About This Study
The purpose of this study is to learn more about using the study drug, CTX001, to treat sickle cell disease in children. We want to see how effective it is and if there are any side effects.
What Activities Are a Part of this Study?
- Informed Consent Review
- Use of Study Treatments or Placebo
- After Study Follow-Up Activities
- Review of Medical History
- Physical Examinations
- Study Visits and Consultations
- Surveys/Questionnaires
- Laboratory and Imaging Tests
- Specimen Sample Collection
Age & Gender
- 2 - 11 years old
- All Genders
Language
- This study enrolls English and Spanish speakers
Additional Study Information
- Compensation Offered
- Reimbursement offered
Study Locations
- Greater Charlotte, NC