Biotin-RBC Transfusion in SCD

Completed · Phase 1

Conditions studied: Sickle Cell Disease

In brief

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. An optional sub-study using INTERCEPT RBCs will mirror the main study but will use INTERCEPT RBCs that have biotinylated for 1 RBC unit.

Key facts

Study ID

NCT04426591

Run by

Marianne Yee

People needed

22

Starts

2021-10-29

Expected to finish

2025-04-30

Last updated by the study team

2026-05-22

Who can join

Age: 2 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Hemoglobinopathy:
• Any sickle cell disease genotype, or
• Transfusion-dependent thalassemia (TDT)
• Receiving CTT for ≥3 months prior to enrollment
• For participants with past BioRBC transfusion exposure, BioRBC antibody screens must have been conducted through at least 6 months post exposure, with negative results

You may not qualify if…

• Anticipated cessation of CTT in the next ≤2 months
• Ongoing consumption of biotin or raw egg dietary supplements
• Antibody specific of INTERCEPT RBCs at baseline (for subjects consenting to the optional arm)
• BioRBC-specific antibodies ever detected in the past, or detected on post-enrollment screening prior to first infusion of Bio-RBC

Where it is running

• Hughes Spalding Children's Hospital — Atlanta, Georgia, United States
• Childrens Healthcare of Atlanta — Atlanta, Georgia, United States
• Grady Health System — Atlanta, Georgia, United States

Full record on ClinicalTrials.gov

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