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Could two old drugs free thalassemia patients from lifelong transfusions?

NCT ID NCT07292259

Disease control Sponsor: Pakistan Blood and Marrow Transplant (PBMT) Group Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This phase 2 trial tested whether combining thalidomide and hydroxyurea can reduce the need for blood transfusions in people with transfusion-dependent beta thalassemia. The study enrolled 100 patients over age 2 in Pakistan, where thalassemia is common and life expectancy is low. Participants took both drugs daily for a year, and researchers tracked how many transfusions they needed compared to before treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
thalidomide and hydroxyurea
What this could lead to
If successful, this drug combination could reduce or eliminate the need for regular blood transfusions in people with severe beta thalassemia, improving quality of life and survival in low-resource settings.
What could go wrong
This is a small, early-phase trial (100 patients) with no control group. Thalidomide has serious risks including birth defects and blood clots, and the combination may cause side effects that limit its use.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

100 people

The number who actually took part.

Started

Jan 2024

Finished

Dec 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 12 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

beta-thalassemia major

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Transfusion-dependent beta-thalassemia

Contacts and locations

Locations

  • AFBMTC (CT&RC), CMH Medical Complex

    Rawalpindi, Punjab Province, 46000, Pakistan

  • AFBMTC (CT&RC), Medical Complex

    Rawalpindi, Punjab Province, 46000, Pakistan

  • AFBMTC (Clinical Trial and Research Cell)

    Rawalpindi, Punjab Province, 46000, Pakistan

  • AFBMTC, CMH Medical Complex

    Rawalpindi, Punjab Province, 46000, Pakistan

  • Armed Forces Bone Marrow Transplant Center

    Islamabad, Punjab Province, 46000, Pakistan

  • Armed Forces Bone Marrow Transplant Center

    Rawalpindi, Punjab Province, 44000, Pakistan

  • Armed Forces Bone Marrow Transplant Center

    Rawalpindi, Punjab Province, 46000, Pakistan

  • Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan

    Rawalpindi, Punjab Province, 46000, Pakistan

  • Armed Forces Bone Marrow Transplant Center Rawalpindi Pakistan

    Rawalpindi, Punjab Province, 60000, Pakistan

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