Clinical Experience of Thalidomide in Thalassemic Patients

Thalassemia, Beta Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05132270
• Other study ID #: CH-0420
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: January 1, 2020
• Est. completion date: January 31, 2021

Study information

• Verified date: November 2021
• Source: Children's Hospital Karachi
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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. 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 135
• Est. completion date: January 31, 2021
• Est. primary completion date: December 31, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

1. Patients with clinical and genetic diagnosis of ß-thalassemia major and intermedia

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

3. Patients who are not the 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

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia, Beta

Intervention

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

Locations

Country	Name	City	State
Pakistan	Children's Hospital Karachi	Karachi	Sindh

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital Karachi

Country where clinical trial is conducted

Pakistan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in transfusion requirements	Change in transfusion needs (measured as blood volume in ml) per month from baseline	6 months on combination therapy
Primary	Change in Hemoglobin levels	Change in hemoglobin level of >1g/dl from baseline	6 months on combination therapy
Primary	Change in the liver function test	Change in SGPT in U/l from baseline	6 months on combination therapy
Primary	Change in liver function test	Change in total, direct, and indirect bilirubin in mg/dl from baseline	6 months on combination therapy
Primary	Change in Kidney Function test	Change in urea and creatinine levels in mg/dl from baseline	6 months on combination therapy
Secondary	Change in Spleen size	Change in size of spleen (in centimeters) from baseline	6 months on combination therapy
Secondary	Change in liver size	Change in size of liver (in centimeters) from baseline	6 months on combination therapy