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Completed

NCT Number: NCT05132270

Clinical Experience of Thalidomide in Thalassemic Patients

Objectives

Primary objective:

• To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.

Secondary objective:

• To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy

A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study.

Sample Size and Population This study included 135 Beta-thalassemia patients.

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Key information

Conditions

Thalassemia, Beta Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia beta-Thalassemia

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Children's Hospital Karachi

Karachi, Sindh, Pakistan

About this study

The purpose of the study was to evaluate the efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. The participants were evaluated on the basis of eligibility criteria, consent was obtained and baseline investigations were performed on the screening visit. Participants were continued on Hydroxyurea (10-20mg/kg/day) for the first six months and then for the next six months thalidomide (2-5 mg/kg/day) was added to the intervention. Aspirin was also added (2-4mg/kg/day).

Efficacy:

Maintenance or rise in Hemoglobin (Hb) levels and changes in transfusion frequency before and after the use of combination therapy was used to evaluate the efficacy of the combination therapy. Good responders were the patients who were on transfusion and went off-transfusion after the combination therapy or individuals who were already off transfusion and after combination therapy demonstrated an increase in Hb of at least 1gm/dl. Responders were those who remained on transfusion on combination therapy but displayed a 50% reduction in blood requirements. Whereas, non-responders, were individuals who were off-transfusion before combination therapy and had improvement in Hb of <1gm/dl, or those who remained on transfusion during combination therapy and did not experience transfusion reduction of at least 50%.

Safety:

Safety of the drug was evaluated on the basis of the following parameters and intervention was discontinued or put on hold if:

  • Creatinine >1.1mg/dl, Urea >43mg/dl),
  • Liver function (SGPT >35mg/L)
  • Absolute Neutrophil counts<2*109/L
  • Platelets < 100*109/L

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with clinical and genetic diagnosis of β-thalassemia major and intermedia
  • Patients who showed partial response or a decline in response to hydroxyurea
  • Patients who are not the candidates for the bone marrow transplant procedure.

Exclusion criteria

  • Married Patients
  • Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
  • Patients allergic to the drug ingredients
  • Patients with mental disorders
  • Patients who are enrolled in other clinical trials
  • Patients with a history of venous or arterial thrombosis

Treatment and study plan

Hydroxyurea and Thalidomide

Drug

Evaluation of hydroxyurea and thalidomide combination use in beta-thalassemia patients

Primary outcomes

  1. Change in transfusion requirements

    Time frame: 6 months on combination therapy

    Change in transfusion needs (measured as blood volume in ml) per month from baseline

  2. Change in Hemoglobin levels

    Time frame: 6 months on combination therapy

    Change in hemoglobin level of >1g/dl from baseline

  3. Change in the liver function test

    Time frame: 6 months on combination therapy

    Change in SGPT in U/l from baseline

  4. Change in liver function test

    Time frame: 6 months on combination therapy

    Change in total, direct, and indirect bilirubin in mg/dl from baseline

  5. Change in Kidney Function test

    Time frame: 6 months on combination therapy

    Change in urea and creatinine levels in mg/dl from baseline

Secondary outcomes

  1. Change in Spleen size

    Time frame: 6 months on combination therapy

    Change in size of spleen (in centimeters) from baseline

  2. Change in liver size

    Time frame: 6 months on combination therapy

    Change in size of liver (in centimeters) from baseline

Sponsors and collaborators

Lead sponsor

Children's Hospital Karachi

Other

Registry information

Official study title

Efficacy and Safety of Combination of Hydroxyurea and Low-dose Thalidomide on Hemoglobin Synthesis in Thalassemia Patients

Important dates

Study start
2020
Primary completion
2020
Study completion
2021
First posted
Nov 24, 2021
Registry last updated
Nov 24, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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