Could two old drugs free thalassemia patients from lifelong transfusions?

NCT ID NCT07292259

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

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