Vertex Pharmaceuticals Incorporated

This study tests a treatment called VX-880 for people with type 1 diabetes who have dangerous low blood sugar episodes. The therapy uses donor cells that can produce insulin, aiming to let participants stop taking insulin shots and avoid severe hypoglycemia. About 52 adults with …

This study tests a new drug, povetacicept, against a standard treatment (calcineurin inhibitor) in 176 adults with primary membranous nephropathy, a kidney disease that can lead to kidney failure. The goal is to see if the new drug can put the disease into remission more safely a…

This study tests an experimental drug called povetacicept in adults with generalized myasthenia gravis, a condition that causes muscle weakness. The goal is to see if the drug safely reduces harmful immune proteins. About 30 participants will receive either the drug or a placebo,…

This study tests an experimental drug called VX-147 in adults and children 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 and protect kidney function. About 466 participants will receive either VX-147…

This study tests an experimental drug called VX-407 in 24 adults with autosomal dominant polycystic kidney disease (ADPKD) who have specific changes in the PKD1 gene. The goal is to see if the drug can slow kidney growth and is safe. Participants take VX-407 tablets by mouth and …

This early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monito…

This early-stage study tests a new drug called VX-433 in 118 people with narcolepsy type 1. The main goal is to check if it is safe and how the body processes it. Researchers will also see how VX-433 interacts with other drugs like midazolam and bupropion. This is a first step to…

This phase 3 study tests VX-880, a stem-cell therapy infused into the liver, in 10 people with type 1 diabetes who have already received a kidney transplant. The main goal is to see if participants can stop using insulin entirely. The therapy is given alongside standard transplan…

This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or s…

This study tests a new medicine called suzetrigine for people with nerve pain from diabetes. About 1,100 adults with diabetic peripheral neuropathy will receive either suzetrigine, a placebo, or an active comparator to see if it safely reduces pain over 12 weeks. The goal is to f…

This Phase 2 trial tests an experimental oral drug called VX-993 for pain caused by diabetic peripheral neuropathy, a common complication of diabetes. About 300 adults with type 1 or type 2 diabetes and nerve pain in both legs for at least a year will receive VX-993, pregabalin (…

This phase 3 trial tests suzetrigine, an experimental oral drug, for pain caused by diabetic peripheral neuropathy. About 734 adults with diabetes and nerve pain in their legs for at least a year will receive either the drug or a placebo. The main goal is to see if suzetrigine re…

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, and the study will track how many stay completely opioid-free.

This early-stage study is testing a new drug called VX-581 in 128 healthy volunteers to see if it is safe and how the body processes it. The study is double-blind and placebo-controlled, meaning some participants get the drug and some get a dummy pill. This is a first step toward…

This early-stage study is testing how much of the pain medication suzetrigine (JOURNAVX) gets into breast milk after a single dose. Twelve healthy breastfeeding women will provide milk samples to measure the drug and its breakdown product. The goal is to estimate how much a nursi…

This Phase 1 trial tests the safety and how the body handles VX-272, an experimental drug for cystic fibrosis. About 128 healthy volunteers will receive single or multiple doses of the drug or a placebo. The study focuses on side effects and drug levels in the blood, not on treat…