title: An Open-Label Extension Of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

summary:
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.

All patients who enroll in this extension study will receive once-daily oral 20 mg Fx-1006A for 12 months; therefore, patients randomized to placebo in Study Fx-005 will cross over to active drug (Fx-1006A 20 mg) during this study. However, patients and their families as well as clinical Investigators and their clinical site staff will remain blinded to the original Fx-005 treatment assignment. It is intended that there will be no interruption in study medication administration between the two studies. The majority of safety and clinical outcomes assessments will be identical to those evaluated in Study Fx-005. Additional assessments for this open-label extension study include 24-hour Holter monitoring and skin biopsy for IENF; patients will be required to provide written informed consent to participate in this open-label extension study prior to having these additional procedures performed.

The values obtained from procedures and evaluations conducted during the Month 18 visit of Study Fx-005 will be used as the Baseline values for this open-label extension study. The Baseline assessments of IENF and Holter monitoring may be conducted at either day of the Month 18 visit days of Study Fx-005, but prior to the first Fx-1006A dose in this open-label extension study. Clinic Visits will be conducted at Week 6 (± 2 days), and Month 3 (± 1 week), Month 6 (± 2 weeks), and Month 12 (± 2 weeks). Monthly telephone contacts (± 1 week of the scheduled date) will be made during months in which no investigative site visits are scheduled (Months 2, 4, 5, 7, 8, 9, 10, and 11) for assessment of adverse events and concomitant medications.

Neurological evaluation by NIS-LL will be performed at Months 6 and 12. The NIS-LL will be assessed by utilizing the average of two successive NIS-LL clinical assessment scores obtained at least 24 hours apart within a one week period for each study visit. A dedicated neurologist will be required to perform NIS-LL scoring across all time-points for each individual patient enrolled in the study.

Quality of life utilizing the Norfolk QOL-DN will be assessed at Months 6 and 12 (based on the total score as well as the five individual domains of the questionnaire).

QST (utilizing CASE IV), NCS, HRDB, mBMI, and echocardiography will be conducted at Months 6 and 12. Holter monitoring will be conducted at Baseline and Months 6 and 12. Biopsies for IENF will be obtained at Baseline only. Assessments of troponin I and NT-pro-BNP levels will be made at each study visit.

Blood samples for pharmacokinetic assessments (Fx-1006A concentrations as well as calculated steady-state parameters) and pharmacodynamic assessments (TTR stabilization) will be collected at Week 6 and Months 6 and 12.

Safety and tolerability will be assessed throughout the study. Vital signs, 12-lead ECG, blood and urine samples for clinical laboratory tests (serum chemistry, hematology, coagulation panel, urinalysis, and urine pregnancy testing), adverse events, and concomitant medications will be assessed at each study visit. Eye examinations (including fundal photography) will be conducted at Months 6 and 12. Abbreviated physical examinations will be conducted at Week 6, and Months 3 and 6, and a complete physical examination will be conducted at Month 12.

All patients will be contacted by telephone 30 days (± 1 week) after the last dose of study medication for assessment of adverse events and concomitant medications.

Patients who complete the Month 12 visit of this open-label study may be allowed to continue receiving Fx-1006A under a compassionate use program.

Patients who discontinue from the study at any time after enrollment (i.e., early termination) will have final safety assessments performed at the time of discontinuation. Any patient discontinuing after the Month 6 visit will have all safety and clinical outcomes assessments scheduled for the Month 12 visit performed.

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

title: Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years

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.

title: A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy

title: Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy

ReferenceCitation: Coelho T, Maia LF, da Silva AM, Cruz MW, Planté-Bordeneuve V, Suhr OB, Conceiçao I, Schmidt HH, Trigo P, Kelly JW, Labaudinière R, Chan J, Packman J, Grogan DR. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol. 2013 Nov;260(11):2802-14. doi: 10.1007/s00415-013-7051-7. Epub 2013 Aug 22.

repository_institution: RCAAP Project by FCT - Portuguese National Funding Agency for Science, Research and Technology - RCAAP Search Portal

location_country: Brazil Sweden France Argentina Portugal Germany

location_facility: FLENI Departamento de Hepatología y Transplante de Organos

location_facility: FLENI-Hepatology and Organ Transplant Dept.

location_facility: Universitatsklinikum Munster, Transplant Hepatology

location_facility: FAP-Teamet Familjar Amyloids, Norrlands universitetssjukhus

location_facility: Servicio de Neurologica Piso 7, Hospital de Santa Maria

location_facility: Unidade Clinica de Paramiloidose-Hospital Santo Antonio

location_facility: Unidade Clinica de Paramiloidose, Hospital Geral de Santo Antonio, Largo Prof Abdel Salazar

location_facility: Serviço de Neurologia-Hospital de Santa Maria

location_facility: Hospital Universitario Clementino Fraga Filho


title: A Randomized, Double-masked, Multicenter, Laser-controlled Phase III Study Assessing the Efficacy and Safety of Ranibizumab (Intravitreal Injections) as Adjunctive and Mono-therapy in Patients With Visual Impairment Due to Diabetic Macular Edema

acronym: RESTORE

summary:
CRFB002D2301: The core study was designed to confirm the efficacy and safety of ranibizumab (0.5 mg) as adjunctive therapy when added to laser photocoagulation and/or mono-therapy in patients with visual impairment due to diabetic macular edema.

CRFB002D2301E1: A 24 month open-label extension study for participants who completed the 12 month core study evaluated the long-term safety and efficacy of ranibizumab (0.5 mg) as symptomatic treatment for visual impairment due to diabetic macular edema.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: derived

ReferenceCitation: Bressler NM, Varma R, Mitchell P, Suñer IJ, Dolan C, Ward J, Ferreira A, Ehrlich JS, Turpcu A. Effect of Ranibizumab on the Decision to Drive and Vision Function Relevant to Driving in Patients With Diabetic Macular Edema: Report From RESTORE, RIDE, and RISE Trials. JAMA Ophthalmol. 2016 Feb;134(2):160-6. doi: 10.1001/jamaophthalmol.2015.4636.

name: Retina Division, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland2Editor, JAMA Ophthalmology

family: Bressler

full_name: Neil M. Bressler

given: Neil M.

sequence: first

name: University of Southern California Eye Institute, Keck School of Medicine, University of Southern California, Los Angeles

family: Varma

full_name: Rohit Varma

given: Rohit

sequence: additional

name: University of Sydney, Sydney, New South Wales, Australia

family: Mitchell

full_name: Paul Mitchell

given: Paul

family: Suñer

full_name: Ivan J. Suñer

given: Ivan J.

family: Dolan

full_name: Chantal Dolan

given: Chantal

name: Genentech Inc, South San Francisco, California

family: Ward

full_name: James Ward

given: James

family: Ferreira

full_name: Alberto Ferreira

given: Alberto

family: Ehrlich

full_name: Jason S. Ehrlich

given: Jason S.

family: Turpcu

full_name: Adam Turpcu

given: Adam

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: JAMA Ophthalmology

oa_host_type: publisher

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

publisher: American Medical Association (AMA)

publisher_dissemination: American Medical Association

publisher_group: American Medical Association

publisher_normalized: American Medical Association

ReferenceCitation: Mitchell P, Massin P, Bressler S, Coon CD, Petrillo J, Ferreira A, Bressler NM. Three-year patient-reported visual function outcomes in diabetic macular edema managed with ranibizumab: the RESTORE extension study. Curr Med Res Opin. 2015 Nov;31(11):1967-75. doi: 10.1185/03007995.2015.1081880. Epub 2015 Oct 6.

name: aDepartment of Ophthalmology and Westmead Millennium Institute, University of Sydney, Sydney, Australia

name: bService d’Ophtalmologie, Hôpital Lariboisière APHP, Université Paris Diderot, Paris, France

family: Massin

full_name: Pascale Massin

given: Pascale

name: cRetina Division, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA

full_name: Susan Bressler

given: Susan

family: Coon

full_name: Cheryl D. Coon

given: Cheryl D.

family: Petrillo

full_name: Jennifer Petrillo

given: Jennifer

journal_name: Current Medical Research and Opinion

oa_host_type: repository

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

host_type: repository

license: cc-by

license_normalized: cc-by

repository_institution: University of Illinois at Chicago - INDIGO

version: submittedVersion

publisher: Informa Healthcare

publisher_dissemination: Informa

publisher_group: Informa

ReferenceCitation: Bressler NM, Varma R, Suñer IJ, Dolan CM, Ward J, Ehrlich JS, Colman S, Turpcu A; RIDE and RISE Research Groups. Vision-related function after ranibizumab treatment for diabetic macular edema: results from RIDE and RISE. Ophthalmology. 2014 Dec;121(12):2461-72. doi: 10.1016/j.ophtha.2014.07.008. Epub 2014 Aug 20.

title: Vision-Related Function after Ranibizumab Treatment for Diabetic Macular Edema

ReferenceCitation: Mitchell P, Bressler N, Tolley K, Gallagher M, Petrillo J, Ferreira A, Wood R, Bandello F; RESTORE Study Group. Patient-reported visual function outcomes improve after ranibizumab treatment in patients with vision impairment due to diabetic macular edema: randomized clinical trial. JAMA Ophthalmol. 2013 Oct;131(10):1339-47. doi: 10.1001/jamaophthalmol.2013.4592.

title: Patient-Reported Visual Function Outcomes Improve After Ranibizumab Treatment in Patients With Vision Impairment Due to Diabetic Macular Edema

ReferenceCitation: Mitchell P, Bandello F, Schmidt-Erfurth U, Lang GE, Massin P, Schlingemann RO, Sutter F, Simader C, Burian G, Gerstner O, Weichselberger A; RESTORE study group. The RESTORE study: ranibizumab monotherapy or combined with laser versus laser monotherapy for diabetic macular edema. Ophthalmology. 2011 Apr;118(4):615-25. doi: 10.1016/j.ophtha.2011.01.031.

title: The RESTORE Study

ReferenceCitation: Schmidt-Erfurth U, Lang GE, Holz FG, Schlingemann RO, Lanzetta P, Massin P, Gerstner O, Bouazza AS, Shen H, Osborne A, Mitchell P; RESTORE Extension Study Group. Three-year outcomes of individualized ranibizumab treatment in patients with diabetic macular edema: the RESTORE extension study. Ophthalmology. 2014 May;121(5):1045-53. doi: 10.1016/j.ophtha.2013.11.041. Epub 2014 Feb 1.

repository_institution: University of Debrecen - University of Debrecen Electronic Archive

title: Three-Year Outcomes of Individualized Ranibizumab Treatment in Patients with Diabetic Macular Edema

location_country:
Italy
Switzerland
Netherlands
Germany
France
Greece
Hungary
Belgium
Australia
Spain
Turkey
Canada
United Kingdom

location_facility:
Novartis Investigative Site
Novartis Investigational Site


title: Comparison of the Safety and Efficacy of Eplivanserin and Lormetazepam in the Treatment of Insomnia Characterized by Sleep Maintenance Difficulties. A 4 Week, Randomized, Double-blind,Comparative, Parallel-group Study

acronym: DREAMS

summary:
The primary objective is to compare the potential for next-day residual effects of eplivanserin 5 mg/day and lormetazepam 1 mg/day by measuring the sleepiness in the morning using the patient's sleep questionnaire during 4 weeks of treatment in patients with chronic primary insomnia and sleep maintenance difficulties.

The secondary objectives are to compare the clinical safety of both products, including the potential for rebound insomnia and withdrawal symptoms after treatment discontinuation, to compare the efficacy of both products on subjective sleep parameters and to compare the effects of both products on patient's daytime functioning.

lead_sponsor: Sanofi

location_country: Chile Netherlands Spain Finland France Norway Portugal Estonia

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


type: Other Identifier

title: Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

acronym: RETRIEVE

summary:
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases.

In this study no therapy is being offered.

study_start_date_type: Actual

study_type: Observational

time_perspective: Other

ipd_sharing: No

lead_sponsor: Idorsia Pharmaceuticals Ltd.

location_country:
Italy
Brazil
Switzerland
Spain
Germany
France
Belgium
Portugal
United States
United Kingdom

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Lysosomal and Rare Disorders Research and Treatment Center

location_facility: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

location_facility: AP-HP - Hôpitaux Universitaires Est Parisien

location_facility: Great Ormond Street Hospital for Children NHS Found. Trust

location_facility: Hospital de Clínicas de Porto Alegre - HCPA

location_facility: Centro Hospitalar Universitario Lisboa Norte, EPE

location_facility: Centro Universitario Hospitalar de São João, EPE

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago


title: Long-term Safety and Tolerability Study of Linerixibat for the Treatment of Cholestatic Pruritus in Participants With Primary Biliary Cholangitis

acronym: LLSAT

summary: This is an open-label, non-comparator, global, multi-center, long-term safety study for evaluating safety and tolerability of linerixibat in participants with cholestatic pruritus in primary biliary cholangitis (PBC) who participated in a prior clinical trial with linerixibat (BAT117123 [NCT01899703], 201000 GLIMMER [NCT02966834] (group 1) or 212620 GLISTEN [NCT00210418]) (group 2). All participants will receive open-label linerixibat for the duration of the study. The duration of the study will be approximately four years until study end, however the total duration of study participation will vary by participant depending upon time of entry relative to study end in their respective country.

status: Recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ipd_sharing: Yes

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

lead_sponsor: GlaxoSmithKline

location_country:
Italy
United States
France
Japan
Poland
Spain
Germany
United Kingdom

status_simplified: Ongoing


type: EudraCT Number

title: A Postmarketing, Multicenter, Longitudinal, Prospective, Pharmacokinetic, Phase 1B Study in Pregnant Women With Chronic Inflammatory Diseases Treated With Cimzia (Certolizumab Pegol)

acronym: CHERISH

summary: The purpose of the study is to assess systemic certolizumab pegol (CZP) exposure, the formation of anti-CZP antibodies and safety of CZP across the course of pregnancy in study participants with chronic inflammatory diseases.

primary_purpose: Basic Science

ipd_sharing_description: Due to the small sample size in this trial, IPD cannot be adequately anonymized i.e., there is a reasonable likelihood that individual participants could be re-identified. For this reason, data from this trial cannot be shared.

lead_sponsor: UCB Biopharma S.P.R.L.

location_country:
United States
Switzerland
Netherlands
France
Spain
Germany
Canada


ReferenceCitation: van Bon AC, Bode BW, Sert-Langeron C, DeVries JH, Charpentier G. Insulin glulisine compared to insulin aspart and to insulin lispro administered by continuous subcutaneous insulin infusion in patients with type 1 diabetes: a randomized controlled trial. Diabetes Technol Ther. 2011 Jun;13(6):607-14. doi: 10.1089/dia.2010.0224. Epub 2011 Apr 2.

name: Department of Internal Medicine, Academic Medical Center, Amsterdam, The Netherlands.

family: van Bon

full_name: Arianne C. van Bon

given: Arianne C.

name: Atlanta Diabetes Associates, Emory University School of Medicine, Atlanta, Georgia.

family: Bode

full_name: Bruce W. Bode

given: Bruce W.

family: Sert-Langeron

full_name: Caroline Sert-Langeron

given: Caroline

family: DeVries

full_name: J. Hans DeVries

given: J. Hans

name: Department of Internal Medicine, Endocrinology and Diabetology, Centre Hospitalier Sud Francilien, Corbeil, France.

family: Charpentier

full_name: Guillaume Charpentier

given: Guillaume

journal_name: Diabetes Technology & Therapeutics

publisher: Mary Ann Liebert Inc

publisher_dissemination: Mary Ann Liebert Inc

publisher_group: Mary Ann Liebert Inc

publisher_normalized: Mary Ann Liebert Inc

title: Insulin Glulisine Compared to Insulin Aspart and to Insulin Lispro Administered by Continuous Subcutaneous Insulin Infusion in Patients with Type 1 Diabetes: A Randomized Controlled Trial

location_country:
Italy
Austria
Sweden
Netherlands
France
Israel
Hungary
Australia
Spain
United States
United Kingdom
Korea, Republic of

acronym: PUMP

source: euctr

title: Effect of Insulin Glulisine Compared to Insulin Aspart and Insulin Lispro When Administered by Continuous Subcutaneous Insulin Infusion (CSII) on Specific Pump Parameters in Patient With Type 1 Diabetes Mellitus

summary:
Primary objective: To demonstrate the superiority of insulin glulisine over insulin aspart and insulin lispro administered by external pump in term of unexplained hyperglycemia and/or infusion set occlusion.

Main Secondary objectives:

To compare insulin glulisine, insulin aspart and insulin lispro on:

Unexplained hyperglycemia
Infusion set occlusion
Hypoglycemic episodes,7-point blood glucose profiles
Episodes of significant ketosis and/or risk level for impending diabetic ketoacidosis
Time to change the infusion set
HbA1c (Glycosylated hemoglobin)
Overall safety: incidence of adverse events


title: A Prospective Controlled Study on the Effect on Bleeding Events and Joint Function in Young Adults With Severe Hemophilia A After a 6 Month Prophylaxis Treatment Compared to on Demand Treatment

summary:
The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design.

In addition, the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment and health-economic data will be assessed.

design_allocation: Non-Randomized

lead_sponsor: Bayer

location_country:
Italy
France
Spain
United States
United Kingdom


title: A Placebo Controlled Randomized, 12-week, Dose-ranging, Double-blind Study Versus Placebo Using Tolterodine as a Study Calibrator, to Evaluate Efficacy and Safety of SSR240600C in Women With Overactive Bladder Including Urge Urinary Incontinence

acronym: BILADY

summary: The primary objective of this study is to evaluate the efficacy of SSR240600C in women with overactive bladder compared to placebo using tolterodine as a study calibrator.

location_country:
United States
Netherlands
France
Portugal
Germany
Czech Republic
Canada

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


title: The RESPOND Outcomes Study - A Study in the RESPOND Consortium (RESPOND: International Cohort Consortium of Infectious Diseases)

summary: The RESPOND Outcomes study is a research study around use of antiretroviral and other relevant drugs and long-term clinical outcomes in patients living with HIV. Data collected in this study will be used to answer key unanswered questions regarding treatment of people living with HIV.

time_perspective: Prospective

ReferenceType: result

ReferenceCitation: Obel N, Omland LH, Kronborg G, Larsen CS, Pedersen C, Pedersen G, Sørensen HT, Gerstoft J. Impact of non-HIV and HIV risk factors on survival in HIV-infected patients on HAART: a population-based nationwide cohort study. PLoS One. 2011;6(7):e22698. doi: 10.1371/journal.pone.0022698. Epub 2011 Jul 25.

family: Obel

full_name: Niels Obel

given: Niels

family: Omland

full_name: Lars Haukali Omland

given: Lars Haukali

family: Kronborg

full_name: Gitte Kronborg

given: Gitte

family: Larsen

full_name: Carsten S. Larsen

given: Carsten S.

family: Pedersen

full_name: Court Pedersen

given: Court

full_name: Gitte Pedersen

family: Sørensen

full_name: Henrik Toft Sørensen

given: Henrik Toft

family: Gerstoft

full_name: Jan Gerstoft

given: Jan

bso_classification: Biology (fond.)

currency_apc_doaj: USD

journal_name: PLoS ONE

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

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

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

evidence: oa repository (semantic scholar lookup)

publisher: Public Library of Science (PLoS)

publisher_dissemination: Public Library of Science

publisher_group: Public Library of Science

publisher_normalized: Public Library of Science

title: Impact of Non-HIV and HIV Risk Factors on Survival in HIV-Infected Patients on HAART: A Population-Based Nationwide Cohort Study

ReferenceCitation: Smith CJ, Ryom L, Weber R, Morlat P, Pradier C, Reiss P, Kowalska JD, de Wit S, Law M, el Sadr W, Kirk O, Friis-Moller N, Monforte Ad, Phillips AN, Sabin CA, Lundgren JD; D:A:D Study Group. Trends in underlying causes of death in people with HIV from 1999 to 2011 (D:A:D): a multicohort collaboration. Lancet. 2014 Jul 19;384(9939):241-8. doi: 10.1016/S0140-6736(14)60604-8.

family: Smith

full_name: Colette J Smith

given: Colette J

family: Ryom

full_name: Lene Ryom

given: Lene

family: Weber

full_name: Rainer Weber

given: Rainer

family: Morlat

full_name: Philippe Morlat

given: Philippe

family: Pradier

full_name: Christian Pradier

given: Christian

family: Reiss

full_name: Peter Reiss

given: Peter

family: Kowalska

full_name: Justyna D Kowalska

given: Justyna D

repository_institution: University College London - UCL Discovery

ReferenceCitation: Schouten J, Wit FW, Stolte IG, Kootstra NA, van der Valk M, Geerlings SE, Prins M, Reiss P; AGEhIV Cohort Study Group. Cross-sectional comparison of the prevalence of age-associated comorbidities and their risk factors between HIV-infected and uninfected individuals: the AGEhIV cohort study. Clin Infect Dis. 2014 Dec 15;59(12):1787-97. doi: 10.1093/cid/ciu701. Epub 2014 Sep 2.

title: Cross-sectional Comparison of the Prevalence of Age-Associated Comorbidities and Their Risk Factors Between HIV-Infected and Uninfected Individuals: The AGEhIV Cohort Study

ReferenceCitation: Chary A, Nguyen NN, Maiton K, Holodniy M. A review of drug-drug interactions in older HIV-infected patients. Expert Rev Clin Pharmacol. 2017 Dec;10(12):1329-1352. doi: 10.1080/17512433.2017.1377610. Epub 2017 Sep 19. Review.

name: Department of Medicine, Veterans Affairs Palo Alto Health Care System, Palo Alto, CA, USA

name: Division of Infectious Diseases and Geographic Medicine, Stanford University School of Medicine, Stanford, CA, USA

name: Department of Pharmacy, Veterans Affairs Palo Alto Health Care System, Palo Alto, CA, USA

name: Department of Pharmacy Practice, Thomas J. Long School of Pharmacy & Health Sciences, University of the Pacific, Stockton, CA, USA

name: Office of Public Health Surveillance & Research, Veterans Affairs Palo Alto Health Care System, Palo Alto, CA, USA

ReferenceCitation: Weber R, Sabin CA, Friis-Møller N, Reiss P, El-Sadr WM, Kirk O, Dabis F, Law MG, Pradier C, De Wit S, Akerlund B, Calvo G, Monforte Ad, Rickenbach M, Ledergerber B, Phillips AN, Lundgren JD. Liver-related deaths in persons infected with the human immunodeficiency virus: the D:A:D study. Arch Intern Med. 2006 Aug 14-28;166(15):1632-41.

ReferenceCitation: Kattakuzhy S, Gross C, Emmanuel B, Teferi G, Jenkins V, Silk R, Akoth E, Thomas A, Ahmed C, Espinosa M, Price A, Rosenthal E, Tang L, Wilson E, Bentzen S, Masur H, Kottilil S; ASCEND Providers. Expansion of Treatment for Hepatitis C Virus Infection by Task Shifting to Community-Based Nonspecialist Providers: A Nonrandomized Clinical Trial. Ann Intern Med. 2017 Sep 5;167(5):311-318. doi: 10.7326/M17-0118. Epub 2017 Aug 8.

name: From University of Maryland School of Medicine, Baltimore, Maryland; Unity Health Care and Family and Medical Counseling Services, Washington, DC; and National Institutes of Health, Bethesda, Maryland.

ReferenceCitation: Falade-Nwulia O, Suarez-Cuervo C, Nelson DR, Fried MW, Segal JB, Sulkowski MS. Oral Direct-Acting Agent Therapy for Hepatitis C Virus Infection: A Systematic Review. Ann Intern Med. 2017 May 2;166(9):637-648. doi: 10.7326/M16-2575. Epub 2017 Mar 21. Review.

name: From Johns Hopkins University School of Medicine, Baltimore, Maryland; University of Florida, Gainesville, Florida; and University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.

ReferenceCitation: van der Meer AJ, Veldt BJ, Feld JJ, Wedemeyer H, Dufour JF, Lammert F, Duarte-Rojo A, Heathcote EJ, Manns MP, Kuske L, Zeuzem S, Hofmann WP, de Knegt RJ, Hansen BE, Janssen HL. Association between sustained virological response and all-cause mortality among patients with chronic hepatitis C and advanced hepatic fibrosis. JAMA. 2012 Dec 26;308(24):2584-93. doi: 10.1001/jama.2012.144878.

title: Association Between Sustained Virological Response and All-Cause Mortality Among Patients With Chronic Hepatitis C and Advanced Hepatic Fibrosis

ReferenceCitation: Friis-Møller N, Ryom L, Smith C, Weber R, Reiss P, Dabis F, De Wit S, Monforte AD, Kirk O, Fontas E, Sabin C, Phillips A, Lundgren J, Law M; D:A:D study group. An updated prediction model of the global risk of cardiovascular disease in HIV-positive persons: The Data-collection on Adverse Effects of Anti-HIV Drugs (D:A:D) study. Eur J Prev Cardiol. 2016 Jan;23(2):214-23. doi: 10.1177/2047487315579291. Epub 2015 Apr 16.

name: Odense University Hospital, Department of Infectious Diseases, Denmark

name: Copenhagen HIV Programme, Department of Infectious Diseases and Rheumatology, University of Copenhagen, Denmark

name: Research Department of Infection and Population Health, UCL, London, UK

name: Division of Infectious Diseases, University Hospital Zurich, University of Zurich, Switzerland

name: Academic Medical Centre, University of Amsterdam, and Stichting HIV Monitoring, The Netherlands

name: Department of Infectious Diseases, Centre Hospitalier Universitaire St Pierre Hospital, Brussels, Belgium

name: Department of Infectious Diseases, Hospital San Paolo, University of Milan, Italy

name: Department de Santé Publique, Centre Hospitalier Universitaire de Nice, France

name: 0The Kirby Institute, University of New South Wales, Sydney, Australia

journal_name: European Journal of Preventive Cardiology

ReferenceCitation: Mocroft A, Lundgren JD, Ross M, Fux CA, Reiss P, Moranne O, Morlat P, Monforte Ad, Kirk O, Ryom L; Data Collection on Adverse events of Anti-HIV Drugs (D:A:D) Study. Cumulative and current exposure to potentially nephrotoxic antiretrovirals and development of chronic kidney disease in HIV-positive individuals with a normal baseline estimated glomerular filtration rate: a prospective international cohort study. Lancet HIV. 2016 Jan;3(1):e23-32. doi: 10.1016/S2352-3018(15)00211-8. Epub 2015 Nov 17.

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: University of Ferrara - Institutional Research Information System University of Ferrara

ReferenceCitation: Bruyand M, Ryom L, Shepherd L, Fatkenheuer G, Grulich A, Reiss P, de Wit S, D Arminio Monforte A, Furrer H, Pradier C, Lundgren J, Sabin C; D:A:D study group. Cancer risk and use of protease inhibitor or nonnucleoside reverse transcriptase inhibitor-based combination antiretroviral therapy: the D: A: D study. J Acquir Immune Defic Syndr. 2015 Apr 15;68(5):568-77. doi: 10.1097/QAI.0000000000000523.

journal_name: JAIDS Journal of Acquired Immune Deficiency Syndromes

publisher: Ovid Technologies (Wolters Kluwer Health)

title: Cancer Risk and Use of Protease Inhibitor or Nonnucleoside Reverse Transcriptase Inhibitor–Based Combination Antiretroviral Therapy

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
France
Georgia
Australia
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility: Austrian HIV Cohort Study (AHIVCOS), Medizinische Universität Innsbruch

location_facility: The ATHENA (AIDS Therapy Evaluation in the Netherlands) national observational HIV cohort, Stichting HIV Monitorin, AMC, University of Amsterdam

location_facility: Italian Cohort Naive Antiretrovirals (ICONA)

location_facility: Swedish InfCare HIV Cohort, Karolinska University Hospital

location_facility: Swiss HIV Cohort Study (SHCS), University Hospital Zurich

location_facility: The Australian HIV Observational Database (AHOD)

location_facility: San Raffaele Scientific Institute, Ospedale San Raffaele

location_facility: PISCIS Cohort Study, Germans Trias i Pujol University Hospital

location_facility: Royal Free HIV Cohort Study, Royal Free Hospital

location_facility: Modena HIV Cohort, Università degli Studi di Modena

location_facility: The EuroSIDA Study, CHIP, Rigshospitalet

location_facility: Nice HIV Cohort, Centre Hospitalier Universitaire de Nice

location_facility: Frankfurt HIV Cohort Study, Goethe-University Frankfurt

location_facility: Georgian National AIDS Health Information System (AIDS HIS), IDACIRC


title: Vascular Access Outcome Measure for Function: a vaLidation Study In haemoDialysis (VALID)

acronym: VALID

summary: A functioning vascular access provides a lifeline for patients requiring haemodialysis but vascular access dysfunction remains one of the leading causes of excessive morbidity, mortality and healthcare costs in this group. Despite increasing numbers of vascular access trials, successful interventions to improve vascular access function have been sparse and compromised by highly variable, often selectively reported outcome measures of limited relevance to patients and health professionals. Through engagement of all relevant stakeholders including patients and caregivers, vascular access function, defined by the need for interventions to enable and maintain the use of a vascular access for haemodialysis, has been identified as one of the most critically important outcome measures for trials in haemodialysis. This prospective, multi-centre, multinational validation study aims to assess the accuracy and feasibility of measuring vascular access function part of routine clinical practice and across different clinical settings to ensure successful global implementation of this core outcome measure in future trials in haemodialysis.

status: Enrolling by invitation

ipd_sharing_description: Individual participant data that underlie the results reported in the primary publication, after deidentification (text, tables, figures and appendices) will be available for individual participant data meta-analysis.

lead_sponsor: The University of Queensland

location_country:
Switzerland
Netherlands
France
Malaysia
Australia
Canada
United Kingdom

location_facility: Centre Hospitalier Régional Universitaire de Tours

lead_sponsor_type: academique

status_simplified: Unknown


title: A Real World Evidence Prospective Cohort Study in the Management of Metastatic Colorectal Cancer: A Clinical and Patient Perspective

acronym: PROMETCO

lead_sponsor: Servier Affaires Médicales

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
Spain
United Kingdom
Croatia
Czechia
France
Greece
Hungary
Argentina
Belgium
Ireland
Portugal
Germany
Slovenia

location_facility: Centro Des Estudos Egas Moniz - Hospital Santa Maria

location_facility: UniversitaetsSpital Zuerich (University Hospital Zurich)

location_facility: Cancer Research UK, Department of Medical Oncology-The Christie NHS Foundation Trust

location_facility: Del-Pesti Centrum Korhaz OHII, Szent Laszlo Korhaz Telephely

location_facility: University Hospital in Hradec Kralove Clinic of Oncology and Radiotherapy

location_facility: The Adelaide & Meath Hospital-Dublin Incorporating The National Children's Hospital

intervention_type: Other


title: E²-RADIatE: EORTC-ESTRO RADiotherapy InfrAstrucTure for Europe

summary: The primary objective is the collection of real-world data of cancer patients treated with radiotherapy, to support radiotherapy research and to provide evidence of the role of radiation oncology in a multidisciplinary approach. This is an open ended prospective non-interventional non-therapeutic multi-cohort study.

ipd_sharing: Undecided

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

location_country: Italy Switzerland Netherlands France Belgium

location_facility: Hôpitaux universitaires de Genève - HUG - site de Cluse-Roseraie

location_facility: Radboudumc - Radboud University Medical Center Nijmegen

location_facility: AZ Groeninge Kortrijk - Campus Kennedylaan


type: Registry Identifier

title: Localized Non-Rhabdomyosarcoma Soft Tissue Sarcomas

summary:
RATIONALE: Drugs used in chemotherapy, such as ifosfamide and doxorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving combination chemotherapy with or without radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving radiation therapy after surgery may kill any tumor cells that remain after surgery.

PURPOSE: This phase III trial is studying how well giving ifosfamide and doxorubicin, radiation therapy, and/or surgery works in treating young patients with localized soft tissue sarcoma.

status: Unknown status

lead_sponsor: European Paediatric Soft Tissue Sarcoma Study Group

location_country:
Austria
Sweden
Switzerland
France
Ireland
Belgium
Spain
Denmark
United Kingdom

location_facility: Rigshospitalet - Copenhagen University Hospital

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Great Ormond Street Hospital for Children

location_facility: Our Lady's Hospital for Sick Children Crumlin

intervention_type: Radiation


title: A Multicenter, Randomized, Double-blind, Parallel-arm, Two-stage Study of the Efficacy and Safety of AVE0005 (VEGF Trap) Administered Intravenously Every 2 Weeks in Patients With Platinum-resistant and topotecan-and/or Liposomal Doxorubicin-resistant Advanced Ovarian Cancer

summary:
This study evaluated outcomes in participants with advanced ovarian epithelial adenocarcinoma receiving aflibercept.

The primary objective was to compare the objective response rate of Aflibercept (ziv-aflibercept, AVE0005, VEGF trap, ZALTRAP®) 4.0 mg/kg and 2.0 mg/kg, administered intravenously (IV) every 2 weeks with historical control in participants with advanced ovarian epithelial (including fallopian tube and primary peritoneal) adenocarcinoma resistant to platinum and topotecan and/or liposomal doxorubicin.

The secondary objectives was to further assess efficacy, safety, pharmacokinetics, potential biological and pharmacogenomic markers of study drug activity, and health-related quality of life.

This study employed an Independent Review Committee (IRC) for radiological tumor assessments. For all tumor assessment-related efficacy variables, two analyses were performed: the primary analysis was based on Independent Review Committee (IRC) reviewed data and the secondary analysis was based on Investigator evaluation. If an endpoint was evaluated by the IRC, the IRC reviewed data is reported for this study.

ReferenceCitation: Tew WP, Colombo N, Ray-Coquard I, Del Campo JM, Oza A, Pereira D, Mammoliti S, Matei D, Scambia G, Tonkin K, Shun Z, Sternas L, Spriggs DR. Intravenous aflibercept in patients with platinum-resistant, advanced ovarian cancer: results of a randomized, double-blind, phase 2, parallel-arm study. Cancer. 2014 Feb 1;120(3):335-43. doi: 10.1002/cncr.28406. Epub 2013 Oct 11.

name: Department of Medicine; Memorial Sloan-Kettering Cancer Center; New York New York

family: Tew

full_name: William P. Tew

given: William P.

name: Gynecologic Oncology, European Institute of Oncology; University of Milan-Bicocca; Milan Italy

family: Colombo

full_name: Nicoletta Colombo

given: Nicoletta

name: Department of Medicine; Leon Berard Center; Lyon France

family: Ray-Coquard

full_name: Isabelle Ray-Coquard

given: Isabelle

name: Department of Oncology, Vall d'Hebron Hospital; Passeig Vall d'Hebron; Barcelona Spain

family: del Campo

full_name: Josep M. del Campo

given: Josep M.

name: Department of Medical Oncology; Princess Margaret Hospital; Toronto Ontario Canada

family: Oza

full_name: Amit Oza

given: Amit

name: Medical Oncology Department; Portuguese Institute of Oncology; Porto Portugal

family: Pereira

full_name: Deolinda Pereira

given: Deolinda

name: Medical Oncology; San Martino Hospital; Genova Italy

family: Mammoliti

full_name: Serafina Mammoliti

given: Serafina

name: Department of Medicine; Indiana University Cancer Center; Indianapolis Indiana

family: Matei

full_name: Daniela Matei

given: Daniela

name: Department of Obstetrics and Gynecology, Gemelli Hospital; Sacro Cuore University; Campobasso Italy

family: Scambia

full_name: Giovanni Scambia

given: Giovanni

name: Division of Medical Oncology; Cross Cancer Institute; Edmonton Alberta Canada

family: Tonkin

full_name: Katia Tonkin

given: Katia

name: Sanofi-Aventis Oncology; Bridgewater New Jersey

family: Shun

full_name: Zhenming Shun

given: Zhenming

family: Sternas

full_name: Lars Sternas

given: Lars

family: Spriggs

full_name: David R. Spriggs

given: David R.

journal_name: Cancer

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Intravenous aflibercept in patients with platinum-resistant, advanced ovarian cancer: Results of a randomized, double-blind, phase 2, parallel-arm study

location_country:
Italy
United States
Sweden
Switzerland
Netherlands
Spain
Germany
France
Australia
Portugal
Canada


title: A Double Blind, Single Dose Study To Explore The Safety, Pharmacokinetics And Pharmacodynamics Of PHA-794428 In Pediatric Patients With Growth Hormone Deficiency

summary: This will be the first clinical study of the development of PHA-794428 in a pediatric population. Since differences in PK and/or PD response may occur between adult and pediatric subjects, it is deemed appropriate to first conduct an exploratory single dose study in pediatric patients to assess safety and tolerability in this patient population. In addition this will add pediatric data to facilitate the prediction of the optimal therapeutic dose to be tested in repeated dose phase 2b trials in children, using PK/PD modeling

status: Terminated

lead_sponsor: Pfizer

location_country:
France
Israel
Belgium
Germany
United Kingdom


title: Randomized Comparison of a Two-month Regimen of Irbesartan Versus Enalapril on Cardiovascular Markers in Patients With Acute Coronary Syndrome Without ST Segment Elevation.

summary:
Primary Objective

The main objective of this study is to assess if a two-month regimen of irbesartan in patients hospitalized for acute coronary syndrome without ST segment elevation can reduce inflammation markers (ie hsCRP), in comparison to a similar regimen of enalapril.

Secondary Objectives

To compare both regimens on several other biological parameters which have demonstrated their relevance and their predictive clinical value (ie BNP, microalbuminuria, troponin I …) in this patient population.
To compare on the above parameters the early initiation of treatment versus the initiation of treatment at hospital discharge.

ReferenceCitation: Montalescot G, Drexler H, Gallo R, Pearson T, Thoenes M, Bhatt DL. Effect of irbesartan and enalapril in non-ST elevation acute coronary syndrome: results of the randomized, double-blind ARCHIPELAGO study. Eur Heart J. 2009 Nov;30(22):2733-41. doi: 10.1093/eurheartj/ehp301. Epub 2009 Aug 21.

family: Montalescot

full_name: G. Montalescot

given: G.

family: Drexler

full_name: H. Drexler

given: H.

family: Gallo

full_name: R. Gallo

given: R.

family: Pearson

full_name: T. Pearson

given: T.

family: Thoenes

full_name: M. Thoenes

given: M.

family: Bhatt

full_name: D. L. Bhatt

given: D. L.

journal_name: European Heart Journal

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

location_country:
Italy
United States
Switzerland
Netherlands
Germany
France
Hungary
Belgium
Spain
Canada
United Kingdom


title: Long-Term Safety Extension of Phase II Study EFC5286 of SR57667B in Patients With Mild-to-Moderate Alzheimer's Disease

summary:
The primary objective is to assess the long term safety/tolerability of 4 mg/day of SR57667B in comparison to placebo in patients with mild-to-moderate Alzheimer's Disease (AD).

A secondary objective is to describe the long term progression of Alzheimer's symptoms in patients treated by 4 mg/day of SR57667B.

location_country:
South Africa
Sweden
Germany
Finland
France
Denmark
Canada


title: Safety Profile of Innohep Versus Subcutaneous Unfractionated Heparin in Elderly Patients With Impaired Renal Function Treated for Acute Deep Vein Thrombosis

summary:
The objective of the study is to compare the safety of innohep® and Unfractionated Heparin (UFH) in terms of clinically relevant bleedings in elderly patients with impaired renal function for initial treatment of acute Deep Venous Thrombosis (DVT).

The primary response criterion is the percentage of patients with clinically relevant bleeding events prior to day 90 +/- 5.

ReferenceCitation: Leizorovicz A, Siguret V, Mottier D; Innohep® in Renal Insufficiency Study Steering Committee, Leizorovicz A, Siguret V, Mottier D, Clonier F, Janas M, Stinson J, Townshend G, Maddalena M. Safety profile of tinzaparin versus subcutaneous unfractionated heparin in elderly patients with impaired renal function treated for acute deep vein thrombosis: the Innohep® in Renal Insufficiency Study (IRIS). Thromb Res. 2011 Jul;128(1):27-34. doi: 10.1016/j.thromres.2011.03.002. Epub 2011 Apr 7.

family: Leizorovicz

full_name: Alain Leizorovicz

given: Alain

family: Siguret

full_name: Virginie Siguret

given: Virginie

family: Mottier

full_name: Dominique Mottier

given: Dominique

journal_name: Thrombosis Research

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Safety profile of tinzaparin versus subcutaneous unfractionated heparin in elderly patients with impaired renal function treated for acute deep vein thrombosis: The Innohep® in Renal Insufficiency Study (IRIS)

lead_sponsor: LEO Pharma

location_country: Serbia Croatia Czechia Bulgaria France Poland Romania Spain Germany

location_facility: Clinical Center Serbia, Institute of Cardiovascular diseases & Centre for Vascular Surgery-Belgrade

location_facility: Service of Geriatry, Hospital Universitario Clínico San Carlos


title: Phase 1b, Multi-arm, Open-label Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndrome

summary: To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients, and to identify recommended doses for future studies.

status: Active, not recruiting

lead_sponsor: Novartis Pharmaceuticals

location_country:
United States
Netherlands
Finland
France
Australia
Spain
Germany
United Kingdom

location_facility:
Oregon Health and Science University
Massachusetts General Hospital
Novartis Investigative Site
MD Anderson Cancer Center


title: An Open-Label, Multicenter, Multinational, Centrally Randomized, Two-Arm Parallel-Group Study to Demonstrate the Non-Inferiority in Clinical Efficacy of Levofloxacin 750mg od in Comparison With Piperacillin/Tazobactam 4g/500mg Every 8 Hours in the Treatment of Mild to Moderate Hospital-Acquired Pneumonia Adult Patients in Both General Wards and ICU (Intensive Care Unit)

summary:
Primary Objective:

The primary objective of the study is to demonstrate the non-inferiority in clinical efficacy at the test of cure (TOC) visit planned 5-7 days after treatment completion of levofloxacin 750 mg once daily (od) in comparison with piperacillin/tazobactam 4 g/500 mg every 8 hours in treating adult patients suffering from mild to moderate hospital-acquired pneumonia.

Secondary Objectives:

The secondary objectives of the study are:

To assess the bacteriological efficacy at the test of cure (TOC) visit
To assess the clinical and bacteriological efficacy at the end of study (EOS) visit, 28 to 32 days after treatment ends
To assess the tolerability of both drugs

location_country:
Italy
Austria
South Africa
Guatemala
Lebanon
Mexico
Netherlands
France
Greece
Venezuela
Romania
Belgium
Russian Federation
Spain
Turkey
Germany
Czech Republic


title: A Disease Registry for Patients With Tenosynovial Giant Cell Tumors (TGCT), Also Known as Pigmented Villonodular Synovitis (PVNS) and Giant Cell Tumor of the Tendon Sheath (GCT-TS)

acronym: TOP

summary:
TGCT is a rare disease that is difficult to manage, surgical resection is the primary treatment currently available. To date no disease registry exists and there is little data available detailing the management of patients with diffuse TGCT, the burden of diffuse TGCT for patients (including pain, joint stiffness, swelling, reduced mobility and quality of life) or the economic impact of diffuse TGCT.

This study aims to collect data by an observational disease registry involving no intervention to the patient or changes to investigators treatment decisions.

ReferenceCitation: Bernthal NM, Spierenburg G, Healey JH, Palmerini E, Bauer S; TOPP Study Group, Gelderblom H, Staals EL, Lopez-Bastida J, Fronk EM, Ye X, Laeis P, van de Sande MAJ. The diffuse-type tenosynovial giant cell tumor (dt-TGCT) patient journey: a prospective multicenter study. Orphanet J Rare Dis. 2021 Apr 29;16(1):191. doi: 10.1186/s13023-021-01820-6.

family: Bernthal

full_name: Nicholas M. Bernthal

given: Nicholas M.

family: Spierenburg

full_name: Geert Spierenburg

given: Geert

family: Healey

full_name: John H. Healey

given: John H.

family: Palmerini

full_name: Emanuela Palmerini

given: Emanuela

family: Bauer

full_name: Sebastian Bauer

given: Sebastian

family: Gelderblom

full_name: Hans Gelderblom

given: Hans

family: Staals

full_name: Eric L. Staals

given: Eric L.

family: Lopez-Bastida

full_name: Julio Lopez-Bastida

given: Julio

family: Fronk

full_name: Eva-Maria Fronk

given: Eva-Maria

family: Ye

full_name: Xin Ye

given: Xin

family: Laeis

full_name: Petra Laeis

given: Petra

family: van de Sande

full_name: Michiel A. J. van de Sande

given: Michiel A. J.

currency_apc_doaj: GBP

journal_name: Orphanet Journal of Rare Diseases

repository_institution: California Digital Library - eScholarship

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ipd_sharing_description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
Italy
Austria
Netherlands
Germany
France
Spain
United States
United Kingdom

location_facility: Istituto Nazionale Tumori-Foundazione IRCCS

location_facility: University of Texas MD Anderson Cancer Center


title: Calcipotriol Plus Betamethasone Dipropionate Gel Compared to Betamethasone Dipropionate in the Gel Vehicle and Calcipotriol in the Gel Vehicle in Scalp Psoriasis

summary:
The purpose of the study is to evaluate whether once daily topical treatment for up to 8 weeks of calcipotriol 50 mcg/g plus betamethasone 0.5 mg/g (as dipropionate) gel is safe and more effective than betamethasone 0.5 mg/g (as dipropionate) in the gel vehicle and calcipotriol 50 mcg/g in the gel vehicle in patients with scalp psoriasis.

The primary response criterion is the number of patients with absence of disease and very mild disease after 8 weeks of treatment.

ReferenceCitation: van de Kerkhof PC, Hoffmann V, Anstey A, Barnes L, Bolduc C, Reich K, Saari S, Segaert S, Vaillant L. A new scalp formulation of calcipotriol plus betamethasone dipropionate compared with each of its active ingredients in the same vehicle for the treatment of scalp psoriasis: a randomized, double-blind, controlled trial. Br J Dermatol. 2009 Jan;160(1):170-6. doi: 10.1111/j.1365-2133.2008.08927.x. Epub 2008 Nov 25.

family: van de Kerkhof

full_name: P.C.M. van de Kerkhof

family: Hoffmann

full_name: V. Hoffmann

given: V.

family: Anstey

full_name: A. Anstey

given: A.

family: Barnes

full_name: L. Barnes

given: L.

family: Bolduc

full_name: C. Bolduc

given: C.

family: Reich

full_name: K. Reich

given: K.

family: Saari

full_name: S. Saari

given: S.

family: Segaert

full_name: S. Segaert

family: Vaillant

full_name: L. Vaillant

journal_name: British Journal of Dermatology

location_country:
Netherlands
Finland
France
Belgium
Germany
United Kingdom
Canada

location_facility: Universitair Ziekenhuis Sint Rafaël, Dienst Dermatologie

location_facility: Policlinic of Dermatology, Medical Reception Centre

location_facility: Georg-August-Universität Göttingen, Abteilung Dermatologie und Venerologie

location_facility: Hôpital Trousseau, Service de Dermatologie CHU Tours

location_facility: Royal Gwent Hospital, Department of Dermatology

location_facility: Universitair Medisch, Centrum St. Radboud, Afdeling Dermatologie


title: A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome

summary: This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric and young adult subjects with Dravet syndrome. Subjects who qualify for the study will be randomized (1:1:1) in a double-blind manner to receive 1 of 2 doses of ZX008 or placebo. All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Total treatment time from the beginning of the Titration Period through the end of the Maintenance Period is 14 weeks.

ReferenceCitation: Cross JH, Galer BS, Gil-Nagel A, Devinsky O, Ceulemans B, Lagae L, Schoonjans AS, Donner E, Wirrell E, Kothare S, Agarwal A, Lock M, Gammaitoni AR. Impact of fenfluramine on the expected SUDEP mortality rates in patients with Dravet syndrome. Seizure. 2021 Nov 2;93:154-159. doi: 10.1016/j.seizure.2021.10.024. [Epub ahead of print]

family: Cross

full_name: J. Helen Cross

given: J. Helen

family: Galer

full_name: Bradley S. Galer

given: Bradley S.

family: Gil-Nagel

full_name: Antonio Gil-Nagel

given: Antonio

family: Devinsky

full_name: Orrin Devinsky

given: Orrin

family: Ceulemans

full_name: Berten Ceulemans

given: Berten

family: Lagae

full_name: Lieven Lagae

given: Lieven

family: Schoonjans

full_name: An-Sofie Schoonjans

given: An-Sofie

family: Donner

full_name: Elizabeth Donner

given: Elizabeth

family: Wirrell

full_name: Elaine Wirrell

given: Elaine

family: Kothare

full_name: Sanjeev Kothare

given: Sanjeev

family: Agarwal

full_name: Anupam Agarwal

given: Anupam

family: Lock

full_name: Michael Lock

given: Michael

family: Gammaitoni

full_name: Arnold R. Gammaitoni

given: Arnold R.

journal_name: Seizure

evidence: open (via page says license)

ReferenceCitation: Sullivan J, Perry MS, Wheless JW, Galer B, Gammaitoni A. Fenfluramine responder analyses and numbers needed to treat: Translating epilepsy trial data into clinical practice. Eur J Paediatr Neurol. 2021 Mar;31:10-14. doi: 10.1016/j.ejpn.2021.01.005. Epub 2021 Jan 22.

family: Sullivan

full_name: Joseph Sullivan

given: Joseph

family: Perry

full_name: M. Scott Perry

given: M. Scott

family: Wheless

full_name: James W. Wheless

given: James W.

full_name: Bradley Galer

given: Bradley

full_name: Arnold Gammaitoni

given: Arnold

journal_name: European Journal of Paediatric Neurology

ReferenceCitation: Lagae L, Sullivan J, Knupp K, Laux L, Polster T, Nikanorova M, Devinsky O, Cross JH, Guerrini R, Talwar D, Miller I, Farfel G, Galer BS, Gammaitoni A, Mistry A, Morrison G, Lock M, Agarwal A, Lai WW, Ceulemans B; FAiRE DS Study Group. Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. Lancet. 2019 Dec 21;394(10216):2243-2254. doi: 10.1016/S0140-6736(19)32500-0. Epub 2019 Dec 17.

repositories_institution: University College London - UCL Discovery

lead_sponsor: Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

location_country:
Italy
France
Japan
Belgium
Australia
Spain
Denmark
Germany
United Kingdom

location_facility: U.O. Neurologia Dipartimento di Neuroscienze Ospedale Pediatrico Bambino Gesù, IRCS

location_facility: Universitaetsklinikum Jena Klinik fuer Kinder- und Jugendmedizin Neuropaediatrie

location_facility: Institute of Neurosciences Queens Elizabeth University Hospital

location_facility: Great Ormonnd Street Hospital for Children NHS Foundation Trust

location_facility: Epilepsiezentrum / Neuropädiatrie Hedwig-von-Rittberg-Zentrum Für Kinder und Jugendliche

location_facility: Kleinwachau Saechsisches Epilepsiezentrum Radeberg gemeinnuetzige GmbH

location_facility: Ospedal Policlinico Giambattista Rossi diBorga Roma

location_facility: National Epilepsy Center Shizuoka Institute

location_facility: Hospital Ruber Internacional Primera Planta Servicio de Neurologia

location_facility: Universitaetsklinik fuer Kinder- und Jugendmedizin Abteilung III

location_facility: The Children's Hospital Westmead Dept. of Neurology and Neurosurgery

location_facility: Istituto Pediatrico Giannina Gaslini Dipartimento di Neurologia

location_facility: Klinik für Neuropädiatrie Universitätsklinikum Schleswig Holstein Campus Kiel

location_facility: Children's Health Queensland Hospital and Health Service at Lady Cilento Children's Hospital

location_facility: Clinica Universitaria de Navarra Fase 4. Segunda planta, Consulta de Pediatria

location_facility: Tokyo Women's Medical University Hospital

location_facility: Krankenhaus Mara Epilepsie-Zentrum Bethel

location_facility: Schoen Klinik Vogtareuth Neuropaediatrie und Neurologische Rehabilitation, Epilepsiezentrum fuer Kinder und Jugendlische, Tagesklinik fuer Neuropaediatrie


title: The RELIEF Trial: REduction or eLimination of mItral rEgurgitation in Degenerative or Functional Mitral Regurgitation With the CardiAQ-Edwards™ Transcatheter Mitral Valve

acronym: RELIEF

summary: Clinical study to evaluate the safety and performance of the CardiAQ-Edwards™ Transcatheter Mitral Valve

status: Withdrawn

lead_sponsor: Edwards Lifesciences

location_country: Italy Switzerland Netherlands Germany France Denmark Canada

location_facility: Centre de recherche Institut Universitaire de Cardiologie et de Pneumologie de Québec (CRIUCPQ)

location_facility: St. Paul's Hospital, University of British Columbia

location_facility: Centre Hospitalier Régional Universitaire de Lille

intervention_type: Device


title: INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger

acronym: INCEPTUS

summary: This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.

lead_sponsor: Audentes Therapeutics

location_country:
United States
France
Germany
United Kingdom
Canada

location_facility: National Institute of Neurological Disorders and Stroke/NIH Porter

location_facility: Ann & Robert H. Lurie Children's Hospital of Chicago


title: An Extension to a Phase II Open-label Study to Determine the Safety and Anti-leukemic Effects of STI571 in Patients With Philadelphia Chromosome-positive Chronic Myeloid Leukemia in Myeloid Blast Crisis

summary: This extension II study allowed for further follow-up of the disease under treatment with imatinib mesylate and allow the participants to continue to receive imatinib mesylate.

location_country:
Italy
Germany
United States
France

location_facility:
University of Chicago
Novartis Investigative Site
MD Anderson Cancer Center
Dana Faber Institute


title: A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients "IMPALA"

acronym: IMPALA

summary: This study evaluates inhaled molgramostim (recombinant human (rh) Granulocyte Macrophage-Colony Stimulating Factor (GM-CSF)) in the treatment of autoimmune pulmonary alveolar proteinosis patients. A third of the patients will receive inhaled molgramostim daily for 24 weeks, a third will receive inhaled molgramostim intermittently (seven days on, seven days off) for 24 weeks and a third will receive inhaled matching placebo for 24 weeks.

ReferenceCitation: Trapnell BC, Inoue Y, Bonella F, Morgan C, Jouneau S, Bendstrup E, Campo I, Papiris SA, Yamaguchi E, Cetinkaya E, Ilkovich MM, Kramer MR, Veltkamp M, Kreuter M, Baba T, Ganslandt C, Tarnow I, Waterer G, Jouhikainen T; IMPALA Trial Investigators. Inhaled Molgramostim Therapy in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2020 Oct 22;383(17):1635-1644. doi: 10.1056/NEJMoa1913590. Epub 2020 Sep 7.

name: From the Translational Pulmonary Science Center, Cincinnati Children’s Hospital Medical Center, Cincinnati (B.C.T.); National Hospital Organization Kinki-Chuo Chest Medical Center, Osaka (Y.I.), Aichi Medical University Hospital, Nagakute, Aichi (E.Y.), and Kanagawa Cardiovascular and Respiratory Center, Yokohama (T.B.) — all in Japan; Outpatients Clinic for Interstitial and Rare Lung Disease, Ruhrlandklinik University Hospital, Essen (F.B.), and Center for Interstitial and Rare Lung Diseases,...

family: Trapnell

full_name: Bruce C. Trapnell

given: Bruce C.

family: Inoue

full_name: Yoshikazu Inoue

given: Yoshikazu

family: Bonella

full_name: Francesco Bonella

given: Francesco

family: Morgan

full_name: Cliff Morgan

given: Cliff

family: Jouneau

full_name: Stéphane Jouneau

given: Stéphane

family: Bendstrup

full_name: Elisabeth Bendstrup

given: Elisabeth

family: Campo

full_name: Ilaria Campo

given: Ilaria

family: Papiris

full_name: Spyros A. Papiris

given: Spyros A.

family: Yamaguchi

full_name: Etsuro Yamaguchi

given: Etsuro

family: Cetinkaya

full_name: Erdogan Cetinkaya

given: Erdogan

family: Ilkovich

full_name: Mikhail M. Ilkovich

given: Mikhail M.

family: Kramer

full_name: Mordechai R. Kramer

given: Mordechai R.

family: Veltkamp

full_name: Marcel Veltkamp

given: Marcel

family: Kreuter

full_name: Michael Kreuter

family: Baba

full_name: Tomohisa Baba

given: Tomohisa

family: Ganslandt

full_name: Cecilia Ganslandt

given: Cecilia

family: Tarnow

full_name: Inge Tarnow

given: Inge

family: Waterer

full_name: Grant Waterer

given: Grant

family: Jouhikainen

full_name: Taneli Jouhikainen

given: Taneli

journal_name: New England Journal of Medicine

oa_colors: green other

evidence: open (via free article)

location_country:
Italy
Switzerland
Netherlands
Spain
Germany
France
Japan
Greece
Israel
Slovakia
Australia
Russian Federation
Portugal
Turkey
Denmark
United States
United Kingdom
Korea, Republic of

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg

location_facility: Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital

location_facility: Asklepios Fachkliniken München - Gauting

location_facility: Niigata University Medical and Dental Hospital

location_facility: II. Pulmonary Department National Institut for TB, Lung Diseases and Chest Surgery

location_facility: Universitätsklinikum Schleswig-Holstein Zentralklinikum, Lübeck Medizinische Klinik III, Pneumologie

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Samsung Medical Center, Division of Pulmonary and Critical Care Medicine

location_facility: Asan Medical Center, Division of Pulmonary and Critical Care Medicine

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Westdeutsches Lungenzentrum am Universitätsklinikum Essen


title: European Network-Paediatric Hodgkin Lymphoma Study Group (EuroNet-PHL) Second International Inter-Group Study for Classical Hodgkin Lymphoma in Children and Adolescents

summary: The EuroNet-PHL-C2 trial is an international, multicentre, randomised controlled trial with the aims to reduce the indication for radiotherapy in newly diagnosed patients with classical Hodgkin lymphoma without compromising cure rates and to investigate a chemotherapy intensification randomisation in intermediate and advanced classical Hodgkin lymphoma to compensate for reduction in radiotherapy.

lead_sponsor: University of Giessen

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
France
Czechia
Poland
Israel
Slovakia
Norway
Ireland
Australia
Belgium
New Zealand
Spain
Denmark
Germany
United Kingdom

location_facility: Starship Blood and Cancer Centre, Starship Children's Hospital

location_facility: Department of Medical Oncology Oslo University Hospital

location_facility: Clinic of Pediatric Oncology University Children's Hospital

location_facility: Department of Pediatric Hematology/Oncology (5054) The Child and Youth Clinic, University Hospital of Copenhagen

location_facility: Head of Department of Pediatric Oncology and Hematology, Polish-American Pediatric Institute, Jagiellonian University Medical Faculty

location_facility: Sección de Onco-Hematología Pediátrica Hospital Universitario Virgen Macarena y Virgen del Rocío

location_facility: CHUV - Centre Hospitalier Universitaire Vaudois = LS, Départment femme - meré - enfant, Service de pédiatrie, Unité d'hématologie-oncologie pédiatrique

location_facility: Pediatric Radiotherapy and Youth Area Unit C.R.O. - Centro di Riferimento Oncologico IRCCS

location_facility: Royal Children's Hospital and Monash Medical Centre Royal Children's Hospital

location_facility: Pediatric Hematology & Oncology Children´s University Hospital

location_facility: Tel Aviv University Schneider Children's Medical Center of Israel The Rina Zaizov Pediatric Hematology Oncology Division

location_facility: Princess Máxima Center for pediatric oncology

location_facility: Service d'Oncohématologie, Hopital d'Ènfants Armand Trousseau

location_facility: Dpt. of Pediatric Hematology and Oncology, Faculty Hospital Motol

location_facility: Paediatric haemato-oncology, University Hospitals of Leuven


title: High Risk Neuroblastoma Study 1 Of Siop-Europe

summary:
RATIONALE: Colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy. Combining chemotherapy with peripheral stem cell transplant may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining isotretinoin and monoclonal antibodies may kill any remaining tumor cells following surgery. It is not yet known which treatment regimen is more effective in treating neuroblastoma.

PURPOSE: This randomized phase III trial is studying how well combination chemotherapy with or without filgrastim before surgery, high-dose chemotherapy, and radiation therapy followed by isotretinoin with or without monoclonal antibody work in treating patients with neuroblastoma.

ReferenceCitation: Ladenstein R, Valteau-Couanet D, Brock P, Yaniv I, Castel V, Laureys G, Malis J, Papadakis V, Lacerda A, Ruud E, Kogner P, Garami M, Balwierz W, Schroeder H, Beck-Popovic M, Schreier G, Machin D, Pötschger U, Pearson A. Randomized Trial of prophylactic granulocyte colony-stimulating factor during rapid COJEC induction in pediatric patients with high-risk neuroblastoma: the European HR-NBL1/SIOPEN study. J Clin Oncol. 2010 Jul 20;28(21):3516-24. doi: 10.1200/JCO.2009.27.3524. Epub 2010 Jun 21.

name: From St. Anna Children's Hospital; Children's Cancer Research Institute; Austrian Institute of Technology, Vienna, Austria; Institut Gustave Roussy, Villejuif, France; Great Ormond Street Hospital, London; The Children's Cancer and Leukaemia Group, University of Leicester, Leicester; Royal Marsden Hospital, Sutton, United Kingdom; Schneider Children's Medical Center of Israel, Petah Tikva, Israel; Hospital Universitario Infantil La Fe, Valencia, Spain; Ghent University Hospital, Ghent, Belgium;...

family: Ladenstein

full_name: Ruth Ladenstein

given: Ruth

family: Valteau-Couanet

full_name: Dominique Valteau-Couanet

given: Dominique

family: Brock

full_name: Penelope Brock

given: Penelope

family: Yaniv

full_name: Isaac Yaniv

given: Isaac

family: Castel

full_name: Victoria Castel

given: Victoria

family: Laureys

full_name: Geneviève Laureys

given: Geneviève

family: Malis

full_name: Josef Malis

given: Josef

family: Papadakis

full_name: Vassilios Papadakis

given: Vassilios

family: Lacerda

full_name: Ana Lacerda

given: Ana

family: Ruud

full_name: Ellen Ruud

given: Ellen

family: Kogner

full_name: Per Kogner

given: Per

family: Garami

full_name: Miklos Garami

given: Miklos

family: Balwierz

full_name: Walentyna Balwierz

given: Walentyna

family: Schroeder

full_name: Henrik Schroeder

given: Henrik

family: Beck-Popovic

full_name: Maja Beck-Popovic

given: Maja

family: Schreier

full_name: Günter Schreier

given: Günter

family: Machin

full_name: David Machin

given: David

family: Pötschger

full_name: Ulrike Pötschger

given: Ulrike

family: Pearson

full_name: Andrew Pearson

given: Andrew

journal_name: Journal of Clinical Oncology

publisher: American Society of Clinical Oncology (ASCO)

title: Randomized Trial of Prophylactic Granulocyte Colony-Stimulating Factor During Rapid COJEC Induction in Pediatric Patients With High-Risk Neuroblastoma: The European HR-NBL1/SIOPEN Study

ReferenceCitation: Veal GJ, Nguyen L, Paci A, Riggi M, Amiel M, Valteau-Couanet D, Brock P, Ladenstein R, Vassal G. Busulfan pharmacokinetics following intravenous and oral dosing regimens in children receiving high-dose myeloablative chemotherapy for high-risk neuroblastoma as part of the HR-NBL-1/SIOPEN trial. Eur J Cancer. 2012 Nov;48(16):3063-72. doi: 10.1016/j.ejca.2012.05.020. Epub 2012 Jun 26.

family: Veal

full_name: G.J. Veal

family: Nguyen

full_name: L. Nguyen

given: L.

family: Paci

full_name: A. Paci

given: A.

family: Riggi

location_country:
Italy
Austria
Sweden
Switzerland
Spain
France
Israel
Norway
Ireland
Belgium
Portugal
Denmark
United Kingdom

location_facility: Instituto Portugues de Oncologia de Francisco Gentil - Centro Regional de Oncologia de Lisboa, SA

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary

location_facility: Institute of Child Health at University of Bristol

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Aarhus Universitetshospital - Aarhus Sygehus

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Fondazione Istituto Nazionale dei Tumori

location_facility: Great Ormond Street Hospital for Children

location_facility: Our Lady's Hospital for Sick Children Crumlin


title: A Phase 1 First-in-Human Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AMG 228 in Subjects With Selected Advanced Solid Tumors

summary: The purpose of this study is to evaluate the safety, pharmacokinetics, anti-tumor activity, and identify a tolerable dose of AMG 228 in subjects with advanced solid tumors.

ReferenceCitation: Tran B, Carvajal RD, Marabelle A, Patel SP, LoRusso PM, Rasmussen E, Juan G, Upreti VV, Beers C, Ngarmchamnanrith G, Schöffski P. Dose escalation results from a first-in-human, phase 1 study of glucocorticoid-induced TNF receptor-related protein agonist AMG 228 in patients with advanced solid tumors. J Immunother Cancer. 2018 Sep 25;6(1):93. doi: 10.1186/s40425-018-0407-x.

family: Tran

full_name: Ben Tran

given: Ben

family: Carvajal

full_name: Richard D. Carvajal

given: Richard D.

family: Marabelle

full_name: Aurelien Marabelle

given: Aurelien

family: Patel

full_name: Sandip Pravin Patel

given: Sandip Pravin

family: LoRusso

full_name: Patricia M. LoRusso

given: Patricia M.

family: Rasmussen

full_name: Erik Rasmussen

given: Erik

family: Juan

full_name: Gloria Juan

given: Gloria

family: Upreti

full_name: Vijay V. Upreti

given: Vijay V.

family: Beers

full_name: Courtney Beers

given: Courtney

family: Ngarmchamnanrith

full_name: Gataree Ngarmchamnanrith

given: Gataree

family: Schöffski

full_name: Patrick Schöffski

given: Patrick

journal_name: Journal for ImmunoTherapy of Cancer

repository_normalized: University of Melbourne - Minerva Access

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

title: Dose escalation results from a first-in-human, phase 1 study of glucocorticoid-induced TNF receptor–related protein agonist AMG 228 in patients with advanced solid tumors

lead_sponsor: Amgen

location_country:
Germany
France
Belgium
Australia
United States


title: Balloon Expandable vs. Self Expanding Transcatheter Valve for Degenerated Bioprosthesis. THE BASELINE TRIAL.

acronym: BASELINE

summary:
Transcatheter aortic valve implantation (TAVI) serves a growing spectrum of patients with symptomatic severe aortic stenosis (AS). Approximately 80% of surgical aortic valve replacements is performed using a bioprosthesis1. Durability of surgical bioprostheses varies based on the patient's age at the moment of implantation, type and size etc2. TAVI has become the preferred treatment for degenerated aortic bioprostheses in elderly patients3. The median time since index surgical aortic valve replacement (SAVR) and for bioprosthetic valve degeneration is typically 8 - 10 years4-6. TAVI in this setting has proven to have equally favorable results as in native aortic valves7. Balloon expandable8 and self-expanding9 transcatheter heart valves (THV) can be used in a degenerated bioprosthesis and each have specific assets and limitations. TAVI in a failed bioprosthesis can cause coronary obstruction, THV migration, paravalvular leakage and prosthesis patient mismatch. The SAPIEN-3 / Ultra and EVOLUT R/Pro are the 2 most commonly used THV platforms in contemporary clinical practice including treatment of failing surgical aortic bioprostheses.

Objective: To compare TAVI with EVOLUT R/Pro vs. SAPIEN-3 / Ultra in terms of device success.

Study design: International multi-center randomized study with 1:1 randomization to TAVI with SAPIEN-3 / Ultra or Evolut R/Pro.

Study population: 440 patients with a failing surgical aortic bioprosthesis (aortic stenosis with or without aortic regurgitation) and selected for transfemoral TAVI by heart-team consensus.

Investigational intervention: Transfemoral TAVI with SAPIEN-3 / Ultra or Evolut R/PRO

Main study parameters/endpoints:

Primary endpoint is device success at 30 days

Defined by

Absence of procedural mortality AND
Correct positioning of a single prosthetic heart valve into the proper anatomical location AND
Intended performance of the prosthetic heart valve (no severe prosthesis- patient mismatch and mean aortic valve gradient < 20 mmHg or peak velocity < 3 m/s, AND no moderate or severe prosthetic valve regurgitation). Severe prosthesis patient mismatch is defined by effective orifice area (EOAi) ≤0.65 cm2/m2
Safety endpoint at 1 year defined by the composite of all-cause death, disabling stroke, rehospitalization for heart failure or valve related problems.

lead_sponsor: Erasmus Medical Center

location_country:
Italy
Austria
Switzerland
Netherlands
Germany
France
Portugal
Denmark
United States
United Kingdom
Canada

location_facility: St Paul's and Vancouver General Hospital


title: A Phase II Study of Blincyto in Children With CD19+ Precursor B-lineage ALL and MRD-Positivity Before and/or Following First Allogeneic HSCT

summary:
A phase II trial of continuous intravenous infusion of Blincyto given over a 28-day cycle. Starting day for patients who are MRD-positive before HSCT is between day +60 and day +100 and for patients who become MRD-positive post HSCT it is between day +60 and day +360 post HSCT.

Patients will be evaluated for response at day +28 (+4 days) (bone marrow morphology and MRD analysis - defined by PCR/FLOW-techniques) after start of Blincyto-treatment at the end of first Blincyto infusion and at regular post-TX-checks (according to FORUM: days +28, +60, +100, +180 and +360 after HSCT).

The dose of Blincyto used in this trial will be 15 mcg/m2/day for 28 days

lead_sponsor: Prof. Christina Peters

location_country: Italy Austria France Czechia Greece Poland Slovakia Norway Belgium Spain Denmark

location_facility: IRCCS Ospedale Pediatrico Bambino Gesù Roma

location_facility: Nicolaus Copernicus University Collegium Medicum

location_facility: University Hospital Gasthuisberg (UZ Leuven)


lead_sponsor: Merck Sharp & Dohme Corp.

location_country:
Italy
France
Israel
Spain
United States
Canada

location_facility: A.O. Universitaria Policlinico S. Orsola-Malpighi ( Site 1400)

location_facility: IRCCS Ospedale San Raffaele ( Site 1402)

location_facility: Centre Hospitalier Lyon-Sud ( Site 0804)

location_facility: Instituto Catalan de Oncologia ICO - Hospital Duran i Reynals ( Site 2000)

location_facility: Hospital Universitari Vall d'Hebron ( Site 2001)

location_facility: Medical Oncology Associates (Summit Cancer Centers) ( Site 2710)

location_facility: John Theurer Cancer Center at Hackensack University Medical Center ( Site 2704)

location_facility: Hospital Universitario de Salamanca ( Site 2002)

location_facility: Hospital Universitario 12 de Octubre ( Site 2003)

location_facility: Hadassah Ein Karem Jerusalem ( Site 1300)

title: A Phase 2 Study to Evaluate the Efficacy and Safety of MK-1026 in Participants With Hematologic Malignancies

summary: The purpose of this study is to evaluate the safety and efficacy of nemtabrutinib (formerly ARQ 531) in participants with hematologic malignancies of chronic lymphocytic leukemia (CLL)/ small lymphocytic lymphoma (SLL), Richter's transformation, marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), follicular lymphoma (FL), and Waldenström's macroglobulinemia (WM).


ipd_sharing_description:
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency.

As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

primary_purpose: Other

location_country:
Italy
United States
Germany
France
Poland
Belgium
New Zealand
Canada
United Kingdom

acronym: INSIGHT

location_facility:
Johns Hopkins University
Irccs Ospedale Maggiore Di Milano
Beth Israel Deaconess Medical Center
Toronto General Hospital
University Medical Center
New Zealand Clinical Research
ID Clinic
Grahame Hayton Unit
UZ Antwerpen
Kings College Hospital
Hôpital Beaujon

title: A Phase 2 Randomized, Open-label, Parallel-group, Multicenter Study to Assess Intrahepatic and Peripheral Changes of Immunologic and Virologic Markers in Response to Combination Regimens Containing JNJ-73763989 and Nucleos(t)Ide Analog With or Without JNJ-56136379 in Patients With Chronic Hepatitis B Virus Infection

summary: The purpose of this study is to assess changes in intrahepatic hepatitis B surface antigen (HBsAg) between baseline and on-treatment liver biopsy in response to JNJ-3989-based combination treatment.


title: BETAEVAL_Global- The New BETACONNECT® Auto-injector: Adherence and EVALuation of MS Patients Treated With Betaferon®

summary:
Considering the significance of an early and consequent Multiple Sclerosis (MS) treatment as well as the challenge to achieve high adherence to treatment, evaluating the benefits of any new measure to improve adherence is important. The data storage capabilities of the BETACONNECT device, including the automated recording of injections, will facilitate the collection of reliable data on patient's injection behavior and adherence, which should be unaffected by recall bias or reporting bias.

To better understand the utilities of the new BETACONNECT device and characterize its contribution to adherence, we plan to prospectively follow-up MS patients using this device for 24 weeks. The study will take place in a real-life setting in Neurology centers across Europe.

lead_sponsor: Bayer

location_country:
Italy
Austria
Bosnia and Herzegovina
Switzerland
Croatia
Czechia
France
Greece
Hungary
Belgium
Spain


title: Multi-center Phase 2 Study to Assess the Safety, Tolerability and Early Signs of Efficacy of Tid Orally Administered BAY63-2521 in Adult Delta F508 Homozygous Cystic Fibrosis Patients

summary: Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi

ReferenceCitation: Derichs N, Taylor-Cousar JL, Davies JC, Fajac I, Tullis E, Nazareth D, Downey DG, Rosenbluth D, Malfroot A, Saunders C, Jensen R, Solomon GM, Vermeulen F, Kaiser A, Willmann S, Saleh S, Droebner K, Sandner P, Bear CE, Hoffmann A, Ratjen F, Rowe SM; Rio-CF Study Group. Riociguat for the treatment of Phe508del homozygous adults with cystic fibrosis. J Cyst Fibros. 2021 Aug 19. pii: S1569-1993(21)01329-1. doi: 10.1016/j.jcf.2021.07.015. [Epub ahead of print]

family: Derichs

full_name: Nico Derichs

given: Nico

family: Taylor-Cousar

full_name: Jennifer L. Taylor-Cousar

given: Jennifer L.

family: Davies

full_name: Jane C. Davies

given: Jane C.

family: Fajac

full_name: Isabelle Fajac

given: Isabelle

family: Tullis

full_name: Elizabeth Tullis

given: Elizabeth

family: Nazareth

full_name: Dilip Nazareth

given: Dilip

family: Downey

full_name: Damian G. Downey

given: Damian G.

family: Rosenbluth

full_name: Daniel Rosenbluth

given: Daniel

family: Malfroot

full_name: Anne Malfroot

given: Anne

family: Saunders

full_name: Clare Saunders

given: Clare

family: Jensen

full_name: Renee Jensen

given: Renee

family: Solomon

full_name: George M. Solomon

given: George M.

family: Vermeulen

full_name: Francois Vermeulen

given: Francois

family: Kaiser

full_name: Andreas Kaiser

given: Andreas

family: Willmann

full_name: Stefan Willmann

given: Stefan

family: Saleh

full_name: Soundos Saleh

given: Soundos

family: Droebner

full_name: Karoline Droebner

given: Karoline

family: Sandner

full_name: Peter Sandner

given: Peter

family: Bear

full_name: Christine E. Bear

given: Christine E.

family: Hoffmann

full_name: Anja Hoffmann

given: Anja

family: Ratjen

full_name: Felix Ratjen

given: Felix

family: Rowe

full_name: Steven M. Rowe

given: Steven M.

location_country:
United States
Netherlands
Germany
France
Belgium
Canada
United Kingdom


title: A Multicenter Randomized Trial, Comparing a 25G EUS Fine Needle Aspiration (FNA) Device With a 20G EUS ProCore Fine Needle Biopsy (FNB) Device

acronym: ASPRO

summary: The aim of this study is to compare the diagnostic accuracy of two EUS-guided tissue acquisition devices; the 25G Echotip Ultra Fine Needle Aspiration (FNA) device and the 20G Echotip ProCore Fine Needle Biopsy (FNB) device.

primary_purpose: Diagnostic

ReferenceCitation: van Riet PA, Giorgio Arcidiacono P, Petrone M, Quoc Nguyen N, Kitano M, Chang K, Larghi A, Iglesias-Garcia J, Giovannini M, van der Merwe S, Santo E, Baldaque-Silva F, Bucobo JC, Bruno MJ, Aslanian HR, Cahen DL, Farrell J. Combined versus single use 20 G fine-needle biopsy and 25 G fine-needle aspiration for endoscopic ultrasound-guided tissue sampling of solid gastrointestinal lesions. Endoscopy. 2020 Jan;52(1):37-44. doi: 10.1055/a-0966-8755. Epub 2019 Jul 22.

name: Gastroenterology and Hepatology, Erasmus MC University Medical Center Rotterdam, Rotterdam, the Netherlands

family: van Riet

full_name: Priscilla A. van Riet

given: Priscilla A.

name: Gastroenterology and Gastrointestinal Endoscopy, Vita Salute San Raffaele University, Milan, Italy

family: Giorgio Arcidiacono

full_name: Paolo Giorgio Arcidiacono

given: Paolo

family: Petrone

full_name: Mariachiara Petrone

given: Mariachiara

name: Gastroenterology and Hepatology, Royal Adelaide Hospital, Adelaide, Australia

family: Quoc Nguyen

full_name: Nam Quoc Nguyen

given: Nam

name: Department of Gastroenterology and Hepatology, Kindai University, Osaka-Sayama, Japan

family: Kitano

full_name: Masayuki Kitano

given: Masayuki

name: Comprehensive Digestive Disease Center, University of California, Irvine, California, United States

family: Chang

full_name: Kenneth Chang

given: Kenneth

name: Digestive Endoscopy Unit, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy

family: Larghi

full_name: Alberto Larghi

given: Alberto

name: Gastroenterology, University Hospital of Santiago de Compostela, Santiago de Compostela, Spain

family: Iglesias-Garcia

full_name: Julio Iglesias-Garcia

given: Julio

name: Institut Paoli-Calmettes, Marseilles, France

family: Giovannini

full_name: Marc Giovannini

given: Marc

name: Department of Hepatology and Biliopancreatic Disease, University Hospital Leuven, Leuven, Belgium

family: van der Merwe

full_name: Schalk van der Merwe

given: Schalk

name: Department of Gastroenterology, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel

family: Santo

full_name: Erwin Santo

given: Erwin

name: Gastro Center, Karolinska University Hospital, Huddinge, Sweden

family: Baldaque-Silva

full_name: Francisco Baldaque-Silva

given: Francisco

name: Division of Gastroenterology, Stony Brook University Hospital, New York, New York, United States

family: Bucobo

full_name: Juan Carlos Bucobo

given: Juan Carlos

family: Bruno

full_name: Marco J. Bruno

given: Marco J.

name: Department of Medicine, Section of Digestive Diseases, Yale University School of Medicine, New Haven, United States

family: Aslanian

full_name: Harry R. Aslanian

given: Harry R.

family: Cahen

full_name: Djuna L. Cahen

given: Djuna L.

family: Farrell

full_name: James Farrell

given: James

journal_name: Endoscopy

publisher: Georg Thieme Verlag KG

publisher_dissemination: Georg Thieme

publisher_group: Georg Thieme

publisher_normalized: Georg Thieme

ReferenceCitation: van Riet PA, Larghi A, Attili F, Rindi G, Nguyen NQ, Ruszkiewicz A, Kitano M, Chikugo T, Aslanian H, Farrell J, Robert M, Adeniran A, Van Der Merwe S, Roskams T, Chang K, Lin F, Lee JG, Arcidiacono PG, Petrone M, Doglioni C, Iglesias-Garcia J, Abdulkader I, Giovannini M, Bories E, Poizat F, Santo E, Scapa E, Marmor S, Bucobo JC, Buscaglia JM, Heimann A, Wu M, Baldaque-Silva F, Moro CF, Erler NS, Biermann K, Poley JW, Cahen DL, Bruno MJ. A multicenter randomized trial comparing a 25-gauge EUS fine-needle aspiration device with a 20-gauge EUS fine-needle biopsy device. Gastrointest Endosc. 2019 Feb;89(2):329-339. doi: 10.1016/j.gie.2018.10.026. Epub 2018 Oct 24.

family: Attili

full_name: Fabia Attili

given: Fabia

family: Rindi

location_country:
Italy
Sweden
Netherlands
France
Japan
Israel
Belgium
Australia
Spain
United States

location_facility: University Hospital of Santiago de Compostella


title: Patient-Specific Simulation Used for Case Rehearsal Prior to EVAR Procedure - Evaluation of Clinical Performance With and Without Prior Rehearsal Using the Procedure Rehearsal Studio. Multi-center Randomized Study

acronym: PRS

summary: The purpose of this study is to evaluate the procedural and clinical benefits of patient specific pre-procedure rehearsal for operators with various experience levels as a tool for optimizing Endovascular Aneurysm Repair (EVAR) procedures.

lead_sponsor: Simbionix US Corporation

location_country:
Italy
United States
France

location_facility: University of South Florida, Tampa General Hospital


ReferenceCitation: Bjornstad P, Laffel L, Tamborlane WV, Simons G, Hantel S, von Eynatten M, George J, Marquard J, Cherney DZI. Acute Effect of Empagliflozin on Fractional Excretion of Sodium and eGFR in Youth With Type 2 Diabetes. Diabetes Care. 2018 Aug;41(8):e129-e130. doi: 10.2337/dc18-0394. Epub 2018 Jun 25.

family: Bjornstad

full_name: Petter Bjornstad

given: Petter

family: Laffel

full_name: Lori Laffel

given: Lori

family: Tamborlane

full_name: William V. Tamborlane

given: William V.

family: Simons

full_name: Gudrun Simons

given: Gudrun

family: Hantel

full_name: Stefan Hantel

given: Stefan

family: von Eynatten

full_name: Maximilian von Eynatten

given: Maximilian

family: George

full_name: Jyothis George

given: Jyothis

family: Marquard

full_name: Jan Marquard

given: Jan

family: Cherney

full_name: David Z.I. Cherney

given: David Z.I.

journal_name: Diabetes Care

license: implied-oa

license_normalized: implied-oa

publisher: American Diabetes Association

publisher_dissemination: American Diabetes Association

publisher_group: American Diabetes Association

publisher_normalized: American Diabetes Association

ReferenceCitation: Laffel LMB, Tamborlane WV, Yver A, Simons G, Wu J, Nock V, Hobson D, Hughan KS, Kaspers S, Marquard J. Pharmacokinetic and pharmacodynamic profile of the sodium-glucose co-transporter-2 inhibitor empagliflozin in young people with Type 2 diabetes: a randomized trial. Diabet Med. 2018 Aug;35(8):1096-1104. doi: 10.1111/dme.13629. Epub 2018 May 6.

name: Harvard Medical School Joslin Diabetes Center Boston MA USA

full_name: L. M. B. Laffel

given: L. M. B.

name: Yale School of Medicine New Haven CT USA

full_name: W. V. Tamborlane

given: W. V.

family: Yver

full_name: A. Yver

given: A.

name: Boehringer Ingelheim International GmbH Biberach an der Riss Germany

full_name: G. Simons

given: G.

name: Boehringer Ingelheim Pharmaceuticals Inc. Ridgefield CT USA

family: Wu

full_name: J. Wu

given: J.

family: Nock

full_name: V. Nock

given: V.

family: Hobson

full_name: D. Hobson

given: D.

name: Division of Pediatric Endocrinology, Metabolism and Diabetes Mellitus Children's Hospital of Pittsburgh University of Pittsburgh School of Medicine Pittsburgh PA USA

family: Hughan

full_name: K. S. Hughan

given: K. S.

name: Boehringer Ingelheim International GmbH Ingelheim Germany

family: Kaspers

full_name: S. Kaspers

given: S.

title: Pharmacokinetic and pharmacodynamic profile of the sodium‐glucose co‐transporter‐2 inhibitor empagliflozin in young people with Type 2 diabetes: a randomized trial

location_country:
South Africa
Mexico
France
Israel
United States

location_facility: 1245.87.33001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01002 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01013 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.97202 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.97203 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.27002 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.52001 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01012 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.27003 Boehringer Ingelheim Investigational Site

location_facility: 1245.87.01004 Boehringer Ingelheim Investigational Site

title: An Open-label, Randomised, Multicentre, Single-dose, Parallel Group Trial to Evaluate Pharmacokinetics and Pharmacodynamics of Empagliflozin in Children and Adolescents From 10 to Less Than 18 Years of Age With Type 2 Diabetes Mellitus

summary: The aim of the study is to generate pharmacokinetic and pharmacodynamic data to identify the safe-effective dose of empagliflozin in children and adolescents aged 10 to less than 18 years with type 2 diabetes mellitus.


title: Long Term Experience With Abatacept in Routine Clinical Practice

acronym: ACTION

summary: The purpose of this study is to estimate the retention rate of Abatacept over 24 months in routine clinical practice in every participating country depending on the treatment line.

ReferenceCitation: Alten R, Mariette X, Lorenz HM, Nüßlein H, Galeazzi M, Navarro F, Chartier M, Heitzmann J, Poncet C, Rauch C, Le Bars M. Predictors of abatacept retention over 2 years in patients with rheumatoid arthritis: results from the real-world ACTION study. Clin Rheumatol. 2019 May;38(5):1413-1424. doi: 10.1007/s10067-019-04449-w. Epub 2019 Feb 21. Erratum in: Clin Rheumatol. 2019 Apr 11;:.

family: Alten

full_name: Rieke Alten

given: Rieke

family: Mariette

full_name: Xavier Mariette

given: Xavier

family: Lorenz

full_name: Hanns-Martin Lorenz

given: Hanns-Martin

family: Nüßlein

full_name: Hubert Nüßlein

given: Hubert

family: Galeazzi

full_name: Mauro Galeazzi

given: Mauro

family: Navarro

full_name: Federico Navarro

given: Federico

family: Chartier

full_name: Melanie Chartier

given: Melanie

family: Heitzmann

full_name: Julia Heitzmann

given: Julia

family: Poncet

full_name: Coralie Poncet

given: Coralie

family: Rauch

full_name: Christiane Rauch

given: Christiane

family: Le Bars

full_name: Manuela Le Bars

given: Manuela

journal_name: Clinical Rheumatology

oa_colors: green hybrid

repository_institution: INFN Catania - INFM-OAR

repository_normalized: Zenodo

ReferenceCitation: Alten R, Mariette X, Lorenz HM, Galeazzi M, Cantagrel A, Nüßlein HG, Chartier M, Elbez Y, Rauch C, Le Bars M. Real-world predictors of 12-month intravenous abatacept retention in patients with rheumatoid arthritis in the ACTION observational study. RMD Open. 2017 Dec 29;3(2):e000538. doi: 10.1136/rmdopen-2017-000538. eCollection 2017.

family: Cantagrel

full_name: Alain Cantagrel

given: Alain

full_name: Hubert G Nüßlein

given: Hubert G

title: Real-world predictors of 12–month intravenous abatacept retention in patients with rheumatoid arthritis in the ACTION observational study

ReferenceCitation: Alten R, Burkhardt H, Feist E, Krüger K, Rech J, Rubbert-Roth A, Voll RE, Elbez Y, Rauch C. Abatacept used in combination with non-methotrexate disease-modifying antirheumatic drugs: a descriptive analysis of data from interventional trials and the real-world setting. Arthritis Res Ther. 2018 Jan 2;20(1):1. doi: 10.1186/s13075-017-1488-5.

repository_institution: Kooperativer Bibliotheksverbund Berlin-Brandenburg (KOBV), on behalf of the Universitätsbibliothek Erlangen-Nürnberg - OPUS FAU - Online-Publikationssystem der Friedrich-Alexander-Universität Erlangen-Nürnberg

ReferenceCitation: Alten R, Nüßlein HG, Mariette X, Galeazzi M, Lorenz HM, Cantagrel A, Chartier M, Poncet C, Rauch C, Le Bars M. Baseline autoantibodies preferentially impact abatacept efficacy in patients with rheumatoid arthritis who are biologic naïve: 6-month results from a real-world, international, prospective study. RMD Open. 2017 Feb 13;3(1):e000345. doi: 10.1136/rmdopen-2016-000345. eCollection 2017.

location_country:
Italy
Austria
Switzerland
Netherlands
Germany
France
Greece
Ireland
Belgium
Spain
Denmark
Canada
Czech Republic


title: A Phase 3b, Prospective, Randomized, Open-label, Blind Evaluator (PROBE) Study Evaluating the Efficacy and Safety of (LMW) Heparin/Edoxaban Versus Dalteparin in Venous Thromboembolism Associated With Cancer

summary: The primary objective is to demonstrate the non-inferiority of edoxaban (preceded by a short course of LMWH) compared with dalteparin for the prevention of the combined outcome of recurrent venous thromboembolism (VTE) or major bleeding in subjects with VTE associated with cancer during a 12-month study period. If non-inferiority is established, LMWH/edoxaban will be compared with dalteparin for superiority.

ReferenceCitation: Raskob GE, van Es N, Verhamme P, Carrier M, Di Nisio M, Garcia D, Grosso MA, Kakkar AK, Kovacs MJ, Mercuri MF, Meyer G, Segers A, Shi M, Wang TF, Yeo E, Zhang G, Zwicker JI, Weitz JI, Büller HR; Hokusai VTE Cancer Investigators. Edoxaban for the Treatment of Cancer-Associated Venous Thromboembolism. N Engl J Med. 2018 Feb 15;378(7):615-624. doi: 10.1056/NEJMoa1711948. Epub 2017 Dec 12.

name: From the University of Oklahoma Health Sciences Center, College of Public Health, Oklahoma City (G.E.R.); the Department of Vascular Medicine, Academic Medical Center, University of Amsterdam (N.E., H.R.B.), and ITREAS, Academic Research Organization (A.S.) — both in Amsterdam; the Department of Vascular Medicine and Hemostasis, University Hospitals Leuven, Leuven, Belgium (P.V.); Ottawa Hospital Research Institute, Ottawa (M.C.), London Health Sciences Centre–Victoria Hospital, London, ON (M.J.K.),...

family: Raskob

full_name: Gary E. Raskob

given: Gary E.

family: van Es

full_name: Nick van Es

given: Nick

family: Verhamme

full_name: Peter Verhamme

given: Peter

family: Carrier

full_name: Marc Carrier

given: Marc

family: Di Nisio

full_name: Marcello Di Nisio

given: Marcello

family: Garcia

full_name: David Garcia

given: David

family: Grosso

full_name: Michael A. Grosso

given: Michael A.

family: Kakkar

full_name: Ajay K. Kakkar

given: Ajay K.

family: Kovacs

full_name: Michael J. Kovacs

given: Michael J.

family: Mercuri

full_name: Michele F. Mercuri

given: Michele F.

family: Meyer

full_name: Guy Meyer

given: Guy

family: Segers

full_name: Annelise Segers

given: Annelise

family: Shi

full_name: Minggao Shi

given: Minggao

family: Wang

full_name: Tzu-Fei Wang

given: Tzu-Fei

family: Yeo

full_name: Erik Yeo

given: Erik

family: Zhang

full_name: George Zhang

given: George

family: Zwicker

full_name: Jeffrey I. Zwicker

given: Jeffrey I.

family: Weitz

full_name: Jeffrey I. Weitz

family: Büller

full_name: Harry R. Büller

given: Harry R.

repository_normalized: UCL Discovery

ReferenceCitation: van Es N, Di Nisio M, Bleker SM, Segers A, Mercuri MF, Schwocho L, Kakkar A, Weitz JI, Beyer-Westendorf J, Boda Z, Carrier M, Chlumsky J, Décousus H, Garcia D, Gibbs H, Kamphuisen PW, Monreal M, Ockelford P, Pabinger I, Verhamme P, Grosso MA, Büller HR, Raskob GE. Edoxaban for treatment of venous thromboembolism in patients with cancer. Rationale and design of the Hokusai VTE-cancer study. Thromb Haemost. 2015 Nov 25;114(6):1268-76. doi: 10.1160/TH15-06-0452. Epub 2015 Aug 13.

location_country:
Italy
Netherlands
France
Hungary
Belgium
United States