Phase 1
N=22
Biotin-RBC Transfusion in SCD
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT04426591 ↗Enrolled (actual)
22
Serious AEs
54.6%
Results posted
May 2026
Primary outcome: Primary: Percentage of Biotin Labeled RBCs — 97.3; 80; 21 percentage of biotin-labeled RBCs
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Biotin Labeled Red Blood Cells (Drug); Pathogen-reduced Biotin Labeled Red Blood Cells (Device)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Marianne Yee
- Primary completion
- Apr 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Biotin Labeled RBCs |
97.3; 80; 21 | — |
| PRIMARY Half-life of Biotinylated RBCs |
60 | — |
| PRIMARY Mean Potential Lifespan (MPL) of Biotinylated RBCs |
— | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT04426591). 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.