NCT04426591CompletedPhase 1
Biotin-RBC Transfusion in SCD
Kinetics of Red Blood Cell Clearance in Chronically Transfused Children With Sickle Cell Disease
Source: ClinicalTrials.gov · Last updated 22 May 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 22 (actual)
- First submitted
- 29 May 2020
- Start date
- 29 Oct 2021
- Primary completion date
- 30 Apr 2025
- Lead sponsor
- Marianne Yee
- Diseases
- Sickle cell disease
- NCT number
- NCT04426591
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 22
- From 29 Oct 2021 to the primary completion date, 30 Apr 2025
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other
- Anticipated cessation of CTT in the next ≤2 months
- Ongoing consumption of biotin or raw egg dietary supplements
Who the study is looking for
- 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
Age in the registry: 2 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Childrens Healthcare of Atlanta Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Grady Health System Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
| Hughes Spalding Children's Hospital Coordinator of this site? Confirm its status | Atlanta | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.