Vertex Pharmaceuticals Incorporated

This early-stage trial tests VX-264, a cell therapy designed to replace the insulin-making cells destroyed in type 1 diabetes. Seven adults with long-standing diabetes will be monitored for safety and for signs that their bodies can produce insulin again. The goal is to see if th…

This study tests an experimental drug called inaxaplin in 42 people with a genetic form of kidney disease (APOL1-mediated). The goal is to see if the drug can reduce protein in the urine, a sign of kidney damage. Participants take the drug and are monitored for side effects and k…

This study looks at the long-term safety and effects of the drug VX-670 in 44 adults with myotonic dystrophy type 1 (DM1). All participants must have already taken VX-670 in an earlier study. Researchers will monitor side effects and measure drug levels in the blood. This is an o…

This study tests the long-term safety and effectiveness of a triple-combination drug (VX-121/TEZ/D-IVA) in people with cystic fibrosis. About 822 participants who completed earlier studies will take the medication and be monitored for side effects, lung function, and other health…

This study tests a new drug called VX-522 in adults with cystic fibrosis whose genetic mutations do not respond to existing CFTR modulator therapies. The main goals are to check if the drug is safe and tolerable, and to see if it improves lung function. About 26 participants aged…

This study looks at the long-term safety and effectiveness of a triple combination drug (vanzacaftor/tezacaftor/deutivacaftor) for people with cystic fibrosis aged 1 year and older. Participants who completed a previous study are invited to join. The goal is to see if the drug is…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…

This study aimed to test a single dose of exa-cel (CTX001) in adolescents and adults with a severe form of sickle cell disease called HbSC. The treatment was designed to increase fetal hemoglobin to reduce pain crises and other complications. However, the study was withdrawn befo…

This study tests a single dose of CTX001, a gene therapy made from the patient's own blood stem cells edited with CRISPR, in 16 children with transfusion-dependent beta-thalassemia. The goal is to see if it can help them produce enough healthy red blood cells to stop needing regu…

This study tests a one-time gene-editing treatment (CTX001) in children with severe sickle cell disease who cannot take hydroxyurea. The treatment uses the child's own blood stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The goal is to prevent severe p…

This study looks at the long-term safety and how well the body handles a drug combination (elexacaftor/tezacaftor/ivacaftor, also known as Trikafta) in people with cystic fibrosis who are 12 months old or older. About 50 participants who completed a previous study will continue t…

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in about 300 people with cystic fibrosis who have certain gene changes. Participants take the medication and are monitored for side effects, lung functio…

This study tests the long-term safety and effectiveness of an experimental drug called suzetrigine for people with pain caused by diabetic peripheral neuropathy. About 455 adults who completed a previous related study will take the drug and be monitored for side effects and pain …

This study is for people with autosomal dominant polycystic kidney disease (ADPKD), a condition that causes cysts to grow in the kidneys. Researchers want to learn how different gene variants affect the disease. About 400 participants will provide health information and samples. …