type: U.S. NIH Grant/Contract

title: Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea Therapy in the Treatment of High Risk Polycythemia Vera (PV) and High Risk Essential Thrombocythemia (ET)

summary:
This research is looking at two conditions, Essential Thrombocythemia (ET) and Polycythemia Vera (PV). ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red blood cells to be made. Platelets are particles which circulate in the blood stream and normally prevent bleeding and bruising. Having too many platelets in the blood increases the risk of developing blood clots, which can result in life threatening events like heart attacks and strokes. When the number of red blood cells is increased in PV this will slow the speed of blood flow in the body and increases the risk of developing blood clots.

The purpose of this study is to look at the effectiveness of giving participants who have been diagnosed with ET or PV one of two different study regimens over time. The study subject will be followed for their condition for about 5 years. The subject will be randomized into one of two study regimens, either Pegylated Interferon Alfa-2a (PEGASYS) or Aspirin and Hydroxyurea (also called Hydroxycarbamide). The subject must be newly diagnosed or already receiving treatment for either PV or ET. Each of the study drugs used in this study is already being used to treat subjects with ET or PV currently, but the investigators are unsure which study drug is better.

ReferenceCitation: Yacoub A, Mascarenhas J, Kosiorek H, Prchal JT, Berenzon D, Baer MR, Ritchie E, Silver RT, Kessler C, Winton E, Finazzi MC, Rambaldi A, Vannucchi AM, Leibowitz D, Rondelli D, Arcasoy MO, Catchatourian R, Vadakara J, Rosti V, Hexner E, Kremyanskaya M, Sandy L, Tripodi J, Najfeld V, Farnoud N, Papaemmanuil E, Salama M, Singer-Weinberg R, Rampal R, Goldberg JD, Barbui T, Mesa R, Dueck AC, Hoffman R. Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea. Blood. 2019 Oct 31;134(18):1498-1509. doi: 10.1182/blood.2019000428.

name: Division of Hematologic Malignancies and Cellular Therapeutics, The University of Kansas Cancer Center, Westwood, KS;

family: Yacoub

full_name: Abdulraheem Yacoub

given: Abdulraheem

name: Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY;

family: Mascarenhas

full_name: John Mascarenhas

given: John

family: Kosiorek

full_name: Heidi Kosiorek

given: Heidi

name: Division of Hematology, University of Utah School of Medicine and

name: Huntsman Cancer Center, Salt Lake City, UT;

family: Prchal

full_name: Josef T. Prchal

given: Josef T.

name: Comprehensive Cancer Center, Wake Forest University Medical Center, Wake Forest Health, Winston-Salem, NC;

family: Berenzon

full_name: Dmitry Berenzon

given: Dmitry

name: Greenebaum Comprehensive Cancer Center, University of Maryland, Baltimore, MD;

family: Baer

full_name: Maria R. Baer

given: Maria R.

name: Division of Hematology and Oncology, Department of Medicine, Richard T. Silver Myeloproliferative Neoplasms Center, Weill Cornell Medical College, New York, NY;

family: Ritchie

full_name: Ellen Ritchie

given: Ellen

family: Silver

full_name: Richard T. Silver

given: Richard T.

name: Georgetown University Medical Center, Washington, DC;

family: Kessler

full_name: Craig Kessler

given: Craig

name: Winship Cancer Institute, Emory University School of Medicine, Atlanta, GA;

family: Winton

full_name: Elliott Winton

given: Elliott

name: Department of Hematology, Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Bergamo, Italy;

family: Finazzi

full_name: Maria Chiara Finazzi

given: Maria Chiara

name: Department of Oncology, University of Milan, Ospedale Papa Giovanni XXIII, Bergamo, Italy;

family: Rambaldi

full_name: Alessandro Rambaldi

name: Center for Research and Innovation of Myeloproliferative Neoplasms, Azienda Ospedaliero-Universitaria Careggi, and

name: Denothe Excellence Center, University of Florence, Florence, Italy;

family: Vannucchi

full_name: Alessandro M. Vannucchi

given: Alessandro M.

name: Department of Oncology, Palo Alto Medical Foundation, Sutter Health, Palo Alto, CA;

family: Leibowitz

full_name: David Leibowitz

given: David

name: Division of Hematology and Oncology, University of Illinois, Chicago, IL;

family: Rondelli

full_name: Damiano Rondelli

given: Damiano

name: Division of Hematology, Duke University School of Medicine, Durham, NC;

family: Arcasoy

full_name: Murat O. Arcasoy

given: Murat O.

name: Department of Oncology, John H. Stroger Jr. Hospital of Cook County, Chicago, IL;

family: Catchatourian

full_name: Rosalind Catchatourian

given: Rosalind

family: Vadakara

full_name: Joseph Vadakara

given: Joseph

name: Laboratory of Biochemistry, Biotechnology, and Advanced Diagnosis, Center for the Study of Myelofibrosis, Istituto Di Ricovero e Cura a Carattere Scientifico, Foundation Policlinico San Matteo, Pavia, Italy;

family: Rosti

full_name: Vittorio Rosti

given: Vittorio

name: Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA;

family: Hexner

full_name: Elizabeth Hexner

given: Elizabeth

family: Kremyanskaya

full_name: Marina Kremyanskaya

given: Marina

family: Sandy

full_name: Lonette Sandy

given: Lonette

name: Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, NY;

family: Tripodi

full_name: Joseph Tripodi

name: Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY;

name: Center for Hematological Malignancies, and

name: Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY;

name: Mayo Clinic Laboratories, Rochester, MN;

name: Department of Environmental Medicine, New York University School of Medicine, New York, NY;

name: UT Health San Antonio Cancer Center, San Antonio, TX

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

location_country:
Italy
United States
United Kingdom
France

location_facility: Wake Forest University Baptist Medical Center

location_facility: Guy's and St. Thomas' NHS Foundation Trust


title: INGEVITY(TM) Active Fixation and Passive Fixation Pace/ Sense Lead Clinical Study

summary: The objective of this study is to gather data to establish the safety, performance and effectiveness of the INGEVITY pace/ sense leads.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Boston Scientific Corporation

location_country:
Italy
Austria
United States
Sweden
Hong Kong
Spain
Germany
France
Malaysia
Thailand
Australia
Belgium
Portugal
Denmark
Canada
United Kingdom

location_facility: Azienda Ospedaliera Universitaria Integrata di Verona

location_facility: Lindner Center for Research and Education at Christ Hosp

location_facility: Kardiologische Gemeinschaftspraxis Kaltofen Schubert Gerner

location_facility: Indiana University Health La Porte Hospital

location_facility: Hopital Hotel Dieu du Centre Hospitalier de l'Universite de Montreal

location_facility: University of Iowa Hospitals and Clinics

location_facility: Cardiovascular Associates of the Delaware Valley

location_facility: Cardiology Associates of Fairfield County

location_facility: Centro Hospitalar do Porto - Hospital de Santo Antonio

location_facility: Hospital Universitario Nuestra Senora de Candelaria

location_facility: Basildon and Thurrock University Hospitals NHS

location_facility: Chiang Mai Heart Center Medical School Hospital

intervention_type: Device

lead_sponsor_type: industriel

status_simplified: Completed


title: A Phase 1 Study of SEA-TGT (SGN-TGT) in Subjects With Advanced Malignancies

summary:
This trial will look at a drug called SEA-TGT (also known as SGN-TGT) to find out whether it is safe for patients with solid tumors and lymphomas. It will study SEA-TGT to find out what its side effects are. A side effect is anything the drug does besides treating cancer. It will also study whether SEA-TGT works to treat solid tumors and lymphomas.

The study will have three parts. Part A of the study will find out how much SEA-TGT should be given to patients. Part B will use the dose found in Part A to find out how safe SEA-TGT is and if it works to treat solid tumors and lymphomas. Part C will study how well SEA-TGT with sasanlimab works to treat solid tumors.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

design_allocation: Non-Randomized

enrollment_type: Anticipated

lead_sponsor: Seagen Inc.

location_country:
Italy
France
Spain
United States
United Kingdom
Canada

location_facility: University of Michigan Comprehensive Cancer Center

location_facility: University of Cincinnati Cancer Institute

location_facility: Texas Oncology - Baylor Sammons Cancer Center

location_facility: Oncology and Hematology Assoc of SW VA DBA Blue Ridge Cancer Care

location_facility: Wake Forest Baptist Medical Center / Wake Forest University

location_facility: University of Pittsburgh Medical Center (UPMC)/Hillman Cancer Center

location_facility: University Health Network, Princess Margaret Hospital

location_facility: University of California at San Francisco

location_facility: Carbone Cancer Center / University of Wisconsin

location_facility: MD Anderson Cancer Center / University of Texas

intervention_type: Drug

status_simplified: Ongoing


title: A Phase 1 Study of SGN-CD228A in Select Advanced Solid Tumors

summary: This trial will study SGN-CD228A to find out whether it is an effective treatment for different kinds of cancer. It will also look at what side effects (unwanted effects) may occur. The study will have two parts. Part 1 of the study will find out how much SGN-CD228A should be given for treatment and how often. Part 2 of the study will use the dose found in Part 1 and look at how safe and effective the treatment is.

ipd_sharing: No

location_country:
Italy
France
Spain
United States
United Kingdom

location_facility: Angeles Clinic and Research Institute, The

location_facility: South Texas Accelerated Research Therapeutics

location_facility: Case Western Reserve University / University Hospitals Cleveland Medical Center


title: Determinants of the Progression and Outcome of Mitral Regurgitation-PROGRAM STUDY

acronym: PROGRAM

summary: Mitral regurgitation (MR) is one of the most frequent valve lesions, both in North America and in Europe, and its prevalence is increasing with the aging of the population. Organic Mitral Regurgitation (OMR) and Ischemic Mitral Regurgitation are the 2 main categories of MR. Organic or primary MR is caused by an anatomic alteration of the valvular or subvalvular mitral apparatus and refers to rheumatic MR and degenerative MR that includes mitral leaflet prolapse and flail leaflet. In the past 20 years, degenerative MR has become, by far, the most frequent cause of severe MR leading to surgery in the western world. However, the best current treatment for OMR remains uncertain and controversial. We have obtained preliminary data showing that OMR is a dynamic lesion. Hence, the echocardiographic evaluation of MR at rest, as generally performed during routine clinical exam, does not necessarily reflect the status of MR during patient's daily activities and thereby does not adequately assess the risk of rapid progression and poor outcome in these patients. The objective of this study is to identify the independent predictors of disease progression and outcome in patients with asymptomatic chronic OMR and to develop and validate novel imaging and circulating biomarkers to improve risk stratification and therapeutic decision-making process in patients with chronic asymptomatic primary OMR.

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Magne J, Mahjoub H, Pibarot P, Pirlet C, Pierard LA, Lancellotti P. Prognostic importance of exercise brain natriuretic peptide in asymptomatic degenerative mitral regurgitation. Eur J Heart Fail. 2012 Nov;14(11):1293-302. doi: 10.1093/eurjhf/hfs114. Epub 2012 Jul 10.

ReferenceCitation: Magne J, Mahjoub H, Pierard LA, O'Connor K, Pirlet C, Pibarot P, Lancellotti P. Prognostic importance of brain natriuretic peptide and left ventricular longitudinal function in asymptomatic degenerative mitral regurgitation. Heart. 2012 Apr;98(7):584-91. doi: 10.1136/heartjnl-2011-301128. Epub 2012 Feb 18.

ReferenceCitation: Lancellotti P, Magne J. Stress testing for the evaluation of patients with mitral regurgitation. Curr Opin Cardiol. 2012 Sep;27(5):492-8. doi: 10.1097/HCO.0b013e3283565c3b. Review.

ReferenceCitation: Sénéchal M, Michaud N, Machaalany J, Bernier M, Dubois M, Magne J, Couture C, Mathieu P, Bertrand OF, Voisine P. Relation of mitral valve morphology and motion to mitral regurgitation severity in patients with mitral valve prolapse. Cardiovasc Ultrasound. 2012 Jan 27;10:3. doi: 10.1186/1476-7120-10-3.

ReferenceCitation: Van de Heyning CM, Magne J, Vrints CJ, Piérard L, Lancellotti P. The role of multi-imaging modality in primary mitral regurgitation. Eur Heart J Cardiovasc Imaging. 2012 Feb;13(2):139-51. doi: 10.1093/ejechocard/jer257. Epub 2011 Nov 29. Review.

ReferenceCitation: Magne J, Lancellotti P, O'Connor K, Van de Heyning CM, Szymanski C, Piérard LA. Prediction of exercise pulmonary hypertension in asymptomatic degenerative mitral regurgitation. J Am Soc Echocardiogr. 2011 Sep;24(9):1004-12. doi: 10.1016/j.echo.2011.04.003. Epub 2011 May 17.

ReferenceCitation: Magne J, Lancellotti P, Piérard LA. Exercise-induced changes in degenerative mitral regurgitation. J Am Coll Cardiol. 2010 Jul 20;56(4):300-9. doi: 10.1016/j.jacc.2009.12.073.

ReferenceCitation: Magne J, Lancellotti P, Piérard LA. Exercise pulmonary hypertension in asymptomatic degenerative mitral regurgitation. Circulation. 2010 Jul 6;122(1):33-41. doi: 10.1161/CIRCULATIONAHA.110.938241. Epub 2010 Jun 21.

ReferenceCitation: Magne J, Mathieu P, Dumesnil JG, Tanné D, Dagenais F, Doyle D, Pibarot P. Impact of prosthesis-patient mismatch on survival after mitral valve replacement. Circulation. 2007 Mar 20;115(11):1417-25. Epub 2007 Mar 5.

ReferenceCitation: Mascle S, Schnell F, Thebault C, Corbineau H, Laurent M, Hamonic S, Veillard D, Mabo P, Leguerrier A, Donal E. Predictive value of global longitudinal strain in a surgical population of organic mitral regurgitation. J Am Soc Echocardiogr. 2012 Jul;25(7):766-72. doi: 10.1016/j.echo.2012.04.009. Epub 2012 May 19.

ReferenceCitation: Donal E, Mascle S, Brunet A, Thebault C, Corbineau H, Laurent M, Leguerrier A, Mabo P. Prediction of left ventricular ejection fraction 6 months after surgical correction of organic mitral regurgitation: the value of exercise echocardiography and deformation imaging. Eur Heart J Cardiovasc Imaging. 2012 Nov;13(11):922-30. doi: 10.1093/ehjci/jes068. Epub 2012 Apr 14.

ReferenceCitation: Magne J, Mahjoub H, Dulgheru R, Pibarot P, Pierard LA, Lancellotti P. Left ventricular contractile reserve in asymptomatic primary mitral regurgitation. Eur Heart J. 2014 Jun 21;35(24):1608-16. doi: 10.1093/eurheartj/eht345. Epub 2013 Sep 7.

ReferenceCitation: Toubal O, Mahjoub H, Thébault C, Clavel MA, Dahou A, Magne J, O'Connor K, Beaudoin J, Bernier M, Le Ven F, Pibarot P. Increasing Pulmonary Arterial Pressure at Low Level of Exercise in Asymptomatic, Organic Mitral Regurgitation. J Am Coll Cardiol. 2018 Feb 13;71(6):700-701. doi: 10.1016/j.jacc.2017.11.062.

ReferenceCitation: Clemenceau A, Bérubé JC, Bélanger P, Gaudreault N, Lamontagne M, Toubal O, Clavel MA, Capoulade R, Mathieu P, Pibarot P, Bosse Y. Deleterious variants in DCHS1 are prevalent in sporadic cases of mitral valve prolapse. Mol Genet Genomic Med. 2018 Jan;6(1):114-120. doi: 10.1002/mgg3.347. Epub 2017 Dec 10.

ReferenceCitation: Dupuis M, Mahjoub H, Clavel MA, Côté N, Toubal O, Tastet L, Dumesnil JG, O'Connor K, Dahou A, Thébault C, Bélanger C, Beaudoin J, Arsenault M, Bernier M, Pibarot P. Forward Left Ventricular Ejection Fraction: A Simple Risk Marker in Patients With Primary Mitral Regurgitation. J Am Heart Assoc. 2017 Oct 27;6(11). pii: e006309. doi: 10.1161/JAHA.117.006309.

lead_sponsor: Laval University

location_country: Canada Belgium France

location_facility: University Hospital (CHU) of Brest, Hôpital La Cavale Blanche

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec

intervention_type: Other

lead_sponsor_type: academique


type: EudraCT Number

title: A Double-blind, Placebo Controlled, Randomized, Phase IIa Study to Assess the Safety, Tolerability, Efficacy, and Pharmacokinetics of Repeated Topical Applications Over 42 Days of AP611074 5% Gel in Condyloma Patients

summary: The purpose of this study is to determine the safety, tolerability and efficacy of the topical application of AP611074 5% gel during 6 weeks on ano-genital warts caused by human papillomavirus (HPV).

design_allocation: Randomized

lead_sponsor: Anaconda Pharma

location_country: Argentina France

location_facility: Hopital Henri Mondor-Dept of Dermatology

location_facility: Hospotal Italinao de Buenos Aires - Dept of Dermatology and Gynecology

location_facility: IADT - Instituto Argentino de Diagnostico y Tratamiento S.A.


ipd_sharing_description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

lead_sponsor: Regeneron Pharmaceuticals

location_country:
United States
United Kingdom
Korea, Republic of
France

ipd_sharing: Yes

source: euctr

title: A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

summary:
The primary objective of this study is to assess the safety and tolerability of REGN7257 in patients with severe aplastic anemia (SAA) that is refractory to or has relapsed while on standard of care immunosuppressive therapy (IST). An additional primary objective (for Part B only) is to evaluate the clinical efficacy of REGN7257 in IST-refractory/relapsed patients.

The secondary objectives of this study are to assess the following for REGN7257:

Clinical response over time
Maintenance of response
Impact on transfusion requirements
Effect on blood counts and cell populations
Pharmacokinetics (PK)
Immunogenicity


title: Targeted Hypothermia Versus Targeted Normothermia After Out-of-hospital Cardiac Arrest (TTM2) - A Randomised Clinical Trial

summary: ILCOR guidelines recommend Target Temperature Management (TTM) to between 32°C and 36°C after out-of-hospital cardiac arrest, based on low quality evidence. In a previous trial, TTM at 33°C did not confer a survival benefit or improved neurological function, compared to TTM at 36°C. A lower target temperature might be beneficial compared with normothermia and early treatment of fever. Therefore the primary purpose of the TTM2-trial will be to study any differences in mortality, neurological function and quality of life between a target temperature of 33°C and standard care avoiding fever.

status: Active, not recruiting

ReferenceCitation: Nielsen N, Wetterslev J, Cronberg T, Erlinge D, Gasche Y, Hassager C, Horn J, Hovdenes J, Kjaergaard J, Kuiper M, Pellis T, Stammet P, Wanscher M, Wise MP, Åneman A, Al-Subaie N, Boesgaard S, Bro-Jeppesen J, Brunetti I, Bugge JF, Hingston CD, Juffermans NP, Koopmans M, Køber L, Langørgen J, Lilja G, Møller JE, Rundgren M, Rylander C, Smid O, Werer C, Winkel P, Friberg H; TTM Trial Investigators. Targeted temperature management at 33°C versus 36°C after cardiac arrest. N Engl J Med. 2013 Dec 5;369(23):2197-206. doi: 10.1056/NEJMoa1310519. Epub 2013 Nov 17.

ReferenceType: derived

ReferenceCitation: Dankiewicz J, Cronberg T, Lilja G, Jakobsen JC, Levin H, Ullén S, Rylander C, Wise MP, Oddo M, Cariou A, Bělohlávek J, Hovdenes J, Saxena M, Kirkegaard H, Young PJ, Pelosi P, Storm C, Taccone FS, Joannidis M, Callaway C, Eastwood GM, Morgan MPG, Nordberg P, Erlinge D, Nichol AD, Chew MS, Hollenberg J, Thomas M, Bewley J, Sweet K, Grejs AM, Christensen S, Haenggi M, Levis A, Lundin A, Düring J, Schmidbauer S, Keeble TR, Karamasis GV, Schrag C, Faessler E, Smid O, Otáhal M, Maggiorini M, Wendel Garcia PD, Jaubert P, Cole JM, Solar M, Borgquist O, Leithner C, Abed-Maillard S, Navarra L, Annborn M, Undén J, Brunetti I, Awad A, McGuigan P, Bjørkholt Olsen R, Cassina T, Vignon P, Langeland H, Lange T, Friberg H, Nielsen N; TTM2 Trial Investigators. Hypothermia versus Normothermia after Out-of-Hospital Cardiac Arrest. N Engl J Med. 2021 Jun 17;384(24):2283-2294. doi: 10.1056/NEJMoa2100591.

name: From the Department of Clinical Sciences Lund, Sections of Cardiology (J. Dankiewicz, D.E.), Neurology (T. Cronberg, G.L.), and Anesthesiology and Intensive Care (H. Levin, O.B.), Skåne University Hospital Lund, Lund University and Clinical Studies Sweden — Forum South, Skåne University Hospital (S.U.), Lund; the Department of Clinical Sciences Lund, Section of Anesthesia and Intensive Care, Skåne University Hospital Malmö, Malmö, (J. Düring, S.S., H.F.); the Department of Clinical Sciences Lund,...

family: Dankiewicz

full_name: Josef Dankiewicz

given: Josef

sequence: first

family: Cronberg

full_name: Tobias Cronberg

given: Tobias

sequence: additional

family: Lilja

full_name: Gisela Lilja

given: Gisela

family: Jakobsen

full_name: Janus C. Jakobsen

given: Janus C.

family: Levin

full_name: Helena Levin

given: Helena

family: Ullén

full_name: Susann Ullén

given: Susann

family: Rylander

full_name: Christian Rylander

given: Christian

family: Wise

full_name: Matt P. Wise

given: Matt P.

family: Oddo

full_name: Mauro Oddo

given: Mauro

family: Cariou

full_name: Alain Cariou

given: Alain

family: Bělohlávek

full_name: Jan Bělohlávek

given: Jan

family: Hovdenes

full_name: Jan Hovdenes

family: Saxena

full_name: Manoj Saxena

given: Manoj

family: Kirkegaard

full_name: Hans Kirkegaard

given: Hans

family: Young

full_name: Paul J. Young

given: Paul J.

family: Pelosi

full_name: Paolo Pelosi

given: Paolo

family: Storm

full_name: Christian Storm

family: Taccone

full_name: Fabio S. Taccone

given: Fabio S.

family: Joannidis

full_name: Michael Joannidis

given: Michael

family: Callaway

full_name: Clifton Callaway

given: Clifton

family: Eastwood

full_name: Glenn M. Eastwood

given: Glenn M.

family: Morgan

full_name: Matt P.G. Morgan

given: Matt P.G.

family: Nordberg

full_name: Per Nordberg

given: Per

ReferenceCitation: Dankiewicz J, Cronberg T, Lilja G, Jakobsen JC, Bělohlávek J, Callaway C, Cariou A, Eastwood G, Erlinge D, Hovdenes J, Joannidis M, Kirkegaard H, Kuiper M, Levin H, Morgan MPG, Nichol AD, Nordberg P, Oddo M, Pelosi P, Rylander C, Saxena M, Storm C, Taccone F, Ullén S, Wise MP, Young P, Friberg H, Nielsen N. Targeted hypothermia versus targeted Normothermia after out-of-hospital cardiac arrest (TTM2): A randomized clinical trial-Rationale and design. Am Heart J. 2019 Nov;217:23-31. doi: 10.1016/j.ahj.2019.06.012. Epub 2019 Jun 26.

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

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

repository_institution: Cardiff University - ORCA Online Research @Cardiff

title: Targeted hypothermia versus targeted Normothermia after out-of-hospital cardiac arrest (TTM2): A randomized clinical trial—Rationale and design

location_country:
Italy
Austria
Sweden
Switzerland
Germany
France
Czechia
Norway
Australia
Belgium
New Zealand
Denmark
United States
United Kingdom

location_facility: Basildon and Thurrock Hospitals NHS Foundation Trust - Essex CTC

location_facility: Royal Bournemouth and Christchurch Hospitals NHS Foundation Trust


title: A Multicenter, Open-Label, Randomized Comparative Study of Tigecycline vs Ceftriaxone Sodium Plus Metronidazole for the Treatment of Hospitalized Subjects With Complicated Intra-abdominal Infection

summary: This is a study of the safety and efficacy of tigecycline to ceftriaxone sodium plus metronidazole in hospitalized subjects with cIAI. Subjects will be followed for efficacy through the test-of-cure assessment. Safety evaluations will occur through the treatment and post-treatment periods and continue through resolution or stability of the adverse event(s).

ReferenceCitation: Qvist N, Warren B, Leister-Tebbe H, Zito ET, Pedersen R, McGovern PC, Babinchak T. Efficacy of tigecycline versus ceftriaxone plus metronidazole for the treatment of complicated intra-abdominal infections: results from a randomized, controlled trial. Surg Infect (Larchmt). 2012 Apr;13(2):102-9. doi: 10.1089/sur.2011.048. Epub 2012 Mar 22.

name: Surgical Department A, Odense University Hospital, Odense, Denmark.

family: Qvist

full_name: Niels Qvist

given: Niels

name: Division of Surgery, Tygerberg Hospital and the University of Stellenbosch, Tygerberg, South Africa.

family: Warren

full_name: Brian Warren

given: Brian

name: Pfizer Inc., Collegeville, Pennsylvania.

family: Leister-Tebbe

full_name: Heidi Leister-Tebbe

given: Heidi

family: Zito

full_name: Edward T. Zito

given: Edward T.

family: Pedersen

full_name: Ronald Pedersen

given: Ronald

family: McGovern

full_name: Paul C. McGovern

given: Paul C.

family: Babinchak

full_name: Tim Babinchak

given: Tim

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Surgical Infections

publisher: Mary Ann Liebert Inc

publisher_dissemination: Mary Ann Liebert Inc

publisher_group: Mary Ann Liebert Inc

publisher_normalized: Mary Ann Liebert Inc

title: Efficacy of Tigecycline versus Ceftriaxone Plus Metronidazole for the Treatment of Complicated Intra-Abdominal Infections: Results from a Randomized, Controlled Trial

lead_sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

location_country:
Italy
South Africa
India
Saudi Arabia
Hong Kong
Spain
Philippines
Switzerland
Finland
France
Greece
Australia
China
Turkey
Denmark
Germany
United Kingdom
Taiwan
Portugal


title: GLOBAL LEADERS: A Clinical Study Comparing Two Forms of Anti-platelet Therapy After Stent Implantation

summary:
After a stent procedure, it is common practice to prescribe anti-platelet medication to prevent the blood from clotting. The main objective of this study is to determine if there is a better medication strategy to prevent blood from clotting and at the same time minimising the number of complications.

There are two medication strategies:

Study group: Dual anti-platelet therapy (ticagrelor combined with aspirin) for 1 month, and then ticagrelor alone for another 23 months OR
Control group: Standard treatment, being dual anti-platelet therapy (ticagrelor or clopidogrel combined with aspirin) for 12 months, and then aspirin alone indefinitely

ReferenceCitation: Vranckx P, Valgimigli M, Jüni P, Hamm C, Steg PG, Heg D, van Es GA, McFadden EP, Onuma Y, van Meijeren C, Chichareon P, Benit E, Möllmann H, Janssens L, Ferrario M, Moschovitis A, Zurakowski A, Dominici M, Van Geuns RJ, Huber K, Slagboom T, Serruys PW, Windecker S; GLOBAL LEADERS Investigators. Ticagrelor plus aspirin for 1 month, followed by ticagrelor monotherapy for 23 months vs aspirin plus clopidogrel or ticagrelor for 12 months, followed by aspirin monotherapy for 12 months after implantation of a drug-eluting stent: a multicentre, open-label, randomised superiority trial. Lancet. 2018 Sep 15;392(10151):940-949. doi: 10.1016/S0140-6736(18)31858-0. Epub 2018 Aug 27.

ReferenceCitation: Vranckx P, Valgimigli M, Odutayo A, Serruys PW, Hamm C, Steg PG, Heg D, Mc Fadden EP, Onuma Y, Benit E, Janssens L, Diletti R, Ferrario M, Huber K, Räber L, Windecker S, Jüni P; GLOBAL LEADERS Investigators. Efficacy and Safety of Ticagrelor Monotherapy by Clinical Presentation: Pre-Specified Analysis of the GLOBAL LEADERS Trial. J Am Heart Assoc. 2021 Sep 21;10(18):e015560. doi: 10.1161/JAHA.119.015560. Epub 2021 Sep 17.

name: Jessa Ziekenhuis Faculty of Medicine and Life Sciences at the Hasselt University Hasselt Belgium

family: Vranckx

full_name: Pascal Vranckx

given: Pascal

name: Department of Cardiology University of BernInselspital Bern Switzerland

family: Valgimigli

full_name: Marco Valgimigli

given: Marco

name: Department of Medicine and Institute of Health Policy, Management and Evaluation Applied Health Research CentreLi Ka Shing Knowledge Institute of St. Michael’s HospitalUniversity of Toronto Canada

family: Odutayo

full_name: Ayodele Odutayo

given: Ayodele

name: Imperial College London London United Kingdom

family: Serruys

full_name: Patrick W. Serruys

given: Patrick W.

name: Kerckhoff Heart and Thorax Center Bad Nauheim Germany

family: Hamm

full_name: Christian Hamm

given: Christian

name: INSERM U‐1148 FACT (French Alliance for Cardiovascular Trials) Hôpital BichatAssistance Publique–Hôpitaux de ParisUniversité de Paris France

name: National Heart and Lung InstituteRoyal Brompton HospitalImperial College London United Kingdom

family: Steg

full_name: Philippe Gabriel Steg

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name: Clinical Trials Unit University of Bern Switzerland

family: Heg

full_name: Dik Heg

given: Dik

name: Cork University Hospital Wilton, Cork Ireland

family: Mc Fadden

full_name: Eugene P. Mc Fadden

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name: Erasmus Medical Center Rotterdam The Netherlands

family: Onuma

full_name: Yoshinobu Onuma

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family: Benit

full_name: Edouard Benit

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family: Janssens

full_name: Luc Janssens

given: Luc

family: Diletti

full_name: Roberto Diletti

given: Roberto

name: UOC Cardiologia Fondazione IRCCS Policlinico San Matteo Pavia Italy

family: Ferrario

full_name: Maurizio Ferrario

given: Maurizio

name: 3rd Department of Medicine, Cardiology and Intensive Care Medicine Medical Faculty Wilhelminen Hospital and Sigmund Freud University Vienna Austria

family: Huber

full_name: Kurt Huber

given: Kurt

family: Räber

full_name: Lorenz Räber

given: Lorenz

family: Windecker

full_name: Stephan Windecker

given: Stephan

family: Jüni

full_name: Peter Jüni

given: Peter

currency_apc_doaj: USD

journal_name: Journal of the American Heart Association

oa_host_type: publisher

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: Efficacy and Safety of Ticagrelor Monotherapy by Clinical Presentation: Pre‐Specified Analysis of the GLOBAL LEADERS Trial

ReferenceCitation: Gao C, Tomaniak M, Takahashi K, Kawashima H, Wang R, Hara H, Ono M, Montalescot G, Garg S, Haude M, Slagboom T, Vranckx P, Valgimigli M, Windecker S, van Geuns RJ, Hamm C, Steg PG, Onuma Y, Angiolillo DJ, Serruys PW. Ticagrelor monotherapy in patients with concomitant diabetes mellitus and chronic kidney disease: a post hoc analysis of the GLOBAL LEADERS trial. Cardiovasc Diabetol. 2020 Oct 16;19(1):179. doi: 10.1186/s12933-020-01153-x.

family: Gao

full_name: Chao Gao

given: Chao

family: Tomaniak

full_name: Mariusz Tomaniak

given: Mariusz

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

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

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repository_institution: University of Zurich - Zurich Open Repository and Archive

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

repositories_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université Paris Descartes

repositories_institution: University Library Bern - BORIS - Bern Open Repository and Information System

repositories_institution: University of Zurich - Zurich Open Repository and Archive

ReferenceCitation: Kawashima H, Tomaniak M, Ono M, Wang R, Hara H, Gao C, Takahashi K, Sharif F, Thury A, Suryapranata H, Walsh S, Cotton J, Carrie D, Sabate M, Steinwender C, Leibundgut G, Wykrzykowska J, de Winter RJ, Garg S, Hamm C, Steg PG, Jüni P, Vranckx P, Valgimigli M, Windecker S, Onuma Y, Serruys PW. Safety and Efficacy of 1-Month Dual Antiplatelet Therapy (Ticagrelor + Aspirin) Followed by 23-Month Ticagrelor Monotherapy in Patients Undergoing Staged Percutaneous Coronary Intervention (A Sub-Study from GLOBAL LEADERS). Am J Cardiol. 2021 Jan 1;138:1-10. doi: 10.1016/j.amjcard.2020.09.057. Epub 2020 Oct 13.

ReferenceCitation: Gao C, Takahashi K, Garg S, Hara H, Wang R, Kawashima H, Ono M, Montalescot G, Haude M, Slagboom T, Vranckx P, Valgimigli M, Windecker S, Hamm C, Steg PG, Storey R, van Geuns RJ, Tao L, Onuma Y, Serruys PW. Regional variation in patients and outcomes in the GLOBAL LEADERS trial. Int J Cardiol. 2021 Feb 1;324:30-37. doi: 10.1016/j.ijcard.2020.09.039. Epub 2020 Sep 15.

title: Regional variation in patients and outcomes in the GLOBAL LEADERS trial

ReferenceCitation: Hara H, Takahashi K, Kogame N, Tomaniak M, Kerkmeijer LSM, Ono M, Kawashima H, Wang R, Gao C, Wykrzykowska JJ, de Winter RJ, Neumann FJ, Plante S, Lemos Neto PA, Garg S, Jüni P, Vranckx P, Windecker S, Valgimigli M, Hamm C, Steg PG, Onuma Y, Serruys PW. Impact of Bleeding and Myocardial Infarction on Mortality in All-Comer Patients Undergoing Percutaneous Coronary Intervention. Circ Cardiovasc Interv. 2020 Sep;13(9):e009177. doi: 10.1161/CIRCINTERVENTIONS.120.009177. Epub 2020 Aug 25.

name: Department of Cardiology, Academic Medical Center, University of Amsterdam, the Netherlands (H.H., K.T., N.K., L.S.M.K., M.O., H.K. J.J.W., R.J.d.W.).

name: Department of Cardiology, Erasmus Medical Center, Erasmus University, Rotterdam, the Netherlands (M.T.).

name: First Department of Cardiology, Medical University of Warsaw, Poland (M.T.).

name: Department of Cardiology, Radboud University, Nijmegen, the Netherlands (R.W., C.G.).

name: Division of Cardiology and Angiology II, University Heart Center Freiburg, Bad Krozingen, Germany (F.-J.N.).

name: Division of Cardiology, Department of Medicine, Southlake Regional Health Center, Newmarket, ON, Canada (S.P.).

name: Heart Institute (InCor), University of Sao Paulo Medical School, Brazil (P.A.L.N.).

name: Department of Cardiology, Royal Blackburn Hospital, Blackburn, United Kingdom (S.G.).

name: Applied Health Research Centre, Li Ka Shing Knowledge Institute, St Michael’s Hospital, University of Toronto, Canada (P.J.).

name: Department of Cardiology and Critical Care, Jessa Ziekenhuis Hasselt, Faculty of Medicine and Life Sciences, University of Hasselt, Belgium (P.V.).

name: Department of Cardiology, Bern University Hospital, Switzerland (S.W., M.V.).

name: Department of Cardiology, Kerckhoff Heart Center, Campus University of Giessen, Bad Nauheim, Germany (C.H.).

name: FACT, Université Paris Diderot, Hôpital Bichat, Assistance Publique-Hôpitaux de Paris, France (P.G.S.).

name: Department of Cardiology, National University of Ireland, Galway (NUIG), Ireland (Y.O., P.W.S.).

name: NHLI, Imperial College London, United Kingdom (P.W.S.).

ReferenceCitation: Hara H, van Klaveren D, Takahashi K, Kogame N, Chichareon P, Modolo R, Tomaniak M, Ono M, Kawashima H, Wang R, Gao C, Niethammer M, Fontos G, Angioi M, Ribeiro VG, Barbato E, Leandro S, Hamm C, Valgimigli M, Windecker S, Jüni P, Steg PG, Verbeeck J, Tijssen JGP, Sharif F, Onuma Y, Serruys PW; GLOBAL LEADERS Trial Investigators. Comparative Methodological Assessment of the Randomized GLOBAL LEADERS Trial Using Total Ischemic and Bleeding Events. Circ Cardiovasc Qual Outcomes. 2020 Aug;13(8):e006660. doi: 10.1161/CIRCOUTCOMES.120.006660. Epub 2020 Jul 30.

name: Department of Cardiology, Academic Medical Center, University of Amsterdam, the Netherlands (H.H., K.T., N.K., P.C., R.M., M.O., H.K., J.G.P.T.).

name: Department of Public Health, Center for Medical Decision Making, Erasmus MC, Rotterdam, the Netherlands (D.v.K.).

name: Predictive Analytics and Comparative Effectiveness Center, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA (D.v.K.).

name: Cardiology Unit, Department of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Thailand (P.C.).

name: Cardiology Division, Department of Internal Medicine, University of Campinas (UNICAMP), Brazil (R.M.).

name: Medizinische Klinik I, Herz-Thorax Zentrum, Klinikum Fulda, Germany (M.N.).

name: Gottsegen Hungarian Institute of Cardiology, Budapest, Hungary (G.F.).

name: Department of Interventional Cardiology Clinique Louis Pasteur Essey-les-Nancy, France (M.A.).

name: Cardiology Department, Gaia Hospital Center, Portugal (V.G.R.).

name: Division of Cardiology, Department of Advanced Biomedical Sciences, University Federico II, Naples, Italy (E.B.).

name: Instituto Nacional De Cardiologia, Rio de Janeiro, Brazil (S.L.).

name: Kerckhoff Heart Center, Campus University of Giessen, Bad Nauheim, Germany (C.H.).

name: Department of Cardiology, Bern University Hospital, Inselspital, University of Bern, Switzerland (M.V., S.W.).

name: Applied Health Research Centre (AHRC), Li Ka Shing Knowledge Institute of St. Michael’s Hospital, Department of Medicine and the Institute of Health Policy, Management and Evaluation at the University of Toronto, Canada (P.J.).

name: FACT (French Alliance for Cardiovascular Clinical Trials), Université de Paris, Hôpital Bichat, Assistance-Publique-Hôpitaux de Paris, and INSERM Unité 1148, France (P.G.S.).

name: Imperial College, Royal Brompton Hospital, London, United Kingdom (P.G.S.).

name: I-Biostat, University of Hasselt, Belgium (J.V.).

name: Department of Cardiology, National University of Ireland, Galway (NUIG), Ireland (F.S., Y.O., P.W.S.).

name: for the GLOBAL LEADERS Trial Investigators

journal_name: Circulation: Cardiovascular Quality and Outcomes

ReferenceCitation: Ono M, Chichareon P, Tomaniak M, Kawashima H, Takahashi K, Kogame N, Modolo R, Hara H, Gao C, Wang R, Walsh S, Suryapranata H, da Silva PC, Cotton J, Koning R, Akin I, Rensing BJWM, Garg S, Wykrzykowska JJ, Piek JJ, Jüni P, Hamm C, Steg PG, Valgimigli M, Windecker S, Storey RF, Onuma Y, Vranckx P, Serruys PW. The association of body mass index with long-term clinical outcomes after ticagrelor monotherapy following abbreviated dual antiplatelet therapy in patients undergoing percutaneous coronary intervention: a prespecified sub-analysis of the GLOBAL LEADERS Trial. Clin Res Cardiol. 2020 Sep;109(9):1125-1139. doi: 10.1007/s00392-020-01604-1. Epub 2020 Jan 31.

repository_institution: University of Bern - Bern Open Repository and Information System

repositories_institution: University of Bern - Bern Open Repository and Information System

ReferenceCitation: Tomaniak M, Chichareon P, Klimczak-Tomaniak D, Takahashi K, Kogame N, Modolo R, Wang R, Ono M, Hara H, Gao C, Kawashima H, Rademaker-Havinga T, Garg S, Curzen N, Haude M, Kochman J, Gori T, Montalescot G, Angiolillo DJ, Capodanno D, Storey RF, Hamm C, Vranckx P, Valgimigli M, Windecker S, Onuma Y, Serruys PW, Anderson R. Impact of renal function on clinical outcomes after PCI in ACS and stable CAD patients treated with ticagrelor: a prespecified analysis of the GLOBAL LEADERS randomized clinical trial. Clin Res Cardiol. 2020 Jul;109(7):930-943. doi: 10.1007/s00392-019-01586-9. Epub 2020 Jan 10.

ReferenceCitation: Tomaniak M, Chichareon P, Takahashi K, Kogame N, Modolo R, Chang CC, Spitzer E, Neumann FJ, Plante S, Hernández Antolin R, Jambrik Z, Gelev V, Brunel P, Konteva M, Beygui F, Morelle JF, Filipiak KJ, van Geuns RJ, Soliman O, Tijssen J, Rademaker-Havinga T, Storey RF, Hamm C, Steg PG, Windecker S, Onuma Y, Valgimigli M, Serruys PW; GLOBAL LEADERS Study Investigators. Impact of chronic obstructive pulmonary disease and dyspnoea on clinical outcomes in ticagrelor treated patients undergoing percutaneous coronary intervention in the randomized GLOBAL LEADERS trial. Eur Heart J Cardiovasc Pharmacother. 2020 Jul 1;6(4):222-230. doi: 10.1093/ehjcvp/pvz052.

name: Department of Cardiology, Erasmus Medical Centre, Erasmus University, Rotterdam, The Netherlands

name: First Department of Cardiology, Medical University of Warsaw, Warsaw, Poland

name: Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands

name: Division of Cardiology, Department of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Songkhla, Thailand

name: Department of Internal Medicine, Cardiology Division, University of Campinas (UNICAMP), Campinas, Brazil

name: Cardialysis Core Laboratories and Clinical Trial Management, Rotterdam, The Netherlands

name: Universitäts-Herzzentrum Freiburg Bad Krozingen, Bad Krozingen, Germany

name: Southlake Regional Health Centre, Newmarket, Ontario, Canada

name: Békés Megyei Pándy Kálmán Kórház County Hospital, Gyula, Hungary

name: Cardiologie Clinique Valmy Hopital Prive Dijon Bourgogne HPDB Dijon, Dijon, France

name: Heart Centre “Pontica”, Burgas, Bulgaria

name: Department of Cardiology, Radboud UMC, Nijmegen, The Netherlands

name: Department of Cardiology, National University of Ireland Galway, Galway, Ireland

name: Department of Cardiovascular Science, University of Sheffield, Sheffield, UK

name: FACT (French Alliance for Cardiovascular Trials), Université Paris Diderot, Hôpital Bichat, Assistance Publique—Hôpitaux de Paris, INSERM U-1148, Paris, France

name: Department of Cardiology, Bern University Hospital, Inselspital, University of Bern, Bern, Switzerland

name: NHLI, Imperial College London, London, UK

name: for the GLOBAL LEADERS Study Investigators

journal_name: European Heart Journal - Cardiovascular Pharmacotherapy

ReferenceCitation: Tomaniak M, Chichareon P, Modolo R, Takahashi K, Chang CC, Kogame N, Spitzer E, Buszman PE, van Geuns RM, Valkov V, Steinwender C, Geisler T, Prokopczuk J, Sabaté M, Zmudka K, Rademaker-Havinga T, Tijssen JGP, Jüni P, Hamm C, Steg PG, Onuma Y, Vranckx P, Valgimigli M, Windecker S, Baber U, Anderson R, Dominici M, Serruys PW. Ticagrelor monotherapy beyond one month after PCI in ACS or stable CAD in elderly patients: a pre-specified analysis of the GLOBAL LEADERS trial. EuroIntervention. 2020 Apr 3;15(18):e1605-e1614. doi: 10.4244/EIJ-D-19-00699.

ReferenceCitation: Chichareon P, Modolo R, Kerkmeijer L, Tomaniak M, Kogame N, Takahashi K, Chang CC, Komiyama H, Moccetti T, Talwar S, Colombo A, Maillard L, Barlis P, Wykrzykowska J, Piek JJ, Garg S, Hamm C, Steg PG, Jüni P, Valgimigli M, Windecker S, Onuma Y, Mehran R, Serruys PW. Association of Sex With Outcomes in Patients Undergoing Percutaneous Coronary Intervention: A Subgroup Analysis of the GLOBAL LEADERS Randomized Clinical Trial. JAMA Cardiol. 2020 Jan 1;5(1):21-29. doi: 10.1001/jamacardio.2019.4296.

name: Heart Center, Department of Clinical and Experimental Cardiology, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands

name: Cardiology Unit, Department of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Songkhla, Thailand

name: Cardiology Division, Department of Internal Medicine, University of Campinas, Campinas, Brazil

name: Erasmus Medical Center, Erasmus University, Rotterdam, the Netherlands

name: Department of Cardiology, Fondazione Cardiocentro Ticino, Lugano, Switzerland

name: Royal Bournemouth and Christchurch National Health Services Trust, Bournemouth, England

name: Division of Interventional Cardiology, Cardio-Thoracic-Vascular Department, San Raffaele Scientific Institute, Milan, Italy

name: GCS ES Axium Rambot, Aix en Provence, France

name: St Vincent’s and Northern Hospitals, Melbourne Medical School, The University of Melbourne, Victoria, Australia

name: East Lancashire Hospitals National Health Services Trust, Blackburn, Lancashire, England

name: Kerckhoff Heart Center, Campus University of Giessen, Bad Nauheim, Germany

name: French Alliance for Cardiovascular Trials; Hôpital Bichat, AP-HP; Université Paris-Diderot; INSERM U-1148; Paris, France

name: Royal Brompton Hospital, Imperial College, London, England

name: Applied Health Research Centre, Li Ka Shing Knowledge Institute, St Michael’s Hospital, Department of Medicine, University of Toronto, Toronto, Canada

name: Department of Cardiology, Bern University Hospital, Bern, Switzerland

name: Cardialysis Clinical Trials Management and Core Laboratories, Westblaak 98, Rotterdam, the Netherlands

name: Interventional Cardiovascular Research and Clinical Trials, The Zena and Michael A. Wiener Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York, New York

name: National Heart and Lung Institute, Imperial College London, London, England

title: Association of Sex With Outcomes in Patients Undergoing Percutaneous Coronary Intervention

ReferenceCitation: Takahashi K, Serruys PW, Chichareon P, Chang CC, Tomaniak M, Modolo R, Kogame N, Magro M, Chowdhary S, Eitel I, Zweiker R, Ong P, Ottesen MM, Tijssen JGP, Wykrzykowska JJ, de Winter RJ, Garg S, Stoll HP, Hamm C, Steg PG, Onuma Y, Valgimigli M, Vranckx P, Carrie D, Windecker S. Efficacy and Safety of Ticagrelor Monotherapy in Patients Undergoing Multivessel PCI. J Am Coll Cardiol. 2019 Oct 22;74(16):2015-2027. doi: 10.1016/j.jacc.2019.08.997.

journal_name: Journal of the American College of Cardiology

title: Efficacy and Safety of Ticagrelor Monotherapy in Patients Undergoing Multivessel PCI

ReferenceCitation: Tomaniak M, Chichareon P, Onuma Y, Deliargyris EN, Takahashi K, Kogame N, Modolo R, Chang CC, Rademaker-Havinga T, Storey RF, Dangas GD, Bhatt DL, Angiolillo DJ, Hamm C, Valgimigli M, Windecker S, Steg PG, Vranckx P, Serruys PW; GLOBAL LEADERS Trial Investigators. Benefit and Risks of Aspirin in Addition to Ticagrelor in Acute Coronary Syndromes: A Post Hoc Analysis of the Randomized GLOBAL LEADERS Trial. JAMA Cardiol. 2019 Nov 1;4(11):1092-1101. doi: 10.1001/jamacardio.2019.3355.

name: Department of Cardiology, Erasmus Medical Center, Erasmus University, Rotterdam, the Netherlands

name: Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands

name: Cardialysis Core Laboratories and Clinical Trial Management, Rotterdam, the Netherlands

name: Department of Internal Medicine, Cardiology Division, University of Campinas, Campinas, Brazil

name: Department of Infection, Immunity and Cardiovascular Disease, University of Sheffield, Sheffield, England

name: Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York, New York

name: Department of Medicine, Brigham and Women’s Hospital Heart and Vascular Center, Boston, Massachusetts

name: Division of Cardiology, University of Florida, College of Medicine, Jacksonville

name: French Alliance for Cardiovascular Trials, Université Paris Diderot, Hôpital Bichat, Assistance Publique Hôpitaux de Paris, and INSERM U-1148, Paris, France

name: Department of Cardiology and Critical Care Medicine, Hartcentrum Hasselt, Jessa Ziekenhuis, Hasselt, Belgium

repository_institution: University of Szeged - SZTE Publicatio Repozitórium - SZTE - Repository of Publications

repositories_institution: University of Szeged - SZTE Publicatio Repozitórium - SZTE - Repository of Publications

title: Benefit and Risks of Aspirin in Addition to Ticagrelor in Acute Coronary Syndromes

ReferenceCitation: Serruys PW, Tomaniak M, Chichareon P, Modolo R, Kogame N, Takahashi K, Chang CC, Spitzer E, Walsh SJ, Adlam D, Hildick-Smith D, Édes I, van de Harst P, Krackhardt F, Tijssen JGP, Rademaker-Havinga T, Garg S, Steg PG, Hamm C, Jüni P, Vranckx P, Onuma Y, Verheugt FWA. Patient-oriented composite endpoints and net adverse clinical events with ticagrelor monotherapy following percutaneous coronary intervention: insights from the randomised GLOBAL LEADERS trial. EuroIntervention. 2019 Dec 20;15(12):e1090-e1098. doi: 10.4244/EIJ-D-19-00202.

ReferenceCitation: Leonardi S, Franzone A, Piccolo R, McFadden E, Vranckx P, Serruys P, Benit E, Liebetrau C, Janssens L, Ferrario M, Zurakowski A, van Geuns RJ, Dominici M, Huber K, Slagboom T, Buszman P, Bolognese L, Tumscitz C, Bryniarski K, Aminian A, Vrolix M, Petrov I, Garg S, Naber C, Prokopczuk J, Hamm C, Steg G, Heg D, Juni P, Windecker S, Valgimigli M. Rationale and design of a prospective substudy of clinical endpoint adjudication processes within an investigator-reported randomised controlled trial in patients with coronary artery disease: the GLOBAL LEADERS Adjudication Sub-StudY (GLASSY). BMJ Open. 2019 Mar 9;9(3):e026053. doi: 10.1136/bmjopen-2018-026053.

repository_institution: Jagiellonian University - Jagiellonian University Repository

repository_institution: University of Hasselt - Document Server@UHasselt

repositories_institution: Jagiellonian University - Jagiellonian University Repository

repositories_institution: University of Hasselt - Document Server@UHasselt

location_country:
Italy
Austria
Brazil
Switzerland
Netherlands
Spain
Germany
France
Bulgaria
Poland
Singapore
Hungary
Australia
Belgium
Portugal
Denmark
Canada
United Kingdom

location_facility: Research centre Villingen - Schwenningen, 4919


title: A Randomized, Double-Blind, Placebo-Controlled Trial Comparing Clopidogrel Plus Acetylsalicylic Acid (ASA) Versus ASA Alone in Subjects With Acute ST Elevation Myocardial Infarction (STEMI) Treated With Fibrinolytic Therapy

summary: The purpose of this study is to determine if the combination of aspirin plus clopidogrel is more effective than aspirin alone in preventing another heart attack, chest pain, stroke or death in people who have already had a heart attack that was treated with fibrinolytic therapy.

primary_purpose: Prevention

ReferenceType: result

ReferenceCitation: Sabatine MS, Cannon CP, Gibson CM, López-Sendón JL, Montalescot G, Theroux P, Claeys MJ, Cools F, Hill KA, Skene AM, McCabe CH, Braunwald E; CLARITY-TIMI 28 Investigators. Addition of clopidogrel to aspirin and fibrinolytic therapy for myocardial infarction with ST-segment elevation. N Engl J Med. 2005 Mar 24;352(12):1179-89. Epub 2005 Mar 9.

ReferenceCitation: Harkness JR, Sabatine MS, Braunwald E, Morrow DA, Sloan S, Wiviott SD, Giugliano RP, Antman EM, Cannon CP, Scirica BM. Extent of ST-segment resolution after fibrinolysis adds improved risk stratification to clinical risk score for ST-segment elevation myocardial infarction. Am Heart J. 2010 Jan;159(1):55-62. doi: 10.1016/j.ahj.2009.10.033.

family: Harkness

full_name: James R. Harkness

given: James R.

family: Sabatine

full_name: Marc S. Sabatine

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family: Braunwald

full_name: Eugene Braunwald

given: Eugene

family: Morrow

full_name: David A. Morrow

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family: Sloan

full_name: Sarah Sloan

given: Sarah

family: Wiviott

full_name: Stephen D. Wiviott

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family: Giugliano

full_name: Robert P. Giugliano

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family: Antman

full_name: Elliott M. Antman

given: Elliott M.

family: Cannon

full_name: Christopher P. Cannon

given: Christopher P.

family: Scirica

full_name: Benjamin M. Scirica

given: Benjamin M.

journal_name: American Heart Journal

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Sanofi

location_country:
Australia
Belgium
Russian Federation
France
Poland
Argentina
Canada
South Africa
Sweden
Netherlands
Hungary
Ireland
Puerto Rico
Portugal
Germany
United States
United Kingdom
Italy
Austria
Brazil
Mexico
Israel
Spain
Turkey

location_facility:
sanofi-aventis France
sanofi-aventis Austria
sanofi-aventis Germany
sanofi-aventis Belgium
Sanofi-Aventis
sanofi-aventis Canada
sanofi-aventis Spain
sanofi-aventis Poland
sanofi-aventis UK
sanofi-aventis Italy
sanofi-aventis Brazil
sanofi-aventis Sweden
Sanofi-Aventis Mexico
Sanofi-Aventis Hungaria
sanofi-aventis Netherlands
sanofi-aventis South Africa


ReferenceCitation: Marabelle A, Le DT, Ascierto PA, Di Giacomo AM, De Jesus-Acosta A, Delord JP, Geva R, Gottfried M, Penel N, Hansen AR, Piha-Paul SA, Doi T, Gao B, Chung HC, Lopez-Martin J, Bang YJ, Frommer RS, Shah M, Ghori R, Joe AK, Pruitt SK, Diaz LA Jr. Efficacy of Pembrolizumab in Patients With Noncolorectal High Microsatellite Instability/Mismatch Repair-Deficient Cancer: Results From the Phase II KEYNOTE-158 Study. J Clin Oncol. 2020 Jan 1;38(1):1-10. doi: 10.1200/JCO.19.02105. Epub 2019 Nov 4.

name: Gustave Roussy, Institut National de la Santé et de la Recherche Médicale U1015, Villejuif, France

family: Marabelle

full_name: Aurelien Marabelle

given: Aurelien

name: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD

family: Le

full_name: Dung T. Le

given: Dung T.

name: Instituto Nazionale Tumori Istituto di Ricovero e Cura a Carattere Scientifico Fondazione Pascale, Naples, Italy

family: Ascierto

full_name: Paolo A. Ascierto

given: Paolo A.

name: Center for Immuno-Oncology, University Hospital of Siena, Italy

family: Di Giacomo

full_name: Anna Maria Di Giacomo

given: Anna Maria

family: De Jesus-Acosta

full_name: Ana De Jesus-Acosta

given: Ana

name: Institut Claudius Regaud, IUCT-Oncopole, Toulouse, France

family: Delord

full_name: Jean-Pierre Delord

given: Jean-Pierre

name: Sourasky Medical Center, Tel Aviv, Israel

family: Geva

full_name: Ravit Geva

given: Ravit

family: Gottfried

full_name: Maya Gottfried

given: Maya

name: Centre Oscar Lambret and Lille University, Lille, France

family: Penel

full_name: Nicolas Penel

given: Nicolas

name: Princess Margaret Cancer Center, Toronto, Ontario, Canada

family: Hansen

full_name: Aaron R. Hansen

given: Aaron R.

name: The University of Texas MD Anderson Cancer Center, Houston, TX

family: Piha-Paul

full_name: Sarina A. Piha-Paul

given: Sarina A.

name: National Cancer Center Hospital East, Kashiwa, Japan

family: Doi

full_name: Toshihiko Doi

given: Toshihiko

name: Blacktown Hospital, Western Sydney Local Health District, Sydney, NSW, Australia

full_name: Bo Gao

given: Bo

name: Yonsei Cancer Center, Seoul, South Korea

family: Chung

full_name: Hyun Cheol Chung

given: Hyun Cheol

name: 12 de Octubre University Hospital and Research Institute, Madrid, Spain

family: Lopez-Martin

full_name: Jose Lopez-Martin

given: Jose

name: Seoul National University College of Medicine, Seoul, South Korea

family: Bang

full_name: Yung-Jue Bang

given: Yung-Jue

name: Chaim Sheba Medical Center, Ramat Gan, Israel

family: Frommer

full_name: Ronnie Shapira Frommer

given: Ronnie Shapira

name: Ohio State University Comprehensive Cancer Center, Columbus, OH

family: Shah

full_name: Manisha Shah

given: Manisha

family: Ghori

full_name: Razi Ghori

given: Razi

family: Joe

full_name: Andrew K. Joe

given: Andrew K.

family: Pruitt

full_name: Scott K. Pruitt

given: Scott K.

name: Memorial Sloan Kettering Cancer Center, New York, NY

family: Diaz Jr

full_name: Luis A. Diaz Jr

given: Luis A.

journal_name: Journal of Clinical Oncology

oa_host_type: repository

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

publisher: American Society of Clinical Oncology (ASCO)

publisher_dissemination: American Society of Clinical Oncology (ASCO)

publisher_group: American Society of Clinical Oncology (ASCO)

publisher_normalized: American Society of Clinical Oncology (ASCO)

title: Efficacy of Pembrolizumab in Patients With Noncolorectal High Microsatellite Instability/Mismatch Repair–Deficient Cancer: Results From the Phase II KEYNOTE-158 Study

ReferenceCitation: Yap TA, Nakagawa K, Fujimoto N, Kuribayashi K, Guren TK, Calabrò L, Shapira-Frommer R, Gao B, Kao S, Matos I, Planchard D, Chatterjee A, Jin F, Norwood K, Kindler HL. Efficacy and safety of pembrolizumab in patients with advanced mesothelioma in the open-label, single-arm, phase 2 KEYNOTE-158 study. Lancet Respir Med. 2021 Jun;9(6):613-621. doi: 10.1016/S2213-2600(20)30515-4. Epub 2021 Apr 6.

ReferenceCitation: Marabelle A, Fakih M, Lopez J, Shah M, Shapira-Frommer R, Nakagawa K, Chung HC, Kindler HL, Lopez-Martin JA, Miller WH Jr, Italiano A, Kao S, Piha-Paul SA, Delord JP, McWilliams RR, Fabrizio DA, Aurora-Garg D, Xu L, Jin F, Norwood K, Bang YJ. Association of tumour mutational burden with outcomes in patients with advanced solid tumours treated with pembrolizumab: prospective biomarker analysis of the multicohort, open-label, phase 2 KEYNOTE-158 study. Lancet Oncol. 2020 Oct;21(10):1353-1365. doi: 10.1016/S1470-2045(20)30445-9. Epub 2020 Sep 10.

ReferenceCitation: Piha-Paul SA, Oh DY, Ueno M, Malka D, Chung HC, Nagrial A, Kelley RK, Ros W, Italiano A, Nakagawa K, Rugo HS, de Braud F, Varga AI, Hansen A, Wang H, Krishnan S, Norwood KG, Doi T. Efficacy and safety of pembrolizumab for the treatment of advanced biliary cancer: Results from the KEYNOTE-158 and KEYNOTE-028 studies. Int J Cancer. 2020 Oct 15;147(8):2190-2198. doi: 10.1002/ijc.33013. Epub 2020 May 2.

name: Department of Investigational Cancer Therapeutics The University of Texas MD Anderson Cancer Center  Houston Texas USA

name: Department of Internal Medicine Seoul National University Hospital, and Cancer Research Institute, Seoul National University College of Medicine  Seoul Republic of Korea

name: Department of Gastroenterology Hepatobiliary and Pancreatic Medical Oncology Division, Kanagawa Cancer Center  Yokohama Japan

name: Département de Médecine Oncologique Gustave Roussy, Université Paris‐Saclay  Villejuif France

name: Department of Medical Oncology, Yonsei Cancer Center Yonsei University College of Medicine  Seoul Republic of Korea

name: Blacktown Cancer and Haematology Centre Blacktown Hospital and University of Sydney  Sydney New South Wales Australia

name: Division of Hematology/Oncology University of California San Francisco  San Francisco California USA

name: Division of Pharmacology Antoni van Leeuwenhoek Ziekenhuis  Amsterdam Netherlands

name: Early phase Trials and Sarcoma Units Institut Bergonié  Bordeaux France

name: Department of Medical Oncology Kindai University Hospital  Osaka Japan

name: Helen Diller Family Comprehensive Cancer Center University of California San Francisco  San Francisco California USA

name: Department of Oncology and Hemato‐Oncology University of Milan and Department of Medical Oncology and Hematology, Fondazione IRCCS Istituto Nazionale Tumori  Milan Italy

name: Department of Drug Development Gustave Roussy, Université Paris‐Saclay  Villejuif France

name: Division of Medical Oncology Princess Margaret Cancer Centre  Toronto Ontario Canada

name: Biostatistical and Research Decision Sciences, Merck & Co., Inc.  Kenilworth New Jersey USA

name: Oncology Late Development, Merck & Co., Inc.  Kenilworth New Jersey USA

name: Department of Experimental Therapeutics/Gastrointestinal Oncology National Cancer Center Hospital East  Kashiwa Japan

title: Efficacy and safety of pembrolizumab for the treatment of advanced biliary cancer: Results from the KEYNOTE ‐158 and KEYNOTE ‐028 studies

ReferenceCitation: Strosberg J, Mizuno N, Doi T, Grande E, Delord JP, Shapira-Frommer R, Bergsland E, Shah M, Fakih M, Takahashi S, Piha-Paul SA, O'Neil B, Thomas S, Lolkema MP, Chen M, Ibrahim N, Norwood K, Hadoux J. Efficacy and Safety of Pembrolizumab in Previously Treated Advanced Neuroendocrine Tumors: Results From the Phase II KEYNOTE-158 Study. Clin Cancer Res. 2020 May 1;26(9):2124-2130. doi: 10.1158/1078-0432.CCR-19-3014. Epub 2020 Jan 24.

publisher: American Association for Cancer Research (AACR)

publisher_dissemination: American Association for Cancer Research

publisher_group: American Association for Cancer Research

publisher_normalized: American Association for Cancer Research

ReferenceCitation: Chung HC, Ros W, Delord JP, Perets R, Italiano A, Shapira-Frommer R, Manzuk L, Piha-Paul SA, Xu L, Zeigenfuss S, Pruitt SK, Leary A. Efficacy and Safety of Pembrolizumab in Previously Treated Advanced Cervical Cancer: Results From the Phase II KEYNOTE-158 Study. J Clin Oncol. 2019 Jun 10;37(17):1470-1478. doi: 10.1200/JCO.18.01265. Epub 2019 Apr 3.

name: Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, South Korea

name: Antoni van Leeuwenhoek Ziekenhuis, Amsterdam, the Netherlands

name: Institut Claudius Regaud and Institut Universitaire du Cancer de Toulouse Oncopole, Toulouse, France

name: Rambam Health Care Campus, Technion - Israel Institute of Technology, Haifa, Israel

name: NN Blokhin National Medical Research Center of Oncology, Moscow, Russia

name: Gustave Roussy Cancer Campus and University of Paris-Saclay, Villejuif, France

location_country:
Australia
Russian Federation
Denmark
Taiwan
France
Japan
Norway
Canada
Korea, Republic of
Colombia
South Africa
Netherlands
Philippines
China
United States
Germany
Italy
Brazil
Mexico
Israel
Spain

location_facility: Call for Information (Investigational Site 0011)

location_facility: Call for Information (Investigational Site 0207)

location_facility: Call for Information (Investigational Site 0202)

location_facility: Call for Information (Investigational Site 0010)

location_facility: Call for Information (Investigational Site 0005)

location_facility: Call for Information (Investigational Site 0008)

location_facility: Call for Information (Investigational Site 0209)

location_facility: Call for Information (Investigational Site 0004)

location_facility: Call for Information (Investigational Site 0015)

location_facility: Call for Information (Investigational Site 0017)

title: A Clinical Trial of Pembrolizumab (MK-3475) Evaluating Predictive Biomarkers in Subjects With Advanced Solid Tumors (KEYNOTE 158)

summary: In this study, participants with multiple types of advanced (unresectable and/or metastatic) solid tumors who have progressed on standard of care therapy will be treated with pembrolizumab (MK-3475).


ReferenceCitation: Geoerger B, Kang HJ, Yalon-Oren M, Marshall LV, Vezina C, Pappo A, Laetsch TW, Petrilli AS, Ebinger M, Toporski J, Glade-Bender J, Nicholls W, Fox E, DuBois SG, Macy ME, Cohn SL, Pathiraja K, Diede SJ, Ebbinghaus S, Pinto N. Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1-2 trial. Lancet Oncol. 2020 Jan;21(1):121-133. doi: 10.1016/S1470-2045(19)30671-0. Epub 2019 Dec 4.

family: Geoerger

full_name: Birgit Geoerger

given: Birgit

family: Kang

full_name: Hyoung Jin Kang

given: Hyoung Jin

family: Yalon-Oren

full_name: Michal Yalon-Oren

given: Michal

family: Marshall

full_name: Lynley V Marshall

given: Lynley V

family: Vezina

full_name: Catherine Vezina

given: Catherine

family: Pappo

full_name: Alberto Pappo

given: Alberto

family: Laetsch

full_name: Theodore W Laetsch

given: Theodore W

family: Petrilli

full_name: Antonio S Petrilli

given: Antonio S

family: Ebinger

full_name: Martin Ebinger

given: Martin

family: Toporski

full_name: Jacek Toporski

given: Jacek

family: Glade-Bender

full_name: Julia Glade-Bender

given: Julia

family: Nicholls

full_name: Wayne Nicholls

given: Wayne

family: Fox

full_name: Elizabeth Fox

given: Elizabeth

family: DuBois

full_name: Steven G DuBois

given: Steven G

family: Macy

full_name: Margaret E Macy

given: Margaret E

family: Cohn

full_name: Susan L Cohn

given: Susan L

family: Pathiraja

full_name: Kumudu Pathiraja

given: Kumudu

family: Diede

full_name: Scott J Diede

given: Scott J

family: Ebbinghaus

full_name: Scot Ebbinghaus

given: Scot

family: Pinto

full_name: Navin Pinto

given: Navin

journal_name: The Lancet Oncology

title: Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1–2 trial

intervention_type: Biological

lead_sponsor: Merck Sharp & Dohme Corp.

location_country:
Italy
Brazil
Sweden
Germany
France
Israel
Australia
United States
United Kingdom
Korea, Republic of

acronym: A Phase I/II trial of MK-3475 (pembrolizumab) in children’s solid tumors and lymphoma

location_facility: Call for Information (Investigational Site 0069)

location_facility: Call for Information (Investigational Site 0012)

location_facility: Call for Information (Investigational Site 0054)

location_facility: Call for Information (Investigational Site 0056)

location_facility: Call for Information (Investigational Site 0019)

location_facility: Call for Information (Investigational Site 0026)

location_facility: Call for Information (Investigational Site 0014)

location_facility: Call for Information (Investigational Site 0025)

location_facility: Call for Information (Investigational Site 0031)

title: A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)

summary:
This is a two-part study of pembrolizumab (MK-3475) in pediatric participants who have any of the following types of cancer:

advanced melanoma (6 months to <18 years of age),
advanced, relapsed or refractory programmed death-ligand 1 (PD-L1)-positive malignant solid tumor or other lymphoma (6 months to <18 years of age),
relapsed or refractory classical Hodgkin lymphoma (rrcHL) (3 years to <18 years of age), or
advanced relapsed or refractory microsatellite-instability-high (MSI-H) solid tumors (6 months to <18 years of age), or
advanced relapsed or refractory tumor-mutational burden-high ≥10 mutation/Mb (TMB-H) solid tumors (6 months to <18 years of age)

Part 1 will find the maximum tolerated dose (MTD)/maximum administered dose (MAD), confirm the dose, and find the recommended Phase 2 dose (RP2D) for pembrolizumab therapy. Part 2 will further evaluate the safety and efficacy at the pediatric RP2D.

The primary hypothesis of this study is that intravenous (IV) administration of pembrolizumab to children with either advanced melanoma; a PD-L1 positive advanced, relapsed or refractory solid tumor or other lymphoma; advanced, relapsed or refractory MSI-H solid tumor; or rrcHL, will result in an Objective Response Rate (ORR) greater than 10% for at least one of these types of cancer. The 10% assessment does not apply to the MSI-H and TMB-H cohorts.

With Amendment 8, enrollment of participants with solid tumors and of participants aged 6 months to <12 years with melanoma were closed. Enrollment of participants aged ≥12 years to ≤18 years with melanoma continues. Enrollment of participants with MSI-H and TMB-H solid tumors also continues.


title: European Dyspnoea Survey in the EMergency Departments

acronym: EuroDEM

summary:
Braunwald defines dyspnoea as an abnormally uncomfortable awareness of breathing. Breathing discomfort, and its varying degrees of severity, is the one of the most disturbing symptoms patients can experience; and it is one of the main complaints in the patients presenting to the Emergency Department (ED). Dyspnea has a variety of underlying etiologies, like cardiac, pulmonary or metabolic etiologies or a combination of them, since several diseases can cause dyspnea like for instance heart failure (HF), asthma and chronic obstructive pulmonary disease (COPD).

Acute heart failure syndrome (AHFS) is collectively defined as a gradual or rapid change in heart failure (HF) signs and symptoms resulting in a need for urgent therapy. Heart failure (HF) is one of the most important causes of morbidity and mortality in the industrialized world. The prevalence of symptomatic HF is estimated to range from 0.4 to 2.0% in general European population. The incidence increases rapidly with age, and in Europe. Characteristics, clinical presentation, treatment, and outcomes of HF patients admitted to hospital have been adequately described, in Europe and in the United States. The Euro Heart Failure Survey (EHFS) I with 11 327 patients described the demographics of acutely hospitalized HF patients. The ADHERE registry has data on over 100 000 hospitalizations for AHF from the USA. In-hospital mortality was 4 and 7%, in ADHERE and EHFS I, respectively.

This same sensation of breathlessness is what also drives patients with asthma and chronic obstructive pulmonary disease (COPD) to the ED. Chronic obstructive pulmonary disease (COPD) exacerbation accounts for approximately 1.5 million ED visits in the United States per year. It is the third most common cause of hospitalization, with an estimated 726 000 hospitalizations in 2000 in the USA. Previous studies have demonstrated important differences between guideline recommendations and actual management of COPD exacerbation, either in the ED or during hospitalization.

The diagnosis in front of a dyspneic patient in the ED remains a challenge, because of a low sensitivity of the clinical signs associated with the aging of the population and the variety of underlying diseases. Little is known about the Epidemiology of dyspneic patients in the ED at the European level. Diagnosis, prevalence and treatment of the patients may vary among European countries.

lead_sponsor: Hopital Lariboisière

location_country:
Italy
Netherlands
Finland
France
Romania
Belgium
Spain
Turkey
Germany
United Kingdom

location_facility:
Country: Netherlands
Country: Turkey
Country: Italy
Country: Belgium
Country: France
Country: Finland
Country: Germany
Country: Spain
Country: Romania
Country: United Kingdom


ReferenceCitation: Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet J Rare Dis. 2014 Dec 18;9:199. doi: 10.1186/s13023-014-0199-0. Erratum in: Orphanet J Rare Dis. 2016;11(1):92.

family: Attarian

full_name: Shahram Attarian

given: Shahram

family: Vallat

full_name: Jean-Michel Vallat

given: Jean-Michel

family: Magy

full_name: Laurent Magy

given: Laurent

family: Funalot

full_name: Benoît Funalot

given: Benoît

family: Gonnaud

full_name: Pierre-Marie Gonnaud

given: Pierre-Marie

family: Lacour

full_name: Arnaud Lacour

given: Arnaud

family: Péréon

full_name: Yann Péréon

given: Yann

family: Dubourg

full_name: Odile Dubourg

given: Odile

family: Pouget

full_name: Jean Pouget

given: Jean

family: Micallef

full_name: Joëlle Micallef

given: Joëlle

family: Franques

full_name: Jérôme Franques

given: Jérôme

family: Lefebvre

full_name: Marie-Noëlle Lefebvre

given: Marie-Noëlle

family: Ghorab

full_name: Karima Ghorab

given: Karima

family: Al-Moussawi

full_name: Mahmoud Al-Moussawi

given: Mahmoud

family: Tiffreau

full_name: Vincent Tiffreau

given: Vincent

family: Preudhomme

full_name: Marguerite Preudhomme

given: Marguerite

family: Magot

full_name: Armelle Magot

given: Armelle

family: Leclair-Visonneau

full_name: Laurène Leclair-Visonneau

given: Laurène

family: Stojkovic

full_name: Tanya Stojkovic

given: Tanya

family: Bossi

full_name: Laura Bossi

given: Laura

family: Lehert

full_name: Philippe Lehert

given: Philippe

family: Gilbert

full_name: Walter Gilbert

given: Walter

family: Bertrand

full_name: Viviane Bertrand

given: Viviane

family: Mandel

full_name: Jonas Mandel

given: Jonas

family: Milet

full_name: Aude Milet

given: Aude

repository_institution: University of Melbourne - Minerva Access

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

ReferenceCitation: Chumakov I, Milet A, Cholet N, Primas G, Boucard A, Pereira Y, Graudens E, Mandel J, Laffaire J, Foucquier J, Glibert F, Bertrand V, Nave KA, Sereda MW, Vial E, Guedj M, Hajj R, Nabirotchkin S, Cohen D. Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy. Orphanet J Rare Dis. 2014 Dec 10;9:201. doi: 10.1186/s13023-014-0201-x.

repositories_institution:
Max Planck Society - MPG.PuRe
PubMed Central - Europe PMC

ReferenceCitation: Mandel J, Bertrand V, Lehert P, Attarian S, Magy L, Micallef J, Chumakov I, Scart-Grès C, Guedj M, Cohen D. A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment. Orphanet J Rare Dis. 2015 Jun 13;10:74. doi: 10.1186/s13023-015-0293-y.

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Inria

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Aix-Marseille Université

ReferenceCitation: Prukop T, Stenzel J, Wernick S, Kungl T, Mroczek M, Adam J, Ewers D, Nabirotchkin S, Nave KA, Hajj R, Cohen D, Sereda MW. Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A (CMT1A). PLoS One. 2019 Jan 16;14(1):e0209752. doi: 10.1371/journal.pone.0209752. eCollection 2019.

ReferenceCitation: Hajj R, Prukop T, Wernick S, Ewers D, Brureau A, Cholet N, Laffaire J, Nave KA, Cohen D, Sereda M. Baclofen, Naltrexone and Sorbitol all contribute to the efficacy of PXT3003 in CMT1A Rats. EMJ Neurol, 2019;7[1]:47-49

ReferenceCitation: Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. Erratum to: An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet J Rare Dis. 2016 Jul 7;11(1):92.

location_country:
United States
Netherlands
Germany
France
Belgium
Spain
Canada
United Kingdom

location_facility: Centre de Référence des Maladies Neuromusculaires, Pôle des Neurosciences Clinique, CHU la Timone

location_facility: Department for Sleep Medicine and Neuromuscular, University Hospital Münster

location_facility: Centro de Diagnostico y Tratamiento, Hospital Universitario Virgen del Rocio

location_facility: Servicio de Neurologia, Hospital Univesitari i Politécnic La Fe

location_facility: Service de Neurologie, Hôpital Kremlin Bicêtre

location_facility: Centre de Référence des Maladies Neuromusculaires; Hôtel Dieu, CHU de Nantes

location_facility: Department of Neurology and Institute for Neuropathology, University Hospital RWTH Aachen

location_facility: Servicio de Neurologia, Hospital Universitario La Paz

location_facility: Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center

location_facility: Department of Neurology, University of Minnesota

location_facility: Service de Neurologie et du Sommeil, CHU Lyon Sud

location_facility: Centre de Référence des Neuropathies Périphériques Rares, Hôpital Dupuytren, CHU Limoges

location_facility: Department of Neurology, Cedars-Sinai Medical Center

location_facility: Centre de Référence des Maladies Neuromusculaires, Hôpital Swynghedauwl, CHU de Lille

location_facility: Department of Neurology, McKnight Brain Institute

location_facility: Departement of Neurology, Academic Medical Center

location_facility: Department of Clinical Neurophysiology, University Medical Center Göttingen

location_facility: Department of Neurology and Psichiatry, Saint Louis University

location_facility: Department of Neurology, Ludwig-Maximillian University, Munich

location_facility: Department of Neurology, Salford Royal NHS Foundation Trust

location_facility: Department of neurology, Hospital Univesitario de Bellvitge

title: International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months

summary: The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.


title: Thrombolysis or Anticoagulation for Cerebral Venous Thrombosis (TOACT)

acronym: TOACT

summary:
Background: Endovascular thrombolysis, with or without mechanical clot removal (ET), may be beneficial for a subgroup of patients with cerebral venous sinus thrombosis (CVT), who have a poor prognosis despite treatment with heparin. Published experience with ET is promising, but only based on case series and not on controlled trials.

Objective: The main objective of the TO-ACT trial is to determine if ET improves the functional outcome of patients with a severe form of CVT

Study design: The TO-ACT trial will be designed as a multi-centre, prospective, randomized, open-label, blinded endpoint (PROBE) trial.

Study population: Patients are eligible if they have a radiologically proven CVT, a high probability of poor outcome (defined by presence of one or more of the following risk factors: mental status disorder, coma, intracranial hemorrhagic lesion or thrombosis of the deep cerebral venous system) and the responsible physician is uncertain if ET or standard anti-coagulant treatment is better.

Intervention: Patients will be randomized to receive either ET or standard therapy (therapeutic doses of heparin). ET consists of local application of alteplase or urokinase within the thrombosed sinuses, and/or mechanical thrombectomy. Glasgow coma score, NIH stroke scale and relevant laboratory parameters will be assessed at baseline.

Endpoints: The primary endpoint is the modified Rankin scale (mRS) at 12 months. The most important secondary outcomes are the mRS, mortality and recanalization rate at 6 months. Major intra- and extracranial hemorrhagic complications within one week following the intervention are the principal safety outcome. Results will be analyzed according to the "intention-to-treat" principle. Assessment of study endpoints will be carried out according to standardized questionnaires by a blinded neurologist or research nurse who is not involved in the treatment of the patient.

Study size: To detect a 50% relative reduction in mRS≥2 (from 40 to 20%), 164 patients (82 in each treatment arm) have to be included (two-sided alpha, 80% power).

Nature and extent of the burden and risks associated with participation, benefit and group relatedness: Included patients may benefit directly from ET. Complications of ET, most notably intracranial hemorrhages, constitute the most important risk of the study.

status: Terminated

ReferenceCitation: Coutinho JM, Stam J. How to treat cerebral venous and sinus thrombosis. J Thromb Haemost. 2010 May;8(5):877-83. doi: 10.1111/j.1538-7836.2010.03799.x. Epub 2010 Feb 9. Review.

ReferenceCitation: Canhão P, Falcão F, Ferro JM. Thrombolytics for cerebral sinus thrombosis: a systematic review. Cerebrovasc Dis. 2003;15(3):159-66. Review.

ReferenceCitation: Ciccone A, Canhão P, Falcão F, Ferro JM, Sterzi R. Thrombolysis for cerebral vein and dural sinus thrombosis. Cochrane Database Syst Rev. 2004;(1):CD003693. Review.

ReferenceCitation: Stam J, Majoie CB, van Delden OM, van Lienden KP, Reekers JA. Endovascular thrombectomy and thrombolysis for severe cerebral sinus thrombosis: a prospective study. Stroke. 2008 May;39(5):1487-90. doi: 10.1161/STROKEAHA.107.502658. Epub 2008 Mar 13.

ReferenceCitation: Coutinho JM, Zuurbier SM, Bousser MG, Ji X, Canhão P, Roos YB, Crassard I, Nunes AP, Uyttenboogaart M, Chen J, Emmer BJ, Roosendaal SD, Houdart E, Reekers JA, van den Berg R, de Haan RJ, Majoie CB, Ferro JM, Stam J; TO-ACT investigators. Effect of Endovascular Treatment With Medical Management vs Standard Care on Severe Cerebral Venous Thrombosis: The TO-ACT Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):966-973. doi: 10.1001/jamaneurol.2020.1022.

name: Department of Neurology, Amsterdam University Medical Centers, Amsterdam, the Netherlands

family: Coutinho

full_name: Jonathan M. Coutinho

given: Jonathan M.

family: Zuurbier

full_name: Susanna M. Zuurbier

given: Susanna M.

name: Department of Neurology, Hôpital Lariboisière, Paris, France

family: Bousser

full_name: Marie-Germaine Bousser

given: Marie-Germaine

name: Department of Interventional Radiology, XuanWu Hospital, Beijing, China

family: Ji

full_name: Xunming Ji

given: Xunming

name: Serviço de Neurologia, Instituto de Medicina Molecular, Hospital Santa Maria/Centro Hospitalar Lisboa Norte, Department of Neurosciences and Mental Health, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal

family: Canhão

full_name: Patricia Canhão

given: Patricia

family: Roos

full_name: Yvo B. Roos

given: Yvo B.

family: Crassard

full_name: Isabelle Crassard

given: Isabelle

name: Department of Neurology, Centro Hospitalar Universitário de Lisboa Central, Lisbon, Portugal

family: Nunes

full_name: Ana Paiva Nunes

given: Ana Paiva

name: Department of Neurology, University Medical Center Groningen, Groningen, the Netherlands

family: Uyttenboogaart

full_name: Maarten Uyttenboogaart

given: Maarten

family: Chen

full_name: Jian Chen

given: Jian

name: Department of Radiology, Amsterdam University Medical Centers, Amsterdam, the Netherlands

family: Emmer

full_name: Bart J. Emmer

given: Bart J.

family: Roosendaal

full_name: Stefan D. Roosendaal

given: Stefan D.

name: Department of Neuroradiology, Hôpital Lariboisière, Paris, France

family: Houdart

full_name: Emmanuel Houdart

given: Emmanuel

family: Reekers

full_name: Jim A. Reekers

given: Jim A.

family: van den Berg

full_name: René van den Berg

given: René

name: Clinical Research Unit, Amsterdam University Medical Centers, Amsterdam, the Netherlands

family: de Haan

full_name: Rob J. de Haan

given: Rob J.

family: Majoie

full_name: Charles B. Majoie

given: Charles B.

family: Ferro

full_name: José M. Ferro

given: José M.

family: Stam

full_name: Jan Stam

given: Jan

journal_name: JAMA Neurology

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via free pdf)

ReferenceCitation: Coutinho JM, Ferro JM, Zuurbier SM, Mink MS, Canhão P, Crassard I, Majoie CB, Reekers JA, Houdart E, de Haan RJ, Bousser MG, Stam J. Thrombolysis or anticoagulation for cerebral venous thrombosis: rationale and design of the TO-ACT trial. Int J Stroke. 2013 Feb;8(2):135-40. doi: 10.1111/j.1747-4949.2011.00753.x. Epub 2012 Feb 20.

name: Department of Neurology, Academic Medical Centre, Amsterdam, The Netherlands

name: Department of Neurology, Hospital Santa Maria, University of Lisbon, Lisbon, Portugal

name: Department of Radiology, Academic Medical Centre, Amsterdam, The Netherlands

name: Clinical Research Unit, Academic Medical Centre, Amsterdam, The Netherlands

title: Thrombolysis or Anticoagulation for Cerebral Venous Thrombosis: Rationale and Design of the TO-ACT Trial

location_country: Switzerland Netherlands France Portugal China Canada

location_facility: Centre hospitalier de l'université de Montréal (CHUM)


title: A 24-Week, Double-Blind, Parallel Group, Placebo and Active Controlled Study to Investigate the Efficacy and Safety of Daily Oral Roflumilast Taken With Low Dose Inhaled Corticosteroids in Patients With Chronic Asthma

summary:
Bronchial asthma is among the world's most prevalent diseases. Roflumilast is a novel, orally active, selective enzyme inhibitor (phosphodiesterase 4 inhibitor), which has shown effectiveness in the treatment of asthma.

The aim of the study is to investigate the effect of roflumilast taken orally together with low dose inhaled corticosteroids on lung function. Roflumilast will be administered at one dose level once daily together with inhaled corticosteroids at one dose level twice daily. The study duration consists of a baseline period (2 to 6 weeks) and a treatment period (24 weeks). The study will provide further data on safety, tolerability, and effectiveness of roflumilast.

ReferenceCitation: Chervinsky P, Meltzer EO, Busse W, Ohta K, Bardin P, Bredenbröker D, Bateman ED. Roflumilast for asthma: Safety findings from a pooled analysis of ten clinical studies. Pulm Pharmacol Ther. 2015 Dec;35 Suppl:S28-34. doi: 10.1016/j.pupt.2015.11.003. Epub 2015 Nov 22.

family: Chervinsky

full_name: P. Chervinsky

given: P.

family: Meltzer

full_name: E.O. Meltzer

family: Busse

full_name: W. Busse

given: W.

family: Ohta

full_name: K. Ohta

given: K.

family: Bardin

full_name: P. Bardin

family: Bredenbröker

full_name: D. Bredenbröker

given: D.

family: Bateman

full_name: E.D. Bateman

journal_name: Pulmonary Pharmacology & Therapeutics

ReferenceCitation: Meltzer EO, Chervinsky P, Busse W, Ohta K, Bardin P, Bredenbröker D, Bateman ED. Roflumilast for asthma: Efficacy findings in placebo-controlled studies. Pulm Pharmacol Ther. 2015 Dec;35 Suppl:S20-7. doi: 10.1016/j.pupt.2015.10.006. Epub 2015 Oct 21.

lead_sponsor: AstraZeneca

location_country:
Finland
Greece
Russian Federation
Czech Republic
Taiwan
India
Pakistan
Croatia
France
Poland
Thailand
Norway
South Africa
Philippines
Singapore
Hungary
Ireland
Portugal
United States
United Kingdom
Italy
Austria
New Zealand
Spain


lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
Brazil
South Africa
Sweden
India
France
Poland
Hungary
Belgium
Turkey
United States

location_facility:
Sites in France
Sites in Poland
Sites in USA
Sites in Sweden
Sites in Belgium
Sites in Turkey
sites in Italy
Sites in Brazil
Sites in Hungary
Sites in South Africa
Sites in India

title: A Randomized, Multicenter, Double-blind, 6 Week Study to Evaluate the Dose Response of Valsartan on Blood Pressure Reduction in Children 6 Months - 5 Years Old With Hypertension, Followed by a 2 Week Placebo Withdrawal Period.

summary: The purpose of this study is to evaluate the safety and efficacy of three doses of valsartan (0.25, 1.0, and 4.0 mg/kg) on mean sitting systolic blood pressure (MSSBP) and mean sitting diastolic blood pressure (MSDBP) in 6 months - 5 year old children with hypertension (sitting systolic blood pressure [SSBP] ≥ 95th percentile ).


title: MOnitoring REsynchronization deviCes and cARdiac patiEnts

acronym: MORE-CARE

summary:
The objective of this study is to compare two different strategies of disease management in heart failure patients treated with cardiac resynchronization therapy devices

Remote monitoring with CareLink Network System
Standard management of the disease by means of scheduled routine in-patient follow-ups;

and to demonstrate that the remote monitoring strategy is superior to the standard strategy, both in terms of clinical effectiveness and total healthcare system utilization.

ReferenceCitation: Burri H, Quesada A, Ricci RP, Boriani G, Davinelli M, Favale S, Da Costa A, Kautzner J, Moser R, Navarro X, Santini M. The MOnitoring Resynchronization dEvices and CARdiac patiEnts (MORE-CARE) study: rationale and design. Am Heart J. 2010 Jul;160(1):42-8. doi: 10.1016/j.ahj.2010.04.005.

ReferenceCitation: Boriani G, Da Costa A, Ricci RP, Quesada A, Favale S, Iacopino S, Romeo F, Risi A, Mangoni di S Stefano L, Navarro X, Biffi M, Santini M, Burri H; MORE-CARE Investigators. The MOnitoring Resynchronization dEvices and CARdiac patiEnts (MORE-CARE) randomized controlled trial: phase 1 results on dynamics of early intervention with remote monitoring. J Med Internet Res. 2013 Aug 21;15(8):e167. doi: 10.2196/jmir.2608.

family: Boriani

full_name: Giuseppe Boriani

given: Giuseppe

family: Da Costa

full_name: Antoine Da Costa

given: Antoine

family: Ricci

full_name: Renato Pietro Ricci

given: Renato Pietro

family: Quesada

full_name: Aurelio Quesada

given: Aurelio

family: Favale

full_name: Stefano Favale

given: Stefano

family: Iacopino

full_name: Saverio Iacopino

given: Saverio

family: Romeo

full_name: Francesco Romeo

given: Francesco

family: Risi

full_name: Arnaldo Risi

given: Arnaldo

family: Mangoni di S Stefano

full_name: Lorenza Mangoni di S Stefano

given: Lorenza

family: Navarro

full_name: Xavier Navarro

given: Xavier

family: Biffi

full_name: Mauro Biffi

given: Mauro

family: Santini

full_name: Massimo Santini

given: Massimo

family: Burri

full_name: Haran Burri

given: Haran

name: On Behalf Of The MORE-CARE Investigators

journal_name: Journal of Medical Internet Research

oa_colors: green gold

license: cc-by

license_normalized: cc-by

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

repository_institution: PubMed Central - Europe PMC

version: submittedVersion

evidence: oa repository (semantic scholar lookup)

publisher: JMIR Publications Inc.

publisher_dissemination: JMIR Publications

publisher_group: JMIR Publications

publisher_normalized: JMIR Publications

title: The MOnitoring Resynchronization dEvices and CARdiac patiEnts (MORE-CARE) Randomized Controlled Trial: Phase 1 Results on Dynamics of Early Intervention With Remote Monitoring

ReferenceCitation: Burri H, da Costa A, Quesada A, Ricci RP, Favale S, Clementy N, Boscolo G, Villalobos FS, Mangoni di S Stefano L, Sharma V, Boriani G; MORE-CARE Investigators. Risk stratification of cardiovascular and heart failure hospitalizations using integrated device diagnostics in patients with a cardiac resynchronization therapy defibrillator. Europace. 2018 May 1;20(5):e69-e77. doi: 10.1093/europace/eux206.

name: Department of Cardiology, University Hospital of Geneva, Rue Gabrielle Perret Gentil 4, 1211 Geneva 14, Switzerland

name: CHU St-Etienne, Hôpital Nord Avenue Albert Raimond, 42270 Saint-Priest-en-Jarez, France

name: University General Hospital, Avda. Tres Cruces, s/n de Valencia, 46014 Valencia, Spain

name: San Filippo Neri Hospital, via Martinotti, 20, 00136 Roma, Italy

name: Azienda Ospedaliera Policlinico di Bari, Piazza Giulio Cesare, 11, Bari 70124, Italy

name: Tours University Hospital, Avenue de la République, 37170 Chambray-lés-Tours, France

name: Chioggia ULSS 14, Strada Madonna Marina 500, 30015 Chioggia (VE), Italy

name: Hospital Universitario Insular, Las Palmas, Avda. Marìtima del Sur s/n, 35016 Las Palmas de Gran Canaria, Spain

name: Medtronic EMEA Regional Clinical Center, Via Aurelia 475-477, 00165 Rome, Italy

name: Medtronic Plc., Mounds View Facility North, 8200 Coral Sea Street N.E., Mounds View, MN 55112, USA

name: University of Bologna, S. Orsola-Malpighi University Hospital, Via Albertoni, 15, Bologna, Italy

name: University of Modena and Reggio Emilia, Policlinico di Modena, via del Pozzo 71, 41124 Modena, Italy

name: On behalf of the MORE-CARE Investigators

ReferenceCitation: Boriani G, Da Costa A, Quesada A, Ricci RP, Favale S, Boscolo G, Clementy N, Amori V, Mangoni di S Stefano L, Burri H; MORE-CARE Study Investigators. Effects of remote monitoring on clinical outcomes and use of healthcare resources in heart failure patients with biventricular defibrillators: results of the MORE-CARE multicentre randomized controlled trial. Eur J Heart Fail. 2017 Mar;19(3):416-425. doi: 10.1002/ejhf.626. Epub 2016 Aug 28.

name: University of Modena and Reggio Emilia; Policlinico di Modena; Modena Italy

name: University of Bologna; S. Orsola-Malpighi University Hospital; Bologna Italy

name: University General Hospital; Valencia Spain

name: Medtronic EMEA Regional Clinical Center; Rome Italy

name: University Hospital of Geneva; Geneva Switzerland

name: on behalf of the MORE-CARE Study Investigators

repository_institution: University of Geneva - Archive ouverte UNIGE

location_country:
Italy
Switzerland
Netherlands
France
Greece
Israel
Slovakia
Hungary
Spain
Czech Republic

location_facility: Institut Klinicke a Experimentalni Mediciny

location_facility: Azienda Ospedaliera Sacro Cuore Don Calabria

location_facility: Ospedale Civile Maggiore di Borgo Trento

location_facility: Centre Hospitalier Universitaire de Angers

location_facility: Azienda Complesso Ospedaliero San Filippo Neri

location_facility: Gottsegen György Országos Kardiológia Intézet GOKI

location_facility: Complejo Hospitalario Universitario de Vigo

location_facility: Policlinico Universitario S. Orsola-Malpighi


title: Safety and Efficacy of Orally Administered Fx-1006A in Patients With Familial Amyloid Polyneuropathy (FAP): A Randomized, Double-blind, Placebo-controlled Study

summary:
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP).

Deposition of TTR amyloid is associated with a variety of human diseases. Deposition of amyloid fibrils of variant TTR (primarily V30M) in peripheral nerve tissue produces the condition called FAP.

The prevention of the formation of amyloid by stabilization of the TTR native state should constitute an effective therapy for amyloid diseases. Therapeutic intervention with a TTR stabilizer drug, such as Fx-1006A, is hypothesized to stop progression of the disease in FAP patients. FAP is a uniformly fatal disease and Fx-1006A is intended to halt the relentless neurological deterioration FAP patients experience.

This Phase 2/3 study will enroll early to mid-stage FAP patients in order to interrupt and stabilize the disease at a point in time where progression of motor and autonomic dysfunction can be maximally effected. Male and female patients with FAP with documented V30M TTR mutation will receive Fx-1006A or placebo once daily for a period of eighteen (18) months.

ReferenceCitation: Merlini G, Coelho T, Waddington Cruz M, Li H, Stewart M, Ebede B. Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years. Neurol Ther. 2020 Jun;9(1):105-115. doi: 10.1007/s40120-020-00180-w. Epub 2020 Feb 27.

family: Merlini

full_name: Giampaolo Merlini

given: Giampaolo

family: Coelho

full_name: Teresa Coelho

given: Teresa

family: Waddington Cruz

full_name: Márcia Waddington Cruz

given: Márcia

family: Li

full_name: Huihua Li

given: Huihua

family: Stewart

full_name: Michelle Stewart

given: Michelle

family: Ebede

full_name: Ben Ebede

given: Ben

journal_name: Neurology and Therapy

license: cc-by-nc

license_normalized: cc-by-nc

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B, Gundapaneni B, Schwartz JH. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-209. doi: 10.1080/13506129.2019.1643714. Epub 2019 Jul 27.

family: Huber

full_name: Peter Huber

given: Peter

family: Flynn

full_name: Alison Flynn

given: Alison

family: Sultan

full_name: Marla B. Sultan

given: Marla B.

family: Rill

full_name: Denise Rill

given: Denise

family: Gundapaneni

full_name: Balarama Gundapaneni

given: Balarama

family: Schwartz

full_name: Jeffrey H. Schwartz

given: Jeffrey H.

journal_name: Amyloid

evidence: open (via crossref license)

ReferenceCitation: Amass L, Li H, Gundapaneni BK, Schwartz JH, Keohane DJ. Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy. Orphanet J Rare Dis. 2018 Dec 17;13(1):225. doi: 10.1186/s13023-018-0947-7.

ReferenceCitation: Gundapaneni BK, Sultan MB, Keohane DJ, Schwartz JH. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2018 Mar;25(3):464-468. doi: 10.1111/ene.13510. Epub 2017 Dec 26.

title: Tafamidis delays neurological progression comparably across Val30Met and non‐Val30Met genotypes in transthyretin familial amyloid polyneuropathy

ReferenceCitation: Keohane D, Schwartz J, Gundapaneni B, Stewart M, Amass L. Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial. Amyloid. 2017 Mar;24(1):30-36. doi: 10.1080/13506129.2017.1301419. Epub 2017 Apr 10.

name: inVentiv Health Inc., Burlington, MA, USA

ReferenceCitation: Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016 Sep;23(3):178-183. Epub 2016 Aug 5.

location_country:
Brazil
Sweden
Spain
Germany
France
Argentina
Portugal
United States
United Kingdom

location_facility: FLENI-Hepatology and Organ Transplant Dept.

location_facility: Universitatsklinikum Munster, Transplant Hepatology

location_facility: Hospital Universitário Prof. Clementino Fraga Filho-UFRJ

location_facility: Unidade Clinica de Paramiloidose-Hospital Santo Antonio

location_facility: Serviço de Neurologia-Hospital de Santa Maria