Sickle Cell Disease Clinical Trials

Study goal:

The main goal of this study is to see if a single dose of exa-cel allows your child’s body to increase the amount of hemoglobin F while reducing the painful effects of sickle cell disease.

Age:

2 to 11 years old

Study goal:

The purpose of this research study is to collect data on sickle cell disease participants from birth to end of life. With these data, researchers will better understand problems caused by sickle cell disease both in children and in adults. 

Study goal:

The main goal of this study is to find out what a successful health care transition looks like and to look at connections between health care transition and factors such as hospitalizations, patients’ disease knowledge and general adjustment. Researchers also want to know how these factors change over time.

Age:

16 to 20 years old

Study goal:

The main goal of this study is to see if a single dose of BEAM-101 allows your body to increase the amount of hemoglobin F while reducing the painful effects of sickle cell disease.

Age:

18 to 35 years old

Study goal:

The main goal of this clinical trial is to find out how well virtual reality therapy lessens acute pain in sickle cell patients.

Age:

Between 6 and 25 years old

Study goal:

The main goal of this trial is to collect DNA from individuals with non-cancerous blood diseases and their family members. Researchers will use the DNA to study how genes cause and influence these diseases. All research data will be confidential.

Study goal:

This study will help us find out how to make the intervention program better and test it with a larger group of caregivers and their children.

Age:

4–6

Study goal:

To find out if a single dose of genetically modified cells is safe and effective to reduce the symptoms of sickle cell disease.

Age:

18–25 years

Study goal:

To see if motixafortide is safe and helps increase collection of stem cells in adults with sickle cell disease. Learn more about the study.

Age:

Age 18 and older

Study goal:

To find out how gene therapy and bone marrow transplant affect the brain function of patients with sickle cell disease

Age:

8–25 years old

Study goal:

To study the safety and use of red blood cells prepared with the INTERCEPT® Blood System for Red Blood Cells as compared with red blood cells that are not prepared with this system.  

Age:

Ages 4 and up