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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called AG-348 in 20 adults with a type of thalassemia that doesn't 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 a day for up to 24 weeks, with an option to continue for up to 10 years.

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Conditions

The condition(s) this trial relates to.

beta-thalassemia intermedia thalassemia

As listed by the trial registrant

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

THALASSEMIA

Contacts and locations

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

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