title: An Exploratory Multicenter, Open-Label, Single Arm Study of the Safety and Tolerability of Pirfenidone (Esbriet®) in Combination With Nintedanib (Ofev®) in Patients With Idiopathic Pulmonary Fibrosis

summary: This clinical study will evaluate the safety and tolerability of combination treatment of nintedanib and pirfenidone in participants with IPF. Eligible participants must have received pirfenidone for at least 16 weeks on a stable dose. Nintedanib will be added on Day 1 of the study as a combination treatment for IPF for 24 weeks.

ReferenceCitation: Flaherty KR, Fell CD, Huggins JT, Nunes H, Sussman R, Valenzuela C, Petzinger U, Stauffer JL, Gilberg F, Bengus M, Wijsenbeek M. Safety of nintedanib added to pirfenidone treatment for idiopathic pulmonary fibrosis. Eur Respir J. 2018 Aug 2;52(2):1800230. doi: 10.1183/13993003.00230-2018. Print 2018 Aug. Erratum In: Eur Respir J. 2018 Oct 4;52(4):

repository_institution: Erasmus University Rotterdam - EUR Research Repository

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

repository_normalized: dnet

publisher_dissemination: European Respiratory Society

location_country:
Germany
Italy
Denmark
Netherlands
United States
Spain
Canada
France

location_facility: David Geffen School of Medicine at UCLA;Division of Pulmonary & Critical Care/ Department of Medic

location_facility: South Health Campus/Alberta Health Services/ University of Calgary

location_facility: Medical University of South Carolina (MUSC); MUSC Pulmonary

location_facility: Hospital del Henares; Medicina Interna. Unidad de Neumología

location_facility: Hospital Universitario de Canarias; Servicio de Neumologia

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Neumologia

location_facility: Fachkrankenhaus Coswig GmbH Zentrum f.Pneumologie Beatmungsmedizin Thorax-u.Gefäßchirurgie

location_facility: Hospital Universitario La Princesa; Servicio de Neumologia

location_facility: Stanford University School of Medicine ; Pulmonary/Critical Care Medicine

location_facility: Ruhrlandklinik Lungenzentrum der UNI Essen Abt.Pneumologie-Allergologie

location_facility: Antonius Ziekenhuis; Dept of Lung Diseases

location_facility: Inova Health Care Services; Advanced Lung Disease Transplant Program

location_facility: A.O. Universitaria San Luigi Gonzaga di Orbassano; Malattie Apparato Respiratorio (MAR2)

location_facility: Klinikum Fulda gAG; Universitätsmedizin Marburg, Campus Fulda

location_facility: Hospital Universitari de Bellvitge ; Servicio de Neumologia

location_facility: Cardio-Pulmonary Associates of St. Luke's Hospital

location_facility: A.O. Univ. Senese Policlinico S. Maria alle Scotte; UOC Malattie Resepiratorie e Trapianto Polmonare

location_facility: Hopital de Pontchaillou; Service de Pneumologie

location_facility: Gentofte Hospital, Lungemedicinsk Afdeling

location_facility: John A. Butler, M.D. - Oregon Pulmonary Associates

location_facility: Complejo Asistencial Universitario de Leon; Pneumology

location_facility: Azienda Ospedaliero Universitaria Pisana; U.O. Pneumologia

location_facility: Hospital General Universitario De Valencia; Servicio de Neumologia


study_start_date_type: Actual

acronym: ASSENT

intervention_type: Drug

lead_sponsor: Daiichi Sankyo, Inc.

summary: This is a Phase 1b/2, double-blind (study participants and Investigators), placebo-controlled, randomized, single-ascending dose, multi-center study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DS-1040b in participants with Acute Ischemic Stroke (AIS).

location_facility: Ospedaliero di Albenga - Pietra Ligure Dept Neurology

location_facility: Hospital Clinico Universitario de Valladolid

location_facility: Hospital Clinico Universitario Lozano Blesa de Zaragoza

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Universittsklinikum Essen AR Klinik fr Neurologie

location_facility: Klinikum der Johann Wolfgang Goethe-Universitat

location_facility: Svet zdravia a.s.,Vseobecna nemocnica Rimavska Sobota

location_facility: Chang Gung Memorial Hospital-Linkou Branch

location_facility: Oregon Health Sciences University Hospital

location_facility: Seoul National University Bundang Hospital

location_facility: University of South Alabama USA Health System

location_facility: Ospedale Guglielmo da Saliceto Via Taverna

location_facility: Hospital Clínico Universitario de Santiago de Compostela

location_facility: Chattanooga Center for Neurologic Research

location_facility: University College Hospitals NHS Foundation Trust

location_facility: University of Kentucky Chandler Medical Center

status: Completed

title: A Phase 1b/2, Multi-Center, Double-Blind (Principal Investigators and Study Subjects Blinded, Sponsor Unblinded), Placebo-Controlled, Randomized, Single-Ascending Dose Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of DS-1040b in Subjects With Acute Ischemic Stroke

primary_purpose: Treatment

type: EudraCT Number

source: euctr

enrollment_type: Actual

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

design_allocation: Randomized

study_completion_date_type: Actual

location_country:
Germany
Italy
Slovakia
United Kingdom
Australia
United States
Korea, Republic of
Spain
Taiwan
Czechia
France

study_type: Interventional

ipd_sharing: Yes

lead_sponsor_type: industriel

status_simplified: Completed


title: A Phase 3, Randomized, Vehicle-Controlled, Double-Blind, Multicenter Study to Evaluate the Safety and Efficacy of Once-Daily CLS001 Topical Gel Versus Vehicle Administered for 12 Weeks to Subjects With Papulopustular Rosacea With a 4 Week Follow-up Period

summary: This study evaluates the safety and efficacy of once-daily application of CLS001 topical gel compared to vehicle gel in subjects with severe papulopustular rosacea.

lead_sponsor: Maruho Co., Ltd.

location_country:
Germany
New Zealand
United Kingdom
Australia
Sweden
Netherlands
United States
Canada
France


title: Confirmatory Validation of Oral Macimorelin as a Growth Hormone (GH) Stimulation Test (ST) for the Diagnosis of Adult Growth Hormone Deficiency (AGHD) in Comparison With the Insulin Tolerance Test (ITT)

summary: The Macimorelin Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, 2-way crossover Trial. The trial will include subjects suspected to have adult growth hormone deficiency (AGHD) and a group of healthy control subjects.

primary_purpose: Diagnostic

ReferenceType: derived

ReferenceCitation: Garcia JM, Biller BMK, Korbonits M, Popovic V, Luger A, Strasburger CJ, Chanson P, Medic-Stojanoska M, Schopohl J, Zakrzewska A, Pekic S, Bolanowski M, Swerdloff R, Wang C, Blevins T, Marcelli M, Ammer N, Sachse R, Yuen KCJ. Macimorelin as a Diagnostic Test for Adult GH Deficiency. J Clin Endocrinol Metab. 2018 Aug 1;103(8):3083-3093. doi: 10.1210/jc.2018-00665.

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via free article)

host_type: publisher

license_normalized: no license

version: publishedVersion

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

host_type: repository

repository_institution: Queen Mary University of London - Queen Mary Research Online

version: submittedVersion

unpaywall_oa_status: bronze

publisher_dissemination: The Endocrine Society

ipd_sharing: Undecided

lead_sponsor: AEterna Zentaris

location_country:
Germany
Italy
Poland
United Kingdom
Austria
United States
Spain
Serbia
France

location_facility: Baylor College of Medicine-Endocrinology

location_facility: Phase I - MTZ Clinical Research Sp. z o.o.

location_facility: Medical University & General Hospital of Vienna, AKH,

location_facility: Klinik für Endokrinologie, Diabetes und Ernährungsmedizin der Charité


lead_sponsor: Hoffmann-La Roche

summary: This early phase, multicenter, open-label, single-arm study evaluated the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary efficacy of atezolizumab in pediatric and young adult participants with solid tumors for which prior treatment was proven to be ineffective.

ReferenceCitation: Geoerger B, Zwaan CM, Marshall LV, Michon J, Bourdeaut F, Casanova M, Corradini N, Rossato G, Farid-Kapadia M, Shemesh CS, Hutchinson KE, Donaldson F, Liao M, Caron H, Trippett T. Atezolizumab for children and young adults with previously treated solid tumours, non-Hodgkin lymphoma, and Hodgkin lymphoma (iMATRIX): a multicentre phase 1-2 study. Lancet Oncol. 2020 Jan;21(1):134-144. doi: 10.1016/S1470-2045(19)30693-X. Epub 2019 Nov 25.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

ReferenceCitation: Shemesh CS, Chanu P, Jamsen K, Wada R, Rossato G, Donaldson F, Garg A, Winter H, Ruppel J, Wang X, Bruno R, Jin J, Girish S. Population pharmacokinetics, exposure-safety, and immunogenicity of atezolizumab in pediatric and young adult patients with cancer. J Immunother Cancer. 2019 Nov 21;7(1):314. doi: 10.1186/s40425-019-0791-x.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

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

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

repositories_concat: PubMed Central

unpaywall_oa_status: gold

publisher_dissemination: British Medical Journal

location_facility: Institut Gustave Roussy; Service Pediatrique

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Universitäts-Kinderspital; Abteilung für Onkologie

location_facility: Leeds General Infirmary; Paediatric Oncology & Haematology

location_facility: Hospital Infantil Universitario Nino Jesus

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori; Struttura Complessa di Pediatria Oncologica

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: The Royal Victoria Infirmary; Paediatric and Adolescent Oncology Unit

location_facility: Centre Léon Bérard, Institut d'Hémato-Oncologie Pédiatrique

location_facility: Rigshospitalet; BØRNEUNGEKLINIKKEN, Ambulatoriet for kræft- og Blodsygdomme hos børn og unge

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

location_facility: University of Texas Health Science Center at San Antonio

location_facility: Erasmus MC / location Sophia Kinderziekenhuis

location_facility: IRCCS Istituto Giannina Gaslini; Unità Operativa Oncologica Pediatrica

location_facility: Azienda Ospedaliera di Padova; Clinica di Onco-ematologia pediatrica

location_facility: Ospedale Pediatrico Bambino Gesù - IRCCS; Dipartimento di Onco-Ematologia Pediatrica

location_facility: Stanford University/Lucile Packard Children's Hospital

status: Terminated

title: An Early-Phase, Multicenter, Open-Label Study of the Safety and Pharmacokinetics of Atezolizumab (MPDL3280A) In Pediatric and Young Adult Patients With Previously Treated Solid Tumors

location_country:
Germany
Italy
Israel
United Kingdom
Denmark
Netherlands
Switzerland
United States
Spain
Canada
France

status_simplified: Unknown


intervention_type: Other

lead_sponsor: Kyowa Kirin, Inc.

summary: The primary efficacy objective of this study is to establish the effect of burosumab treatment compared with placebo on increasing serum phosphorus levels in adults with XLH.

ReferenceType: result

ReferenceCitation: Insogna KL, Briot K, Imel EA, Kamenicky P, Ruppe MD, Portale AA, Weber T, Pitukcheewanont P, Cheong HI, Jan de Beur S, Imanishi Y, Ito N, Lachmann RH, Tanaka H, Perwad F, Zhang L, Chen CY, Theodore-Oklota C, Mealiffe M, San Martin J, Carpenter TO; AXLES 1 Investigators. A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti-FGF23 Antibody, in Adults With X-Linked Hypophosphatemia: Week 24 Primary Analysis. J Bone Miner Res. 2018 Aug;33(8):1383-1393. doi: 10.1002/jbmr.3475. Epub 2018 Jun 26.

oa_colors: green hybrid

evidence: open (via crossref license)

repository_institution: Indiana University - Purdue University Indianapolis - IUPUI ScholarWorks

repository_institution: Indiana University - IUScholarWorks

unpaywall_oa_status: hybrid

publisher_dissemination: Wiley

ReferenceCitation: Portale AA, Carpenter TO, Brandi ML, Briot K, Cheong HI, Cohen-Solal M, Crowley R, Jan De Beur S, Eastell R, Imanishi Y, Imel EA, Ing S, Ito N, Javaid M, Kamenicky P, Keen R, Kubota T, Lachmann R, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Zhang L, Theodore-Oklota C, Mealiffe M, San Martin J, Insogna K. Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period. Calcif Tissue Int. 2019 Sep;105(3):271-284. doi: 10.1007/s00223-019-00568-3. Epub 2019 Jun 4.

repository_institution: University of Edinburgh - Edinburgh Research Explorer

repository_institution: University of Oxford - Oxford University Research Archive (ORA)

publisher_dissemination: Springer-Nature

ReferenceCitation: Brandi ML, Jan de Beur S, Briot K, Carpenter T, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Imel EA, Ing SW, Insogna K, Ito N, Javaid K, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Portale A, Ralston SH, Tanaka H, Weber TJ, Yoo HW, Sun W, Williams A, Nixon A, Takeuchi Y. Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study. Calcif Tissue Int. 2022 Oct;111(4):409-418. doi: 10.1007/s00223-022-01006-7. Epub 2022 Aug 4.

ReferenceCitation: Briot K, Portale AA, Brandi ML, Carpenter TO, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Ing S, Insogna K, Ito N, Jan de Beur S, Javaid MK, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Nixon A, Nixon M, Sun W, Williams A, Imel EA. Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension. RMD Open. 2021 Sep;7(3):e001714. doi: 10.1136/rmdopen-2021-001714.

license: cc-by-nc

license_normalized: cc-by-nc

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

version: acceptedVersion

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

location_facility: University of Edinburgh Edinburgh Clinical Trials Unit (ECTU) - Western General Hospital

location_facility: CHU de Bicêtre, Service d'Endocrinologie et des Maladies de la Reproduction

location_facility: Hôpital Lariboisière, Dept. of Rheumatology

location_facility: University of California-San Francisco Medical Center

location_facility: The Ohio State University Wexner Medical Center

location_facility: Indiana University Department of Medicine

location_facility: Azienda Ospedaliero Universitaria Careggi - Neurofarba

location_facility: Northern General Hospital, Metabolic Bone Centre (Sorby Wing)

title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study With Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

location_country:
Ireland
Italy
United Kingdom
Japan
United States
Korea, Republic of
France


title: An Open-label, Single Arm, Phase III Study to Assess the Self-administration of AOP2014 Using a Pre-filled Pen, Developed for the Treatment of Polycythemia Vera Patients

acronym: PEN-PV

summary:
Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells. The "thickening" of the blood in relation with a modified function of the cells has several consequences like increased blood pressure, pruritus of the skin, fatigue, disturbed blood circulation in the brain as well as fingers and toes and an increased risk of arterial and venous thrombosis (thrombosis is the formation of a blood clot in a vessel); like stroke, cardiac infarction, deep vein thrombosis in the legs. In case of a strong increase of platelets there is an additional risk of bleedings. As the disease progresses the size of spleen and liver increased in most cases and the bone marrow shows signs of fibrosis. In some cases of PV a progression at a later time point to a leukemia (increased formation of white blood cells) can occur.

The aim of this study is to assess the ease of AOP2014 self-administration using dedicated questionnaires.

To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study.
To assess maintenance of the blood efficacy parameters Hct (Hematocrit), WBC (white blood cells) and PLTs (platelets) and spleen size (comparing values at Visit P7 vs. values at Visit P1).
To assess the feasibility of AOP2014 self-administration: defined as the ability of the patients to use the pen as a self-administration tool (ease of handling, safety, tolerability and efficacy).

enrollment_type: Anticipated

lead_sponsor: AOP Orphan Pharmaceuticals AG

collaborators: PharmaEssentia Corporation (Co-Sponsor for USA)

location_country:
Poland
Slovakia
Hungary
Austria
Bulgaria
Ukraine
Czech Republic
France

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Saint Cyril and Metod University Hospital Bratislava

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: St Istvan and St Laszlo Hospital of Budapest

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

location_facility: University of Szeged, Albert Szent-Gyorgyi Clinical Center, Koranyi fasor 6

location_facility: Multiprofile Hospital for Active Treatment - Hristo Botev, Vratsa, First Department of Internal Medicine

location_facility: Andrzej Mielecki Independent Public Clinical Hospital of Medical University of Silesia in Katowice

location_facility: National Research Center for Radiation Medicine, Institute of Clinical Radiology

location_facility: Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center

location_facility: University Hospital with Outpatient Clinic F.D. Roosevelt

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: Institute of Hematology and Blood Transfusion

location_facility: Bekes County Pandy Kalman Hospital, 1st Department of Medicine, Hematology

location_facility: Fryderyk Chopin Provincial Specialized Hospital

location_facility: Institute of Hematology and Transfusion Medicine


title: A Multi-center, Double-blind, Randomized, Three-Arm, Parallel-Group, Placebo Controlled Study to Assess the Efficacy and Safety of NTRA-2112 on Intestinal Malabsorption in Preterm Infants

summary: The study will evaluate the effect of NTRA-2112 on intestinal malabsorption in preterm infants.

status: Unknown status

study_completion_date_type: Anticipated

ReferenceCitation: Mank E, Saenz de Pipaon M, Lapillonne A, Carnielli VP, Senterre T, Shamir R, van Toledo L, van Goudoever JB; FIT-04 Study Group. Efficacy and Safety of Enteral Recombinant Human Insulin in Preterm Infants: A Randomized Clinical Trial. JAMA Pediatr. 2022 May 1;176(5):452-460. doi: 10.1001/jamapediatrics.2022.0020.

oa_host_type: repository

repository_institution: University of Debrecen - University of Debrecen Electronic Archive

repositories_concat: PubMed Central;dea.lib.unideb.hu

unpaywall_oa_status: green

publisher_dissemination: American Medical Association

ipd_sharing: No

lead_sponsor: Nutrinia

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

location_facility: UMC Groningen (UMCG), Beatrix Kinderziekenhuis

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: Ospedale F. Del Ponte - Azienda Ospedaliera di Varese

location_facility: CHUS - Complejo Hospitalario Universitario de Santiago

location_facility: Ospedale dei Bambini "V.Buzzi"- Azienda ICP

location_facility: University Hospital of Leicester NHS Trust- Royal Infirmary

location_facility: Kinderklinik, Evangelisches Waldkrankenhaus Spandau

location_facility: Klinik und Poliklinik fur Kinder-und Jugendmedizin, Universitaetsklinikum Carl Gustav Carus Dresden

location_facility: Fejéktató Kórhz Újszülött, Csecsemő és Gyermekosztály - Megyei Szent György Egyetemi

location_facility: Klinik fur Neugeborene, Kinder und Jugendliche, Universitatsklinik der Paracelsus Medizinischen Privatuniversitat

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: University of Oklahoma Health Sciences Center

location_facility: Presidio Ospedaliero G. Salesi - AOUI Ospedali Riuniti di Ancona

location_facility: Klinik fuer Kinder und Jugendliche, Helios Klinikum Pforzheim

location_facility: Szegedi Tudomanyegyetem Szent Gyorgyi Albert Klinikai Kozpont Gyermekgyogyaszati klinika Koraszulott Intenzív Osztaly


title: A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

acronym: ESSENCE

summary: The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

status: Active, not recruiting

ReferenceCitation: Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.

lead_sponsor: Sarepta Therapeutics, Inc.

location_country:
Germany
Ireland
Poland
Argentina
Australia
Czechia
Canada
Serbia
France
Hungary
Denmark
United States
India
Belgium
Italy
United Kingdom
Sweden
Mexico
Korea, Republic of
Spain
Greece
Bulgaria
Russian Federation
Israel

location_facility: Reference Centre for Neuromuscular Diseases

location_facility: Instituto de Investigaciones Clínicas para la Salud A.C

location_facility: Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: Children's and Women's Health Centre of British Columbia

location_facility: University of Rochester Clinical Research Center

location_facility: Rigshospitalet Copenhagen University Hospital

location_facility: Stanford University School of Medicine/Medical Center

location_facility: Pusan National University Yangsan Hospital

location_facility: State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg

location_facility: Az Ospedaliera Universitaria Policlinico G Martino

location_facility: Samodzielny Publiczny Centralny Szpital Kliniczny

location_facility: Schneider Children's Medical Center of Israel

location_facility: Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation

location_facility: Center for Integrative Rare Disease Research (CIRDR)

location_facility: Clinic for Neurology and Psychiatry for Children and Youth

location_facility: Rady Children's Hospital San Diego/ UCSD

location_facility: Children's Hospital of the King's Daughters

location_facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

location_facility: Children's Hospital of Pittsburgh of UPMC

location_facility: Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

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

location_facility: Drottning Silvias Barn Och Ungdomssjukhus

location_facility: Ippokratio General Hospital of Thessaloniki

location_facility: Cincinnati Children's Hospital Medical Center (CCHMC)

status_simplified: Ongoing


type: Other Identifier

type: Registry Identifier

title: A Multi-centre, Randomised, Placebo Controlled, Double Blinded, Multiple Dose Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of Concizumab Administered Subcutaneously to Haemophilia A Subjects (Explorer™3)

summary: This trial is conducted globally. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of concizumab administered subcutaneously to haemophilia A subjects.

lead_sponsor: Novo Nordisk A/S

location_country:
Germany
Poland
Israel
United Kingdom
Australia
Netherlands
Austria
United States
Spain
Thailand
Malaysia
Croatia
Turkey
Ukraine
France


lead_sponsor: UCB BIOSCIENCES, Inc.

summary: The purpose of this trial is to assess the efficacy, safety and tolerability of lacosamide administered as add-on therapy with 1 to 3 anti-seizure medications. This trial is for children aged 1 month to less than 4 years with epilepsy who currently have uncontrolled partial-onset seizures.

title: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Lacosamide as Adjunctive Therapy in Subjects With Epilepsy >=1 Month to <4 Years of Age With Partial-Onset Seizures

location_country:
Poland
Argentina
Brazil
Thailand
Croatia
Czechia
Serbia
France
Portugal
Slovakia
Hungary
United States
Ukraine
Romania
China
Moldova, Republic of
Philippines
Italy
Mexico
Korea, Republic of
Greece
Taiwan
Bulgaria
Georgia
Russian Federation
Israel


intervention_type: Biological

lead_sponsor: Novartis Pharmaceuticals

summary: This was a multicenter, randomized, double-blind, placebo- and active-controlled (etanercept in single blinded arm) study in pediatric subjects aged 6 years to less than 18 years with severe chronic plaque psoriasis. Approximately 160 subjects aged 6 years to <18 years were enrolled, of which at least 30 were 6 years to <12 years old. Subjects were enrolled at approximately 70 study sites worldwide.

title: A Randomized, Double-blind, Placebo- and Active Controlled Multicenter Trial to Demonstrate Efficacy of Subcutaneous Secukinumab Compared to Placebo and Etanercept (in a Single-blinded Arm) After Twelve Weeks of Treatment, and to Assess the Safety, Tolerability, and Long-term Efficacy in Subjects From 6 to Less Than 18 Years of Age With Severe Chronic Plaque Psoriasis

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
Poland
Japan
France
Colombia
Hungary
Switzerland
United States
Latvia
Romania
Belgium
Estonia
Egypt
Italy
Guatemala
United Kingdom
Spain
Russian Federation
Israel


title: A Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 in Cystic Fibrosis

summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).

ReferenceCitation: Chmiel JF, Flume P, Downey DG, Dozor AJ, Colombo C, Mazurek H, Sapiejka E, Rachel M, Constantine S, Conley B, Dgetluck N, Dinh Q, White B, Elborn JS; Lenabasum JBT101-CF-001 Study Group. Safety and efficacy of lenabasum in a phase 2 randomized, placebo-controlled trial in adults with cystic fibrosis. J Cyst Fibros. 2021 Jan;20(1):78-85. doi: 10.1016/j.jcf.2020.09.008. Epub 2020 Oct 1.

oa_host_type: publisher

license: publisher-specific-oa

license_normalized: publisher-specific

lead_sponsor: Corbus Pharmaceuticals Inc.

location_country:
Germany
Italy
Poland
United Kingdom
Belgium
United States
France

location_facility: University Hospitals Cleveland Medical Center

location_facility: Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

location_facility: Rutgers Robert-Wood Johnson Medical School

location_facility: Long Beach Memorial Medical Center/Miller Children's and Women's Hospital

location_facility: Texas Children's Hospital Clinical Care Center

location_facility: Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: The University of Texas Southwestern Medical Center

location_facility: Christiane Herzog CF-Zentrum Frankfurt am Main

location_facility: Szpital Dziecięcy Polanki im. Macieja Płażyńskiego w Gdańsku Spółka z o.o. Poradnia Leczenia Mukowiscydozy

location_facility: Podkarpacki Ośrodek Pulmunologii i Alergologii

location_facility: AOUI di Verona - Ospedale Borgo Trento UOC Fibrosi Cistica

location_facility: University of Arkansas for Medical Sciences

location_facility: Instytut Gruźlicy i Chorób Płuc I Klinika Chorób Płuc


title: A Multicenter Phase II Clinical Trial of Lurbinectedin (PM01183) in Selected Advanced Solid Tumors

summary: Multicenter, open-label, exploratory, phase II clinical trial to evaluate the efficacy and safety of PM01183 in previously treated patients with advanced solid tumors

ReferenceCitation: Boni V, Pistilli B, Brana I, Shapiro GI, Trigo J, Moreno V, Castellano D, Fernandez C, Kahatt C, Alfaro V, Siguero M, Zeaiter A, Longo F, Zaman K, Anton A, Paredes A, Huidobro G, Subbiah V. Lurbinectedin, a selective inhibitor of oncogenic transcription, in patients with pretreated germline BRCA1/2 metastatic breast cancer: results from a phase II basket study. ESMO Open. 2022 Oct;7(5):100571. doi: 10.1016/j.esmoop.2022.100571. Epub 2022 Aug 28.

repositories_concat: PubMed Central;www.repositoriosalud.es

ReferenceCitation: Longo-Munoz F, Castellano D, Alexandre J, Chawla SP, Fernandez C, Kahatt C, Alfaro V, Siguero M, Zeaiter A, Moreno V, Sanz-Garcia E, Awada A, Santaballa A, Subbiah V. Lurbinectedin in patients with pretreated neuroendocrine tumours: Results from a phase II basket study. Eur J Cancer. 2022 Sep;172:340-348. doi: 10.1016/j.ejca.2022.06.024. Epub 2022 Jul 10.

ReferenceCitation: Subbiah V, Brana I, Longhi A, Boni V, Delord JP, Awada A, Boudou-Rouquette P, Sarantopoulos J, Shapiro GI, Elias A, Ratan R, Fernandez C, Kahatt C, Cullell-Young M, Siguero M, Zeaiter A, Chawla SP. Antitumor Activity of Lurbinectedin, a Selective Inhibitor of Oncogene Transcription, in Patients with Relapsed Ewing Sarcoma: Results of a Basket Phase II Study. Clin Cancer Res. 2022 Jul 1;28(13):2762-2770. doi: 10.1158/1078-0432.CCR-22-0696.

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

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

repositories_concat: PubMed Central;dipot.ulb.ac.be

publisher_dissemination: American Association for Cancer Research

ReferenceCitation: Fernandez-Teruel C, Fudio S, Lubomirov R. Integrated exposure-response analysis of efficacy and safety of lurbinectedin to support the dose regimen in small-cell lung cancer. Cancer Chemother Pharmacol. 2022 May;89(5):585-594. doi: 10.1007/s00280-021-04366-3. Epub 2021 Nov 5.

ReferenceCitation: Subbiah V, Paz-Ares L, Besse B, Moreno V, Peters S, Sala MA, Lopez-Vilarino JA, Fernandez C, Kahatt C, Alfaro V, Siguero M, Zeaiter A, Zaman K, Lopez R, Ponce S, Boni V, Arrondeau J, Delord JP, Martinez M, Wannesson L, Anton A, Valdivia J, Awada A, Kristeleit R, Olmedo ME, Rubio MJ, Sarantopoulos J, Chawla SP, Mosquera-Martinez J, D' Arcangelo M, Santoro A, Villalobos VM, Sands J, Trigo J. Antitumor activity of lurbinectedin in second-line small cell lung cancer patients who are candidates for re-challenge with the first-line treatment. Lung Cancer. 2020 Dec;150:90-96. doi: 10.1016/j.lungcan.2020.10.003. Epub 2020 Oct 10.

repository_institution: University of Zaragoza Repository - Zaguan

ReferenceCitation: Trigo J, Subbiah V, Besse B, Moreno V, Lopez R, Sala MA, Peters S, Ponce S, Fernandez C, Alfaro V, Gomez J, Kahatt C, Zeaiter A, Zaman K, Boni V, Arrondeau J, Martinez M, Delord JP, Awada A, Kristeleit R, Olmedo ME, Wannesson L, Valdivia J, Rubio MJ, Anton A, Sarantopoulos J, Chawla SP, Mosquera-Martinez J, D'Arcangelo M, Santoro A, Villalobos VM, Sands J, Paz-Ares L. Lurbinectedin as second-line treatment for patients with small-cell lung cancer: a single-arm, open-label, phase 2 basket trial. Lancet Oncol. 2020 May;21(5):645-654. doi: 10.1016/S1470-2045(20)30068-1. Epub 2020 Mar 27. Erratum In: Lancet Oncol. 2020 Dec;21(12):e553.

lead_sponsor: PharmaMar

location_country:
Germany
Italy
United Kingdom
Sweden
Switzerland
Belgium
United States
Spain
France

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Charité Universitätsmedizin Berlin - Campus Benjamin Franklin - Comprehensive Cancer Center

location_facility: Hospital Universitario Puerta de Hierro Majadahonda

location_facility: Hospital Universitari i Polotècnic la Fe

location_facility: Hospital Universitario Madrid Sanchinarro

location_facility: AUSL Romagna - Ospedale Santa Maria delle Croci

location_facility: Complejo Hospitalario Regional Reina Sofía

location_facility: ASST Monza - Ospedale San Gerardo di Monza Struttura Complessa di Oncologia Medica

location_facility: Complexo Hospitalario Universitario A Coruña

location_facility: Hopsital Universitario Donostia - Donostia Unibertsitate Ospitalea

location_facility: Institute for Drug Development, Cancer Therapy & Research Center at University of Texas Health Science Center

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Istituto Oncologico della Svizzera Italiana (IOSI)

location_facility: Complejo Hospitalario de Especialidades Virgen de la Victoria

location_facility: Complexo Hospitalario Universitario de Santiago

location_facility: The University of Texas MD Anderson Cancer Center

location_facility: Hospital Universitario Central de Asturias

location_facility: Hospital Universitario Fundación Jiménez Díaz


lead_sponsor: Eli Lilly and Company

summary: The main purpose of this study is to evaluate the effectiveness of the study drug known as abemaciclib versus docetaxel in participants with stage IV squamous non-small cell lung cancer (NSCLC) previously treated with platinum-based chemotherapy.

location_facility: Washington University School of Medicine

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

location_facility: St-Petersburg scientifical practical center of specialized kinds of medical care (oncological)

location_facility: Volgograd regional clinical oncology dispensary

title: A Randomized Phase 2 Study of Abemaciclib (LY2835219) Versus Docetaxel in Patients With Stage IV Squamous Non-Small Cell Lung Cancer Previously Treated With Platinum-Based Chemotherapy

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

location_country:
Germany
Italy
Poland
Hungary
Romania
Australia
United States
Korea, Republic of
Spain
Taiwan
Ukraine
Russian Federation
France


title: A Phase 3, Multicenter, Long Term, Extension Study of the Safety and Efficacy of AVP-786 (Deuterated [d6] Dextromethorphan Hydrobromide [d6-DM]/Quinidine Sulfate [Q]) for the Treatment of Agitation in Patients With Dementia of the Alzheimer's Type

summary: This is an extension study of the Phase 3 Studies 15-AVP-786-301, 15-AVP-786-302, and 17-AVP-786-305, which also allows participants from the Phase 2 Study 12-AVR-131 to be included.

status: Recruiting

design_allocation: Non-Randomized

ipd_sharing_description: Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.

lead_sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

location_country:
Italy
Poland
Hungary
United States
Spain
South Africa
Czechia
Bulgaria
Canada
France

location_facility: Eastside Comprehensive Medical Center, LLC

location_facility: Debreceni Egyetem Kenezy Gyula Egyetemi Korhaz

location_facility: Clinical Research Associates of South Florida #2

location_facility: Neurology Research Institute Palm Beach, LLC #2

location_facility: Cleveland Clinic Lou Ruvo Center for Brain Health at Lakewood Hospital

location_facility: Columbus Research & Wellness Institute, INC

location_facility: Wielospecjalistyczna Poradnia Lekarska Synapsis

location_facility: New Foundation Medical Group Clinical Trials

location_facility: Rhode Island Mood & Memory Research Institute

location_facility: Premier Clinical Research Institute, Inc. #1

location_facility: Mental Health Centre Prof. Nikola Shipkovenski

location_facility: Pszichiatriai es Pszichiatriai Rehabilitacios Osztaly

location_facility: Psychiatry and Alzheimer's Care of Rochester, PLLC

location_facility: Dr. Kenessey Albert Kórház-Rendelőintézet

location_facility: Nathan S. Kline Institute for Psychiatric Research #571

location_facility: Alzheimer's Research and Treatment Center #2

location_facility: Flora Baker Center for Alzheimer's Disease and Related Disorders

location_facility: Clinical Neuroscience Research Associates, Inc. dba The Memory Clinic

location_facility: Houston Methodist Neurological Institute

location_facility: Fondazione Santa Lucia - Istituto di Ricovero e Cura a Carattere Scientifico

location_facility: Alzheimer's Research and Treatment Center #1

location_facility: Research Center for Clinical Studies, Inc.

location_facility: The Birches at Newton / Family Medical Associates

location_facility: Clinical Research Associates of South Florida #1

location_facility: Complejo Asistencial de Zamora Hospital Virgen de la Concha

location_facility: University Texas Southwestern Medical Center

location_facility: Torrance Clinical Research Institute, Inc.

location_facility: Michigan State University Department of Neurology

location_facility: Negron Research Services / Humanity Clinical Research Site# 766

location_facility: Centre Hospitalier Universitaire Dijon Bourgogn

location_facility: University Multiprofile Hospital for Active Treatment Alexandrovska

location_facility: Alzheimer's Research and Treatment Center #3

location_facility: Josephson Wallack Munshower Neurology, PC

location_facility: Nathan S. Kline Institute for Psychiatric Research #1

location_facility: Centre Hospitalier Universitaire de Saint-Étienne - Hôpital Nord

location_facility: Alexian Brothers Neurosciences Institute

location_facility: Veteran Affairs Medical Center, Salem Virginia

location_facility: Alliance for Wellness, Inc dba Alliance for Research

location_facility: Pharmaceuticals Research Associates, Inc.

location_facility: Neurology and Neuroscience Associates, Inc.

location_facility: Centre Hospitalier Universitaire Toulouse - Casselardit Ancely

location_facility: The Samuel & Alexia Bratton Memory Clinic

location_facility: Homestead Associates in Research Site# 797

location_facility: Medical Research & Health Education Foundation, Inc.

location_facility: Miami Dade Medical Research Institute, LLC

location_facility: Collaborative Neuroscience Network, LLC.

location_facility: Texas Medical Research Associates, L.L.C.

location_facility: ANI Neurology PLLC dba Alzheimer's Memory Center

location_facility: MidAmerica Neuroscience Research Foundation

location_facility: Premier Clinical Research Institute, Inc. #2

location_facility: Neurology Research Institute Palm Beach, LLC #1

location_facility: Lytle and Weiss, PLLC dba Clinical Trials of the Rockies


title: Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy

acronym: PAVO

summary: Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.

primary_purpose: Other

sponsor_collaborators:
Novartis Pharmaceuticals
University of Pennsylvania

location_country:
Finland
Germany
Italy
Israel
Norway
Denmark
Australia
Japan
Netherlands
Austria
Belgium
Singapore
United States
Spain
United Kingdom
Taiwan
Canada
France

location_facility: University of Chicago Medical Center, Hematology & Oncology

location_facility: Childrens Hospital Los Angeles SC CTL019

location_facility: Mayo Clinic Arizona Mayo Clinic Building

location_facility: Duke Unversity Medical Center SC - CTL019B2205J

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: The Childrens Hospital of Philadelphia CHOP

location_facility: Children's Healthcare of Atlanta SC CTL019

location_facility: University of Michigan Health System SC CTL019

location_facility: Stanford Universtiy Medical Center SC - CTL019B2205J - B2206

location_facility: Ohio State Comprehensive Cancer Center/James Cancer Hospital Ohio State Univ.

location_facility: University of Wisconsin Hospital and Clinics

location_facility: University of Utah Clinical Trials Office

location_facility: Emory University School of Medicine/Winship Cancer Institute

intervention_type: Genetic


lead_sponsor: Adienne SA

summary: The objectives of this study are to determine the efficacy and safety of BEGEDINA® in subjects with steroid resistant acute graft versus host disease (GvHD). GvHD is a rare and complex immunological disease occurring in some recipients of allogeneic hematopoietic stem cell transplants (HSCTs) and affecting principally the skin, liver and gastrointestinal (GI) tissues. The disease is life threatening and may be acute or chronic and the first choice treatment for patients with acute GvHD (Grade II or higher) is the immunosuppressive corticosteroid hormone methylprednisolone. However, some GvHD patients may be resistant to this treatment leading to disease progression and a high rate of morbidity and mortality, primarily from infections and/or multi-organ failure. There are currently no other satisfactory therapies. BEGEDINA® is a therapeutic monoclonal antibody that recognises and binds to CD26 on CD4+ T lymphocytes. BEGEDINA® reduces the activity of CD26 in these cells and inhibits the immune response leading to improvement in patients that have shown steroid resistance. This study is therefore aimed at demonstrating that BEGEDINA® is a safe and effective treatment for steroid-resistant GvHD patients where no other such treatments are currently available.

sponsor_collaborators:
Adienne SA
ADIENNE S.r.l. S.U.

location_facility: Centre Hospitalier Universitaire de Grenoble

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: Huntsman Cancer Center, University of Utah

location_facility: Ospedale Casa Sollievo della Sofferenza, IRCCS

location_facility: University of Kansas Cancer Center and Medical Pavilion

location_facility: A.O. Universitaria Policlinico Tor Vergata

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: Hospital Saint-Louis APHP (Hôpitaux Universitaires Sant-Louis)

location_facility: Washington University School of Medicine

location_facility: Hackensack University Medical Center - John Theurer Cancer Center

location_facility: Policlinico S.Orsola Malpighi, AOU di Bologna

location_facility: City of Hope Comprehensive Cancer Center

title: Prospective, Phase II/III, Randomized Clinical Study to Compare BEGEDINA® Versus "Conventional Treatment" for Treating Steroid Resistant Acute Graft-versus Host Disease

location_country:
Germany
Italy
United Kingdom
Switzerland
United States
Spain
France


summary: This randomized, open-label study was designed to evaluate and compare the safety and efficacy of atezolizumab with gemcitabine + cisplatin or carboplatin in PD-L1 selected participants with chemotherapy-naive, Stage IV squamous NSCLC. The study was closed due to low patient enrollment and the Sponsor's decision to include patients with squamous NSCLC into the GO29431 study, NCT02409342. Therefore the planned objectives of this study are no longer applicable and formal analyses of efficacy or safety have not been performed.

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

location_facility: Clin Hospital Center - Kragujevac; Pulmonary Diseases

location_facility: Arkhangelsk Regional Clinical Oncology Dispensary

location_facility: University of Pecs, I st Dept of Internal Medicine

location_facility: Chao Family Comprehensive Cancer Center; UC Irvine Medical Center

location_facility: Szabolcs-Szatmar-Bereg Megyei; Korhazak es Egyetemi Oktatokorhaz

location_facility: IRST Istituto Scientifico Romagnolo Per Lo Studio E Cura Dei Tumori, Sede Meldola; Oncologia Medica

location_facility: Penn State Milton S. Hershey Medical Center

location_facility: Jasz-Nagykun-Szolnok Megyei Hetenyi Geza Korhaz-Rend.Int.

location_facility: Eastern Connecticut Hematology and Oncology Associates; (ECHO)

location_facility: California Cancer Associates for Research & Excellence, Inc.

location_facility: Azienda Ospedaliera Istituti Ospitalieri

location_facility: Hospital Universitari Germans Trias i Pujol; Servicio de Oncologia

location_facility: A.O.U.I. VERONA-OSPEDALE BORGO TRENTO; ONCOLODIA MEDICA-d.O.

location_facility: Sibiu Emergency Clinical County Hospital

location_facility: Kursk Regional Clinical Oncology Dispensary

location_facility: Clinical Hospital Center Bezanijska kosa; Clinic for Oncology

location_facility: MultiCare Regional Cancer Center - Auburn

location_facility: Institutul Regional de Oncologie Iasi; Clinica de Hematologie

location_facility: Teo Health SA - Saint Constantin Hospital

location_facility: Seoul National University Bundang Hospital

location_facility: Chonnam National University Hwasun Hospital

location_facility: Georgios Papanikolaou General Hosp. of Thessaloniki

location_facility: Irccs Centro Di Riferimento Oncologico (CRO)

location_facility: Filial #1 Regional Oncology Dispensary of Nizhniy Novgorod

location_facility: Fundacion Investigacion Hospital La Fe de Valencia

location_facility: The Catholic University of Korea St. Vincent's Hospital

location_facility: Irccs Istituto Nazionale Dei Tumori (Int);S.C. Medicina Oncologica 2

location_facility: Carolina Oncology Specialists, PA - Hickory

location_facility: Dr Constantin Opris Emergency County Hospital Baia Mare

title: A Phase III, Open-Label, Randomized Study of Atezolizumab (Anti-PD-L1 Antibody) Compared With Gemcitabine+Cisplatin or Carboplatin for PD-L1-Selected, Chemotherapy Naive Patients With Stage IV Squamous Non-Small Cell Lung Cancer

location_country:
Germany
Italy
Poland
Hungary
United Kingdom
Romania
United States
Korea, Republic of
Spain
Greece
Czechia
Serbia
Russian Federation
France


acronym: VALUE

summary: Assessment of long-term safety and efficacy of oral lacosamide (LCM) as an adjunctive therapy for uncontrolled primary generalized tonic-clonic seizures (PGTCS) in subjects >= 4 years of age with idiopathic generalized epilepsy (IGE). This study will enroll subjects from the LCM SP0982 [NCT02408523] study.

title: An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Lacosamide as Adjunctive Therapy for Uncontrolled Primary Generalized Tonic-Clonic Seizures in Subjects With Idiopathic Generalized Epilepsy

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

location_country:
Germany
Poland
Australia
Brazil
Japan
Czechia
France
Portugal
Slovakia
Hungary
United States
Romania
China
Italy
Mexico
Korea, Republic of
Spain
Taiwan
Bulgaria
Russian Federation
Israel


summary:
The purpose of this "first-in-human" study of PDR001 was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of PDR001 administered i.v. as a single agent to adult patients with solid tumors.

By blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2, PDR001 inhibits the PD-1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells.

ReferenceCitation: Capdevila J, Wirth LJ, Ernst T, Ponce Aix S, Lin CC, Ramlau R, Butler MO, Delord JP, Gelderblom H, Ascierto PA, Fasolo A, Fuhrer D, Hutter-Kronke ML, Forde PM, Wrona A, Santoro A, Sadow PM, Szpakowski S, Wu H, Bostel G, Faris J, Cameron S, Varga A, Taylor M. PD-1 Blockade in Anaplastic Thyroid Carcinoma. J Clin Oncol. 2020 Aug 10;38(23):2620-2627. doi: 10.1200/JCO.19.02727. Epub 2020 May 4.

evidence: open (via page says license)

repository_institution: Universitat Autònoma de Barcelona - Dipòsit Digital de Documents de la UAB

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

publisher_dissemination: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Naing A, Gainor JF, Gelderblom H, Forde PM, Butler MO, Lin CC, Sharma S, Ochoa de Olza M, Varga A, Taylor M, Schellens JHM, Wu H, Sun H, Silva AP, Faris J, Mataraza J, Cameron S, Bauer TM. A first-in-human phase 1 dose escalation study of spartalizumab (PDR001), an anti-PD-1 antibody, in patients with advanced solid tumors. J Immunother Cancer. 2020 Mar;8(1):e000530. doi: 10.1136/jitc-2020-000530.

location_facility: Oregon Health and Science University SC-10

location_facility: The Sidney Kimmel Cancer Center at Johns Hopkins Hospital Johns Hopkins

location_facility: Huntsman Cancer Institute Univ. of Utah HCI

location_facility: University of Texas MD Anderson Cancer Center MD Anderson PSC

title: Open Label Multicenter Phase I/II Study of the Safety and Efficacy of PDR001 Administered to Patients With Advanced Malignancies

location_country:
Germany
Italy
Poland
Hungary
Norway
Netherlands
Lebanon
United States
Spain
Thailand
Turkey
Taiwan
Canada
France


title: A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

summary: The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

ReferenceCitation: Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3.

ReferenceCitation: Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3.

lead_sponsor: Scioderm, Inc.

sponsor_collaborators:
Scioderm, Inc.
Amicus Therapeutics

location_country:
Germany
Italy
Lithuania
Poland
Israel
United Kingdom
Australia
Netherlands
Austria
United States
Spain
Serbia
France


title: A Twelve-Week, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Multicenter Study of the Safety and Efficacy of JZP-110 [(R)-2-amino-3-phenylpropylcarbamate Hydrochloride] in the Treatment of Excessive Sleepiness in Subjects With Obstructive Sleep Apnea (OSA)

acronym: OSA

summary: This trial is a 12 week, randomized, double-blind, placebo controlled, multicenter, 5-arm parallel group study of safety and efficacy of JZP-110 in the treatment of excessive sleepiness in adult subjects with OSA.

ReferenceCitation: Rosenberg R, Thorpy MJ, Dauvilliers Y, Schweitzer PK, Zammit G, Gotfried M, Bujanover S, Scheckner B, Malhotra A. Incidence and duration of common early-onset adverse events in randomized controlled trials of solriamfetol for treatment of excessive daytime sleepiness in obstructive sleep apnea and narcolepsy. J Clin Sleep Med. 2022 Jan 1;18(1):235-244. doi: 10.5664/jcsm.9550.

evidence: open (via free pdf)

repository_institution: California Digital Library - eScholarship

repositories_concat: PubMed Central;escholarship.org

publisher_dissemination: American Academy of Sleep Medicine (AASM)

ReferenceCitation: Schweitzer PK, Mayer G, Rosenberg R, Malhotra A, Zammit GK, Gotfried M, Chandler P, Baladi M, Strohl KP. Randomized Controlled Trial of Solriamfetol for Excessive Daytime Sleepiness in OSA: An Analysis of Subgroups Adherent or Nonadherent to OSA Treatment. Chest. 2021 Jul;160(1):307-318. doi: 10.1016/j.chest.2021.02.033. Epub 2021 Feb 22.

ReferenceCitation: Weaver TE, Menno DM, Bron M, Crosby RD, Morris S, Mathias SD. Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea. Sleep Breath. 2021 Sep;25(3):1707-1715. doi: 10.1007/s11325-020-02270-3. Epub 2021 Jan 4.

ReferenceCitation: Rosenberg R, Baladi M, Bron M. Clinically relevant effects of solriamfetol on excessive daytime sleepiness: a posthoc analysis of the magnitude of change in clinical trials in adults with narcolepsy or obstructive sleep apnea. J Clin Sleep Med. 2021 Apr 1;17(4):711-717. doi: 10.5664/jcsm.9006. Erratum In: J Clin Sleep Med. 2021 Nov 1;17(11):2343.

ReferenceCitation: Weaver TE, Drake CL, Benes H, Stern T, Maynard J, Thein SG, Andry JM Sr, Hudson JD, Chen D, Carter LP, Bron M, Lee L, Black J, Bogan RK. Effects of Solriamfetol on Quality-of-Life Measures from a 12-Week Phase 3 Randomized Controlled Trial. Ann Am Thorac Soc. 2020 Aug;17(8):998-1007. doi: 10.1513/AnnalsATS.202002-136OC.

publisher_dissemination: American Thoracic Society

ReferenceCitation: Schweitzer PK, Rosenberg R, Zammit GK, Gotfried M, Chen D, Carter LP, Wang H, Lu Y, Black J, Malhotra A, Strohl KP; TONES 3 Study Investigators. Solriamfetol for Excessive Sleepiness in Obstructive Sleep Apnea (TONES 3). A Randomized Controlled Trial. Am J Respir Crit Care Med. 2019 Jun 1;199(11):1421-1431. doi: 10.1164/rccm.201806-1100OC.

location_country:
Germany
Netherlands
United States
Canada
France

location_facility: University of Illinois at Chicago College of Nursing

location_facility: Northwestern University Feinberg School of Medicine

location_facility: Henry Ford Hospital Sleep Disorders & Research Center

location_facility: Mercy St. Anne & Mercy St. Charles Sleep Disorders Center

location_facility: Sleep Medicine & Research Center, St. Luke's Hospital

location_facility: So Cal Institute For Respiratory Diseases, Inc.

location_facility: Universitätsklinikum Münster Department für Neurologie

location_facility: Somni bene GmbH Institut für Medizinische Forschung und Schlafmedizin Schwerin GmbH

location_facility: medbo Bezirksklinikum Regensburg Schlafmedizinisches Zentrum

location_facility: Stanford University Center for Narcolepsy

location_facility: Ohio Sleep Medicine & Neuroscience Institute

location_facility: Southwest Cleveland Sleep Research Center

location_facility: Hickory Research Center, ARSM Research, LLC

location_facility: Clinical Research Group of St. Petersburg


lead_sponsor: Jazz Pharmaceuticals

summary: This trial is a 12-week, randomized, double-blind, placebo controlled, multicenter, 4-treatment parallel group study of the safety and efficacy of JZP-110 in the treatment of excessive sleepiness in adult subjects with narcolepsy.

repositories_institution: California Digital Library - eScholarship

ReferenceCitation: Dauvilliers Y, Shapiro C, Mayer G, Lammers GJ, Emsellem H, Plazzi G, Chen D, Carter LP, Lee L, Black J, Thorpy MJ. Solriamfetol for the Treatment of Excessive Daytime Sleepiness in Participants with Narcolepsy with and without Cataplexy: Subgroup Analysis of Efficacy and Safety Data by Cataplexy Status in a Randomized Controlled Trial. CNS Drugs. 2020 Jul;34(7):773-784. doi: 10.1007/s40263-020-00744-2.

repository_institution: Leiden University - Leiden Repository

ReferenceCitation: Emsellem HA, Thorpy MJ, Lammers GJ, Shapiro CM, Mayer G, Plazzi G, Chen D, Carter LP, Villa KF, Lee L, Menno D, Black J, Dauvilliers Y. Measures of functional outcomes, work productivity, and quality of life from a randomized, phase 3 study of solriamfetol in participants with narcolepsy. Sleep Med. 2020 Mar;67:128-136. doi: 10.1016/j.sleep.2019.11.1250. Epub 2019 Dec 4.

location_facility: Northwestern University, Feinberg School of Medicine

location_facility: University of Illinois Chicago, College of Nursing

location_facility: Critical Care Pulmonary & Sleep Associates, LLC

title: A Twelve-week, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multicenter Study of the Safety and Efficacy of JZP-110 [(R)-2-amino-3-phenylpropylcarbamate Hydrochloride] in the Treatment of Excessive Sleepiness in Subjects With Narcolepsy

location_country:
Germany
Finland
Netherlands
United States
Canada
France


lead_sponsor: Kite, A Gilead Company

summary:
This study will be separated into 3 distinct phases designated as the Phase 1 study, Phase 2 pivotal study (Cohort 1 and Cohort 2), and Phase 2 safety management study (Cohort 3 and Cohort 4, Cohort 5 and Cohort 6).

The primary objectives of this study are:

Phase 1 Study: Evaluate the safety of axicabtagene ciloleucel regimens
Phase 2 Pivotal Study; Evaluate the efficacy of axicabtagene ciloleucel
Phase 2 Safety Management Study: Assess the impact of prophylactic regimens or earlier interventions on the rate and severity of cytokine release syndrome (CRS) and neurologic toxicities

Subjects who received an infusion of KTE-C19 will complete the remainder of the 15 year follow-up assessments in a separate long-term follow-up study, KT-US-982-5968.

ReferenceCitation: Topp MS, van Meerten T, Wermke M et al. Preliminary Results of Earlier Steroid Use with Axicabtagene Ciloleucel in Patients With Relapsed/Refractory Large B Cell Lymphoma. Poster presented at the American Society of Presented at: American Society of Clinical Oncology Annual Meeting. May 31-June 4, 2019; Chicago, Illinois; Abstract 7558.

ReferenceCitation: Topp, M, van Meerten T, Houot R, et al. (2019). Earlier Steroid Use with Axicabtagene Ciloleucel (Axi-Cel) in Patients with Relapsed/Refractory Large B Cell Lymphoma. Blood (ASH Annual Meeting Abstracts) 134(Suppl 1): 243. Abstract 626.

ReferenceCitation: Abstract: Oluwole OO, et al. Prophylactic corticosteroid use with axicabtagene ciloleucel in patients with relapsed/refractory large B-cell lymphoma. Transplantation and Cellular Therapy 2021.

ReferenceCitation: Oluwole OO, Bouabdallah K, Munoz J, De Guibert S, Vose JM, Bartlett NL, Lin Y, Deol A, McSweeney PA, Goy AH, Kersten MJ, Jacobson CA, Farooq U, Minnema MC, Thieblemont C, Timmerman JM, Stiff P, Avivi I, Tzachanis D, Kim JJ, Bashir Z, McLeroy J, Zheng Y, Rossi JM, Johnson L, Goyal L, van Meerten T. Prophylactic corticosteroid use in patients receiving axicabtagene ciloleucel for large B-cell lymphoma. Br J Haematol. 2021 Aug;194(4):690-700. doi: 10.1111/bjh.17527. Epub 2021 Jul 22.

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

repository_institution: University of California - eScholarship University of California

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

ReferenceCitation: Santa Monica, Calif. New Four-Year Data Show Long-Term Survival in Patients With Large B-Cell Lymphoma Treated With Yescarta® in ZUMA-1 Trial. Data presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. December 5, 2020; Abstract 1187

ReferenceCitation: Max S. Topp, Tom van Meerten, Martin Wermke, Pieternella J. Lugtenburg, Monique C. Minnema, Kevin W. Song, Catherine Thieblemont, Yizhou Jiang, Vicki Plaks, Anne Kerber, Marie José Kersten. Preliminary Results of Earlier Steroid Use With Axicabtagene Ciloleucel in Patients With Relapsed/ Refractory Large B Cell Lymphoma. Poster presented at ASCO 2019 Annual Meeting. June 3, 2019; Abstract 7558

ReferenceCitation: Sattva S. Neelapu, Caron A. Jacobson, Olalekan O. Oluwole, Javier Munoz, Abhinav Deol, David B. Miklos, Nancy L. Bartlett, Ira Braunschweig, Yizhou Jiang, Jenny J. Kim, Lianqing Zheng, John M. Rossi, Frederick L. Locke. Axicabtagene Ciloleucel (Axi-Cel) In Refractory Large B Cell Lymphoma: Outcomes in Patients ≥ or < 65 Years of Age in the Pivotal Phase 1/2 ZUMA-1 Study. Poster presented at EHA 2019. June 15, 2019; Abstract 1066

ReferenceCitation: Max S. Topp, Tom van Meerten, Martin Wermke, Pieternella J. Lugtenburg, Monique C. Minnema, Kevin W. Song, Catherine Thieblemont, Yizhou Jiang, Vicki Plaks, Anne Kerber, Marie José Kersten. Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory Large B Cell Lymphoma: Preliminary Results of Earlier Steroid Use. Poster presented at EHA 2019. June 15, 2019; Abstract 1067

ReferenceCitation: Sattva S. Neelapu, John M. Rossi, Caron A. Jacobson, Frederick L. Locke, David B. Miklos, Patrick M. Reagan, Scott Rodig, Lazaros J. Lekakis, Ian W. Flinn, Lianqing Zheng, Francesca Milletti, Edmund Chang, Allen Xue, Vicki Plaks, Jenny J. Kim, Adrian Bot. CD19-Loss With Preservation of Other B Cell Lineage Features in Patients With Large B Cell Lymphoma Who Relapsed Post-Axi-Cel. Blood (ASH Annual Meeting Abstracts) 134(Suppl 1): 203. Abstract 704.

ReferenceCitation: Sattva S. Neelapu, Frederick L. Locke, Nancy L. Bartlett, Lazaros J. Lekakis, Patrick Reagan, David B. Miklos, Caron A. Jacobson, Ira Braunschweig, Olalekan Oluwole, Tanya Siddiqi, Yi Lin, Michael Crump, John Kuruvilla, Eric Van Den Neste, Umar Farooq, Lynn Navale, Venita DePuy, Jenny J. Kim, Christian Gisselbrecht. A Comparison of Two-Year Outcomes in ZUMA-1 (Axicabtagene Ciloleucel) and SCHOLAR-1 in Patients With Refractory Large B Cell Lymphoma. Blood (ASH Annual Meeting Abstracts) 134(Suppl 1): 4095. Abstract 626.

ReferenceCitation: Ernst M, Oeser A, Besiroglu B, Caro-Valenzuela J, Abd El Aziz M, Monsef I, Borchmann P, Estcourt LJ, Skoetz N, Goldkuhle M. Chimeric antigen receptor (CAR) T-cell therapy for people with relapsed or refractory diffuse large B-cell lymphoma. Cochrane Database Syst Rev. 2021 Sep 13;9(9):CD013365. doi: 10.1002/14651858.CD013365.pub2.

repositories_concat: PubMed Central;ora.ox.ac.uk

repositories_institution: University of Oxford - Oxford University Research Archive (ORA)

ReferenceCitation: Maloney DG, Kuruvilla J, Liu FF, Kostic A, Kim Y, Bonner A, Zhang Y, Fox CP, Cartron G. Matching-adjusted indirect treatment comparison of liso-cel versus axi-cel in relapsed or refractory large B cell lymphoma. J Hematol Oncol. 2021 Sep 8;14(1):140. doi: 10.1186/s13045-021-01144-9.

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

repository_normalized: HAL

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

repository_institution: University of Nottingham - Repository@Nottingham

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

repositories:
HAL
PubMed Central
nottingham-repository.worktribe.com

ReferenceCitation: Westin JR, Kersten MJ, Salles G, Abramson JS, Schuster SJ, Locke FL, Andreadis C. Efficacy and safety of CD19-directed CAR-T cell therapies in patients with relapsed/refractory aggressive B-cell lymphomas: Observations from the JULIET, ZUMA-1, and TRANSCEND trials. Am J Hematol. 2021 Oct 1;96(10):1295-1312. doi: 10.1002/ajh.26301. Epub 2021 Aug 13.

ReferenceCitation: Upadhyay R, Boiarsky JA, Pantsulaia G, Svensson-Arvelund J, Lin MJ, Wroblewska A, Bhalla S, Scholler N, Bot A, Rossi JM, Sadek N, Parekh S, Lagana A, Baccarini A, Merad M, Brown BD, Brody JD. A Critical Role for Fas-Mediated Off-Target Tumor Killing in T-cell Immunotherapy. Cancer Discov. 2021 Mar;11(3):599-613. doi: 10.1158/2159-8290.CD-20-0756. Epub 2020 Dec 17.

repository_institution: University of Illinois at Chicago - INDIGO

ReferenceCitation: Locke FL, Rossi JM, Neelapu SS, Jacobson CA, Miklos DB, Ghobadi A, Oluwole OO, Reagan PM, Lekakis LJ, Lin Y, Sherman M, Better M, Go WY, Wiezorek JS, Xue A, Bot A. Tumor burden, inflammation, and product attributes determine outcomes of axicabtagene ciloleucel in large B-cell lymphoma. Blood Adv. 2020 Oct 13;4(19):4898-4911. doi: 10.1182/bloodadvances.2020002394.

ReferenceCitation: Locke FL, Ghobadi A, Jacobson CA, Miklos DB, Lekakis LJ, Oluwole OO, Lin Y, Braunschweig I, Hill BT, Timmerman JM, Deol A, Reagan PM, Stiff P, Flinn IW, Farooq U, Goy A, McSweeney PA, Munoz J, Siddiqi T, Chavez JC, Herrera AF, Bartlett NL, Wiezorek JS, Navale L, Xue A, Jiang Y, Bot A, Rossi JM, Kim JJ, Go WY, Neelapu SS. Long-term safety and activity of axicabtagene ciloleucel in refractory large B-cell lymphoma (ZUMA-1): a single-arm, multicentre, phase 1-2 trial. Lancet Oncol. 2019 Jan;20(1):31-42. doi: 10.1016/S1470-2045(18)30864-7. Epub 2018 Dec 2.

ReferenceCitation: Neelapu SS, Locke FL, Bartlett NL, Lekakis LJ, Miklos DB, Jacobson CA, Braunschweig I, Oluwole OO, Siddiqi T, Lin Y, Timmerman JM, Stiff PJ, Friedberg JW, Flinn IW, Goy A, Hill BT, Smith MR, Deol A, Farooq U, McSweeney P, Munoz J, Avivi I, Castro JE, Westin JR, Chavez JC, Ghobadi A, Komanduri KV, Levy R, Jacobsen ED, Witzig TE, Reagan P, Bot A, Rossi J, Navale L, Jiang Y, Aycock J, Elias M, Chang D, Wiezorek J, Go WY. Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma. N Engl J Med. 2017 Dec 28;377(26):2531-2544. doi: 10.1056/NEJMoa1707447. Epub 2017 Dec 10.

location_facility: University of California San Diego (UCSD)

location_facility: Cleveland Clinic - Taussig Cancer Institute

location_facility: University of Iowa Hospitals and Clinics

location_facility: University of California Los Angeles (UCLA)

location_facility: Sarah Cannon-Methodist Healthcare System - San Antonio

location_facility: Washington University School of Medicine

title: A Phase 1/2 Multicenter Study Evaluating the Safety and Efficacy of KTE-C19 in Adults With Refractory Aggressive Non-Hodgkin Lymphoma

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

location_country:
Germany
France
Netherlands
United States
Canada
Israel


title: A Phase 3b, Randomized, Double-Blind Study to Evaluate Switching From a Regimen Consisting of Efavirenz/Emtricitabine/Tenofovir Disoproxil Fumarate (EFV/FTC/TDF) Fixed Dose Combination (FDC) to Emtricitabine/Rilpivirine/ Tenofovir Alafenamide (FTC/RPV/TAF) FDC in Virologically-Suppressed, HIV-1 Infected Subjects

summary: The primary objective of this study is to evaluate the non-inferiority of switching to emtricitabine/rilpivirine/tenofovir alafenamide (FTC/RPV/TAF) fixed dose combination (FDC) as compared to continuing the non-nucleoside reverse transcriptase inhibitor (NNRTI) regimen of efavirenz /FTC/tenofovir disoproxil fumarate (EFV/FTC/TDF) FDC in virologically-suppressed HIV-1 infected participants.

ReferenceCitation: Hagins D, Orkin C, Daar ES, Mills A, Brinson C, DeJesus E, Post FA, Morales-Ramirez J, Thompson M, Osiyemi O, Rashbaum B, Stellbrink HJ, Martorell C, Liu H, Liu YP, Porter D, Collins SE, SenGupta D, Das M. Switching to coformulated rilpivirine (RPV), emtricitabine (FTC) and tenofovir alafenamide from either RPV, FTC and tenofovir disoproxil fumarate (TDF) or efavirenz, FTC and TDF: 96-week results from two randomized clinical trials. HIV Med. 2018 Nov;19(10):724-733. doi: 10.1111/hiv.12664. Epub 2018 Aug 12.

repository_institution: PubMed Central - Europe PMC

ReferenceCitation: Mills A, Brinson C, Martorell C, Crofoot G, Daar E, Osiyemi O, et al. Switching to RPV/FTC/TAF from RPV/FTC/TDF or EFV/FTC/TDF: Week 96 Results. Conference on Retroviruses and Opportunistic Infections, Boston. March 4-7, 2018, Abstract 504.

ReferenceCitation: Arribas JR, Rockstroh J, Post, Yazdanpanah Y, Cavassini, DeJesus E, et al. Bone and renal safety of switching to rilpivirine/emtricitabine/tenofovir alafenamide (RPV/FTC/TAF) from single-tablet regimens (STRs) containing efavirenz/emtricitabine/tenofovir disoproxil fumarate (EFV/FTC/TDF) or rilpivirine/emtricitabine/tenofovir disoproxil fumarate (RPV/FTC/TDF): Week 48 subgroup analysis in patients at risk of or with comorbidities. Abstract accepted for presentation at the 16th European AIDS Conference, 2017 25-27 October Milan, Italy.

ReferenceCitation: Porter DP, KulkarniR, Cao H, SenGupta D, and White KL. No Emergent Resistance in HIV-1 Virologically-Suppressed Subjects Who Switched to RPV/FTC/TAF [Poster 1381]. ID Week 2017 4-8 October; San Diego, California.

ReferenceCitation: E DeJesus, M Ramgopal, G Crofoot, P Ruane, A LaMarca. J-M Molina et al. Efficacy and Safety of Switching to RPV/FTC/TAF in Older Adults. 8th International Workshop on HIV and Aging 2017 2-3 October, New York, New York.

ReferenceCitation: Molina JM, DeJesus E, Rijnders B, Post FAV, B., Stoeckle M, ThalmeA, et al. Efficacy and Safety of Switching From RPV/FTC/TDF or EFV/FTC/TDF to RPV/FTC/TAF in Black Adults [Presentation MOPEB0291]. 9th IAS Conference on HIV Science 2017 23-26 July Paris, France.

ReferenceCitation: Rockstroh J, Orkin C, Yazdanpanah Y, Di Perri GDS, P. E., Arribas JR, Brinkman K, et al. Switching From TDF to TAF Improves Bone and Renal Safety Independent of Age, Sex, Race, or 3rd Agent: Results From Pooled Analysis (N=3816) of Virologically Suppressed HIV-1 Infected Adults [Presentation MOPEB0289]. 9th IAS Conference on HIV Science; 2017 23 26 July Paris, France.

ReferenceCitation: DeJesus E, Ramgopal M, Crofoot G, Ruane P, LaMarca A, Mills A, Martorell CT, de Wet J, Stellbrink HJ, Molina JM, Post FA, Valero IP, Porter D, Liu Y, Cheng A, Quirk E, SenGupta D, Cao H. Switching from efavirenz, emtricitabine, and tenofovir disoproxil fumarate to tenofovir alafenamide coformulated with rilpivirine and emtricitabine in virally suppressed adults with HIV-1 infection: a randomised, double-blind, multicentre, phase 3b, non-inferiority study. Lancet HIV. 2017 May;4(5):e205-e213. doi: 10.1016/S2352-3018(17)30032-2. Epub 2017 Mar 2.

ReferenceCitation: Majeed SR, Shao Y, Garner W, Scott J, Pérez-Ruixo C, SenGupta D, et al. Evaluation of RPV/FTC/TAF Exposure-Efficacy and Exposure-Safety Relationships [Poster 427]. Conference on Retroviruses and Opportunistic Infections (CROI) 2017 13-16 February; Seattle, Washington.

ReferenceCitation: Hagins D, Mills A, Martorell C, Walmsley S, Gallant J, Tebas P, et al. Efficacy and Safety of Switching to RPV/FTC/TAF in Women [Abstract 12]. 7th International Workshop on HIV & Women; 2017 11-12 February; Seattle, Washington.

ReferenceCitation: Orkin C, DeJesus E, Ramgopal M, Crofoot G, Ruane P, LaMarca A, et al. 48 Week Results from two studies: Switching to RPV/FTC/TAF from EFV/FTC/TDF (Study 1160) or RPV/FTC/TDF (Study 1216) [Presentation]. HIV Glasgow; 2016 23-26 October; Glasgow, United Kingdom.

lead_sponsor: Gilead Sciences

location_country:
Germany
United Kingdom
Switzerland
Belgium
United States
Spain
Canada
Puerto Rico
France

location_facility: North Texas Infectious Diseases Consultants

location_facility: Long Beach Education and Research Consultants

location_facility: Center for HIV and Hepatogastroenterology

location_facility: University of Puerto Rico School of Medicine

location_facility: AIDS Arms, Inc./Trinity Health & Wellness Center

location_facility: AIDS Research & Treatment Center of the Treasure Coast

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Infectious Disease Specialists of Atlanta

location_facility: University of North Carolina at Chapel Hill

location_facility: Infectious Disease Research Institute Inc.

location_facility: Midway Immunology & Research Center, LLC

location_facility: Clinical Alliance for Research & Education - Infectious Diseases, LLC (CARE-ID)

location_facility: World Health Clinicians' CIRCLE CARE Center

location_facility: Columbia University Medical Center/ New York Presbyterian

location_facility: Hospital General Universitario de Alicante

location_facility: St. Joseph's Comprehensive Research Institute

location_facility: Zentrum für Infektiologie Berlin Prenzlauer Berg GmbH (zibp)

location_facility: Baystate Infectious Diseases Clinical Research

location_facility: La Playa Medical Group and Clinical Research

location_facility: Southwest Infectious Disease Clinical Research, Inc.

location_facility: Infectious Diseases Associates of NW Florida, P.A.

location_facility: Trinity Health and Wellness Center/AIDS Arms, Inc.

location_facility: Los Angeles BioMedical Institute at Harbor-UCLA Medical Center


title: A Multicenter, Randomized, Open-Label Study to Assess the Impact of Natalizumab Versus Fingolimod on Central Nervous System Tissue Damage and Recovery in Active Relapsing-Remitting Multiple Sclerosis Subjects

acronym: REVEAL

summary: The primary objective of this study is to assess the effect of natalizumab compared to fingolimod on the evolution of new on-treatment T1-gadolinium-enhancing (Gd+) lesions to persistent black holes (PBH) over 52 weeks. The secondary objectives of this study in this study population are to assess the effect of natalizumab compared to fingolimod on: magnetic resonance imaging (MRI) measures of central nervous system (CNS) tissue destruction as measured by the number of new T1-Gd+ lesions; various other MRI measures of disease activity; No Evidence of Disease Activity (NEDA); Relapse on treatment over 52 weeks; The change in information processing speed as measured by the Symbol Digit Modalities Test (SDMT).

ReferenceCitation: Butzkueven H, Licata S, Jeffery D, Arnold DL, Filippi M, Geurts JJ, Santra S, Campbell N, Ho PR; REVEAL Investigators. Natalizumab versus fingolimod for patients with active relapsing-remitting multiple sclerosis: results from REVEAL, a prospective, randomised head-to-head study. BMJ Open. 2020 Oct 20;10(10):e038861. doi: 10.1136/bmjopen-2020-038861.

repositories_concat: PubMed Central;University of Melbourne - Minerva Access

lead_sponsor: Biogen

location_country:
Germany
Italy
United Kingdom
Australia
Sweden
United States
Spain
Czechia
France


title: A Multi-national, Multi-center, Prospective, Randomized, Double Blinded, Placebo-controlled Trial to Evaluate the Efficacy of HyperBox Cyclical Topical Wound Oxygen Therapy (TWO2) in the Treatment of Chronic Diabetic Foot Ulcers

summary: The purpose of this study is to evaluate the efficacy, safety and economical benefits of Cyclical Pressure Topical Wound Oxygen (TWO2) Therapy in the treatment of chronic diabetic foot ulcers. Subjects will wear a standardized off-loading device and use advanced moist wound therapy (AMWT) dressings. Following a 2 week run-in period with the standardized care and after meeting all the eligibility criteria, half the subjects will use the TWO2 device, while the other half will use a sham device.

ReferenceCitation: Frykberg RG, Franks PJ, Edmonds M, Brantley JN, Teot L, Wild T, Garoufalis MG, Lee AM, Thompson JA, Reach G, Dove CR, Lachgar K, Grotemeyer D, Renton SC; TWO2 Study Group. A Multinational, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy of Cyclical Topical Wound Oxygen (TWO2) Therapy in the Treatment of Chronic Diabetic Foot Ulcers: The TWO2 Study. Diabetes Care. 2020 Mar;43(3):616-624. doi: 10.2337/dc19-0476. Epub 2019 Oct 16.

publisher_dissemination: American Diabetes Association

lead_sponsor: AOTI Ltd.

location_country:
Germany
United Kingdom
United States
Luxembourg
France

location_facility:
Montpellier University Hospital
Carl T. Hayden VA Medical Center
Avicenne Hospital
Kirchberg Hospital
Advanced Foot & Ankle Center
Northwick Park Hospital
NorthBay Center for Wounds
Hospital Simone Veil
Städtisches Klinikum Dessau
Hunter Holmes McGuire VA Medical Center
King's College Hospital
Edward Hines, Jr. VA Hospital
Advanced Research Institute of Miami
Salem VA Medical Center
The Research Center Inc.
Washington DC VA Medical Center

intervention_type: Device


title: Open-Label, Multicenter, Dose Escalation Phase Ib Study With Expansion Phase to Evaluate the Safety, Pharmacokinetics, and Activity of RO5509554 (Emactuzumab) and MPDL3280A (Atezolizumab) Administered in Combination in Patients With Advanced Solid Tumors

summary:
This Phase 1, open-label, multicenter, global study will evaluate the safety, pharmacokinetics, and activity of emactuzumab and atezolizumab administered in combination in participants with selected locally advanced or metastatic solid tumors that are not amenable to standard treatment.

Participants who receive emactuzumab and atezolizumab will continue to receive study drug as long as they experience clinical benefit in the opinion of the investigator or until unacceptable toxicity or symptomatic deterioration attributed to disease progression as determined by the investigator after an integrated assessment of radiographic data, biopsy results (if available), and clinical status, or withdrawal of consent.

ReferenceCitation: Gomez-Roca C, Cassier P, Zamarin D, Machiels JP, Perez Gracia JL, Stephen Hodi F, Taus A, Martinez Garcia M, Boni V, Eder JP, Hafez N, Sullivan R, Mcdermott D, Champiat S, Aspeslagh S, Terret C, Jegg AM, Jacob W, Cannarile MA, Ries C, Korski K, Michielin F, Christen R, Babitzki G, Watson C, Meneses-Lorente G, Weisser M, Ruttinger D, Delord JP, Marabelle A. Anti-CSF-1R emactuzumab in combination with anti-PD-L1 atezolizumab in advanced solid tumor patients naive or experienced for immune checkpoint blockade. J Immunother Cancer. 2022 May;10(5):e004076. doi: 10.1136/jitc-2021-004076.

repository_institution: Universitat Pompeu Fabra - Repositori digital de la UPF

repository_institution: Vrije Universiteit Brussel - VUBIR

repository_normalized: vubissmart

repositories:
PubMed Central
repositori.upf.edu
vubissmart

lead_sponsor: Hoffmann-La Roche

location_country:
United States
Spain
Belgium
France

location_facility: Institut Claudius Regaud; Departement Oncologie Medicale

location_facility: Memorial Sloan-Kettering Cancer Center Breast & Imaging Center

location_facility: Clinica Universitaria de Navarra; Servicio de Oncologia

location_facility: Centro Integral Oncológico Clara Campal Ensayos Clínicos START

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale

location_facility: Centre Leon Berard; Departement Oncologie Medicale


title: A Randomized, Open-label, 2-arm Parallel-group, Multicenter, 26-week Study Assessing the Safety and Efficacy of H0E901-U300 Versus Lantus in Older Patients With Type 2 Diabetes Inadequately Controlled on Antidiabetic Regimens Either Including no Insulin, or With Basal Insulin as Their Only Insulin

acronym: SENIOR

summary:
Primary Objective:

To demonstrate the non-inferiority of H0E901-U300 to Lantus, in change of glycated hemoglobin A1c (HbA1c).

Secondary Objectives:

To demonstrate the superiority of H0E901-U300 in comparison with Lantus in:

Percentage of participants with at least one severe and/or confirmed (by plasma glucose ≤70mg/dL [3.9mmol/L]) hypoglycemia event from 22:00 to 08:59 next morning
Percentage of participants with at least one nocturnal (from 00:00-05:59) severe and/or confirmed (≤70mg/dL [3.9mmol/L]) hypoglycemia event
Percentage of participants with at least one severe and/or confirmed (by plasma glucose ≤70mg/dL [3.9mmol/L]) hypoglycemia event occurring at any time of day
HbA1c change

ReferenceCitation: Ritzel R, Harris SB, Baron H, Florez H, Roussel R, Espinasse M, Muehlen-Bartmer I, Zhang N, Bertolini M, Brulle-Wohlhueter C, Munshi M, Bolli GB. A Randomized Controlled Trial Comparing Efficacy and Safety of Insulin Glargine 300 Units/mL Versus 100 Units/mL in Older People With Type 2 Diabetes: Results From the SENIOR Study. Diabetes Care. 2018 Aug;41(8):1672-1680. doi: 10.2337/dc18-0168. Epub 2018 Jun 12. Erratum In: Diabetes Care. 2019 Aug;42(8):1604.

lead_sponsor: Sanofi

location_country:
Germany
Italy
Poland
Colombia
Hungary
Argentina
Romania
Australia
Japan
Sweden
Mexico
United Kingdom
United States
Korea, Republic of
Spain
Peru
Canada
France

location_facility:
Investigational Site Number 840065
Investigational Site Number 276005
Investigational Site Number 410004
Investigational Site Number 840028
Investigational Site Number 170007
Investigational Site Number 840012
Investigational Site Number 484004
Investigational Site Number 276003
Investigational Site Number 032002
Investigational Site Number 724004
Investigational Site Number 840029
Investigational Site Number 036002
Investigational Site Number 392004
Investigational Site Number 170004
Investigational Site Number 380003
Investigational Site Number 724008
Investigational Site Number 392008
Investigational Site Number 124006
Investigational Site Number 840070
Investigational Site Number 840003
Investigational Site Number 642003
Investigational Site Number 724001
Investigational Site Number 840038
Investigational Site Number 840064
Investigational Site Number 124007
Investigational Site Number 642006
Investigational Site Number 392013
Investigational Site Number 840036
Investigational Site Number 840020
Investigational Site Number 840018
Investigational Site Number 724005
Investigational Site Number 840055
Investigational Site Number 616003
Investigational Site Number 348001
Investigational Site Number 840013
Investigational Site Number 348005
Investigational Site Number 348003
Investigational Site Number 840051
Investigational Site Number 840062
Investigational Site Number 840002
Investigational Site Number 840001
Investigational Site Number 840037
Investigational Site Number 840023
Investigational Site Number 840014
Investigational Site Number 724007
Investigational Site Number 826004
Investigational Site Number 484001
Investigational Site Number 840039
Investigational Site Number 032006
Investigational Site Number 840025
Investigational Site Number 840040
Investigational Site Number 036003
Investigational Site Number 124002
Investigational Site Number 410002
Investigational Site Number 410003
Investigational Site Number 840047
Investigational Site Number 410005
Investigational Site Number 124003
Investigational Site Number 840054
Investigational Site Number 840060
Investigational Site Number 642007
Investigational Site Number 840017
Investigational Site Number 348007
Investigational Site Number 840072
Investigational Site Number 616002
Investigational Site Number 724003
Investigational Site Number 840024
Investigational Site Number 752004
Investigational Site Number 642002
Investigational Site Number 840021
Investigational Site Number 840041
Investigational Site Number 840069
Investigational Site Number 124010
Investigational Site Number 032004
Investigational Site Number 642001
Investigational Site Number 604004
Investigational Site Number 124004
Investigational Site Number 840026
Investigational Site Number 840009
Investigational Site Number 250004
Investigational Site Number 840030
Investigational Site Number 604006
Investigational Site Number 724006
Investigational Site Number 840032
Investigational Site Number 276002
Investigational Site Number 840022
Investigational Site Number 604007
Investigational Site Number 840059
Investigational Site Number 840048
Investigational Site Number 484003
Investigational Site Number 348008
Investigational Site Number 642005
Investigational Site Number 250005
Investigational Site Number 348004
Investigational Site Number 036004
Investigational Site Number 642004
Investigational Site Number 124008
Investigational Site Number 840006
Investigational Site Number 616005
Investigational Site Number 840046
Investigational Site Number 604003
Investigational Site Number 604005
Investigational Site Number 840057
Investigational Site Number 276001
Investigational Site Number 124005
Investigational Site Number 840050
Investigational Site Number 840033
Investigational Site Number 348009
Investigational Site Number 752003
Investigational Site Number 752002
Investigational Site Number 032001
Investigational Site Number 276004
Investigational Site Number 036001
Investigational Site Number 840045
Investigational Site Number 840053
Investigational Site Number 380005
Investigational Site Number 484002
Investigational Site Number 840004
Investigational Site Number 826005
Investigational Site Number 840042
Investigational Site Number 840016
Investigational Site Number 826001
Investigational Site Number 032003
Investigational Site Number 170001
Investigational Site Number 724002
Investigational Site Number 170005
Investigational Site Number 124009
Investigational Site Number 616004
Investigational Site Number 250006
Investigational Site Number 616001
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Investigational Site Number 840007
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Investigational Site Number 124001
Investigational Site Number 348002
Investigational Site Number 840019
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Investigational Site Number 380001
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Investigational Site Number 380004
Investigational Site Number 826006
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Investigational Site Number 826003
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Investigational Site Number 392002
Investigational Site Number 392003
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Investigational Site Number 604002
Investigational Site Number 380002
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Investigational Site Number 392012
Investigational Site Number 604001
Investigational Site Number 752001
Investigational Site Number 840005
Investigational Site Number 840015
Investigational Site Number 170003
Investigational Site Number 410001


acronym: COBALT

lead_sponsor: Intercept Pharmaceuticals

summary: Primary Biliary Cholangitis (PBC) is a serious, life-threatening, bile acid related liver disease of unknown cause. Without treatment, it frequently progresses to liver fibrosis and eventual cirrhosis requiring liver transplantation or resulting in death. The investigational drug, Obeticholic Acid (OCA) is a modified bile acid and FXR agonist that is derived from the primary human bile acid chenodeoxycholic acid. The key mechanisms of action of OCA, including its choleretic, anti-inflammatory, and anti-fibrotic properties, underlie its hepatoprotective effects and result in attenuation of injury and improved liver function in a cholestatic liver disease such as PBC. The study will assess the effect of OCA compared to placebo, combined with stable standard care, on clinical outcomes in PBC participants.

ReferenceType: background

ReferenceCitation: Lindor KD, Gershwin ME, Poupon R, Kaplan M, Bergasa NV, Heathcote EJ; American Association for Study of Liver Diseases. Primary biliary cirrhosis. Hepatology. 2009 Jul;50(1):291-308. doi: 10.1002/hep.22906. No abstract available.

ReferenceCitation: European Association for the Study of the Liver. EASL Clinical Practice Guidelines: management of cholestatic liver diseases. J Hepatol. 2009 Aug;51(2):237-67. doi: 10.1016/j.jhep.2009.04.009. Epub 2009 Jun 6. No abstract available.

location_facility: Gastrocentro/ Universidade Estadual de Campinas (UNICAMP)

location_facility: Azienda Socio-Sanitarla Territoriale (ASST) Santi Paolo e Carlo

location_facility: Southern Therapy and Advanced Research (STAR)

location_facility: University Of Debrecen, Department Of Medicine, Division Of Gastroenterology

location_facility: Department of Gastroenterology & Hepatology, Nepean Hospital

location_facility: Virgen De La Victoria University Hospital

location_facility: Amsterdam University Medical Centre (AMC)

location_facility: Hospital Privado Universitario de Cordoba S.A.

location_facility: Wojewodzki Szpital Specjalistyczny im. J. Gromkowskiego, Pierwszy Oddzial Chorob Zakaznych

location_facility: Nottingham University Hospital Nhs Trust

location_facility: USZ, Klinik für Gastroenterologie und Hepatologie

location_facility: UT Health The University of Texas Health Science Center at Houston

location_facility: University of Colorado Denver and Hospital

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

location_facility: AOU Ospedale Civile S. Agostino Estense - UO Medicina Metabolica

location_facility: Azienda Ospedaliero Universitaria Careggi Universita di Firenze

location_facility: Instituto Hospital de Base do Distrito Federal-IHBDF

location_facility: Hospital de Gastroenterologia Dr. Carlos Bonorino Udaondo

location_facility: Hospital Universitario Professor Edgard Santos

location_facility: Baylor College of Medicine - Advanced Liver Therapies

location_facility: Northwestern University Feinberg School of Medicine

location_facility: Azienda Ospedaliero - Universita di Padova

location_facility: NZ Liver Transplant Unit, Auckland Hospital

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Medicinsk klinik for mave, tarm-og leversygdomme

location_facility: University of Alberta, Walter C. Mackenzie Health Sciences Centre (WMC)

location_facility: Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)

location_facility: East Tallinn Central Hospital Gastroenterology Center

location_facility: Kantonsspital St. Gallen, Clinic for Gastroeterologie and Hepatologie

location_facility: Vilnius University Hospital Santaros klinikos

location_facility: Békés Megyei Kozponti Korhaz - Dr. Rethy Pal Tagkorhaz

location_facility: Universidade Federal do Estado do Rio de Janeiro - Hospital Universitario Gaffree e Guinle/HUGG/UNIRIO

location_facility: Departamento De Gastroenterologia. Instituto Nacional De Ciencias Medicas Y Nutricion Salvador Zubiran

location_facility: University of Munich, LMU Klinikum Grosshadern

location_facility: Azienda Ospedaliero - Universitaria di Cagliari - Centro per lo Studio delle Malattie del Fegato

location_facility: Indianapolis Gastroenterology Research Foundation

location_facility: Policlinico Universitario Agostino Gemelli - Universita Cattolica del Sacro Cuore

location_facility: Inselspital, University Of Bern, UVCM, DMLL

location_facility: Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo-HCFMUSP

location_facility: University Of Manitoba, Health Sciences Centre

location_facility: Medical University of Warsaw Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie (SPCSK in Warsaw

location_facility: Hospital De Clinicas University Of Buenos Aires

location_facility: London Health Sciences Centre-University Hospital

location_facility: Hadassah Hebrew University Medical Center

location_facility: Università Politecnica delle Marche - Azienda Ospedaliero

location_facility: Klinikum der Johann-Wolfgang Goethe, Universitaet Frankfurt am Main

location_facility: CIPREC - Centro de Investigacion y Prevencion Cardiovascular S.A.

location_facility: Ankara University School of Medicine Gastroenterology Dept.

location_facility: University of California Davis, Davis Medical Center

location_facility: Cambrigde University Hospitals NHS Foundation Trust

location_facility: Universitaetsklinikum Heidelberg Medizinische Universitaetsklinik

location_facility: Fiona Stanley Hospital, Gastroenterology Department

location_facility: University of Calgary Liver Unit (Heritage Medical Research Clinic)

location_facility: The Liver Institute at Methodist Dallas Medical Center

location_facility: Seoul National University Bundang Hospital

location_facility: Turku University Central Hospital, Gastroenterology Outpatient Clinic

location_facility: Univeritatsklinikum Hamburg Eppendorf, Medizinische Klinik und Poliklinik

location_facility: Centrum Onkologii - Instytut im. Marii Skłodowskiej - Curie, Klinika Gastroenterologii Onkologicznej

location_facility: Gastrocentro. Unidad Estadual de CAmpinas UNICAMP

location_facility: Odense University Hospital Afdeling for medicinske mave-tarmsygdomme S

location_facility: Aarhus University Hospital Department of Hepatology and Gastroenterology

location_facility: American Research Corporation at Texas Liver Institute

location_facility: University Health Network, Toronto General Hospital

location_facility: Hospital Universitario Marqués De Valdecilla

location_facility: Ege University School of Medicine Gastroenterology Dept.

location_facility: Centro De Investigaciones Clínicas Viña Del Mar

location_facility: Saint Louis University Gastroenterology & Hepatology

location_facility: Azienda Ospedaliero Universitaria Sant´ Orsola Malpighi

location_facility: Plymouth Hospitals NHS Trust, Derriford Hospital

location_facility: Hospital of Lithuanian University of Health Sciences, Kauno klinikos

location_facility: Azienda Socio Sanitaria Territoriale (ASST) di Monza

location_facility: AW Morrow Gastroenterology and Liver Centre, Royal Prince Alfred Hospital

location_facility: Consultorio Médico de la Dra Alma Laura Ladrón de Guevara Cetina

location_facility: University of Louisville, Medical Dental Complex

location_facility: Department of Gastroenterology and Hepatology, Royal Adelaide Hospital

location_facility: Medizinische Hochschule Hannover, Klinik für Gastroenterologie, Hepatologie und Endokrinologie

location_facility: Hospital of the University of Pennsylvania

location_facility: AOU Policlinico Paolo Giaccone - Di.Bi.M.I.S

location_facility: Schiff Center for Liver Diseases/University of Miami

location_facility: Universität Leipzig KöR, Medizinische Fakultät

location_facility: Vrije Universiteit Medisch Centrum (VUMC)

location_facility: Hospital Universitario Puerta De Hierro Majadahonda

location_facility: Baylor Scott and White Research Institute

location_facility: Oddział Gastr. I Hepat. Uniwersyteckie Centrum Kliniczne Im. Prof. K. Gibińskiego Sum

location_facility: Clinic For Gastroenterology And Hepatology Clinical Center Of Serbia

title: A Phase 4, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Effect of Obeticholic Acid on Clinical Outcomes in Patients With Primary Biliary Cholangitis

location_country:
Finland
Germany
Poland
New Zealand
Argentina
Australia
Brazil
Turkey
Chile
Canada
Serbia
France
Lithuania
Portugal
Hungary
Hong Kong
Denmark
Switzerland
United States
Belgium
Estonia
Italy
United Kingdom
Sweden
Netherlands
Austria
Mexico
Korea, Republic of
Spain
Bulgaria
Israel


lead_sponsor: BioCryst Pharmaceuticals

summary: This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in subjects with hereditary angioedema (HAE). Eligible subjects will be randomized to receive one of two doses of BCX4161 or placebo for 12 weeks. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical outcomes, and the safety and tolerability of each dose of BCX4161 compared to placebo.

ReferenceCitation: Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.

title: OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema

primary_purpose: Prevention

location_country:
Germany
Italy
Hungary
United Kingdom
Belgium
United States
Canada
France