NCT05675436Active, not recruiting
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Observational Study Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Source: ClinicalTrials.gov · Last updated 29 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 6 (actual)
- First submitted
- 6 Jan 2023
- Start date
- 9 May 2024
- Primary completion date
- 27 Oct 2027
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Diseases
- Sickle cell disease, Thalassaemia
- NCT number
- NCT05675436
What you commit to
- Study type: Observational
- Participants enrolled: 6
- From 9 May 2024 to the primary completion date, 27 Oct 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other
- Subjects that did not meet eligibility criteria to the parent protocol (AG348-C-020) will not be eligible to enroll for this study.
Criteria the registry did not label
- INCLUSION CRITERIA:
- All subjects enrolled at NIH meeting eligibility for the parent study (AG348-C-020) are eligible for this study.
Age in the registry: 16 years to 80 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| National Institutes of Health Clinical Center Coordinator of this site? Confirm its status | Bethesda | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.