Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients

Not Applicable

Completed

• Conditions: Thalassemia
• Interventions: Drug: Thalidomide

• Registration Number: NCT06888232
• Lead Sponsor: Muhammad Aamir Latif

Brief Summary

This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.

Detailed Description

Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.

Study Timeline

• Study Start: 2023-08-01
• Primary Completion: 2024-12-31
• Study Completion: 2024-12-31
• Status Verified: 2025-03
• Study First Submitted: 2025-03-15
• Study First Submitted QC: 2025-03-15
• Last Update Submitted: 2025-03-15
• Study First Posted: 2025-03-21
• Last Update Posted: 2025-03-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 121

Inclusion Criteria

• Children of both genders
• Aged 2-5 years
• With transfusion-dependent thalassemia (TDT)
• Whose parents/guardians showed willingness to adhere to follow-up visits

Exclusion Criteria

• Children with uncontrolled infections
• Significant hepatic or renal dysfunction
• Malignancy
• Known contraindications to thalidomide

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Thalidomide group	Thalidomide	Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.

Primary Outcome Measures

Name	Time	Method
Efficacy of treatment	6 months	Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin \<1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months.
Safety of treatment	6 months	Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.

Trial Locations

Locations (1)

The Children's Hospital & Institute of Child Health; 🇵🇰 Multan, Punjab, Pakistan