Could two old drugs free thalassemia patients from lifelong transfusions?

NCT ID NCT07292259

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

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.

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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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