Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

100

participants targeted

Target at P50-P75 for phase_2

Timeline

Completed

Started Jan 2024

Geographic Reach

1 country

9 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

2 years study duration

Study Start

First participant enrolled

January 1, 2024

Completed

1.9 years until next milestone

First Submitted

Initial submission to the registry

November 13, 2025

Completed

1 month until next milestone

First Posted

Study publicly available on registry

December 18, 2025

Completed

12 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2025

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2025

Completed

Last Updated

May 5, 2026

Status Verified

December 1, 2025

Enrollment Period

2 years

First QC Date

November 13, 2025

Last Update Submit

April 29, 2026

Conditions

Transfusion Dependent Beta Thalassemia

Keywords

Thalidomide, Hydoxyurea, Transfusion dependent thalassemia

Outcome Measures

Primary Outcomes (1)

Number of Red Blood Cell Transfusions after starting a combination of Thalidomide and hydroxyurea
The frequency of Red Blood cell transfusions before the start of intervention will be noted. At 03 months, 06 months, and 12 months of intervention (Thalidomide and hydroxyurea) number of Red Blood cell transfusions will be documented.
01 Year

Secondary Outcomes (3)

To document the spectrum and frequency of significant adverse drug reactions.
01 Year
To assess changes in spleen size during the intervention.
01 Year
Monitoring of Serum Ferritin levels in ng/ml during the time of intervention.
01 Year

Study Arms (1)

A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT

EXPERIMENTAL

* The trial include 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.

Drug: ADDITION OF THALIDOMIDE AND HYDROXYUREA

Interventions

ADDITION OF THALIDOMIDE AND HYDROXYUREADRUG

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.

Also known as: Hydrea and Thalidomide

A combination of thalidomide and hydroxyurea is added to patients diagnosed with TDT

Eligibility Criteria

Age2 Years - 12 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Patients suffering from Transfusion Dependent β-thalassemia (TDBT) more than two years of age of either sex will be included in the study.

You may not qualify if:

Age less than two years.
Patients having cardiac, hepatic, pulmonary, renal or neurological dysfunction or history of thrombosis.
Both male and female participants of childbearing potential will be excluded due to the teratogenicity of thalidomide.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pakistan Blood and Marrow Transplant (PBMT) Grouplead

Study Sites (9)

Armed Forces Bone Marrow Transplant Center

Islamabad, Punjab Province, 46000, Pakistan

Location

Armed Forces Bone Marrow Transplant Center

Rawalpindi, Punjab Province, 44000, Pakistan

Location

AFBMTC (Clinical Trial and Research Cell)