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Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

NCT05675436 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 6

Last updated 2026-04-29

No results posted yet for this study

Summary

Background:

Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys. SCD can lead to serious illness and death. Treatments such as bone marrow transplants and gene therapies can cure SCD, but they are not widely available. Current drug treatments for SCD are not always effective. This natural history study will examine how a study drug (mitapivat) affects red blood cells in people with SCD.

Objective:

To learn how mitapivat affects red blood cells in people with SCD.

Eligibility:

People with SCD who are enrolled in the parent study, NIH protocol IRB001565-H.

Design:

Procedures for this study will be done during visits already scheduled for the parent study.

Participants will have additional blood drawn during study visits. The additional amount will be about 3.5 teaspoons.

Participants will undergo a test called near infrared spectroscopy (NIRS) up to 9 times. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or to hold their breath during these measurements. NIRS measures oxygen levels, blood flow, and the makeup of skin and muscle.

Researchers will draw additional information for this study from participants medical records.

Conditions

  • Sickle Cell Anemia
  • Sickle Cell Thalassemia
  • Sickle Cell Pain
  • Hbss
  • Hbsc
  • Sickle Beta Thalassemia
  • Sickle Beta Zero Thalassemia
  • Sickle Cell Syndrome Variant

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • Swee Lay Thein, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Eligibility

Min Age
16 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-05-09
Primary Completion
2027-10-27
Completion
2027-10-27

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05675436 on ClinicalTrials.gov