Clinical Trials

his trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice.

The main goal of this study is to determine whether the treatment will lead to a complete absence of severe vaso-occlusive events (VOEs) in patients with severe SCD.

Has your child received a Fontan palliation operation? If so, they may be able to join a clinical trial that is testing whether exercise can help patients who have had this procedure. Your child must be between 8 and 19 years old, had a Fontan palliation, and can exercise. A study team member will observe the participants doing aerobic and resistance exercises live on video. The study is taking place in Palo Alto, California.

Are you an adult with sickle cell disease? This study aims to determine whether monthly exchange blood transfusions (removing blood and replacing it with transfused red blood cells) can improve health and prevent or reduce the number of pain crises. To participate in this study, you must be at least 18 years old, have sickle cell disease, and have not been on a chronic exchange transfusion program for at least 2 months. This study is located in Pittsburgh, Pennsylvania.

Are you or your child between the ages of 3 and 21 and experiencing pain episodes? This study tests a treatment called arginine therapy for pain episodes in children with sickle cell disease. Researchers are investigating how safe and effective this treatment is, as well as how arginine is metabolized in the body and its effects on cells. To participate in this study, you or your child must have sickle cell disease and pain that requires medical attention. This study takes place in multiple locations in the United States.