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Could a pill cut transfusions for sickle cell patients?

NCT ID NCT07656415

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

First seen Jun 26, 2026 · Last updated Aug 05, 2026 · Updated 5 times

Summary

This Phase 3 trial tests whether the drug mitapivat can reduce the need for blood transfusions in people with sickle cell disease. About 159 participants aged 12 and older will receive either mitapivat or a placebo daily for a year. The main goal is to see if more people can avoid transfusions while taking the drug.

What this could mean

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

Active substance
Mitapivat (a drug taken as a tablet)
What this could lead to
If it works, mitapivat could reduce or eliminate the need for frequent blood transfusions in people with sickle cell disease, improving their quality of life.
What could go wrong
This is an early-stage Phase 3 trial, so results are not yet known. The drug may not work better than placebo, and side effects are possible. It is not a cure.

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.

sickle cell disease

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Indiana Hemophilia & Thrombosis Center

    RECRUITING

    Indianapolis, Indiana, 46260, United States

  • Southern Specialty Clinic

    RECRUITING

    Flowood, Mississippi, 39232, United States

  • The Cancer and Hematology Centers (CHC)

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

More trials for these conditions

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