What side effects are associated with this type of intervention?

The most common treatments for Sickle Cell Disease include hydroxyurea and red blood cell (RBC) transfusions. Hydroxyurea can cause bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia, as well as gastrointestinal disturbances and, rarely, skin ulcers. Chronic RBC transfusions carry risks such as iron overload, alloimmunization, and transfusion reactions. Investigational therapies like biotinylated RBCs are used to track RBC survival and share similar transfusion-related risks.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease, focusing on reducing vaso-occlusive pain and other complications. Hydroxyurea is a well-established medication that reduces the frequency of pain episodes and other complications by increasing fetal hemoglobin production. Additionally, L-glutamine has been approved to reduce acute complications of SCD. While specific FDA approvals for biotinylated RBCs are not mentioned, RBC transfusion remains a common therapeutic approach for managing severe anemia and preventing stroke in SCD patients. These transfusions are critical in managing acute complications and improving the quality of life for individuals with SCD.

What other types of research exist?

Promising novel alternatives to Biotinylated RBCs for tracking RBC survival in Sickle Cell Disease patients include the use of recombinant fusion proteins like rIX-FP and glycoPEGylated recombinant factors such as nonacog beta pegol (N9-GP). These alternatives offer prolonged half-life and enhanced pharmacokinetic properties, which can improve the efficacy of transfusions. Additionally, hematopoietic stem cell transplantation (HCT) remains a potential curative option, especially for patients with matched related donors. These approaches not only aim to improve treatment outcomes but also address complications like iron overload and alloimmunization.

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Red Blood Cell Survival Study for Sickle Cell Disease

Phase 1

Recruiting

Led By Marianne Yee, MD

Research Sponsored by Emory University

Eligibility Criteria Checklist

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

Must have

receiving CTT for ≥3 months prior to enrollment

HbSS or HbSβ0 thalassemia SCD

Must not have

concurrent hydroxyurea therapy

automated RBC exchange therapy within 3 months prior to enrollment or anticipated within the next 3 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1, weeks 1-12

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to understand how long transfused red blood cells (RBCs) last in patients with sickle cell disease (SCD) who receive frequent blood transfusions. Researchers will label a small portion of the transfused RBCs with biotin to track their survival. The study will help identify factors that influence the effectiveness of these transfusions and improve treatment strategies.

Who is the study for?

This trial is for individuals with Sickle Cell Disease (SCD), specifically HbSS or HbSβ0 thalassemia, who have been receiving regular blood transfusions for at least 3 months. It's not suitable for those on hydroxyurea therapy, taking high-dose biotin or raw egg supplements, expecting to stop transfusions soon, having had certain blood exchange therapies or reactions recently, or are currently pregnant.

What is being tested?

The study tests how long transfused red blood cells last in patients with SCD undergoing chronic transfusion therapy. Participants will receive biotin-labeled RBCs during a normal transfusion and then be monitored regularly up to six months. An optional part of the study uses INTERCEPT-treated RBCs to compare survival rates.

What are the potential side effects?

While the main focus is on observing RBC survival rather than side effects, potential risks may include allergic reactions from labeled RBCs and complications related to frequent blood draws such as bruising or infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been on hormone therapy for at least 3 months.

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I have sickle cell disease, either HbSS or HbSβ0 thalassemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking hydroxyurea.

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I have not had, nor plan to have, a red blood cell exchange in the last or next 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1, weeks 1-12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1, weeks 1-12

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1, weeks 1-12 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in Number of Biotin Labeled RBCs

Half-life of Biotinylated RBCs

Mean Potential Lifespan (MPL) of Biotinylated RBCs

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: Biotin labeled Red Blood CellsExperimental Treatment2 Interventions

Participants receiving a transfusion with biotin labeled RBCs. Samples will be taken for 12 weeks after the biotinylated transfusion. During this time participants will continue to receive regular monthly transfusions (non-biotinylated) as part of CTT.

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, which increases fetal hemoglobin (Hb F) production to reduce red blood cell sickling and vaso-occlusive events, and red blood cell (RBC) transfusions, which provide normal RBCs to improve oxygen delivery and reduce complications. Investigational therapies like biotinylated RBCs involve labeling RBCs with biotin to track their survival in vivo, aiding in understanding the longevity and efficacy of transfused cells. These treatments are essential for SCD patients as they help reduce pain, prevent complications, and improve overall quality of life.

Hydroxyurea treatment for sickle cell disease.