This trial is evaluating whether VX-121/TEZ/D-IVA will improve 1 primary outcome and 3 secondary outcomes in patients with Cystic Fibrosis. Measurement will happen over the course of From Baseline Through Week 24.
This trial requires 400 total participants across 2 different treatment groups
This trial involves 2 different treatments. VX-121/TEZ/D-IVA is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.
"A mutation in a cystic fibrosis transmembrane conductance regulator (CFTR) gene provides a reasonable explanation for about 70% of cases. Around 20% of patients with CF have a G551D mutation in the CFTR gene that confers a milder phenotype, and is almost entirely the cause of milder cases. In more severe cases there are mutations in other genes, notably the SLC26A4 gene. The gene may be involved in the development of the pancreas, sweat glands, parasympathetic nerves, sperm or pancreas. About 6% of cases have normal CFTR genes but are also carriers of a second mutated gene from both parents." - Anonymous Online Contributor
"In the United States, the approximate prevalence of cystic fibrosis in children, including infants, is one in 3,650, as diagnosed by a physician. It is not clear which populations are at risk for developing disease; however, a few subgroups are at increased risk. For instance, persons of Ashkenazi Jewish descent with a known history of cystic fibrosis are much more likely to be affected than persons of similarly descended non-Jewish ethnicity. This information can be utilized to identify high-risk populations, and may be valuable in establishing appropriate epidemiologic studies and, ultimately, in targeting interventions and prevention efforts. [provided by Ref." - Anonymous Online Contributor
"Signs of CF include problems with breathing, the digestive system, and the skin. These symptoms may appear as early as the newborn stage of life and may be as noticeable in children as young as 12 months.\n" - Anonymous Online Contributor
"While current treatments for CF do help prolong survival, they do not eliminate the cause of the disease. In the future, research teams may develop more effective and less invasive treatments that could eventually help cure CF." - Anonymous Online Contributor
"The aim of treatment of CF is to keep patients as healthy as possible and maintain lung function. Patients often require supplemental oxygen for their survival, and if left untreated, can develop irreversible pulmonary disease. The disease can be cured with a lung transplant but is limited by the number of available donors. Regular screening, early diagnosis, and treatment with CFTR modulators are key to minimize complications.\n\nPalliative therapy in oncology are available for patients that are in the late stages of their disease and include end-of-life interventions. Palliative therapy can be used to treat [chronic pain](https://www.withpower.com/clinical-trials/chronic-pain) and symptoms during end-of-life, particularly for patients with advanced cancer that have intractable pain." - Anonymous Online Contributor
"CF affects the entire body, causing irreversible consequences including chronic infection by pathogens and impaired nutrition uptake. The most severe manifestations include chronic obstructive pulmonary disease that can eventually lead to death. There are three types of CF, with different symptoms and outcome. The autosomal dominance transmission makes it easy for parents to pass the gene to their children, and almost any combination of those three genetic mutations causes CF. Cystic fibrosis is caused by a mutation of the CF transmembrane conductance regulator gene. It is the most prevalent genetic error underlying cystic fibrosis, and is a rare disease. Cystic fibrosis can be found with or without symptoms. In most cases, infants and children are asymptomatic." - Anonymous Online Contributor
"Vx-121/TEZ/DIVA provides a significant number of patients with more favorable patient-rated QoL outcomes compared to placebo. Overall, patients treated with Vx-121/TEZ/DIVA had similar results to patients treated with IBN-025 on most QoL outcomes. Results from a recent paper support the potential therapeutic role of Vx-121/TEZ/DIVA in the clinical management of patients with cystic fibrosis." - Anonymous Online Contributor
"There is no evidence from these three families in Australia for a genetic link between the parents. If such a link is actually present, it may be small and not evident. The finding that the parents have no CF-causing mutation in CF genes and only those people with mutations will have children and family members is consistent with the expectation and the current theories of disease." - Anonymous Online Contributor
"A single iv injection of Vx-121 (100 ng/kg) leads to an increase in plasma sodium levels by about 2.5mEq/L, resulting in a decrease by about 0.8mEq/l in urine ionized chloride (p<0.001). The sodium levels of the treatment groups appear to return to baseline four hours thereafter. TSD (130 μg/d D-iva) produces no change in any of these parameters, but the placebo group showed a slight, small increase in urine chloride." - Anonymous Online Contributor
"Data from a recent study, VX-121/TEZ/IVA was shown to be effective in reducing severity and prolonging survival when used as part of multi-drug, high-dose, inhaled antibiotic treatment regimen for patients with bronchiectasis due to cystic fibrosis." - Anonymous Online Contributor
"Some people (for example, parents, siblings and medical professionals) may assume that cystic fibrosis is a result of a particular environmental risk factor or a random genetic event. This assumption is not supported by current evidence since almost all people with CF have a common genetic cause. Although the underlying mechanisms are still not fully understood, it is likely that many genetic and environmental risk factors combine in a variety of ways to cause disease. At present it is not possible to tell in advance which environmental factors could cause CF.\n\nThe most likely cause of cystic fibrosis is a mutation in the CFTR gene which stops the production of the CFTR protein in the cells that line the lungs and pancreas." - Anonymous Online Contributor
"In patients with cystic fibrosis, vx-121/tez/d-iva reduces the need for medications to control diarrhea by about 29-67%, compared with placebo and vx-121/tez/d-iva reduced the need for sputum exchange by about 12-45%. However, the improvement in overall care does not translate into significant improvements in clinical outcome. Vx-121/tez/d-iva was poorly well tolerated. Adverse effects were similar to those observed with placebo and included nausea, vomiting, diarrhea, diarrhea with fever, abdominal pain, constipation and anemia. Drug interactions were also not altered by vx-121/tez/d-iva." - Anonymous Online Contributor