Vertex Pharmaceuticals Incorporated

This study looked at the long-term safety of a triple combination therapy (VX-445, tezacaftor, and ivacaftor) in 507 people with cystic fibrosis who have the F508del mutation. Participants had already completed a previous study and continued treatment to monitor side effects and …

This study looked at the long-term safety and effectiveness of the triple-combination drug Trikafta (elexacaftor/tezacaftor/ivacaftor) in 71 children aged 2 and older with cystic fibrosis. All participants had already taken the drug in an earlier study. The main goal was to track…

This study tested a combination of two drugs, VX-661 and ivacaftor, in 40 adults with cystic fibrosis who have the most common genetic mutation (F508del). The goal was to see if the treatment is safe and improves lung function over 12 weeks. Participants were randomly assigned to…

This study tested a one-time treatment using CRISPR gene editing to fix a patient's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the modified cells after a short course of chemotherapy. …

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The goal was to see how the drug moves through the body and to check its safety. All children had at least one specific gene mutation th…

This study looked at how the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) affects coughing and physical activity in 82 people with cystic fibrosis aged 12 and older. Participants wore activity trackers to measure cough frequency and step counts over 12 weeks. The …

This study tested a new drug called VX-147 in 16 adults with a specific genetic form of kidney disease (APOL1-mediated FSGS). The goal was to see if the drug could reduce protein in the urine, a sign of kidney damage, and to check for side effects. Participants had a kidney biops…

This study tested an experimental drug called VX-548 in 218 adults with painful lumbosacral radiculopathy, a condition where a nerve in the lower back is irritated, causing leg pain. The goal was to see if the drug reduces leg pain and improves sleep compared to a placebo. The st…

This study tested a drug called suzetrigine for managing acute pain after specific surgeries, like laparoscopic or arthroscopic procedures. It involved 47 adults and looked at how well the drug worked and its safety when used with other pain treatments. The goal was to see if pat…

This study tested a medicine called suzetrigine for treating short-term pain after cosmetic or reconstructive surgery. About 100 adults took the drug as part of a combination of pain treatments. The goal was to see if it worked well and was safe.

This study tested a new drug called VX-407 in 32 healthy adults to see if it is safe and how the body handles it. The drug is being developed for a kidney condition called ADPKD. This early research does not offer treatment but gathers important safety information for future stud…

This study followed 79 infants with cystic fibrosis under 6 months old to see how their pancreas function changed over the first year of life. Researchers measured a stool enzyme called fecal elastase-1 to track pancreatic health. No treatment was given; the goal was simply to un…

This early-stage study tested a new granule (powder) version of three cystic fibrosis drugs (vanzacaftor, tezacaftor, and deutivacaftor) in 34 healthy adults. The goal was to see how well the body absorbs the granule form, whether food changes its effects, and if different doses …

This study looked at how a cystic fibrosis drug combination (vanzacaftor/tezacaftor/deutivacaftor) affects the levels of a common cholesterol drug (rosuvastatin) in the body. 18 healthy adults took both medications, and researchers measured drug levels and checked for side effect…

This study looked at how different versions of the drug povetacicept are absorbed by the body in 136 healthy adults. The goal was to compare the drug's bioavailability and check that different forms of the drug work the same way. Researchers also monitored safety and side effects…

This completed Phase 1 study looked at how a new drug called VX-407 changes the levels of various birth control pills in the body. 74 healthy adults participated to measure drug levels and check safety. The results will help doctors understand if birth control pills work properly…