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New pill could help kids with thalassemia fight anemia without transfusions

NCT ID NCT07517133

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

First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 2 times

Summary

This Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mitapivat raises hemoglobin levels by at least 1.0 g/dL compared to placebo.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
mitapivat
What this could lead to
If it works, this could provide a new oral treatment option to improve anemia and reduce disease burden in children with thalassemia who do not need regular blood transfusions.
What could go wrong
This is still an early-stage Phase 3 trial with only 45 participants, so results may not apply to all patients. The drug may not work better than placebo, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

alpha thalassemia spectrum beta thalassemia beta-thalassemia intermedia hemoglobin H disease

As listed by the trial registrant

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

NON-TRANSFUSION-DEPENDENT ALPHA-THALASSEMIA NON-TRANSFUSION-DEPENDENT BETA-THALASSEMIA

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

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