Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Mar 14, 2024

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ClinConnect Summary

This clinical trial is studying how long red blood cells (RBCs) last in people with sickle cell disease (SCD) and other blood disorders, including those who have undergone a bone marrow transplant. Researchers want to understand the survival rate of these cells before and after treatment, which is important for improving care for patients with these conditions. The trial is looking for adults aged 18 and older who have SCD, thalassemia, or another inherited blood disorder. People who have been cured with a bone marrow transplant can also participate.

If you join the trial, you will go through a screening process that includes a physical exam and blood tests. You’ll have about 7 tablespoons of blood drawn, which will be treated with a vitamin called biotin that helps track your RBCs. The next day, your biotin-labeled RBCs will be returned to your body. Over the next 20 weeks, you will have smaller blood samples taken regularly to see how many of your cells still have the biotin label. It's important to note that you should not take biotin supplements or have certain medical conditions to qualify for the study. If you're interested, please speak with your healthcare provider for more information.

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Eligibility criteria

• * INCLUSION CRITERIA:
• In order to be eligible to participate in this study, an individual must meet all of the following criteria:
• • 1. Provision of signed and dated informed consent form
• • 2. Stated willingness to comply with all study procedures and availability for the duration of the study
• • 3. 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.
• • 4. Be at steady state for their underlying disease (e.g. SCD or thalassemia) or post-bone marrow transplantation status, as evidenced by medical history.
• • 5. Ability to have blood samples drawn.
• • 6. 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.
• • 7. 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:
• • 1. Consumption of biotin supplements or raw eggs within the last 30 days.
• • 2. Blood loss within the previous 8 weeks (\>540 mL).
• • 3. Treatment with chronic transfusion therapy for their underlying SCD and/or thalassemia.
• • a. Participants with history of chronic transfusion therapy can be eligible three months following their last transfusion.
• • 4. Patients on hemodialysis, due to possibility of early removal of biotinylated RBCs.
• • 5. Pregnancy, lactation or absence of adequate contraception for fertile female subjects.
• • 6. Pediatric subjects will not participate in this study.
• • 7. Known allergic reactions to biotin, due to risk of possible life-threatening allergic reaction.
• • 8. Current diagnosis of malignancy (liquid and/or solid).