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, skin and nail changes, and potential long-term risks like infertility in men. RBC transfusions can result in complications such as alloimmunization, iron overload, and transfusion reactions, including acute and delayed hemolytic reactions, transfusion-associated circulatory overload (TACO), and transfusion-related acute lung injury (TRALI). These treatments require careful monitoring to manage these side effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease (SCD), including hydroxyurea and red blood cell transfusions. Hydroxyurea is a disease-modifying therapy that helps reduce the frequency of painful episodes and other vaso-occlusive complications. Red blood cell transfusions are commonly used to manage severe complications such as acute chest syndrome and to prevent stroke. These transfusions can involve either simple transfusion or exchange transfusion techniques. Both treatments aim to improve the quality of life and reduce the morbidity associated with SCD.

What other types of research exist?

Promising alternatives to red blood cell transfusion for sickle cell disease patients include hydroxyurea therapy and the combination of hydroxyurea with phlebotomy. Hydroxyurea has been shown to reduce the frequency of pain crises and the need for transfusions. Additionally, gene therapy and CRISPR-based treatments are emerging as potential curative approaches by directly addressing the genetic mutations responsible for SCD. These alternatives aim to reduce complications such as iron overload and alloimmunization associated with chronic transfusions. While the lifespan of transfused RBCs from G6PD-deficient donors may be shorter, these novel treatments focus on reducing the need for transfusions altogether, thereby mitigating related risks.

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Red Blood Cell Transfusion

Red Blood Cell Transfusion Impact for Sickle Cell Disease (RBC Survival Trial)

Phase 2

Recruiting

Led By Matthew Karafin, MD, MS

Research Sponsored by Versiti

Eligibility Criteria Checklist

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

Must have

Receiving chronic transfusions (i.e regular transfusion every 4-8 weeks).

Age 18-60 years

Must not have

Hepato- or splenomegaly

Known G6PD deficiency

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 hours post-transfusion

Awards & highlights

No Placebo-Only Group

Summary

This trial gives sickle cell patients blood from two types of donors to see if the lifespan of the transfused cells differs. It aims to find out if a specific condition in donors affects how long the blood cells last in the patient's body. This will help improve future blood transfusions for sickle cell patients.

Who is the study for?

This trial is for adults aged 18-60 with Sickle Cell Disease who are in a stable condition and regularly receive blood transfusions. It's not for those with organ enlargement, G6PD deficiency, HIV, language barriers, multiple blood transfusion allergies or antibodies, pregnant/nursing women, or other conditions that affect study participation.

What is being tested?

The study tests if red blood cells from donors without the G6PD enzyme have different lifespans when given to sickle cell patients compared to cells from donors with this deficiency. The survival of these cells post-transfusion is measured.

What are the potential side effects?

While specific side effects aren't listed for this trial, general risks may include allergic reactions to transfused blood and complications related to mismatched blood types.

Eligibility Criteria

Inclusion Criteria

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

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I regularly get blood transfusions every 4-8 weeks.

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I am between 18 and 60 years old.

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I have sickle cell disease (HbSS or HbSβ0-thalassemia).

Exclusion Criteria

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

Select...

I have an enlarged liver or spleen.

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I have been diagnosed with G6PD deficiency.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 hours post-transfusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 hours post-transfusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 hours post-transfusion for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Percentage of Red Blood Cells Surviving

Secondary study objectives

Mean Percent Change in Hemoglobin A

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: G6PD Deficient Red Blood Cell Transfusion, then Non-G6PD deficient Red Blood Cell TransfusionExperimental Treatment2 Interventions

Transfusion of a red blood cell unit that has been identified by local laboratory procedures to be deficient in G6PD enzyme activity followed after 4 months by transfusion of a red blood cell unit that has been identified by local laboratory procedures to not be deficient in G6PD enzyme activity.

Group II: Non-G6PD deficient Red Blood Cell Transfusion, then G6PD Deficient Red Blood Cell Transfusion,Active Control2 Interventions

Transfusion of a red blood cell unit that has been identified by local laboratory procedures to not be deficient in G6PD enzyme activity followed after 4 months by transfusion of a red blood cell unit that has been identified by local laboratory procedures to be deficient in G6PD enzyme activity

0 / 5Eligibility 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

The most common treatments for Sickle Cell Disease (SCD) include hydroxyurea and red blood cell (RBC) transfusions. Hydroxyurea increases fetal hemoglobin (HbF) production, which helps prevent the sickling of red blood cells and reduces the frequency of painful vaso-occlusive episodes. RBC transfusions provide normal red blood cells to improve oxygen delivery and reduce complications such as stroke and acute chest syndrome. The quality of transfused RBCs, such as those from donors with or without G6PD deficiency, can impact the effectiveness and safety of the transfusions. These treatments are vital for managing SCD and improving patient outcomes.

Magnesium for treating sickle cell disease.Hydroxyurea treatment for sickle cell disease.