Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

This study seeks to understand the safety of a gene editing therapy called CRISPR_SCD001 and if this treatment can help people with severe sickle cell disease get better.

Locations

2 United States of America sites

Age

>

12-35 years

Years

Genotypes

SCD type SS

Phase

1/2

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RISE UP: Studying whether an investigational drug can increase hemoglobin and reduce pain crises

The RISE UP Study seeks to determine if an investigational molecule called “mitapivat” has the potential to improve the health of red blood cells in individuals living with sickle cell disease by improving the energy and reducing the sickling of red blood cells.

Locations

13 USA sites

6 France sites

5 Belgium sites

4 UK sites

3 Canada sites

2 Netherlands sites

2 Germany sites

1 Italy site

1 Oman site

1 Kenya site

Age

>

>16 years

Years

Genotypes

SCD type SS, SCD type SC, SCD type Sβ0 thalassemia, SCD type Sβ+ thalassemia

Phase

3

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Using Community Based Research Practices to Advance Anti-Racism in Sickle Cell Disease Clinical Care

This study is being conducted by Sickle Cell 101, UCSF Benioff Children's Hospitals, and BCH Diversity, Equity, Inclusion and Anti-Racism Council to address racism in SCD care.

Locations

Global & Remote

Age

>

>18 years

Years

Genotypes

All types of SCD

Phase

N/A

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Attributional Style and Adherence to Hydroxyurea in Adults with Sickle Cell Disease (HU Adherence Study)

This study, conducted by Lance Archer, a doctoral student in the School of Psychology at Fielding Graduate University, Santa Barbara, CA, aims to understand why some adults with sickle cell disease (SCD) may have difficulties taking Hydroxyurea medication.

Locations

United States and Canada

Age

18 – 65 years

- 65 Years

Genotypes

All types of SCD

Phase

N/A

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Exercise Collapse Associated with Sickle Cell Trait (ECAST)

This study aims to understand why some SCT carriers are at higher risk for Exercise Collapse Associated with Sickle Cell Trait (ECAST).

Locations

US participation only

Age

18 – 45 years

- 45 Years

Genotypes

Hb AS - sickle cell trait

Phase

N/A

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KNOWledge and Understanding of Sickle Cell Trait (KNOWUrSCT): A Global Health Disparity Research Study

This study is being conducted by Sickle Cell 101 to understand our sickle cell and global community’s thoughts on sickle cell trait, genotype awareness, and educational gaps associated with the evolving research about potential complications of sickle cell trait.

Locations

Global & Remote

Age

>

>18 years

Years

Genotypes

Open to everyone

Phase

N/A

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ASCENT1 Study

Hemoglobin levels in people living with sickle cell disease are typically lower than the average person.

Locations

11 US sites

2 Canada sites

Age

>

18

Years

Genotypes

SCD type SS, SCD type SC, SCD type Sβ0 thalassemia, SCD type Sβ+ thalassemia

Phase

2

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An Electronic Patient Reported Pain Assessment in Sickle Cell

“The Electronic Patient Reported Pain Assessment in Sickle Cell” study aims to gather insights into painful crises in people living with sickle cell disease.

Locations

1 USA site

Age

>

18

Years

Genotypes

SCD type SS, SCD type Sβ0 thalassemia

Phase

N/A

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Hibiscus Study: An oral investigational medication for SCD

The Hibiscus Study is enrolling adults and adolescents 12 to 65 years old with a clinical diagnosis of SCD.

Age

12

- 65 Years

Genotypes

All types of SCD

Phase

2/3

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