title: A Three Arms Prospective, Randomized Phase II Study to Evaluate the Best Sequential Approach With Combo Immunotherapy (Ipilimumab/Nivolumab) and Combo Target Therapy (LGX818/MEK162) in Patients With Metastatic Melanoma and BRAF Mutation

acronym: SECOMBIT

summary: To evaluate the best sequencing approach with the combination of target agents (LGX818 plus MEK162) and the combination of immunomodulatory antibodies (ipilimumab plus nivolumab) in patients with metastatic melanoma and BRAF V600 mutation.

ReferenceCitation: Ascierto PA, Mandala M, Ferrucci PF, Guidoboni M, Rutkowski P, Ferraresi V, Arance A, Guida M, Maiello E, Gogas H, Richtig E, Fierro MT, Lebbe C, Helgadottir H, Queirolo P, Spagnolo F, Tucci M, Del Vecchio M, Gonzales Cao M, Minisini AM, De Placido S, Sanmamed MF, Mallardo D, Curvietto M, Melero I, Palmieri G, Grimaldi AM, Giannarelli D, Dummer R, Chiarion Sileni V. Sequencing of Ipilimumab Plus Nivolumab and Encorafenib Plus Binimetinib for Untreated BRAF-Mutated Metastatic Melanoma (SECOMBIT): A Randomized, Three-Arm, Open-Label Phase II Trial. J Clin Oncol. 2023 Jan 10;41(2):212-221. doi: 10.1200/JCO.21.02961. Epub 2022 Sep 1.

publisher_dissemination: American Society of Clinical Oncology (ASCO)

lead_sponsor: Fondazione Melanoma Onlus

sponsor_collaborators:
Fondazione Melanoma Onlus
Clinical Research Technology S.r.l.

location_country:
Germany
Italy
Poland
United Kingdom
Sweden
Switzerland
Austria
Spain
Greece
France

location_facility: Karl Landsteiner University of Health Sciencies - University Clinic

location_facility: IEO - Istituto Europeo di Oncologia - IRCCS

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori "Fondazione Giovanni Pascale"

location_facility: Azienda Ospedaliera Universitaria - Città della Salute e della Scienza di Torino

location_facility: Istituto Dermopatico dell'Immacolata - IDI - IRCCS

location_facility: IRCCS - Istituto Scientifico Romagnolo per la Cura e lo Studio dei Tumori (I.R.S.T) S.r.l.

location_facility: Fondazione I.R.C.C.S. Istituto Nazionale dei Tumori

location_facility: Maria Sklodowska-Curie Institute - Oncology Center

location_facility: Azienda Ospedaliero Universitaria Federico II

location_facility: Azienda Ospedaliera Universitaria Integrata di Udine

location_facility: Università degli Studi di Bari Aldo Moro


title: A Phase I, Multicenter, Open-Label Safety, Pharmacokinetic and Preliminary Efficacy Study of Wild-type Sparing EGFR Inhibitor, AC0010MA, in Adult Patients With Previously Treated EGFRmut and Acquired T790M Mutation Non-Small Cell Lung Cancer (NSCLC)

summary: AC0010MA is a new, irreversible, Epidermal Growth Factor Receptor (EGFR) mutation selective Tyrosine Kinase Inhibitor. Aim at local advanced or metastatic non-small cell lung cancer patients with EGFR mutation or T790M drug-resistant mutation. The molecular mechanism: by irreversible combining the EGFR-RTKs ATP binding site of cell, selectively suppress the activities of EGFR tyrosine kinase phosphorylation, block the signal transduction pathway of EGFR and inhibit the function of ras/raf/MAPK downstream, thus block the tumor cell growth by EGFR induction, and promotes apoptosis. AC0010MA Maleate Capsules has three characters: 1. Irreversible binding to EGFR; 2. Effectively suppresses the tumor cell with EGFR mutant while has no suppression to EGFR wild-type cell; 3. Efficient suppress the tumor cell with EGFR T790M drug-resistant mutation.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Non-Randomized

primary_purpose: Treatment

enrollment_type: Actual

ipd_sharing: No

lead_sponsor: ACEA Therapeutics, Inc.

location_country:
United States
Spain
France

location_facility: City of Hope Comprehensive Cancer Center

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


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.

status: Completed

ReferenceType: result

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; Societe Francaisedes 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 Societe Francaise 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. doi: 10.1200/JCO.2006.06.1572.

lead_sponsor: Children's Cancer and Leukaemia Group

location_country:
Ireland
United Kingdom
France

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Sir James Spence Institute of Child Health

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Royal Marsden NHS Foundation Trust - Surrey

location_facility: Institute of Child Health at University of Bristol

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

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

location_facility: Great Ormond Street Hospital for Children NHS Trust

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

status_simplified: Completed


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
United Kingdom
France

intervention_type: Device


title: A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

summary: Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. This 3-year, non-interventional, two-part, natural history study is designed to gain insight into total body HO, FOP disease progression, the impact of FOP on subjects' physical functioning, and clinical features and biomarkers that may be useful in the diagnosis and monitoring of disease progression. This natural history study will also provide important information to inform the design of subsequent interventional trials.

study_type: Observational

time_perspective: Prospective

ReferenceType: background

ReferenceCitation: Connor JM, Evans DA. Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J Bone Joint Surg Br. 1982;64(1):76-83. doi: 10.1302/0301-620X.64B1.7068725.

ReferenceCitation: Zhang W, Zhang K, Song L, Pang J, Ma H, Shore EM, Kaplan FS, Wang P. The phenotype and genotype of fibrodysplasia ossificans progressiva in China: a report of 72 cases. Bone. 2013 Dec;57(2):386-91. doi: 10.1016/j.bone.2013.09.002. Epub 2013 Sep 17.

ReferenceCitation: Cohen RB, Hahn GV, Tabas JA, Peeper J, Levitz CL, Sando A, Sando N, Zasloff M, Kaplan FS. The natural history of heterotopic ossification in patients who have fibrodysplasia ossificans progressiva. A study of forty-four patients. J Bone Joint Surg Am. 1993 Feb;75(2):215-9. doi: 10.2106/00004623-199302000-00008.

ReferenceType: derived

ReferenceCitation: Warner SE, Kaplan FS, Pignolo RJ, Smith SE, Hsiao EC, De Cunto C, Di Rocco M, Harnett K, Grogan D, Genant HK. Whole-body Computed Tomography Versus Dual Energy X-ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva. Calcif Tissue Int. 2021 Dec;109(6):615-625. doi: 10.1007/s00223-021-00877-6. Epub 2021 Jul 31.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

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

license_normalized: no license

repository_institution: University of California - eScholarship University of California

evidence: oa repository (via pmcid lookup)

repositories_concat: PubMed Central;escholarship.org

unpaywall_oa_status: hybrid

publisher_dissemination: Springer-Nature

ReferenceCitation: Kou S, De Cunto C, Baujat G, Wentworth KL, Grogan DR, Brown MA, Di Rocco M, Keen R, Al Mukaddam M, le Quan Sang KH, Masharani U, Kaplan FS, Pignolo RJ, Hsiao EC. Patients with ACVR1R206H mutations have an increased prevalence of cardiac conduction abnormalities on electrocardiogram in a natural history study of Fibrodysplasia Ossificans Progressiva. Orphanet J Rare Dis. 2020 Jul 29;15(1):193. doi: 10.1186/s13023-020-01465-x.

oa_colors: green gold

evidence: oa journal (via doaj)

repositories_concat: PubMed Central

unpaywall_oa_status: gold

ReferenceCitation: Towler OW, Shore EM, Kaplan FS. Skeletal malformations and developmental arthropathy in individuals who have fibrodysplasia ossificans progressiva. Bone. 2020 Jan;130:115116. doi: 10.1016/j.bone.2019.115116. Epub 2019 Oct 23.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

ReferenceCitation: Pignolo RJ, Baujat G, Brown MA, De Cunto C, Di Rocco M, Hsiao EC, Keen R, Al Mukaddam M, Sang KLQ, Wilson A, White B, Grogan DR, Kaplan FS. Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes. Orphanet J Rare Dis. 2019 May 3;14(1):98. doi: 10.1186/s13023-019-1068-7. Erratum In: Orphanet J Rare Dis. 2019 May 23;14(1):113.

repository_institution: Queensland University of Technology - QUT ePrints

repository_institution: King's College London - Research Portal

lead_sponsor: Clementia Pharmaceuticals Inc.

location_country:
Italy
United Kingdom
Argentina
Australia
United States
France

location_facility: The Royal National Orthopaedic Hospital, Brockley Hill

location_facility: University of Pennsylvania, Center for FOP & Related Bone Disorders

location_facility: Hospital Italiano de Buenos Aires, Department of Pediatrics

location_facility: Hôpital Necker-Enfants Malades, Department of Genetics

location_facility: Gaslini Institute, Unit of Rare Diseases, Department of Pediatrics

location_facility: University of California San Francisco, Division of Endocrinology and Metabolism

location_facility: Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)


title: Longitudinal Observational Study to Determine the Correlation Between Patient's Pharmacodynamic Response to Immunosuppressants Measured in Vitro With IMMUNOBIOGRAM and of Rejection in Graft Biopsies in Patients With Renal Transplantation

acronym: BIOTRAIM

summary:
Immunobiogram (IMBG) is a novel in vitro diagnostic bioassay developed by Biohope Scientific Solutions for Human Health SL, that allows to measure the pharmacodynamic response to individual immunosuppressive drugs in patients with a renal transplantation. Pharmacodynamics can complement the already available pharmacokinetic information on immunosuppressants and enable a more individualized evaluation of the immunosuppressive therapy.

The aim of this study is to evaluate the association between the pharmacodynamic response to individual immunosuppressants taken by the patient measured in vitro with IMBG and the existence of signs of graft rejection in biopsies (upon indication or protocol) performed in a sample of kidney transplant patients.

The main hypothesis is that a lower sensitivity to the immunosuppressive drugs taken by the patient will be associated with a higher probability of rejection.

status: Recruiting

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ReferenceCitation: Portoles JM, Jimenez C, Janeiro D, Lopez-Oliva MO, Ortega-Carrion A, Blanquez D, Arribas L, Gomez C, Diez T, Pascual J, Portero I. The Immunobiogram, a Novel In Vitro Assay to Evaluate Treatment Resistance in Patients Receiving Immunosuppressive Therapy. Front Immunol. 2021 Jan 25;11:618202. doi: 10.3389/fimmu.2020.618202. eCollection 2020.

ReferenceCitation: Pascual J, Jimenez C, Krajewska M, Seron D, Kotton CN, Portoles J, Witzke O, Sorensen SS, Andres A, Crespo M, Paz-Artal E, Diez T, Ortega A, Portero I. The Immunobiogram, a novel in vitro diagnostic test to measure the pharmacodynamic response to immunosuppressive therapy in kidney transplant patients. Transpl Immunol. 2022 Dec;75:101711. doi: 10.1016/j.trim.2022.101711. Epub 2022 Sep 9.

ReferenceCitation: Pascual J, Crespo M, Portoles J, Jimenez C, Ortega-Carrion A, Diez T, Portero I. The IMBG Test for Evaluating the Pharmacodynamic Response to Immunosuppressive Therapy in Kidney Transplant Patients: Current Evidence and Future Applications. Int J Mol Sci. 2023 Mar 8;24(6):5201. doi: 10.3390/ijms24065201.

lead_sponsor: Biohope Scientific Solutions for Human Health, S.L.

location_country: Germany France Brazil Spain Sweden

location_facility: Hospital Universitario Marqués de Valdecilla

intervention_type: Diagnostic Test

status_simplified: Ongoing


type: Other Identifier

title: Attitudes and Unmet Needs Among Type 2 Diabetes Patients

summary: The main purpose of this study is to explore experiences and understand the unmet needs of people with Type 2 Diabetes (T2D) among those who are being managed with basal insulin and those who are insulin naïve in order to understand perspectives for a once weekly insulin option; including any potential barriers to being managed with insulin therapy overall. Study participants will be recruited to complete a 15-minute self-administered online survey.

time_perspective: Cross-Sectional

ipd_sharing: Yes

ipd_sharing_description: "According to the Novo Nordisk disclosure commitment on novonordisk-trials.com"

lead_sponsor: Novo Nordisk A/S

location_country:
India
United Kingdom
Australia
Mexico
Korea, Republic of
Saudi Arabia
France

intervention_type: Other


title: A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)

summary: The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.

ipd_sharing_description: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

lead_sponsor: Alexion Pharmaceuticals, Inc.

location_country:
Italy
Japan
United States
Spain
Serbia
France


title: A Phase III Trial of Risk-stratified, Reduced Intensity Adjuvant Treatment in Patients Undergoing Transoral Surgery for Human Papillomavirus (HPV)-Positive Oropharyngeal Cancer

acronym: PATHOS

summary:
The main objectives of the PATHOS study are:

To assess whether swallowing function can be improved following transoral resection of HPV-positive OPSCC, by reducing the intensity of adjuvant treatment protocols. The aim is to personalise treatment, based on disease biology (HPV status and pathology findings), to optimise patient outcomes.

To demonstrate the non-inferiority of reducing the intensity of adjuvant treatment protocols in terms of overall survival in the reduced intensity treatment arms.

design_allocation: Randomized

ReferenceCitation: Owadally W, Hurt C, Timmins H, Parsons E, Townsend S, Patterson J, Hutcheson K, Powell N, Beasley M, Palaniappan N, Robinson M, Jones TM, Evans M. PATHOS: a phase II/III trial of risk-stratified, reduced intensity adjuvant treatment in patients undergoing transoral surgery for Human papillomavirus (HPV) positive oropharyngeal cancer. BMC Cancer. 2015 Aug 27;15:602. doi: 10.1186/s12885-015-1598-x.

repository_institution: Newcastle Univesity - Newcastle University ePrints

repository_institution: Cardiff University - ORCA Online Research @Cardiff

version: acceptedVersion

lead_sponsor: Lisette Nixon

collaborators: Princess Alexandra Hospital, Brisbane, Australia

location_country:
Germany
United Kingdom
Australia
United States
France

location_facility: Royal United Hospitals Bath NHS Foundation Trust

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: HPV Research Group Section of Pathology Cardiff University ,School of Medicine

location_facility: Central Manchester University Hospital NHS Foundation Trust

location_facility: Board of Trustees of the Leland Stanford Junior University

location_facility: Newcastle upon Tyne Hospitals NHS Foundation Trust

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: Derby Teaching Hospitals NHS Foundation Trust

location_facility: Oxford University Hospitals NHS Foundation Trust

location_facility: University College London Hospitals NHS Foundation Trust

location_facility: University Hospitals Dorset NHS Foundation

location_facility: City Hospitals Sunderland NHS Foundation Trust

location_facility: Guys and St Thomas's NHS Foundation Trust

location_facility: Swansea Bay University Local Health Board

location_facility: Royal Devon University Health Care NHS Foundation Trust

location_facility: Cardiff and Vale University Local Health Board


intervention_type: Biological

lead_sponsor: Kyowa Kirin, Inc.

summary:
The objectives of the study are to:

Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants
Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile
Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients
Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients
Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function
Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients
Evaluate the long-term safety and efficacy of burosumab

ReferenceCitation: Linglart A, Imel EA, Whyte MP, Portale AA, Hogler W, Boot AM, Padidela R, Van't Hoff W, Gottesman GS, Chen A, Skrinar A, Scott Roberts M, Carpenter TO. Sustained Efficacy and Safety of Burosumab, a Monoclonal Antibody to FGF23, in Children With X-Linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Feb 17;107(3):813-824. doi: 10.1210/clinem/dgab729.

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

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

repositories_concat: PubMed Central;pure.rug.nl

publisher_dissemination: The Endocrine Society

ReferenceCitation: Mao M, Carpenter TO, Whyte MP, Skrinar A, Chen CY, San Martin J, Rogol AD. Growth Curves for Children with X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2020 Oct 1;105(10):3243-9. doi: 10.1210/clinem/dgaa495.

ReferenceCitation: Carpenter TO, Whyte MP, Imel EA, Boot AM, Hogler W, Linglart A, Padidela R, Van't Hoff W, Mao M, Chen CY, Skrinar A, Kakkis E, San Martin J, Portale AA. Burosumab Therapy in Children with X-Linked Hypophosphatemia. N Engl J Med. 2018 May 24;378(21):1987-1998. doi: 10.1056/NEJMoa1714641.

oa_colors: green other

evidence: open (via free article)

repository_institution: Indiana University - Purdue University Indianapolis - IUPUI ScholarWorks

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

unpaywall_oa_status: bronze

publisher_dissemination: Massachusetts Medical Society

sponsor_collaborators:
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

location_facility:
University of California San Francisco
Royal Manchester Hospital
Yale University School of Medicine
Shriners Hospital for Children
University Medical Center Groningen
Bicetre Hospital
Birmingham Children's University
Great Ormond Street Hospital
Indiana University Hospital

title: A Randomized, Open-Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the Anti-FGF23 Antibody, KRN23, in Pediatric Patients With X-linked Hypophosphatemia (XLH)

source: euctr

location_country:
United States
United Kingdom
Netherlands
France


title: Patisiran-LNP Pregnancy Surveillance Program

summary: The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

lead_sponsor: Alnylam Pharmaceuticals

location_country:
Germany
Italy
Portugal
Netherlands
United States
Spain
France


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, Hadj-Rabia S, Faschingbauer F, Bodemer C, Grange DK, Norton ME, Cavalli R, Tadini G, Stepan H, Clarke A, Guillen-Navarro E, Maier-Wohlfart S, Bouroubi A, Porte F. Protocol for the Phase 2 EDELIFE Trial Investigating the Efficacy and Safety of Intra-Amniotic ER004 Administration to Male Subjects with X-Linked Hypohidrotic Ectodermal Dysplasia. Genes (Basel). 2023 Jan 6;14(1):153. doi: 10.3390/genes14010153.

ReferenceCitation: Schneider H, Faschingbauer F, Schuepbach-Mallepell S, Korber 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.

repository_institution: Université de Lausanne - SERVAL

ReferenceCitation: Schneider H, Schweikl C, Faschingbauer F, Hadj-Rabia S, Schneider P. A Causal Treatment for X-Linked Hypohidrotic Ectodermal Dysplasia: Long-Term Results of Short-Term Perinatal Ectodysplasin A1 Replacement. Int J Mol Sci. 2023 Apr 12;24(8):7155. doi: 10.3390/ijms24087155.

repository_institution: Kooperativer Bibliotheksverbund Berlin-Brandenburg - OPUS Repository

publisher_dissemination: MDPI

lead_sponsor: EspeRare Foundation

collaborators:
Pierre Fabre Medicament
Iqvia Pty Ltd

sponsor_collaborators:
EspeRare Foundation
Pierre Fabre Medicament
Iqvia Pty Ltd

location_country:
Germany
Italy
United Kingdom
United States
Spain
France

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

location_facility: Hospital Universitario Virgen de la Arrixaca


type: EudraCT Number

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:
United Kingdom
Denmark
Korea, Republic of
Spain
France

location_facility: Guys and St Thomas Hospital; OHCT Clinical Trials

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Clinica Universidad de Navarra Madrid; Servicio de Oncología

location_facility: Christie Hospital NHS Trust; Experimental Cancer Medicine Team

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

location_facility: START Madrid-FJD, Hospital Fundacion Jimenez Diaz

location_facility: Western General Hospital; Edinburgh Cancer Center


title: Correlate - Safety and Effectiveness of Regorafenib in Routine Clinical Practice Settings

acronym: Correlate

summary: This study is a prospective observational cohort study. The study will be conducted in routine clinical practice settings. It is planned to enroll 1000 patients with metastatic Colorectal Cancer (mCRC) who have been previously treated with other approved treatments for metastatic disease and for whom a decision has been made by the physician to treat with regorafenib according to local health authority approved label. It is the aim of this observational cohort study to further characterize safety and effectiveness of regorafenib in routine clinical practice setting. Healthcare resource utilization in the routine provision of care is becoming increasingly important from a health economics and outcomes research perspective. Therefore, another aim of this observational cohort study is to capture healthcare resource associated with the management of treatment emergent adverse events in the real world setting. The primary objective of this study is to further characterize safety of regorafenib use in routine clinical practice settings. The secondary objective of this study is to assess the effectiveness of regorafenib in routine clinical practice settings as measured by Overall Survival (OS), Progression Free Survival (PFS) and Disease control rate (DCR). Health Related Quality of Life (HRQoL) Data will be collected in applicable countries.

ReferenceCitation: Yeh KH, Yang TS, Hsu TC, Tzu-Liang Chen W, Chen HH, Teng HW, Lin BW, Kuan FC, Chiang FF, Duann CW, Li YS, Lin MT, Fiala-Buskies S, Ducreux M, Wang JY. Real-world evidence of the safety and effectiveness of regorafenib in Taiwanese patients with metastatic colorectal cancer: CORRELATE Taiwan. J Formos Med Assoc. 2021 Nov;120(11):2023-2031. doi: 10.1016/j.jfma.2020.12.015. Epub 2021 Jan 7.

oa_host_type: publisher

ReferenceCitation: Ducreux M, Petersen LN, Ohler L, Bergamo F, Metges JP, de Groot JW, Wang JY, Garcia Paredes B, Dochy E, Fiala-Buskies S, Cervantes A, O'Connor JM, Falcone A; CORRELATE Investigators. Safety and effectiveness of regorafenib in patients with metastatic colorectal cancer in routine clinical practice in the prospective, observational CORRELATE study. Eur J Cancer. 2019 Dec;123:146-154. doi: 10.1016/j.ejca.2019.09.015. Epub 2019 Nov 4.

repository_institution: INFN Catania - INFM-OAR

repository_normalized: Zenodo

repositories_concat: Zenodo

lead_sponsor: Bayer

location_country: Italy Argentina Denmark Singapore Netherlands Austria Mexico Switzerland Spain Luxembourg Taiwan Czechia France


title: European TauroPace Registry - University of Schleswig-Holstein Prospective Observational Clinical Study

acronym: ETPR

summary: University of Schleswig-Holstein Campus Kiel is sponsoring the European TauroPace Registry. This investigator-initiated local, prospective, non-interventional, multi-center study includes patients undergoing cardiac implantable electronic placement including and not limited to placement, revision with the aim to upgrade or downgrade, generator substitution, additional lead placement, revision or extraction (procedure) with adjunct TauroPace. It is planned to include a total of at least 2300 procedures (valid for safety analysis).

ipd_sharing_description: participant data is recorded in a pseudonymous manner in the centre, before statistical analysis data is anonymized

lead_sponsor: University of Kiel

sponsor_collaborators:
University of Kiel
University of Luebeck

location_country:
Germany
Italy
United Kingdom
Austria
France

location_facility: Great Western Hospitals NHS Foundation Trust

lead_sponsor_type: academique


title: European Geriatric Emergency Departments Registry Study

acronym: EGERS

summary: Geriatric presentations to emergency services comprise a significant percentage of all emergency service presentations in Europe and it has been reported that 3-23% of all emergency service presentations from various regions of the countries. There are specific management practices for patients who are 65 years and older at emergency services. On the other hand several risk-scoring systems have been developed to define the severity class of the patient during their initial evaluation at emergency services.Only a few studies in the literature have evaluated risk-scoring systems for the geriatric patient group.Several studies have reported that risk-scoring systems, such as Identification of Seniors at Risk and Triage Risk Screening Tool, which are specifically developed for geriatric patients over 65 years who present to emergency services, are not sufficiently effective for evaluating patients in more severe conditions. Previously the TEDGeS (Turkish Emergency Departments Geriatric Scoring Study) pilot study was carried out and published by some of the investigators of this project.This pilot study enrolled all geriatric patients (age ≥ 65 years) and carried out in 13 centers from different cities of Turkey. This pilot study showed that geriatric patients not only constitute significant proportion of emergency department presentations but also these patients need more hospitalization. The predictive powers of the Modified Early Warning Score, Rapid Emergency Medicine Score and The Vital PAC Early Warning Score for hospitalization and mortality in geriatric patients those presented to emergency department are significantly high and might be concerned in the emergency department triage of these patients. Within the light of these pilot study results, the investigators have decided to execute this prospective, multinational, multicentric study with the main objective to determine the epidemiological and age related characteristics of geriatric patients presenting to the emergency department across Europe and evaluate early warning scoring systems systems regarding hospitalization, ICU admission and in-hospital mortality for geriatric patients.

status: Unknown status

ReferenceCitation: Dundar ZD, Ergin M, Karamercan MA, Ayranci K, Colak T, Tuncar A, Cander B, Gul M. Modified Early Warning Score and VitalPac Early Warning Score in geriatric patients admitted to emergency department. Eur J Emerg Med. 2016 Dec;23(6):406-412. doi: 10.1097/MEJ.0000000000000274.

ReferenceCitation: Cei M, Bartolomei C, Mumoli N. In-hospital mortality and morbidity of elderly medical patients can be predicted at admission by the Modified Early Warning Score: a prospective study. Int J Clin Pract. 2009 Apr;63(4):591-5. doi: 10.1111/j.1742-1241.2008.01986.x. Epub 2009 Feb 11.

ReferenceCitation: Dundar ZD, Ayranci MK. Presenting symptoms of older emergency department patients: a single-center experience of 10,692 patients in Turkey. Acta Clin Belg. 2020 Dec;75(6):405-410. doi: 10.1080/17843286.2019.1655215. Epub 2019 Aug 12.

ReferenceCitation: Lamantia MA, Stewart PW, Platts-Mills TF, Biese KJ, Forbach C, Zamora E, McCall BK, Shofer FS, Cairns CB, Busby-Whitehead J, Kizer JS. Predictive value of initial triage vital signs for critically ill older adults. West J Emerg Med. 2013 Sep;14(5):453-60. doi: 10.5811/westjem.2013.5.13411.

ReferenceCitation: Buurman BM, van den Berg W, Korevaar JC, Milisen K, de Haan RJ, de Rooij SE. Risk for poor outcomes in older patients discharged from an emergency department: feasibility of four screening instruments. Eur J Emerg Med. 2011 Aug;18(4):215-20. doi: 10.1097/MEJ.0b013e328344597e.

ReferenceCitation: Pines JM, Mullins PM, Cooper JK, Feng LB, Roth KE. National trends in emergency department use, care patterns, and quality of care of older adults in the United States. J Am Geriatr Soc. 2013 Jan;61(1):12-7. doi: 10.1111/jgs.12072.

ReferenceCitation: Platts-Mills TF, Travers D, Biese K, McCall B, Kizer S, LaMantia M, Busby-Whitehead J, Cairns CB. Accuracy of the Emergency Severity Index triage instrument for identifying elder emergency department patients receiving an immediate life-saving intervention. Acad Emerg Med. 2010 Mar;17(3):238-43. doi: 10.1111/j.1553-2712.2010.00670.x.

ReferenceCitation: Subbe CP, Kruger M, Rutherford P, Gemmel L. Validation of a modified Early Warning Score in medical admissions. QJM. 2001 Oct;94(10):521-6. doi: 10.1093/qjmed/94.10.521.

ReferenceCitation: Wheeler I, Price C, Sitch A, Banda P, Kellett J, Nyirenda M, Rylance J. Early warning scores generated in developed healthcare settings are not sufficient at predicting early mortality in Blantyre, Malawi: a prospective cohort study. PLoS One. 2013;8(3):e59830. doi: 10.1371/journal.pone.0059830. Epub 2013 Mar 29. Erratum In: PLoS One. 2014;9(2):e91623.

lead_sponsor: Gazi University

collaborators: European Society for Emergency Medicine (EUSEM) Research Network

location_country: Germany Ireland Greece Croatia Turkey France

location_facility: Necmettin Erbakan University Meram Medical Faculty


title: Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5 and WP6) Part 2: a Neurobehavioural and MRI Study

summary: The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.

lead_sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

collaborators:
Newcastle-upon-Tyne Hospitals NHS Trust
Leiden University Medical Center
Stichting Kempenhaeghe
Region Hovedstadens Apotek
Institut Necker Enfants Malades
Catholic University of the Sacred Heart
Università degli Studi di Ferrara
Universidad Complutense de Madrid

location_country:
Italy
United Kingdom
Denmark
Netherlands
Spain
France

location_facility: STICHTING KEMPENHAEGHE/Leiden University Medical Center

location_facility: Imagine Institut Des Maladies Genetiques Necker Enfants Malades Fondation


title: Brain INvolvement in Dystrophinopathies (BIND): Deep Functional Phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Patients (WP5) Part 1: a Multicentre Online Phenotyping and Neurobehavioural Data Collection Study

summary: The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.

ipd_sharing: Undecided

ipd_sharing_description: Pseudonymised data will be shared amongst collaborating partners in the project, but details on data sharing are still under discussion.

sponsor_collaborators: Great Ormond Street Hospital for Children NHS Foundation Trust


title: Comprehensive Long-term Follow up of Adults With Arterial Switch Operation - European Collaboration for Prospective Outcome Research in Congenital Heart Disease

acronym: EPOCH-ASO

summary:
Background: Long-term outcomes in adults with prior arterial switch operation (ASO) have not yet been well defined. The aim of this study is to elucidate incidence and predictors of adverse cardiac outcomes in a prospectively followed cohort of adults after their ASO.

Methods: The comprehensive long-term follow up of adults with ASO is a project within the European collaboration for prospective outcome research in congenital heart disease (EPOCH). It is designed as a prospective, international multicenter cohort study. Consecutive patients (aged 16 years or more) with prior ASO will be included at 11 European tertiary care centers. Participants will be followed according to a standardized protocol following international recommendations, including standardized protocols for imaging and for exercise testing. Main outcome measures are all-cause and cardiac-related mortality, rate of cardiac re-intervention, neo-aortic dissection, myocardial infarction, stroke, infective endocarditis, sustained atrial and ventricular arrhythmias, new-onset or worsening pulmonary hypertension and new-onset heart failure. Secondary endpoints are frequency and progression of right ventricular outflow tract stenosis, neo-aortic root dilatation, neo-aortic valve regurgitation and ventricular dysfunction. The impact of demographic, anatomic (e.g. coronary artery anatomy) and functional variables on the above-mentioned outcomes, as well as quality of life and incidence of pregnancy related complications will also be assessed.

Aim: The prospective, international, multicenter EPOCH-ASO study will provide a better understanding of adverse outcomes and their predictors in adults after ASO. The results of the EPOCH-ASO study may help to optimize future care of this novel patient cohort in adult cardiology.

status: Active, not recruiting

lead_sponsor: University of Zurich

location_country: Netherlands Austria Switzerland Spain France

location_facility: Hospital Universitario y Politécnico La Fe Valencia

location_facility: CHU Paris IdF Ouest - HEGP Hôpital Européen Georges Pompidou


title: A Randomised, Double-blind, Placebo-controlled, Multi-national, Phase IIIb Study to Assess the Sustained Clinical Effect and Safety of Sublingual Immunotherapy Administered as Birch Pollen Extract Solution at a Dose of 300 IR Once Daily to Patients Suffering From Birch Pollen-induced Rhinoconjunctivitis

summary: The purpose of this 2-year study is to assess the sustained clinical efficacy and safety of 300 IR/day of a sublingual solution of birch pollen allergen extract starting 4 months prior to the birch pollen season and continuing over the birch pollen season compared with placebo for reduction of rhinoconjunctivitis-related symptoms and anti-allergy medication usage.

ReferenceCitation: Worm M, Rak S, de Blay F, Malling HJ, Melac M, Cadic V, Zeldin RK. Sustained efficacy and safety of a 300IR daily dose of a sublingual solution of birch pollen allergen extract in adults with allergic rhinoconjunctivitis: results of a double-blind, placebo-controlled study. Clin Transl Allergy. 2014 Feb 11;4(1):7. doi: 10.1186/2045-7022-4-7.

publisher_dissemination: Wiley

lead_sponsor: Stallergenes Greer

sponsor_collaborators:
Stallergenes Greer
Quintiles, Inc.
Aptuit
Cenduit LLC
PHT Corporation

location_country:
Finland
Germany
Lithuania
Poland
Slovakia
Denmark
Sweden
Latvia
Estonia
Czech Republic
France

location_facility: Universitätsmedizin Berlin - Allergie-Centrum-Charité

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny Nr 1im.N.Barlickiego w Uniwersystetu Medycznego w Łodzi

location_facility: Sahlgrenska Universitetssjukhuset - Avd för Lungmedicin och Allergi

location_facility: Centre of Investigations and Treatment of Allergic Diseases

location_facility: National University Hospital - Allergy Unit 4222

location_facility: NHC, Hôpitaux Universitaires de Strasbourg

location_facility: Allergic Diseases Diagnostics and Treatment Centre


title: A Phase III Single Arm Study to Evaluate the Efficacy, Safety and Local Tolerability of a Subcutaneous 3-month Formulation of Triptorelin Pamoate (11.25 mg) in Patients With Locally Advanced or Metastatic Prostate Cancer

acronym: DKP 3M SC

summary: Assess the efficacy and safety of Triptorelin pamoate 3M formulation (11.25mg) when administered by subcutaneous route.

lead_sponsor: Ipsen

location_country: Poland Romania Latvia Bulgaria France


title: A Multicenter, Open-label, Dose Escalation, Phase I Study of LJM716 Administered Intravenously in Combination With Trastuzumab in Patients With HER2 Overexpressing Metastatic Breast Cancer or Gastric Cancer

summary: This is a multicenter, open-label, dose escalation, phase I study to estimate the Maximum Tolerated Dose (MTD) or a lower Recommended Dose for Expansion (RDE) of LJM716 in combination with trastuzumab in patients with Human Epidermal growth factor Receptor 2 (HER2) overexpressing Metastatic Breast Cancer (MBC) or gastric cancer (MGC). The study consists of a dose escalation part and a dose expansion part. LJM716 will be administered intravenously once weekly unless a less frequent dosing regimen such as every 2 weeks or once every 4 weeks is introduced. Patients will continue on their trastuzumab dosing, administered intravenously once weekly at 2mg/kg. During dose escalation, a minimum of 15 patients are anticipated to be treated in successive cohorts. The dose escalation will continue until the MTD/RDE is declared. The RDE dose selected will either be the MTD or a dose below the MTD based on safety and Pharmacokinetic/Pharmacodynamic (PK/PD) considerations. Following the MTD/RDE declaration, approximately 20 MBC and 20 MGC patients will be enrolled in separate arms in the dose expansion part and treated at the MTD/RDE to further assess the safety, tolerability, and anti-tumor activity of the combination.

lead_sponsor: Novartis Pharmaceuticals

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


title: Efficacy of the Combination of Simvastatin Plus Rifaximin in Patients With Decompensated Cirrhosis to Prevent ACLF Development: a Multicenter, Double-blind, Placebo Controlled Randomized Clinical Trial

summary: The aim of this study is to assess the efficacy of oral administration of simvastatin plus rifaximin in patients with decompensated cirrhosis to halt the progression of the disease as assessed by prevention of the development of ACLF

ReferenceCitation: Marrache MK, Rockey DC. Statins for treatment of chronic liver disease. Curr Opin Gastroenterol. 2021 May 1;37(3):200-207. doi: 10.1097/MOG.0000000000000716.

oa_host_type: repository

unpaywall_oa_status: green

publisher_dissemination: Wolters Kluwer Health

lead_sponsor: Judit Pich

location_country:
Germany
Italy
United Kingdom
Netherlands
Belgium
Spain
France

location_facility:
Hospital Clínic de Barcelona
Bologna University Hospital
Academic Medical Centre
Hospital Gregorio Marañón
Universitatsklinikum Frankurt
San Giovanni Battista Hospital
Hospital Moisés Broggi
Hospital Parc Taulí
Royal Free Hospital
Beajuon Hospital
Hospital de Sant Pau
University Hospitals Leuven
Padova University Hospital
Hospital Universitari Vall d'Hebrón


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, Rodriguez de Rivera Garrido FJ, Schols 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.

oa_colors: green diamond

repository_institution: Imperial College London - Spiral

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories:
LIRIAS (KU Leuven)
PubMed Central
UCL Discovery
spiral.imperial.ac.uk

repositories_concat: LIRIAS (KU Leuven);PubMed Central;UCL Discovery;spiral.imperial.ac.uk

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

sponsor_collaborators:
RWTH Aachen University
Assistance Publique - Hôpitaux de Paris

location_country:
Germany
Italy
United Kingdom
Austria
Spain
France

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: Service de génétique médicale - Hôpital La Pitié Salpetrière


title: Prospective, Multicenter, Single Arm Feasibility and Safety Study of the Endologix Fenestrated Stent Graft System for the Endovascular Repair of Juxtarenal/Pararenal (JAA/PAA) Aneurysms

summary: The aim of this study is to evaluate the safety and feasibility of the Endologix Fenestrated Stent Graft System for the endovascular repair of juxtarenal or pararenal aortic aneurysms.

ReferenceCitation: Mertens R, Bergoeing M, Marine L, Valdes F, Kramer A, Vergara J. Ventana fenestrated stent-graft system for endovascular repair of juxtarenal aortic aneurysms. J Endovasc Ther. 2012 Apr;19(2):173-8. doi: 10.1583/11-3706.1.

lead_sponsor: Endologix

location_country:
New Zealand
United Kingdom
Netherlands
United States
Chile
France

location_facility:
Hospital Universidad Catolica
UCLA
Bureau de Recherche Clinique
University of Indiana
Auckland City Hospital
St. George's Vascular Institute
Rijnstate Hospital
Cleveland Clinic Foundation


title: Prognostic Imaging Biomarkers for Diabetic Kidney Disease

acronym: iBEAt

summary:
Diabetic kidney disease (DKD) is a common complication of diabetes, and is now the most common form of chronic kidney disease. DKD is the leading cause of kidney disease requiring dialysis or kidney transplantation, and its global incidence and prevalence have reached epidemic levels. While the risk of developing DKD can be ameliorated by tight blood glucose and blood pressure control, it is not fully preventable and once established DKD cannot be cured. Therefore many patients are left with poor and worsening health and with increased mortality risk. Developing new ways to treat DKD requires healthcare professionals to be able to identify those patients most in need of treatment.

One promising approach for identifying patients that are at risk is the use of imaging measurements (called "biomarkers") derived from Magnetic Resonance Imaging (MRI) and Ultrasound (US) of the kidneys. Evidence from early studies shows that such imaging biomarkers can identify underlying problems in DKD such as blood supply, oxygen supply, kidney scarring and kidney function, in ways that are better than those currently available.

The investigators think that imaging biomarkers will improve the identification of patients who are likely to decline from DKD in the short term. The changes found by imaging may even happen before effects on the blood and urine.

The investigators plan to test this hypothesis by performing a study observing 500 patients with early stage DKD, recruited in 5 sites across Europe. All patients will have detailed assessment at the start of their involvement, including clinical assessment, blood and urine samples, and MRI and US scans. The investigators will look at whether imaging biomarkers are associated with other measures that predict progression in DKD, and follow patients every year for 3 years (4 years total study participation) to see if the imaging biomarkers predict worsening DKD.

ReferenceCitation: Grenier N, Merville P, Combe C. Radiologic imaging of the renal parenchyma structure and function. Nat Rev Nephrol. 2016 Jun;12(6):348-59. doi: 10.1038/nrneph.2016.44. Epub 2016 Apr 12.

ReferenceCitation: Gooding KM, Lienczewski C, Papale M, Koivuviita N, Maziarz M, Dutius Andersson AM, Sharma K, Pontrelli P, Garcia Hernandez A, Bailey J, Tobin K, Saunavaara V, Zetterqvist A, Shelley D, Teh I, Ball C, Puppala S, Ibberson M, Karihaloo A, Metsarinne K, Banks RE, Gilmour PS, Mansfield M, Gilchrist M, de Zeeuw D, Heerspink HJL, Nuutila P, Kretzler M, Welberry Smith M, Gesualdo L, Andress D, Grenier N, Shore AC, Gomez MF, Sourbron S; BEAt-DKD consortium. Prognostic imaging biomarkers for diabetic kidney disease (iBEAt): study protocol. BMC Nephrol. 2020 Jun 29;21(1):242. doi: 10.1186/s12882-020-01901-x.

repository_institution: University of Michigan - Deep Blue

repository_institution: Royal Devon and Exeter - Royal Devon and Exeter Research Repository

repository_institution: Cold Spring Harbor Laboratory - medRxiv

repository_normalized: medRxiv

version: submittedVersion

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

lead_sponsor: University of Leeds

sponsor_collaborators:
University of Leeds
University of Exeter
University Hospital, Bordeaux
University of Bari
University of Turku
Lund University
Swiss Institute of Bioinformatics

location_country:
Finland
Italy
France
United Kingdom
Switzerland
Sweden

location_facility: Centre Hospitalier Universitaire de Bordeaux

location_facility: Università degli Studi di Bari Aldo Moro

intervention_type: Procedure


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:
Germany
United Kingdom
Denmark
Netherlands
Spain
France

location_facility: Hospital Universitario Central de Asturias

location_facility: Vall d'Hebron University Hospital - Barcelona


title: Randomized Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) Versus Expectant Management During Pregnancy in Fetuses With Left Sided and Isolated Congenital Diaphragma Hernia and Severe Pulmonary Hypoplasia.

acronym: TOTAL

summary: This trial investigates whether prenatal intervention improves survival rate of fetuses with isolated congenital diaphragmatic hernia and severe pulmonary hypoplasia, as compared to expectant management during pregnancy, both followed by standardized postnatal care.

ReferenceCitation: Jani JC, Nicolaides KH, Gratacos E, Valencia CM, Done E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.

ReferenceCitation: Deprest J, Breysem L, Gratacos E, Nicolaides K, Claus F, Debeer A, Smet MH, Proesmans M, Fayoux P, Storme L. Tracheal side effects following fetal endoscopic tracheal occlusion for severe congenital diaphragmatic hernia. Pediatr Radiol. 2010 May;40(5):670-3. doi: 10.1007/s00247-010-1579-9. Epub 2010 Mar 30. No abstract available.

ReferenceCitation: Deprest JA, Gratacos E, Nicolaides K, Done E, Van Mieghem T, Gucciardo L, Claus F, Debeer A, Allegaert K, Reiss I, Tibboel D. Changing perspectives on the perinatal management of isolated congenital diaphragmatic hernia in Europe. Clin Perinatol. 2009 Jun;36(2):329-47, ix. doi: 10.1016/j.clp.2009.03.004.

ReferenceCitation: Deprest JA, Hyett JA, Flake AW, Nicolaides K, Gratacos E. Current controversies in prenatal diagnosis 4: Should fetal surgery be done in all cases of severe diaphragmatic hernia? Prenat Diagn. 2009 Jan;29(1):15-9. doi: 10.1002/pd.2108. No abstract available.

ReferenceCitation: Deprest JA, Flemmer AW, Gratacos E, Nicolaides K. Antenatal prediction of lung volume and in-utero treatment by fetal endoscopic tracheal occlusion in severe isolated congenital diaphragmatic hernia. Semin Fetal Neonatal Med. 2009 Feb;14(1):8-13. doi: 10.1016/j.siny.2008.08.010. Epub 2008 Oct 8.

ReferenceCitation: Jani JC, Benachi A, Nicolaides KH, Allegaert K, Gratacos E, Mazkereth R, Matis J, Tibboel D, Van Heijst A, Storme L, Rousseau V, Greenough A, Deprest JA; Antenatal-CDH-Registry group. Prenatal prediction of neonatal morbidity in survivors with congenital diaphragmatic hernia: a multicenter study. Ultrasound Obstet Gynecol. 2009 Jan;33(1):64-9. doi: 10.1002/uog.6141.

ReferenceCitation: Jani J, Nicolaides KH, Keller RL, Benachi A, Peralta CF, Favre R, Moreno O, Tibboel D, Lipitz S, Eggink A, Vaast P, Allegaert K, Harrison M, Deprest J; Antenatal-CDH-Registry Group. Observed to expected lung area to head circumference ratio in the prediction of survival in fetuses with isolated diaphragmatic hernia. Ultrasound Obstet Gynecol. 2007 Jul;30(1):67-71. doi: 10.1002/uog.4052.

ReferenceCitation: Jani J, Keller RL, Benachi A, Nicolaides KH, Favre R, Gratacos E, Laudy J, Eisenberg V, Eggink A, Vaast P, Deprest J; Antenatal-CDH-Registry Group. Prenatal prediction of survival in isolated left-sided diaphragmatic hernia. Ultrasound Obstet Gynecol. 2006 Jan;27(1):18-22. doi: 10.1002/uog.2688.

ReferenceCitation: Deprest J, Gratacos E, Nicolaides KH; FETO Task Group. Fetoscopic tracheal occlusion (FETO) for severe congenital diaphragmatic hernia: evolution of a technique and preliminary results. Ultrasound Obstet Gynecol. 2004 Aug;24(2):121-6. doi: 10.1002/uog.1711. Erratum In: Ultrasound Obstet Gynecol. 2004 Oct;24(5):594.

ReferenceCitation: Rodrigues HC, Deprest J, v d Berg PP. When referring physicians and researchers disagree on equipoise: the TOTAL trial experience. Prenat Diagn. 2011 Jun;31(6):589-94. doi: 10.1002/pd.2756. Epub 2011 Apr 11.

ReferenceCitation: Deprest J, Flake A. How should fetal surgery for congenital diaphragmatic hernia be implemented in the post-TOTAL trial era: A discussion. Prenat Diagn. 2022 Mar;42(3):301-309. doi: 10.1002/pd.6091. Epub 2022 Jan 22.

ReferenceCitation: Van Calster B, Benachi A, Nicolaides KH, Gratacos E, Berg C, Persico N, Gardener GJ, Belfort M, Ville Y, Ryan G, Johnson A, Sago H, Kosinski P, Bagolan P, Van Mieghem T, DeKoninck PLJ, Russo FM, Hooper SB, Deprest JA. The randomized Tracheal Occlusion To Accelerate Lung growth (TOTAL)-trials on fetal surgery for congenital diaphragmatic hernia: reanalysis using pooled data. Am J Obstet Gynecol. 2022 Apr;226(4):560.e1-560.e24. doi: 10.1016/j.ajog.2021.11.1351. Epub 2021 Nov 19.

evidence: open (via free pdf)

ReferenceCitation: Deprest JA, Nicolaides KH, Benachi A, Gratacos E, Ryan G, Persico N, Sago H, Johnson A, Wielgos M, Berg C, Van Calster B, Russo FM; TOTAL Trial for Severe Hypoplasia Investigators. Randomized Trial of Fetal Surgery for Severe Left Diaphragmatic Hernia. N Engl J Med. 2021 Jul 8;385(2):107-118. doi: 10.1056/NEJMoa2027030. Epub 2021 Jun 8.

repository_institution: Radboud University - Radboud Repository

repository_institution: University of Zurich - Zurich Open Repository and Archive

lead_sponsor: University Hospital, Gasthuisberg

collaborators: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

collaborators: Ospedale Pediatrico Bambino Gesù, Rome (IT)

collaborators: National Center for Child Health and Development, Tokyo (JP)

collaborators: The University of Texas Health Science Center, Houston

location_country:
Germany
Italy
Poland
United Kingdom
Australia
Japan
Belgium
United States
Spain
Canada
France

location_facility: 1st Department of Obstetrics and Gynecology, Medical University of Warsaw


acronym: GLOW

lead_sponsor: AstraZeneca

summary: The purpose of this study is to assess the safety and effectiveness of AZD4547 in combination with fulvestrant vs. fulvestrant alone in ER+ breast cancer patients with FGFR1 polysomy (FISH4/5) or gene amplification (FISH 6)

title: A Randomised Double-blind Phase IIa Study (With Combination Safety Run-in) to Assess the Safety and Efficacy of AZD4547 in Combination With Fulvestrant vs. Fulvestrant Alone in ER+ Breast Cancer Patients With FGFR1 Polysomy or Gene Amplification Who Have Progressed Following Treatment With Prior Endocrine Therapy (Adjuvant or First-line Metastatic) (GLOW)

location_country:
Germany
Italy
Hungary
United Kingdom
Romania
Belgium
Czech Republic
France


title: GARDIAN, Gadovist in Routine Diagnostic Magnetic Resonance Imaging Administration in Non-selected Patients

acronym: GARDIAN

summary: Prospective, non-interventional, multi-center study. The observation period for each subject covers the treatment period with Gadovist®. For each patient, the treating physician or nurse documents demographics, medical data, safety parameters and treatment signs and symptoms at the visit. Patients with severe renal impairment will be followed-up after 3 month by phone call from the investigator if in line with routine practice. Data audit/monitoring by source data verification will be done in a subset of sites and patients

location_country:
Germany
Poland
Bosnia and Herzegovina
Thailand
Vietnam
Canada
Kyrgyzstan
Czech Republic
France
Hungary
Hong Kong
China
Italy
Korea, Republic of
Spain
Greece
South Africa
Taiwan
Russian Federation
Kazakhstan


acronym: IMPALA-X

lead_sponsor: Savara Inc.

summary:
Clinical trial for subjects with autoimmune pulmonary alveolar proteinosis (aPAP) who have completed the IMPALA trial (NCT02702180).

At the Baseline visit, eligible subjects may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.

Subject will be treated with inhaled molgramostim for up to 36 months.

During the trial, whole lung lavage will be applied as rescue therapy.

location_facility: CHU Rennes Hospital Pontchaillou, Service de Pneumologie

location_facility: Dept. Of Intensive Care Unit Royal Brompton Hospital London

location_facility: Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens

location_facility: Rabin Medical Center Institute of Pulomonary Medicine

location_facility: Asklepios Fachkliniken München - Gauting Klinik für Pneumologie

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin

location_facility: Pavlov first Saint Petersburg State Medical Univerisity

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

location_facility: Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH

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

location_facility: S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo

title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients

location_country:
Germany
France
Italy
United Kingdom
Denmark
Netherlands
Greece
Turkey
Russian Federation
Israel


acronym: SHIELD

summary: The purpose of this pivotal trial is to evaluate subcutaneous (SQ) AIN457 as an adjunctive therapy to reduce the rate of exacerbations of posterior uveitis or panuveitis secondary to Behçet's disease during the 24 weeks of study therapy as compared to standard of care alone.

ReferenceCitation: Dick AD, Tugal-Tutkun I, Foster S, Zierhut M, Melissa Liew SH, Bezlyak V, Androudi S. Secukinumab in the treatment of noninfectious uveitis: results of three randomized, controlled clinical trials. Ophthalmology. 2013 Apr;120(4):777-87. doi: 10.1016/j.ophtha.2012.09.040. Epub 2013 Jan 3.

title: A 24 Week Multicenter, Randomized, Double-masked, Placebo Controlled Study to Assess the Difference in the Rate of Recurrent Exacerbations in Behçet¿s Patients With Posterior or Panuveitis Treated With AIN457 vs Placebo Adjunctive to Standard-of-care Immunosuppressive Therapy

location_country:
Germany
Italy
Taiwan
India
Israel
Hong Kong
Singapore
Switzerland
Tunisia
United States
Korea, Republic of
Spain
Greece
Turkey
Jordan
Egypt
France


title: A Phase 1/2, Open-Label Study Of Bosutinib Administered In Combination With Capecitabine In Subjects With Solid Tumor And ErbB2 Negative Locally Advanced Or Metastatic Breast Cancer

summary:
This is a research study in 2 parts assessing the following parameters of the combination of the study drug called bosutinib, and a drug called capecitabine: the safety, how well the subject's body handles the study drug, and preliminary anti-tumor activity as treatment for different types of cancers in part 1, and breast cancer only in part 2.

In part 1, subjects will receive bosutinib and capecitabine daily at different dose levels of each drug in order to determine the highest tolerated dose of the combination study treatment. In part 2, subjects will receive bosutinib and capecitabine at this highest tolerated dose to see how well the study treatment works to treat breast cancer. In addition, genetic research testing (research analyses involving genes and gene products) will be performed on biological samples from subjects.

ReferenceCitation: Isakoff SJ, Wang D, Campone M, Calles A, Leip E, Turnbull K, Bardy-Bouxin N, Duvillie L, Calvo E. Bosutinib plus capecitabine for selected advanced solid tumours: results of a phase 1 dose-escalation study. Br J Cancer. 2014 Nov 25;111(11):2058-66. doi: 10.1038/bjc.2014.508. Epub 2014 Oct 7.

evidence: open (via page says license)

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: Springer

lead_sponsor: Pfizer

location_country:
Hong Kong
Australia
Belgium
United States
Spain
France


title: Treatment of Patients With Acute Sinusitis in Daily Practice

summary: For each patient, an initial visit and at least one follow-up visit at the end of treatment should be documented by the treating physician in the case report form.

ReferenceCitation: Mosges R, Desrosiers M, Arvis P, Heldner S. Characterisation of patients receiving moxifloxacin for acute bacterial rhinosinusitis in clinical practice: results from an international, observational cohort study. PLoS One. 2013 Apr 23;8(4):e61927. doi: 10.1371/journal.pone.0061927. Print 2013.

evidence: oa repository (semantic scholar lookup)

repositories_concat: PubMed Central;pdfs.semanticscholar.org

publisher_dissemination: Public Library of Science

lead_sponsor: Bayer

location_country:
Germany
Kuwait
Yemen
Pakistan
France
Bahrain
Indonesia
Romania
Lebanon
China
Malaysia
Philippines
Egypt
Singapore
Netherlands
Austria
United Arab Emirates
Saudi Arabia
Jordan


title: Computer-aided Diagnosis of Endoscopic Ultrasound Elastography Used in the Differentiation of Focal Pancreatic Masses

acronym: EUS-EG

summary: The aim of the study is to assess elastography during EUS examinations of focal pancreatic masses, and to consequently differentiate benign versus malignant pancreatic masses in a prospective multi-center design.

ReferenceCitation: Frey H. [Realtime elastography. A new ultrasound procedure for the reconstruction of tissue elasticity]. Radiologe. 2003 Oct;43(10):850-5. doi: 10.1007/s00117-003-0943-2. German.

ReferenceCitation: Ophir J, Alam SK, Garra B, Kallel F, Konofagou E, Krouskop T, Varghese T. Elastography: ultrasonic estimation and imaging of the elastic properties of tissues. Proc Inst Mech Eng H. 1999;213(3):203-33. doi: 10.1243/0954411991534933.

ReferenceCitation: Itoh A, Ueno E, Tohno E, Kamma H, Takahashi H, Shiina T, Yamakawa M, Matsumura T. Breast disease: clinical application of US elastography for diagnosis. Radiology. 2006 May;239(2):341-50. doi: 10.1148/radiol.2391041676. Epub 2006 Feb 16.

ReferenceCitation: Miyanaga N, Akaza H, Yamakawa M, Oikawa T, Sekido N, Hinotsu S, Kawai K, Shimazui T, Shiina T. Tissue elasticity imaging for diagnosis of prostate cancer: a preliminary report. Int J Urol. 2006 Dec;13(12):1514-8. doi: 10.1111/j.1442-2042.2006.01612.x.

ReferenceCitation: Rago T, Santini F, Scutari M, Pinchera A, Vitti P. Elastography: new developments in ultrasound for predicting malignancy in thyroid nodules. J Clin Endocrinol Metab. 2007 Aug;92(8):2917-22. doi: 10.1210/jc.2007-0641. Epub 2007 May 29.

ReferenceCitation: Janssen J, Schlorer E, Greiner L. EUS elastography of the pancreas: feasibility and pattern description of the normal pancreas, chronic pancreatitis, and focal pancreatic lesions. Gastrointest Endosc. 2007 Jun;65(7):971-8. doi: 10.1016/j.gie.2006.12.057.

ReferenceCitation: Saftoiu A, Vilmann P, Ciurea T, Popescu GL, Iordache A, Hassan H, Gorunescu F, Iordache S. Dynamic analysis of EUS used for the differentiation of benign and malignant lymph nodes. Gastrointest Endosc. 2007 Aug;66(2):291-300. doi: 10.1016/j.gie.2006.12.039.

ReferenceCitation: Saftoiu A, Vilmann P, Gorunescu F, Janssen J, Hocke M, Larsen M, Iglesias-Garcia J, Arcidiacono P, Will U, Giovannini M, Dietrich CF, Havre R, Gheorghe C, McKay C, Gheonea DI, Ciurea T; European EUS Elastography Multicentric Study Group. Efficacy of an artificial neural network-based approach to endoscopic ultrasound elastography in diagnosis of focal pancreatic masses. Clin Gastroenterol Hepatol. 2012 Jan;10(1):84-90.e1. doi: 10.1016/j.cgh.2011.09.014. Epub 2011 Oct 1.

ReferenceCitation: Saftoiu A, Vilmann P, Gorunescu F, Janssen J, Hocke M, Larsen M, Iglesias-Garcia J, Arcidiacono P, Will U, Giovannini M, Dietrich C, Havre R, Gheorghe C, McKay C, Gheonea DI, Ciurea T; European EUS Elastography Multicentric Study Group. Accuracy of endoscopic ultrasound elastography used for differential diagnosis of focal pancreatic masses: a multicenter study. Endoscopy. 2011 Jul;43(7):596-603. doi: 10.1055/s-0030-1256314. Epub 2011 Mar 24.

lead_sponsor: University of Medicine and Pharmacy Craiova

collaborators: University Hospital, Gentofte, Copenhagen

location_country:
Germany
Italy
Norway
United Kingdom
Denmark
Romania
Netherlands
Belgium
Spain
France

location_facility: HELIOS Klinikum Wuppertal, University of Witten/Herdecke


title: Phase II of Immunotherapy Plus Local Tumor Ablation (RFA or Stereotactic Radiotherapy) in Patients With Colorectal Cancer Liver Metastases

acronym: ILOC

summary:
This is a single-arm, open-label, multi-center early phase II study. This proof of concept study will investigate whether the combined use of local tumor ablation/radiation plus immunomodulating drugs may induce a significant immune response in patient with incurable liver metastases from colorectal cancer (CRC) (+/- limited extrahepatic disease) being stable or in partial remission after completion of 4-6 months first line systemic therapy.

The primary objective of the study is to show an overall response rate of lesions not treated by ablation/radiotherapy including the extrahepatic lesions (according to iRECIST criteria) higher than 10%. With the continuation of first line systemic treatment, no further responses are expected.

Secondary objectives are:

To establish the feasibility and safety of the combined treatment modalities;
To study the impact of the local technique (RFA/Radiotherapy) on the results;
To investigate biomarkers to predict response to the combined treatment

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

location_country: Germany France Netherlands Austria Switzerland Sweden

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Medical University Vienna - General Hospital AKH

location_facility: Karolinska University Hospital - Karolinska Institutet - Danderyds Hospital

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

location_facility: Radboud University Medical Center Nijmegen


title: An Open, Qualitative, Prospective, Multicenter Trial of a Novel Transanal Irrigation System in Spinal Cord Injured Patients.

summary: This study is designed as an open, prospective, non-controlled, qualitative, multicentre study of a novel transanal irrigation system performed in a population of 150 subjects suffering from spinal cord injury and confirmed neurological bowel dysfunction. The study is expected to last for a total of 1 year (treatment period) with a planned 12- month recruitment period and three scheduled site visits.

primary_purpose: Other

ReferenceCitation: Emmanuel A, Kurze I, Krogh K, Ferreiro Velasco ME, Christensen P, Del Popolo G, Bazzocchi G, Hultling C, Perrouin Verbe B, Bothig R, Glott T, Gonzalez Viejo MA. An open prospective study on the efficacy of Navina Smart, an electronic system for transanal irrigation, in neurogenic bowel dysfunction. PLoS One. 2021 Jan 29;16(1):e0245453. doi: 10.1371/journal.pone.0245453. eCollection 2021.

repositories:
PubMed Central
UCL Discovery

repositories_concat: PubMed Central;UCL Discovery

repositories_institution: University College London - UCL Discovery

lead_sponsor: Dentsply International

sponsor_collaborators:
Dentsply International
Wellspect HealthCare

location_country:
Germany
Italy
Norway
United Kingdom
Denmark
Sweden
Spain
France

location_facility: Unidad de Lesionados Medulares Hospital Traumatología

location_facility: Complejo Hospitalario Universitario A Coruña

location_facility: Neurologiska kliniken, Avd R18 Karolinska Universitetssjukhuset, Solna

location_facility: Montecatone Rehabilitation Institute, Università di Bologna

location_facility: Azienda Ospedaliera- Universitaria Careggi

location_facility: Gastrointestinal Physiology Unit, University College London Hospital


title: Diagnostic Accuracy of On-line Quantitative Flow Ratio. Functional Assessment by Virtual Online Reconstruction (The FAVOR II Europe-Japan Study)

acronym: FAVOR II EJ

summary: Quantitative Flow Ratio (QFR) is a novel method for evaluating the functional significance of coronary stenosis. QFR is assessed by calculation of the pressure in the vessel based on two angiographic projections. The purpose of the FAVOR II study is to evaluate the diagnostic accuracy of on-line QFR compared to 2D Quantitative Coronary Angiography (QCA) with FFR as gold standard.

ReferenceCitation: Tu S, Westra J, Yang J, von Birgelen C, Ferrara A, Pellicano M, Nef H, Tebaldi M, Murasato Y, Lansky A, Barbato E, van der Heijden LC, Reiber JHC, Holm NR, Wijns W; FAVOR Pilot Trial Study Group. Diagnostic Accuracy of Fast Computational Approaches to Derive Fractional Flow Reserve From Diagnostic Coronary Angiography: The International Multicenter FAVOR Pilot Study. JACC Cardiovasc Interv. 2016 Oct 10;9(19):2024-2035. doi: 10.1016/j.jcin.2016.07.013.

ReferenceCitation: Westra J, Andersen BK, Campo G, Matsuo H, Koltowski L, Eftekhari A, Liu T, Di Serafino L, Di Girolamo D, Escaned J, Nef H, Naber C, Barbierato M, Tu S, Neghabat O, Madsen M, Tebaldi M, Tanigaki T, Kochman J, Somi S, Esposito G, Mercone G, Mejia-Renteria H, Ronco F, Botker HE, Wijns W, Christiansen EH, Holm NR. Diagnostic Performance of In-Procedure Angiography-Derived Quantitative Flow Reserve Compared to Pressure-Derived Fractional Flow Reserve: The FAVOR II Europe-Japan Study. J Am Heart Assoc. 2018 Jul 6;7(14):e009603. doi: 10.1161/JAHA.118.009603.

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

repository_institution: Red Federada de Repositorios Institucionales de Publicaciones Científicas - LA Referencia

repository_normalized: dnet

lead_sponsor: Aarhus University Hospital Skejby

location_country:
Germany
Italy
United Kingdom
Denmark
Japan
Netherlands
France

location_facility: Azienda ospedaliera Sant'Anna e S. sebastiano di Caserta

location_facility: Institut Cardiovasculaire Paris Sud Massy

location_facility: Azienda Ospedaliero-Universitaria di Ferrara, University of Ferrara


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

summary:
Primary objective :

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

Secondary objectives:

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

lead_sponsor: Sanofi

location_country:
Montenegro
Netherlands
Croatia
Serbia
Bulgaria
Estonia
Czech Republic
France


title: An Open-Label, Multicenter, Phase 1 Study With Expansion Cohorts of Ramucirumab or Necitumumab in Combination With Osimertinib in Patients With Advanced T790M-Positive EGFR-Mutant Non-Small Cell Lung Cancer After Progression on First-Line EGFR TKI Therapy

summary: The main purpose of this study is to evaluate the safety of ramucirumab or necitumumab in combination with osimertinib in participants with non-small cell lung cancer (NSCLC).

ReferenceCitation: Yu HA, Paz-Ares LG, Yang JC, Lee KH, Garrido P, Park K, Kim JH, Lee DH, Mao H, Wijayawardana SR, Gao L, Hozak RR, Chao BH, Planchard D. Phase I Study of the Efficacy and Safety of Ramucirumab in Combination with Osimertinib in Advanced T790M-positive EGFR-mutant Non-small Cell Lung Cancer. Clin Cancer Res. 2021 Feb 15;27(4):992-1002. doi: 10.1158/1078-0432.CCR-20-1690. Epub 2020 Oct 12.

evidence: oa repository (via page says license)

repository_institution: University of Illinois at Chicago - INDIGO

publisher_dissemination: American Association for Cancer Research

lead_sponsor: Eli Lilly and Company

sponsor_collaborators:
Eli Lilly and Company
AstraZeneca

location_country:
United States
Spain
Korea, Republic of
Taiwan
France

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.


title: Watchman FLX Left Atrial Appendage Closure Device Post Approval Study

lead_sponsor: Boston Scientific Corporation

sponsor_collaborators:
Boston Scientific Corporation
Premier Research Group plc
Iqvia Pty Ltd

location_country:
Germany
Ireland
Italy
Poland
United Kingdom
Denmark
Netherlands
Spain
France

location_facility: Cardio Vasculares Centrum Sankt Katharinen

location_facility: Clinical Hospital University of Medicine


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

acronym: EUMDS

summary:
Study Objectives:

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

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

To disseminate results of the studies to all stakeholders involved.

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

license: cc-by-nc

license_normalized: cc-by-nc

repositories_institution:
PubMed Central - Europe PMC
Radboud University - Radboud Repository

publisher_dissemination: Ferrata Storti Foundation (Haematologica)

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

publisher_dissemination: American Society of Hematology

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

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

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

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

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

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

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

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

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

location_facility: Instituto Português de Oncologia de Lisboa,

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

location_facility: Leeds General Infirmary Dept of Hematology

location_facility: Radboud University Nijmegen Medical Centre, dept of Hematology

location_facility: Fundeni CLinical Institute, Clinic of Hematology

location_facility: University of Pavia Medical SChool, Dept of Hematology

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

location_facility: Hopital Avicenne Universite Paris, Dept of Hematology

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

location_facility: Institute of Haematology and Blood Transfusion U nemocnice


summary:
The primary objective is to estimate the Complete Response rate of docetaxel to the combination of cisplatin-5-fluorouracil (TCF) compared to cisplatin-5-fluorouracil (CF) in the Induction treatment of Nasopharyngeal Carcinoma (NPC).

The secondary objectives are to determine:

the safety of TCF in comparison to CF after induction treatment of NPC,
the pharmacokinetics of docetaxel when added to CF,
the Overall Response rate of TCF and CF on completion of induction and consolidation (chemo-radiotherapy) treatment of NPC, and to compare overall survival between TCF and CF.

location_facility:
Investigational Site Number 156005
Investigational Site Number 360001
Investigational Site Number 356003
Investigational Site Number 504003
Investigational Site Number 764002
Investigational Site Number 484001
Investigational Site Number 250001
Investigational Site Number 356004
Investigational Site Number 788003
Investigational Site Number 380001
Investigational Site Number 410002
Investigational Site Number 076002
Investigational Site Number 504002
Investigational Site Number 356002
Investigational Site Number 410003
Investigational Site Number 792003
Investigational Site Number 792001
Investigational Site Number 788002
Investigational Site Number 504001
Investigational Site Number 356001
Investigational Site Number 764001
Investigational Site Number 608002
Investigational Site Number 012001
Investigational Site Number 076001
Investigational Site Number 792002
Investigational Site Number 410001

title: International Randomized Study to Evaluate the Addition of Docetaxel to the Combination of Cisplatin-5-fluorouracil (TCF) vs. Cisplatin-5-fluorouracil (CF) in the Induction Treatment of Nasopharyngeal Carcinoma (NPC) in Children and Adolescents

location_country:
Italy
India
Algeria
Brazil
Tunisia
Mexico
Korea, Republic of
China
Philippines
Thailand
Turkey
Morocco
Indonesia
France


acronym: ALFACHIN

summary:
The primary objective of the study was to evaluate the efficacy of Alfuzosin in comparison to Placebo on the detrusor Leak Point Pressure (LPP) in children and adolescents 2-16 years of age with elevated detrusor LPP of neuropathic etiology and detrusor LPP ≥ 40 cm H2O.

Secondary objectives were:

To investigate the safety and tolerability of two doses of Alfuzosin in comparison to Placebo in children and adolescents,

To evaluate the effects of the two doses of Alfuzosin in comparison to Placebo on:

Detrusor compliance,
Urinary tract infection,
To investigate the pharmacokinetics of Alfuzosin (population kinetics),
To evaluate the 12-month long-term safety of Alfuzosin 0.1 mg/kg/day and 0.2 mg/kg/day.

The study consisted of 2 periods:

a 12-week double blind treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day or placebo then,
a 40-week open label extension treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day.

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

title: 12-week, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Investigate the Efficacy, Pharmacodynamic and Safety of 2 Doses of Alfuzosin (0.1 mg/kg/Day, 0.2 mg/kg/Day) in the Treatment of Children and Adolescents 2-16 Years With Elevated Detrusor Leak Point Pressure of Neuropathic Etiology Followed by a 40-week Open-label Extension

location_country:
Germany
Poland
Russian Federation
India
Portugal
Slovakia
Singapore
Estonia
United States
Spain
Malaysia
Turkey
Taiwan
Bulgaria
Canada
Serbia
Philippines
France


title: Efficacy and Safety of 2 mg/Day M100907 on Sleep Maintenance Insomnia: a 6-week, Multicenter, Randomized, Double-blind, Placebo-controlled Polysomnographic Study

summary: The purpose of this study is to assess efficacy and safety of volinanserin in the population of patients complaining of sleep maintenance insomnia. The patients suffering from that condition frequently wake up during the night, their sleep is non restorative and they suffer from a significant distress or impairment in their daily activities consecutive to insomnia.

location_country:
Germany
Australia
Austria
United States
Canada
Russian Federation
France


title: Treatment Protocol for Relapsed Anaplastic Large Cell Lymphoma of Childhood and Adolescence

summary:
RATIONALE: Giving combination chemotherapy and total-body irradiation before a peripheral stem cell transplant that uses the patient's or a donor's stem cells, helps stop both the growth of cancer cells and the patient's immune system from rejecting the stem cells. When the stem cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving combination chemotherapy and total-body irradiation followed by a stem cell transplant may be an effective treatment for anaplastic large cell lymphoma.

PURPOSE: This clinical trial is studying how well combination chemotherapy followed by stem cell transplant works in treating young patients with progressive or relapsed anaplastic large cell lymphoma.

ReferenceCitation: Knorr F, Brugieres L, Pillon M, Zimmermann M, Ruf S, Attarbaschi A, Mellgren K, Burke GAA, Uyttebroeck A, Wrobel G, Beishuizen A, Aladjidi N, Reiter A, Woessmann W; European Inter-Group for Childhood Non-Hodgkin Lymphoma. Stem Cell Transplantation and Vinblastine Monotherapy for Relapsed Pediatric Anaplastic Large Cell Lymphoma: Results of the International, Prospective ALCL-Relapse Trial. J Clin Oncol. 2020 Dec 1;38(34):3999-4009. doi: 10.1200/JCO.20.00157. Epub 2020 Jul 30.

lead_sponsor: European Inter-group Cooperation on Childhood and Adolescent Non Hodgkin Lymphoma

location_country:
Germany
Ireland
Italy
Poland
United Kingdom
Sweden
Netherlands
Austria
Belgium
Switzerland
Czech Republic
France

location_facility: Helios Kliniken Wuppertal University Hospital

location_facility: Charite University Hospital - Campus Virchow Klinikum

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Institute of Child Health at University of Bristol

location_facility: Krankenanstalt Mutterhaus der Borromaerinnen

location_facility: Klinik und Poliklinik Fuer Kinder-und Jugendmedizin - Universitaetsklinikum Greifswald

location_facility: Universitaetskinderklinik - Universitaetsklinikum Freiburg

location_facility: Kinderklinik - Universitaetsklinikum Rostock

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Klinik und Poliklinik fuer Kinder und Jugendmedizin - Universitaetsklinikum Muenster

location_facility: Comprehensive Cancer Center Ulm at Universitaetsklinikum Ulm

location_facility: Kinderklinik - Universitaetsklinikum Aachen

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Kliniken der Stadt Koeln gGmbH - Kinderkrankenhaus Riehl

location_facility: University Hospital Schleswig-Holstein - Kiel Campus

location_facility: Dr. von Haunersches Kinderspital der Universitaet Muenchen

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

location_facility: University Medical Center Hamburg - Eppendorf

location_facility: Great Ormond Street Hospital for Children

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

location_facility: Kinderklink Siegen Deutsches Rotes Kreuz


title: A Randomized, Double Blind, Two-arm Placebo Controlled, 12-Month Study of the Effects of Rimonabant 20mg Once Daily on the Amount and the Activity of Visceral Fat in Abdominally Obese Patients With Metabolic Syndrome.

acronym: VICTORIA

summary:
Primary objective:

To assess the effect of rimonabant on visceral fat area over a period of 12 months when prescribed with a mild hypocaloric diet in abdominally obese patients with metabolic syndrome

Secondary objectives:

To assess the effect of rimonabant over a period of 12 months on:

Liver fat content using CT scan (Computed Tomography scan)
Anthropometric measures (weight, waist circumference, body composition using Dual Energy X-ray Absorptiometry (DEXA))
Lipid, lipoprotein profile
Glycemia, insulinemia and HbA1c
Adipokines, inflammatory and hemostatic markers
To evaluate the percentage of patients with metabolic syndrome at 12 months
To evaluate the safety and tolerability of rimonabant in these patients

In four selected US sites the effect of rimonabant at 12 months will be also assessed on:

Basal lipolysis and insulin suppressed lipolysis (euglycemic hyperinsulinemic clamp).
Resting metabolic rate and substrate oxidation at rest using indirect calorimetry.
Adipose tissue histology and expression of genes involved in glucose and lipid metabolism (superficial adipose tissue biopsy).

ReferenceCitation: Triay J, Mundi M, Klein S, Toledo FG, Smith SR, Abu-Lebdeh H, Jensen M. Does rimonabant independently affect free fatty acid and glucose metabolism? J Clin Endocrinol Metab. 2012 Mar;97(3):819-27. doi: 10.1210/jc.2011-2486. Epub 2011 Dec 14.

location_country:
Finland
Italy
United Kingdom
Denmark
Sweden
United States
Spain
Canada
France


title: A Double Blind, Randomized, Phase II-III Maintenance Study of SR48692 Versus Placebo in Patients With Extensive Stage Small Cell Lung Cancer Following a First Line Chemotherapy With Cisplatin + Etoposide

acronym: SESAME

summary:
To demonstrate an increase in overall survival for patients with newly diagnosed extended stage small cell lung cancer when treated with SR48692 versus placebo, after an initial response (complete or partial response or stable) to first line cisplatin plus etoposide.

Primary objective: comparison of overall survival between patients in the control arm and the meclinertant arm.

Secondary objectives: comparison of the progression free survival, the time to progression, the clinical benefit, the quality of life, the toxicity and safety between patients in the control arm and the meclinertant arm.

location_country:
Germany
Italy
Poland
Hungary
Argentina
United Kingdom
Australia
Brazil
Netherlands
Mexico
Belgium
Spain
Russian Federation
France

location_facility:
Sanofi-Aventis Administrative Office
Sanofi-aventis adminsitrative office


summary: The purpose of this study was to determine the safety and effectiveness of different doses of botulinum toxin Type A in treating lower urinary tract symptoms due to benign prostatic hyperplasia.

ReferenceCitation: Marberger M, Chartier-Kastler E, Egerdie B, Lee KS, Grosse J, Bugarin D, Zhou J, Patel A, Haag-Molkenteller C. A randomized double-blind placebo-controlled phase 2 dose-ranging study of onabotulinumtoxinA in men with benign prostatic hyperplasia. Eur Urol. 2013 Mar;63(3):496-503. doi: 10.1016/j.eururo.2012.10.005. Epub 2012 Oct 12.

lead_sponsor: Allergan

location_country:
Germany
Italy
Slovakia
United Kingdom
Australia
Austria
Korea, Republic of
Taiwan
Canada
Czech Republic
France


title: Cooperative Multicenter Study for Children and Adolescents With Low Grade Glioma

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Drugs used in chemotherapy, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This clinical trial is studying giving radiation therapy or combination chemotherapy to see how well it works in treating patients with clinically or radiologically progressive low-grade gliomas.

ReferenceCitation: Gnekow AK, Walker DA, Kandels D, Picton S, Giorgio Perilongo, Grill J, Stokland T, Sandstrom PE, Warmuth-Metz M, Pietsch T, Giangaspero F, Schmidt R, Faldum A, Kilmartin D, De Paoli A, De Salvo GL; of the Low Grade Glioma Consortium and the participating centers. A European randomised controlled trial of the addition of etoposide to standard vincristine and carboplatin induction as part of an 18-month treatment programme for childhood (</=16 years) low grade glioma - A final report. Eur J Cancer. 2017 Aug;81:206-225. doi: 10.1016/j.ejca.2017.04.019. Epub 2017 Jun 22. Erratum In: Eur J Cancer. 2017 Dec 13;:

repository_institution: University of Nottingham - Repository@Nottingham

repository_institution: Augsburg University - OPUS - Augsburg University Publication Server

repository_institution: Umeå University - DiVA at Umeå University

ReferenceCitation: Kandels D, Pietsch T, Bison B, Warmuth-Metz M, Thomale UW, Kortmann RD, Timmermann B, Hernaiz Driever P, Witt O, Schmidt R, Gnekow AK. Loss of efficacy of subsequent nonsurgical therapy after primary treatment failure in pediatric low-grade glioma patients-Report from the German SIOP-LGG 2004 cohort. Int J Cancer. 2020 Dec 15;147(12):3471-3489. doi: 10.1002/ijc.33170. Epub 2020 Jul 11. Erratum In: Int J Cancer. 2021 Jul 15;149(2):E3-E4.

repository_institution: Würzburg University - Online Publication Service of Würzburg University

lead_sponsor: Societe Internationale d'Oncologie Pediatrique

location_country:
Germany
Italy
Poland
Portugal
Norway
United Kingdom
Denmark
Switzerland
Austria
Belgium
Croatia
France

location_facility: Allg. Krankenhaus der Stadt Wien Universitaets-Kinderklinik