About this study
Sickle cell disease is a serious blood illness. It can cause pain, damage to organs, and other health problems. The only cure right now is a bone marrow transplant. Most children do not have a matching donor. New treatments like gene therapy are being studied. These treatments often use genetic testing. Genetic testing looks for changes in a person’s DNA.
Researchers do not know what parents and teens with sickle cell disease think about genetic testing or gene therapy. Some families may not understand these studies. Some may feel worried about them. Because of this, families may choose not to take part. This can slow research and make it harder to find better treatments.
This study will ask parents and teens to share their thoughts about genetic testing and research. The goal is to learn what families know and what worries they have. Researchers will also learn how much families trust doctors and researchers. They want to know how easy it is for families to understand medical information. This will help researchers make studies that are clear and helpful for families.
Parents and teens will help test learning tools, such as a website and a storybook. These tools explain treatment choices for sickle cell disease, including gene therapy. Researchers will also talk with families from different parts of the country to learn what families need to know before joining a gene therapy study.
What families share through this study will help researchers make better learning tools. It will also help them design studies that are easier to understand. This can help families make informed choices about future treatments.
Eligibility overview
- Parent of a child (1–18 years old) with sickle cell disease
- Patient age 13–18 with sickle cell disease
- Participant has been previously approached for the SCCRIP study (for survey/interview group)
- Speaks English