Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis.

Background: Ivacaftor (IVA) has been shown to be safe and efficacious in children aged ≥4 months with cystic fibrosis (CF) and CFTR gating variants. We evaluated safety, pharmacokinetics (PK), and efficacy of IVA in a small cohort of infants aged 1 to <4 months with CF.

Methods: In this phase 3, open-label study, infants 1 to <4 months with CF and an IVA-responsive CFTR variant received an initial low dose of IVA based on age and weight.

Application of machine learning to predict reduction in total PANSS score and enrich enrollment in schizophrenia clinical trials.

Clinical trial efficiency, defined as facilitating patient enrollment, and reducing the time to reach safety and efficacy decision points, is a critical driving factor for making improvements in therapeutic development. The present work evaluated a machine learning (ML) approach to improve phase II or proof-of-concept trials designed to address unmet medical needs in treating schizophrenia. Diagnostic data from the Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE) trial were used to develop a binary classification ML model predicting individual patient response as either "improvement," defined as greater than 20% reduction in total Positive and Negative Syndrome Scale (PANSS) score, or "no improvement," defined as an inadequate treatment response (<20% reduction in total PANSS).

Prediction of Nephropathy in Type 2 Diabetes: An Analysis of the ACCORD Trial Applying Machine Learning Techniques.

Applying data mining and machine learning (ML) techniques to clinical data might identify predictive biomarkers for diabetic nephropathy (DN), a common complication of type 2 diabetes mellitus (T2DM). A retrospective analysis of the Action to Control Cardiovascular Risk in Diabetes (ACCORD) trial was intended to identify such factors using ML. The longitudinal data were stratified by time after patient enrollment to differentiate early and late predictors.

Early Systemic Alterations in Severe Spinal Cord Injury: An Experimental Study on the Impact of Injury Level on Renal Function.

Study Design: An experimental model of spinal cord injury (SCI) intended to characterize changes in renal function.

Objective: The aim of this study was to evaluate the possible influence of SCI level on renal function during spinal shock.

Summary Of Background Data: SCI triggers multiple systemic and metabolic alterations.

A novel, simple and inexpensive procedure for the simultaneous determination of iopamidol and p-aminohippuric acid for renal function assessment from plasma samples in awake rats.

The purpose of the current study was to design, validate and implement a novel analytical method for the simultaneous plasma measurement of iopamidol and p-aminohippuric acid (PAH) to estimate renal function in awake rats. A reverse-phase high performance liquid chromatographic (RP-HPLC) method for the simultaneous measurement of iopamidol (for glomerular filtration rate estimation, GFR) and PAH (for tubular secretion determination, TS) was designed and validated using a C-18 column, 0.1M acetic acid-10% acetonitrile (90:10, v/v) as mobile phase, at a flow rate of 0.