Ensayos clínicos para la enfermedad de células falciformes

Meta de estudio:

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. 

Meta de estudio:

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.

Edad:

16 to 20 years old

Meta de estudio:

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

Edad:

Between 6 and 25 years old

Meta de estudio:

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.

Meta de estudio:

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.

Edad:

3.5–6.5

Meta de estudio:

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

Edad:

18–25 years

Meta de estudio:

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

Edad:

Age 18 and older

Meta de estudio:

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

Edad:

8–25 years old

Meta de estudio:

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.  

Edad:

Ages 4 and up

Meta de estudio:

To study the long-term health of patients with hemoglobin disorders who receive either a stem cell transplant or gene therapy product.

Edad:

5 years and older

Meta de estudio:

To better understand why some people with sickle cell disease still have serious complications even with high hemoglobin F (HbF) levels.

Edad:

Older than 5 years

Meta de estudio:

To measure coronary flow reserve using PET imaging in people with sickle cell anemia and in healthy people.

Edad:

18–21 years