Vertex Pharmaceuticals Incorporated

This study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…

This study is testing a new three-drug combination therapy for children aged 1 to 11 years old who have cystic fibrosis. The main goals are to see how the drugs move through the children's bodies, check if the treatment is safe, and look for early signs that it might help control…

This clinical trial is testing whether an investigational drug called VX-147 can help slow kidney damage in people who have a specific genetic risk (APOL1 mutations) that causes protein to leak into their urine. The study will enroll 466 adults and children to compare VX-147 agai…

This study is testing a new drug called povetacicept for people with primary membranous nephropathy, a serious autoimmune disease that damages the kidneys. Researchers will compare it to a standard treatment to see if it's better at putting the disease into remission. The goal is…

This study is testing an investigational cell therapy called VX-880 in people who have type 1 diabetes and have already received a kidney transplant. The main goal is to see if the treatment can help participants stop needing insulin injections. Participants must already be on st…

This early-stage study is testing a new drug called VX-522 in adults with cystic fibrosis (CF) who have genetic mutations that don't respond to current CFTR modulator therapies. The main goals are to check if the drug is safe and tolerable, and to see if it improves lung function…

This study is testing an investigational drug called Inaxaplin for people with a specific genetic form of kidney disease (APOL1-mediated). The main goal is to see if the drug safely reduces the amount of protein leaking into the urine, which is a key sign of kidney damage. The tr…

This clinical trial is testing an experimental cell therapy called VX-880 for adults with Type 1 diabetes who experience dangerous low blood sugar episodes. The treatment involves infusing specially engineered cells that may help the body produce its own insulin again. Researcher…

This early-stage study aims to check the safety and how the body processes a new drug called VX-828. It will test VX-828 alone and in combinations with other CF medications in 255 healthy volunteers and people with cystic fibrosis. The main goal is to see if the drug is safe and …

This study is testing an investigational drug called VX-407 in people with a specific genetic type of autosomal dominant polycystic kidney disease (ADPKD). The main goal is to see if the drug can slow the growth of kidney cysts, which is a key driver of the disease. Researchers w…

This early-stage study is testing a new drug called VX-670 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. Researchers will give different doses to 44 participants to check if the drug is safe and how it behaves in the body. The study will also measure…

This study is testing a new oral medication, VX-993, to see if it can safely reduce pain caused by nerve damage from diabetes. It will involve about 300 adults who have had this type of pain in their feet or legs for at least a year. Researchers will compare different doses of th…

This study is testing if an investigational drug called Suzetrigine can safely reduce pain caused by nerve damage from diabetes. About 734 adults with long-term diabetic foot and leg pain will receive either the drug or a placebo for 12 weeks. The main goal is to see if Suzetrigi…

This study aims to see if a new drug called Suzetrigine can safely reduce pain in people with diabetic peripheral neuropathy, a common and painful nerve condition. It will involve about 1100 adults with type 1 or type 2 diabetes who have significant daily pain in their feet or le…

This is a very early study to check if a new drug called VX-581 is safe and how it moves through the body. It involves 128 healthy volunteers who will receive either the drug or a placebo. The main goal is to understand the drug's safety profile and how the body processes it befo…

This study aims to measure how much of an experimental pain medication called suzetrigine gets into breast milk. It will involve 12 healthy women who are currently breastfeeding. The goal is to understand how much of the drug a nursing infant might be exposed to and to check for …