Vertex Pharmaceuticals Incorporated

This study tested 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 of the same drug. The main goal was to see how safe and…

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

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 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 a medicine called suzetrigine for treating acute pain after keyhole surgery (laparoscopy) or joint surgery (arthroscopy). 47 adults took part. The goal was to see how well the medicine worked and if it was safe when used with other pain treatments.

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 safely reduces leg pain and improves sleep compared to a placebo.…

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 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…

This early-stage study tested the safety and how the body handles VX-407 in 32 healthy adults. Participants received either the drug or a placebo to check for side effects and measure drug levels in the blood. The results will help decide if VX-407 should move into future trials …

This study tested a new granule (small particle) form of three cystic fibrosis medicines in 34 healthy adults. The goal was to see how well the body absorbs the medicine and whether food changes that. This is an early-stage study to gather information, not a treatment for patient…

This study looked at how the body absorbs different versions of the drug povetacicept in 136 healthy adults. The goal was to compare the drug's formulations and check for safety. No treatment was given—just careful measurement of drug levels in the blood.

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

This study followed 79 infants with cystic fibrosis (CF) under 6 months old to see how their pancreas function changed during 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 simpl…