Explore sickle cell clinical trials and research studies happening near you and across the globe. Stay informed on the latest opportunities for participation.
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PRESENCE Study
PRESENCE is a national study testing a digital CBT program to help 16 to 30-year-olds with sickle cell manage chronic pain, stress, and the emotional toll it can take.
16 to 30 years old
Survey
Yes
All types of SCD
United States
The Pioneer Study
This study explores the potential benefits of a daily oral medicine for sickle cell disease called Pociredir.
18 to 65 years
Clinical
Yes
1
SCD type SS
SCD type SC
SCD type Sβ0 thalassemia
....
United States
South Africa
Nigeria
...
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
The goal of this study, conducted by UCSF and UCLA, is to determine if a potential gene therapy, called CRISPR_SCD001, is safe for and improves the health of individuals with SCD.
12-35 years
Clinical
Yes
1/2
SCD type SS
United States of America
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.
>18 years
Survey
Yes
N/A
All types of SCD
Global & Remote
KNOWledge and Understanding of Sickle Cell Trait (KNOWUrSCT): A Global Health Disparity Research Study
This study aims to explore perceptions of sickle cell trait (SCT), SCT testing, understand related complications/experiences, and develop community-driven resources for SCT.
>18 years
Survey
Yes
N/A
Open to everyone
Global & Remote
ASCENT1 Study
A research study investigating how well NDec works in people with sickle cell disease.
18
Clinical
Yes
2
SCD type SS
SCD type SC
SCD type Sβ0 thalassemia
....
US
Canada
31 Mar 2026
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that it will pursue U.S. accelerated approval for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease, following completion of its pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration (FDA).
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23 Feb 2026
The new path aims to remove barriers for bespoke medicines designed for patients with rare diseases, potentially allowing data in a few patients to support approvals in broader populations by targeting specific genetic, cellular or molecular abnormalities, according to the FDA release.
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24 Dec 2025
On December 23, 2025, the U.S. Food and Drug Administration (FDA) approved AQVESME (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
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