title: A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

acronym: PEAPOD

summary: The study will aim to estimate the efficacy of apremilast compared with placebo in the treatment of juvenile psoriatic arthritis (JPsA) in pediatric participants 5 to less than 18 years of age.

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

lead_sponsor: Amgen

location_country:
Germany
Italy
Lithuania
Poland
Portugal
United Kingdom
Romania
Netherlands
Austria
Belgium
Spain
Greece
South Africa
Turkey
France

location_facility: Ziekenhuis Netwerk Antwerpen Jan Palfijn

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: Charite - Universitaetsmedizin Berlin, Campus Virchow

location_facility: Narodowy Instytut Geriatrii Reumatologii i Rehabilitacji im prof dr hab med Eleonory Reicher

location_facility: Wojewodzki Specjalistyczny Szpital Dzieciecy im sw Ludwika w Krakowie

location_facility: Spitalul Clinic de Urgenta pentru Copii Louis Turcanu Timisoara

location_facility: Hospices Civils de Lyon Hopital Femme Mere Enfant

location_facility: Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz

location_facility: Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

location_facility: Spitalul Clinic de Urgenta pentru Copii Cluj

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Centro Hospitalar Universtario de Sao Joao, EPE

location_facility: General Hospital of Thessaloniki Ippokrateio

location_facility: Asklepios Kinderklinik Sankt Augustin GmbH

location_facility: SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi

location_facility: Viesoji istaiga Vilniaus universiteto ligonine Santaros klinikos, Vaiku ligonine

location_facility: Centre Hospitalier Regional de la Citadelle

location_facility: Centro Hospitalar Universitario de Lisboa Norte, EPE - Hospital de Santa Maria

location_facility: Hacettepe Universitesi Tip Fakultesi Hastanesi

location_facility: Azienda Socio Sanitaria Territoriale Centro Specialistico Ortopedico Traumatologico Gaetano Pini


title: DiamondTemp Global Registry

summary: The DiamondTemp™ Global Registry is a prospective, global, multi-center, observational post-market registry.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Germany
Italy
Poland
Slovenia
United Kingdom
Australia
Netherlands
Switzerland
Belgium
Spain
France

location_facility: John Paul II Upper Silesian Medical Centre

location_facility: University Hospitals Coventry & Warwickshire

location_facility: Ospedale di Conegliano - S. Maria dei Battuti

location_facility: Herz- und Diabeteszentrum NRW - Ruhr-Universität Bochum

intervention_type: Device

lead_sponsor_type: industriel

status_simplified: Ongoing


title: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis

summary: The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.

study_type: Interventional

primary_purpose: Treatment

ipd_sharing: No

lead_sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

location_country:
Germany
Italy
United Kingdom
Romania
Lebanon
United Arab Emirates
United States
Spain
Morocco
France

intervention_type: Drug


title: A First-in-human, Two-part Clinical Study to Assess the Safety, Tolerability and Activity of IV Doses of ICT01 as Monotherapy and in Combination With a Checkpoint Inhibitor, in Patients With Advanced-stage, Relapsed/Refractory Cancer

acronym: EVICTION

summary: Part 1 will be a dose escalation study of IV ICT01 (a monoclonal antibody targeting BTN3A) as monotherapy in patients with advanced solid or hematologic tumors, followed by a cohort examining the combination of ICT01 plus pembrolizumab (Keytruda). Part 2 will be a cohort expansion into 2 solid tumor indications and one hematologic malignancy for ICT01 monotherapy, and 3 solid tumor indications for the combination of ICT01 plus pembrolizumab.

design_allocation: Randomized

lead_sponsor: ImCheck Therapeutics

location_country:
Germany
United Kingdom
Belgium
United States
Spain
France

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: University Carl Gustav Carus Clinical Trial Unit

intervention_type: Biological


acronym: ILLUMINATE-C

lead_sponsor: Alnylam Pharmaceuticals

summary: The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

ReferenceType: derived

ReferenceCitation: Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2023 Feb;81(2):145-155.e1. doi: 10.1053/j.ajkd.2022.05.012. Epub 2022 Jul 14.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

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

host_type: repository

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Nantes

repository_normalized: HAL

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

repositories_concat: HAL

unpaywall_oa_status: hybrid

publisher_dissemination: Elsevier

status: Active, not recruiting

title: ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (PH1)

type: EudraCT Number

source: euctr

enrollment_type: Actual

location_country:
France
Italy
Australia
Netherlands
Lebanon
Belgium
United Arab Emirates
United States
Turkey
Jordan
Israel


title: Stereotactic Ablative Body Radiotherapy (SABR) Combined With Immunotherapy (L19-IL2) in Stage IV NSCLC Patients, ImmunoSABR: a Multicentre, Randomised Controlled Open-label Phase II Trial

summary:
This will be a phase II trial testing if the combination of stereotactic ablative body radiotherapy (SABR) and L19-IL2 improve the progression-free survival in patients with limited metastatic non-small cell lung cancer (NSCLC). The trial consists of one cohort with two arms; C-arm and an E-arm.

Patients with oligometastatic disease will receive SABR to minimal 1 and max all metastatic sites (max 5 sites irradiated) and patients with diffuse metastatic lesions (6 to max 10) will receive radiotherapy to max 5 sites. In the experimental arm, immunotherapy will be given after irradiation.

status: Unknown status

ReferenceCitation: Olivo Pimentel V, Marcus D, van der Wiel AM, Lieuwes NG, Biemans R, Lieverse RI, Neri D, Theys J, Yaromina A, Dubois LJ, Lambin P. Releasing the brakes of tumor immunity with anti-PD-L1 and pushing its accelerator with L19-IL2 cures poorly immunogenic tumors when combined with radiotherapy. J Immunother Cancer. 2021 Mar;9(3):e001764. doi: 10.1136/jitc-2020-001764.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

repository_institution: ETH Zurich - Repository for Publications and Research Data

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

unpaywall_oa_status: gold

publisher_dissemination: British Medical Journal

ReferenceCitation: Lieverse RIY, Van Limbergen EJ, Oberije CJG, Troost EGC, Hadrup SR, Dingemans AC, Hendriks LEL, Eckert F, Hiley C, Dooms C, Lievens Y, de Jong MC, Bussink J, Geets X, Valentini V, Elia G, Neri D, Billiet C, Abdollahi A, Pasquier D, Boisselier P, Yaromina A, De Ruysscher D, Dubois LJ, Lambin P. Stereotactic ablative body radiotherapy (SABR) combined with immunotherapy (L19-IL2) versus standard of care in stage IV NSCLC patients, ImmunoSABR: a multicentre, randomised controlled open-label phase II trial. BMC Cancer. 2020 Jun 15;20(1):557. doi: 10.1186/s12885-020-07055-1.

repository_institution: PubMed Central - Europe PMC

repository_institution: Technical University of Denmark, DTU - Technical University of Denmark, DTU Orbit

repository_normalized: Pure (Denmark)

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repository_institution: Erasmus University Rotterdam - EUR Research Repository

repository_institution: Radboud University - Radboud Repository

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

repository_institution: Erasmus University, Rotterdam - RePub

repository_institution: University of Antwerp - Institutional Repository University of Antwerp

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

repository_institution: University College London - UCL Discovery

repositories_concat: LIRIAS (KU Leuven);PubMed Central;Pure (Denmark);UCL Discovery;archive.ugent.be;irua;pure.eur.nl;repository.ubn.ru.nl;repub.eur.nl;www.research-collection.ethz.ch

collaborators: Cliniques universitaires Saint-Luc- Université Catholique de Louvain

collaborators: Institut du Cancer de Montpellier - Val d'Aurelle

location_country:
Germany
Italy
United Kingdom
Netherlands
Belgium
France

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS Università Cattolica del Sacro Cuore

location_facility: Academisch Ziekenhuis Maastricht (Leading Centre)

location_facility: INSTITUT régional du CANCER MONTPELLIER - ICM - VAL d'AURELLE


acronym: LEADER 75

lead_sponsor: Bayer

summary:
The study was conducted to gain knowledge about a new dose of a diagnostic drug that is used for contrast-enhanced Magnetic Resonance Imaging (MRI) of the human central nervous system (CNS). MRI can visualize the anatomy of the body and is used to detect medical conditions. Diagnostic drugs like gadobutrol and gadoterate contain an element called gadolinium that is applied to improve the analysability of MRI-images.

The purpose of this study was to examine if contrast-enhanced MRI using a reduced dose of the gadolinium-based contrast agent gadobutrol delivers images of similar quality to those obtained when a full dose of the gadolinium-based contrast agent gadoterate was used.

ReferenceCitation: Liu BP, Rosenberg M, Saverio P, Weon YC, Peters S, Ardellier FD, Boeckenhoff A, Endrikat J. Clinical Efficacy of Reduced-Dose Gadobutrol Versus Standard-Dose Gadoterate for Contrast-Enhanced MRI of the CNS: An International Multicenter Prospective Crossover Trial (LEADER-75). AJR Am J Roentgenol. 2021 Nov;217(5):1195-1205. doi: 10.2214/AJR.21.25924. Epub 2021 Jun 16.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: American Roentgen Ray Society

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Universitätsklinikum Schleswig-Holstein / AÖR

location_facility: University of Texas Southwestern Medical Center

location_facility: Universität Rostock - Medizinische Fakultät

status: Completed

title: LowEr Administered Dose With highEr Relaxivity: Gadovist vs Dotarem (LEADER 75)

primary_purpose: Diagnostic

design_allocation: Non-Randomized

study_completion_date_type: Actual

location_country:
Germany
Italy
United Kingdom
Switzerland
United States
Korea, Republic of
France

status_simplified: Completed


summary: In this study researchers want to gather more information about safety and effectiveness of BAY 2599023 (DTX201), a drug therapy that delivers the human factor VIII gene into the human body by use of a viral vector to treat the disease. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own.

sponsor_collaborators:
Bayer
Ultragenix pharmaceutical

location_facility: SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD

location_facility: Hopital Necker les enfants malades - Paris

title: A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A

type: Other Identifier

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

location_country:
Germany
United Kingdom
Netherlands
United States
Bulgaria
France


title: Trial to Evaluate Treatment With Abbott Transcatheter Clip Repair System in Patients With Moderate or Greater Tricuspid Regurgitation (TRILUMINATE)

acronym: TRILUMINATE

summary: The primary purpose of this study is to evaluate safety and effectiveness of the Tricuspid Valve Repair System (TVRS) for treating symptomatic moderate or greater tricuspid regurgitation (TR) in patients currently on medical management and who are deemed appropriate for percutaneous transcatheter intervention.

ReferenceCitation: Lurz P, Stephan von Bardeleben R, Weber M, Sitges M, Sorajja P, Hausleiter J, Denti P, Trochu JN, Nabauer M, Tang GHL, Biaggi P, Ying SW, Trusty PM, Dahou A, Hahn RT, Nickenig G; TRILUMINATE Investigators. Transcatheter Edge-to-Edge Repair for Treatment of Tricuspid Regurgitation. J Am Coll Cardiol. 2021 Jan 26;77(3):229-239. doi: 10.1016/j.jacc.2020.11.038.

ReferenceCitation: Nickenig G, Weber M, Lurz P, von Bardeleben RS, Sitges M, Sorajja P, Hausleiter J, Denti P, Trochu JN, Nabauer M, Dahou A, Hahn RT. Transcatheter edge-to-edge repair for reduction of tricuspid regurgitation: 6-month outcomes of the TRILUMINATE single-arm study. Lancet. 2019 Nov 30;394(10213):2002-2011. doi: 10.1016/S0140-6736(19)32600-5. Epub 2019 Nov 7. Erratum In: Lancet. 2020 Mar 14;395(10227):870.

lead_sponsor: Abbott Medical Devices

location_country:
Germany
Italy
Switzerland
United States
Spain
France

location_facility: Universitatsmedizin der Johannes Gutenberg-Universitat Mainz

location_facility: Ludwig-Maximilian University of Munich (LMU)

location_facility: HerzKlinik Hirslanden - Klinik Hirslanden

location_facility: Azienda Ospedaliero Universitaria Policlinico Vittorio Emanuele - Presidio Ferrarotto

location_facility: Clinical and Provincial Hospital of Barcelona


title: A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva

summary:
This is a three period study design consisting of a 6-month, randomized, double-blind placebo-controlled treatment (period 1) followed by a 6-month, open-label treatment (period 2) and a follow-up treatment period (period 3).

Primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).

Primary efficacy objective of the study is to assess the effect of REGN2477 versus placebo on the change from baseline in heterotopic ossification (HO) in patients with FOP, as determined by 18-NaF uptake in HO lesions by positron emission tomography (PET) and in total volume of HO lesions by computed tomography (CT).

Key Secondary objectives are:

To compare the effect of REGN2477 versus placebo on pain due to FOP, as measured by the area under the curve (AUC) for pain based on daily pain numeric rating scale (NRS) scores
To assess the effect of REGN2477 versus placebo on the change from baseline in HO, as determined by the number of new HO lesions identified by 18F-NaF PET or by CT
To assess the effect of REGN2477 versus placebo on the change from baseline in 18F-NaF standardized uptake value maximum (SUVmax) of individual active HO site(s) by PET
To assess the effect of REGN2477, between week 28 and week 56, on the number, activity, and volume of HO lesions identified by 18F-NaF PET or by CT in patients who switch from placebo to REGN2477 at week 28 versus the same patients between baseline and week 28
To assess the effect of REGN2477 versus placebo on the change from baseline in biochemical markers of bone formation
To characterize the concentrations of total activin A at baseline and over time following the first dose of study drug
To characterize the concentration-time profile (pharmacokinetics [PK]) of REGN2477 in patients with FOP
To assess the immunogenicity of REGN2477

ReferenceCitation: Vanhoutte F, Liang S, Ruddy M, Zhao A, Drewery T, Wang Y, DelGizzi R, Forleo-Neto E, Rajadhyaksha M, Herman G, Davis JD. Pharmacokinetics and Pharmacodynamics of Garetosmab (Anti-Activin A): Results From a First-in-Human Phase 1 Study. J Clin Pharmacol. 2020 Nov;60(11):1424-1431. doi: 10.1002/jcph.1638. Epub 2020 Jun 18.

repositories_concat: PubMed Central

publisher_dissemination: Wiley

ReferenceCitation: Aykul S, Corpina RA, Goebel EJ, Cunanan CJ, Dimitriou A, Kim HJ, Zhang Q, Rafique A, Leidich R, Wang X, McClain J, Jimenez J, Nannuru KC, Rothman NJ, Lees-Shepard JB, Martinez-Hackert E, Murphy AJ, Thompson TB, Economides AN, Idone V. Activin A forms a non-signaling complex with ACVR1 and type II Activin/BMP receptors via its finger 2 tip loop. Elife. 2020 Jun 9;9:e54582. doi: 10.7554/eLife.54582.

publisher_dissemination: eLife Sciences Publications, Ltd

lead_sponsor: Regeneron Pharmaceuticals

location_country:
Italy
Poland
United Kingdom
Netherlands
United States
Spain
Canada
France

location_facility: Hopital Lariboisiere,Hospitalier Universitaire Nord

location_facility: Royal National Orthopaedic Hospital, Brockley Hill


acronym: Expanded treatment protocol for CTL019.

lead_sponsor: Novartis Pharmaceuticals

summary: This is a single arm, open-label, multi-center, phase III B study to determine the safety and efficacy of CTL019 in pediatric/young adult patients with r/r B-cell Acute Lymphoblastic Leukemia (ALL).

ReferenceCitation: Laetsch TW, Maude SL, Balduzzi A, Rives S, Bittencourt H, Boyer MW, Buechner J, De Moerloose B, Qayed M, Phillips CL, Pulsipher MA, Hiramatsu H, Tiwari R, Grupp SA. Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2022 Jun;36(6):1508-1515. doi: 10.1038/s41375-022-01550-z. Epub 2022 Apr 14.

publisher_dissemination: Springer-Nature

title: Phase IIIb Study for Relapsed/Refractory Pediatric/Young Adult Acute Lymphoblastic Leukemia Patients to be Treated With CTL019

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

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

location_country: Germany Italy Norway Japan Austria Belgium Spain Canada France

ipd_sharing: Yes


title: A Real World Evaluation of the ELUVIA Drug Eluting Stent in All-Comers With Superficial Femoral Artery and Proximal Popliteal Artery Disease

acronym: REGAL

summary: The REGAL study is a European, prospective, multi-center Post-Market Clinical Follow-up (PMCF) trial providing additional data including health economics data to support the use of the ELUVIA stent in the treatment of lesions located in the femoropopliteal arteries.

ipd_sharing: Undecided

lead_sponsor: Boston Scientific Corporation

location_country: Italy Austria Belgium Spain France

location_facility: Centre Hospitalier Universitaire Grenoble-Alpes

location_facility: Hospital Azienda Ospedaliera Universitaria Senese

location_facility: Foundation for the Aragonese Healthcare Research Institute

location_facility: Hosp. Complejo Hosp Universitario (CHUO)


title: A Phase 2 Randomized Open-Label Study of Oral Darolutamide (ODM-201) vs. Androgen Deprivation Therapy (ADT) With LHRH Agonists or Antagonist in Men With Hormone Naive Prostate Cancer

summary:
This is an open label non-comparative controlled randomized phase II study. The experimental arm is the group receiving ODM-201. The group receiving androgen-deprivation therapy (ADT) is included as an internal control.

The primary trial objective is to demonstrate that ODM-201 produces prostate-specific antigen (PSA) response rates at 24 weeks (defined as ≥80% reduction compared to baseline) that are in the range of those achieved with 24 weeks of ADT.

In total, this 1:1 randomized study will therefore require randomization of at least 250 patients, 125 to each arm.

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

location_country: Italy Spain Belgium France

location_facility: Institut Catala d'Oncologia - ICO Badalona - Hospital Germans Trias i Pujol

location_facility: Hospital Universitario Virgen De La Victoria

location_facility: Hopitaux Universitaires Bordet-Erasme - Hopital Universitaire Erasme

location_facility: CHU de Dijon - Centre Georges-Francois-Leclerc

location_facility: Fundacion Instituto Valenciano De Oncologia

location_facility: Azienda Ospedaliera Citta della Salute e della Scienza di Torino - Ospedale Molinette


title: MERIT - A Phase II Marker Identification Trial for Tarceva in Second Line NSCLC Patients

acronym: MERIT

summary: This study will assess potentially predictive markers of efficacy in participants with NSCLC receiving oral erlotinib (Tarceva) therapy. The anticipated time on study treatment is until disease progression, unacceptable toxicity or death.

lead_sponsor: Hoffmann-La Roche

location_country:
Germany
Ireland
Italy
Poland
United Kingdom
Hong Kong
Singapore
Belgium
Spain
Taiwan
Bulgaria
Estonia
Russian Federation
France


title: A Phase I Dose Finding Study of Oral LXH254 in Adult Patients With Advanced Solid Tumors Harboring MAPK Pathway Alterations

summary: A Phase I Study of LXH254 in Patients With Advanced Solid Tumors That Harbor MAPK Pathway Alterations.

status: Terminated

location_country:
Germany
Italy
Japan
Netherlands
Switzerland
United States
Korea, Republic of
Spain
Canada
France

location_facility: Memorial Sloan Kettering Cancer Center SC - LXH254X2101

location_facility: Massachusetts General Hospital MGH Cancer Center

location_facility: UT M.D Anderson Cancer Center SC - LXH254X2101

status_simplified: Unknown


title: An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy

acronym: FSHD

summary: The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with early onset FSHD.

lead_sponsor: aTyr Pharma, Inc.

location_country:
United States
Italy
France

location_facility: Centre d'nvestigation Clinique - Centre de Pharmacologie Clinique et d'Evaluations Thérapeutiques (CICCPCET)

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


title: A Phase I, Multicenter, Open-label, Single-sequence Drug-drug Interaction Study to Assess the Effect of INC280 on the Pharmacokinetics of Midazolam and Caffeine in Patients With cMET-dysregulated Advanced Solid Tumors

summary: Drg-drug Interaction (DDI) study to assess the effect of INC280 on the pharmacokinetics of midazolam and caffeine in patients with cMET-dysregulated advanced solid tumors

location_country:
Italy
United Kingdom
Denmark
United States
Spain
Bulgaria
France

location_facility: Emory University School of Medicine/Winship Cancer Institute SC-2

location_facility: Washington University School of Medicine


acronym: MORA

lead_sponsor: Norgine

summary: This study evaluates the efficacy, safety and tolerability of NER1006 versus MOVIPREP in adult patients requiring bowel cleansing prior to any procedure that requires a clean bowel, using a 2-Day evening/morning Split-Dosing and 1-Day morning only Split-Dosing regimens. Approximately 810 patients will be randomised with the aim of achieving a minimum of 245 patients in each of the 3 groups.

ReferenceCitation: Cash BD, Allen C, Poppers DM. Transient alterations in plasma sodium concentrations with NER1006 bowel preparation: an analysis of three phase III, randomized clinical trials. BMC Gastroenterol. 2022 Sep 5;22(1):412. doi: 10.1186/s12876-022-02484-7.

ReferenceCitation: Bisschops R, Manning J, Clayton LB, Ng Kwet Shing R, Alvarez-Gonzalez M; MORA Study Group. Colon cleansing efficacy and safety with 1 L NER1006 versus 2 L polyethylene glycol + ascorbate: a randomized phase 3 trial. Endoscopy. 2019 Jan;51(1):60-72. doi: 10.1055/a-0638-8125. Epub 2018 Jul 19.

oa_host_type: repository

version: acceptedVersion

repositories_concat: LIRIAS (KU Leuven)

unpaywall_oa_status: green

publisher_dissemination: Georg Thieme

location_facility: Ospedale Valduce U.O. Gastroenterologia e Endoscopia

location_facility: Uniwersytecki Szpital Kliniczny w Bialymstoku

location_facility: Royal Albert Edward Infirmary Department

location_facility: Centro di Riferimento Oncologico (C.R.O.) S.O.C Gastroenterologia

location_facility: Klinikum der Friedrich Schiller Universität Jena

location_facility: Praxis für Innere Medizin, Gastroenterologie und Allg. Medizin

location_facility: Centralny Szpital Kliniczny Ministerstwa Spraw Wewnetrznych

location_facility: Kliniken Essen-Mitte; Abteilung für Gastroenterologie

location_facility: Pol. Univ. A. Gemelli U.O. di Endoscopia Digestiva Chirurgica

location_facility: Hospital General Universitario de Alicante

location_facility: Hospital Ramon y Cajal - Ctra. De Colmenar km. 9, 100

location_facility: Gabinet Internistyczny dr n. med. Krzysztof Janik

location_facility: A.O.U. di Bologna - Policlinico S. Orsola-Malpighi

location_facility: NZOZ Centrum Medyczne-Szpital Swietej Rodziny

location_facility: Hôpital Avicenne- Service de Gastro-Entérologie

location_facility: Hospital Universitari Germans Trias i Pujol

title: A Multicenter Randomized Parallel Group Phase III Study Comparing the Bowel Cleansing Efficacy, Safety and Tolerability of NER1006 (a Low Volume Bowel Cleansing Solution) Versus MOVIPREP Using 2-Day Split-Dosing and 1-Day Morning Split-Dosing Regimen in Adults.

primary_purpose: Screening

location_country:
Germany
Italy
Poland
United Kingdom
Belgium
Spain
France


title: A Prospective Colorectal Liver Metastasis Database With an Integrated Quality Assurance Program

acronym: CLIMB

summary:
This prospective database has two main objectives;

to evaluate the complication rates, 30-day and 90-day mortality from different surgical strategies for unresectable, borderline resectable or initially unresectable liver metastasis from colorectal cancer.
to establish baseline quality parameters for different surgical strategies for unresectable, borderline and initially unresectable colorectal liver metastasis (CRLM) patients.

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

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

location_facility: Der Wiener Krankenanstaltenverbund - Krankenanstalt Rudolfstiftung

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

intervention_type: Other


title: An Open Label Study to Evaluate the Effects of Ezogabine/Retigabine Added to Existing Anti-epileptic Drug(s) on Urinary Voiding Function in Subjects With Partial Onset Seizures

summary:
This is a multicentre, open label study to examine the effect of ezogabine/retigabine on the voiding function of adult subjects with drug-resistant partial onset seizures (POS). Subjects fulfilling the study entry criteria at Screening and at Baseline including a comprehensive eye examination by an ophthalmologist or retina specialist and a skin assessment by the investigator will receive ezogabine/retigabine.

The starting dose of ezogabine/retigabine will be 300 mg/day. Subjects will be up titrated by 150 mg/day weekly up to the maximum ezogabine/retigabine daily dose of 1200 mg (or the highest tolerated dose). During the 49 days of the treatment phase, subjects will undergo three repeat non-invasive assessments of voiding function. In addition, subjects who meet pre-determined criteria for voiding dysfunction will undergo multichannel cystometry in order to characterise bladder hypocontractility, bladder outlet obstruction or a combination of events which clinically is manifest with difficulty emptying the bladder or acute urinary retention.

At the end of the Treatment Phase, all subjects will enter the Taper Phase, a 3-week down titration period. Subjects who have new findings of abnormal pigmentation of the retina, unexplained vision loss, pigmentation of non-retinal ocular tissue or discoloration of skin, lip, nail, or mucosa since baseline will be asked to enter the Safety Follow-Up / Continuation Phase. All subjects will undergo 6-monthly comprehensive eye examinations during the Safety Follow-Up / Continuation Phase. Subjects who have not developed abnormal discoloration of the skin, lips, nails or mucosa will continue to undergo skin assessments by the investigator. Any subject who has developed abnormal discoloration of the skin, lips, nails or mucosa since baseline will be referred to a dermatologist for evaluation and 6-monthly follow up assessments. All subjects will continue to be followed until the pigmentation and/or discoloration has resolved or stabilised, as defined by no change over 2 consecutive 6-monthly assessments conducted over at least 12 months after discontinuation of ezogabine/retigabine.

lead_sponsor: GlaxoSmithKline

location_country:
United States
Poland
France


title: An Open Label, 52-week, Safety and Tolerability Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

summary: This study was to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects with Familial Chylomicronemia Syndrome (FCS) (HLP type I).

location_country:
Germany
United Kingdom
Netherlands
United States
South Africa
Canada
France


title: An Open-label, Multi-center Phase I Dose-finding Study of RAD001 (Everolimus, Afinitor®) in Combination With BEZ235 in Patients With Advanced Solid Tumors

summary:
Study has two parts:

Dose-finding: to determine the maximum tolerated dose (MTD) and to evaluate the safety and tolerability of RAD001 (everolimus , Afinitor®) in combination with BEZ235 in patients with advanced solid tumors.
Dose-expansion: to assess safety and tolerability of RAD001 and BEZ235 at the MTD in patients with ER+/HER2- metastatic breast cancer and metastatic renal cell cancer

location_country:
Italy
New Zealand
United Kingdom
Belgium
United States
Spain
France

location_facility: Highlands Oncology Group Dept of Highlands Oncology Grp

location_facility: Washington University School of Medicine Washington University (16)


title: A Multi-arm Dose-finding Phase Ib Multicenter Study of Imatinib in Combination With the Oral Phosphatidyl-inositol 3-kinase (PI3-K) Inhibitor BKM120 in Patients With Gastrointestinal Stromal Tumor (GIST) Who Failed Prior Therapy With Imatinib and Sunitinib

summary: The purpose of this study is to determine a maximum tolerated dose and/or recommended phase 2 dose of a combination of imatinib and BKM120 in the treatment of 3rd line GIST patients.

ReferenceCitation: Gelderblom H, Jones RL, George S, Valverde Morales C, Benson C, Jean-Yves Blay, Renouf DJ, Doi T, Le Cesne A, Leahy M, Hertle S, Aimone P, Brandt U, Schӧffski P. Imatinib in combination with phosphoinositol kinase inhibitor buparlisib in patients with gastrointestinal stromal tumour who failed prior therapy with imatinib and sunitinib: a Phase 1b, multicentre study. Br J Cancer. 2020 Apr;122(8):1158-1165. doi: 10.1038/s41416-020-0769-y. Epub 2020 Mar 9.

repositories_concat: LIRIAS (KU Leuven);PubMed Central;scholarlypublications.universiteitleiden.nl

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

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

location_facility:
Novartis Investigative Site
Dana Farber Cancer Institute SC (2)
Seattle Cancer Care Alliance Onc


title: A Multi-center, Open Label, First in Human Phase I Dose Escalation Study of Single Agent RO5503781, a Small Molecule MDM2 Antagonist, Administered Orally in Patients With Advanced Malignancies, Except Leukemia

summary: This multicenter, open label, dose-escalating study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of RO5503781, administered once daily (QD) or once weekly (QW) in participants with advanced malignancies except leukemia. Participants will receive multiple escalating oral doses in two different dosing schedules (Sch) until disease progression or unacceptable toxicity occurs.

ReferenceCitation: Italiano A, Miller WH Jr, Blay JY, Gietema JA, Bang YJ, Mileshkin LR, Hirte HW, Higgins B, Blotner S, Nichols GL, Chen LC, Petry C, Yang QJ, Schmitt C, Jamois C, Siu LL. Phase I study of daily and weekly regimens of the orally administered MDM2 antagonist idasanutlin in patients with advanced tumors. Invest New Drugs. 2021 Dec;39(6):1587-1597. doi: 10.1007/s10637-021-01141-2. Epub 2021 Jun 28.

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

repositories_concat: PubMed Central;University of Melbourne - Minerva Access;pure.rug.nl

location_country:
Australia
Netherlands
Korea, Republic of
Canada
France


title: A Multicentre, Open-Label, Long-Term, Safety and Tolerability Study of Retigabine Immediate Release (IR) in Adults With Partial-Onset Seizures (Extension of Study RGB113905)

acronym: IR

summary: The purpose of this Phase III study is to assess the long-term safety, tolerability and efficacy of flexibly dosed retigabine Immediate Release (IR) as adjunctive therapy in adult subjects with partial-onset seizures. In addition, those subjects who successfully completed 20 weeks of adjunctive treatment with retigabine IR in the parent study, RGB113905, and who were thought to have benefitted from treatment will be provided continued access to retigabine IR.

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

location_country:
Germany
Italy
Poland
Netherlands
Belgium
Thailand
Bulgaria
Ukraine
Russian Federation
France


title: A Proof of Concept, Open-label, Forced Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of 10-weeks Treatment of LCI699 in Patients With Cushing's Disease

summary:
This exploratory study is a proof of concept study to determine whether LCI699 can safely reduce the level of urinary free cortisol in patients with Cushing's disease.

In addition, this study evaluated the long term efficacy and safety of LCI699 including an additional 12 week of treatment followed by a 12 month long term optional extension.

A second extension provided patients who were clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 was commercially available and reimbursed or through the availability of a local access program.

ReferenceCitation: Bertagna X, Pivonello R, Fleseriu M, Zhang Y, Robinson P, Taylor A, Watson CE, Maldonado M, Hamrahian AH, Boscaro M, Biller BM. LCI699, a potent 11beta-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing's disease: results from a multicenter, proof-of-concept study. J Clin Endocrinol Metab. 2014 Apr;99(4):1375-83. doi: 10.1210/jc.2013-2117. Epub 2013 Dec 11.

oa_host_type: publisher

evidence: open (via free pdf)

unpaywall_oa_status: bronze

publisher_dissemination: The Endocrine Society

location_country:
United States
Italy
Japan
France

location_facility: Northwestern University Endo, Metabolism and Molecular

location_facility: Massachusetts General Hospital Neuroendocrine Unit


title: A 2-year, Multicenter, Double-masked, Randomized, Parallel Study of the Safety of LUMIGAN® 0.1 mg/mL Compared With LUMIGAN® 0.3 mg/mL in Patients With Glaucoma or Ocular Hypertension

summary: This study will evaluate the long-term safety of bimatoprost 0.01% ophthalmic solution compared with bimatoprost 0.03% ophthalmic solution in patients with glaucoma or ocular hypertension.

lead_sponsor: Allergan

location_country:
Germany
Italy
Poland
Hungary
Israel
United Kingdom
Belgium
Spain
Czechia
France

location_facility: Dipartimento di Scienze Otorino-Odonto-Oftalmologiche e Cerv

location_facility: Fundacion Oftalmologica Del Mediterraneo

location_facility: University of Szeged Szent-Gyorgyi Albert Clinical Center

location_facility: Glaucoma Service The Rabin Medical Center

location_facility: Akad. Med. w Warszawie - Ketedra I Klin. Okulistki Wyzialu L

location_facility: Huntingdon Glaucoma Diagnostic & Research Centre

location_facility: Centre Hospitalier Universitaire de Bordeaux

location_facility: Fondazione G.B. Bietti per l'Oftalmologia I.R.C.C.S.

location_facility: Osrodek Badan Klinicznych Euromedis Sp. z o.o.

location_facility: Department of Ophthalmology, University of Tuebingen

location_facility: Szpital Kliniczny Przemienienia Panskiego Uniwersytetu

location_facility: Azlenda Ospedaliero Universitaria Careggi Viale


title: A Phase 1/2 Combined Dose Ranging and Randomized, Open-label, Comparative Study of the Efficacy and Safety of Plerixafor in Addition to Standard Regimens for Mobilization of Haematopoietic Stem Cells Into Peripheral Blood, and Subsequent Collection by Apheresis, Versus Standard Mobilization Regimens Alone in Pediatric Patients, Aged 1 to <18 Years, With Solid Tumours Eligible for Autologous Transplants.

summary:
This is a multi-site study with plerixafor in pediatric cancer patients. The study will be conducted in 2 stages:

Stage 1 is a dose-escalation study.
Stage 2 is an open-label, randomized, comparative study using the appropriate dosing regimen identified in the Stage 1 dose-escalation study.

All participating patients will receive a standard mobilization regimen as per study site practice guidelines (either chemotherapy plus once daily granulocyte-colony stimulating factor (G-CSF) or once daily G-CSF alone). The only change to the standard mobilization regimen is the addition of plerixafor treatment prior to apheresis for all patients in Stage 1 (dose escalation), and for those patients randomized to the plerixafor plus standard mobilization treatment arm in Stage 2 (randomized, comparative).

Stage 1 will enroll at least 27 patients. Stage 2 will enroll at least 40 patients.

ReferenceCitation: Sebastien B, Cheverton P, Magnin C, Aouni J, Castan R. Development and validation of a predictive model to guide the use of plerixafor in pediatric population. Bone Marrow Transplant. 2022 Dec;57(12):1827-1832. doi: 10.1038/s41409-022-01831-2. Epub 2022 Sep 26.

ReferenceCitation: Morland B, Kepak T, Dallorso S, Sevilla J, Murphy D, Luksch R, Yaniv I, Bader P, Rossler J, Bisogno G, Maecker-Kolhoff B, Lang P, Zwaan CM, Sumerauer D, Krivan G, Bernard J, Liu Q, Doyle E, Locatelli F. Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC). Bone Marrow Transplant. 2020 Sep;55(9):1744-1753. doi: 10.1038/s41409-020-0836-2. Epub 2020 Mar 3.

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

repositories_institution: Erasmus University Rotterdam - EUR Research Repository

lead_sponsor: Genzyme, a Sanofi Company

sponsor_collaborators:
Genzyme, a Sanofi Company
Sanofi

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

location_facility:
Investigational Site Number 81
Investigational Site Number 93
Investigational Site Number 71
Investigational Site Number 35
Investigational Site Number 51
Investigational Site Number 36
Investigational Site Number 82
Investigational Site Number 13
Investigational Site Number 42
Investigational Site Number 11
Investigational Site Number 31
Investigational Site Number 43
Investigational Site Number 83
Investigational Site Number 33
Investigational Site Number 85
Investigational Site Number 22
Investigational Site Number 84
Investigational Site Number 26
Investigational Site Number 72
Investigational Site Number 34
Investigational Site Number 24
Investigational Site Number 21
Investigational Site Number 92
Investigational Site Number 94
Investigational Site Number 61
Investigational Site Number 91
Investigational Site Number 23


title: European Ankylosing Spondylitis Infliximab Cohort (EASIC) Follow up Registration Study

acronym: EASIC registry

summary: Long term data on efficacy and safety of anti-TNF treatment with infliximab in patients with ankylosing spondylitis (AS) beyond 5 years is lacking. These data are important because patients with AS usually are younger and withdrawal of anti-TNF therapy in these patients almost always leads to a disease relapse. Furthermore it is still unclear whether long term anti-TNF treatment in AS patients can inhibit radiographic progression. Patients who participated in the EASIC and the DIKAS trial respectively who were treated with infliximab within these studies for 7 and 10 years respectively are followed up by using clinical outcome parameters every 6 months assessing efficacy and safety of long term treatment. Furthermore radiographs of the spine, if done for clinical indication, are analyzed. It is hypothesized that anti-TNF treatment with infliximab is effective and safe over a time period of 9 and 12 years respectively and that long term anti-TNF therapy may inhibit radiographic progression of the spine.

ReferenceType: background

ReferenceCitation: Zochling J, van der Heijde D, Burgos-Vargas R, Collantes E, Davis JC Jr, Dijkmans B, Dougados M, Geher P, Inman RD, Khan MA, Kvien TK, Leirisalo-Repo M, Olivieri I, Pavelka K, Sieper J, Stucki G, Sturrock RD, van der Linden S, Wendling D, Bohm H, van Royen BJ, Braun J; 'ASsessment in AS' international working group; European League Against Rheumatism. ASAS/EULAR recommendations for the management of ankylosing spondylitis. Ann Rheum Dis. 2006 Apr;65(4):442-52. doi: 10.1136/ard.2005.041137. Epub 2005 Aug 26.

ReferenceCitation: Baraliakos X, Listing J, Brandt J, Zink A, Alten R, Burmester G, Gromnica-Ihle E, Kellner H, Schneider M, Sorensen H, Zeidler H, Rudwaleit M, Sieper J, Braun J. Clinical response to discontinuation of anti-TNF therapy in patients with ankylosing spondylitis after 3 years of continuous treatment with infliximab. Arthritis Res Ther. 2005;7(3):R439-44. doi: 10.1186/ar1693. Epub 2005 Feb 21. Erratum In: Arthritis Res Ther. 2005;7(3):113. Zink, Angela [added];Alten, Rieke [added]; Burmester, Gerd [added]; Gromnica-Ihle, Erika [added]; Kellner, Herbert [added]; Schneider, Matthias [added]; Sorensen, Helmut [added]; Zeidler, Henning [added]; Rudwaleit, Martin [added].

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Alten R, Burmester G, Gromnica-Ihle E, Haibel H, Schewe S, Schneider M, Sorensen H, Zeidler H, Visvanathan S, Sieper J, Braun J. Safety and efficacy of readministration of infliximab after longterm continuous therapy and withdrawal in patients with ankylosing spondylitis. J Rheumatol. 2007 Mar;34(3):510-5. Epub 2007 Feb 1.

ReferenceCitation: Braun J, Baraliakos X, Listing J, Fritz C, Alten R, Burmester G, Krause A, Schewe S, Schneider M, Sorensen H, Zeidler H, Sieper J. Persistent clinical efficacy and safety of anti-tumour necrosis factor alpha therapy with infliximab in patients with ankylosing spondylitis over 5 years: evidence for different types of response. Ann Rheum Dis. 2008 Mar;67(3):340-5. doi: 10.1136/ard.2007.075879. Epub 2007 Oct 29.

ReferenceCitation: Dijkmans B, Emery P, Hakala M, Leirisalo-Repo M, Mola EM, Paolozzi L, Salvarani C, Sanmarti R, Sibilia J, Sieper J, Van Den Bosch F, van der Heijde D, van der Linden S, Wajdula J. Etanercept in the longterm treatment of patients with ankylosing spondylitis. J Rheumatol. 2009 Jun;36(6):1256-64. doi: 10.3899/jrheum.081033. Epub 2009 May 1. Erratum In: J Rheumatol. 2010 Oct;37(10):2198.

ReferenceCitation: Baraliakos X, Listing J, Rudwaleit M, Brandt J, Sieper J, Braun J. Radiographic progression in patients with ankylosing spondylitis after 2 years of treatment with the tumour necrosis factor alpha antibody infliximab. Ann Rheum Dis. 2005 Oct;64(10):1462-6. doi: 10.1136/ard.2004.033472. Epub 2005 Mar 18.

ReferenceCitation: van der Heijde D, Landewe R, Baraliakos X, Houben H, van Tubergen A, Williamson P, Xu W, Baker D, Goldstein N, Braun J; Ankylosing Spondylitis Study for the Evaluation of Recombinant Infliximab Therapy Study Group. Radiographic findings following two years of infliximab therapy in patients with ankylosing spondylitis. Arthritis Rheum. 2008 Oct;58(10):3063-70. doi: 10.1002/art.23901.

ReferenceCitation: Essers I, van Tubergen A, Heldmann F, Baraliakos X, Braun J, Kiltz U, Boonen A. Do patients with ankylosing spondylitis adapt to their disease? Evidence from a 'then-test' in patients treated with TNF inhibitors. RMD Open. 2015 Nov 17;1(1):e000164. doi: 10.1136/rmdopen-2015-000164. eCollection 2015.

license: cc-by-nc

license_normalized: cc-by-nc

lead_sponsor: Rheumazentrum Ruhrgebiet

sponsor_collaborators:
Rheumazentrum Ruhrgebiet
Centocor Ortho Biotech Services, L.L.C.

location_country:
Finland
Germany
United Kingdom
Netherlands
Belgium
France

location_facility: Klinik für Immunologie und Rheumatologie der MHH

location_facility: University Central Hospital, Division of Rheumatology

location_facility: Klinikum der Universität München, Rheumaeinheit

location_facility: Universitair Ziekenhuis, Afdeling Rheumatologie


title: A phase1 Multi-center, Open Label, Dose-escalation Study of Oral LEE011 in Patients With Advanced Solid Tumors or Lymphoma

summary:
LEE011 is a new oral drug designed to inhibit the activity of an enzyme known as CDK4/6. CDK4/6 is involved in the process that allows both normal and cancer cells to divide and multiply. Cancer cells are often driven to divide and multiply by abnormalities that increase the activity of CDK4. Hence there is hope that blocking the activity of CDK4 may slow the growth of some cancers. LEE011 has shown anti-cancer activity in several different tumor models in animals.

Because CDK4 is important in both normal and cancerous cells, LEE011 is expected to decrease the ability of the bone marrow to make white blood cells, platelets, and red blood cells. Although these effects are expected to be reversible, they can increase the risk of infection, bleeding and fatigue.

The primary purpose of this study is to find the highest dose of LEE011 that can be safely given to adult patients with advanced solid tumors or lymphomas for which no further effective standard treatment is available. It will provide information about the side effects that may occur following treatment. The study will also possibly provide early evidence for LEE011's anti-tumor activity.

location_country:
United States
Netherlands
France

location_facility: Sarah Cannon Research Institute DeptofSarahCannonRes.Inst. (2)

location_facility: University of Michigan Comprehensive Cancer Center SC


title: A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease

summary: A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.

lead_sponsor: BioMarin Pharmaceutical

location_country:
Germany
United Kingdom
Australia
United States
France

location_facility: Hôpital de I´Archet- Centre Hospitalier Universitaire Nice

location_facility: University of Florida College of Medicine

location_facility: Old Queen Elizabeth Hospital, Department of Medicine

location_facility: Univ of California San Diego School of Medicine


title: Double Blind Randomized Phase III Study of Maintenance Pazopanib Versus Placebo in NSCLC Patients Non Progressive After First Line Chemotherapy. MAPPING, an EORTC Lung Group Study.

summary:
RATIONALE: Pazopanib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor. It is not yet known whether pazopanib hydrochloride is more effective than a placebo in treating patients with non-small cell lung cancer that has not progressed after first-line chemotherapy.

PURPOSE: This randomized phase II/III trial is studying how well giving pazopanib hydrochloride works and compares it with giving a placebo in treating patients with non-small cell lung cancer who have received first-line chemotherapy.

location_country:
Germany
Slovenia
United Kingdom
Netherlands
Belgium
Greece
Egypt
France

location_facility: Centre Hospitalier Regional De La Citadelle

location_facility: The Netherlands Cancer Institute-Antoni Van Leeuwenhoekziekenhuis

location_facility: Assistance Publique - Hôpitaux de Marseille - Assistance Publique - Hôpitaux de Marseille - Hopital Nord

location_facility: Nottingham University Hospitals NHS Trust


type: Registry Identifier

title: A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.

summary:
This trial is conducted in Europe, Japan and the United States of America (USA).

The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.

ReferenceCitation: Negrier C, Knobe K, Tiede A, Giangrande P, Moss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.

ReferenceCitation: Collins PW, Moss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost. 2012 Nov;10(11):2305-12. doi: 10.1111/jth.12000.

lead_sponsor: Novo Nordisk A/S

location_country:
Germany
United Kingdom
Denmark
Japan
Sweden
United States
Spain
France


summary: This trial is conducted in Europe. The aim of this clinical trial is to investigate the safety, tolerability, pharmacokinetics (the determination of the concentration of the administered medication in blood over time) and pharmacodynamics (the determination of the effect over time and the duration of action) of a long-acting growth hormone (NNC126-0083) in growth hormone deficient children.

ReferenceType: result

ReferenceCitation: de Schepper J, Rasmussen MH, Gucev Z, Eliakim A, Battelino T. Long-acting pegylated human GH in children with GH deficiency: a single-dose, dose-escalation trial investigating safety, tolerability, pharmacokinetics and pharmacodynamics. Eur J Endocrinol. 2011 Sep;165(3):401-9. doi: 10.1530/EJE-11-0536. Epub 2011 Jul 1.

ReferenceCitation: Papathanasiou T, Agerso H, Damholt BB, Hojby Rasmussen M, Kildemoes RJ. Population Pharmacokinetics and Pharmacodynamics of Once-Daily Growth Hormone Norditropin(R) in Children and Adults. Clin Pharmacokinet. 2021 Sep;60(9):1217-1226. doi: 10.1007/s40262-021-01011-3. Epub 2021 Apr 17.

title: A Randomised, Open-labelled, Single Dose, Dose-escalation Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pegylated Long-acting Human Growth Hormone (NNC126-0083) Compared to Norditropin NordiFlex® in Growth Hormone Deficient Children

location_country: Macedonia, The Former Yugoslav Republic of


title: Clinical Investigation of the Freedom SOLO Stentless Heart Valve

summary: This is a trial to demonstrate the safety and effectiveness of the Freedom SOLO heart valve when used to replace a diseased or dysfunctional aortic valve or aortic valve prosthesis.

ReferenceCitation: Thalmann M, Grubitzsch H, Matschke K, Glauber M, Tan E, Francois K, Amorim MJ, Hensens AG, Cesari F, Feyrer R, Diegeler A, Veit F, Repossini A; Freedom Solo Investigators. A European Multicenter Study of 616 Patients Receiving the Freedom Solo Stentless Bioprosthesis. Ann Thorac Surg. 2016 Jan;101(1):100-8. doi: 10.1016/j.athoracsur.2015.06.096. Epub 2015 Oct 9.

lead_sponsor: Corcym S.r.l

location_country: Germany Italy Portugal Netherlands Austria Belgium Switzerland France

location_facility: Ospedale del Cuore, Fondazione G. Monasterio

location_facility: Herzzentrum Dresden Universitätsklinik an der Technischen Universität Dresden

location_facility: Universitätsklinikum Erlangen, Klinik für Herzchirurgie


title: An Open Label, Single-arm, Baseline-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Immunogenicity of Subcutaneous HX575 Administered Once a Week (qw) and Once Every Two Weeks (q2w) in the Treatment of Anemia Associated With Chronic Kidney Disease in Pre-dialysis and Dialysis Subjects

acronym: SWEEP

summary:
This is an open-label, single-arm, baseline-controlled, multicenter efficacy and safety switch study involving 500 CKD subjects suffering from anemia and treated previously with a stable dose of ESA s.c.

Correction of anemia will be maintained by s.c. administration of HX575 in two frequencies (i.e. qw and q2w), in order to maintain an Hb target range of 10.0-12.0 g/dL.

status: Withdrawn

lead_sponsor: Sandoz

location_country: Germany Romania Spain Bulgaria France

location_facility: Spitalul Clinic de Nefrologie Dr. Carol Davila

location_facility: Spitalul Universitar de Urgenta Bucuresti

location_facility: Nierenzentrum Weinheim Kreiskrankenhaus Weinheim

location_facility: Centre Hospitalier Universitaire Limoges, Hôpital Dupuytren

location_facility: Gemeinschaftspraxis Dr. Spitthöver, Dr. Knee, Dr. Gröschel

location_facility: Gesundheitszentrum Alzey Internistische Gemeinschaftspraxis

location_facility: Spitalul Clinic Municipal "Dr. Gavril Curteanu"


lead_sponsor: Bayer

summary: This is a uncontrolled, open-label, non-randomized Phase II study of oral BAY73-4506 to evaluate the response rate of BAY73-4506 in previously untreated patients with metastatic or unresectable renal cell cancer (RCC).

ReferenceCitation: Eisen T, Joensuu H, Nathan PD, Harper PG, Wojtukiewicz MZ, Nicholson S, Bahl A, Tomczak P, Pyrhonen S, Fife K, Bono P, Boxall J, Wagner A, Jeffers M, Lin T, Quinn DI. Regorafenib for patients with previously untreated metastatic or unresectable renal-cell carcinoma: a single-group phase 2 trial. Lancet Oncol. 2012 Oct;13(10):1055-62. doi: 10.1016/S1470-2045(12)70364-9. Epub 2012 Sep 6.

title: A Phase II Uncontrolled Study of BAY73-4506 in Previously Untreated Patients With Metastatic or Unresectable Renal Cell Cancer (RCC)

location_country:
Germany
Finland
Poland
United Kingdom
United States
France


title: Efficacy Evaluation of Intra-articular Hyaluronic Acid (Sinovial®) vs Synvisc® in the Treatment of Symptomatic Knee Osteoarthritis. A Double-blind, Controlled, Randomised, Parallel-group Non-inferiority Study

summary: The purpose of this study is to prove the non-inferiority of the intra-articular injection of hyaluronic acid (Sinovial®) in the symptomatic treatment of knee OA in comparison to Synvisc®.

ReferenceCitation: Theiler R, Bruhlmann P. Overall tolerability and analgesic activity of intra-articular sodium hyaluronate in the treatment of knee osteoarthritis. Curr Med Res Opin. 2005 Nov;21(11):1727-33. doi: 10.1185/030079905X65547.

ReferenceCitation: Castellacci E, Polieri T. Antalgic effect and clinical tolerability of hyaluronic acid in patients with degenerative diseases of knee cartilage: an outpatient treatment survey. Drugs Exp Clin Res. 2004;30(2):67-73.

ReferenceCitation: Jordan KM, Arden NK, Doherty M, Bannwarth B, Bijlsma JW, Dieppe P, Gunther K, Hauselmann H, Herrero-Beaumont G, Kaklamanis P, Lohmander S, Leeb B, Lequesne M, Mazieres B, Martin-Mola E, Pavelka K, Pendleton A, Punzi L, Serni U, Swoboda B, Verbruggen G, Zimmerman-Gorska I, Dougados M; Standing Committee for International Clinical Studies Including Therapeutic Trials ESCISIT. EULAR Recommendations 2003: an evidence based approach to the management of knee osteoarthritis: Report of a Task Force of the Standing Committee for International Clinical Studies Including Therapeutic Trials (ESCISIT). Ann Rheum Dis. 2003 Dec;62(12):1145-55. doi: 10.1136/ard.2003.011742.

ReferenceCitation: Recommendations for the medical management of osteoarthritis of the hip and knee: 2000 update. American College of Rheumatology Subcommittee on Osteoarthritis Guidelines. Arthritis Rheum. 2000 Sep;43(9):1905-15. doi: 10.1002/1529-0131(200009)43:93.0.CO;2-P. No abstract available.

ReferenceCitation: Altman RD. Status of hyaluronan supplementation therapy in osteoarthritis. Curr Rheumatol Rep. 2003 Feb;5(1):7-14. doi: 10.1007/s11926-003-0077-6.

ReferenceCitation: Lo GH, LaValley M, McAlindon T, Felson DT. Intra-articular hyaluronic acid in treatment of knee osteoarthritis: a meta-analysis. JAMA. 2003 Dec 17;290(23):3115-21. doi: 10.1001/jama.290.23.3115.

ReferenceCitation: Wang CT, Lin J, Chang CJ, Lin YT, Hou SM. Therapeutic effects of hyaluronic acid on osteoarthritis of the knee. A meta-analysis of randomized controlled trials. J Bone Joint Surg Am. 2004 Mar;86(3):538-45. doi: 10.2106/00004623-200403000-00012.

ReferenceCitation: Bellamy N, Campbell J, Robinson V, Gee T, Bourne R, Wells G. Viscosupplementation for the treatment of osteoarthritis of the knee. Cochrane Database Syst Rev. 2006 Apr 19;2006(2):CD005321. doi: 10.1002/14651858.CD005321.pub2.

ReferenceCitation: Recommendations for the registration of drugs used in the treatment of osteoarthritis. Group for the respect of ethics and excellence in science (GREES): osteoarthritis section. Ann Rheum Dis. 1996 Aug;55(8):552-7. doi: 10.1136/ard.55.8.552. No abstract available.

ReferenceCitation: Altman R, Brandt K, Hochberg M, Moskowitz R, Bellamy N, Bloch DA, Buckwalter J, Dougados M, Ehrlich G, Lequesne M, Lohmander S, Murphy WA Jr, Rosario-Jansen T, Schwartz B, Trippel S. Design and conduct of clinical trials in patients with osteoarthritis: recommendations from a task force of the Osteoarthritis Research Society. Results from a workshop. Osteoarthritis Cartilage. 1996 Dec;4(4):217-43. doi: 10.1016/s1063-4584(05)80101-3. No abstract available.

ReferenceCitation: Strand V, Kelman A. Outcome measures in osteoarthritis: randomized controlled trials. Curr Rheumatol Rep. 2004 Feb;6(1):20-30. doi: 10.1007/s11926-004-0080-6.

ReferenceCitation: Kirchner M, Marshall D. A double-blind randomized controlled trial comparing alternate forms of high molecular weight hyaluronan for the treatment of osteoarthritis of the knee. Osteoarthritis Cartilage. 2006 Feb;14(2):154-62. doi: 10.1016/j.joca.2005.09.003. Epub 2005 Oct 19.

ReferenceCitation: Bellamy N, Buchanan WW, Goldsmith CH, Campbell J, Stitt LW. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. J Rheumatol. 1988 Dec;15(12):1833-40.

ReferenceCitation: Bellamy N. WOMAC: a 20-year experiential review of a patient-centered self-reported health status questionnaire. J Rheumatol. 2002 Dec;29(12):2473-6. No abstract available.

ReferenceCitation: KELLGREN JH, LAWRENCE JS. Radiological assessment of osteo-arthrosis. Ann Rheum Dis. 1957 Dec;16(4):494-502. doi: 10.1136/ard.16.4.494. No abstract available.

ReferenceCitation: Altman R, Asch E, Bloch D, Bole G, Borenstein D, Brandt K, Christy W, Cooke TD, Greenwald R, Hochberg M, et al. Development of criteria for the classification and reporting of osteoarthritis. Classification of osteoarthritis of the knee. Diagnostic and Therapeutic Criteria Committee of the American Rheumatism Association. Arthritis Rheum. 1986 Aug;29(8):1039-49. doi: 10.1002/art.1780290816.

ReferenceCitation: Lockman LE. Practice tips. Knee joint injections and aspirations: the triangle technique. Can Fam Physician. 2006 Nov;52(11):1403-4. No abstract available.

ReferenceCitation: Lequesne MG, Mery C, Samson M, Gerard P. Indexes of severity for osteoarthritis of the hip and knee. Validation--value in comparison with other assessment tests. Scand J Rheumatol Suppl. 1987;65:85-9. doi: 10.3109/03009748709102182. Erratum In: Scand J Rheumatol 1988;17(3):following 241. Scand J Rheumatol Suppl 1988;73:1.

ReferenceCitation: Lequesne M, Samson M, Gerard P, Mery C. [Pain-function indices for the follow-up of osteoarthritis of the hip and the knee]. Rev Rhum Mal Osteoartic. 1990 Oct 30;57(9 ( Pt 2)):32S-36S. French.

ReferenceCitation: Ali Y, Weinstein M, Jokl P. Acute pseudogout following intra-articular injection of high molecular weight hyaluronic acid. Am J Med. 1999 Dec;107(6):641-2. doi: 10.1016/s0002-9343(99)00255-7. No abstract available.

ReferenceCitation: Luzar MJ, Altawil B. Pseudogout following intraarticular injection of sodium hyaluronate. Arthritis Rheum. 1998 May;41(5):939-40. doi: 10.1002/1529-0131(199805)41:53.0.CO;2-D. No abstract available.

ReferenceCitation: Maillefert JF, Hirschhorn P, Pascaud F, Piroth C, Tavernier C. Acute attack of chondrocalcinosis after an intraarticular injection of hyaluronan. Rev Rhum Engl Ed. 1997 Oct;64(10):593-4. No abstract available.

ReferenceCitation: Allen E, Krohn K. Adverse reaction to Hylan GF-20. J Rheumatol. 2000 Jun;27(6):1572. No abstract available.

ReferenceCitation: Chen AL, Desai P, Adler EM, Di Cesare PE. Granulomatous inflammation after Hylan G-F 20 viscosupplementation of the knee : a report of six cases. J Bone Joint Surg Am. 2002 Jul;84(7):1142-7.

ReferenceCitation: Zardawi IM, Chan I. Synvisc perisynovitis. Pathology. 2001 Nov;33(4):519-20. doi: 10.1080/00313020120083296.

ReferenceCitation: Hamburger MI, Lakhanpal S, Mooar PA, Oster D. Intra-articular hyaluronans: a review of product-specific safety profiles. Semin Arthritis Rheum. 2003 Apr;32(5):296-309. doi: 10.1053/sarh.2002.50008.

ReferenceCitation: Ehrich EW, Davies GM, Watson DJ, Bolognese JA, Seidenberg BC, Bellamy N. Minimal perceptible clinical improvement with the Western Ontario and McMaster Universities osteoarthritis index questionnaire and global assessments in patients with osteoarthritis. J Rheumatol. 2000 Nov;27(11):2635-41.

ReferenceCitation: Angst F, Aeschlimann A, Michel BA, Stucki G. Minimal clinically important rehabilitation effects in patients with osteoarthritis of the lower extremities. J Rheumatol. 2002 Jan;29(1):131-8.

ReferenceCitation: Bijlsma JW. Patient centred outcomes in osteoarthritis. Ann Rheum Dis. 2005 Jan;64(1):1-2. doi: 10.1136/ard.2004.025072. No abstract available.

ReferenceCitation: Tubach F, Ravaud P, Baron G, Falissard B, Logeart I, Bellamy N, Bombardier C, Felson D, Hochberg M, van der Heijde D, Dougados M. Evaluation of clinically relevant changes in patient reported outcomes in knee and hip osteoarthritis: the minimal clinically important improvement. Ann Rheum Dis. 2005 Jan;64(1):29-33. doi: 10.1136/ard.2004.022905. Epub 2004 Jun 18.

ReferenceCitation: Pavelka K, Uebelhart D. Efficacy evaluation of highly purified intra-articular hyaluronic acid (Sinovial((R))) vs hylan G-F20 (Synvisc((R))) in the treatment of symptomatic knee osteoarthritis. A double-blind, controlled, randomized, parallel-group non-inferiority study. Osteoarthritis Cartilage. 2011 Nov;19(11):1294-300. doi: 10.1016/j.joca.2011.07.016. Epub 2011 Aug 16.

lead_sponsor: IBSA Institut Biochimique SA

location_country:
Germany
Italy
Slovakia
Switzerland
Czech Republic
France

location_facility: Centrum léčby pohybového aparátu spol. s.r.o.

location_facility: Unità Operativa di Reumatologia - Istituto Ortopedico Gaetano Pini

location_facility: Sezione Dipartimentale a valenza provinciale di Reumatologia - AzUSL 8

location_facility: University Hospital Centre Bois-Guillaume

location_facility: University Hospital Centre of Limoges - Rheumatology Service

location_facility: Interni hemato-onkologická klinika, Fakultní nemocnice

location_facility: Department of Rheumatology and Institute of Physical Medicine - Zurich University

location_facility: Orthopädische Universitätsklinik und Poliklinik der RWTH Aachen

location_facility: National Institute of Rheumatic Diseases

location_facility: Medicina e Traumatologia dello Sport - AzUSL 2 Lucca

location_facility: Unità Operativa di Reumatologia - Az. Ospedaliera Universitaria Senese - Policlinico "Le Scotte"


title: Clinical Study on Tacrolimus Ointment Over the Long-term. "CONTROL Study - Children"

summary: A long-term 0.03% tacrolimus ointment based regimen comprising of up to 6 weeks of initial twice daily treatment and subsequent twice weekly prophylactic application can effectively treat active lesions of atopic dermatitis, and prevent, delay, and reduce flares.

ReferenceCitation: Thaci D, Reitamo S, Gonzalez Ensenat MA, Moss C, Boccaletti V, Cainelli T, van der Valk P, Buckova H, Sebastian M, Schuttelaar ML, Ruzicka T; European Tacrolimus Ointment Study Group. Proactive disease management with 0.03% tacrolimus ointment for children with atopic dermatitis: results of a randomized, multicentre, comparative study. Br J Dermatol. 2008 Dec;159(6):1348-56. doi: 10.1111/j.1365-2133.2008.08813.x. Epub 2008 Sep 6.

lead_sponsor: Astellas Pharma Inc

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


title: A Double-blind, Randomised Study Comparing the Safety and Tolerance of SMOFlipid 20% in Long-term Treatment With Parenteral Nutrition

summary: The aim of this study is to evaluate the safety and tolerance of SMOFlipid 20% in comparison to a standard lipid emulsion Intralipid 20% in patients requiring long-term parenteral nutrition. The safety and tolerance will be evaluated by biochemistry, hematology and coagulation variables, vital signs and adverse events. Further objectives to evaluate are the influence of SMOFlipid 20% on inflammation processes, the efficacy of anti-oxidative properties of vitamin E supplemented to SMOFlipid 20%, and the fatty acid pattern in red blood cells and serum.

lead_sponsor: Fresenius Kabi

location_country:
France
Poland
United Kingdom
Denmark
Australia
Netherlands
Israel

location_facility: Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego

location_facility: Beilinson Medical Center, Department of Surgery "B"

location_facility: Wydzial Lekarski I Katedra Chirurgii Ogolnej

location_facility: University Hospital, Queen's Medical Centre

location_facility: Hopital Edouard Herriot, Unite de Nutrition a Domicile Lyon

location_facility: Rigshospitalet, Department of Medical Gastroenterology

location_facility: Aalborg University Hospital, Department of Medical Gastroenterology


title: A Randomized, Double-Blind Study to Evaluate the Safety of Continued Treatment With CellCept in Patients With Well-Controlled Myasthenia Gravis Receiving a Stable Dose of Prednisone

summary: This 2 arm study will provide optional continuation of double-blind treatment with CellCept or placebo, in patients with myasthenia gravis who have achieved good symptom control in study WX17798. Patients who have completed 36 weeks of treatment in study WX17798, with stable prednisone dosing for the last 4 weeks, can continue on blinded treatment with CellCept (1g bid) or placebo until the database for WX17798 is locked and unblinded. The anticipated time on study treatment is 3-12 months, and the target sample size is 100-500 individuals.

sponsor_collaborators:
Hoffmann-La Roche
Aspreva Pharmaceuticals

location_country:
Germany
Italy
United Kingdom
United States
Former Serbia and Montenegro
Ukraine
France


title: A Phase I Dose Escalation Study of BMS-690514 in Patients With Advanced or Metastatic Solid Tumors

summary: The purpose of this research study is to determine the highest dose of the drug (BMS-690514) that can be safely given to patients with advanced cancer.

ReferenceCitation: Soria JC, Baselga J, Hanna N, Laurie SA, Bahleda R, Felip E, Calvo E, Armand JP, Shepherd FA, Harbison CT, Berman D, Park JS, Zhang S, Vakkalagadda B, Kurland JF, Pathak AK, Herbst RS. Phase I-IIa study of BMS-690514, an EGFR, HER-2 and -4 and VEGFR-1 to -3 oral tyrosine kinase inhibitor, in patients with advanced or metastatic solid tumours. Eur J Cancer. 2013 May;49(8):1815-24. doi: 10.1016/j.ejca.2013.02.012. Epub 2013 Mar 13.

lead_sponsor: Bristol-Myers Squibb

location_country:
United States
Spain
Canada
France

location_facility: The University Of Texas Md Anderson Cancer Center


title: An Extension to a Phase II Study to Determine the Safety and Anti-Leukemic Effects of STI571 in Adult Patients With Philadelphia Chromosome Positive Leukemia Including Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Accelerated Phase Chronic Myeloid Leukemia

summary:
The objectives of Part 1 of the study were:

To determine the rate of hematologic response (HR) lasting ≥4 weeks in participants with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the accelerated phase (AP).
To evaluate duration of HR, overall survival, cytogenetic response (CyR), time to blast crisis in CML participants in the AP, improvement of symptomatic parameters, tolerability and safety of STI571 treatment.

The objective of the extension (Part 2) was:

-To enable participants to have access to study drug and continue study treatment and to decrease data collection to include only overall survival and serious adverse events.

lead_sponsor: Novartis Pharmaceuticals

location_country:
Germany
Italy
United Kingdom
United States
France

location_facility: MD Anderson Cancer Center, University of Texas


title: A Randomized Phase II-III Trial Evaluating the Efficacy of Capecitabine and Vinorelbine in Anthracycline and Taxane Pre-Treated Metastatic Breast Cancer

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known if capecitabine is more effective than vinorelbine in treating metastatic breast cancer.

PURPOSE: Randomized phase II/III trial to compare the effectiveness of capecitabine with that of vinorelbine in treating women who have metastatic breast cancer that has been previously treated with chemotherapy.

ReferenceCitation: Pajk B, Cufer T, Canney P, Ellis P, Cameron D, Blot E, Vermorken J, Coleman R, Marreaud S, Bogaerts J, Basaran G, Piccart M. Anti-tumor activity of capecitabine and vinorelbine in patients with anthracycline- and taxane-pretreated metastatic breast cancer: findings from the EORTC 10001 randomized phase II trial. Breast. 2008 Apr;17(2):180-5. doi: 10.1016/j.breast.2007.09.002. Epub 2007 Oct 31.

ReferenceCitation: Hoon SN, Lau PK, White AM, Bulsara MK, Banks PD, Redfern AD. Capecitabine for hormone receptor-positive versus hormone receptor-negative breast cancer. Cochrane Database Syst Rev. 2021 May 26;5(5):CD011220. doi: 10.1002/14651858.CD011220.pub2.

repository_institution: UWA - UWA Profiles and Research Repository

repositories:
PubMed Central
Pure (Denmark)

repositories_concat: PubMed Central;Pure (Denmark)

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

location_country:
Germany
Slovenia
United Kingdom
Belgium
France

location_facility: Cancer Research Centre at Weston Park Hospital


title: Randomized Phase III Trial of Two Investigational Schedules of Ifosfamide vs. Standard Dose Doxorubicin in Patients With Advanced or Metastatic Soft Tissue Sarcoma

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known whether ifosfamide or doxorubicin is more effective for advanced or metastatic soft tissue sarcoma.

PURPOSE: Randomized phase III trial to compare the effectiveness of ifosfamide with that of doxorubicin in treating patients who have advanced or metastatic soft tissue sarcoma.

ReferenceCitation: Lorigan P, Verweij J, Papai Z, Rodenhuis S, Le Cesne A, Leahy MG, Radford JA, Van Glabbeke MM, Kirkpatrick A, Hogendoorn PC, Blay JY; European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. Phase III trial of two investigational schedules of ifosfamide compared with standard-dose doxorubicin in advanced or metastatic soft tissue sarcoma: a European Organisation for Research and Treatment of Cancer Soft Tissue and Bone Sarcoma Group Study. J Clin Oncol. 2007 Jul 20;25(21):3144-50. doi: 10.1200/JCO.2006.09.7717.

location_country:
Germany
Poland
Slovakia
Hungary
United Kingdom
Denmark
Netherlands
Belgium
Spain
France

location_facility: Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology

location_facility: Middlesex Hospital- Meyerstein Institute


title: Phase II Study of the Efficacy of rH Interleukin-2 in Patients With Slowly Progressing Acute Myelogenous Leukemia (AML) and With Limited Bone Marrow Blastosis After Autologous Stem Cell Transplantation or Chemotherapy

summary:
RATIONALE: Interleukin-2 may stimulate a person's white blood cells to kill acute myelogenous leukemia cells.

PURPOSE: Phase II trial to study the effectiveness of interleukin-2 in treating patients with acute myelogenous leukemia that has relapsed following previous treatment.

location_country: Italy Netherlands Austria Belgium Croatia France

location_facility:
Institut Jules Bordet
University Medical Center Nijmegen
Universitair Ziekenhuis Antwerpen
Leiden University Medical Center
Innsbruck Universitaetsklinik
University Hospital Rebro
Hotel Dieu de Paris
Institut Gustave Roussy
Azienda Policlinico Umberto Primo
Ospedale San Eugenio


title: A Phase 1, 2-Part, Multicenter, First-In-Human Dose-Escalation and Dose-Expansion Study of DS-1103a Combination Therapy in Subjects With Advanced Solid Tumors

summary: This study will evaluate the safety and efficacy of DS-1103a combination therapy in patients with advanced solid tumors.

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

lead_sponsor: Daiichi Sankyo, Inc.

sponsor_collaborators:
Daiichi Sankyo, Inc.
AstraZeneca

location_facility: Princess Margaret Cancer Centre, University Health Network


title: A Phase IIa Multicenter, Open-Label, 12-Week Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype

acronym: Aldebaran

summary: This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion Angelman Syndrome (AS) aged 5-17 years (inclusive) will be enrolled in the study.

ipd_sharing_description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
Australia
United States
Spain
France

location_facility: Ospedale Pediatrico Bambino Gesù; Dip. Neuroscienze e Neuroriabilitazione

location_facility: IRCCS Istituto G. Gaslini; UOC Neurologia Pediatrica e Malattie Muscolari

location_facility: Hopital la Timone Enfants; Service de Pediatrie et Neurologie Pediatrique

location_facility: Carolina Institute for Development Disabilities University of North Carolina/School of Medicine

location_facility: IRCCS Eugenio Medea; U.O. di Epilessia e Neurofisiologia clinica

location_facility: Groupe Hospitalier Necker Enfants Malades


title: A Phase 1 Study of the SHP2 Inhibitor BBP-398 (Formerly Known as IACS-15509) in Combination With the KRAS-G12C Inhibitor Sotorasib in Patients With Advanced Solid Tumors and a KRAS-G12C Mutation

acronym: Argonaut

summary:
This is a Phase 1 study of BBP-398, a SHP2 inhibitor, in combination with sotorasib, a KRAS-G12C inhibitor (KRAS-G12Ci), in patients with a KRAS-G12C mutation.

The study involves 2 parts: Phase 1a Dose Escalation and Phase 1b Dose Expansion/Optimization.

lead_sponsor: Navire Pharma Inc., a BridgeBio company

sponsor_collaborators:
Navire Pharma Inc., a BridgeBio company
Amgen

location_country: Italy Australia Spain France

location_facility: Peninsula & South Eastern Haematology and Oncology Group

location_facility: Hospital Universitario Virgen De La Macarena

location_facility: Southern Oncology Clinical Research Unit


title: A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)

summary: The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).

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
Korea, Republic of
Spain
Canada
France


title: An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia)

acronym: HELIA

summary: PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.

lead_sponsor: ProQR Therapeutics

location_country:
United States
Canada
France

location_facility: Centre de maladies rares CHNO des Quinze Vingts

location_facility: Casey Eye Institute, Oregon Health & Science University

location_facility: University of Michigan, Kellogg Eye Center

location_facility: Centre for Innovative Medicine, Department of Paediatric Surgery, Montreal Children's Hospital at the McGill University Health Centre

location_facility: Center for Clinical Research Operations, Massachusetts Eye and Ear

location_facility: Hôpital Gui de Chauliac - CHRU de Montpellier - Maladies Sensorielles Génétique


title: A Phase 1b Study to Evaluate the Efficacy and Safety of JNJ-64251330, a Janus Kinase (JAK) Inhibitor, in Participants With Familial Adenomatous Polyposis

summary: The purpose of this study is to determine the effect of JNJ-64251330 in participants with Familial Adenomatous Polyposis (FAP) on colorectal polyp burden (sum of the polyp diameters).

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
Germany
Netherlands
United States
Korea, Republic of
Spain
Puerto Rico
France

location_facility: Severance Hospital, Yonsei University Health System

location_facility: University of Texas MD Anderson Cancer Center

location_facility: Pan American Center for Oncology Trials LLC

location_facility: Academisch Medisch Centrum Universiteit van Amsterdam


title: A Modular Phase II, Open-Label, Multicentre Study to Assess the Efficacy and Safety of Capivasertib in Patients With Relapsed or Refractory B-cell Non-Hodgkin Lymphoma (CAPITAL)

acronym: CAPITAL

summary: This study is an open-label, multicenter Phase II study of capivasertib administered orally in participants with Relapsed or Refractory (R/R) B-cell Non-Hodgkin's Lymphoma (NHL).

ipd_sharing_description:
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal. All requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com /ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

lead_sponsor: AstraZeneca

sponsor_collaborators: AstraZeneca Parexel

location_country:
United Kingdom
Denmark
United States
Korea, Republic of
Spain
Canada
France


lead_sponsor: Ultragenyx Pharmaceutical Inc

summary: The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

location_facility: University of Texas, Health Science Center of Houston

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

location_facility: Institut de Myologie - Hopital Antoine Béclère

title: A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III

location_country:
Italy
United Kingdom
United States
Spain
Canada
France


title: A Multicentre, Prospective, Open-label, Uncontrolled Phase 3 Study to Assess the Efficacy, Safety and Pharmacokinetics of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

summary: Assess the incidence of the composite of thrombotic events (TEs) and thromboembolic events (TEEs) in patients with congenital antithrombin deficiency under cover of Atenativ for surgical procedures or parturition.

primary_purpose: Prevention

lead_sponsor: Octapharma

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

location_facility: Universitätsklinik für Innere Medizin Klinische Abteilung für Hämatologie und Hämostaseologie

location_facility: Universita degli Studi di Milano - IRCCS Ospedale Maggiore Policlinico - Centro Emofilia e Trombosi Angelo Bianchi Bonomi

location_facility: Klinik fur Angiologie Hamostaseologie Haus 12 A Gerinnungssprechstunde

location_facility: University of Debrecen, Medical and Health Science Centre

location_facility: Centre Hospitalier Universitaire de Rouen (CHU de Rouen) - Centre de Traitement des Maladies Hemorragiques (CRTH) (Centre d'Hemophiles)

location_facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

location_facility: Bleeding and Clotting Disorders Institute

location_facility: Unita Strutturale Complessa di Immunoematologia e Medicina Trasfusionale -Dipartimento Interaziendale di Medicina Trasfusionale ed Ematologia - ASST Papa Giovanni XXIII ematologia Piazza OMS, 1


acronym: HERO

lead_sponsor: Myovant Sciences GmbH

summary: The purpose of this study is to determine the efficacy and safety of relugolix 120 milligrams (mg) orally once daily for 48 weeks on maintaining serum testosterone suppression to castrate levels (< 50 nanograms/deciliter [ng/dL]) in participants with androgen-sensitive advanced prostate cancer.

ReferenceCitation: Shore ND, Saad F, Cookson MS, George DJ, Saltzstein DR, Tutrone R, Akaza H, Bossi A, van Veenhuyzen DF, Selby B, Fan X, Kang V, Walling J, Tombal B; HERO Study Investigators. Oral Relugolix for Androgen-Deprivation Therapy in Advanced Prostate Cancer. N Engl J Med. 2020 Jun 4;382(23):2187-2196. doi: 10.1056/NEJMoa2004325. Epub 2020 May 29.

ReferenceCitation: Shore ND, Sutton J. Plain language summary of the HERO study comparing relugolix with leuprolide for men with advanced prostate cancer. Future Oncol. 2022 Jul;18(21):2575-2584. doi: 10.2217/fon-2022-0172. Epub 2022 May 19.

evidence: open (via page says license)

publisher_dissemination: Future Science Ltd

location_facility:
Daegu
Valencia
Sapporo
Orange
Greensboro
Passo Fundo
London
Scunthorpe
Brick
Yokohama
Rhyl
Omaha
Brussels
Cremona
Kaohsiung City
Nitra
Lyon
Salamanca
Teresina
Planegg
Tucson
Barcelona
Alborg
Wahroonga
Jeffersonville
Suzhou
Tauranga
San Antonio
Siedlce
Vejle
Kosice
Nashville
Bunkyō-Ku
Quebec
Osaka
Sherbrooke
Braunschweig
Nottingham
Calgary
Milano
Orebro
Rome
Herlev
Pompano Beach
Nakano-ku
Sumida-ku
Creteil
Hiroshima
Ube
Montreal
Goyang-Si
Middleburg Heights
Itabuna
Tampere
Porto Alegre
Maebashi
Gent
Des Moines
Chongqing
Nagasaki
Martin
Plainview
Oviedo
Durham
Taiyuan
Uppsala
Exeter
Beijing
Hwasun
Pierre Benite
Kita-gun
Suita
Fukuoka
Bratislava
Sneek
Ijuí
Beijing Shi
Seinajoki
Lanzhou
Strasbourg
Las Vegas
Turku
Presov
Tokyo
Baltimore
Kanazawa-shi
Kortrijk
Taipei
Tweed Heads
Myrtle Beach
Lublin
Malmo
Busan
Albany
Syracuse
Poughkeepsie
Helsinki
Linz
Warszawa
Vancouver
Cincinnati
Seoul
A Coruna
Hangzhou
Nanjing
Amsterdam
Orbassano
Candiolo
Emmendingen
Nanchang
Aarhus
Salvador
Arezzo
Denver
Munster
Trencin
Meldola
Hamilton
Katowice
Curitiba
Sala
Wichita
Changchun
Southport
Dunedin
Gdynia
Redcliffe
Joinville
Sendai
Camperdown
Lancaster
Halifax
Christchurch
Eindhoven
Poprad
Stockholm
Dresden
Chiba
Kyoto
São José Do Rio Preto
Shanghai
Oklahoma City
Natal
Troy
Lubeck
Madrid
Garden City
Albuquerque
Osaka-sayama

title: HERO: A Multinational Phase 3 Randomized, Open-label, Parallel Group Study to Evaluate the Safety and Efficacy of Relugolix in Men With Advanced Prostate Cancer

location_country:
Finland
Germany
Poland
New Zealand
Australia
Brazil
Japan
Canada
France
Slovakia
Denmark
United States
Belgium
China
Italy
United Kingdom
Netherlands
Austria
Korea, Republic of
Spain
Taiwan
Sweden


acronym: ASPEN

lead_sponsor: BeiGene

summary: This study evaluated the safety, efficacy and clinical benefit of BGB-3111 (zanubrutinib) vs ibrutinib in participants with MYD88 Mutation Waldenström's Macroglobulinemia.

ReferenceCitation: Tam CS, LeBlond V, Novotny W, Owen RG, Tedeschi A, Atwal S, Cohen A, Huang J, Buske C. A head-to-head Phase III study comparing zanubrutinib versus ibrutinib in patients with Waldenstrom macroglobulinemia. Future Oncol. 2018 Sep;14(22):2229-2237. doi: 10.2217/fon-2018-0163. Epub 2018 Jun 5.

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

location_facility: Hospital Duran i Reynals, Instituto Catalán de Oncología

location_facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz

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

location_facility: PO A.Ferrarotto, AOU Policlinico-Vittorio Emanuele Catania

location_facility: Hospital Universitari i Politècnic La Fe

location_facility: Universitätsklinikum Münster Hämatologie und Onkologie

location_facility: The Royal Bournemouth and Christchurch Hospitals NHS Foundation

location_facility: Azienda Ospedaliera "Maggiore della Carità" di Novara

location_facility: AZ Sint-Jan Brugge - Oostende - Campus Sint-Jan

location_facility: Szpital Specjalist. w Brzozowie,Podkarpacki Ośrodek Onkologiczny

location_facility: PU A. Gemelli, Universität Cattolica del Sacro Cuore

location_facility: Barwon Health, University Hospital Geelong

location_facility: Desert Hematology Oncology Medical Group Inc.

location_facility: Niguarda Cancer Center Division of Hematology

location_facility: Uniwersytecki Szpital Kliniczny w Białymstoku

location_facility: Szpitale Pomorskie Sp. z o.o., Szpital Morski im. PCK Gdansk

location_facility: Università degli Studi di Roma "La Sapienza"

location_facility: Instytut Hematologii i Transfuzjologii w Warszawie

location_facility: Germans Trias i Pujol University Hospital

location_facility: Complejo Asistencial Universitario de Salamanca

location_facility: Azienda Ospedaliera Spedali Civili Di Brescia

location_facility: University of California San Diego (UCSD) - Moores Cancer Center

location_facility: Hospital Universitario Fundación Jiménez Díaz

location_facility: Nottingham University Hospitals NHS Trust

title: A Phase 3, Randomized, Open-Label, Multicenter Study Comparing the Efficacy and Safety of the Bruton's Tyrosine Kinase (BTK) Inhibitors BGB-3111 and Ibrutinib in Subjects With Waldenström's Macroglobulinemia (WM)

location_country:
Germany
Italy
Poland
United Kingdom
Australia
Sweden
Netherlands
Belgium
United States
Spain
Greece
Czechia
France


summary: This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

location_facility: Radboud University Nijmegen Medical Centre; Ophthalmology

location_facility: National Center of Neurology and Psychiatry

location_facility: Yale University School of Medicine ; Pulmonary & Critical Care

location_facility: Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia

location_facility: Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital

location_facility: Alder Hey Children s Hospital; Department of Pediatrics

location_facility: Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia

location_facility: National Hospital Organization Osaka Toneyama Medical Center

location_facility: Hotel Dieu; Service Pharmacie Essais Cliniques

location_facility: Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.

location_facility: UCL Institute of Child Health & Great Ormond Street Hospital for Children

location_facility: University of California Davis Medical Center

location_facility: Children's Hospital Westmead; Paediatrics & Child Health

location_facility: Fondazione Serena Onlus - CENTRO CLINICO NEMO

location_facility: Universitatsklinikum Essen; Innere Klinik

location_facility: University of Massachusetts Memorial Childrens Medical Center; Department of Neurology

location_facility: Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy

location_facility: Lady Cilento Children's Hospital; Neurosciences Department

location_facility: Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie

location_facility: Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin

location_facility: London Health Sciences Centre; Children's Hospital; Pediatrics

title: A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

location_country:
Germany
Italy
United Kingdom
Argentina
Australia
Japan
Netherlands
Sweden
Belgium
United States
Spain
Canada
France


lead_sponsor: GlaxoSmithKline

summary:
COPD is characterized by an airflow limitation, which is not fully reversible, usually progressive and accompanied by chronic cough, sputum production and dyspnea, which can be a major cause of disability and anxiety associated with the disease. In addition, COPD is associated with poor health-related quality of life (HRQoL). Pharmacologic therapy is used to improve lung function, reduce symptoms, reduce the frequency and severity of exacerbations, and also to improve health status and exercise tolerance.

This is a multi-center, randomized, double blind, double dummy, 3-arm parallel group study to compare umeclidinium/vilanterol (62.5/25 microgram [mcg], once daily), umeclidinium (62.5 mcg, once daily), and salmeterol (50 mg, twice daily) in male and female subjects with COPD. The primary purpose of this study is to demonstrate improvements in lung function for subjects treated with UMEC/VI compared with UMEC for 24 weeks.

Approximately 2424 subjects will be randomized across 3 parallel arms in 1:1:1 ratio. Subjects will be stratified based on long-acting bronchodilator usage during the run-in period (none, one or 2 long-acting bronchodilators per day). Subjects will receive either UMEC/VI inhalation powder (62.5/25 microgram [mcg] once daily) administered via the ELLIPTA® dry powder inhaler (DPI) and placebo twice daily via DISKUS® DPI; or UMEC (62.5 mcg once daily) administered via the ELLIPTA DPI and placebo twice daily via DISKUS DPI or salmeterol (50 mcg twice daily [BID]) administered via the DISKUS DPI and placebo once daily via ELLIPTA DPI. The duration of the study will be 29 to 31 weeks including a pre-screening period of 2 weeks, run-in period of 4 weeks, treatment period of 24 weeks and follow-up period of 1 week.

ELLIPTA and DISKUS are trademarks of GSK group of companies.

ReferenceCitation: Maltais F, Bjermer L, Kerwin EM, Jones PW, Watkins ML, Tombs L, Naya IP, Boucot IH, Lipson DA, Compton C, Vahdati-Bolouri M, Vogelmeier CF. Efficacy of umeclidinium/vilanterol versus umeclidinium and salmeterol monotherapies in symptomatic patients with COPD not receiving inhaled corticosteroids: the EMAX randomised trial. Respir Res. 2019 Oct 30;20(1):238. doi: 10.1186/s12931-019-1193-9.

ReferenceCitation: Bjermer LH, Boucot IH, Vogelmeier CF, Maltais F, Jones PW, Tombs L, Compton C, Lipson DA, Kerwin EM. Efficacy and Safety of Umeclidinium/Vilanterol in Current and Former Smokers with COPD: A Prespecified Analysis of The EMAX Trial. Adv Ther. 2021 Sep;38(9):4815-4835. doi: 10.1007/s12325-021-01855-y. Epub 2021 Aug 4.

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

repositories_concat: PubMed Central;openaccess.sgul.ac.uk

ReferenceCitation: Maltais F, Naya IP, Vogelmeier CF, Boucot IH, Jones PW, Bjermer L, Tombs L, Compton C, Lipson DA, Kerwin EM. Salbutamol use in relation to maintenance bronchodilator efficacy in COPD: a prospective subgroup analysis of the EMAX trial. Respir Res. 2020 Oct 22;21(1):280. doi: 10.1186/s12931-020-01451-8.

ReferenceCitation: Kerwin EM, Boucot IH, Vogelmeier CF, Maltais F, Naya IP, Tombs L, Jones PW, Lipson DA, Keeley T, Bjermer L. Early and sustained symptom improvement with umeclidinium/vilanterol versus monotherapy in COPD: a post hoc analysis of the EMAX randomised controlled trial. Ther Adv Respir Dis. 2020 Jan-Dec;14:1753466620926949. doi: 10.1177/1753466620926949.

publisher_dissemination: SAGE Publications

title: A 24-week Treatment, Multi-center, Randomized, Double-blind, Double-dummy, Parallel Group Study to Compare Umeclidinium/Vilanterol, Umeclidinium, and Salmeterol in Subjects With Chronic Obstructive Pulmonary Disease (COPD)

location_country:
Germany
Italy
Argentina
Australia
Sweden
Netherlands
Mexico
United States
Spain
South Africa
Canada
France