Understanding red blood cell lifespan in sickle cell disease and hemoglobin disorders

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

This study is trying to see how long red blood cells last in people with sickle cell disease and other blood disorders to help improve their treatment.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 18 Years to 100 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (NIH)
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT06313398 on ClinicalTrials.gov

What this trial studies

This study aims to determine the lifespan of red blood cells (RBCs) in individuals with sickle cell disease (SCD) and other hemoglobinopathies using biotin labeling techniques. Participants will undergo screening and provide blood samples, which will be analyzed to compare RBC survival before and after treatments such as bone marrow transplantation. The research seeks to understand how various factors influence RBC longevity in these patients, contributing to better management of their conditions.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of sickle cell disease, thalassemia, or other inherited hemoglobinopathies.

Not a fit: Patients with non-inherited blood disorders or those under 18 years old may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment strategies for patients with sickle cell disease and other hemoglobinopathies.

How similar studies have performed: Previous studies have shown success in determining red blood cell lifespan using similar biotin labeling techniques, indicating a promising approach.

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. 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.
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).

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: John F Tisdale, M.D. — National Heart, Lung, and Blood Institute (NHLBI)
• Study coordinator: Christina C Luckett

Conditions Sickle Cell Disease; Thalassemia; Hemoglobinopathy; Red Cell; Biotin