Search results for "Interim analysis"
showing 10 items of 59 documents
Add-on salmeterol compared to double dose fluticasone in pediatric asthma: A double-blind, randomized trial (VIAPAED)
2009
Rationale In asthmatic children whose symptoms are uncontrolled on standard doses of inhaled corticosteroids (ICS), guidelines recommend to either increase the ICS dose or to add further controller medication, e.g. a long acting s2-agonist (LABA). The aim of this study was to compare the efficacy and safety of doubling the dose of ICS (fluticasone proprionate FP 200 µg twice daily) with adding a long-acting beta-2 agonist to the ICS (SFC, salmeterol 50 µg/ FP 100 µg twice daily) in children with uncontrolled asthma. Methods Children between 4 and 16 years of age were eligible for this multicenter, randomized, double blind, double dummy, parallel-group study. During a 14-day run-in phase, al…
NEONAX trial: Neoadjuvant plus adjuvant or only adjuvant nab-paclitaxel plus gemcitabine for resectable pancreatic cancer, a phase II study of the AI…
2019
4128 Background: Survival in pancreatic cancer (PDAC) is still poor even after curatively intended resection. Perioperative treatment approaches improve outcome in various tumor entities. Data on perioperative treatment in resectable PDAC are limited and there is a debate whether neoadjuvant treatment might impair subsequent surgery by adding perioperative morbidity or mortality. Methods: NEONAX is a randomized phase II study (planned 166 patients) of perioperative gemcitabine/nab-paclitaxel (Arm A: 2 pre- and 4 post-operative cycles, Arm B: 6 cycles adjuvant) for patients with primarily resectable PDAC. Primary objective is DFS at 18 months after randomization. Secondary objectives are 3-…
Real-World Management of Myelofibrosis with Ruxolitinib: Initial Analysis of an Italian Observational Study (ROMEI)
2018
Abstract Introduction ROMEI (CINC424AIT04 Ruxolitinib Observational study in Myelofibrosis treated patiEnts in Italy) is a prospective observational study that aims to bridge the knowledge gap between the clinical experience of registration trials and routine patient management by following roughly 200 myelofibrosis (MF) patients (pts) treated with ruxolitinib in everyday clinical practice. Enrollment began in April 2017 and ended in May 2018. Methods The primary endpoint is to evaluate changes in symptoms and quality of life during treatment with ruxolitinib through the Myeloproliferative Neoplasm 10 (MPN-10) disease-specific questionnaire and EuroQoL-5D-5L (EQ-5D-5L) general health questi…
Sorafenib in advanced hepatocellular carcinoma
2008
none 25 BACKGROUND: No effective systemic therapy exists for patients with advanced hepatocellular carcinoma. A preliminary study suggested that sorafenib, an oral multikinase inhibitor of the vascular endothelial growth factor receptor, the platelet-derived growth factor receptor, and Raf may be effective in hepatocellular carcinoma. METHODS: In this multicenter, phase 3, double-blind, placebo-controlled trial, we randomly assigned 602 patients with advanced hepatocellular carcinoma who had not received previous systemic treatment to receive either sorafenib (at a dose of 400 mg twice daily) or placebo. Primary outcomes were overall survival and the time to symptomatic progression. Seconda…
Interim Analysis of Overall Survival Per Subgroups in the Prospective, Non-Interventional Insight Study in Patients with Hepatocellular Carcinoma Tre…
2012
ABSTRACT Background The efficacy of Sorafenib in patients (pts) with hepatocellular carcinoma (HCC) has been proven in randomized, controlled trials. INSIGHT is a prospective, non-interventional study, conducted in Germany and Austria in pts with HCC. The objectives of this study are the evaluation of safety and efficacy under practice conditions in both hospitals and private practices. Enrollment into INSIGHT is not restricted to a particular tumor stage. Recruitment into the study is ongoing. Methods The second interim analysis (data cut-off 23 FEB 2012) evaluated overall survival and safety data in relevant subgroups. All patients with HCC were observed for the duration of their sorafeni…
Sorafenib in advanced hepatocellular carcinoma – We have won a battle not the war
2008
Sorafenib in advanced hepatocellular carcinoma. Llovet JM, Ricci S, Mazzaferro V, Hilgard P, Gane E, Blanc JF, de Oliveira AC, Santoro A, Raoul JL, Forner A, Schwartz M, Porta C, Zeuzem S, Bolondi L, Greten TF, Galle PR, Seitz JF, Borbath I, Haussinger D, Giannaris T, Shan M, Moscovici M, Voliotis D, Bruix J; SHARP Investigators Study Group. Background No effective systemic therapy exists for patients with advanced hepatocellular carcinoma. A preliminary study suggested that sorafenib, an oral multi-kinase inhibitor of the vascular endothelial growth factor receptor, the platelet-derived growth factor receptor, and Raf may be effective in hepatocellular carcinoma. Methods In this multicente…
Performance of adaptive sample size adjustment with respect to stopping criteria and time of interim analysis
2006
The benefit of adjusting the sample size in clinical trials on the basis of treatment effects observed in interim analysis has been the subject of several recent papers. Different conclusions were drawn about the usefulness of this approach for gaining power or saving sample size, because of differences in trial design and setting. We examined the benefit of sample size adjustment in relation to trial design parameters such as 'time of interim analysis' and 'choice of stopping criteria'. We compared the adaptive weighted inverse normal method with classical group sequential methods for the most common and for optimal stopping criteria in early, half-time and late interim analyses. We found …
Adaptive Modifications of Hypotheses After an Interim Analysis
2001
It is investigated how one can modify hypotheses in a trial after an interim analysis such that the type I error rate is controlled. If only a global statement is desired, a solution was given by Bauer (1989). For a general multiple testing problem, Kieser, Bauer and Lehmacher (1999) and Bauer and Kieser (1999) gave solutions, by means of which the initial set of hypotheses can be reduced after the interim analysis. The same techniques can be applied to obtain more flexible strategies, as changing weights of hypotheses, changing an a priori order, or even including new hypotheses. It is emphasized that the application of these methods requires very careful planning of a trial as well as a c…
The Artificial Kidney Initiation in Kidney Injury 2 (AKIKI2): study protocol for a randomized controlled trial
2019
Abstract Background The Artificial Kidney Initiation in Kidney Injury (AKIKI) trial showed that a delayed renal replacement therapy (RRT) strategy for severe acute kidney injury (AKI) in critically ill patients was safe and associated with major reduction in RRT initiation compared with an early strategy. The five criteria which mandated RRT initiation in the delayed arm were: severe hyperkalemia, severe acidosis, acute pulmonary edema due to fluid overload resulting in severe hypoxemia, serum urea concentration > 40 mmol/l and oliguria/anuria > 72 h. However, duration of anuria/oliguria and level of blood urea are still criteria open to debate. The objective of the study is to compar…
Results of the TOP Study: Prospectively Randomized Multicenter Trial of an Ex Vivo Tacrolimus Rinse Before Transplantation in EDC Livers
2016
Background. Organ shortage results in the transplantation of extended donor criteria (EDC) livers which is associated with increased ischemia-reperfusion injury (IRI). Experimental studies indicate that an organ rinse with the calcineurin inhibitor tacrolimus before implantation protects against IRI. The tacrolimus organ perfusion study was initiated to examine the effects of ex vivo tacrolimus perfusion on IRI in transplantation of EDC livers. Methods. A prospective randomized multicenter trial comparing ex vivo perfusion of marginal liver grafts (>= 2 EDC according to Eurotransplant manual) with tacrolimus (20 ng/mL) or histidine-tryptophane-ketoglutarate solution (control) was carried ou…