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

NCT06888232 | Completed

• 1 other identifier: DRASLAMCHMULTAN
• Study Type: interventional
• Target: 121
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Thalassemia

Outcome Measures

Primary Outcomes (2)

• Efficacy of treatment

  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.

  6 months

• Safety of treatment

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

  6 months

Study Arms (1)

Thalidomide group

EXPERIMENTAL

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.

Drug: Thalidomide

Interventions

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.

Thalidomide group

Eligibility Criteria

• Age: 2 Years - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Muhammad Aamir Latif: lead

Study Sites (1)

The Children's Hospital & Institute of Child Health

Multan, Punjab Province, 66000, Pakistan

Location

MeSH Terms

Conditions

Thalassemia

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Muhammad Aslam, FCPS

  Children's Hospital and institute of Child Health Multan, Punjab, Pakistan

  PRINCIPAL INVESTIGATOR

• Sara Rubab, FCPS

  Children's Hospital and institute of Child Health Multan, Punjab, Pakistan

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Research Consultant

Study Record Dates

• First Submitted: March 15, 2025
• First Posted: March 21, 2025
• Study Start: August 1, 2023
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: March 21, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)