SICKLE CELL DISEASE

This early-stage study is testing a one-time gene therapy injection called KL003 for people with severe sickle cell disease. The treatment involves collecting a patient's own blood stem cells, modifying them in a lab to produce healthy hemoglobin, and then returning them to the p…

This study is testing if adding a drug called ruxolitinib to a stem cell transplant can help prevent the body from rejecting the new cells, which is a major risk. It aims to improve the chance of a cure for children and young adults with severe sickle cell disease. Up to 24 parti…

This study is testing if adding a new drug called SIL-8301 (senicapoc) to standard sickle cell treatment can improve anemia and reduce disease complications. It will involve about 105 young adults (16-35) with a specific type of sickle cell disease who have low blood counts despi…

This study is testing if a high dose of a drug called plerixafor can safely and effectively collect enough stem cells from people with sickle cell disease. These collected cells are a necessary first step for future gene therapy treatments, which aim to correct the disease at its…

This study is testing whether an oral drug called mitapivat can help protect the kidneys of people with sickle cell disease. It will enroll about 40 participants who already show signs of early kidney damage. Researchers will measure protein levels in urine over 6 months to see i…

This study aims to understand the long-term safety of a daily medication called pociredir for adults with sickle cell disease. It will follow 50 participants who previously benefited from the drug in an earlier study, monitoring them for up to 4 years. The main goal is to see if …

This study is testing a new program called SickleFit, which combines personalized exercise with nutrition counseling for adults aged 35 and older who have sickle cell disease. The main goal is to see if the program is safe, practical, and acceptable to participants. Researchers w…

This study is testing whether a drug called iloprost can help adults with sickle cell disease during painful vaso-occlusive crises. Researchers will give 144 hospitalized patients either iloprost or a placebo through an IV to see if it reduces their pain and how much opioid pain …

This study aims to see if giving a single dose of ketamine within the first hour of a sickle cell pain crisis can help patients avoid being admitted to the hospital. It will also check if starting ketamine early during a hospital stay reduces the need for strong opioid painkiller…

This study aims to learn if a non-invasive light therapy, aimed at the forehead, can improve thinking skills and oxygen use in the brains of children with sickle cell disease. Researchers will enroll 60 children, aged 8-17, who will be randomly assigned to receive either the real…

This study aims to understand how long specially treated, pathogen-reduced red blood cells last in the body of adults with sickle cell disease. It will follow five patients who regularly receive blood exchange therapy, tracking the treated cells with special markers. The goal is …

This study aims to understand how often children with sickle cell disease experience painful episodes called vaso-occlusive crises and what complications result from them. Researchers will look back at the medical records of 25 children, aged 1 to 18, who have been hospitalized f…