Sickle Cell Program

Clinical Trials for Sickle Cell Disease

Dana-Farber/Children's Hospital Cancer Center (DFCHCC) is one of the top pediatric research centers in the world, and our scientific advances are propelled by our unique clinical trials. Learn about two of the open trials for Sickle Cell Disease that are currently accepting new patients at DFCHCC.

Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease

Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) then people without SCD. This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions.

Sickle-shaped cells are a problem because they often get stuck in blood vessels blocking the flow of blood, and cause inflammation and injury to the important areas in the body. Lexiscan is drug that may prevent this inflammation and injury caused by the sickle shaped cells.

This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making it beat faster. Lexiscan has never been studied in patients with SCD and has never been given as a long infusion.

Evaluating the Safety and Effectiveness of Stem Cell Transplants From Unrelated Donors in Children With Sickle Cell Disease (BMT CTN #0601) (The SCURT Study)

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant is a treatment option for someone with a severe form of the disease.

Prior to undergoing a transplant, people typically receive a conditioning regimen of high doses of chemotherapy and other medications to prepare the body to accept the transplant. A conditioning regimen that uses lower doses of chemotherapy and medications may be safer for transplant recipients.

This study will evaluate the safety and effectiveness of blood stem cell transplants, using either bone marrow or umbilical cord blood from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant.

Learn about other clinical trials for pediatric cancers and blood disorders at DF/CHCC.