What the trial was testing
The trial enrolled 17 patients with sickle cell disease. The study was sponsored by National Institutes of Health Clinical Center and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Hemoglobin rose 1.2 g/dL — and 56% had a meaningful response.
Blood · 2022 · NCT04000165
These findings — that in hemoglobin on mitapivat 50 mg twice daily in adults with sickle cell disease — were published in the Blood and represent the headline result of the study.
Researchers tracked outcomes across 17 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with sickle cell disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Mitapivat (Pyrukynd) is FDA-approved for pyruvate kinase deficiency anemia but is not yet approved for sickle cell disease. The Phase 3 RISE UP trial was positive, and FDA review is ongoing as of early 2026. Several FDA-approved sickle cell options exist: hydroxyurea, L-glutamine, crizanlizumab, and gene therapies. Ask your hematologist about access.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open sickle cell disease trials
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.
Study of Erythrocyte Parameters and Hypercoagulability in Sickle Cell Disease (SCD-TGA)
Sickle cell disease (SCD) is an inherited haemoglobinopathy disorder caused by mutations in HBB gene with amino-acid substitution on β globin chain. The consequence is synthesis of altered haemoglobin S (HbS) which polymerises in red blood cell (RBC) at deoxygenated state. SCD is associated with chronic haemolytic anaemia, vaso-occlusive crisis (VOC) leading to frequent hospitalisation. The aim of the study was to to investigate whether a combination of routine laboratory biomarkers of haemolysis could be used to predict VOC development in confirmed SCD patients.