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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06313398
Other study ID # 10001883
Secondary ID 001883-H
Status Recruiting
Phase Early Phase 1
First received
Last updated
Start date May 17, 2024
Est. completion date June 15, 2029

Study information

Verified date May 14, 2024
Source National Institutes of Health Clinical Center (CC)
Contact Christina C Luckett
Phone (301) 827-7901
Email christina.luckett@nih.gov
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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....


Description:

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Study Design


Intervention

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

Locations

Country Name City State
United States National Institutes of Health Clinical Center Bethesda Maryland

Sponsors (2)

Lead Sponsor Collaborator
National Heart, Lung, and Blood Institute (NHLBI) Children's National Research Institute

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. Enumeration of biotin-labeled RBCS (BioRBCs) will be performed by flow cytometry. F-cell and/or S-cell measurement will be done by flow cytometry. BioRBCs will be isolated from post-infusion blood samples using flow cytometry separation. The collected BioRBCs and the whole blood fraction will be analyzed by high performance liquid chromatography (HPLC) for hemoglobin content. 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.
Secondary Validate the association of RBC survival with known markers of increased survival. We will measure the relationship of RBC survival to hematologic parameters known to be associated with survival, specifically hemolysis markers (e.g. absolute reticulocyte count, lactate dehydrogenase, total bilirubin, and aspartate aminotransferase), hemoglobin F or hemoglobin A percentage, and alpha globin gene mutation status obtained at baseline and during scheduled lab draws (except for alpha globin gene mutation analysis which is only done at baseline). 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.
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