This study is currently recruiting participants.
National Heart, Lung and Blood Institute (NHLBI)
Type: Enrolling by Invitation Gender: Male & Female Min Age: 16 Years Max Age: N/A
Referral Letter Required
Sickle Cell Pain Crisis; Sickle Cell Anemia; Sickle Cell Treatment; Sickle Cell Therapy; Hemoglobin; Natural History
Sickle Cell Anemia; Sickle Cell Thalassemia; Sickle Cell Pain; Hbss; Hbsc; Sickle Beta Thalassemia; Sickle Beta Zero Thalassemia; Sickle Cell Syndrome Variant
National Heart, Lung, and Blood Institute
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.
To learn how mitapivat affects red blood cells in people with SCD.
People with SCD who are enrolled in the parent study, NIH protocol 000997.
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.
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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.
All subjects enrolled at NIH meeting eligibility for the parent study (AG348-C-020) are eligible for this study.
Subjects that did not meet eligibility criteria to the parent protocol (AG348-C-020) will not be eligible to enroll for this study.
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