Tiny study probes Mitapivat's inner workings in sickle cell

NCT ID NCT05675436

First seen Jun 25, 2026 · Last updated Jun 25, 2026

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.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

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.

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

As listed by the trial registrant

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