Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

NCT06490627 | Recruiting Phase 2 DMC

• 1 other identifier: NIBD/IRB-266/01-2024
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 1

Brief Summary

The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects. Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.

Conditions

Fetal Hemoglobin; Thalassemia Major; Genetic Disease

Keywords

thalidomide; safety and efficacy; thalassemia; fetal hemoglobin inducer

Outcome Measures

Primary Outcomes (1)

• transfusion dependency

  Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event

  1 year

Secondary Outcomes (16)

• hemoglobin levels.

  2 years

• total leukocyte count.

  2 years

• reticulocyte count

  2 years

• Platelets

  2 years

• impact of genetic modifiers (e.g., HBB mutation, XMN polymorphism, BCL11A polymorphism, and co-inheritance of alpha chain) on the treatment outcomes with thalidomide.

  2 years

Other Outcomes (1)

• Safety Profile:

  2 years

Study Arms (3)

Control

NO INTERVENTION

in this group, patient will not receive any interventional drug or treatment drug so simply they will blood transfusion dependent.

Group 1 Thalidomide

EXPERIMENTAL

In this group , thalassemia patients will receive thalidomide at the dose of 5-6mg/kg/day at night with aspirin.

Drug: Thalidomide

Group 2 Thalidomide

ACTIVE COMPARATOR

In this group , thalassemia patients will receive thalidomide at the dose of 7-8mg/kg/day at night with aspirin

Drug: Thalidomide

Interventions

Thalidomide DRUG

Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 5-6mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.

Group 1 Thalidomide

Eligibility Criteria

• Age: 8 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Know case of beta thalassemia major/ intermediate ( transfusion dependent)
• willing to give informed consent

You may not qualify if:

• Patients with comorbidities such as liver dysfunction
• Married patients
• Lactating mother
• H/O thrombosis and fits

Sponsors & Collaborators

• National Institute of Blood and Marrow Transplant (NIBMT), Pakistan: lead

Study Sites (1)

National Institute of blood disease and bone marrow transplant

Karachi, Sindh, Pakistan

RECRUITING

Related Publications (1)

• Swabb EA, Sugerman AA, Platt TB, Pilkiewicz FG, Frantz M. Single-dose pharmacokinetics of the monobactam azthreonam (SQ 26,776) in healthy subjects. Antimicrob Agents Chemother. 1982 Jun;21(6):944-9. doi: 10.1128/AAC.21.6.944.

  PMID: 7202343 BACKGROUND

MeSH Terms

Conditions

beta-Thalassemia; Genetic Diseases, Inborn; Thalassemia

Interventions

Thalidomide

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; 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 Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Doctor

Model Details: in this study three groups have been made. one is control group which received no intervention and on transfusion only. whereas other two groups are interventional both received thalidomide. one received thalidomide at 5-6mg/kg/day and second at 7-8 mg/kg/day.

Study Record Dates

• First Submitted: June 20, 2024
• First Posted: July 8, 2024
• Study Start: April 22, 2024
• Primary Completion: April 22, 2025
• Study Completion: April 22, 2026
• Last Updated: May 30, 2025

Record last verified: 2025-05

Locations

PA (1)