Tiny study probes Mitapivat's inner workings in sickle cell

NCT ID NCT05675436

Ongoing Knowledge-focused Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 11 times

Summary

This observational study will examine how the drug mitapivat changes red blood cells in 6 people with sickle cell disease. Researchers will measure oxygen binding and cell survival markers from blood samples and muscle oxygen tests. The goal is to understand the drug's effects, not to test it as a treatment.

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 successful, this study could reveal how mitapivat improves red blood cell function in sickle cell disease, potentially guiding future treatments.
What could go wrong: This is a very small observational study (6 participants) that does not test treatment outcomes directly. Results may not apply to all patients or lead to new therapies.

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.

hemoglobin H disease Hemoglobin SC Disease sickle cell disease sickle cell disease due to hemoglobin S and a non-S/non-C hemoglobin variant sickle cell-beta plus-thalassemia sickle cell-beta zero-thalassemia sickle cell-beta-thalassemia disease syndrome sickle cell-hemoglobin c disease syndrome

As listed by the trial registrant

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

HBSC HBSS SICKLE BETA THALASSEMIA SICKLE BETA ZERO THALASSEMIA SICKLE CELL ANEMIA SICKLE CELL PAIN SICKLE CELL SYNDROME VARIANT SICKLE CELL THALASSEMIA

Contacts and locations

Locations

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

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