducer
hydroxyurea
Lead sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sponsor class: OTHER_GOV
First posted: 2024-07-08
Enrollment: 30
Sex: ALL
Minimum age (years): 8
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: study participants were divided into 2 groups. one was treated with combination therapy that includes thalidomide and hydroxyurea whereas another group was treated with thalidomide alone.
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility 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

Primary outcomes:
measure: hemoglobin levels
timeFrame: 3 years
measure: red blood cell count.
timeFrame: 3 years
measure: leukocyte count
timeFrame: 3 years
measure: reticulocyte count
timeFrame: 3 years
measure: Transfusion Frequency:
timeFrame: 3 years
Secondary outcomes:
measure: Spleen and Liver Size
timeFrame: 3 years
measure: Serum Ferritin Levels
timeFrame: 3 years
measure: Genetic Modifiers:
timeFrame: 3 years
measure: bilirubin
timeFrame: 3 years
measure: lactate dehydrogenase.
timeFrame: 3 years
measure: Serum Glutamate Pyruvate Transaminase,
timeFrame: 3 years
measure: creatinine
timeFrame: 3 years
Other outcomes:
measure: Safety Profile:
timeFrame: 3 years
Locations:
city: Karachi
country: Pakistan
facility: National Institute of blood disease and bone marrow transplant
state: Sindh
Study officials:
References:
citation: Henson DE. Loss of p53-immunostaining intensity in breast cancer. J Natl Cancer Inst. 1996 Aug 7;88(15):1015-6. doi: 10.1093/jnci/88.15.1015. No abstract available.