At the Initiative, we believe clinical trial participation is one of the most important ways we can move towards a future without sickle cell disease.
About Clinical Trials
Clinical trials are medical studies that help find safe and effective ways to prevent, detect, or treat diseases. The Cure Sickle Cell Initiative (CureSCi) is working with researchers across the country to move promising genetic therapies into clinical trials.
CureSCi clinical trials focus first on the safety of the new treatment (Phase 1). In a later stage clinical trial (Phase 2), the same treatment is tested to see if it can cure or reduce the severity of sickle cell disease. Each phase of the clinical trial will answer important questions about safety and effectiveness.
Ways You Can Help
One of the most significant ways to support the Cure Sickle Cell Initiative is to learn more about the importance of clinical trials and to make an informed decision about participating. How to overcome barriers to progress, which includes exploring beliefs about clinical trials participation, is an important conversation in the sickle cell disease community.
CureSCi Supported Clinical Trials
Although genetic therapies are a promising option for many rare diseases, the technique is still being studied to make sure that it is safe and effective. The National Heart, Lung, and Blood Institute, part of the National Institutes of Health, is partnering with researchers through CureSCi to design and fund genetic therapy clinical trials. These trials are investigational, and treatments are not currently approved by the U.S. Food and Drug Administration (FDA).
CURRENT CLINICAL TRIALS
Trial phase 2
Trial Phase 12
TRIAL PHASE 1/2
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD-001) in Patients with Severe Sickle Cell Disease