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We list hundreds of Clinical Trials about "Vertex Receives CHMP Positive Opinion ORKAMBI lumacaftor ivacaftor" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.
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The present study is designed to support dose selection of N91115 in a planned Phase 2 study in CF patients who are on co-formulated lumacaftor/ivacaftor (Orkambi) treatment. Rifampin is being used in this drug-drug interaction (DDI) study to assess its effects on the PK of N91115, since rifampin's metabolic properties are believed to be similar to lumacaftor. The Orakambi NDA was approved July 1, 2015. It is important to understand the potential effects of Orkambi (lumacafto...
The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society...
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor affects glycemia in patient with cystic fibrosis.
In 2015, VERTEX company - producing already KALYDECO (IVACAFTOR, VX-770) potentiator molecule that is recommended for the treatment of CF patients aged ≥ 6 y, with CFTR mutation altering the channel regulation (class III mutations) as G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549Nou S549R) - was allowed by the Federal Drug Administration (FDA) and European Medicines Agency (EMEA) for producing and using the association LUMACAFTOR-IVACAFTOR in clinical trials to ma...
This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva. Clinical and laboratory measurements will be measured at baseline, 1 month, 3 months, and 6 months after initiation of tez/iva. The length of study participation will be approximately 6 months.
Shortness of breath (dyspnea) during exercise is a major source of distress and is a commonly reported symptom in patients with cystic fibrosis (CF). A recent treatment option known as Orkambi, which combines the drugs lumacaftor and ivacaftor, may be used in patients with CF to help improve lung health. However, the effects of this combination therapy on dyspnea and exercise performance, a known predictor of survival in CF, are not clear. The investigators aim to understand th...
The academic investigator - initiated trial will evaluate in a postapproval setting whether, and if yes, to what extent and variability, the treatment with lumacaftor in combination with ivacaftor reverses the p.Phe508del CFTR - mediated basic defect in p.Phe508del homozygous subjects with cystic fibrosis under real life conditions.
The purpose of this research study is to find out if the combined therapy lumacaftor-ivacaftor effects how people with cystic fibrosis respond to an oral glucose tolerance test, a test for diabetes.
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of Lumacaftor/Ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with Cystic Fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if patient meet the eligibility criteria.
Cystic Fibrosis related diabetes (CFRD), a major factor of morbid-mortality in CF, is characterized by a preclinical phase of glucose intolerance particularly long reaching up to 10 years. At the physiopathology level, insulin secretion is determinant in the glucose tolerance abnormalities in CF. Indeed insulin secretion is dependent of the CFTR activity at the beta cell surface and inhibition of CFTR leads to a decrease in insulin secretion. Recently, the combination o...
This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of N91115 in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.
Orkambi is currently approved for patients with CF carrying two copies of the common F508del variant, but not for those with the P67L mutation. This is a clinical study of 22 subjects carrying P67L, each given Orkambi for approximately four weeks. The researchers will monitor clinical endpoints that include FEV1, sweat chloride, and nasal potential difference. Cutaneous punch biopsy material will be obtained from each subject so that iPS cells can be differentiated into airway ...
This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with CF who are homozygous for the F508del CFTR mutation.
The primary purpose of study is to evaluate the treatment effect of VX-661 in combination with ivacaftor (VX-661/ivacaftor) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of VX-661/ivacaftor through Week 72
The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are
The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation
A Phase 3 Study of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are
To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 in combination with ivacaftor (IVA) (VX-661/IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del. Our goal is to perform the exercise and vascular measurements every 3 months after a patient starts taking Orkambi.
This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.
This is an observational study for children with Cystic Fibrosis (CF) who are eligible based on their CF gene type. One group will be called the treatment group because they have the gene type (homozygous F508del) that makes them clinically eligible through their CF care provider to begin treatment with the new FDA approved CF drug called orkambi. For the control group, children will be enrolled who have a similar CF gene type (heterozygous F508del) but are not eligible to be p...