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Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia

NCT07292259 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 100

Last updated 2026-05-05

No results posted yet for this study

Summary

Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation. Around 23,000 babies are affected by BTM each year, of which around 90% belong to low- or middle-income nations.

In Pakistan, the carrier prevalence of thalassemia is 5-7% resulting in a significant population of approximately 10 million carriers in the general population. There are 50,000 thalassemia patients registered in treatment facilities around the country, one of the highest global prevalence rates for transfusion dependent BTM. The average life expectancy of BTM patients in Pakistan is around 10 years of age, while life expectancy in developed countries is around 50 to 60 years. This difference is due to poor transfusion support, transfusion-transmitted infections (TTIs) and inadequate iron chelation leading to hepatotoxicity and cardiac failure.

The standard of care for BTM remains bone marrow transplantation or lifelong blood transfusions followed by iron chelation therapies. While standard care involves, challenges such as limited resources, lack of access to transplant services, and transfusion-related complications persist, particularly in low-and-middle-income countries.

Conditions

  • Transfusion Dependent Beta Thalassemia

Interventions

DRUG

ADDITION OF THALIDOMIDE AND HYDROXYUREA

The intervention includes Hydroxyurea (HU) and Thalidomide in combination. The starting dose of Hydroxyurea will be 20 mg/kg once daily and of thalidomide will be 2.5-3 mg/kg once a day adjusted to nearest multiple of 10, at bedtime. Among those with partial response (PR) or no response (NR) after two months, the dose of thalidomide will be escalated in increments of 1 mg/kg/day at four weeks interval to a maximum of 5 mg/kg/day (maximum dose 100 mg/day). * To prevent thrombosis, aspirin (2-4 mg/kg per day) will be used. All patients will receive Folic acid 2 to 5 mg once daily. * Patients will also continue the iron chelation therapy (Deferasirox, Deferiprone or Deferoxamine) in case of iron overload.

Sponsors & Collaborators

  • Pakistan Blood and Marrow Transplant (PBMT) Group

    lead OTHER

Principal Investigators

  • Tariq Ghafoor, FCPS,FRCP · National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-01
Primary Completion
2025-12-30
Completion
2025-12-30

Countries

  • Pakistan

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07292259 on ClinicalTrials.gov