Biotin Labeling for Sickle Cell Anemia

Phase < 1

Recruiting

Led By John F Tisdale, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up patients w/o transplant: lab draw every 2 weeks until biotin-labeled cells are no longer detected. patients post-transplant: lab draw every four weeks, until week 12. lab draw every 2 weeks until biotin-labeled cells are no longer detected.

Awards & highlights

No Placebo-Only Group

Summary

"This trial aims to determine the lifespan of red blood cells in patients with sickle cell disease and other blood disorders, before and after receiving treatments such as stem cell transplantation. The study will use a labeling

Who is the study for?

This trial is for individuals with sickle cell disease or other hemoglobin disorders like thalassemia. It's open to those who are on disease-modifying treatments or have had a stem cell transplant. The study aims to understand how long their red blood cells live using biotin labeling.

What is being tested?

The trial tests the lifespan of red blood cells in patients with sickle cell and related disorders by marking them with biotin, a vitamin. Researchers will compare this before and after treatments like transplants, looking at how different therapies affect red cell survival.

What are the potential side effects?

Since the intervention involves labeling cells with biotin, which is generally considered safe and is a common dietary supplement, significant side effects are not expected. However, individual reactions may vary.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ patients w/o transplant: lab draw every 2 weeks until biotin-labeled cells are no longer detected. patients post-transplant: lab draw every four weeks, until week 12. lab draw every 2 weeks until biotin-labeled cells are no longer detected.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~patients w/o transplant: lab draw every 2 weeks until biotin-labeled cells are no longer detected. patients post-transplant: lab draw every four weeks, until week 12. lab draw every 2 weeks until biotin-labeled cells are no longer detected.

This trial's timeline: 3 weeks for screening, Varies for treatment, and patients w/o transplant: lab draw every 2 weeks until biotin-labeled cells are no longer detected. patients post-transplant: lab draw every four weeks, until week 12. lab draw every 2 weeks until biotin-labeled cells are no longer detected. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

To determine and compare red blood cell survival, by virtue of the mean number of days of detectable biotin-labeled RBCs, in participants with SCD and/or other hemoglobinopathies.

Secondary study objectives

Validate the association of RBC survival with known markers of increased survival.

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RBC survival in patients with SCDExperimental Treatment1 Intervention

RBC lifespan, determined by the mean number of days from biotin-labeled RBC infusion until biotin-labeled RBCs are below limit of detection, in patients with inherited hemoglobinopathies prior to and post initiation of disease modifying therapy or HSCT

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