CompletedPhase 1

Biotin-RBC Transfusion in SCD

United States22 participantsStarted 2021-10-29

Plain-language 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.

Who can participate

Age range

2 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of Biotin Labeled RBCs

Timeframe: Posttransfusion 24-hour recovery (PTR-24), 28-day recovery, and 90-day recovery

Half-life of Biotinylated RBCs

Timeframe: Day 1 (24 hours post-transfusion) up to Week 12

Mean Potential Lifespan (MPL) of Biotinylated RBCs

Timeframe: Up to Day 70

Trial details

NCT IDNCT04426591

SponsorMarianne Yee

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2025-04-30

Results submitted2026-04-29

View on ClinicalTrials.gov More Sickle Cell Disease trials