Vertex Pharmaceuticals Incorporated

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 previously taken the drug in an earlier study. Researchers tracked sid…

This study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…

This study tested a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) in 70 toddlers aged 12 to 24 months with cystic fibrosis. The goal was to check how the drug moves through the body, its safety, and any side effects. The trial has finished, and results will help dete…

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 its safety. The drug is taken by mouth and aims t…

This study tested a combination drug (elexacaftor/tezacaftor/ivacaftor) in 82 people with cystic fibrosis aged 12 and older. Researchers used wearable devices to measure how much the drug reduced coughing and increased physical activity over 12 weeks. The goal was to see if the t…

This phase 3 study tested the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (Trikafta) in 507 people with cystic fibrosis who have the F508del mutation. Participants had already completed a previous study and continued taking the medi…

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

This completed Phase 2 trial tested the drug VX-993 in 367 people who had bunion surgery. The goal was to see if it safely reduces acute pain compared to a placebo. Participants took VX-993 tablets or a placebo, and researchers measured pain intensity over 48 hours.

This study tested a new painkiller called suzetrigine (Journavx) in 47 adults who had laparoscopic or arthroscopic surgery. The goal was to see if it works well and is safe as part of a combination of pain treatments. Participants took the tablet by mouth, and the study is now co…

This study tested the effectiveness and safety of a medicine called suzetrigine, used together with other pain treatments, for acute pain after cosmetic or reconstructive surgery. About 100 adults took part. The goal was to see if patients rated their pain control as good, very g…

This completed Phase 2 trial tested the drug VX-548 (Suzetrigine) in 218 adults with painful lumbosacral radiculopathy, a condition where a pinched nerve in the lower back causes leg pain. The study compared the drug to a placebo to see if it safely reduces leg pain and improves …

This completed Phase 1 study tested how a cystic fibrosis drug combo (vanzacaftor/tezacaftor/deutivacaftor) affects the body's processing of the statin rosuvastatin. 18 healthy adults took both medications to check for interactions and safety. The goal was to see if the drug comb…

This study looked at how a new drug called VX-407 changes the way birth control pills work in the body. 74 healthy adults took VX-407 along with different types of oral contraceptives. The goal was to see if VX-407 affects the levels of these contraceptives, which is important fo…

This early-stage study tested the safety and how the body handles a new drug called VX-407 in 32 healthy adults. The goal was to check for side effects and measure drug levels in the blood. No treatment benefit was expected, as this study was designed to gather safety information…

This completed study followed 79 infants with cystic fibrosis during their first year of life to see how well their pancreas works over time. Researchers measured a substance called fecal elastase-1 in stool samples to track pancreatic function. The goal was to learn more about t…

This 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 absorption, and if different doses work as expec…

This early-stage study tested different versions of the drug povetacicept in 136 healthy adults to see how well the body absorbs each form. The goal was to compare the drug's bioavailability and check that different presentations work the same way. Researchers also monitored safe…