Vertex Pharmaceuticals Incorporated

This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…

This phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…

This study looks at the long-term safety and effectiveness of the drug combination Trikafta (elexacaftor/tezacaftor/ivacaftor) in people with cystic fibrosis who have certain non-F508del gene mutations. About 297 participants who completed a prior study will continue taking the m…

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects a…

This phase 3 study is testing the long-term safety and effectiveness of the drug combination elexacaftor/tezacaftor/ivacaftor (known as Trikafta) in 50 people with cystic fibrosis who are at least 12 months old. Participants previously completed a related study and will continue …

This early-phase trial tests VX-264, a therapy made from stem cells that produce insulin, in 7 people with long-standing type 1 diabetes. The goal is to see if it is safe and can help control blood sugar. If successful, it may reduce or replace the need for insulin injections.

This study tests a triple-combination drug (VX-121/tezacaftor/deutivacaftor) in children aged 1 to 11 with cystic fibrosis who have a specific genetic mutation. The goal is to check how the medicine works in the body, its safety, and how well children tolerate it. Around 210 chil…

This early-stage study tests the safety and how the body handles a new drug called VX-828, alone or combined with other CF medicines. It involves about 165 healthy adults and people with cystic fibrosis. The goal is to see if VX-828 is safe enough for further testing.

This early-stage trial tests an inhaled mRNA therapy called VX-522, combined with an existing drug (ivacaftor), in 26 adults with cystic fibrosis who have genetic mutations that do not respond to current modulator treatments. The main goals are to check safety and tolerability, a…

This study tests a drug called inaxaplin in 42 people 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, a sign of kidney damage, and to check for side effects. Participants take the drug as tablets, and…

This study tests a triple-combination drug (VX-121/TEZ/D-IVA) for people with cystic fibrosis, a genetic condition that affects the lungs and digestion. The goal is to see if the treatment is safe and works well over a long period. About 822 participants who completed earlier stu…

This phase 3 study tests the long-term safety and effectiveness of a triple combination drug (vanzacaftor/tezacaftor/deutivacaftor) in 174 people with cystic fibrosis aged 1 and older. Participants who completed a prior study will receive the drug to see if it helps control sympt…

This study was designed to test a single dose of exa-cel, a gene-edited cell therapy, in people with severe sickle cell disease who have the HbSC genotype. Participants would have received their own modified stem cells after chemotherapy. However, the trial was withdrawn before a…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…

This study tests a drug called suzetrigine (VX-548) for long-term safety and pain relief in people with diabetic peripheral neuropathy. About 455 adults who completed a prior 12-week study will take the drug for up to one year. Researchers will track side effects and changes in p…

This early-stage study is testing how a new pain medication called suzetrigine interacts with efavirenz, a drug used to treat HIV. The trial involves 18 healthy adults and measures how the body processes suzetrigine when taken alone versus with efavirenz. The goal is to check for…

This study looks at 401 people with autosomal dominant polycystic kidney disease (ADPKD) to see how different versions of the PKD1 gene relate to patient traits like age at diagnosis and body size. Researchers are not testing any treatment—they are simply gathering information to…