title: Clinical and Health Economic Evaluation of a New Baha® Abutment, With a Minimally Invasive Surgical Technique. An International Multicentre, Open, Randomised, Comparative, Parallel Group, Investigation. 1y Investigation, 2y Follow-up

summary:
To demonstrate that the minimally invasive surgical procedure in combination with the use of the Cochlear Baha BA400 abutment is associated with a reduction of inflammation/infection, overgrowth, pain and numbness at the site of implantation compared to the traditional surgical procedure in combination with the use of the standard Baha abutment (Cochlear Baha BA300 Abutment).

To demonstrate that the minimally invasive surgical procedure in combination with the use of the Cochlear Baha BA400 abutment is associated with a reduction in direct medical costs, due to shorter surgical procedures, faster wound healing and less complications compared to the traditional surgical procedure in combination with the use of the standard Baha abutment (Cochlear Baha BA300 Abutment).

ReferenceCitation: van Hoof M, Wigren S, Ivarsson Blechert J, Molin M, Andersson H, Mateijsen DJM, Bom SJH, Calmels MN, van der Rijt AJM, Flynn MC, van Tongeren J, Hof JR, Brunings JW, Anteunis LJC, Marco Algarra J, Stokroos RJ, Joore MA. A Multinational Cost-Consequence Analysis of a Bone Conduction Hearing Implant System-A Randomized Trial of a Conventional vs. a Less Invasive Treatment With New Abutment Technology. Front Neurol. 2020 Mar 13;11:106. doi: 10.3389/fneur.2020.00106. eCollection 2020.

lead_sponsor: Cochlear Bone Anchored Solutions

location_country: Netherlands France Spain Sweden

location_facility: Otorhinolaryngology, Hospital Clinico Universitario De Valencia


ipd_sharing: No

status: Terminated

primary_purpose: Treatment

intervention_type: Drug

title: An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)

study_completion_date_type: Actual

lead_sponsor: PTC Therapeutics

type: EudraCT Number

source: euctr

study_start_date_type: Actual

study_type: Interventional

enrollment_type: Actual

location_country:
Spain
Italy
United States
France
Israel
Belgium
Sweden

location_facility: Karolinska University Hospital, Huddinge

location_facility: Hadassah University Hospital - Mount Scopus

location_facility: Hôpital Universitaire des Enfants Reine Fabiola

summary: The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).

lead_sponsor_type: industriel

status_simplified: Unknown


type: Other Identifier

title: POLARIS: A Non-interventional Study in Patients With Diabetic Macular Edema (DME) With Central Involvement to Assess the Effectiveness of Existing Anti-vascular Endothelial Growth Factor (Anti-VEGF) Treatment Regimens

acronym: POLARIS

summary: POLARIS : A non-interventional study in patients with diabetic macular edema (DME) with central involvement to assess the effectiveness of existing anti-vascular endothelial growth factor (anti-VEGF) treatment regimens.

status: Completed

study_type: Observational

lead_sponsor: Bayer

location_country:
Portugal
Spain
Greece
Russian Federation
United Kingdom
France
Germany
Slovakia

status_simplified: Completed


summary: This is a prospective, randomised, open label, controlled pilot study to evaluate the safety and preliminary effectiveness of the ReCell Autologous Cell Harvesting Device (ReCell) for the management of chronic leg ulcers associated with venous insufficiency. Outcome will be compared between study participants receiving standard care (debridement, compression therapy) and participants receiving ReCell in addition to standard care.

design_allocation: Randomized

lead_sponsor: Avita Medical

location_country:
France
United Kingdom

location_facility: Cambridge University Hospitals - Addenbrooke's Hospital

location_facility: The Leeds Teaching Hospitals - James's University Hospital

location_facility: University Hospital of South Manchester - Wythenshawe Hospital

intervention_type: Other


title: Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.

summary: The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.

lead_sponsor: Swedish Orphan Biovitrum

location_country:
Germany
France
United Kingdom

location_facility: Evelina Children's Hospital, St Thomas' Hospital


type: Other Grant/Funding Number

title: International Validation and Testing of a Supplementary Questionnaire Module for Assessment of Oral and Dental Health in Cancer; the QLQ-OH17

summary:
Assessment of oral and dental problems is seldom routine in clinical oncology, despite the potential negative impact of these problems on nutritional status, social function and quality of life (QoL). A brief, assessment tool for oral/dental health and related QoL-issues to improve symptom management has been requested. The present study will be conducted on behalf of and with support from the European Organisation for Research and Treatment of Cancer - (EORTC) Quality of Life Group (QLG). The study represents phase IV, the final step, in the development of an international, symptom specific questionnaire module, focusing on oral and dental problems in relation to cancer and its treatment. Phase I-III of this stepwise development process was conducted from 2008 to 2011, as an international collaboration and conducted according to the guidelines for module development set forth by the EORTC QLG. The resulting module, the QLQ-OH17, is now subject to an international field testing and validation study as described in this project description.

The present version of the QLQ-OH17 consists of 17 items conceptualized into four multi-item scales (pain/discomfort, xerostomia, eating and information) and three single items related to use of dentures and future worries. The aim of the present study is to conduct phase IV; an international field study to confirm the psychometric properties of the QLQ-OH17

time_perspective: Cross-Sectional

ReferenceType: background

ReferenceCitation: Hjermstad MJ, Bergenmar M, Bjordal K, Fisher SE, Hofmeister D, Montel S, Nicolatou-Galitis O, Pinto M, Raber-Durlacher J, Singer S, Tomaszewska IM, Tomaszewski KA, Verdonck-de Leeuw I, Yarom N, Winstanley JB, Herlofson BB; EORTC QoL Group. International field testing of the psychometric properties of an EORTC quality of life module for oral health: the EORTC QLQ-OH15. Support Care Cancer. 2016 Sep;24(9):3915-24. doi: 10.1007/s00520-016-3216-0. Epub 2016 Apr 25.

ReferenceType: result

ReferenceCitation: Hjermstad MJ, Bergenmar M, Fisher SE, Montel S, Nicolatou-Galitis O, Raber-Durlacher J, Singer S, Verdonck-de Leeuw I, Weis J, Yarom N, Herlofson BB. The EORTC QLQ-OH17: a supplementary module to the EORTC QLQ-C30 for assessment of oral health and quality of life in cancer patients. Eur J Cancer. 2012 Sep;48(14):2203-11. doi: 10.1016/j.ejca.2012.04.003. Epub 2012 May 8.

ReferenceType: derived

ReferenceCitation: Johns SA, Brown LF, Beck-Coon K, Talib TL, Monahan PO, Giesler RB, Tong Y, Wilhelm L, Carpenter JS, Von Ah D, Wagner CD, de Groot M, Schmidt K, Monceski D, Danh M, Alyea JM, Miller KD, Kroenke K. Randomized controlled pilot trial of mindfulness-based stress reduction compared to psychoeducational support for persistently fatigued breast and colorectal cancer survivors. Support Care Cancer. 2016 Oct;24(10):4085-96. doi: 10.1007/s00520-016-3220-4. Epub 2016 May 17.

ipd_sharing: Yes

ipd_sharing_description: Phase III and Phase IV reports approved by the EORTC Quality of Life Group Executive Committee. First publication in Eur J Cancer. 2012 Sep;48(14):2203-11. doi: 10.1016/j.ejca.2012.04.003. Second publication accepted for publication in Supp Care Cancer, April 2016

lead_sponsor: Oslo University Hospital

location_country:
Poland
United Kingdom
Italy
Greece
Netherlands
Sweden
France
Israel
Norway
Germany

location_facility: Istituto Nazionale Tumori "Fondazione G. Pascale"- IRCCS,

lead_sponsor_type: academique


title: A Phase II, Randomized, Double-Blind, Placebo-Controlled Bronchoscopy Study to Evaluate the Effects of Lebrikizumab on Airway Eosinophilic Inflammation in Patients With Uncontrolled Asthma on Inhaled Corticosteroids and a Second Controller Medication

summary: This Phase II, randomized, double-blind, placebo-controlled, multicenter study will evaluate the effects of lebrikizumab on airway eosinophilic inflammation in participants with uncontrolled asthma who are using inhaled corticosteroid (ICS) treatment and a second controller medication. Enrolled participants will undergo a 3-week screening period during which assessments, including a bronchoscopy procedure, will be made. Participants will subsequently be randomized to receive lebrikizumab or placebo by subcutaneous (SC) injection on Day 1, Day 8, Week 4, and Week 8. Participants will continue their standard of care therapy throughout the study. End of treatment assessments will be taken at Week 12. Total study period, including screening and follow-up, is expected to last 23 weeks.

ReferenceCitation: Austin CD, Gonzalez Edick M, Ferrando RE, Solon M, Baca M, Mesh K, Bradding P, Gauvreau GM, Sumino K, FitzGerald JM, Israel E, Bjermer L, Bourdin A, Arron JR, Choy DF, Olsson JK, Abreu F, Howard M, Wong K, Cai F, Peng K, Putnam WS, Holweg CTJ, Matthews JG, Kraft M, Woodruff PG; CLAVIER Investigators. A randomized, placebo-controlled trial evaluating effects of lebrikizumab on airway eosinophilic inflammation and remodelling in uncontrolled asthma (CLAVIER). Clin Exp Allergy. 2020 Dec;50(12):1342-1351. doi: 10.1111/cea.13731. Epub 2020 Oct 4.

lead_sponsor: Hoffmann-La Roche

location_country:
United Kingdom
Ireland
Canada
United States
France
Sweden

location_facility: Wake Forest University Baptist Medical Center; Gastroenterology & Digestive Health

location_facility: University of Miami School of Medicine - Sylvester at Deerfield

location_facility: University of California Davis Health System; Division of Pulmonary and Critical Care Medicine

location_facility: McMaster University Health Sciences Center

location_facility: Brigham and Women's Hospital; Pulmonary Division

location_facility: University of Iowa Hospitals & Clinics; Internal Medicine

location_facility: Temple University Hospital ; Lung Center

location_facility: University of Pittsburgh Medical Center Health System

location_facility: Groupe Hospitalier Sud - Hôpital Haut Lévêque

location_facility: Queen's University Belfast; NICRN Respiratory Research Office


title: A Phase II, Open-label, Single-arm, Exploratory Pharmacogenomic Study of Single Agent Eribulin (HALAVEN®) as Neoadjuvant Treatment for Operable Stage I-II HER2 Non-overexpressing Breast Cancer.

acronym: NeoEribulin

summary: This is a prospective, non-randomized, open-label, multicenter, single-arm exploratory pharmacogenomic study of single agent eribulin as neoadjuvant therapy in patients with operable Stage III HER2 non-overexpressing breast cancer.

ReferenceCitation: Prat P, Llombart A, de la Peña L, Di Cosimo S, Oliveira M, Ortega V, Rubio I, Muñoz E, Harbeck N, Cortés J. NeoEribulin: A Phase II, non-randomized, open-label, single-arm, multicenter, exploratory pharmacogenomic study of single agent eribulin as neoadjuvant treatment for operable Stage I-II HER2 non-overexpressing breast cancer. Poster session presented at: 35th Annual San Antonio Breast Cancer Symposium (SABCS); 2012 December 4th-8th; San Antonio, Texas, United States.

lead_sponsor: SOLTI Breast Cancer Research Group

location_country: Germany France Portugal Spain

location_facility: Instituto Portugues de Oncologia de Porto Francisco Gentil, EPE

location_facility: Hospital Universitario Clínico San Carlos

location_facility: Hospital Universitario Puerta de Hierro de Majadahonda

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Instituto Portugues de Oncologia de Coimbra Francisco Gentil, EPE

location_facility: Klinikum des Landkreises Deggendorf Frauenklinik Mammazentrum

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Brustzentrum im Krankenhaus Köln-Holweide Priv. Doz.

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: Klinikum Südstadt Rostock, Universitätsfrauenklinik und Poliklinik

location_facility: Complejo Hospitalario San Pedro de Alcántara

location_facility: Hospital Universitari Arnau de Vilanova de Lleida


title: European Multicenter Study Using Hybrid Staged Operating Room and Interventional Catheter Ablation Techniques to Treat Chronic AF

acronym: HISTORIC-AF

summary:
Prospective, multi-center, investigator-driven trial. This study hypothesizes that combining surgical endoscopic and transcatheter techniques in a staged fashion provides superior clinical outcomes than isolated surgical/EP approaches in patients with persistent AF lasting > 1 year but > 5 years.

The proposed procedure involves the creation of cardiac lesions with epicardially applied radiofrequency (RF) ablation through a minimally invasive surgical (MIS) approach followed by a delayed EP ablation procedure performed at 1-2 months from the surgical operation.

status: Unknown status

study_completion_date_type: Anticipated

time_perspective: Prospective

enrollment_type: Anticipated

ReferenceCitation: Bisleri G, Curnis A, Bottio T, Mascioli G, Muneretto C. The need of a hybrid approach for the treatment of atrial fibrillation. Heart Surg Forum. 2005;8(5):E326-30. Review.

ReferenceCitation: Muneretto C, Bisleri G, Rosati F, Krakor R, Giroletti L, Di Bacco L, Repossini A, Moltrasio M, Curnis A, Tondo C, Polvani G. European prospective multicentre study of hybrid thoracoscopic and transcatheter ablation of persistent atrial fibrillation: the HISTORIC-AF trial. Eur J Cardiothorac Surg. 2017 Oct 1;52(4):740-745. doi: 10.1093/ejcts/ezx162.

lead_sponsor: Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia

location_country:
Poland
United Kingdom
Italy
France
Germany

location_facility:
Hammersmith Hospital
Stadtische Kliniken
University Hospital
Royal Brompton
Univ.Hosp. Molinette
Ospedale Gavazzeni
Louis Pradel Hospital
Hamburg Uke
Heart Center Brandenburg- Immanuel
Univ. Hosp. Spedali Civili


title: Medtronic PERIcardial SurGical AOrtic Valve ReplacemeNt Pivotal Trial A Multi-center, Non-randomized Trial to Determine the Safety and Effectiveness of the Model 400 Aortic Valve Bioprosthesis in Patients With Aortic Valve Disease

acronym: PERIGON

summary: To evaluate the safety and effectiveness of the Model 400 aortic valve bioprosthesis.

status: Recruiting

ReferenceCitation: Klautz RJM, Kappetein AP, Lange R, Dagenais F, Labrousse L, Bapat V, Moront M, Misfeld M, Zeng C, Sabik Iii JF; PERIGON Investigators. Safety, effectiveness and haemodynamic performance of a new stented aortic valve bioprosthesis. Eur J Cardiothorac Surg. 2017 Sep 1;52(3):425-431. doi: 10.1093/ejcts/ezx066.

lead_sponsor: Medtronic Cardiac Surgery

location_country:
United Kingdom
Italy
Netherlands
Canada
Switzerland
United States
France
Germany

location_facility: Universitäts Klinikum Frankfurt - Goethe-Universität

location_facility: University of Southern California (USC) University Hospital

location_facility: Leids Universitair Medisch Centrum (LUMC)

location_facility: London Health Sciences Centre - University Campus

location_facility: Guy's & St Thomas' NHS Foundation Trust - St Thomas' Hospital

location_facility: Riverside Methodist Hospital (OhioHealth)

location_facility: University of Michigan Cardiovascular Center

location_facility: Cardiothoracic and Vascular Surgeons (CTVS)

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Québec (IUCPQ)

location_facility: New York-Presbyterian Hospital/Columbia University Medical Center

intervention_type: Device

status_simplified: Ongoing


title: Efficacy of Oral Alitretinoin Treatment in Patients With Palmo-plantar Pustulosis (PPP) Inadequately Responding to Standard Topical Treatment

summary: The main purpose of this study is to demonstrate the improvement in the skin condition rate of patients receiving alitretinoin compared to patients receiving placebo.

ipd_sharing_description: Patient-level data for this study will be made available through www.clinicalstudydatarequest.com following the timelines and process described on this site.

lead_sponsor: Stiefel, a GSK Company

location_country:
Germany
France
Netherlands
United Kingdom


title: A Phase III, Multicentre, Double Blinded Study In Patients With Chronic Hepatitis C Who Are Non-Responders To Prior Peginterferon Alpha + Ribavirin Therapy Comparing Treatment With Thymosin Alpha 1 + Peginterferon Alpha-2a Plus Ribavirin With Peginterferon Alpha-2a + Ribavirin + Placebo

summary: The purpose of the study was to determine safety and efficacy of 48 weeks treatment with Thymosin alpha 1 (Talpha1) in combination with pegylated interferon (PEGIFN) alpha2a and ribavirin (RBV) in adult patients with chronic hepatitis C (CHC) already treated with, and not responding to previous courses of PEGIFN alpha plus RBV combination therapy, in comparison with a concurrent group treated with PEG IFN alpha2a in combination with RBV and placebo.

ReferenceCitation: Ciancio A, Andreone P, Kaiser S, Mangia A, Milella M, Solà R, Pol S, Tsianos E, De Rosa A, Camerini R, McBeath R, Rizzetto M. Thymosin alpha-1 with peginterferon alfa-2a/ribavirin for chronic hepatitis C not responsive to IFN/ribavirin: an adjuvant role? J Viral Hepat. 2012 Jan;19 Suppl 1:52-9. doi: 10.1111/j.1365-2893.2011.01524.x.

lead_sponsor: sigma-tau i.f.r. S.p.A.

location_country: Spain Italy Greece France Germany

location_facility: Dipartimento Medico Chirurgico delle Malattie dell'Apparato Digerente e della Nutrizione, Azienda Ospedaliera S Giovanni Battista

intervention_type: Biological


status: Active, not recruiting

title: A Phase I, Multicenter, Open-label Study of Oral ABL001 in Patients With Chronic Myelogenous Leukemia (CML) or Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL)

lead_sponsor: Novartis Pharmaceuticals

design_allocation: Non-Randomized

ReferenceCitation: Li YF, Combes FP, Hoch M, Lorenzo S, Sy SKB, Ho YY. Population Pharmacokinetics of Asciminib in Tyrosine Kinase Inhibitor-Treated Patients with Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic and Acute Phases. Clin Pharmacokinet. 2022 Oct;61(10):1393-1403. doi: 10.1007/s40262-022-01148-9. Epub 2022 Jun 28. Erratum in: Clin Pharmacokinet. 2022 Aug 17;:.

ReferenceCitation: Hughes TP, Mauro MJ, Cortes JE, Minami H, Rea D, DeAngelo DJ, Breccia M, Goh YT, Talpaz M, Hochhaus A, le Coutre P, Ottmann O, Heinrich MC, Steegmann JL, Deininger MWN, Janssen JJWM, Mahon FX, Minami Y, Yeung D, Ross DM, Tallman MS, Park JH, Druker BJ, Hynds D, Duan Y, Meille C, Hourcade-Potelleret F, Vanasse KG, Lang F, Kim DW. Asciminib in Chronic Myeloid Leukemia after ABL Kinase Inhibitor Failure. N Engl J Med. 2019 Dec 12;381(24):2315-2326. doi: 10.1056/NEJMoa1902328.

location_country:
Spain
Italy
Australia
Netherlands
Korea, Republic of
United States
France
Singapore
Japan
Germany

location_facility: Oregon Health and Science University SC-6

location_facility: Dana Farber Cancer Institute Hematology / Oncology

location_facility: Memorial Sloan Kettering Cancer Center Memorial Sloan Kettering

location_facility: University of Michigan Comprehensive Cancer Center SC

location_facility: University of Texas/MD Anderson Cancer Center UT MD Anderson

summary: The design of a phase I, open label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent ABL001 in CML and Ph+ ALL patients who are relapsed or refractory to or are intolerant of TKIs, and of ABL001+Nilotinib, ABL001+Imatinib and ABL001+Dasatinib in Ph positive CML patients who are relapsed or refractory to TKIs.


title: An Open-label, Multi-center, Extension Study to Evaluate the Long-term Safety of Subcutaneous 240mg QGE031 Given Every 4 Weeks for 52 Weeks in Allergic Asthma Patients Who Completed Study CQGE031B2201

summary: This study planned to assess long-term safety of QGE031 during 12 months treatment in asthma patients who completed study CQGE031B2201.

location_country:
Poland
Portugal
Romania
United Kingdom
Italy
Russian Federation
Finland
Argentina
Canada
Hungary
Korea, Republic of
United States
France
Israel
South Africa
Mexico
Turkey
Germany
Slovakia
Czechia


title: Crossover From IDE Trial CLN0009, Lung Volume Reduction Coil Treatment in Patients With Emphysema (RENEW) Study, IDE G110066

acronym: CROSSOVER

summary: Crossover study for patients who were randomized to the Control Group in CLN0009 (NCT01608490).

lead_sponsor: Boston Scientific Corporation

location_country:
United Kingdom
Canada
United States
France
Netherlands

location_facility: Yale University School of Medicine - Yale New Haven Hospital

location_facility: University of Wisconsin School of Medicine & Public Health

location_facility: Emphysema COPD Research Center, University of Pittsburgh

location_facility: Pulmonary, Critical Care & Sleep Medicine Consultants,PCCS/St. Luke's Episcopal Hospital

location_facility: Centre Hospitalier Universitaire de Nice

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Quebec (IUCPQ)

location_facility: University of Alabama Lung Health Center

location_facility: University of Texas Health Sciences Center at San Antonio

location_facility: Royal Brompton Hospital & Chelsea Westminster

location_facility: New York Presbyterian Columbia University Medical Center

location_facility: Illinois Lung and Critical Care Institute

location_facility: El Camino Hospital/Palo Alto Medical Foundation


acronym: Illuminate

title: A Randomized, 6-Week, Double-blind, Placebo-Controlled, Flexible Dose, Parallel-Group Study to Evaluate the Efficacy and Safety of Lurasidone in Children and Adolescent Subjects With Bipolar I Depression

lead_sponsor: Sunovion

ReferenceCitation: DelBello MP, Goldman R, Phillips D, Deng L, Cucchiaro J, Loebel A. Efficacy and Safety of Lurasidone in Children and Adolescents With Bipolar I Depression: A Double-Blind, Placebo-Controlled Study. J Am Acad Child Adolesc Psychiatry. 2017 Dec;56(12):1015-1025. doi: 10.1016/j.jaac.2017.10.006. Epub 2017 Oct 13.

location_country:
Poland
Mexico
Ukraine
Hungary
Korea, Republic of
United States
France
Puerto Rico
Colombia
Philippines
Bulgaria

location_facility: Wojewodzki Szpital Zespolony im. L. Rydygiera w Toruniu

location_facility: Consultorio Especializado Psiquiatría Infantil y Adolescentes

location_facility: CI Kherson Regional Psychiatric Hospital of Kherson RC

location_facility: Severance Hospital, Yonsei University Health System

location_facility: CI Odesa Regional Medical Center of Mental Health

location_facility: West Visayas State University Medical Center

location_facility: Centro para el Desarrollo de la Medicina y de Asistencia Medica Especializada S.C.

location_facility: Family Psychiatry of The Woodlands, P.A.

location_facility: CI Lviv Regional Clinical Psychiatric Hospital

location_facility: SI Institute of Children and Adolescents Healthcare of NAMSU

location_facility: SI Institute of Neurology, Psychiatry and Narcology of NAMSU

location_facility: Hôpitaux Pédiatriques de Nice CHU-Lenval

location_facility: Centro de Investigaciones y Proyectos en Neurociencias CIPNA

location_facility: Ternopil RCCPH Dept of Psychiatry #9 (adolescent)& #8 (pediatric) Ternopil I.Ya. Gorbachevskyi SMU

location_facility: Chair of Psychiatry and Narcology, Vinnytsia National Medical University, O.I. Yushchenko Regional Psychoneurological Hospital

location_facility: Centro de Investigaciones del Sistema Nervioso Limitada - Grupo CISNE Ltda

location_facility: Instituto de Informacion de Investigacion en Salud Mental

location_facility: Instituto de Investigaciones Aplicadas a la Neurociencia A.C.

location_facility: O.F. Maltcev Poltava RCPsH Children Dept Ukrainian Medical Stomatological Academy

location_facility: University Hospitals Case Medical Center

location_facility: RPsH #3 Сhildren Dept SHEI Ivano-Frankivsk SMU

location_facility: Seoul National University Bundang Hospital

location_facility: Vadaskert Alapitvany a Gyermekek Lelki Egeszsegeert

summary: A study to evaluate efficacy and safety of flexibly dosed Lurasidone in children and adolescents with bipolar I depression


title: Influence of Preoperative Hemodialysis or Intraoperative Modified Ultrafiltration on Postoperative Outcome for Patients With Severe Renal Dysfunction Undergoing Open Heart Surgery: Randomized, Controlled, Multicenter Clinical Trial

acronym: SeRenaD-CPB

summary: The purpose of this study is to determine whether preoperative hemodialysis or intraoperative modified ultrafiltration are effective for patients with non-dialysis dependent severe renal dysfunction undergoing open heart surgery.

ReferenceCitation: Foley RN, Parfrey PS, Sarnak MJ. Epidemiology of cardiovascular disease in chronic renal disease. J Am Soc Nephrol. 1998 Dec;9(12 Suppl):S16-23. Review.

ReferenceCitation: Schiffrin EL, Lipman ML, Mann JF. Chronic kidney disease: effects on the cardiovascular system. Circulation. 2007 Jul 3;116(1):85-97. Review.

ReferenceCitation: Levin A, Foley RN. Cardiovascular disease in chronic renal insufficiency. Am J Kidney Dis. 2000 Dec;36(6 Suppl 3):S24-30. Review.

ReferenceCitation: Anderson RJ, O'brien M, MaWhinney S, VillaNueva CB, Moritz TE, Sethi GK, Henderson WG, Hammermeister KE, Grover FL, Shroyer AL. Renal failure predisposes patients to adverse outcome after coronary artery bypass surgery. VA Cooperative Study #5. Kidney Int. 1999 Mar;55(3):1057-62.

ReferenceCitation: Cooper WA, O'Brien SM, Thourani VH, Guyton RA, Bridges CR, Szczech LA, Petersen R, Peterson ED. Impact of renal dysfunction on outcomes of coronary artery bypass surgery: results from the Society of Thoracic Surgeons National Adult Cardiac Database. Circulation. 2006 Feb 28;113(8):1063-70. Epub 2006 Feb 20.

ReferenceCitation: Zakeri R, Freemantle N, Barnett V, Lipkin GW, Bonser RS, Graham TR, Rooney SJ, Wilson IC, Cramb R, Keogh BE, Pagano D. Relation between mild renal dysfunction and outcomes after coronary artery bypass grafting. Circulation. 2005 Aug 30;112(9 Suppl):I270-5.

ReferenceCitation: Weerasinghe A, Hornick P, Smith P, Taylor K, Ratnatunga C. Coronary artery bypass grafting in non-dialysis-dependent mild-to-moderate renal dysfunction. J Thorac Cardiovasc Surg. 2001 Jun;121(6):1083-9.

ReferenceCitation: Ibáñez J, Riera M, Saez de Ibarra JI, Carrillo A, Fernández R, Herrero J, Fiol M, Bonnin O. Effect of preoperative mild renal dysfunction on mortality and morbidity following valve cardiac surgery. Interact Cardiovasc Thorac Surg. 2007 Dec;6(6):748-52. Epub 2007 Sep 21.

ReferenceCitation: Filsoufi F, Rahmanian PB, Castillo JG, Chikwe J, Carpentier A, Adams DH. Early and late outcomes of cardiac surgery in patients with moderate to severe preoperative renal dysfunction without dialysis. Interact Cardiovasc Thorac Surg. 2008 Feb;7(1):90-5. Epub 2007 Nov 22.

ReferenceCitation: Devbhandari MP, Duncan AJ, Grayson AD, Fabri BM, Keenan DJ, Bridgewater B, Jones MT, Au J; North West Quality Improvement Programme in Cardiac Interventions. Effect of risk-adjusted, non-dialysis-dependent renal dysfunction on mortality and morbidity following coronary artery bypass surgery: a multi-centre study. Eur J Cardiothorac Surg. 2006 Jun;29(6):964-70. Epub 2006 May 3.

ReferenceCitation: Kilo J, Margreiter JE, Ruttmann E, Laufer G, Bonatti JO. Slightly elevated serum creatinine predicts renal failure requiring hemofiltration after cardiac surgery. Heart Surg Forum. 2005;8(1):E34-8.

ReferenceCitation: Mangano CM, Diamondstone LS, Ramsay JG, Aggarwal A, Herskowitz A, Mangano DT. Renal dysfunction after myocardial revascularization: risk factors, adverse outcomes, and hospital resource utilization. The Multicenter Study of Perioperative Ischemia Research Group. Ann Intern Med. 1998 Feb 1;128(3):194-203.

ReferenceCitation: Shen I, Giacomuzzi C, Ungerleider RM. Current strategies for optimizing the use of cardiopulmonary bypass in neonates and infants. Ann Thorac Surg. 2003 Feb;75(2):S729-34.

ReferenceCitation: Naik SK, Knight A, Elliott MJ. A successful modification of ultrafiltration for cardiopulmonary bypass in children. Perfusion. 1991;6(1):41-50.

ReferenceCitation: Naik SK, Knight A, Elliott M. A prospective randomized study of a modified technique of ultrafiltration during pediatric open-heart surgery. Circulation. 1991 Nov;84(5 Suppl):III422-31.

ReferenceCitation: Luciani GB, Menon T, Vecchi B, Auriemma S, Mazzucco A. Modified ultrafiltration reduces morbidity after adult cardiac operations: a prospective, randomized clinical trial. Circulation. 2001 Sep 18;104(12 Suppl 1):I253-9.

ReferenceCitation: Boodhwani M, Williams K, Babaev A, Gill G, Saleem N, Rubens FD. Ultrafiltration reduces blood transfusions following cardiac surgery: A meta-analysis. Eur J Cardiothorac Surg. 2006 Dec;30(6):892-7. Epub 2006 Oct 13. Review.

ReferenceCitation: Bogă M, Islamoğlu, Badak I, Cikirikçioğlu M, Bakalim T, Yağdi T, Büket S, Hamulu A. The effects of modified hemofiltration on inflammatory mediators and cardiac performance in coronary artery bypass grafting. Perfusion. 2000 Mar;15(2):143-50.

ReferenceCitation: Capuano F, Bianchini R, Goracci M, Roscitano A, Luciani R, Simon C, Giusti L, Sinatra R. Intraoperative veno-arterial hemofiltration during miniaturized extracorporeal bypass. Ann Thorac Surg. 2007 Jun;83(6):2215-6.

ReferenceCitation: Durmaz I, Yagdi T, Calkavur T, Mahmudov R, Apaydin AZ, Posacioglu H, Atay Y, Engin C. Prophylactic dialysis in patients with renal dysfunction undergoing on-pump coronary artery bypass surgery. Ann Thorac Surg. 2003 Mar;75(3):859-64.

ReferenceCitation: Bingol H, Akay HT, Iyem H, Bolcal C, Oz K, Sirin G, Demirkilic U, Tatar H. Prophylactic dialysis in elderly patients undergoing coronary bypass surgery. Ther Apher Dial. 2007 Feb;11(1):30-5.

ReferenceCitation: Moher D, Schulz KF, Altman DG. The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomised trials. Lancet. 2001 Apr 14;357(9263):1191-4.

lead_sponsor: University Hospital, Geneva

location_country: Spain Switzerland France Turkey Germany

location_facility: University of Lyon, Hopital Cardiothoracique Louis Pradel

location_facility: Ankara University, Department of Cardiovascular Surgery

location_facility: University Hospital of Geneva, Service for Cardiovascular Surgery

location_facility: Hospital Clinico, University of Barcelona, Department of Cardiovascular Surgery

location_facility: Gaziantep University, Department of Cardiovascular Surgery

location_facility: Pamukkale University, Department of Cardiovascular Surgery

intervention_type: Procedure


type: Registry Identifier

title: A Phase 4 Randomised, Double-Blind, Placebo-Controlled, Parallel-Group, MultiCentre Study of Colesevelam as Add-on Therapy in Patients With Familial Hypercholesterolaemia

summary: This study is designed to assess whether colesevelam given as third line treatment added to a maximal tolerated and stable dose of a statin and ezetimibe is able to further decrease the level of LDL cholesterol in a safe and efficient manner in difficult to treat Familial Hypercholesterolaemia patients who are not at their target level of LDL cholesterol.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
United Kingdom
Netherlands
Sweden
France
Germany

location_facility: Department of Medicine, University of Manchester

location_facility: Service d'Endocrinologie, Metabolisme Prevention des Maladies Cardio-Vasculaires


title: A Multicentre Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency

lead_sponsor: CSL Behring

ReferenceCitation: Jolles S, Bernatowska E, de Gracia J, Borte M, Cristea V, Peter HH, Belohradsky BH, Wahn V, Neufang-Hüber J, Zenker O, Grimbacher B. Efficacy and safety of Hizentra(®) in patients with primary immunodeficiency after a dose-equivalent switch from intravenous or subcutaneous replacement therapy. Clin Immunol. 2011 Oct;141(1):90-102. doi: 10.1016/j.clim.2011.06.002. Epub 2011 Jun 12.

ReferenceCitation: Borte M, Pac M, Serban M, Gonzalez-Quevedo T, Grimbacher B, Jolles S, Zenker O, Neufang-Hueber J, Belohradsky B. Efficacy and safety of hizentra®, a new 20% immunoglobulin preparation for subcutaneous administration, in pediatric patients with primary immunodeficiency. J Clin Immunol. 2011 Oct;31(5):752-61. doi: 10.1007/s10875-011-9557-z. Epub 2011 Jun 15.

location_country:
Poland
Spain
Italy
United Kingdom
Switzerland
Sweden
France
Romania
Germany

summary: The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).


acronym: RISE

title: A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome

location_facility:
UMC St Radboud Centraal
Hopital Avicenne/University
Royal Bournemouth Hospital
Medizinische Hochschule Hannover
St. Johannes-Hospital Duisburg
St. James Hospital
King's College Hospital

summary: This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who have never been treated for MDS (i.e., treatment-naïve patients).


title: An Open-Label, Phase II Dose Titration Study of ACZ885 (Human Anti-IL-1β Monoclonal Antibody) to Assess the Clinical Efficacy, Safety, Pharmacokinetics and Pharmacodynamics in Patients With NALP3 Mutations

ReferenceCitation: Kuemmerle-Deschner JB, Ramos E, Blank N, Roesler J, Felix SD, Jung T, Stricker K, Chakraborty A, Tannenbaum S, Wright AM, Rordorf C. Canakinumab (ACZ885, a fully human IgG1 anti-IL-1β mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS). Arthritis Res Ther. 2011 Feb 28;13(1):R34. doi: 10.1186/ar3266.

location_country:
United Kingdom
Spain
France
India
Germany

location_facility:
Novartis Investigative site
Novartis Investigator Site
Novartis investigative site

summary: This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are either untreated or insufficiently treated and require medical intervention.


title: A Multicenter, Open, Single Sequence Crossover Study to Assess the Safety and Efficacy of a Tacrolimus Modified Release, FK506E (MR4), Based Immunosuppressive Regimen in Stable Kidney Transplant Patients Converted From a Prograf® Based Immunosuppressive Regimen

summary: Assessment of the safety and the efficacy of a tacrolimus modified release (MR4) based immunosuppressive regimen in stable kidney transplant subjects converted on a 1:1 (mg:mg) basis from a Prograf® based immunosuppressive regimen.

lead_sponsor: Astellas Pharma Inc

location_country:
Poland
Spain
United Kingdom
Netherlands
France
Germany


title: Phase II Study of the Combination of Cisplatin + Temozolomide in Malignant Glial Tumours in Children and Adolescents at Diagnosis or in Relapse

summary:
RATIONALE: Drugs used in chemotherapy, such as cisplatin and temozolomide, 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.

PURPOSE: This phase II trial is studying how well giving cisplatin together with temozolomide works in treating young patients with malignant glioma.

lead_sponsor: Children's Cancer and Leukaemia Group

location_country:
France
United Kingdom
Ireland

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Central Manchester and Manchester Children's University Hospitals NHS Trust

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Marsden NHS Foundation Trust - Surrey

location_facility: Sir James Spence Institute of Child Health

location_facility: Southampton University Hospital NHS Trust

location_facility: Great Ormond Street Hospital for Children NHS Trust

location_facility: Addenbrooke's Hospital at Cambridge University Hospitals NHS Foundation Trust

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

location_facility: Royal Belfast Hospital for Sick Children

intervention_type: Genetic


title: An Open International Multicentre Long-Term Follow Up Study to Evaluate Safety of Prolonged Release OROS Methlyphenidate in Adults With Attention Deficit Hyperactivity Disorder

summary:
Trial 42603ATT3004 is an open-label extension study to clinical trial 42603ATT3002 (NCT00246220). In trial 42603ATT3002 the efficacy and safety of OROS methylphenidate was assessed in adult subjects with Attention Deficit Hyperactivity Disease (ADHD). ADHD is a developmental disorder beginning in childhood and characterized by developmentally inappropriate inattention, hyperactivity and impulsiveness. Data on the number of adult patients with ADHD is limited, but it is estimated that approximately 50% of children with ADHD will have symptoms also in adhulthood. The drug tested in this trial is OROS methylphenidate. The active ingredient is methylphenidate and the tablet is designed to release the active ingredient gradually to ensure an effect, which lasts up to 12 hours. Trial 42603ATT3002 consisted of a 5-week period, where subjects were assigned to either receive placebo (empty drug) or one out of three different dosages of OROS methylphenidate. This 5-week period was followed by a 7-week period, where patients received OROS methylphenidate at their optimal dose. In study 42603ATT3004, subjects who complete 42603ATT3002 are followed for a period of at least 52 weeks to evaluate safety and tolerability of OROS methylphenidate in patients who are treated with OROS methylphenidate over a long period of time.

Amendment: At the end of the open-label period of the present study 42603ATT3004, patients are enrolled into a double-blind placebo-controlled period, which lasts an additional 4 weeks. The purpose of this double-blind placebo-controlled period is to evaluate the maintenance of effect under continued treatment with OROS methlyphenidate in comparison to treatment cessation in those patients, who are randomized into the placebo-group.

ReferenceCitation: Boesen K, Paludan-Müller AS, Gøtzsche PC, Jørgensen KJ. Extended-release methylphenidate for attention deficit hyperactivity disorder (ADHD) in adults. Cochrane Database Syst Rev. 2022 Feb 24;2:CD012857. doi: 10.1002/14651858.CD012857.pub2. Review.

ReferenceCitation: Buitelaar JK, Trott GE, Hofecker M, Waechter S, Berwaerts J, Dejonkheere J, Schäuble B. Long-term efficacy and safety outcomes with OROS-MPH in adults with ADHD. Int J Neuropsychopharmacol. 2012 Feb;15(1):1-13. doi: 10.1017/S1461145711001131. Epub 2011 Jul 29.

lead_sponsor: Janssen-Cilag International NV

location_country: Portugal Spain Netherlands Switzerland France Norway Germany


title: A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease

summary: The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country: Spain Italy Netherlands Switzerland Sweden France Turkey Germany

location_facility:
University Regensburg
Karolinska University Hospital
Hospital Clínico Universitario
University Hospital
Ospedale San Martino
Hopital St. Louis
University Faculty of Medicine


title: Phase II Study of Temozolomide (Temodal) in Children Over 1 Year of Age With Relapsed or Refractory High Risk Neuroblastoma

summary:
RATIONALE: Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying how well temozolomide works in treating patients with high-risk relapsed or refractory neuroblastoma.

ReferenceCitation: Rubie H, Chisholm J, Defachelles AS, Morland B, Munzer C, Valteau-Couanet D, Mosseri V, Bergeron C, Weston C, Coze C, Auvrignon A, Djafari L, Hobson R, Baunin C, Dickinson F, Brisse H, McHugh K, Biassoni L, Giammarile F, Vassal G; Société Françaisedes Cancers de l'Enfant; United Kingdom Children Cancer Study Group-New Agents Group Study. Phase II study of temozolomide in relapsed or refractory high-risk neuroblastoma: a joint Société Française des Cancers de l'Enfant and United Kingdom Children Cancer Study Group-New Agents Group Study. J Clin Oncol. 2006 Nov 20;24(33):5259-64.


title: Observational Registry: The Effectiveness of Pain Management Using the ARCHIMEDES® Implantable Constant-Flow Infusion Pump System for Intrathecal Delivery

summary: The purpose of this observational registry is to collect a continuum of meaningful clinical data on the ARCHIMEDES implantable pump in pain management

lead_sponsor: Codman & Shurtleff

location_country:
Germany
France
United Kingdom


title: A Randomized, Parallel Group, Open-label, Multicenter Study to Investigate the Efficacy and Safety of Oral BAY85-3934 and Active Comparator (Darbepoetin Alfa) in the Maintenance Treatment of Anemia in Pre-dialysis Subjects With Chronic Kidney Disease on Darbepoetin Treatment in Europe and Asia Pacific

acronym: DIALOGUE 2

summary:
Anaemia is a condition in which blood has a lower than normal number of red blood cells. It can also occur if red blood cells do not contain enough haemoglobin, an oxygen carrying part of blood. Anaemia is common in patients with chronic kidney disease. Healthy kidneys produce a hormone called erythropoietin, which stimulates the bone marrow to produce the proper number of red blood cells needed to carry oxygen to vital organs. Chronic kidney disease is a general term that means that the kidneys are not functioning to their full potential. The study drug, BAY85-3934, is being evaluated as a drug to increase the body's ability to produce erythropoietin.

The purpose of this study is to find out if the study drug, a tablet taken orally, is safe and effective for the treatment of anaemia associated with chronic kidney disease.

The study will enroll 120 patients at multiple locations in Europe, Asia and Australia. Participation will involve a screening visit and between 12 and 15 study visits scheduled over a period of approximately 5 to 7 months. The estimated total duration of study treatment will be 16 weeks. During these scheduled visits patients will undergo a number of procedures to confirm efficacy and safety of the study drug, including measurement of heart rate and blood pressure, physical examination, Electrocardiogram and blood/urine sample collection for laboratory tests.

The study will be conducted at 3 hospitals in the UK. Bayer HealthCare AG is funding this research.

ReferenceCitation: Natale P, Palmer SC, Jaure A, Hodson EM, Ruospo M, Cooper TE, Hahn D, Saglimbene VM, Craig JC, Strippoli GF. Hypoxia-inducible factor stabilisers for the anaemia of chronic kidney disease. Cochrane Database Syst Rev. 2022 Aug 25;8:CD013751. doi: 10.1002/14651858.CD013751.pub2. Review.

lead_sponsor: Bayer

location_country:
Poland
Spain
Italy
United Kingdom
Australia
Hungary
Korea, Republic of
France
Israel
Turkey
Romania
Japan
Germany
Bulgaria

location_facility:
Ankara Univ. Medical Faculty
Sifa University Medical Faculty
Baskent University Medical Faculty


title: MMT 98 Study For Metastatic Disease Rhabdomyosarcoma And Other Malignant Soft Tissue Sarcoma Of Childhood

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy in treating children with metastatic rhabdomyosarcoma or other malignant mesenchymal tumors.

ReferenceCitation: McDowell HP, Foot AB, Ellershaw C, Machin D, Giraud C, Bergeron C. Outcomes in paediatric metastatic rhabdomyosarcoma: results of The International Society of Paediatric Oncology (SIOP) study MMT-98. Eur J Cancer. 2010 Jun;46(9):1588-95. doi: 10.1016/j.ejca.2010.02.051. Epub 2010 Mar 24.

lead_sponsor: Societe Internationale d'Oncologie Pediatrique

location_facility: Meyerstein Institute of Oncology at University College of London Hospitals


title: An International Field Study Of The Reliability And Validity Of The EORTC QLQ-C30 And A Disease-Specific Questionnaire Module (The QLQ-STO22) In Assessing The Quality Of Life Of Patients With Gastric Cancer

summary:
RATIONALE: Assessing quality of life in patients who are undergoing cancer treatment may help determine the effect of treatment on these patients.

PURPOSE: Clinical trial to study the effectiveness of a quality of life assessment in patients who are receiving treatment for stomach cancer.

ReferenceCitation: Oñate-Ocaña LF, Alcántara-Pilar A, Vilar-Compte D, García-Hubard G, Rojas-Castillo E, Alvarado-Aguilar S, Carrillo JF, Blazeby JM, Aiello-Crocifoglio V. Validation of the Mexican Spanish version of the EORTC C30 and STO22 questionnaires for the evaluation of health-related quality of life in patients with gastric cancer. Ann Surg Oncol. 2009 Jan;16(1):88-95. doi: 10.1245/s10434-008-0175-9. Epub 2008 Nov 1.

ReferenceCitation: Sadighi S, Montazeri A, Sedighi Z, Mohagheghi MA, Froutan H. Quality of life in patients with gastric cancer: translation and psychometric evaluation of the Iranian version of EORTC QLQ-STO22. BMC Cancer. 2009 Aug 28;9:305. doi: 10.1186/1471-2407-9-305.

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

location_country:
United Kingdom
Spain
Sweden
France
Germany

location_facility:
Centre Alexis Vautrin
Royal Infirmary
Sahlgrenska University Hospital
Bristol Haematology and Oncology Centre
Universitaetsklinikum Charite
Hospital De Navarra
University of Marburg


title: The Observational Clinical Registry of the European Reference Network on Rare Adult Solid Cancers: the Protocol for the Rare Head and Neck Cancers

acronym: EURACAN

summary:
Cancer care for head and neck cancers is multidisciplinary and complex and knowledge on the rare ones is limited. There is a wide consensus that to support clinical research on rare cancers, clinical registries should be developed within networks specializing in rare cancers. Our hypothesis is that our head and neck cancer registry established in the framework of the European reference network on rare adults solid cancers will help to: describe the natural history of rare head and neck cancers; evaluate factors that influence prognosis; assess treatment effectiveness; measure indicators of quality of care.

The registry is a prospective observational real-world registry. It collects data from already available registries/database and/or directly from expert health care providers (HCP). Information are prospectively collected on patient characteristics; exposure, outcomes and potential confounders (https://euracan.eu/research/starter/rare-head-and-neck-cancer-registry/#codebook).

The registry if federated (i.e. data are stored by the data provider). Analyses will be performed using the federated learning approach which split computations into a local part and a central part. The data providers will share sub-computations only. Data quality checks are envisioned to assess whether data value are present, valid and believable. Validity and plausibility checks are embedded in the electronic case report form (CRF) in the form of alerts and errors during the data input. Additional checks are implemented in R and run using the federated learning to ensure a central data quality monitoring. The data analyses will include descriptive statistics showing frequency and patterns of patients' and cancers' variables; analytical analyses investigating the association of patients/disease and/or treatment characteristics and health outcomes.

Fondazione IRCCS Istituto Nazionale dei Tumori (INT) is the coordinator of the EURACAN registry as well as a data provider. At the INT, and at each HCP involved, responsible investigators ensure that the EURACAN registry will be implemented in compliance with the protocol, following the instructions and procedures described herein. Each HCP is a controller and will identify a data processor. The processing of patients' personal data taking part in the registry is compliant with local privacy legislation and the General Data Protection Regulation 2016/679 of the EU.

ReferenceCitation: Gatta G, Capocaccia R, Botta L, Mallone S, De Angelis R, Ardanaz E, Comber H, Dimitrova N, Leinonen MK, Siesling S, van der Zwan JM, Van Eycken L, Visser O, Žakelj MP, Anderson LA, Bella F, Kaire I, Otter R, Stiller CA, Trama A; RARECAREnet working group. Burden and centralised treatment in Europe of rare tumours: results of RARECAREnet-a population-based study. Lancet Oncol. 2017 Aug;18(8):1022-1039. doi: 10.1016/S1470-2045(17)30445-X. Epub 2017 Jul 4. Erratum in: Lancet Oncol. 2017 Aug;18(8):e433.

ReferenceCitation: Gatta G, Trama A, Capocaccia R; RARECARENet Working Group. Epidemiology of rare cancers and inequalities in oncologic outcomes. Eur J Surg Oncol. 2019 Jan;45(1):3-11. doi: 10.1016/j.ejso.2017.08.018. Epub 2017 Sep 19.

ReferenceCitation: Siesling S, Louwman WJ, Kwast A, van den Hurk C, O'Callaghan M, Rosso S, Zanetti R, Storm H, Comber H, Steliarova-Foucher E, Coebergh JW. Uses of cancer registries for public health and clinical research in Europe: Results of the European Network of Cancer Registries survey among 161 population-based cancer registries during 2010-2012. Eur J Cancer. 2015 Jun;51(9):1039-49. doi: 10.1016/j.ejca.2014.07.016. Epub 2014 Aug 15.

ReferenceCitation: Trama A, Botta L, Foschi R, Visser O, Borras JM, Žagar T, Primic-Žakelj M, Bella F, Dimitrova N, Gatta G, Licitra L. Quality of Care Indicators for Head and Neck Cancers: The Experience of the European Project RARECAREnet. Front Oncol. 2019 Aug 28;9:837. doi: 10.3389/fonc.2019.00837. eCollection 2019.

ReferenceCitation: Orlandi E, Alfieri S, Simon C, Trama A, Licitra L; RARECAREnet Working Group. Treatment challenges in and outside a network setting: Head and neck cancers. Eur J Surg Oncol. 2019 Jan;45(1):40-45. doi: 10.1016/j.ejso.2018.02.007. Epub 2018 Feb 14. Review.

ReferenceCitation: Gliklich RE, Dreyer NA, Leavy MB, editors. Registries for Evaluating Patient Outcomes: A User's Guide [Internet]. 3rd edition. Rockville (MD): Agency for Healthcare Research and Quality (US); 2014 Apr. Available from http://www.ncbi.nlm.nih.gov/books/NBK208616/

ipd_sharing_description: The governance (link https://euracan.eu/research/starter/european-registry-governance/)set the data access rules to the federated registry

lead_sponsor: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

location_country: Spain Italy France Germany Czechia

location_facility: Assistance Publique-Hôpitaux de Paris - Hôpital Tenon

location_facility: Centro Nazionale di Adroterapia Oncologica


title: International Wilson's Disease Patient Registry (iWilson Registry)

summary: Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.

lead_sponsor: Orphalan

location_country:
Poland
Spain
United Kingdom
France
Belgium
Germany

location_facility: University Hospital Clínic de Barcelona, C. de Villarroel

location_facility: The Children's Memorial Health Institute

location_facility: Hospital Universitario Y Politécnico La Fe


title: REmote iSchemic condItioning in Lymphoma PatIents REceiving ANthraCyclinEs

acronym: RESILIENCE

summary: Multinational, prospective, proof of concept phase II, double-blinded, sham-controlled, randomized clinical trial (RCT) to evaluate the efficacy and safety of Remote Ischaemic PreConditioning (RIPC) in Non-Hodgkin lymphoma (NHL) patients receiving anthracyclines.

primary_purpose: Prevention

lead_sponsor: Fundación Centro Nacional de Investigaciones Cardiovasculares Carlos III

location_country: Denmark Portugal Spain Netherlands France Germany

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Hospital Clinico Universitario de Valladolid

location_facility: Centro Nacional de Investigaciones Cardiovasculares (CNIC)

location_facility: Hospital da Luz Learning Health (GLSMED)

location_facility: Hospital Universitario Clínico San Carlos


title: A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency

acronym: SPARTA

summary: This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase, Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.

ReferenceCitation: Sorrells S, Camprubi S, Griffin R, Chen J, Ayguasanosa J. SPARTA clinical trial design: exploring the efficacy and safety of two dose regimens of alpha1-proteinase inhibitor augmentation therapy in alpha1-antitrypsin deficiency. Respir Med. 2015 Apr;109(4):490-9. doi: 10.1016/j.rmed.2015.01.022. Epub 2015 Feb 13.

lead_sponsor: Grifols Therapeutics LLC

location_country:
Denmark
Poland
Brazil
Spain
Russian Federation
Australia
Estonia
Argentina
Canada
Finland
New Zealand
Sweden
United States
France
Romania
Germany

location_facility: Washington University School of Medicine

location_facility: University of Miami Miller School of Medicine

location_facility: Hôpital Cardio-Vasculaire et Pneumologique Louis Pradel

location_facility: Queen Elizabeth II Health Sciences Centre

location_facility: New Zealand Respiratory and Sleep institute

location_facility: University of Texas Health Center at Tyler

location_facility: SAIH of Yaroslavl region "Clinical Hospital of Emergency Medical Care n.a. N. V. Solovyev

location_facility: UNIFESP - Universidade Federal de São Paulo

location_facility: Medical Research of Central Florida, LLC

location_facility: FSI Scientific Research Institute of Pulmonology

location_facility: Groupe Hospitalier Sud - Hôpital Haut-Lévêque

location_facility: Universitaetsklinikum Giessen und Marburg GmbH

location_facility: Hospital Universitario Central de Asturias

location_facility: Instytut Gruzlicy i Chorob Pluc w Warszawie

location_facility: St. Joseph's Hospital and Medical Center

location_facility: Penn State Univ. Milton S. Hershey Medical Center

location_facility: Hospital del Torax Dr. Antonio A. Cetrangolo

location_facility: Spitalul Clinic de Boli Infectioase si Pneumoftiziologie

location_facility: Turku University Central Hospital, Department of Pulmonary Diseases

location_facility: SBEI HPE Altai State Medical University of MoH and SD


title: A Phase 3, Randomized, Open-Label Study Evaluating the Efficacy and Safety of Idelalisib in Combination With Obinutuzumab Compared to Chlorambucil in Combination With Obinutuzumab for Previously Untreated Chronic Lymphocytic Leukemia

summary:
The primary objective of this study is to evaluate the effects of idelalisib with obinutuzumab versus the combination of chlorambucil and obinutuzumab on progression-free survival (PFS) in participants with previously untreated chronic lymphocytic leukemia (CLL).

An increased rate of deaths and serious adverse events (SAEs) among participants with front-line CLL and early-line indolent non-Hodgkin lymphoma (iNHL) treated with idelalisib in combination with standard therapies was observed by the independent data monitoring committee (DMC) during regular review of 3 Gilead Phase 3 studies. Gilead reviewed the unblinded data and terminated those studies in agreement with the DMC recommendation and in consultation with the US Food and Drug Administration (FDA). All front-line studies of idelalisib, including this study, were also terminated.

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at http://www.gilead.com/research/disclosure-and-transparency.

lead_sponsor: Gilead Sciences

location_country:
Poland
Spain
United Kingdom
Australia
Canada
United States
France
Belgium

location_facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej Ministerstwa Spraw Wewnetrznych z Warminsko-Mazurskim Centrum Onkologii w Olsztynie Oddzial Hematologii

location_facility: East Kent Hospitals University NHS Foundation Trust

location_facility: Wojewodzki Szpital Specjalistyczny, im. M. Kopernika Klinika Hematologii Uniwersytetu Medycznego

location_facility: Royal Victoria Regional Health Centre - Simcoe Musk

location_facility: Szpital Specjalistyczny w Brzozowie, Oddzial Hematologii Onkologicznej

location_facility: Wojewódzki Szpital Specjalistyczny w Legnicy

location_facility: UCLA Jonsson Comprehensive Cancer Center


title: A Prospective, Open-label, Genotype-match Controlled, Multicenter Clinical Trial to Investigate the Efficacy and Safety of Intra-amniotic ER004 as a Prenatal Treatment for Male Subjects With XLHED

acronym: EDELIFE

summary: This is an open-label, prospective, genotype-match controlled for primary estimand, non randomized, multicenter, international Phase 2 clinical trial designed to investigate the efficacy and safety of ER004 administered intraamniotically as a treatment for unborn XLHED male subjects.

ReferenceCitation: Schneider H, Faschingbauer F, Schuepbach-Mallepell S, Körber I, Wohlfart S, Dick A, Wahlbuhl M, Kowalczyk-Quintas C, Vigolo M, Kirby N, Tannert C, Rompel O, Rascher W, Beckmann MW, Schneider P. Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia. N Engl J Med. 2018 Apr 26;378(17):1604-1610. doi: 10.1056/NEJMoa1714322.

lead_sponsor: EspeRare Foundation

location_country:
Spain
Italy
United Kingdom
United States
France
Germany

location_facility: University Hospital of Wales Cardiff and Vale University Local Health

location_facility: Hospital Universitario Virgen de la Arrixaca


title: An Open-Label, Multicenter, Dose-Escalation and Expansion, Phase I Study to Evaluate Safety, Pharmacokinetics, And Anti-Tumor Activity of RO7300490, A Fibroblast Activation Protein-α (FAP) Targeted CD40 Agonist, as Single Agent or in Combination With Atezolizumab in Participants With Advanced and/or Metastatic Solid Tumors

summary: A study to evaluate the safety, pharmacokinetics and anti-tumor activity of RO7300490 as a single agent or in combination with atezolizumab. The study will consist of 3 parts: [Part 1] Dose-Escalation of RO7300490 as a single agent; [Part 2] Dose-Escalation of RO7300490 in combination with atezolizumab and [Part 3] Dose-Expansion of RO7300490 in combination with atezolizumab in selected cancer types.

lead_sponsor: Hoffmann-La Roche

location_country:
Denmark
Spain
United Kingdom
Korea, Republic of
France

location_facility: Clinica Universidad de Navarra Madrid; Servicio de Oncología

location_facility: Christie Hospital NHS Trust; Experimental Cancer Medicine Team

location_facility: Western General Hospital; Edinburgh Cancer Center

location_facility: Vall d´Hebron Institute of Oncology (VHIO), Barcelona

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Guys and St Thomas Hospital; OHCT Clinical Trials


title: Development and Internal Validation of a Clinical Nomogram to Diagnose Prostatic Inflammation in Men With Lower Urinary Tract Symptoms

acronym: PIN

summary:
Rationale: Histological inflammation of the prostate is a common finding in the results of the histopathological examinations after a prostate biopsy or a transurethral or open prostatectomy.

Several studies have investigated the role of prostatic inflammation in the development of prostatic enlargement and pathogenesis of Lower Urinary Tract Symptoms (LUTS). Therefore, prostatic inflammation could be a potential treatment target for men with LUTS.

Objective: The aim of the study is the development and the validation of a nomogram based on clinical parameters that could predict the presence of prostatic inflammation.

Study design: Non-interventional, multicentric, cross-sectional, observational prospective study.

Study population: Men, age ≥ 40 yrs, with LUTS who will undergo any prostatic surgery for BPH (Open, laparoscopic, robotic, transurethral resection/enucleation, laser prostatectomy) or TRUS-biopsy according to the standard clinical practice of the participating urologists

Intervention: All included males receive standard care for their symptoms according to the physician's practice. For this study, baseline demographic and clinical characteristics of the patients are recorded and correlated with the histological outcome.

Main study parameters/endpoints: Development and validation of the Prostatic Inflammation Nomogram

Nature and extent of the burden and risks associated with participation, benefit and group relatedness: No additional treatment or intervention related to the study is required.

Therefore no negative outcomes are expected as the standard treatment is unchanged. There is no additional burden for the patients.

ReferenceCitation: Nickel JC, Roehrborn CG, O'Leary MP, Bostwick DG, Somerville MC, Rittmaster RS. The relationship between prostate inflammation and lower urinary tract symptoms: examination of baseline data from the REDUCE trial. Eur Urol. 2008 Dec;54(6):1379-84. Epub 2007 Nov 20.

ReferenceCitation: Robert G, Descazeaud A, Nicolaïew N, Terry S, Sirab N, Vacherot F, Maillé P, Allory Y, de la Taille A. Inflammation in benign prostatic hyperplasia: a 282 patients' immunohistochemical analysis. Prostate. 2009 Dec 1;69(16):1774-80. doi: 10.1002/pros.21027.

ReferenceCitation: Irani J, Levillain P, Goujon JM, Bon D, Doré B, Aubert J. Inflammation in benign prostatic hyperplasia: correlation with prostate specific antigen value. J Urol. 1997 Apr;157(4):1301-3.

ReferenceCitation: Grundy SM, Cleeman JI, Daniels SR, Donato KA, Eckel RH, Franklin BA, Gordon DJ, Krauss RM, Savage PJ, Smith SC Jr, Spertus JA, Costa F; American Heart Association; National Heart, Lung, and Blood Institute. Diagnosis and management of the metabolic syndrome: an American Heart Association/National Heart, Lung, and Blood Institute Scientific Statement. Circulation. 2005 Oct 25;112(17):2735-52. Epub 2005 Sep 12. Review. Erratum in: Circulation. 2005 Oct 25;112(17):e297. Circulation. 2005 Oct 25;112(17):e298.

ReferenceCitation: Iasonos A, Schrag D, Raj GV, Panageas KS. How to build and interpret a nomogram for cancer prognosis. J Clin Oncol. 2008 Mar 10;26(8):1364-70. doi: 10.1200/JCO.2007.12.9791. Review.

ipd_sharing: Undecided

ipd_sharing_description: Not yet decided when and what kind of IPD and supporting information will be available to researchers

lead_sponsor: Institute for Monitoring of Urogenital Diseases

location_country: Portugal Spain Italy Greece France Cyprus

location_facility: Dept of Urology, University Hospital of Larissa

location_facility: Dept of Urology, University Hospital of Florence

location_facility: Dept of Urology, General Hospital of Larissa


title: A Multicenter, Open-label, Long-term Extension Study to Investigate the Efficacy and Safety of Lacosamide as Adjunctive Therapy in Pediatric Subjects With Epilepsy With Partial-Onset Seizures

lead_sponsor: UCB BIOSCIENCES, Inc.

ipd_sharing_description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

location_country:
Croatia
Serbia
United Kingdom
Italy
Lithuania
Ukraine
United States
Belgium
Philippines
Romania
Czechia
Montenegro
Poland
Portugal
Slovenia
Russian Federation
Australia
Argentina
Israel
Mexico
Slovakia
Brazil
Hungary
China
Bulgaria
Moldova, Republic of
Taiwan
Greece
Georgia
Estonia
Latvia
Thailand
Korea, Republic of
France
Colombia

summary: The purpose of this study is to evaluate the long-term safety, tolerability and efficacy of lacosamide (LCM) in pediatric subjects.


title: Diagnostic Performance of Prostate HistoScanningTM (PHS) in Men at Risk of Prostate Cancer Scheduled for an Initial Prostate Biopsy

summary:
The primary objective of the PHSTT-01 trial is to determine if prostate HistoScanning (HS) analysis can be used to improve the detection of clinically significant prostate cancer (PCa), and potentially reduce the burden and number of biopsies in routine clinical practice. Prostate HS is an ultrasound-based tissue characterization technology specifically developed to detect, visualize, and locate tissue suspected of harboring PCa. These suspicious tissues are displayed as red areas on an imaging monitor. Recently a new biopsy guidance tool has been developed that uses the results of the prostate HS analysis.

The subjects that will participate in this study are all scheduled for a first biopsy of the prostate. They will initially be imaged using transrectal ultrasound (TRUS) to obtain data for prostate HS analysis. The results of HS analysis will be used later in the procedure. Subjects will then undergo a routine systematic 10- to 12-core biopsy procedure using TRUS. This will be followed by a TRUS-guided biopsy that uses the result of prostate HS analysis and new biopsy guidance tool.

primary_purpose: Diagnostic

lead_sponsor: Advanced Medical Diagnostics s.a.

location_country:
Denmark
Spain
Italy
Austria
Estonia
Latvia
Lithuania
Czech Republic
Netherlands
Russian Federation
Switzerland
Hungary
France
Turkey
United Kingdom
Belgium
Germany
Bulgaria

location_facility: University Medical Center Nuffield Health - University Hospitals Bristol (UHB) - Bristol Royal Infirmary and Southmead Hospitals

location_facility: • University Medical Center Cliniques Universitaires Saint Luc

location_facility: Medical Center St. Elisabeth Krankenhaus

location_facility: Medical Center Martini Klinik - Prostate Cancer Center

location_facility: Medical Center Institut Mutualiste Montouris

location_facility: Medical Center Antoni Van Leeuwenhoek Ziekenhuis - Nederlands Kanker Instituut

location_facility: University Medical Center Všeobecná fakultní nemocnice v Praze (VFN) a 1. -General University Hospital and First Faculty of Medicine Charles University

location_facility: Medical Center Urologická klinika - Fakultní nemocnice

location_facility: University Medical Center Moscow State University of Medicine and Dentistry named after A.I.Evdokimov

location_facility: Medical Center Onze Lieve Vrouw Ziekenhuis OLVZ

location_facility: University Medical Center University Vita-Salute, Scientific Institute H. San Raffaele

location_facility: Medical Center Med.Landeskrankenhaus Vöcklabruck

location_facility: Medical Center Klinikum Wolfsburg Urologie

location_facility: Medical Center North-Estonian Medical Center Foundation

location_facility: University Medical Center Vilniaus Universiteto Onkologijos Institutas - Santariškiu Clinics

location_facility: Medical Center Spire Washington Hospital

location_facility: University Medical Center CHU Saint Etienne

location_facility: University Medical Center Vall d'Hebron University Hospital - Autonoma Universitat Barcelona

location_facility: • University Medical Center Urinary Tract Surgery - Urology Dpt - Odense Universitetshospital Svendborg Sygehus

location_facility: Medical Center Acıbadem Kozyatağı Hospital

location_facility: Medical Center Blackpool Victoria Hospital

location_facility: Medical Center Cancer Center - Prostatazentrum

location_facility: University Medical Center St. Marina University Hospital

location_facility: Medical Center URO-TIP Urological Diagnosis Center


title: A Phase 3, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of Tenofovir Alafenamide (TAF) 25 mg QD Versus Tenofovir Disoproxil Fumarate (TDF) 300 mg QD for the Treatment of HBeAg-Negative, Chronic Hepatitis B

summary: The primary objective of this study is to compare the efficacy, safety, and tolerability of tenofovir alafenamide (TAF) versus tenofovir disoproxil fumarate (TDF) in treatment-naive and treatment-experienced adults with hepatitis B e antigen (HBeAg)-negative chronic hepatitis B virus (HBV) infection.

ReferenceCitation: Cathcart AL, Chan HL, Bhardwaj N, Liu Y, Marcellin P, Pan CQ, Shalimar, Buti M, Cox S, Parhy B, Zhou E, Martin R, Chang S, Lin L, Flaherty JF, Kitrinos KM, Gaggar A, Izumi N, Lim YS. No Resistance to Tenofovir Alafenamide Detected through 96 Weeks of Treatment in Patients with Chronic Hepatitis B Infection. Antimicrob Agents Chemother. 2018 Sep 24;62(10). pii: e01064-18. doi: 10.1128/AAC.01064-18. Print 2018 Oct.

ReferenceCitation: Seto WK, Asahina Y, Brown TT, Peng CY, Stanciu C, Abdurakhmanov D, Tabak F, Nguyen TT, Chuang WL, Inokuma T, Ikeda F, Santantonio TA, Habersetzer F, Ramji A, Lau AH, Suri V, Flaherty JF, Wang H, Gaggar A, Subramanian GM, Mukewar S, Brunetto MR, Fung S, Chan HL. Improved Bone Safety of Tenofovir Alafenamide Compared to Tenofovir Disoproxil Fumarate Over 2 Years in Patients With Chronic HBV Infection. Clin Gastroenterol Hepatol. 2018 Jun 20. pii: S1542-3565(18)30633-5. doi: 10.1016/j.cgh.2018.06.023. [Epub ahead of print]

ReferenceCitation: Agarwal K, Brunetto M, Seto WK, Lim YS, Fung S, Marcellin P, Ahn SH, Izumi N, Chuang WL, Bae H, Sharma M, Janssen HLA, Pan CQ, Çelen MK, Furusyo N, Shalimar D, Yoon KT, Trinh H, Flaherty JF, Gaggar A, Lau AH, Cathcart AL, Lin L, Bhardwaj N, Suri V, Mani Subramanian G, Gane EJ, Buti M, Chan HLY; GS-US-320-0110; GS-US-320-0108 Investigators. 96 weeks treatment of tenofovir alafenamide vs. tenofovir disoproxil fumarate for hepatitis B virus infection. J Hepatol. 2018 Apr;68(4):672-681. doi: 10.1016/j.jhep.2017.11.039. Epub 2018 Jan 17.

ReferenceCitation: Buti M, Gane E, Seto WK, Chan HL, Chuang WL, Stepanova T, Hui AJ, Lim YS, Mehta R, Janssen HL, Acharya SK, Flaherty JF, Massetto B, Cathcart AL, Kim K, Gaggar A, Subramanian GM, McHutchison JG, Pan CQ, Brunetto M, Izumi N, Marcellin P; GS-US-320-0108 Investigators. Tenofovir alafenamide versus tenofovir disoproxil fumarate for the treatment of patients with HBeAg-negative chronic hepatitis B virus infection: a randomised, double-blind, phase 3, non-inferiority trial. Lancet Gastroenterol Hepatol. 2016 Nov;1(3):196-206. doi: 10.1016/S2468-1253(16)30107-8. Epub 2016 Sep 22. Erratum in: Lancet Gastroenterol Hepatol. 2016 Nov;1(3):e2.

ipd_sharing_description: Qualified external researchers may request IPD for this study after study completion. For more information, please visit our website at https://www.gileadclinicaltrials.com/transparency-policy/

location_country:
Poland
Taiwan
Hong Kong
Spain
Italy
Russian Federation
Australia
New Zealand
Canada
United Kingdom
Korea, Republic of
United States
France
India
Turkey
Romania
Japan

location_facility: Vancouver Infectious Disease Research and Care Centre

location_facility: SPZOZ, Wojewódzki Specjalistyczny Szpital

location_facility: 1st Moscow State Medical University University Clinical Hospital #3

location_facility: Ege Universitesi Tip Fakultesi Hastanesi

location_facility: Gastrointestinal Research Institute (GIRI)

location_facility: Institutul National de Boli Infectioase Prof.Dr. Matei Bals

location_facility: Centrum Badan Klinicznych - Przychodnia Badan

location_facility: Postgraduate Institute of Medical Education and Research

location_facility: Pusan National University Yangsan Hospital

location_facility: Uludag Üniversitesi Tıp Fakültesi Gastroenteroloji

location_facility: Hopital Civil de Strasbourg- CHU Service

location_facility: Medical Hospital of Tokyo Medical and Dental University

location_facility: Seth GS Medical College and KEM Hospital

location_facility: State Novosibirsk Regional Clinical Hospital

location_facility: S. R Kalla Memorial Gastro & General Hospital

location_facility: Kobe City Medical Center General Hospital

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku Klinika

location_facility: Clinical Infectious Hospital named after S.P.Botkin

location_facility: Azienda Ospedaliero- Universitaria Pisana

location_facility: Centrul Medical de Diagnostic si Tratament "Dr. Victor Babes"

location_facility: Centre for Liver Research & Diagnostic, Deccan College of Medical Sciences and Allied Hospitals

location_facility: University California San Francisco (UCSF)

location_facility: Szpital Specjalistyczny w Chorzowie Oddział

location_facility: Infection Center LLC Building 20, MSH #163 Territory Koltsovo

location_facility: Institute of Post Graduate Medical Education And Research

location_facility: The Hospital of Hyogo College of Medicine

location_facility: Infectious Clinical Hospital #1 of Moscow Healthcare Department

location_facility: Institutul National de Boli Infectioase "Prof. Dr. Matei Bals"

location_facility: Dicle University Medical Faculty Department of Infectious Diseases

location_facility: Istanbul Universitesi Cerrahpassa Tip Fakultesi Hastanesi

location_facility: Barts and The London NHS Trust Royal London Hospital

location_facility: IRCCS Ospedale Casa Sollievo della Sofferenza

location_facility: Global Hospital Super Specialty & Transplant Centre

location_facility: Azienda Ospedaliero Universitaria Ospedali

location_facility: National Hospital Organization Nagasaki Medical Center

location_facility: Spitatul Clinic de Boli Infectioase Constanta

location_facility: Scientific Research Institute of Clinical Immunology

location_facility: Keimyung University Dongsan Medical Center

location_facility: Gordon and Leslie Diamond Health Care Centre

location_facility: Azienda Ospedaliera S. Orsola - Malpighi

location_facility: Nottingham University Hospitals NHS Trust

location_facility: Limited Liability Company "Modern Medicine Clinic"

location_facility: Ankara Üniversitesi Gastroenteroloji Bilim Dalı Cebeci

location_facility: Zeidler Ledcor Centre Division of Gastroenterology


title: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

acronym: NICOFA

summary: Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia. It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene (FXN) and results in decreased levels of FXN protein. FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty. Patients gradually lose coordination, become dysarthric and are frequently wheel-chair bound as adolescents. There is no disease modifying therapy and many patients die prematurely of cardiomyopathy. It was subsequently found that the FXN gene is silenced at the chromatin level by the formation of heterochromatin and that this heterochromatin formation can be antagonized by histone deacetylase inhibitors (HDACi) (Chan et al., 2013). A recent proof-of-concept clinical study on ten patients with Friedreich ataxia demonstrated that FXN levels can be restored to those seen in asymptomatic carriers using the class III HDACi nicotinamide at a dose that is well tolerated by patients (Libri et al., 2014). Since carriers are asymptomatic, this degree of restoration of FXN expression might be expected to halt disease progression. Nicotinamide readily crosses the blood brain barrier and has previously been given at high doses for long periods to normal individuals without serious adverse effects (Gale et al., 2004; Knip et al., 2000). This study will be the first to provide clinical evidence for the efficacy and safety of nicotinamide in patients with Friedreich´s ataxia.

study_start_date_type: Anticipated

status: Not yet recruiting

ReferenceCitation: Reetz K, Hilgers RD, Isfort S, Dohmen M, Didszun C, Fedosov K, Kistermann J, Mariotti C, Durr A, Boesch S, Klopstock T, Rodríguez de Rivera Garrido FJ, Schöls L, Klockgether T, Pandolfo M, Korinthenberg R, Lavin P, Molenberghs G, Libri V, Giunti P, Festenstein R, Schulz JB; EFACTS or NICOFA study group. Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA). Neurol Res Pract. 2019 Oct 15;1:33. doi: 10.1186/s42466-019-0038-9. eCollection 2019.

ipd_sharing_description: The study results will be published in appropriate international scientific journals, and publishing details will be given in the clinical study agreement. The study will be registered and study results will be disclosed by the principal investigator in one or more public clinical study registry(ies), according to national/international use.

lead_sponsor: RWTH Aachen University

location_country:
Spain
Italy
Austria
United Kingdom
France
Germany

location_facility: Service de génétique médicale - Hôpital La Pitié Salpetrière

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta


title: Phase IIIB, Open-label, Multi-Center Study of the Efficacy and Safety of Rigosertib Administered as 72-hour Continuous Intravenous Infusions in Patients With Myelodysplastic Syndrome With Excess Blasts Progressing On or After Azacitidine or Decitabine

summary: This study will examine the effect intravenously administered rigosertib has on the relationship between bone marrow blasts response and overall survival in myelodysplastic syndromes (MDS) patients who have 5-30% bone marrow blasts and who progressed on or after treatment with azacitidine or decitabine.

ReferenceCitation: Olnes MJ, Shenoy A, Weinstein B, Pfannes L, Loeliger K, Tucker Z, Tian X, Kwak M, Wilhelm F, Yong AS, Maric I, Maniar M, Scheinberg P, Groopman J, Young NS, Sloand EM. Directed therapy for patients with myelodysplastic syndromes (MDS) by suppression of cyclin D1 with ON 01910.Na. Leuk Res. 2012 Aug;36(8):982-9. doi: 10.1016/j.leukres.2012.04.002. Epub 2012 Apr 21.

ReferenceCitation: Seetharam M, Fan AC, Tran M, Xu L, Renschler JP, Felsher DW, Sridhar K, Wilhelm F, Greenberg PL. Treatment of higher risk myelodysplastic syndrome patients unresponsive to hypomethylating agents with ON 01910.Na. Leuk Res. 2012 Jan;36(1):98-103. doi: 10.1016/j.leukres.2011.08.022. Epub 2011 Sep 14.

ReferenceCitation: Silverman LR, Greenberg P, Raza A, Olnes MJ, Holland JF, Reddy P, Maniar M, Wilhelm F. Clinical activity and safety of the dual pathway inhibitor rigosertib for higher risk myelodysplastic syndromes following DNA methyltransferase inhibitor therapy. Hematol Oncol. 2015 Jun;33(2):57-66. doi: 10.1002/hon.2137. Epub 2014 Apr 29.

ReferenceCitation: Al-Kali A. Relationship of bone marrow blast (BMBL) response to overall survival (OS) in a multicenter study of rigosertib (Rigo) in patients (pts) with myelodysplastic syndrome (MDS) with excess blasts progressing on or after treatment with a hypomethylating agent (HMA). Journal of Clinical Oncology 2017 35:15_suppl, 7056-7056 ASCO 2017

ReferenceCitation: Garcia-Manero G, Fenaux P. Comprehensive Analysis of Safety: Rigosertib in 557 Patients with Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). Blood Dec 2016, 128 (22) 2011; ASH 2016.

lead_sponsor: Onconova Therapeutics, Inc.

location_country:
Denmark
Spain
Italy
Australia
Sweden
United States
France
Germany

location_facility: Universitätsklinikum Frankfurt, Goethe Universität

location_facility: Universitätsklinikum Köln Klinik I für Innere Medizin

location_facility: University of Texas Southwestern Medical Center-Parkland Hospital

location_facility: Karolinska University Hospital, Huddinge

location_facility: AOU Maggiore della Carità, SCUD Ematologia

location_facility: Policlinico Umberto 1, Universita "Sapienza"

location_facility: New York Presbyterian Hospital-Weill Cornell Medical College

location_facility: University of Kansas Cancer Center and Medical Pavilion

location_facility: Greenbaum Cancer Center University of Maryland

location_facility: Hôpital Saint-Louis, Service d'Hématologie

location_facility: Technische Universität München, III. Medizinische Klinik

location_facility: Rigshospitalet, Department of Hematology


title: Post-Market Clinical Follow-up Study to Monitor Device Performance and Outcomes of the CENTERA Transcatheter Heart Valve System

summary: This study will monitor device performance and safety of the Edwards CENTERA Transcatheter Heart Valve (THV) system in patients with severe, symptomatic aortic valve stenosis who are indicated for aortic valve replacement.

lead_sponsor: Edwards Lifesciences

location_country:
Denmark
Spain
United Kingdom
Netherlands
France
Germany

location_facility: Vall d'Hebron University Hospital - Barcelona

location_facility: Hospital Universitario Central de Asturias


acronym: CheckMate 069

title: Phase 2, Randomized, Double Blinded, Study of Nivolumab (BMS-936558) in Combination With Ipilimumab vs Ipilimumab Alone in Subjects With Previously Untreated, Unresectable or Metastatic Melanoma

lead_sponsor: Bristol-Myers Squibb

ReferenceCitation: Mantia CM, Werner L, Stwalley B, Ritchings C, Tarhini AA, Atkins MB, McDermott DF, Regan MM. Sensitivity of treatment-free survival to subgroup analyses in patients with advanced melanoma treated with immune checkpoint inhibitors. Melanoma Res. 2022 Feb 1;32(1):35-44. doi: 10.1097/CMR.0000000000000793.

ReferenceCitation: Hodi FS, Chesney J, Pavlick AC, Robert C, Grossmann KF, McDermott DF, Linette GP, Meyer N, Giguere JK, Agarwala SS, Shaheen M, Ernstoff MS, Minor DR, Salama AK, Taylor MH, Ott PA, Horak C, Gagnier P, Jiang J, Wolchok JD, Postow MA. Combined nivolumab and ipilimumab versus ipilimumab alone in patients with advanced melanoma: 2-year overall survival outcomes in a multicentre, randomised, controlled, phase 2 trial. Lancet Oncol. 2016 Nov;17(11):1558-1568. doi: 10.1016/S1470-2045(16)30366-7. Epub 2016 Sep 9.

ReferenceCitation: Postow MA, Chesney J, Pavlick AC, Robert C, Grossmann K, McDermott D, Linette GP, Meyer N, Giguere JK, Agarwala SS, Shaheen M, Ernstoff MS, Minor D, Salama AK, Taylor M, Ott PA, Rollin LM, Horak C, Gagnier P, Wolchok JD, Hodi FS. Nivolumab and ipilimumab versus ipilimumab in untreated melanoma. N Engl J Med. 2015 May 21;372(21):2006-17. doi: 10.1056/NEJMoa1414428. Epub 2015 Apr 20. Erratum in: N Engl J Med. 2018 Nov 29;379(22):2185.

location_country:
United States
France

location_facility: Washington University School Of Medicine

location_facility: University Of Louisville Medical Center, Inc., Dba

location_facility: University Of Wisconsin Paul P Carbone Comprehensive Ca Ctr

summary: The primary purpose of this study is to compare the objective response rate, as determined by investigators, of Nivolumab combined with Ipilimumab versus Ipilimumab monotherapy in patients with untreated, unresectable, or metastatic melanoma


title: A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease

summary: Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive BMN 701 20 mg/kg by IV infusion every other week. All participants will receive active drug. No dose of existing therapy will be missed - experimental drug is started immediately.

lead_sponsor: BioMarin Pharmaceutical

location_country:
Portugal
United Kingdom
Italy
Netherlands
United States
France
Belgium
Germany

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

location_facility: Villa Metabolica, ZKJM MC University Mainz

location_facility: The Ohio State University - Wexner Medical Center

location_facility: National Hospital for Neurology and Neurosurgery

location_facility: Azienda Ospedaliera Universitaria Policlinico "G. Martino" - Messina


title: A 6-Week Randomized, Parallel, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Evaluate The Efficacy and Safety of Lurasidone in Adolescent Subjects With Schizophrenia

lead_sponsor: Sunovion

location_country:
Poland
Belgium
Romania
Spain
United Kingdom
Russian Federation
Mexico
Ukraine
Hungary
Korea, Republic of
United States
France
Puerto Rico
Colombia
Philippines
Malaysia
Bulgaria

location_facility: West Visayas State University Medical Center

location_facility: Nizhny Novgorod Regional State Institution of Healthcare

location_facility: Family Psychiatry of The Woodlands, P.A.

location_facility: CI Lviv Regional Clinical Psychiatric Hospital

location_facility: Centro de Investigaciones y Proyectos en Neurociencias CIPNA

location_facility: Ternopil RCCPH Dept of Psychiatry #9 (adolescent)& #8 (pediatric) Ternopil I.Ya. Gorbachevskyi SMU

location_facility: Centro de Investigaciones del Sistema Nervioso Limitada - Grupo CISNE Ltda

location_facility: Medical Research Group of Central Florida

location_facility: Spitalul Clinic de Psihiatrie Prof. Dr. Alexandru Obregia

location_facility: University Hospitals Case Medical Center

location_facility: Spitalul Clinic de Urgenta pentru Copii "Louis Turcanu" Timisoara

location_facility: CI Odesa Regional Medical Center of Mental Health

location_facility: Sverdlov regional Psychiatric Clinical Hospital

location_facility: Centro para el Desarrollo de la Medicina y de Asistencia Medica Especializada S.C.

location_facility: Neuropsychiatric Research Center of Orange County

location_facility: CITrials, Inc. - Riverside & San Bernardino County

location_facility: California Pharmaceutical Research Institute, Inc

location_facility: Wojewodzki Szpital Zespolony im. L. Rydygiera w Toruniu

location_facility: CI Kherson Regional Psychiatric Hospital of Kherson RC

location_facility: Centro de Investigacion Clinica Psiquiatrica

location_facility: Severance Hospital, Yonsei University Health System

location_facility: St. Petersburg State Healthcare Institution (SPSHI)

location_facility: SHI Regional Clinical Psychiatry Hospital of St. Sofia

location_facility: SI Institute of Neurology, Psychiatry and Narcology of NAMSU

location_facility: O.F. Maltcev Poltava RCPsH Children Dept Ukrainian Medical Stomatological Academy

location_facility: Vadaskert Alapitvany a Gyermekek Lelki Egeszsegeert

location_facility: Alexian Brothers Health and Wellness Center

location_facility: TMA Psychiatry in Kyiv Center of NT & Rehabilitation of Psychotic Conditions

location_facility: SI Institute of Children and Adolescents Healthcare of NAMSU

location_facility: Hôpitaux Pédiatriques de Nice CHU-Lenval

location_facility: Regional Government Institution Kipetsk Regional Psychoneurology Hospital

location_facility: M.I. Pyrogov VNMU Ch of Psych&Nar BO CI O.I. Yuschenko VRPsH

location_facility: FSBI "Bekhterev Psychoneurological Research Institute SPb Russia"

location_facility: Instituto de Informacion de Investigacion en Salud Mental

location_facility: FSBSI "Scientific Research Institute of Mental Health"

location_facility: RPsH #3 Сhildren Dept SHEI Ivano-Frankivsk SMU

summary: Efficacy and Safety study of Lurasidone in pediatric patients.


acronym: Basalt 3

title: A Phase Ib/II Study of Docetaxel With or Without Buparlisib as Second Line Therapy for Patients With Advanced or Metastatic Squamous Non-small Cell Lung Cancer

lead_sponsor: Novartis Pharmaceuticals

location_country:
Spain
Italy
Sweden
Korea, Republic of
United States
France
Belgium
Germany

location_facility: Reliant Medical Group Reliant Medical Group

location_facility: H. Lee Moffitt Cancer Center & Research Institute H Lee Moffitt

location_facility: Virginia Oncology Associates Virginia Oncology Assoc. (2)

summary:
This is a multi-center, open-label Phase Ib dose escalation part followed by a randomized double-blinded placebo controlled Phase II part.

The Phase Ib part will determine the Maximum Tolerated Dose (MTD)/Recommended Phase II Dose (RP2D) of buparlisib in combination with docetaxel. Subsequently the MTD/RP2D will be investigated in a Phase II randomized trial in patients with advanced or metastatic squamous NSCLC.


title: A Randomized, Double-Blind, Multi-Centre Study to Evaluate the Efficacy and Safety of Adding Mirabegron to Solifenacin in Incontinent OAB Subjects Who Have Received Solifenacin for 4 Weeks and Warrant Additional Relief for Their OAB Symptoms

acronym: BESIDE

summary: The purpose of this study was to see if adding a new type of medication recently approved to treat overactive bladder (mirabegron) to an antimuscarinic treatment (solifenacin) would be more effective in controlling incontinence than when using the antimuscarinic treatment alone.

ReferenceCitation: Gibson W, MacDiarmid S, Huang M, Siddiqui E, Stölzel M, Choudhury N, Drake MJ. Treating Overactive Bladder in Older Patients with a Combination of Mirabegron and Solifenacin: A Prespecified Analysis from the BESIDE Study. Eur Urol Focus. 2017 Dec;3(6):629-638. doi: 10.1016/j.euf.2017.08.008. Epub 2017 Sep 12.

ReferenceCitation: Drake MJ, Chapple C, Esen AA, Athanasiou S, Cambronero J, Mitcheson D, Herschorn S, Saleem T, Huang M, Siddiqui E, Stölzel M, Herholdt C, MacDiarmid S; BESIDE study investigators. Efficacy and Safety of Mirabegron Add-on Therapy to Solifenacin in Incontinent Overactive Bladder Patients with an Inadequate Response to Initial 4-Week Solifenacin Monotherapy: A Randomised Double-blind Multicentre Phase 3B Study (BESIDE). Eur Urol. 2016 Jul;70(1):136-145. doi: 10.1016/j.eururo.2016.02.030. Epub 2016 Mar 8.

ipd_sharing_description: Access to anonymized individual participant level data collected during the trial, in addition to study-related supporting documentation, is planned for trials conducted with approved product indications and formulations, as well as compounds terminated during development. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com.

lead_sponsor: Astellas Pharma Europe Ltd.

location_country:
Spain
Armenia
Italy
United Kingdom
United States
Belgium
Romania
Czechia
Poland
Portugal
Slovenia
Austria
Australia
Russian Federation
Israel
Turkey
Norway
Sweden
Slovakia
Lebanon
Canada
Switzerland
Hungary
Germany
Denmark
Greece
Georgia
Ireland
Finland
France
Netherlands

location_facility: PMG Research of Raleigh, dba PMG Research of Cary

location_facility: Premier Medical Group of the Hudson Valley PC

location_facility: Associated Pharmaceutical Research Center, Inc.


title: A Randomized, Placebo-controlled Study to Investigate the Efficacy and Safety of Circadin® to Alleviate Sleep Disturbances in Children With Neurodevelopmental Disabilities

lead_sponsor: Neurim Pharmaceuticals Ltd.

ReferenceCitation: Malow BA, Findling RL, Schroder CM, Maras A, Breddy J, Nir T, Zisapel N, Gringras P. Sleep, Growth, and Puberty After 2 Years of Prolonged-Release Melatonin in Children With Autism Spectrum Disorder. J Am Acad Child Adolesc Psychiatry. 2021 Feb;60(2):252-261.e3. doi: 10.1016/j.jaac.2019.12.007. Epub 2020 Jan 23.

ReferenceCitation: Gringras P, Nir T, Breddy J, Frydman-Marom A, Findling RL. Efficacy and Safety of Pediatric Prolonged-Release Melatonin for Insomnia in Children With Autism Spectrum Disorder. J Am Acad Child Adolesc Psychiatry. 2017 Nov;56(11):948-957.e4. doi: 10.1016/j.jaac.2017.09.414. Epub 2017 Sep 19.

location_country:
United Kingdom
Finland
United States
France
Netherlands

location_facility: Southwest Autism Research and Resource Center (SARRC)

location_facility: Clinical research center of New Jersey, LLC

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: Blackpool Victoria Teaching Hospitals NHS Foundation Trust

location_facility: Birmingham Childrens Hospital NHS FOUNDATION TRUST

location_facility: Strasbourg University Hospital Depatment of Child Psychiatry & Neurology

location_facility: Guy's & St. Thomas's NHS Foundation Trust of St Thomas's Hospital

location_facility: Attalla Consultants LLC, dba Institue for Behabiovral medicine

summary: The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy of a double-blind, 13 week treatment period with Circadin 2/5mg in improving maintenance of sleep, sleep latency and additional parameters in children with neurodevelopmental disabilities. The efficacy and safety of Circadin 2/5 mg will continue to be assessed during an open-label extension period of 13 weeks.


acronym: Chime

title: A Randomized, Open-Label Phase 2 Study Evaluating LY2875358 Plus Erlotinib and LY2875358 Monotherapy in MET Diagnostic Positive NSCLC Patients With Acquired Resistance to Erlotinib

lead_sponsor: Eli Lilly and Company

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

location_country:
Spain
Italy
United Kingdom
Netherlands
Korea, Republic of
United States
France
Israel
Belgium
Germany

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

location_facility: University of California, Davis - Health Systems

location_facility: Georgetown University Medical Center IRB

location_facility: University of Colorado Health Sciences Center

summary: The primary purpose of this study is to evaluate the efficacy of the study drug known as LY2875358, administered alone or in combination with a second drug named Erlotinib, in participants affected by a defined type of lung cancer (MET biomarker diagnostic positive Non-Small-Cell Lung Cancer) that experienced a disease progression during the most recent treatment with Erlotinib.


title: A Randomized, Double-Masked and Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sarilumab Administered Subcutaneously Every 2 Weeks in Patients With Non-Infectious, Intermediate, Posterior or Pan-Uveitis (NIU)

acronym: SARILNIUSATURN

summary:
Primary Objective:

To evaluate the efficacy of sarilumab at Week 16 in participants with non-infectious uveitis (NIU).

Secondary Objectives:

To evaluate the change in best corrected visual acuity (BCVA). To evaluate the safety of subcutaneous sarilumab in participants with NIU. To evaluate the change in macular edema. To evaluate the change in other signs of ocular inflammation. To evaluate the effect on retinal vessel leakage. To evaluate the effect of sarilumab on reducing concomitant immunosuppressant therapy.

To evaluate the change in ocular inflammation in the anterior chamber. To evaluate the pharmacokinetics of sarilumab in NIU participants. To evaluate the immunogenicity with anti-drug antibodies (ADA).

lead_sponsor: Sanofi

location_country:
Spain
Italy
United States
France
Turkey
Czechia

location_facility:
Investigational Site Number 792006
Investigational Site Number 380003
Investigational Site Number 792002
Investigational Site Number 250002
Investigational Site Number 724001
Investigational Site Number 203002
Investigational Site Number 380004
Investigational Site Number 840006
Investigational Site Number 724003
Investigational Site Number 840005
Investigational Site Number 792003
Investigational Site Number 792004
Investigational Site Number 840008
Investigational Site Number 203001
Investigational Site Number 792005
Investigational Site Number 250001
Investigational Site Number 792001
Investigational Site Number 840007
Investigational Site Number 380001
Investigational Site Number 840009