Vertex Pharmaceuticals Incorporated

This study tests a single dose of a gene-edited stem cell treatment (CTX001) in 13 children with severe sickle cell disease who cannot take or did not benefit from hydroxyurea. The goal is to see if the treatment can prevent severe pain crises and hospital stays for at least a ye…

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain gene mutations not previously studied. About 300 participants who completed an earlier study will take th…

This study looks at the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (known as Trikafta) in people with cystic fibrosis who are at least 12 months old. Participants must have completed a previous related study. The main goal is to tr…

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 will continue taking the medication to monitor side ef…

This early-stage trial tests VX-264, a cell therapy designed to help people with type 1 diabetes who have had the condition for at least 5 years. The main goals are to check safety and see if the treatment can improve the body's own insulin production. About 7 participants will b…

This study tests a one-time gene-editing treatment (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder. The therapy uses the child's own stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The main goal is to see if …

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

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…

This study looks at the long-term safety and effects of a drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). It is for people who already completed a previous study of the same drug. The main goal is to check for side effects over time.

This study tests an experimental drug called inaxaplin in 42 people with a genetic form of kidney disease linked to the APOL1 gene. The goal is to see if the drug can reduce protein in the urine, a sign of kidney damage, and slow disease progression. Participants must have a spec…

This study aimed to test a single dose of exa-cel, a gene therapy, in people with severe sickle cell disease who have the HbSC genotype. The goal was to see if it could safely raise fetal hemoglobin levels to reduce complications. However, the study was withdrawn before enrolling…

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 a genetic kidney disease called ADPKD. Researchers want to learn how different gene changes (variants) affect the disease. About 400 participants will provide health information and samples. The goal is to better understand the disease, not to test a…