Vertex Pharmaceuticals Incorporated

This was a Phase 3 trial designed to see if a single dose of a gene therapy called exa-cel could cure severe sickle cell disease in adolescents and adults. The treatment aimed to permanently modify a patient's own blood stem cells to stop the disease. The study was withdrawn befo…

This study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…

This study follows 297 cystic fibrosis patients to see how safe and effective a triple-combination drug remains over the long term. The treatment targets specific genetic mutations beyond the most common one. Participants had previously taken the medication in earlier studies and…

This study is testing the long-term safety and effectiveness of a three-drug combination therapy for people with cystic fibrosis aged 1 year and older. It follows participants who have already taken the medication in a previous trial to see how they do over a longer period. The m…

This study is testing the long-term safety and effectiveness of a three-drug combination therapy (VX-121/TEZ/D-IVA) for people with cystic fibrosis. It follows 822 participants who previously completed earlier studies of this treatment. The main goals are to monitor side effects …

This study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…

This study aims to learn about the long-term safety and effects of the drug VX-670 in adults with myotonic dystrophy type 1 (DM1). It is an extension study for 44 people who have already completed a previous trial with this drug. The main goal is to monitor participants for side …

This study is testing a one-time treatment called CTX001 in children with severe sickle cell disease who don't respond well to the standard medication, hydroxyurea. The treatment uses the patient's own modified stem cells (a type of gene therapy using CRISPR technology) to try to…

This study is checking the long-term safety and effectiveness of a three-drug combination treatment (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis. It involves about 50 people aged 12 months and older who have already taken the drug in a previous trial. The main goal is t…

This study is testing the long-term safety and effectiveness of an experimental drug called suzetrigine for people with painful nerve damage caused by diabetes. The trial includes 455 participants who previously completed a related 12-week study. Researchers will monitor side eff…

This study aims to learn more about how different versions of the PKD1 gene affect people with autosomal dominant polycystic kidney disease (ADPKD). Researchers are observing about 400 people with ADPKD to see how common different gene variants are and what health characteristics…

This early-stage study aims to understand how a new triple-drug combination for cystic fibrosis (VNZ/TEZ/D-IVA) affects the body's processing of a common cholesterol drug called rosuvastatin. It involves 18 healthy volunteers to check for any safety issues when these medications …

This early-stage study aims to see if an experimental drug for a genetic kidney disease (VX-407) changes how the body processes common birth control pills. Researchers are testing this in 74 healthy volunteers to check for safety and measure drug levels in the blood. The goal is …

This early-stage study is testing the safety of an experimental drug called VX-407 in healthy volunteers. Researchers want to understand how the body processes the medication and what side effects might occur. The results will help determine if VX-407 is safe enough to test in pe…