American journal of respiratory and critical care medicine
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Am. J. Respir. Crit. Care Med. · Jun 2013
Randomized Controlled Trial Multicenter StudyEfficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation.
Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been shown to improve lung function, pulmonary exacerbation rate, respiratory symptoms, and weight gain compared with placebo in patients with cystic fibrosis aged 12 years or older with a G551D-CFTR mutation. ⋯ In patients who are younger and healthier than those in previously studied populations, ivacaftor demonstrated a significant improvement in pulmonary function, weight, and CFTR activity compared with placebo. Clinical trial registered with www.clinicaltrials.gov (NCT00909727).
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Am. J. Respir. Crit. Care Med. · Jun 2013
Multicenter Study Comparative StudyResearch recruitment practices and critically ill patients. A multicenter, cross-sectional study (the Consent Study).
Limited cross-sectional data exist to characterize the challenges of enrolling critically ill patients into research studies. ⋯ A large gap exists between eligibility and the frequency with which consent encounters occur in intensive care unit research. Recruitment is susceptible to design and procedural inefficiencies that hinder recruitment and to personnel availability, given the need to interact with SDMs. Current enrollment practices may underrepresent potential study populations.