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Tiny study probes Mitapivat's inner workings in sickle cell

NCT ID NCT05675436

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 17, 2026 · Updated 17 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.

Study facts

What this study's own registry entry says, in plain language.

Participants

6 people

The number who actually took part.

Started

May 2024

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

primary clinical

Ages

16 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* INCLUSION CRITERIA: All subjects enrolled at NIH meeting eligibility for the parent study (AG348-C-020) are eligible for this study. EXCLUSION CRITERIA: Subjects that did not meet eligibility criteria to the parent protocol (AG348-C-020) will not be eligible to enroll for this study. Subjects will be screened for implanted metal objects or devices that may be incompatible with MRI (i.e. cerebral aneurysm clip, cochlear implant, pacemaker, etc.) These subjects will be eligible to proceed with study enrollment, but will not undergo the optional MRI study.

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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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