Early Phase 1
N=22
Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Sickle Cell Disease · Sickle Cell Anemia
Bottom Line
View on ClinicalTrials.gov: NCT04476277 ↗Enrolled (actual)
22
Serious AEs
0.0%
Results posted
Feb 2024
Primary outcome: Primary: Mean Red Blood Cells Lifespan in Participants — 64.1; 113.4; 126.0; 123.7 day — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Early Phase 1
- Interventions
- Biotin label (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Red Blood Cells Lifespan in Participants |
64.1; 113.4; 126.0; 123.7 | <0.001 sig |
| SECONDARY Number of Participants With Antibody Detection |
0; 0; 0; 0 | — |
| SECONDARY Mean White Blood Cell Count |
9.0; 6.5 | 0.28 |
| SECONDARY Mean Red Blood Cell Count |
3.0; 4.3 | 0.04 sig |
| SECONDARY Mean Hemoglobin Value |
8.8; 13.0 | 0.01 sig |
| SECONDARY Mean Hematocrit Value |
25.0; 38.3 | 0.01 sig |
| SECONDARY Mean Value of Mean Corpuscular Volume |
86.7; 89.4 | 0.65 |
| SECONDARY Mean Absolute Reticulocyte Count |
192.7; 152.9 | 0.48 |
| SECONDARY Mean Aspartate Aminotransferase Value |
26.1; 26.1 | 0.99 |
| SECONDARY Mean Total Bilirubin Value |
7.1; 1.1 | 0.39 |
| SECONDARY Mean Lactate Dehydrogenase Value |
323.9; 253.7 | 0.43 |
| SECONDARY Mean Adult Hemoglobin Percentage |
2.8; 59.6 | <0.01 sig |
| SECONDARY Mean Sickle Hemoglobin Percentage |
68.7; 36.7 | 0.01 sig |
| SECONDARY Mean Fetal Hemoglobin Percentage |
10.4; .5 | 0.04 sig |
Summary
Background:
Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body. In people with SCD, some RBCs are abnormal and die early. This leaves a shortage of healthy RBCs. Researchers want to learn more about how long RBCs live in the human body.
Objective:
To study how long RBCs live in people with and without SCD.
Eligibility:
People age 18 and older who either have SCD, had SCD but were cured with a bone marrow transplant, have the sickle cell trait (SCT), or are a healthy volunteer without SCD or SCT
Design:
Participants will be screened with a medical history and physical exam. They will give a blood sample.
Participants will have a small amount of blood drawn from a vein. In the laboratory, the blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs without changing their function, shape, or overall lifetime. This process is known as biotin labeling of RBCs. The biotin labeled RBCs will be returned to the participant via vein injection.
Participants will give frequent blood samples. Their RBCs will be studied to see how many biotin labeled RBCs remain over time. This shows how long the RBCs live. Participants will give blood samples until no biotin labeled RBCs can be detected.
During the study visits, participants will report any major changes to their health.
Participation lasts for up to 6 months.
Eligibility Criteria
INCLUSION CRITERIA
- Age 18 or greater with a confirmed diagnosis of homozygous SCD (HbSS, HbSC, HbSB0), sickle cell trait (HbAS), or healthy volunteer (HbA)
- Normal renal function: creatinine 540mL
- Pregnancy
- Pre-existing, naturally occurring antibodies against biotin
Data sourced from ClinicalTrials.gov (NCT04476277). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.