A5034938538- Cystic Fibrosis Research Advances
- Statistical Methods in Clinical Trials
- Nematode management and characterization studies
- Legume Nitrogen Fixing Symbiosis
- Optimal Experimental Design Methods
- Gene expression and cancer classification
Vertex Pharmaceuticals (United States)
2019-2023
Background In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients cystic fibrosis (CF) ≥12 years age who have at least one F508del allele. The aim this study is assess long-term safety efficacy ELX/TEZ/IVA these patients. Methods 3, open-label, single-arm extension study, participants –minimal function (from a 24-week parent study; n=399) or – 4-week n=107) genotypes receive the same dose (ELX 200 mg...
<h2>Abstract</h2> Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with cystic fibrosis (CF) heterozygous for <i>F508del</i> a minimal function mutation (<i>F</i>/MF) or homozygous (<i>F</i>/<i>F</i>) two pivotal Phase 3 trials, significantly improving percentage predicted forced expiratory volume 1 second, Cystic Fibrosis Questionnaire–Revised, Respiratory Domain (CFQ-R RD) scores, sweat chloride concentration. Here, we analyzed the 11...
Elexacaftor/tezacaftor/ivacaftor is highly effective in treating people with cystic fibrosis (pwCF) who have ≥ 1 responsive mutation. Liver disease occurs approximately 10%–20% of pwCF. The objective this study was to assess the safety and pharmacokinetics elexacaftor/tezacaftor/ivacaftor moderate hepatic impairment, which necessary inform on its use guide dosing recommendations. were evaluated subjects without CF impairment versus matched healthy controls. Twenty-two (11 11 subjects)...
For multiple rare diseases as defined by a common biomarker signature, or disease with subtypes of low frequency, it is often possible to provide confirmatory evidence for these (baskets) combined group. A novel drug, second generation, may have marginal improvement in efficacy overall but superior some baskets. In this situation, appealing test hypotheses both non-inferiority and superiority on certain The challenge designing study efficient address questions one trial. two-stage adaptive...