Recruiting
Early Phase 1

Red Cell Survival

Sponsor:

National Heart, Lung, and Blood Institute (NHLBI)

Code:

NCT06313398

Conditions

Sickle Cell Disease

Thalassemia

Hemoglobinopathy

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

Biotin-labeled red blood cells

Study Details

Brief summary:

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....

Conditions

Sickle Cell Disease

Thalassemia

Hemoglobinopathy

Study ID

NCT06313398

Start date

May 17, 2024

Status verified date

Aug 27, 2026

Completion date

Jun 15, 2029

Anticipated

Primary completion date

May 1, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

  • 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).

Study Design

Enrollment

100 participants

Anticipated

Intervention Model

Single group

Primary purpose

Other

Interventions and Outcome Measures

Arms

experimental: RBC survival in patients with SCD

RBC lifespan, determined by the mean number of days from biotin-labeled RBC infusion until biotin-labeled RBCs are below limit of detection, in patients with inherited hemoglobinopathies prior to and post initiation of disease modifying therapy or HSCT

Interventions

Biotin-labeled red blood cells

Cellular Product (patient's own red blood cells washed in Biotin and infused back to patient)

Primary outcome measure

  • To determine and compare red blood cell survival, by virtue of the mean number of days of detectable biotin-labeled RBCs, in participants with SCD and/or other hemoglobinopathies. [ Time Frame: Patients w/o transplant: lab draw every 2 weeks until biotin-labeled cells are no longer detected. Patients Post-transplant: lab draw every four weeks, until week 12. Lab draw every 2 weeks until biotin-labeled cells are no longer detected. ]

Central Contacts and Locations

Central contacts

Christina C Luckett

(301) 529-7863christina.luckett@nih.gov

John F Tisdale, M.D.

(301) 402-6497johntis@mail.nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

NIH Clinical Center Office of Patient Recruitment (OPR)

800-411-1222ccopr@nih.gov

More Information

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Last update posted

Aug 31, 2026

Last verified

Aug 27, 2026

Keywords

  • Red Cell
  • Hemoglobinopathy
  • Thalassemia
  • Sickle Cell Disease
  • Biotin

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI) on 2026-08-31.