New pill shows promise for thalassemia patients in early trial

NCT ID NCT03692052

Ongoing Disease control Sponsor: Agios Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 32 times

Summary

This study tested an experimental drug called AG-348 in 20 adults with a form of thalassemia that does not require regular blood transfusions. The goal was to see if the drug could safely raise hemoglobin levels, which carry oxygen in the blood. Participants took the pill twice daily for up to 24 weeks, with some continuing treatment for up to 10 years.

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Contacts and locations

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Locations

Imperial College Healthcare NHS Trust (Hammersmith Hospital)

London, W12 0HS, United Kingdom
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
University Health Network (Toronto General Hospital)

Toronto, Ontario, M5G 2C4, Canada

Conditions

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THALASSEMIA