Measuring how long transfused red blood cells survive in sickle cell disease patients
Kinetics of Donor Red Blood Cell Survival in Sickle Cell Disease
This study is testing how long transfused red blood cells last in people with sickle cell disease who are getting regular blood transfusions.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 2 Years to 65 Years |
| Sex | All |
| Sponsor | Emory University Academic / other |
| Locations | 3 sites (Atlanta, Georgia and 2 other locations) |
| Trial ID | NCT04426591 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to investigate the survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) who are undergoing chronic transfusion therapy. Participants will receive regular blood transfusions, during which a small portion of the RBCs will be labeled with biotin to track their survival over time. The study will involve follow-up visits at various intervals to measure how long these labeled RBCs remain in circulation. Additionally, an optional sub-study will explore the use of INTERCEPT RBCs to further understand their survival dynamics.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with any genotype of sickle cell disease or transfusion-dependent thalassemia who have been receiving chronic transfusion therapy for at least three months.
Not a fit: Patients who are expected to stop chronic transfusion therapy within the next two months or those with specific antibody responses to INTERCEPT RBCs may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved chronic transfusion strategies that enhance the efficacy of treatment for sickle cell disease.
How similar studies have performed: While the approach of measuring RBC survival in SCD patients is novel, previous studies have indicated variability in transfused RBC survival, suggesting that this area of research is both relevant and necessary.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Atlanta, Georgia and 2 other locations
- Hughes Spalding Children's Hospital — Atlanta, Georgia, United States (Recruiting)
- Childrens Healthcare of Atlanta — Atlanta, Georgia, United States (Recruiting)
- Grady Health System — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Marianne Yee, MD — Emory University
- Study coordinator: Marianne Yee, MD
- Email: Marianne.Yee@choa.org
- Phone: 404-785-6190
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.