Vertex Pharmaceuticals Incorporated

This study tests an experimental drug called povetacicept in about 30 adults with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see if the drug can safely lower certain immune proteins (IgG) and improve symptoms. Participants will receive …

This early-stage trial tests the safety and tolerability of a new drug called VX-670 in 44 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either single or multiple doses of VX-670 or a placebo.…

This study tests a triple combination medicine (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to see how the drug moves through the body, how safe it is, and if it helps control the disease. About …

This study tests a treatment called VX-880 in 10 people who have both type 1 diabetes and a kidney transplant. The goal is to see if it can help them stop taking insulin while keeping their blood sugar under control. Participants will continue taking lifelong anti-rejection drugs…

This study tests a single dose of a CRISPR-based gene therapy (CTX001) in 26 adults with transfusion-dependent beta-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, which are edited to produce more fetal hemoglobin, potentially red…

This study tests an experimental drug called VX-407 in 24 adults with autosomal dominant polycystic kidney disease (ADPKD) who have certain changes in the PKD1 gene. The goal is to see if the drug can slow or reduce kidney growth and check for side effects. Participants will take…

This study tests whether a new drug, povetacicept, works better than a standard treatment (calcineurin inhibitor) for people with primary membranous nephropathy, a kidney disease. About 176 adults will take part. The goal is to see if the new drug can help more patients achieve c…

This study tests an experimental drug called VX-147 in people with a specific genetic form of kidney disease caused by the APOL1 gene. The goal is to see if the drug can reduce protein in the urine and protect kidney function. About 466 adults and children will take either VX-147…

This study tests a new stem cell treatment called VX-880 for people with type 1 diabetes who have dangerous low blood sugar episodes. The goal is to see if a single infusion can help the body make its own insulin again, reducing or eliminating the need for insulin shots. About 52…

This early-stage study tests a new experimental drug, VX-828, in healthy adults and people with cystic fibrosis (CF). The main goal is to check if the drug is safe and how the body processes it. Researchers will also test VX-828 together with other CF medicines. About 255 partici…

This study tests a new medicine called suzetrigine for people with diabetes who have long-term nerve pain in their legs. About 734 adults will take either the drug or a placebo for 12 weeks to see if it reduces daily pain. The goal is to find a safe, non-opioid option for this co…

This study is testing a new pain treatment for people having total knee replacement surgery. The goal is to see if it can help patients use fewer opioids after the operation. About 60 adults will take part. The study is currently recruiting.

This study tests an oral medication called VX-993 for people with diabetic peripheral neuropathy, a common complication of diabetes that causes pain in the legs and feet. About 300 adults with type 1 or type 2 diabetes will receive different doses of VX-993 or an active comparato…

This study tests a new medicine called suzetrigine for people with nerve pain from diabetes. About 1,100 adults will receive either the study drug, a placebo, or an active comparator to see if it safely reduces pain. The main goal is to measure pain relief after 12 weeks.

This early-stage study tests whether VX-272 is safe and how the body processes it. About 128 healthy adults will receive either the drug or a placebo. The goal is to gather safety data before testing in people with cystic fibrosis.

This study looks at how a pain medication called suzetrigine gets into breast milk. Twelve healthy breastfeeding women will take one dose, then pump and test their milk. The goal is to find out how much drug the baby might get and whether it is safe.

This early-stage study is testing a new medicine called VX-581 in 128 healthy volunteers to see if it is safe and how the body processes it. Participants will receive either the drug or a placebo, and researchers will monitor for side effects and measure drug levels in the blood.…