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 an investigational drug called VX-147 for people with kidney disease caused by specific changes in the APOL1 gene. It aims to see if the drug can reduce protein loss in urine and slow the decline of kidney function over 48 weeks. The study will enroll about …

This study is testing a new three-drug combination (VX-121/tezacaftor/deutivacaftor) for children aged 1 to 11 with cystic fibrosis who have specific genetic mutations. The main goals are to see how the drugs move through the body, check for side effects, and measure if the treat…

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

This early-stage study is testing a new drug called VX-522 for adults with cystic fibrosis who have genetic mutations that don't respond to current treatments. Researchers will check if the drug is safe and if it helps improve lung function. The study involves about 39 participan…

This clinical trial is testing VX-880, an investigational cell therapy, for adults with Type 1 diabetes who experience severe and dangerous low blood sugar episodes. The study aims to see if the treatment can help patients become independent from insulin injections while eliminat…

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 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 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 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 diabetic nerve damage (neuropathy). About 300 adults with long-term pain in their feet and legs from diabetes will take the drug or a comparison medication for 12 weeks. The main go…

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 …