Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Recruiting · NCT06313398 · Interventional (participants receive a specific treatment) · Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI)
View the official record on ClinicalTrials.gov →Interventions studied
What this trial is about
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how long RBCs survive in people with SCD and other blood disorders before and after treatment compared to those who had a bone marrow transplant. Objective: To learn how long RBCs survive in the body in people with SCD and other blood disorders compared to those whose disease was cured with a bone marrow transplant. Eligibility: People aged 18 years or older with SCD or another inherited blood disorder. People whose SCD or blood disorder was cured with a bone marrow transplant are also needed. Design: Participants will be screened. They will have a physical exam with blood and urine tests. Participants will have about 7 tablespoons of blood drawn. In the lab, this blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs. This process is called "biotin labeling of RBCs." The next day, the participant s own biotin-labeled RBCs will be returned to their bloodstream. Participants will return regularly to have smaller blood samples (about 2 teaspoons) drawn. These samples will be tested to detect the percentage of cells that have biotin labels. These visits may be every 2 weeks, 4 weeks, or some other interval. Participants will continue this schedule for up to 20 weeks or until biotin can no longer be detected....
Who can take part
Inclusion criteria
- In order to be eligible to participate in this study, an individual must meet all of the following criteria:
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged 18 years or greater with confirmed diagnosis of SCD (all genotypes), thalassemia (beta and/or alpha), or other inherited hemoglobinopathy not otherwise specified.
- Be at steady state for their underlying disease (e.g. SCD or thalassemia) or post-bone marrow transplantation status, as evidenced by medical history.
- Ability to have blood samples drawn.
- For female participants of child-bearing potential, agree to use birth control during study participation. Female subjects of child-bearing potential must agree to use a medically acceptable method of birth control such as an oral contraceptive, intrauterine device, barrier and spermicide, or contraceptive implant/injection from start of screening through 4 months after infusion.
- Agreement to adhere to Lifestyle Considerations throughout study duration
Exclusion criteria
- An individual who meets any of the following criteria will be excluded from participation in this study:
- Consumption of biotin supplements or raw eggs within the last 30 days.
- Blood loss within the previous 8 weeks (\>540 mL).
- Red cell transfusion for their underlying SCD and/or thalassemia within the last 3 months.
- a. Participants may be eligible after three months following their last transfusion.
- Patients on hemodialysis, due to possibility of early removal of biotinylated RBCs.
- Pregnancy, lactation or absence of adequate contraception for fertile female subjects.
- Pediatric subjects will not participate in this study.
- Known allergic reactions to biotin, due to risk of possible life-threatening allergic reaction.
- Current diagnosis of malignancy (liquid and/or solid).
Where it is running
1 location listed across 1 US state.
- National Institutes of Health Clinical Center - Bethesda, Maryland, United States