Old drugs, new hope: could a thalidomide combo free thalassemia patients from transfusions?

NCT ID NCT07673302

Recruiting now Disease control Sponsor: Riphah International University Source: ClinicalTrials.gov ↗

First seen Jun 29, 2026 · Last updated Aug 07, 2026 · Updated 3 times

Summary

This study tests whether a combination of two drugs, thalidomide and hydroxyurea, can safely boost fetal hemoglobin levels in people with beta thalassemia major. The goal is to improve overall hemoglobin levels and reduce the need for regular blood transfusions. The trial includes 240 participants of all ages and will track changes in hemoglobin, transfusion frequency, and quality of life over 12 months.

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 combination could reduce or eliminate the need for regular blood transfusions in people with beta thalassemia.
What could go wrong: This is a single-arm, non-randomized study with no placebo group, so results may be less reliable. Thalidomide and hydroxyurea have known side effects, including birth defects and bone marrow suppression.

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Conditions

The condition(s) this trial relates to.

beta-thalassemia major thalassemia

As listed by the trial registrant

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

THALASSEMIA

Contacts and locations

Locations

Riphah International University
RECRUITING

Rawalpindi, Punjab Province, Pakistan

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