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RecruitingEarly Phase 1

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 100 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Urine samples, Physical exams, Blood draws

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "They will have a physical exam with blood and urine tests."
  • "Participants will return regularly to have smaller blood samples (about 2 teaspoons) drawn."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study uses biotin, a vitamin, to track how long red blood cells live in the body. It is for adults with sickle cell disease or other inherited blood disorders, including those cured by a bone marrow transplant. Researchers will compare how long the cells live before and after treatment.

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....

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older with a confirmed inherited blood disorder, such as sickle cell disease or thalassemia, or have had it cured by a bone marrow transplant.
  • Have a stable blood disorder or stable health after a bone marrow transplant, based on your medical history.
  • Sign and date a form agreeing to join the study.
  • Be willing to follow study steps and be available for the whole study.
  • Be able to have blood samples taken.
  • If you are a woman who can become pregnant, use an accepted birth control method from screening until 4 months after your labeled blood cells are returned.
  • Agree to follow the study’s lifestyle rules.

Who can't join

  • Have taken biotin supplements or eaten raw eggs in the past 30 days.
  • Have lost more than 540 mL of blood in the past 8 weeks.
  • Have received red blood cells for sickle cell disease or thalassemia in the past 3 months. You may qualify after 3 months.
  • Receive hemodialysis, a treatment that uses a machine to clean your blood.
  • Are pregnant or breastfeeding, or are a woman who can become pregnant but are not using adequate birth control.
  • Are younger than 18.
  • Have a known allergy to biotin, which could cause a life-threatening reaction.
  • Currently have cancer, including blood cancers or solid tumors.

Simplified for readability. The official criteria below are what the study team uses.

Official 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).

Study sites (1)

MD