Formal Trial Name

A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)

Status

Active, not recruiting

Trial Funding

This study is funded by the National Institutes for Health (NIH), National Heart, Lung, and Blood Institute (NHLBI) and California Institute for Regenerative Medicine (CIRM).

Trial Purpose

The main goal of the clinical trial is to see if the study treatment (gene therapy) can completely prevent severe pain episodes caused by sickle cell disease between 6 and 24 months post-infusion of the gene therapy. Patients in the study will be closely followed for two years after receiving the gene therapy to monitor their health and see how well the treatment works.

Trial Background

This study is testing a new gene therapy treatment for people with severe sickle cell disease, ages 13 to 40, across the United States. The treatment uses the patient’s own blood stem cells, which are collected and then treated in the lab to include a specific gene designed to help the body make more fetal hemoglobin, which can prevent the red blood cells from becoming sickle-shaped and causing pain crises. Before getting the treatment, patients receive blood transfusions for at least three months to help prepare their bodies for the collection of their stem cells. After the stem cells are treated in the lab and are ready, patients go through a standard preparation process, including strong medicine to make room in their bone marrow for the new cells. The treated stem cells are then given back to the patient through an intravenous injection.

At this time, the recruitment of patients for the study has been completed. All patients have been treated, and are being followed for a period of two years to determine the safety and efficacy of the gene therapy.