Overview

Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients

Status:
Completed

Trial end date:
2023-07-30

Target enrollment:
0

Participant gender:
All

Summary

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. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide was started. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during, and end of the study. The assessment of treatment outcomes was conducted at the 1-year, 2-year, and 3-year follow-up points during the combination therapy period, categorizing patients as either "good responders," "responders," or "non-responders."

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Children's Hospital Karachi

Treatments:

Hydroxyurea
Thalidomide

Criteria

Inclusion Criteria:

1. Patients with clinical and genetic diagnoses of β-thalassemia major and intermedia

2. Patients who showed partial response or a decline in response to hydroxyurea

3. Patients who are not candidates for the bone marrow transplant procedure

Exclusion Criteria:

1. Married Patients

2. Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney
diseases

3. Patients allergic to the drug ingredients

4. Patients with mental disorders

5. Patients who are enrolled in other clinical trials

6. Patients with a history of venous or arterial thrombosis