The Cure Sickle Cell Initiative (CureSCi) is pleased to announce the launch of the Gene Therapy to Reduce All Sickle Pain (GRASP) Trial, part of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). This Phase 2, multi-center trial is designed to test whether the gene therapy approach (lentiviral vector shmiR), which decreases expression of the BCL11A gene, can improve or eliminate sickle cell pain episodes.
Trial participants will include people living with sickle cell disease ages 13 to 40 years old who have had four or more pain episodes or acute chest syndrome requiring hospitalizations or emergency room visits in the past two years.
The phase 2 trial is co-funded by the National Heart, Lung, and Blood Institute (NHLBI) and the California Institute for Regenerative Medicine (CIRM) and is based on a pilot/phase 1 study which showed early safety and efficacy in the initial patients enrolled in the trial. The BMT CTN infrastructure is being utilized to enroll at 10 sites across the United States.
For more information about the trial, including inclusion/exclusion criteria and trial locations, please visit the CureSCi GRASP trial page.
