Long Term Beta Thalassemia Treatment: Findings From The Extension Period

NCT06490601 | Active Not Recruiting Phase 2

• 1 other identifier: NIBD/IRB-242/12-2022
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Fetal Hemoglobin; Beta-Thalassemia; Hemoglobinopathies

Keywords

thalidomide; safety and efficacy; thalassemia; fetal hemoglobin inducer; hydroxyurea

Outcome Measures

Primary Outcomes (5)

• hemoglobin levels

  Measure the improvement in hemoglobin levels Hemoglobin levels will be assessed using a complete blood count (CBC) test, measured in grams per deciliter (g/dL) of blood. This test is conducted through a venous blood sample, which is then analyzed using an automated hematology analyzer to determine the hemoglobin concentration.

  3 years

• red blood cell count.

  Measure the improvement in red blood cell count. Red blood cell count will be assessed using a complete blood count (CBC) test, measured in millions of cells per microliter (million cells/µL) of blood. This test is conducted through a venous blood sample, analyzed using an automated hematology analyzer to determine the number of red blood cells present.

  3 years

• leukocyte count

  Measure the effect on in leukocyte count Leukocyte count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the total number of white blood cells present.

  3 years

• reticulocyte count

  Measure the effect on reticulocyte count Reticulocyte count will be assessed using a complete blood count (CBC) test, measured as a percentage of the total red blood cells or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which identifies and quantifies reticulocytes using specific staining techniques.

  3 years

• Transfusion Frequency:

  Document and compare the frequency of blood transfusions required by patients in both treatment arms over the study period. Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event.

  3 years

Secondary Outcomes (7)

• Spleen and Liver Size

  3 years

• Serum Ferritin Levels

  3 years

• Genetic Modifiers:

  3 years

• bilirubin

  3 years

• lactate dehydrogenase.

  3 years

Other Outcomes (1)

• Safety Profile:

  3 years

Study Arms (2)

combination therapy (thalidomide and hydroxyurea)

EXPERIMENTAL

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.

Drug: thalidomide and hydroxyurea

thalidomide alone

ACTIVE COMPARATOR

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

Drug: Thalidomide

Interventions

thalidomide and hydroxyurea DRUG

Thalidomide: Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor- alpha Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Hydroxyurea: Hydroxyurea or hydroxycarbamide (HU) lies in the category of antimetabolite. Mechanism of fetal hemoglobin induction includes increase in erythropoietin and nitric oxide production, apoptosis induction and potentiating in granulocyte cycling activity.

Also known as: combination therapy in beta thalassemia

combination therapy (thalidomide and hydroxyurea)

Thalidomide DRUG

Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor -alpha , Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Investigating the impact of thalidomide on transfusion-dependent beta thalassemia patients is essential for discerning its therapeutic efficacy and safety profile.

Also known as: fetal hemoglobin inducer

thalidomide alone

Eligibility Criteria

• Age: 8 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

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

Study Sites (1)

National Institute of blood disease and bone marrow transplant

Karachi, Sindh, Pakistan

Location

Related Publications (1)

• 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.

  PMID: 8683628 BACKGROUND

MeSH Terms

Conditions

beta-Thalassemia; Hemoglobinopathies; Thalassemia

Interventions

Thalidomide; Hydroxyurea; Combined Modality Therapy

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Urea; Amides; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 21, 2024
• First Posted: July 8, 2024
• Study Start: April 1, 2022
• Primary Completion: April 1, 2024
• Study Completion: July 1, 2025
• Last Updated: May 30, 2025

Record last verified: 2025-05

Locations

PA (1)