title: A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System (BVS EECSS) in the Treatment of Patients With de Novo Native Coronary Artery Lesions.

acronym: ABSORB B

summary:
The purpose of this study is to assess the safety and performance of the BVS Everolimus Eluting Coronary Stent System (EECSS) in the treatment of patients with a maximum of two de novo native coronary artery lesions located in two different major epicardial vessels.

Currently in development at Abbott Vascular. Not available for sale in the United States.

ReferenceCitation: Zeng Y, Cavalcante R, Collet C, Tenekecioglu E, Sotomi Y, Miyazaki Y, Katagiri Y, Asano T, Abdelghani M, Nie S, Bourantas CV, Bruining N, Onuma Y, Serruys PW. Coronary calcification as a mechanism of plaque/media shrinkage in vessels treated with bioresorbable vascular scaffold: A multimodality intracoronary imaging study. Atherosclerosis. 2018 Feb;269:6-13. doi: 10.1016/j.atherosclerosis.2017.11.002. Epub 2017 Dec 2.

family: Zeng

full_name: Yaping Zeng

given: Yaping

family: Cavalcante

full_name: Rafael Cavalcante

given: Rafael

family: Collet

full_name: Carlos Collet

given: Carlos

family: Tenekecioglu

full_name: Erhan Tenekecioglu

given: Erhan

family: Sotomi

full_name: Yohei Sotomi

given: Yohei

family: Miyazaki

full_name: Yosuke Miyazaki

given: Yosuke

family: Katagiri

full_name: Yuki Katagiri

given: Yuki

family: Asano

full_name: Taku Asano

given: Taku

family: Abdelghani

full_name: Mohammad Abdelghani

given: Mohammad

family: Nie

full_name: Shaoping Nie

given: Shaoping

family: Bourantas

full_name: Christos V. Bourantas

given: Christos V.

family: Bruining

full_name: Nico Bruining

given: Nico

family: Onuma

full_name: Yoshinobu Onuma

given: Yoshinobu

family: Serruys

full_name: Patrick W. Serruys

given: Patrick W.

journal_name: Atherosclerosis

ReferenceCitation: Onuma Y, Grundeken MJ, Nakatani S, Asano T, Sotomi Y, Foin N, Ng J, Okamura T, Wykrzykowska JJ, de Winter RJ, van Geuns RJ, Koolen J, Christiansen EH, Whitbourn R, McClean D, Smits P, Windecker S, Ormiston JA, Serruys PW. Serial 5-Year Evaluation of Side Branches Jailed by Bioresorbable Vascular Scaffolds Using 3-Dimensional Optical Coherence Tomography: Insights From the ABSORB Cohort B Trial (A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System in the Treatment of Patients With De Novo Native Coronary Artery Lesions). Circ Cardiovasc Interv. 2017 Sep;10(9). pii: e004393. doi: 10.1161/CIRCINTERVENTIONS.116.004393. Erratum in: Circ Cardiovasc Interv. 2018 Jan;11(1):e000031. Christiansen, Evald [corrected to Christiansen, Evald H].

name: From the Thoraxcenter, Erasmus MC, Rotterdam, the Netherlands (Y.O., S.N., R.-J.v.G.); Cardialysis, Rotterdam, the Netherlands (Y.O.); Academic Medical Center, University of Amsterdam, the Netherlands (M.J.G., T.A., Y.S., J.J.W., R.J.d.W.); National Heart Centre Singapore (N.F., J.N.); National University of Singapore (J.N.); Department of Medicine and Clinical Science, Yamaguchi University Graduate School of Medicine, Yamaguchi-Ube, Japan (T.O.); Catharina Ziekenhuis, Eindhoven, the Netherlands (J...

family: Grundeken

full_name: Maik J. Grundeken

given: Maik J.

family: Nakatani

full_name: Shimpei Nakatani

given: Shimpei

family: Foin

full_name: Nicolas Foin

given: Nicolas

family: Ng

full_name: Jaryl Ng

given: Jaryl

publisher: Ovid Technologies (Wolters Kluwer Health)

ReferenceCitation: Zeng Y, Tateishi H, Cavalcante R, Tenekecioglu E, Suwannasom P, Sotomi Y, Collet C, Nie S, Jonker H, Dijkstra J, Radu MD, Räber L, McClean DR, van Geuns RJ, Christiansen EH, Fahrni T, Koolen J, Onuma Y, Bruining N, Serruys PW. Serial Assessment of Tissue Precursors and Progression of Coronary Calcification Analyzed by Fusion of IVUS and OCT: 5-Year Follow-Up of Scaffolded and Nonscaffolded Arteries. JACC Cardiovasc Imaging. 2017 Oct;10(10 Pt A):1151-1161. doi: 10.1016/j.jcmg.2016.11.016. Epub 2017 Mar 15.

ReferenceCitation: Onuma Y, Collet C, van Geuns RJ, de Bruyne B, Christiansen E, Koolen J, Smits P, Chevalier B, McClean D, Dudek D, Windecker S, Meredith I, Nieman K, Veldhof S, Ormiston J, Serruys PW; ABSORB Investigators. Long-term serial non-invasive multislice computed tomography angiography with functional evaluation after coronary implantation of a bioresorbable everolimus-eluting scaffold: the ABSORB cohort B MSCT substudy. Eur Heart J Cardiovasc Imaging. 2017 May 1;18(8):870-879. doi: 10.1093/ehjci/jex022.

name: Department of Interventional Cardiology, ThoraxCenter, Erasmus University Medical Center,’s-Gravendijkwal 230, 3015 CE Rotterdam, The Netherlands

name: Cardiology Department, Academic Medical Center, Meibergdreef 9, 1105 AZ Amsterdam-Zuidoost, Amsterdam, The Netherlands

name: Cardiology Department, Academic Medical Center, Amsterdam, Cardialysis, Westblaak 98, 3012 KM Rotterdam, The Netherlands

name: Department of Cardiology, Onze-Lieve-Vrouwziekenhuis, Moorselbaan 164, 9300 Aalst, Belgium

name: Department of Cardiology, Skejby Sygehus, Aarhus Universitet Skejby Sygehus, Brendstrupgårdsvej 100, 8200 Aarhus N, Denmark

name: Department of Cardiology, Catharina Ziekenhuis, Michelangelolaan 2, 5623 EJ Eindhoven, the Netherlands

name: Department of Cardiology, Maasstad Ziekenhuis, Maasstadweg 21, 3079 DZ, Rotterdam, the Netherlands

name: Department of Interventional Cardiology, Institut Hospital Jacques Cartier, 6 Avenue du Noyer Lambert, 91300 Massy, France

name: Department of Cardiology, Christchurch Hospital, 2 Riccarton Ave, Christchurch Central, Christchurch 4710, New Zealand

name: Jagiellonian University Institute of Cardiology, University Hospital Krakow, Mikołaja Kopernika 36, 31-501 Kraków, Poland

name: Universitätsklinik für Kardiologie, Inselspital, Freiburgstrasse 4, 3010 Bern, Switzerland

name: Monash Heart, Monash Medical Centre, 246 Clayton Rd, Clayton VIC 3168, Melbourne, Australia

name: Clinical Development, Abbott Vascular, Diegem, Belgium

name: Department of Cardiology, Auckland City Hospital, 2 Park Rd, Grafton, Auckland 1023, New Zealand

name: Imperial College London Kensington, London SW7 2AZ, UK

journal_name: European Heart Journal - Cardiovascular Imaging

ReferenceCitation: Serruys PW, Ormiston J, van Geuns RJ, de Bruyne B, Dudek D, Christiansen E, Chevalier B, Smits P, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Wasungu L, Ediebah D, Veldhof S, Onuma Y. A Polylactide Bioresorbable Scaffold Eluting Everolimus for Treatment of Coronary Stenosis: 5-Year Follow-Up. J Am Coll Cardiol. 2016 Feb 23;67(7):766-76. doi: 10.1016/j.jacc.2015.11.060.

journal_name: Journal of the American College of Cardiology

ReferenceCitation: Ishibashi Y, Nakatani S, Sotomi Y, Suwannasom P, Grundeken MJ, Garcia-Garcia HM, Bartorelli AL, Whitbourn R, Chevalier B, Abizaid A, Ormiston JA, Rapoza RJ, Veldhof S, Onuma Y, Serruys PW. Relation Between Bioresorbable Scaffold Sizing Using QCA-Dmax and Clinical Outcomes at 1 Year in 1,232 Patients From 3 Study Cohorts (ABSORB Cohort B, ABSORB EXTEND, and ABSORB II). JACC Cardiovasc Interv. 2015 Nov;8(13):1715-26. doi: 10.1016/j.jcin.2015.07.026.

repositories_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

ReferenceCitation: Karanasos A, Garcia-Garcia HM, van Geuns RJ, Regar E. Fate of side-branch jailing and a malapposed platinum marker after resorption of an everolimus-eluting bioresorbable vascular scaffold: serial optical coherence tomography observations. JACC Cardiovasc Interv. 2015 Mar;8(3):e53-e54. doi: 10.1016/j.jcin.2014.10.020.

title: Fate of Side-Branch Jailing and a Malapposed Platinum Marker After Resorption of an Everolimus-Eluting Bioresorbable Vascular Scaffold

ReferenceCitation: Onuma Y, Serruys PW, Muramatsu T, Nakatani S, van Geuns RJ, de Bruyne B, Dudek D, Christiansen E, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Garcia-Garcia HM, Veldhof S, Rapoza R, Ormiston JA. Incidence and imaging outcomes of acute scaffold disruption and late structural discontinuity after implantation of the absorb Everolimus-Eluting fully bioresorbable vascular scaffold: optical coherence tomography assessment in the ABSORB cohort B Trial (A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System in the Treatment of Patients With De Novo Native Coronary Artery Lesions). JACC Cardiovasc Interv. 2014 Dec;7(12):1400-11. doi: 10.1016/j.jcin.2014.06.016.

title: Incidence and Imaging Outcomes of Acute Scaffold Disruption and Late Structural Discontinuity After Implantation of the Absorb Everolimus-Eluting Fully Bioresorbable Vascular Scaffold

ReferenceCitation: Zhang YJ, Iqbal J, Nakatani S, Bourantas CV, Campos CM, Ishibashi Y, Cho YK, Veldhof S, Wang J, Onuma Y, Garcia-Garcia HM, Dudek D, van Geuns RJ, Serruys PW; ABSORB Cohort B Study Investigators. Scaffold and edge vascular response following implantation of everolimus-eluting bioresorbable vascular scaffold: a 3-year serial optical coherence tomography study. JACC Cardiovasc Interv. 2014 Dec;7(12):1361-9. doi: 10.1016/j.jcin.2014.06.025. Epub 2014 Nov 12.

title: Scaffold and Edge Vascular Response Following Implantation of Everolimus-Eluting Bioresorbable Vascular Scaffold

ReferenceCitation: Muramatsu T, Onuma Y, van Geuns RJ, Chevalier B, Patel TM, Seth A, Diletti R, García-García HM, Dorange CC, Veldhof S, Cheong WF, Ozaki Y, Whitbourn R, Bartorelli A, Stone GW, Abizaid A, Serruys PW; ABSORB Cohort B Investigators; ABSORB EXTEND Investigators; SPIRIT FIRST Investigators; SPIRIT II Investigators; SPIRIT III Investigators; SPIRIT IV Investigators. 1-year clinical outcomes of diabetic patients treated with everolimus-eluting bioresorbable vascular scaffolds: a pooled analysis of the ABSORB and the SPIRIT trials. JACC Cardiovasc Interv. 2014 May;7(5):482-93. doi: 10.1016/j.jcin.2014.01.155. Epub 2014 Apr 16.

title: 1-Year Clinical Outcomes of Diabetic Patients Treated With Everolimus-Eluting Bioresorbable Vascular Scaffolds

ReferenceCitation: Serruys PW, Onuma Y, Garcia-Garcia HM, Muramatsu T, van Geuns RJ, de Bruyne B, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Rapoza R, Ormiston JA. Dynamics of vessel wall changes following the implantation of the absorb everolimus-eluting bioresorbable vascular scaffold: a multi-imaging modality study at 6, 12, 24 and 36 months. EuroIntervention. 2014 Mar 20;9(11):1271-84. doi: 10.4244/EIJV9I11A217.

title: Dynamics of vessel wall changes following the implantation of the Absorb everolimus-eluting bioresorbable vascular scaffold: a multi-imaging modality study at 6, 12, 24 and 36 months

ReferenceCitation: Ormiston JA, Serruys PW, Onuma Y, van Geuns RJ, de Bruyne B, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Rapoza R, Garcia-Garcia HM. First serial assessment at 6 months and 2 years of the second generation of absorb everolimus-eluting bioresorbable vascular scaffold: a multi-imaging modality study. Circ Cardiovasc Interv. 2012 Oct;5(5):620-32. doi: 10.1161/CIRCINTERVENTIONS.112.971549. Epub 2012 Oct 9.

name: From the Auckland City Hospital, Auckland, New Zealand (J.A.O.); ThoraxCenter, Erasmus Medical Center, Rotterdam, the Netherlands (P.W.S., Y.O., R.-J.v.G.); Cardiovascular Center, Aalst, Belgium (B.d.B.); Jagiellonian University, Krakow, Poland (D.D.); Skejby Sygehus University Hospital, Aarhus, Denmark (L.T.); Maasstad Hospital, Rotterdam, the Netherlands (P.C.S.); Institut Jacques Cartier, Massy, France (B.C.); Christchurch Hospital, Christchurch, New Zealand (D.M.C.); Catharina Hospital,...

title: First Serial Assessment at 6 Months and 2 Years of the Second Generation of Absorb Everolimus-Eluting Bioresorbable Vascular Scaffold

ReferenceCitation: Gutiérrez-Chico JL, Gijsen F, Regar E, Wentzel J, de Bruyne B, Thuesen L, Ormiston J, McClean DR, Windecker S, Chevalier B, Dudek D, Whitbourn R, Brugaletta S, Onuma Y, Serruys PW. Differences in neointimal thickness between the adluminal and the abluminal sides of malapposed and side-branch struts in a polylactide bioresorbable scaffold: evidence in vivo about the abluminal healing process. JACC Cardiovasc Interv. 2012 Apr;5(4):428-35. doi: 10.1016/j.jcin.2011.12.015.

title: Differences in Neointimal Thickness Between the Adluminal and the Abluminal Sides of Malapposed and Side-Branch Struts in a Polylactide Bioresorbable Scaffold

ReferenceCitation: Brugaletta S, Radu MD, Garcia-Garcia HM, Heo JH, Farooq V, Girasis C, van Geuns RJ, Thuesen L, McClean D, Chevalier B, Windecker S, Koolen J, Rapoza R, Miquel-Hebert K, Ormiston J, Serruys PW. Circumferential evaluation of the neointima by optical coherence tomography after ABSORB bioresorbable vascular scaffold implantation: can the scaffold cap the plaque? Atherosclerosis. 2012 Mar;221(1):106-12. doi: 10.1016/j.atherosclerosis.2011.12.008. Epub 2011 Dec 13.

title: Circumferential evaluation of the neointima by optical coherence tomography after ABSORB bioresorbable vascular scaffold implantation: Can the scaffold cap the plaque?

ReferenceCitation: Gutiérrez-Chico JL, Radu MD, Diletti R, Sheehy A, Kossuth MB, Oberhauser JP, Glauser T, Harrington J, Rapoza RJ, Onuma Y, Serruys PW. Spatial distribution and temporal evolution of scattering centers by optical coherence tomography in the poly(L-lactide) backbone of a bioresorbable vascular scaffold. Circ J. 2012;76(2):342-50. Epub 2011 Nov 19.

ReferenceCitation: Serruys PW, Onuma Y, Dudek D, Smits PC, Koolen J, Chevalier B, de Bruyne B, Thuesen L, McClean D, van Geuns RJ, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Sudhir K, Garcia-Garcia HM, Ormiston JA. Evaluation of the second generation of a bioresorbable everolimus-eluting vascular scaffold for the treatment of de novo coronary artery stenosis: 12-month clinical and imaging outcomes. J Am Coll Cardiol. 2011 Oct 4;58(15):1578-88. doi: 10.1016/j.jacc.2011.05.050.

title: Evaluation of the Second Generation of a Bioresorbable Everolimus-Eluting Vascular Scaffold for the Treatment of De Novo Coronary Artery Stenosis

ReferenceCitation: Gomez-Lara J, Radu M, Brugaletta S, Farooq V, Diletti R, Onuma Y, Windecker S, Thuesen L, McClean D, Koolen J, Whitbourn R, Dudek D, Smits PC, Regar E, Veldhof S, Rapoza R, Ormiston JA, Garcia-Garcia HM, Serruys PW. Serial analysis of the malapposed and uncovered struts of the new generation of everolimus-eluting bioresorbable scaffold with optical coherence tomography. JACC Cardiovasc Interv. 2011 Sep;4(9):992-1001. doi: 10.1016/j.jcin.2011.03.020.

title: Serial Analysis of the Malapposed and Uncovered Struts of the New Generation of Everolimus-Eluting Bioresorbable Scaffold With Optical Coherence Tomography

ReferenceCitation: Gomez-Lara J, Brugaletta S, Farooq V, van Geuns RJ, De Bruyne B, Windecker S, McClean D, Thuesen L, Dudek D, Koolen J, Whitbourn R, Smits PC, Chevalier B, Morel MA, Dorange C, Veldhof S, Rapoza R, Garcia-Garcia HM, Ormiston JA, Serruys PW. Angiographic geometric changes of the lumen arterial wall after bioresorbable vascular scaffolds and metallic platform stents at 1-year follow-up. JACC Cardiovasc Interv. 2011 Jul;4(7):789-99. doi: 10.1016/j.jcin.2011.04.009.

title: Angiographic Geometric Changes of the Lumen Arterial Wall After Bioresorbable Vascular Scaffolds and Metallic Platform Stents at 1-Year Follow-Up

ReferenceCitation: Gutiérrez-Chico JL, Serruys PW, Girasis C, Garg S, Onuma Y, Brugaletta S, García-García H, van Es GA, Regar E. Quantitative multi-modality imaging analysis of a fully bioresorbable stent: a head-to-head comparison between QCA, IVUS and OCT. Int J Cardiovasc Imaging. 2012 Mar;28(3):467-78. doi: 10.1007/s10554-011-9829-y. Epub 2011 Feb 26.

journal_name: The International Journal of Cardiovascular Imaging

repository_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

ReferenceCitation: Serruys PW, Onuma Y, Ormiston JA, de Bruyne B, Regar E, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Miquel-Hebert K, Rapoza R, García-García HM. Evaluation of the second generation of a bioresorbable everolimus drug-eluting vascular scaffold for treatment of de novo coronary artery stenosis: six-month clinical and imaging outcomes. Circulation. 2010 Nov 30;122(22):2301-12. doi: 10.1161/CIRCULATIONAHA.110.970772. Epub 2010 Nov 15.

name: From the ThoraxCenter, Erasmus Medical Center, Rotterdam, the Netherlands (P.W.S., Y.O., E.R., H.M.G.-G.); Cardiovascular Center, Aalst, Belgium (B.d.B.); Jagiellonian University, Krakow, Poland (D.D.); Skejby Sygehus, Aarhus University Hospital, Skejby, Denmark (L.T.); Maasstad Hospital, Rotterdam, the Netherlands (P.C.S.); Institut Jacques Cartier, Massy, France (B.C.); Christchurch Hospital, Christchurch, New Zealand (D.M.); Catharina Hospital, Eindhoven, the Netherlands (J.K.); Bern University...

title: Evaluation of the Second Generation of a Bioresorbable Everolimus Drug-Eluting Vascular Scaffold for Treatment of De Novo Coronary Artery Stenosis

ReferenceCitation: Gomez-Lara J, Garcia-Garcia HM, Onuma Y, Garg S, Regar E, De Bruyne B, Windecker S, McClean D, Thuesen L, Dudek D, Koolen J, Whitbourn R, Smits PC, Chevalier B, Dorange C, Veldhof S, Morel MA, de Vries T, Ormiston JA, Serruys PW. A comparison of the conformability of everolimus-eluting bioresorbable vascular scaffolds to metal platform coronary stents. JACC Cardiovasc Interv. 2010 Nov;3(11):1190-8. doi: 10.1016/j.jcin.2010.07.016.

title: A Comparison of the Conformability of Everolimus-Eluting Bioresorbable Vascular Scaffolds to Metal Platform Coronary Stents

ReferenceCitation: Okamura T, Onuma Y, García-García HM, Regar E, Wykrzykowska JJ, Koolen J, Thuesen L, Windecker S, Whitbourn R, McClean DR, Ormiston JA, Serruys PW; ABSORB Cohort B Investigators. 3-Dimensional optical coherence tomography assessment of jailed side branches by bioresorbable vascular scaffolds: a proposal for classification. JACC Cardiovasc Interv. 2010 Aug;3(8):836-44. doi: 10.1016/j.jcin.2010.05.011.

title: 3-Dimensional Optical Coherence Tomography Assessment of Jailed Side Branches by Bioresorbable Vascular Scaffolds

location_country:
Switzerland
Netherlands
France
Poland
Belgium
Australia
New Zealand
Denmark

location_facility:
St. Vincent's Hospital
Institut Hospitalier Jacques Cartier
Maasstad Ziekenhuis
Monash Heart
Auckland City Hospital
Catharina ZH Eindhoven
Inselspital Bern, Kardiologie
Christchurch Hospital
Erasmus Medical Center
Jagiellonian University
Skejby Sygehus
Onze-Lieve VrouweZiekenhuis


type: EudraCT Number

title: Clofarabine in Combination With a Standard Remission Induction Regimen (AraC and Idarubicin) in Patients 18-60 Years Old With Previously Untreated Intermediate and Bad Risk Acute Myelogenous Leukemia (AML) or High Risk Myelodysplasia (MDS) : a Phase I-II Study of the EORTC-LG and GIMEMA (AML-14A Trial)

summary:
RATIONALE: Drugs used in chemotherapy, such as clofarabine, cytarabine, and idarubicin, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells.

PURPOSE: This randomized phase I/II trial is studying the side effects and best dose of clofarabine and to see how well it works when given together with cytarabine and idarubicin in treating patients with intermediate-risk or high-risk acute myeloid leukemia or high-risk myelodysplasia.

status: Unknown status

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Anticipated

lead_sponsor: European Organisation for Research and Treatment of Cancer - EORTC

location_country: Italy Netherlands Croatia France Belgium

location_facility: Azienda Ospedallera Universitaria - Policlinico Tor Vergata

location_facility: Radboud University Nijmegen Medical Center

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: A Double-Blind, Multicenter Study Evaluating the Efficacy and Safety of One Fixed Dose of SR58611A (700 mg/Day) Versus Placebo and Paroxetine (20 mg/Day) in Patients With a Recurrent Major Depressive Episode.

summary:
Primary objective :

To demonstrate the antidepressant efficacy on the Hamilton Depression Rating Scale of SR58611A 700mg/day compared with placebo in the treatment of patients with a recurrent major depressive episode.

Secondary objectives:

To assess the safety profile of SR58611A 700 mg/d in comparison to placebo and to assess plasma concentrations of the active metabolite.

status: Completed

study_completion_date_type: Actual

enrollment_type: Actual

lead_sponsor: Sanofi

location_country:
Serbia
Netherlands
Croatia
Bulgaria
France
Montenegro
Estonia
Czech Republic

status_simplified: Completed


title: A Prospective Observational Registry on the Use of NovoSeven® (Activated Recombinant Human Factor VIIa) for on Demand Treatment of Mild to Moderate Bleeds in Haemophilia A and B Patients With Inhibitors

acronym: ONE

summary: This study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.

study_type: Observational

time_perspective: Prospective

lead_sponsor: Novo Nordisk A/S

location_country:
Italy
Austria
South Africa
Sweden
Saudi Arabia
United Kingdom
Netherlands
France
Poland
Venezuela
Belgium
Algeria
Portugal
Turkey
Germany
Czech Republic


title: A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (MDS), Including Acute Myeloid Leukaemia With 20-<30 Percent Marrow Blasts (Former RAEB-t), and Chronic Myelomonocytic Leukaemia (CMML)

acronym: EUMDS

summary:
Study Objectives:

To collect and describe demographics, disease-management, and treatment outcomes of Myelodysplastic Syndromes (MDS) patients who are newly diagnosed and classified according to the World Health Organization (WHO) criteria.

To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples, and to present relevant research outcomes in the fields of diagnosis and prognostication, health related quality of life issues, health economics, and risk stratification for newly developed classes of drugs.

To disseminate results of the studies to all stakeholders involved.

status: Recruiting

study_completion_date_type: Anticipated

ReferenceType: derived

ReferenceCitation: de Swart L, Crouch S, Hoeks M, Smith A, Langemeijer S, Fenaux P, Symeonidis A, Cermâk J, Hellström-Lindberg E, Stauder R, Sanz G, Mittelman M, Holm MS, Malcovati L, Mądry K, Germing U, Tatic A, Savic A, Almeida AM, Gredelj-Simec N, Guerci-Bresler A, Beyne-Rauzy O, Culligan D, Kotsianidis I, Itzykson R, van Marrewijk C, Blijlevens N, Bowen D, de Witte T; EUMDS Registry Participants. Impact of red blood cell transfusion dose density on progression-free survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2020 Mar;105(3):632-639. doi: 10.3324/haematol.2018.212217. Epub 2019 Jun 6.

family: de Swart

full_name: Louise de Swart

given: Louise

sequence: first

family: Crouch

full_name: Simon Crouch

given: Simon

sequence: additional

family: Hoeks

full_name: Marlijn Hoeks

given: Marlijn

family: Smith

full_name: Alex Smith

given: Alex

family: Langemeijer

full_name: Saskia Langemeijer

given: Saskia

family: Fenaux

full_name: Pierre Fenaux

given: Pierre

family: Symeonidis

full_name: Argiris Symeonidis

given: Argiris

family: Cermâk

full_name: Jaroslav Cermâk

given: Jaroslav

family: Hellström-Lindberg

full_name: Eva Hellström-Lindberg

given: Eva

family: Stauder

full_name: Reinhard Stauder

given: Reinhard

family: Sanz

full_name: Guillermo Sanz

given: Guillermo

family: Mittelman

full_name: Moshe Mittelman

given: Moshe

family: Holm

full_name: Mette Skov Holm

given: Mette Skov

family: Malcovati

full_name: Luca Malcovati

given: Luca

family: Mądry

full_name: Krzysztof Mądry

given: Krzysztof

family: Germing

full_name: Ulrich Germing

given: Ulrich

family: Tatic

full_name: Aurelia Tatic

given: Aurelia

family: Savic

full_name: Aleksandar Savic

given: Aleksandar

family: Almeida

full_name: Antonio Medina Almeida

given: Antonio Medina

family: Gredelj-Simec

full_name: Njetocka Gredelj-Simec

given: Njetocka

family: Guerci-Bresler

full_name: Agnes Guerci-Bresler

given: Agnes

family: Beyne-Rauzy

full_name: Odile Beyne-Rauzy

given: Odile

family: Culligan

full_name: Dominic Culligan

given: Dominic

family: Kotsianidis

name: on behalf of the EUMDS Registry Participants

publisher: Ferrata Storti Foundation (Haematologica)

publisher_dissemination: Ferrata Storti Foundation (Haematologica)

publisher_group: Ferrata Storti Foundation (Haematologica)

publisher_normalized: Ferrata Storti Foundation (Haematologica)

ReferenceCitation: Itzykson R, Crouch S, Travaglino E, Smith A, Symeonidis A, Hellström-Lindberg E, Sanz G, Čermák J, Stauder R, Elena C, Germing U, Mittelman M, Langemeijer S, Mądry K, Tatic A, Holm MS, Almeida AM, Savic A, Šimec NG, Luño E, Culligan D, Guerci-Bresler A, Malcovati L, van Marrewijk C, Bowen D, de Witte T, Fenaux P; European MDS Registry members. Early platelet count kinetics has prognostic value in lower-risk myelodysplastic syndromes. Blood Adv. 2018 Aug 28;2(16):2079-2089. doi: 10.1182/bloodadvances.2018020495.

name: Département d'Hématologie, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris and Université Paris Diderot, Paris, France;

name: Epidemiology and Cancer Statistics Group, Department of Health Sciences, University of York, York, United Kingdom;

name: Department of Hematology Oncology, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia, Italy;

name: Division Hematology, Department of Internal Medicine, University of Patras Medical School, Patras, Greece;

name: Division Hematology, Department of Medicine, Karolinska Institute, Stockholm, Sweden;

name: Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain;

name: Centro de Investigación Biomédica en Red Cáncer, Instituto Carlos III, Madrid, Spain;

name: Department of Clinical Hematology, Institute of Hematology and Blood Transfusion, Praha, Czech Republic;

name: Department of Internal Medicine V (Haematology and Oncology), Innsbruck Medical University, Innsbruck, Austria;

name: Department of Haematology, Oncology and Clinical Immunology, Universitätsklinik Düsseldorf, Düsseldorf, Germany;

name: Department of Medicine A, Tel Aviv Sourasky (Ichilov) Medical Center, Sackler Medical Faculty, Tel Aviv University, Tel Aviv, Israel;

name: Department of Hematology, Radboud University Medical Center, Nijmegen, The Netherlands;

name: Department of Haematology, Oncology and Internal Medicine, Warsaw Medical University, Warsaw, Poland;

name: Center of Hematology and Bone Marrow Transplantation, Fundeni Clinical Institute, Bucharest, Romania;

name: Department of Haematology, Aarhus University Hospital, Aarhus, Denmark;

name: Department of Hematology, Hospital da Luz, Lisbon, Portugal;

name: Clinic of Hematology–Clinical Center of Vojvodina, Faculty of Medicine, University of Novi Sad, Novi Sad, Serbia;

name: Division of Hematology, Department of Internal Medicine, Merkur University Hospital, Zagreb, Croatia;

name: Servicio d'Hematología. Hospital Universitario Central de Asturias, Oviedo, Spain;

name: Department of Haematology, Aberdeen Royal Infirmary, Aberdeen, United Kingdom;

name: Service d'Hématologie, Centre Hospitalier Universtaire Brabois Vandoeuvre, Nancy, France;

name: St. James's Institute of Oncology, Leeds Teaching Hospitals, Leeds, United Kingdom; and

name: Department of Tumor Immunology–Nijmegen Center for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands

repositories_institution: DANS - Data Archiving and Networked Services - NARCIS - National Academic Research and Collaborations Information System

ReferenceCitation: de Swart L, Reiniers C, Bagguley T, van Marrewijk C, Bowen D, Hellström-Lindberg E, Tatic A, Symeonidis A, Huls G, Cermak J, van de Loosdrecht AA, Garelius H, Culligan D, Macheta M, Spanoudakis M, Panagiotidis P, Krejci M, Blijlevens N, Langemeijer S, Droste J, Swinkels DW, Smith A, de Witte T; EUMDS Steering Committee. Labile plasma iron levels predict survival in patients with lower-risk myelodysplastic syndromes. Haematologica. 2018 Jan;103(1):69-79. doi: 10.3324/haematol.2017.171884. Epub 2017 Nov 9.

repository_institution: University of Groningen / Centre for Information Technology - University of Groningen research database

ReferenceCitation: Garelius HK, Johnston WT, Smith AG, Park S, de Swart L, Fenaux P, Symeonidis A, Sanz G, Čermák J, Stauder R, Malcovati L, Mittelman M, van de Loosdrecht AA, van Marrewijk CJ, Bowen D, Crouch S, de Witte TJ, Hellström-Lindberg E. Erythropoiesis-stimulating agents significantly delay the onset of a regular transfusion need in nontransfused patients with lower-risk myelodysplastic syndrome. J Intern Med. 2017 Mar;281(3):284-299. doi: 10.1111/joim.12579. Epub 2016 Dec 7.

name: Department of Medicine; Section of Hematology and Coagulation; Sahlgrenska University Hospital; Gothenburg Sweden

name: Epidemiology and Cancer Statistics Group; Department of Health Sciences; University of York; York UK

name: Clinique Universitaire d'hématologie; CHU de Grenoble; Université Grenoble; Grenoble France

name: Department of Hematology; Radboud university medical center; Nijmegen the Netherlands

name: Service d'Hématologie; Hôpital Saint-Louis; Assistance Publique des Hôpitaux de Paris (AP-HP) and Université Paris 7; Paris France

name: Department of Medicine; Division of Hematology; University of Patras Medical School; Patras Greece

name: Department of Hematology; Hospital Universitario y Politécnico La Fe; Valencia Spain

name: Department of Clinical Hematology; Institute of Hematology & Blood Transfusion; Praha Czech Republic

name: Department of Internal Medicine V (Hematology and Oncology); Innsbruck Medical University; Innsbruck Austria

name: Department of Hematology Oncology; Fondazione IRCCS Policlinico San Matteo; University of Pavia; Pavia Italy

name: Department of Medicine A; Tel Aviv Sourasky (Ichilov) Medical Center; Tel Aviv Israel

name: Department of Hematology; VU Institute of Cancer and Immunology; VU University Medical Center; Amsterdam the Netherlands

name: St. James's Institute of Oncology; Leeds Teaching Hospitals; Leeds UK

name: Department of Tumor Immunology; Nijmegen Center for Molecular Life Sciences; Radboud university medical center; Nijmegen the Netherlands

name: Department of Medicine; Division of Hematology; Karolinska Institutet; Stockholm Sweden

evidence: oa repository (via OAI-PMH title and first author match)

repository_institution: University of Leeds, The University of Sheffield, University of York - White Rose Research Online

ipd_sharing_description: The study results (aggregated data) will be published in an academic journal and presented at scientific meetings. We are working on an update of the website: www.eumds.org. EUMDS related activities and results are also shared via www.mds-europe.eu.

location_country:
Italy
Austria
Sweden
Serbia
Switzerland
Netherlands
Spain
Croatia
Czechia
France
Greece
Israel
Poland
Romania
Portugal
Denmark
Germany
United Kingdom

location_facility: Hopital Avicenne Universite Paris, Dept of Hematology

location_facility: University of Pavia Medical SChool, Dept of Hematology

location_facility: Radboud University Nijmegen Medical Centre, dept of Hematology

location_facility: Leeds General Infirmary Dept of Hematology

location_facility: Institute of Haematology and Blood Transfusion U nemocnice

location_facility: Tel-Aviv Sourasky Medical Center, Dept. of Medicine A

location_facility: Medizinische Klinik und Poliklinik fur Haematologie, Onkologie und Klinische Immunologie

location_facility: Instituto Português de Oncologia de Lisboa,

location_facility: University of Patras Medical School, Haematology Division, Dept of Internal Medicine

location_facility: Karolinsky Institute Huddinge University Hospital, Dept of Medicine Division HAematology

location_facility: Fundeni CLinical Institute, Clinic of Hematology

location_facility: Medical University, Dept of Hematology, Oncology and Internal Medicine

location_facility: University of Innsbruck Fachartz fur Innere Medizin, Dept of Hematology


title: CONTRAST (Can cONTrast Injection Better Approximate FFR compAred to Pure reSTing Physiology?)

acronym: CONTRAST

summary: The purpose of this study is to determine the diagnostic performances of iodine contrast medium and resting conditions to predict fractional flow reserve (FFR). Reference FFR will be measured using standard adenosine. We hypothesize that contrast FFR will offer superior diagnostic agreement compared to resting conditions.

ReferenceType: background

ReferenceCitation: De Bruyne B, Pijls NH, Barbato E, Bartunek J, Bech JW, Wijns W, Heyndrickx GR. Intracoronary and intravenous adenosine 5'-triphosphate, adenosine, papaverine, and contrast medium to assess fractional flow reserve in humans. Circulation. 2003 Apr 15;107(14):1877-83. Epub 2003 Mar 31.

ReferenceType: result

ReferenceCitation: Johnson NP, Jeremias A, Zimmermann FM, Adjedj J, Witt N, Hennigan B, Koo BK, Maehara A, Matsumura M, Barbato E, Esposito G, Trimarco B, Rioufol G, Park SJ, Yang HM, Baptista SB, Chrysant GS, Leone AM, Berry C, De Bruyne B, Gould KL, Kirkeeide RL, Oldroyd KG, Pijls NHJ, Fearon WF. Continuum of Vasodilator Stress From Rest to Contrast Medium to Adenosine Hyperemia for Fractional Flow Reserve Assessment. JACC Cardiovasc Interv. 2016 Apr 25;9(8):757-767. doi: 10.1016/j.jcin.2015.12.273.

family: Johnson

full_name: Nils P. Johnson

given: Nils P.

family: Jeremias

full_name: Allen Jeremias

given: Allen

family: Zimmermann

full_name: Frederik M. Zimmermann

given: Frederik M.

family: Adjedj

full_name: Julien Adjedj

given: Julien

family: Witt

full_name: Nils Witt

given: Nils

family: Hennigan

full_name: Barry Hennigan

given: Barry

family: Koo

full_name: Bon-Kwon Koo

given: Bon-Kwon

family: Maehara

full_name: Akiko Maehara

given: Akiko

family: Matsumura

full_name: Mitsuaki Matsumura

given: Mitsuaki

family: Barbato

full_name: Emanuele Barbato

given: Emanuele

family: Esposito

full_name: Giovanni Esposito

given: Giovanni

family: Trimarco

full_name: Bruno Trimarco

given: Bruno

family: Rioufol

full_name: Gilles Rioufol

given: Gilles

family: Park

full_name: Seung-Jung Park

given: Seung-Jung

family: Yang

full_name: Hyoung-Mo Yang

given: Hyoung-Mo

family: Baptista

full_name: Sérgio B. Baptista

given: Sérgio B.

family: Chrysant

full_name: George S. Chrysant

given: George S.

family: Leone

full_name: Antonio M. Leone

given: Antonio M.

family: Berry

full_name: Colin Berry

given: Colin

family: De Bruyne

full_name: Bernard De Bruyne

given: Bernard

family: Gould

full_name: K. Lance Gould

given: K. Lance

family: Kirkeeide

full_name: Richard L. Kirkeeide

given: Richard L.

family: Oldroyd

full_name: Keith G. Oldroyd

given: Keith G.

repository_institution: Hospital Prof. Dr. Fernando Fonseca - The Institutional Repository of Hospital Prof. Dr. Fernando Fonseca

repository_institution: The University of Glasgow - Enlighten: Publications

ReferenceCitation: Johnson NP, Matsuo H, Nakayama M, Eftekhari A, Kakuta T, Tanaka N, Christiansen EH, Kirkeeide RL, Gould KL. Combined Pressure and Flow Measurements to Guide Treatment of Coronary Stenoses. JACC Cardiovasc Interv. 2021 Sep 13;14(17):1904-1913. doi: 10.1016/j.jcin.2021.07.041.

ReferenceCitation: Johnson NP, Collet C. Can FFR After Stenting Help Reduce Target Vessel Failure? JACC Cardiovasc Interv. 2021 Sep 13;14(17):1901-1903. doi: 10.1016/j.jcin.2021.08.001.

ReferenceCitation: Gould KL, Johnson NP, Roby AE, Nguyen T, Kirkeeide R, Haynie M, Lai D, Zhu H, Patel MB, Smalling R, Arain S, Balan P, Nguyen T, Estrera A, Sdringola S, Madjid M, Nascimbene A, Loyalka P, Kar B, Gregoric I, Safi H, McPherson D. Regional, Artery-Specific Thresholds of Quantitative Myocardial Perfusion by PET Associated with Reduced Myocardial Infarction and Death After Revascularization in Stable Coronary Artery Disease. J Nucl Med. 2019 Mar;60(3):410-417. doi: 10.2967/jnumed.118.211953. Epub 2018 Aug 16.

ReferenceCitation: Johnson NP, Kirkeeide RL, Gould KL. Same Lesion, Different Artery, Different FFR!? JACC Cardiovasc Imaging. 2019 Apr;12(4):718-721. doi: 10.1016/j.jcmg.2017.11.029. Epub 2018 Jan 17.

ReferenceCitation: Kobayashi Y, Johnson NP, Zimmermann FM, Witt N, Berry C, Jeremias A, Koo BK, Esposito G, Rioufol G, Park SJ, Nishi T, Choi DH, Oldroyd KG, Barbato E, Pijls NHJ, De Bruyne B, Fearon WF; CONTRAST Study Investigators. Agreement of the Resting Distal to Aortic Coronary Pressure With the Instantaneous Wave-Free Ratio. J Am Coll Cardiol. 2017 Oct 24;70(17):2105-2113. doi: 10.1016/j.jacc.2017.08.049.

ReferenceCitation: Nishi T, Johnson NP, De Bruyne B, Berry C, Gould KL, Jeremias A, Oldroyd KG, Kobayashi Y, Choi DH, Pijls NHJ, Fearon WF; CONTRAST Study Investigators. Influence of Contrast Media Dose and Osmolality on the Diagnostic Performance of Contrast Fractional Flow Reserve. Circ Cardiovasc Interv. 2017 Oct;10(10). pii: e004985. doi: 10.1161/CIRCINTERVENTIONS.117.004985.

name: From the Stanford University Medical Center, CA (T.N., Y.K., D.-H.C., W.F.F.); Stanford Cardiovascular Institute, CA (T.N., Y.K., D.-H.C., W.F.F.); University of Texas Medical School and Memorial Hermann Hospital, Houston (N.P.J., K.L.G.); Cardiovascular Center Aalst, Belgium (B.D.B.); West of Scotland Heart and Lung Center, Golden Jubilee National Hospital, Clydebank, Scotland (C.B., K.G.O.); British Heart Foundation Glasgow Cardiovascular Research Center, Institute of Cardiovascular and Medical...

ReferenceCitation: Matsumura M, Johnson NP, Fearon WF, Mintz GS, Stone GW, Oldroyd KG, De Bruyne B, Pijls NHJ, Maehara A, Jeremias A. Accuracy of Fractional Flow Reserve Measurements in Clinical Practice: Observations From a Core Laboratory Analysis. JACC Cardiovasc Interv. 2017 Jul 24;10(14):1392-1401. doi: 10.1016/j.jcin.2017.03.031.

ReferenceCitation: Gargiulo G, Stabile E, Ferrone M, Barbato E, Zimmermann FM, Adjedj J, Hennigan B, Matsumura M, Johnson NP, Fearon WF, Jeremias A, Trimarco B, Esposito G; CONTRST Study Investigators. Diabetes does not impact the diagnostic performance of contrast-based fractional flow reserve: insights from the CONTRAST study. Cardiovasc Diabetol. 2017 Jan 13;16(1):7. doi: 10.1186/s12933-016-0494-2.

name: On behalf of the CONTRST Study Investigators

ReferenceCitation: Johnson NP, Gould KL, Di Carli MF, Taqueti VR. Invasive FFR and Noninvasive CFR in the Evaluation of Ischemia: What Is the Future? J Am Coll Cardiol. 2016 Jun 14;67(23):2772-2788. doi: 10.1016/j.jacc.2016.03.584. Review.

ReferenceCitation: Gould KL, Johnson NP. Coronary Blood Flow After Acute MI: Alternative Truths. JACC Cardiovasc Interv. 2016 Mar 28;9(6):614-7. doi: 10.1016/j.jcin.2016.02.009.

title: Coronary Blood Flow After Acute MI

ReferenceCitation: Gould KL, Johnson NP. Myocardial Bridges: Lessons in Clinical Coronary Pathophysiology. JACC Cardiovasc Imaging. 2015 Jun;8(6):705-9. doi: 10.1016/j.jcmg.2015.02.013.

ReferenceCitation: Gould KL, Johnson NP, Kaul S, Kirkeeide RL, Mintz GS, Rentrop KP, Sdringola S, Virmani R, Narula J. Patient selection for elective revascularization to reduce myocardial infarction and mortality: new lessons from randomized trials, coronary physiology, and statistics. Circ Cardiovasc Imaging. 2015 May;8(5). pii: e003099. doi: 10.1161/CIRCIMAGING.114.003099. Review.

name: From the Division of Cardiology, Department of Medicine, Weatherhead PET Center for Preventing and Reversing Atherosclerosis, University of Texas Medical School at Houston and Memorial Hermann Hospital (K.L.G., N.P.J., R.L.K., S.S.); Division of Cardiology, Cedars-Sinai Medical Center, Los Angeles, CA (S.K.); Cardiovascular Research Foundation, New York, NY (G.S.M.); Department of Medicine, Cardiovascular Disease, New York Presbyterian Hospital, The University Hospital of Columbia and Cornell, New...

title: Patient Selection for Elective Revascularization to Reduce Myocardial Infarction and Mortality

lead_sponsor: The University of Texas Health Science Center, Houston

location_country:
Italy
Sweden
Netherlands
France
Belgium
Portugal
United States
United Kingdom
Korea, Republic of

location_facility: Seoul National University College of Medicine


title: Long-term Follow-up of a Multicentre, Randomized, Double-blind, Placebo-controlled Trial (AN004T) Assessing the Efficacy and Tolerability of 2 Dosing Regimens of AllerT in Adult Subjects Allergic to Birch Pollen

summary: Study aiming to evaluate the efficacy of a 2-month pre-seasonal treatment with an AllerT 50 µg or 100 µg maintenance dose administered in previous study AN004T in reducing symptoms of allergic rhinoconjunctivitis during the 2nd following birch pollen season.

lead_sponsor: Anergis

location_country: Sweden Switzerland Poland France Lithuania Denmark Latvia

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: Center of examination and treatment of allergic diseases

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Centre Hospitalier Universitaire Vaudois


title: Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia

summary: The proposed natural history study will enroll male and female patients, ages 36 months and younger, who have a diagnosis of XLHED based on genetic testing and who have not received an investigational study drug. The study protocol will include collection of all relevant medical history and documentation of clinical outcomes using age-appropriate, minimally invasive technologies. Data will be collected both retrospectively, back to pregnancy assessments that may be available, and prospectively through age 5 yrs.

study_start_date_type: Actual

time_perspective: Other

lead_sponsor: Edimer Pharmaceuticals

location_country:
Italy
United States
France
Germany
United Kingdom

location_facility: Azienda Ospedaliera-Polo Universitario "Luigi Sacco"

location_facility: Washington University School of Medicine


title: A Phase Ib/II Study of LEE011 in Combination With Fulvestrant and BYL719 or BKM120 in the Treatment of Postmenopausal Women With Hormone Receptor Positive, HER2 Negative Locally Recurrent or Advanced Metastatic Breast Cancer

summary:
The purpose of this trial is to explore the clinical utility of the three investigational agents in HR+, HER2- breast cancer. LEE011 (CDK4/6 inhibitor), BKM120 (PI3K-pan class I-inhibitor) and BYL719 (PI3K-alpha specific class I inhibitor) in combination with fulvestrant.

This is a multi-center, open-label Phase Ib/II study. The Phase Ib portion of the study is a dose escalation to estimate the MTD and/or RP2D for three regimens: LEE011 with fulvestrant; LEE011 and BKM120 with fulvestrant; LEE011and BYL719 with fulvestrant.

The Phase II portion of the study was planned to be a randomized study to assess the anti-tumor activity as well as safety and tolerability of LEE011 with fulvestrant to LEE011 and BKM120 with fulvestrant, and LEE011 and BYL719 with fulvestrant in patients with ER+/HER2- locally advanced or metastatic breast cancer.

Approximately 216 adult women with ER+/HER2- locally advanced or metastatic breast cancer were planned to be enrolled.

design_allocation: Non-Randomized

ReferenceCitation: Tolaney SM, Im YH, Calvo E, Lu YS, Hamilton E, Forero-Torres A, Bachelot T, Maur M, Fasolo A, Tiedt R, Nardi L, Stammberger U, Abdelhady AM, Ruan S, Lee SC. Phase Ib Study of Ribociclib plus Fulvestrant and Ribociclib plus Fulvestrant plus PI3K Inhibitor (Alpelisib or Buparlisib) for HR(+) Advanced Breast Cancer. Clin Cancer Res. 2021 Jan 15;27(2):418-428. doi: 10.1158/1078-0432.CCR-20-0645. Epub 2020 Sep 4.

family: Tolaney

full_name: Sara M. Tolaney

given: Sara M.

family: Im

full_name: Young-Hyuck Im

given: Young-Hyuck

family: Calvo

full_name: Emiliano Calvo

given: Emiliano

family: Lu

full_name: Yen-Shen Lu

given: Yen-Shen

family: Hamilton

full_name: Erika Hamilton

given: Erika

family: Forero-Torres

full_name: Andres Forero-Torres

given: Andres

family: Bachelot

full_name: Thomas Bachelot

given: Thomas

family: Maur

full_name: Michela Maur

given: Michela

family: Fasolo

full_name: Angelica Fasolo

given: Angelica

family: Tiedt

full_name: Ralph Tiedt

given: Ralph

family: Nardi

full_name: Lisa Nardi

given: Lisa

family: Stammberger

full_name: Uz Stammberger

given: Uz

family: Abdelhady

full_name: Ahmed M. Abdelhady

given: Ahmed M.

family: Ruan

full_name: Shiling Ruan

given: Shiling

family: Lee

full_name: Soo Chin Lee

given: Soo Chin

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Clinical Cancer Research

publisher: American Association for Cancer Research (AACR)

title: Phase Ib Study of Ribociclib plus Fulvestrant and Ribociclib plus Fulvestrant plus PI3K Inhibitor (Alpelisib or Buparlisib) for HR+ Advanced Breast Cancer

ipd_sharing: Undecided

ipd_sharing_description:
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
France
Singapore
Spain
United States
United Kingdom
Korea, Republic of
Taiwan

location_facility: Sarah Cannon Research Institute Onc Dept

location_facility: University of Alabama at Birmingham/ Kirklin Clinic Dept Onc


title: A Phase I Open-label, Dose Escalation Study to Investigate The Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK2879552 Given Orally in Subjects With Relapsed/Refractory Small Cell Lung Carcinoma

summary: GSK2879552 is a potent, selective, mechanism-based inactivator of Lysine Specific Demethylase 1 (LSD1)/ CoRepressor for Element-1-Silencing Transcription factor (CoREST) activity. This is a phase I, open-label, multi-center, non-randomized, 2-part first time in human (FTIH) study for GSK2879552. Part 1 is a dose escalation phase to determine the recommended phase 2 dose (RP2D) for GSK2879552 based on the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) profiles observed after oral administration of GSK2879552. Any dose level(s) may be expanded up to 12 subjects in order to collect additional data on PK and PD.The safety and PK/PD data will be reviewed prior to the dose decision, and the dose escalation will be guided by the Neuenschwander -continuous reassessment method (N-CRM). Built-in safety constraints are in place to prevent exposing subjects to undue risk of toxicity. Once RP2D is identified, an expansion cohort (Part 2) of up to 30 subjects will be enrolled to further evaluate the clinical activity and tolerability of GSK2879552 in subjects with relapsed/refractory SCLC.

status: Terminated

ReferenceCitation: Bauer TM, Besse B, Martinez-Marti A, Trigo JM, Moreno V, Garrido P, Ferron-Brady G, Wu Y, Park J, Collingwood T, Kruger RG, Mohammad HP, Ballas MS, Dhar A, Govindan R. Phase I, Open-Label, Dose-Escalation Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of GSK2879552 in Relapsed/Refractory SCLC. J Thorac Oncol. 2019 Oct;14(10):1828-1838. doi: 10.1016/j.jtho.2019.06.021. Epub 2019 Jun 28.

ipd_sharing: Yes

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: GlaxoSmithKline

location_country:
United States
France
Spain


type: Other Identifier

title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

summary:
The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa.

The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

status: Active, not recruiting

ReferenceCitation: Thurberg BL, Diaz GA, Lachmann RH, Schiano T, Wasserstein MP, Ji AJ, Zaher A, Peterschmitt MJ. Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. Mol Genet Metab. 2020 Sep - Oct;131(1-2):245-252. doi: 10.1016/j.ymgme.2020.06.010. Epub 2020 Jun 24.

family: Thurberg

full_name: Beth L. Thurberg

given: Beth L.

family: Diaz

full_name: George A. Diaz

given: George A.

family: Lachmann

full_name: Robin H. Lachmann

given: Robin H.

family: Schiano

full_name: Thomas Schiano

family: Wasserstein

full_name: Melissa P. Wasserstein

given: Melissa P.

family: Ji

full_name: Allena J. Ji

given: Allena J.

family: Zaher

full_name: Atef Zaher

given: Atef

family: Peterschmitt

full_name: M. Judith Peterschmitt

given: M. Judith

journal_name: Molecular Genetics and Metabolism

oa_host_type: publisher

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

ReferenceCitation: Wasserstein MP, Diaz GA, Lachmann RH, Jouvin MH, Nandy I, Ji AJ, Puga AC. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months. J Inherit Metab Dis. 2018 Sep;41(5):829-838. doi: 10.1007/s10545-017-0123-6. Epub 2018 Jan 5.

family: Jouvin

full_name: Marie-Hélène Jouvin

given: Marie-Hélène

family: Nandy

full_name: Indrani Nandy

given: Indrani

family: Puga

full_name: Ana Cristina Puga

given: Ana Cristina

journal_name: Journal of Inherited Metabolic Disease

oa_host_type: repository

evidence: oa repository (via OAI-PMH doi match)

host_type: repository

license: cc-by

license_normalized: cc-by

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

location_country:
Italy
Brazil
Germany
France
Belgium
United States
United Kingdom

location_facility:
Investigational Site Number 826002
Investigational Site Number 840001
Investigational Site Number 380002
Investigational Site Number 380001
Investigational Site Number 250002
Investigational Site Number 076001
Investigational Site Number 056001
Investigational Site Number 826001
Investigational Site Number 276002


title: An Open-Label, Multi-Center, Dose-Escalation, Phase I Study With an Expansion Phase, to Evaluate Safety, Pharmacokinetics and Therapeutic Activity of RO6895882, an Immunocytokine, Consisting of a Variant of Interleukin-2 (IL-2v) Targeting Carcinoembryonic Antigen (CEA) Administered Intravenously, in Patients With Advanced and/or Metastatic Solid Tumors

summary: This open-label, multi-center, dose-escalation study will evaluate the safety, pharmacokinetics, and therapeutic activity of RO6895882 in participants with Carcinoembryonic Antigen (CEA)-positive solid tumors who have progressed on the standard of care therapy. The study will be conducted in 3 parts. Part 1 will be a single ascending dose study in single participant cohort at low RO6895882 dose (less than or equal to [</=] 6 milligrams [mg]). Part 2 will be a dose-escalation study of RO6895882 monotherapy given every week (qw), every 2 weeks (q2w), and possibly every 3 weeks (q3w). Part 3 will be an expansion phase of the qw, q2w, and possibly q3w at maximum tolerated dose (MTD) (as determined in Part 2). Part 3 will only be conducted if the risk/benefit assessment, as evaluated by the Sponsor and the investigators, is in favor of the participants. Participants will be treated until disease progression, unacceptable toxicity or withdrawal from treatment for other reasons or death for a maximum duration of 24 months.

lead_sponsor: Hoffmann-La Roche

location_country:
Switzerland
Netherlands
Finland
France
Spain
Denmark
United States
United Kingdom

location_facility: Oxford University Hospitals NHS Trust - Churchill Hospital

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Clinica Universitaria de Navarra; Servicio de oncología

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: VU MEDISCH CENTRUM; Dept. of Medical Oncology

location_facility: Helsinki University Central Hospital; Dept of Oncology


title: Multicenter International Study of Oxaliplatin/ 5FU-LV in the Adjuvant Treatment of Colon Cancer

summary:
To evaluate the FOLFOX regimen versus LV5FU2 in the adjuvant treatment of stage II and III colon cancer.

Primary objective: Disease Free Survival (DFS) Secondary objective: Overall Survival (OS), safety (including long term toxicity)

ReferenceCitation: André T, Boni C, Mounedji-Boudiaf L, Navarro M, Tabernero J, Hickish T, Topham C, Zaninelli M, Clingan P, Bridgewater J, Tabah-Fisch I, de Gramont A; Multicenter International Study of Oxaliplatin/5-Fluorouracil/Leucovorin in the Adjuvant Treatment of Colon Cancer (MOSAIC) Investigators. Oxaliplatin, fluorouracil, and leucovorin as adjuvant treatment for colon cancer. N Engl J Med. 2004 Jun 3;350(23):2343-51.

ReferenceCitation: Cohen R, Taieb J, Fiskum J, Yothers G, Goldberg R, Yoshino T, Alberts S, Allegra C, de Gramont A, Seitz JF, O'Connell M, Haller D, Wolmark N, Erlichman C, Zaniboni A, Lonardi S, Kerr R, Grothey A, Sinicrope FA, André T, Shi Q. Microsatellite Instability in Patients With Stage III Colon Cancer Receiving Fluoropyrimidine With or Without Oxaliplatin: An ACCENT Pooled Analysis of 12 Adjuvant Trials. J Clin Oncol. 2021 Feb 20;39(6):642-651. doi: 10.1200/JCO.20.01600. Epub 2020 Dec 23.

name: Sorbonne Université, Department of Medical Oncology, Saint-Antoine Hospital, AP-HP, Paris, France

name: Department of Health Science Research, Mayo Clinic, Rochester, MN

family: Cohen

full_name: Romain Cohen

given: Romain

name: Sorbonne Paris Cité, Paris Descartes University Georges Pompidou European Hospital, Paris, France

family: Taieb

full_name: Julien Taieb

given: Julien

family: Fiskum

full_name: Jack Fiskum

given: Jack

name: Department of Biostatistics, University of Pittsburgh, Pittsburgh, PA

family: Yothers

full_name: Greg Yothers

given: Greg

name: WVU Cancer Institute, West Virginia University, Morgantown, WV

family: Goldberg

full_name: Richard Goldberg

given: Richard

name: Department of Gastrointestinal Oncology, National Cancer Center Hospital East, Kashiwa, Japan

family: Yoshino

full_name: Takayuki Yoshino

given: Takayuki

name: Department of Oncology, Mayo Clinic, Rochester, MN

family: Alberts

full_name: Steven Alberts

given: Steven

name: Department of Medicine and University of Florida Shands Cancer Center, FL

family: Allegra

full_name: Carmen Allegra

given: Carmen

name: Department of Medical Oncology, Franco-British Institute, Levallois-Perret, France

family: de Gramont

full_name: Aimery de Gramont

given: Aimery

family: Seitz

full_name: Jean-Francois Seitz

given: Jean-Francois

family: O'Connell

full_name: Michael O'Connell

given: Michael

name: Division of Hematology/Oncology, University of Pennsylvania, Philadelphia, PA

family: Haller

full_name: Daniel Haller

given: Daniel

family: Wolmark

full_name: Norman Wolmark

given: Norman

family: Erlichman

full_name: Charles Erlichman

given: Charles

name: Medical Oncology Unit, Fondazione Poliambulanza, Brescia, Italy

family: Zaniboni

full_name: Alberto Zaniboni

given: Alberto

name: Department of Oncology, Veneto Institute of Oncology IOV - IRCCS, Padova, Italy

family: Lonardi

full_name: Sara Lonardi

given: Sara

name: University of Oxford, Oxford, United Kingdom

family: Kerr

full_name: Rachel Kerr

given: Rachel

family: Grothey

full_name: Axel Grothey

given: Axel

family: Sinicrope

full_name: Frank A. Sinicrope

given: Frank A.

family: André

full_name: Thierry André

given: Thierry

family: Shi

full_name: Qian Shi

given: Qian

journal_name: Journal of Clinical Oncology

publisher: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Salem ME, Yin J, Goldberg RM, Pederson LD, Wolmark N, Alberts SR, Taieb J, Marshall JL, Lonardi S, Yoshino T, Kerr RS, Yothers G, Grothey A, Andre T, De Gramont A, Shi Q. Evaluation of the change of outcomes over a 10-year period in patients with stage III colon cancer: pooled analysis of 6501 patients treated with fluorouracil, leucovorin, and oxaliplatin in the ACCENT database. Ann Oncol. 2020 Apr;31(4):480-486. doi: 10.1016/j.annonc.2019.12.007. Epub 2020 Jan 16.

family: Salem

full_name: M.E. Salem

ReferenceCitation: Auclin E, André T, Taieb J, Banzi M, Van Laethem JL, Tabernero J, Hickish T, de Gramont A, Vernerey D. Association of post-operative CEA with survival and oxaliplatin benefit in patients with stage II colon cancer: a post hoc analysis of the MOSAIC trial. Br J Cancer. 2019 Aug;121(4):312-317. doi: 10.1038/s41416-019-0521-7. Epub 2019 Jul 12.

ReferenceCitation: André T, de Gramont A, Vernerey D, Chibaudel B, Bonnetain F, Tijeras-Raballand A, Scriva A, Hickish T, Tabernero J, Van Laethem JL, Banzi M, Maartense E, Shmueli E, Carlsson GU, Scheithauer W, Papamichael D, Möehler M, Landolfi S, Demetter P, Colote S, Tournigand C, Louvet C, Duval A, Fléjou JF, de Gramont A. Adjuvant Fluorouracil, Leucovorin, and Oxaliplatin in Stage II to III Colon Cancer: Updated 10-Year Survival and Outcomes According to BRAF Mutation and Mismatch Repair Status of the MOSAIC Study. J Clin Oncol. 2015 Dec 10;33(35):4176-87. doi: 10.1200/JCO.2015.63.4238. Epub 2015 Nov 2.

name: Thierry André and Jean-François Fléjou, Hôpital St Antoine; Thierry André and Jean-François Fléjou, University Pierre et Marie Curie Paris VI; Thierry André, Benoist Chibaudel, Annemilaï Tijeras-Raballand, Soudhir Colote, and Aimery de Gramont, Groupe Coopérateur Multdisciplinaire en Ocologie (GERCOR) Oncology Multidisciplinary Group and GERCOR-Innovative Research Consortium; Christophe Louvet, Institut Mutualiste Montsouris; Alex Duval, L'Institut National de la Santé et de la Recherche Médicale UMRS...

ReferenceCitation: Tournigand C, André T, Bonnetain F, Chibaudel B, Lledo G, Hickish T, Tabernero J, Boni C, Bachet JB, Teixeira L, de Gramont A. Adjuvant therapy with fluorouracil and oxaliplatin in stage II and elderly patients (between ages 70 and 75 years) with colon cancer: subgroup analyses of the Multicenter International Study of Oxaliplatin, Fluorouracil, and Leucovorin in the Adjuvant Treatment of Colon Cancer trial. J Clin Oncol. 2012 Sep 20;30(27):3353-60. doi: 10.1200/JCO.2012.42.5645. Epub 2012 Aug 20.

name: Christophe Tournigand, Thierry André, Benoist Chibaudel, Luis Teixeira, Aimery de Gramont, Hôpital Saint-Antoine, Paris, France; Christophe Tournigand, Thierry André, Benoist Chibaudel, Jean-Baptiste Bachet, Luis Teixeira, Aimery de Gramont, Université Pierre et Marie Curie, Paris, France; Christophe Tournigand, Thierry André, Franck Bonnetain, Benoist Chibaudel, Gérard Lledo, Luis Teixeira, Aimery de Gramont, Groupe Coopérateur Multidisciplinaire en Oncologie, Paris, France; Christophe Tournigand,...

title: Adjuvant Therapy With Fluorouracil and Oxaliplatin in Stage II and Elderly Patients (between ages 70 and 75 years) With Colon Cancer: Subgroup Analyses of the Multicenter International Study of Oxaliplatin, Fluorouracil, and Leucovorin in the Adjuvant Treatment of Colon Cancer Trial

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Spain
France
Poland
Greece
Israel
Norway
Hungary
Australia
Belgium
Singapore
Portugal
Denmark
Germany
United Kingdom

location_facility: sanofi-aventis Australia & New Zealand administrative office


title: EURopean Trial In Atrial Fibrillation or Flutter Patients Receiving Dronedarone for the maIntenance of Sinus Rhythm (EURIDIS)

acronym: EURIDIS

summary:
To assess the efficacy of dronedarone versus placebo for the maintenance of normal sinus rhythm after electrical, pharmacological or spontaneous conversion of atrial fibrillation/atrial flutter (AF/AFL).

To assess the efficacy of dronedarone versus placebo on AF/AFL-related symptoms.

To assess the efficacy of dronedarone versus placebo on ventricular rate control in case of AF/AFL recurrence.

ReferenceCitation: Singh BN, Connolly SJ, Crijns HJ, Roy D, Kowey PR, Capucci A, Radzik D, Aliot EM, Hohnloser SH; EURIDIS and ADONIS Investigators. Dronedarone for maintenance of sinus rhythm in atrial fibrillation or flutter. N Engl J Med. 2007 Sep 6;357(10):987-99.

location_country:
Italy
United Kingdom
Netherlands
Finland
France
Poland
Hungary
Belgium
Spain
Denmark
Germany
Czech Republic

location_facility:
Sanofi- Aventis Administrative Office
Sanofi-Aventis Administrative Office


location_country:
Austria
Germany
France
Japan
United States
Taiwan

source: euctr

title: A Randomized, Double-blind, Placebo Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients With Bullous Pemphigoid With Disease Refractory to Oral Steroid Treatment

summary: To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.


title: Prognostic Factors and Therapeutic Effects of Surgical Treatment for Traumatic Spinal Column Injury With Spinal Cord Injury: A Prospective, Observational European Multi-center Study.

acronym: SCI-POEM

summary: Despite many years of research, an incomprehensible amount of scientific efforts worldwide and billions of dollars invested, no effective therapy resulting in major neurological or functional recovery is available to date for traumatic spinal cord injury (tSCI). Although there is increasing experimental evidence from animal models that surgical decompression of the spinal cord improves recovery after tSCI, clinical studies have not shown conclusive data yet. The main explanations for this lack of convincing evidence are relatively small sample sizes in previous studies, their predominantly retrospective nature, suboptimal measurement methods for the assessment of neurological deficits, and inappropriate recording and documentation of potential confounding factors.

lead_sponsor: AOSpine Europe

location_country:
Italy
Austria
Serbia
Netherlands
Croatia
Bulgaria
France
Romania
Ireland
Turkey
Germany
United Kingdom

location_facility: National Institute of Emergency Medicine "N.I. Pirogov"

intervention_type: Procedure


title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Biological Activity of SAR405838 in Patients With Advanced Cancer

summary:
Primary Objectives:

To determine safety and the maximum tolerated dose (MTD) of SAR405838 through the characterization of dose-limiting toxicities (DLTs).
To assess biological activities in patients with dedifferentiated liposarcoma during MTD cohort expansion.

Secondary Objectives:

Pharmacokinetic (PK) profile of SAR405838.
Biomarkers in association with SAR405838.
Anti-tumor activity in response to SAR405838.
Food effect on SAR405838 PK.
Compliance with SAR405838 treatment.
Cytochrome P450 3A4/5 (CYP3A4/5) activity.

ReferenceCitation: de Jonge M, de Weger VA, Dickson MA, Langenberg M, Le Cesne A, Wagner AJ, Hsu K, Zheng W, Macé S, Tuffal G, Thomas K, Schellens JH. A phase I study of SAR405838, a novel human double minute 2 (HDM2) antagonist, in patients with solid tumours. Eur J Cancer. 2017 May;76:144-151. doi: 10.1016/j.ejca.2017.02.005. Epub 2017 Mar 17.

family: de Jonge

full_name: Maja de Jonge

given: Maja

family: de Weger

full_name: Vincent A. de Weger

given: Vincent A.

family: Dickson

full_name: Mark A. Dickson

given: Mark A.

family: Langenberg

full_name: Marlies Langenberg

given: Marlies

family: Le Cesne

full_name: Axel Le Cesne

family: Wagner

full_name: Andrew J. Wagner

given: Andrew J.

family: Hsu

full_name: Karl Hsu

given: Karl

family: Zheng

full_name: Wei Zheng

given: Wei

family: Macé

full_name: Sandrine Macé

given: Sandrine

family: Tuffal

full_name: Gilles Tuffal

given: Gilles

family: Thomas

full_name: Koruth Thomas

given: Koruth

family: Schellens

full_name: Jan H.M. Schellens

given: Jan H.M.

journal_name: European Journal of Cancer

location_country:
United States
France
Netherlands

location_facility:
Investigational Site Number 840001
Investigational Site Number 528003
Investigational Site Number 840101
Investigational Site Number 528002
Investigational Site Number 840002
Investigational Site Number 250001
Investigational Site Number 528001


ReferenceCitation: Brogan PA, Hofer M, Kuemmerle-Deschner JB, Koné-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penadés I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9.

name: UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust London UK

family: Brogan

full_name: Paul A. Brogan

given: Paul A.

name: Centre Hospitalier Universitaire Vaudois, University of Lausanne, Lausanne, Switzerland, and Hôpitaux Universitaires de Genève Geneva Switzerland

family: Hofer

full_name: Michael Hofer

given: Michael

name: University Hospital Tuebingen Tuebingen Germany

family: Kuemmerle‐Deschner

full_name: Jasmin B. Kuemmerle‐Deschner

given: Jasmin B.

name: CeRéMAIA AP‐HP Bicêtre Hospital University of Paris Sud Le Kremlin‐Bicêtre France

family: Koné‐Paut

full_name: Isabelle Koné‐Paut

given: Isabelle

name: Universitätsklinikum Carl Gustav Carus Dresden Dresden Germany

family: Roesler

full_name: Joachim Roesler

given: Joachim

name: Charité University Berlin Berlin Germany

family: Kallinich

full_name: Tilmann Kallinich

given: Tilmann

name: Asklepios Klinik Sankt Augustin GmbH, St. Augustin, Germany, and University of Cologne Cologne Germany

family: Horneff

full_name: Gerd Horneff

given: Gerd

name: Hospital Universitario y Politécnico La Fe Valencia Spain

family: Calvo Penadés

full_name: Inmaculada Calvo Penadés

given: Inmaculada

family: Sevilla‐Perez

full_name: Belén Sevilla‐Perez

given: Belén

name: Hôpital Universitaire Des Enfants Reine FabiolaUniversité Libre de Bruxelles Brussels Belgium

family: Goffin

full_name: Laurence Goffin

given: Laurence

name: Cliniques Universitaires Saint‐Luc and Université Catholique de Louvain Brussels Belgium

family: Lauwerys

full_name: Bernard R. Lauwerys

given: Bernard R.

name: University College London Royal Free Campus London UK

family: Lachmann

full_name: Helen J. Lachmann

given: Helen J.

name: Meir Medical CenterSackler School of MedicineTel Aviv University Tel Aviv Israel

family: Uziel

full_name: Yosef Uziel

given: Yosef

name: China Novartis Institutes for Biomedical Research Company, Ltd Beijing China

family: Wei

full_name: Xiaoling Wei

given: Xiaoling

name: University of Toronto and the Hospital for Sick Children Toronto Ontario Canada

family: Laxer

full_name: Ronald M. Laxer

given: Ronald M.

journal_name: Arthritis & Rheumatology

oa_colors: green hybrid

oa_host_type: publisher;repository

license_normalized: no license

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

repository_institution: Université de Lausanne - SERVAL

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

title: Rapid and Sustained Long‐Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin‐Associated Periodic Syndrome Ages Five Years and Younger

location_country:
Switzerland
France
Belgium
Spain
Germany
United Kingdom
Canada

title: An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)

summary: This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study


title: A Multi-centre, Randomised, Double-blind, Parallel Group, Placebo Controlled Trial to Evaluate the Efficacy and Safety of Activated Recombinant Factor VII (rFVIIa/NovoSeven®/ Niastase®) in the Treatment of Bleeding in Severely Injured Trauma Subjects

summary: This trial is conducted in Africa, Asia, Europe, Oceania and North America. The aim of this trial is to evaluate the efficacy of activated recombinant human factor VII given in conjunction with standard therapy in the treatment of massive bleeding in subjects with severe blunt and/or penetrating trauma injury.

ReferenceCitation: Rossaint R, Christensen MC, Choong PI, Boffard KD, Riou B, Rizoli S, Kluger Y, Lefering R, Morris S; NovoSeven® Trauma Study Group. Cost-Effectiveness of Recombinant Activated Factor VII as Adjunctive Therapy for Bleeding Control in Severely Injured Trauma Patients in Germany. Eur J Trauma Emerg Surg. 2007 Oct;33(5):528-38. doi: 10.1007/s00068-007-6210-x. Epub 2007 Jul 20.

family: Rossaint

full_name: Rolf Rossaint

given: Rolf

name: on behalf of the NovoSeven® Trauma Study Group

family: Christensen

full_name: Michael C. Christensen

given: Michael C.

family: Choong

full_name: Philip Iau Tsau Choong

given: Philip Iau Tsau

family: Boffard

full_name: Kenneth D. Boffard

given: Kenneth D.

family: Riou

full_name: Bruno Riou

given: Bruno

family: Rizoli

full_name: Sandro Rizoli

given: Sandro

family: Kluger

full_name: Yoram Kluger

given: Yoram

family: Lefering

full_name: Rolf Lefering

family: Morris

full_name: Stephen Morris

given: Stephen

journal_name: European Journal of Trauma and Emergency Surgery

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer

publisher_group: Springer

publisher_normalized: Springer

ReferenceCitation: Boffard KD, Riou B, Warren B, Choong PI, Rizoli S, Rossaint R, Axelsen M, Kluger Y; NovoSeven Trauma Study Group. Recombinant factor VIIa as adjunctive therapy for bleeding control in severely injured trauma patients: two parallel randomized, placebo-controlled, double-blind clinical trials. J Trauma. 2005 Jul;59(1):8-15; discussion 15-8.

ReferenceCitation: Rizoli SB, Boffard KD, Riou B, Warren B, Iau P, Kluger Y, Rossaint R, Tillinger M; NovoSeven Trauma Study Group. Recombinant activated factor VII as an adjunctive therapy for bleeding control in severe trauma patients with coagulopathy: subgroup analysis from two randomized trials. Crit Care. 2006;10(6):R178.

ReferenceCitation: Morris S, Ridley S, Munro V, Christensen MC. Cost effectiveness of recombinant activated factor VII for the control of bleeding in patients with severe blunt trauma injuries in the United Kingdom. Anaesthesia. 2007 Jan;62(1):43-52.

ReferenceCitation: Champion HR, Fingerhut A, Escobar MA, Weiskopf RB. The role of data and safety monitoring in acute trauma resuscitation research. J Am Coll Surg. 2007 Jan;204(1):73-83.

ReferenceCitation: Klitgaard T, Tabanera y Palacios R, Boffard KD, Iau PT, Warren B, Rizoli S, Rossaint R, Kluger Y, Riou B; NovoSeven Trauma Study Group. Pharmacokinetics of recombinant activated factor VII in trauma patients with severe bleeding. Crit Care. 2006;10(4):R104.

ReferenceCitation: Kluger Y, Riou B, Rossaint R, Rizoli SB, Boffard KD, Choong PI, Warren B, Tillinger M. Safety of rFVIIa in hemodynamically unstable polytrauma patients with traumatic brain injury: post hoc analysis of 30 patients from a prospective, randomized, placebo-controlled, double-blind clinical trial. Crit Care. 2007;11(4):R85.

ReferenceCitation: Hsia CC, Chin-Yee IH, McAlister VC. Use of recombinant activated factor VII in patients without hemophilia: a meta-analysis of randomized control trials. Ann Surg. 2008 Jul;248(1):61-8. doi: 10.1097/SLA.0b013e318176c4ec.

family: Hsia

full_name: Cyrus C. Hsia

given: Cyrus C.

family: Chin-Yee

full_name: Ian H. Chin-Yee

given: Ian H.

family: McAlister

full_name: Vivian C. McAlister

given: Vivian C.

journal_name: Annals of Surgery

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: Use of Recombinant Activated Factor VII in Patients Without Hemophilia

ReferenceCitation: Levy JH, Fingerhut A, Brott T, Langbakke IH, Erhardtsen E, Porte RJ. Recombinant factor VIIa in patients with coagulopathy secondary to anticoagulant therapy, cirrhosis, or severe traumatic injury: review of safety profile. Transfusion. 2006 Jun;46(6):919-33.

location_country:
Austria
South Africa
Germany
France
Israel
Singapore
Australia
Canada
United Kingdom


title: Observational Post-marketing Study on the Safety and Efficacy of Dotarem® (SECURE Study)

summary: The main purpose of this study is to prospectively assess the general safety profile of Dotarem

lead_sponsor: Guerbet

location_country:
Italy
Austria
India
Saudi Arabia
Spain
France
Argentina
China
Germany
United Kingdom

location_facility: Bombay Hospital Institute of Medical Sciences


summary: This study will evaluate the safety and efficacy of an intravitreal implant of dexamethasone for the treatment of diabetic macular edema.

ReferenceCitation: Rittiphairoj T, Mir TA, Li T, Virgili G. Intravitreal steroids for macular edema in diabetes. Cochrane Database Syst Rev. 2020 Nov 17;11:CD005656. doi: 10.1002/14651858.CD005656.pub3.

name: Department of Epidemiology; Johns Hopkins Bloomberg School of Public Health; Baltimore Maryland USA

family: Rittiphairoj

full_name: Thanitsara Rittiphairoj

given: Thanitsara

name: Wilmer Eye Institute; Johns Hopkins University School of Medicine; Baltimore Maryland USA

family: Mir

full_name: Tahreem A Mir

given: Tahreem A

name: Department of Ophthalmology; University of Colorado Denver Anschutz Medical Campus; Aurora CO USA

family: Li

full_name: Tianjing Li

given: Tianjing

name: Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA); University of Florence; Florence Italy

name: Centre for Public Health; Queen's University Belfast; Belfast UK

family: Virgili

full_name: Gianni Virgili

given: Gianni

journal_name: Cochrane Database of Systematic Reviews

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Yoon YH, Boyer DS, Maturi RK, Bandello F, Belfort R Jr, Augustin AJ, Li XY, Bai Z, Hashad Y; Ozurdex MEAD Study Group. Natural history of diabetic macular edema and factors predicting outcomes in sham-treated patients (MEAD study). Graefes Arch Clin Exp Ophthalmol. 2019 Dec;257(12):2639-2653. doi: 10.1007/s00417-019-04464-2. Epub 2019 Oct 25.

family: Yoon

full_name: Young Hee Yoon

given: Young Hee

name: on behalf of the Ozurdex MEAD Study Group

family: Boyer

full_name: David S. Boyer

given: David S.

family: Maturi

full_name: Raj K. Maturi

given: Raj K.

family: Bandello

full_name: Francesco Bandello

given: Francesco

family: Belfort

full_name: Rubens Belfort

given: Rubens

suffix: Jr

family: Augustin

full_name: Albert J. Augustin

given: Albert J.

full_name: Xiao-Yan Li

given: Xiao-Yan

family: Bai

full_name: Zhanying Bai

given: Zhanying

family: Hashad

full_name: Yehia Hashad

given: Yehia

journal_name: Graefe's Archive for Clinical and Experimental Ophthalmology

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

ReferenceCitation: Danis RP, Sadda S, Li XY, Cui H, Hashad Y, Whitcup SM. Anatomical effects of dexamethasone intravitreal implant in diabetic macular oedema: a pooled analysis of 3-year phase III trials. Br J Ophthalmol. 2016 Jun;100(6):796-801. doi: 10.1136/bjophthalmol-2015-306823. Epub 2015 Nov 18.

family: Danis

full_name: Ronald P Danis

given: Ronald P

family: Sadda

full_name: Srinivas Sadda

given: Srinivas

family: Cui

full_name: Harry Cui

repository_institution: University of Oxford - Oxford Academic Notes & Queries

ReferenceCitation: Augustin AJ, Kuppermann BD, Lanzetta P, Loewenstein A, Li XY, Cui H, Hashad Y, Whitcup SM; Ozurdex MEAD Study Group. Dexamethasone intravitreal implant in previously treated patients with diabetic macular edema: subgroup analysis of the MEAD study. BMC Ophthalmol. 2015 Oct 30;15:150. doi: 10.1186/s12886-015-0148-2.

repository_normalized: University of Melbourne - Minerva Access

repository_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

repository_institution: University of Udine - Institutional Research Information System

repository_institution: University of California - eScholarship University of California

location_country:
Italy
Brazil
India
France
Korea, Republic of
Poland
Singapore
Hungary
New Zealand
United States
United Kingdom
Canada
Taiwan
Colombia


name: UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust London UK

family: Brogan

full_name: Paul A. Brogan

given: Paul A.

name: Centre Hospitalier Universitaire Vaudois, University of Lausanne, Lausanne, Switzerland, and Hôpitaux Universitaires de Genève Geneva Switzerland

family: Hofer

full_name: Michael Hofer

given: Michael

name: University Hospital Tuebingen Tuebingen Germany

family: Kuemmerle‐Deschner

full_name: Jasmin B. Kuemmerle‐Deschner

given: Jasmin B.

name: CeRéMAIA AP‐HP Bicêtre Hospital University of Paris Sud Le Kremlin‐Bicêtre France

family: Koné‐Paut

full_name: Isabelle Koné‐Paut

given: Isabelle

name: Universitätsklinikum Carl Gustav Carus Dresden Dresden Germany

family: Roesler

full_name: Joachim Roesler

given: Joachim

family: Kallinich

full_name: Tilmann Kallinich

given: Tilmann

family: Horneff

full_name: Gerd Horneff

given: Gerd

family: Calvo Penadés

full_name: Inmaculada Calvo Penadés

given: Inmaculada

family: Sevilla‐Perez

full_name: Belén Sevilla‐Perez

given: Belén

family: Goffin

full_name: Laurence Goffin

given: Laurence

family: Lauwerys

full_name: Bernard R. Lauwerys

given: Bernard R.

family: Lachmann

full_name: Helen J. Lachmann

given: Helen J.

family: Uziel

full_name: Yosef Uziel

given: Yosef

family: Wei

full_name: Xiaoling Wei

given: Xiaoling

family: Laxer

full_name: Ronald M. Laxer

given: Ronald M.

repositories_institution: University College London - UCL Discovery

repositories_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

title: A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS)

summary: This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)


title: Early Therapy of Atrial Fibrillation for Stroke Prevention Trial (EAST).

acronym: EAST

summary:
EAST prospectively tests the hypothesis that an early, structured rhythm control therapy based on antiarrhythmic drugs and catheter ablation can prevent atrial fibrillation (AF) related complications in patients with AF when compared to usual care.

Patients will be randomized to early therapy or usual care. In the early therapy group, patients will receive either catheter ablation (usually by pulmonary vein isolation), or adequate antiarrhythmic drug therapy at an early time point. The initial therapy will be selected by the local investigator. Upon AF recurrence, both modalities will be combined.

Usual care will be conducted following the 2010European Society of Cardiology ( ESC )guidelines for AF treatment. Early rhythm control therapy will be guided by Electrocardiogram (ECG) monitoring.

primary_purpose: Prevention

ReferenceCitation: International Landmark EAST Study Inverstigates Treatment Options for Atrial Fibrillation

ReferenceCitation: Willems S, Borof K, Brandes A, Breithardt G, Camm AJ, Crijns HJGM, Eckardt L, Gessler N, Goette A, Haegeli LM, Heidbuchel H, Kautzner J, Ng GA, Schnabel RB, Suling A, Szumowski L, Themistoclakis S, Vardas P, van Gelder IC, Wegscheider K, Kirchhof P. Systematic, early rhythm control strategy for atrial fibrillation in patients with or without symptoms: the EAST-AFNET 4 trial. Eur Heart J. 2021 Aug 27. pii: ehab593. doi: 10.1093/eurheartj/ehab593. [Epub ahead of print]

name: Asklepios Hospital St. Georg, Department of Cardiology and Internal intensive care medicine, Faculty of Medicine, Semmelweis University Campus Hamburg, Hamburg, Germany

name: DZHK (German Center for Cardiovascular Research), Partner Site Hamburg/Kiel/Luebeck, Berlin, Germany

family: Willems

full_name: Stephan Willems

given: Stephan

name: Department of Cardiology, University Heart and Vascular Center, University Medical Center Hamburg, Martinistraße 52, Hamburg 20246, Germany

family: Borof

full_name: Katrin Borof

given: Katrin

family: Brandes

full_name: Axel Brandes

given: Axel

family: Breithardt

full_name: Günter Breithardt

given: Günter

name: Cardiology Clinical Academic Group, Molecular and Clinical Sciences Research Institute, St. George’s University of London, UK

family: Camm

full_name: A John Camm

given: A John

name: Department of Cardiology, Maastricht University Medical Center and Cardiovascular Research Institute Maastricht, Netherlands

family: Crijns

full_name: Harry J G M Crijns

given: Harry J G M

family: Eckardt

full_name: Lars Eckardt

given: Lars

family: Gessler

full_name: Nele Gessler

given: Nele

family: Goette

full_name: Andreas Goette

given: Andreas

family: Haegeli

full_name: Laurent M Haegeli

given: Laurent M

name: University Hospital Antwerp and Antwerp University, Antwerp, Belgium

family: Heidbuchel

full_name: Hein Heidbuchel

given: Hein

name: Institute for Clinical and Experimental Medicine, Prague, Czech Republic

family: Kautzner

full_name: Josef Kautzner

given: Josef

name: Department of Cardiovascular Sciences, University of Leicester, National Institute for Health Research Leicester Biomedical Research Centre, Glenfield Hospital, Leicester, UK

family: Ng

full_name: G André Ng

given: G André

family: Schnabel

full_name: Renate B Schnabel

given: Renate B

name: Institute of Medical Biometry and Epidemiology, University Medical Center Hamburg, Eppendorf, Germany

family: Suling

full_name: Anna Suling

given: Anna

name: Arrhythmia Center of the National Institute of Cardiology, Medical Division of Cardinal Stefan Wyszynski University in Warsaw, Warsaw, Poland

family: Szumowski

full_name: Lukasz Szumowski

given: Lukasz

name: Department of Cardiology, Ospedale dell’Angelo, Venice, Italy

family: Themistoclakis

full_name: Sakis Themistoclakis

given: Sakis

name: Heart Sector, Hygeia Hospitals Group, Athens, Greece

family: Vardas

full_name: Panos Vardas

given: Panos

name: University of Groningen, University Medical Center Groningen, Groningen, Netherlands

family: van Gelder

full_name: Isabelle C van Gelder

given: Isabelle C

family: Wegscheider

full_name: Karl Wegscheider

given: Karl

family: Kirchhof

full_name: Paulus Kirchhof

given: Paulus

journal_name: European Heart Journal

repository_institution: St George's University of London - St George's Online Research Archive

ReferenceCitation: Rillig A, Magnussen C, Ozga AK, Suling A, Brandes A, Breithardt G, Camm AJ, Crijns HJGM, Eckardt L, Elvan A, Goette A, Gulizia M, Haegeli L, Heidbuchel H, Kuck KH, Ng A, Szumowski L, van Gelder I, Wegscheider K, Kirchhof P. Early Rhythm Control Therapy in Patients With Atrial Fibrillation and Heart Failure. Circulation. 2021 Sep 14;144(11):845-858. doi: 10.1161/CIRCULATIONAHA.121.056323. Epub 2021 Jul 30.

name: Department of Cardiology, Odense University Hospital, Denmark; Department of Clinical Research, University of Southern Denmark, Odense, Denmark

name: Atrial Fibrillation Network (AFNET), Münster, Germany; Department of Cardiology II (Electrophysiology), University Hospital Münster, Germany

name: Cardiology Clinical Academic Group, Molecular and Clinical Sciences Research Institute, St. George's University of London, UK

name: Isala Hospital and Diagram Research, Zwolle, The Netherlands

name: Atrial Fibrillation Network (AFNET), Münster, Germany; St. Vincenz Hospital, Paderborn, Germany; Working Group of Molecular Electrophysiology, University Hospital Magdeburg, Germany

name: University Hospital Zurich, Zurich, Switzerland; Division of Cardiology, Medical University Department, Kantonsspital Aarau, Switzerland

name: German Center for Cardiovascular Research, Partner Site Hamburg/Luebeck/Kiel, Germany; Atrial Fibrillation Network (AFNET), Münster, Germany; LANS Cardio, Hamburg, Germany

name: Arrhythmia Center of the National Institute of Cardiology; Medical Division of Cardinal Stefan Wyszynski University in Warsaw, Warsaw, Poland

name: German Center for Cardiovascular Research, Partner Site Hamburg/Luebeck/Kiel, Germany; Institute of Medical Biometry and Epidemiology, University Medical Center Hamburg-Eppendorf, Germany; Atrial Fibrillation Network (AFNET), Münster, Germany

name: Department of Cardiology, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Germany; German Center for Cardiovascular Research, Partner Site Hamburg/Luebeck/Kiel, Germany; Atrial Fibrillation Network (AFNET), Münster, Germany;  Institute of Cardiovascular Sciences, University of Birmingham, Birmingham, UK

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

repositories:
PubMed Central
irua
openaccess.sgul.ac.uk

title: Early Rhythm Control Therapy in Patients with Atrial Fibrillation and Heart Failure

ReferenceCitation: Kirchhof P, Camm AJ, Goette A, Brandes A, Eckardt L, Elvan A, Fetsch T, van Gelder IC, Haase D, Haegeli LM, Hamann F, Heidbüchel H, Hindricks G, Kautzner J, Kuck KH, Mont L, Ng GA, Rekosz J, Schoen N, Schotten U, Suling A, Taggeselle J, Themistoclakis S, Vettorazzi E, Vardas P, Wegscheider K, Willems S, Crijns HJGM, Breithardt G; EAST-AFNET 4 Trial Investigators. Early Rhythm-Control Therapy in Patients with Atrial Fibrillation. N Engl J Med. 2020 Oct 1;383(14):1305-1316. doi: 10.1056/NEJMoa2019422. Epub 2020 Aug 29.

name: From the Department of Cardiology, University Heart and Vascular Center (P.K.), and Institute of Medical Biometry and Epidemiology (A.S., E.V., K.W.), University Medical Center Hamburg–Eppendorf, LANS Cardio (K.-H.K.), and the Department of Cardiology, Asklepios Klinik St. Georg (S.W.), Hamburg, Atrial Fibrillation Network (AFNET) (P.K., A.G., L.E., T.F., D.H., K.-H.K., N.S., U.S., J.T., K.W., S.W., G.B.) and the Department of Cardiology II (Electrophysiology), University Hospital Münster (L.E., G.B.),...

repository_institution: University of Birmingham - University of Birmingham Research Portal

repository_institution: University of Southern Denmark - University of Southern Denmark Research Portal

repositories_institution: University of Groningen / Centre for Information Technology - University of Groningen research database

ReferenceCitation: Kirchhof P, Breithardt G, Camm AJ, Crijns HJ, Kuck KH, Vardas P, Wegscheider K. Improving outcomes in patients with atrial fibrillation: rationale and design of the Early treatment of Atrial fibrillation for Stroke prevention Trial. Am Heart J. 2013 Sep;166(3):442-8. doi: 10.1016/j.ahj.2013.05.015. Epub 2013 Jul 30.

title: Improving outcomes in patients with atrial fibrillation: Rationale and design of the Early treatment of Atrial fibrillation for Stroke prevention Trial

ipd_sharing_description:
We will share all data that support published results of the trial. Additional data that have not been published will be withheld until six months after publication of these results.

Data will be made available as required for specific, approved analyses. Data will be provided from locked, cleaned and de-identified study database. Requests will be reviewed by AFNET as per the predefined subanalysis plan.

location_country:
Italy
Switzerland
Netherlands
France
Czechia
Poland
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility:
14 Sites
13 Sites
10 Sites
51 Sites
12 Sites
2 Sites
22 Sites
5 Sites
4 Sites


title: Patient Reported Outcomes in Chronic Myeloid Leukemia. GIMEMA QoL - CML0310.

summary:
Although the impact of disease and treatment related burden on health-related quality of life (HRQOL) in patients with solid tumours has been well studied, with several clinical trials that included HRQOL as an endpoint, the general understanding in patients with Chronic Myeloid Leukaemia (CML) is lacking in comparison.

The literature shows that patients' perspective is unique and should always be measured with methodologically sound instruments that are devised for this purpose.

The main scope of this project is develop to an international validated questionnaire for the purpose of HRQOL assessment; such a tool will then be used to provide important data, from the patients' perspective, to make more informed treatment decisions.

time_perspective: Cross-Sectional

ReferenceCitation: Efficace F, Baccarani M, Breccia M, Saussele S, Abel G, Caocci G, Guilhot F, Cocks K, Naeem A, Sprangers M, Oerlemans S, Chie W, Castagnetti F, Bombaci F, Sharf G, Cardoni A, Noens L, Pallua S, Salvucci M, Nicolatou-Galitis O, Rosti G, Mandelli F. International development of an EORTC questionnaire for assessing health-related quality of life in chronic myeloid leukemia patients: the EORTC QLQ-CML24. Qual Life Res. 2014 Apr;23(3):825-36. doi: 10.1007/s11136-013-0523-5. Epub 2013 Sep 13.

lead_sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country:
Italy
Austria
Netherlands
Germany
France
Greece
Iraq
Belgium
United States
Taiwan

location_facility: Policlinico S. Orsola - Malpighi, Università di Bologna

location_facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori- IRST

location_facility: Ematologia - Sapienza Università di Roma

location_facility: Dana-Farber Cancer Institute Harvard University

location_facility: Centre Hospitalier Universitaire of Poitiers

intervention_type: Other


title: Phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy

summary: This is a phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients with Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy to determine the antitumor activity of Zalypsis.

lead_sponsor: PharmaMar

location_country:
Italy
United States
France

location_facility:
Istituto Clinico Humanitas
St. Jude Children 's Research Hospital
Centre Léon Bérard
Istituto Ortopedici Rizzoli
Istituto Nazionale dei Tumori
Sarcoma Oncology Center
Seattle Cancer Care Alliance


title: Prospective Observational Study on NovoSeven® Room Temperature Stable (VII25) in Patients With Haemophilia A or B

summary: This study is conducted in Europe and Asia. The aim of this observational study is to monitor antibody formation towards the room temperature stable formulation of NovoSeven® (activated recombinant human factor VII).

location_country:
Italy
Austria
Sweden
France
Poland
Slovakia
Hungary
Argentina
Belgium
Ireland
Portugal
Germany
United Kingdom
Iran, Islamic Republic of


title: A Randomized, Controlled Study to Evaluate the Safety and Effectiveness of EVICEL as an Adjunct to Sutured Dural Repair

summary: The objective of this study is to evaluate the safety and efficacy of EVICEL for use as an adjunct to dura sutures in elective cranial surgery to provide intraoperative watertight closure.

lead_sponsor: Ethicon, Inc.

location_country:
Netherlands
Finland
France
Belgium
Germany
United Kingdom

location_facility: Department of Neurosurgery, Ziekenhuis Oost Limburg

location_facility: Department of Neurosurgery, University Giessen-Marburg

location_facility: Department of Neurosurgery, Ninewells Hospital & Medical School

location_facility: Department of Neurosurgery, University Hospital of Liège

location_facility: Klinik für Neurochirurgie, Universitätsklinikum Essen

location_facility: Service de Neurochirurgie B, Hopital Neurologique de Lyon

location_facility: Department of Neurosciences and Rehabilitation, Tampere University Hospital

location_facility: Department of Neurosurgery, Klinikum Ingolstadt GmbH

location_facility: Department of Neurosurgery University Clinics of Schleswig-Holstein Campus Kiel

location_facility: Department of Neurosurgery, John Radcliffe Hospital

location_facility: Edinburgh Centre for Neuro-Oncology, Western General Hospital


title: A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy

summary: The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.

ReferenceCitation: Voit T, Topaloglu H, Straub V, Muntoni F, Deconinck N, Campion G, De Kimpe SJ, Eagle M, Guglieri M, Hood S, Liefaard L, Lourbakos A, Morgan A, Nakielny J, Quarcoo N, Ricotti V, Rolfe K, Servais L, Wardell C, Wilson R, Wright P, Kraus JE. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014 Oct;13(10):987-96. doi: 10.1016/S1474-4422(14)70195-4. Epub 2014 Sep 7.

family: Voit

full_name: Thomas Voit

given: Thomas

family: Topaloglu

full_name: Haluk Topaloglu

given: Haluk

family: Straub

full_name: Volker Straub

given: Volker

family: Muntoni

full_name: Francesco Muntoni

given: Francesco

family: Deconinck

full_name: Nicolas Deconinck

given: Nicolas

family: Campion

full_name: Giles Campion

given: Giles

family: De Kimpe

full_name: Sjef J De Kimpe

given: Sjef J

family: Eagle

full_name: Michelle Eagle

given: Michelle

family: Guglieri

full_name: Michela Guglieri

given: Michela

family: Hood

full_name: Steve Hood

given: Steve

family: Liefaard

full_name: Lia Liefaard

given: Lia

family: Lourbakos

full_name: Afrodite Lourbakos

given: Afrodite

family: Morgan

full_name: Allison Morgan

given: Allison

family: Nakielny

full_name: Joanna Nakielny

given: Joanna

family: Quarcoo

full_name: Naashika Quarcoo

given: Naashika

family: Ricotti

full_name: Valeria Ricotti

given: Valeria

family: Rolfe

full_name: Katie Rolfe

given: Katie

family: Servais

full_name: Laurent Servais

given: Laurent

family: Wardell

full_name: Claire Wardell

given: Claire

family: Wilson

full_name: Rosamund Wilson

given: Rosamund

family: Wright

full_name: Padraig Wright

given: Padraig

family: Kraus

full_name: John E Kraus

given: John E

journal_name: The Lancet Neurology

location_country:
Netherlands
France
Israel
Belgium
Australia
Spain
Turkey
Germany
United Kingdom


summary: This study will evaluate the safety and efficacy of intraprostatic administration of botulinum toxin Type A (BOTOX®) compared with placebo to treat urinary tract symptoms due to benign prostatic hyperplasia.

ReferenceCitation: McVary KT, Roehrborn CG, Chartier-Kastler E, Efros M, Bugarin D, Chen R, Patel A, Haag-Molkenteller C. A multicenter, randomized, double-blind, placebo controlled study of onabotulinumtoxinA 200 U to treat lower urinary tract symptoms in men with benign prostatic hyperplasia. J Urol. 2014 Jul;192(1):150-6. doi: 10.1016/j.juro.2014.02.004. Epub 2014 Feb 7. Erratum in: J Urol. 2015 Jan;193(1):374.

lead_sponsor: Allergan

location_country:
United States
Philippines
France
Czechia
Korea, Republic of
Poland
Germany
Canada


title: An Investigator-initiated Study on rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors - European Initiative to Prevent Joint Damage in Hemophilia A Children With Inhibitors

acronym: ENJOIH

summary: The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.

lead_sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

location_country:
Italy
United States
France
Romania
Spain
Germany

location_facility: Unitat Hemofilia, Hospital Vall d'Hebron

location_facility: Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center

location_facility: Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento

location_facility: Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy

location_facility: National Institute for Transfusional Hematology

location_facility: Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital

location_facility: Klinikum der Johann Wolfgang Goethe-Universitat

location_facility: Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco

location_facility: Centro de Hemofilia, Hospital Universitario La Paz

location_facility: Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe