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Completed

NCT Number: NCT06888232

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

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

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Key information

Conditions

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

Age range

2 year–5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

The Children's Hospital & Institute of Child Health

Multan, Punjab Province, 66000, Pakistan

About this study

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.

Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

thalidomide

Drug

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 outcomes

  1. Efficacy of treatment

    Time frame: 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.

  2. Safety of treatment

    Time frame: 6 months

    Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.

Sponsors and collaborators

Lead sponsor

Muhammad Aamir Latif

Other

Registry information

Official study title

Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients: A Single Center Experience From South Punjab, Pakistan

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Mar 21, 2025
Registry last updated
Mar 21, 2025

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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