Vertex Pharmaceuticals Incorporated

This study tests an investigational drug called povetacicept in 30 adults with generalized myasthenia gravis, a condition causing muscle weakness. The goal is to see if the drug safely lowers certain immune proteins (IgG) linked to the disease. Participants receive either the dru…

This study tests a single dose of a CRISPR-modified stem cell therapy (CTX001) in 26 people with transfusion-dependent β-thalassemia or severe sickle cell disease. The treatment uses the patient's own blood stem cells, edited to produce fetal hemoglobin, aiming to reduce or elimi…

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 the growth of kidney cysts and check for side effects. Participants will …

This early-stage study tests a new investigational drug called VX-828, alone or combined with other CF medicines, in healthy adults and people with cystic fibrosis. The main goal is to check safety and how the drug moves through the body. About 255 participants will take part to …

This study tests a triple combination medicine (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific gene change. The goal is to see if the drug is safe and how it works in young bodies. About 210 kids will take part, and the study is…

This study tests a treatment called VX-880 for people with type 1 diabetes who have severe low blood sugar episodes. The therapy uses special cells that can make insulin, aiming to reduce or eliminate the need for insulin injections. About 52 adults will receive the treatment and…

This study tests a new drug, povetacicept, against a standard treatment for primary membranous nephropathy, a kidney disease that can lead to kidney failure. About 176 adults will take part to see if the new drug can put the disease into remission more safely and effectively. The…

This study tests a new drug called VX-522 in adults aged 18 and older with cystic fibrosis whose genetic mutations do not respond to current CFTR modulator therapies. The goal is to see if the drug is safe and can improve lung function. About 39 participants will receive the drug…

This study tests an experimental drug called VX-147 in people with a type of kidney disease caused by a specific gene change (APOL1). 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 phase 3 study tests VX-880, a cell therapy, in 10 adults with type 1 diabetes who have already received a kidney transplant. The main goal is to see if participants can stop taking insulin while staying on lifelong anti-rejection drugs. The study will measure insulin indepen…

This early-stage trial tests the safety and tolerability of a new drug, VX-670, in 44 adults with myotonic dystrophy type 1 (DM1). Participants receive either the drug or a placebo in single or multiple doses. The study aims to find the right dose and understand how the drug move…

This study tests a new medicine called suzetrigine for people with diabetic peripheral neuropathy, a type of nerve pain in the legs and feet caused by diabetes. About 734 adults with diabetes and long-term nerve pain will receive either the drug or a placebo for 12 weeks. The goa…

This study tests a new medicine called suzetrigine for people with painful nerve damage 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 over 12 weeks. The goal is to find a new option for ma…

This study is testing a new pain treatment for people having total knee replacement surgery. The goal is to see if it can help them avoid or use fewer opioid painkillers after the operation. The study will involve 60 adults who are scheduled for knee replacement and will compare …

This study tests an experimental oral medication called VX-993 for people with painful nerve damage from diabetes (diabetic peripheral neuropathy). About 300 adults will receive either VX-993, an active comparator, or placebo to see if it safely reduces daily pain over 12 weeks. …

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

This study looks at how much of the pain medicine suzetrigine and its breakdown product enter breast milk. Twelve healthy women who are breastfeeding will take a single dose and provide milk samples. The goal is to estimate the dose a nursing baby might receive and check for any …

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 drug is being developed for cystic fibrosis, but this trial does not involve people with the disease. The goal is to gather safety informat…