Biotin-RBC Transfusion in SCD

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

Conditions

• Sickle Cell Disease

Interventions

DRUG

Biotin Labeled Red Blood Cells

On the day of transfusion, a 20 mL aliquot is sterilely withdrawn from each RBC unit, washed and labeled with sulfo-NHS-biotin for 30 minutes, washed to stop the labeling reaction, then resuspended in plasma to a hematocrit of \~60%. The biotin-labeled RBC (BioRBC) is transfused along with the remainder of the RBC unit (unlabeled volume). Standard blood bank and CTT protocols and minor antigen matching for SCD patients are followed. Exact transfusion volume is determined based on pre-transfusion hemoglobin (Hb), sickle cell hemoglobin (HbS), and body weight, per clinical protocol.

DEVICE

Pathogen-reduced Biotin Labeled Red Blood Cells

Participants taking part in this optional intervention have one transfusion episode with blood using the INTERCEPT Blood System. For this transfusion, a portion of each blood unit is biotin-labeled and one of those units has the INTERCEPT treatment. In addition to the blood drawn for the main study, individuals participating in this optional intervention have additional tubes of peripheral venous blood drawn for evaluating treatment-emergent antibodies specific to INTERCEPT RBCs and acridine surface label monitoring. Tests for treatment-emergent antibodies specific to INTERCEPT RBCs are performed according to procedures developed by Cerus Corporation. This optional study activity examines the survival of transfused RBCs with and without the INTERCEPT treatment.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Cerus Corporation

  collaborator INDUSTRY

• Marianne Yee

  lead OTHER

Principal Investigators

• Marianne Yee, MD · Emory University

Study Design

Allocation

NA

Purpose

BASIC_SCIENCE

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Max Age

65 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2021-10-29

Primary Completion

2025-04-30

Completion

2025-04-30

FDA Drug

Yes

FDA Device

Yes

Countries

• United States

Study Locations