Red Blood Cell Survival in Sickle Cell Disease

• Status: Recruiting
• Trial end date: 2025-05-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Emory University
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Biotin

Criteria

Inclusion Criteria:

• HbSS or HbSβ0 thalassemia SCD

• receiving CTT for ≥3 months prior to enrollment

Exclusion Criteria:

• anticipated cessation of CTT in the next ≤2 months

• concurrent hydroxyurea therapy

• automated RBC exchange therapy within 3 months prior to enrollment or anticipated within the next 3 months

• delayed hemolytic transfusion reaction in the past 3 months

• consuming high-dose biotin or raw egg supplements

• current pregnancy