title: An Evaluation of Effectiveness and Safety of the CorPath® GRX System in Endovascular Embolization Procedures of Cerebral Aneurysms

summary: The primary objective of this study is to evaluate the effectiveness and safety of robotic-assisted endovascular embolization procedures compared to objective performance criteria for traditional, manual operation based on the scientific literature.

ReferenceCitation: Lawton MT, Vates GE. Subarachnoid Hemorrhage. N Engl J Med. 2017 Jul 20;377(3):257-266. doi: 10.1056/NEJMcp1605827. Review.

ReferenceCitation: Lantigua H, Ortega-Gutierrez S, Schmidt JM, Lee K, Badjatia N, Agarwal S, Claassen J, Connolly ES, Mayer SA. Subarachnoid hemorrhage: who dies, and why? Crit Care. 2015 Aug 31;19:309. doi: 10.1186/s13054-015-1036-0.

ReferenceCitation: Connolly ES Jr, Rabinstein AA, Carhuapoma JR, Derdeyn CP, Dion J, Higashida RT, Hoh BL, Kirkness CJ, Naidech AM, Ogilvy CS, Patel AB, Thompson BG, Vespa P; American Heart Association Stroke Council; Council on Cardiovascular Radiology and Intervention; Council on Cardiovascular Nursing; Council on Cardiovascular Surgery and Anesthesia; Council on Clinical Cardiology. Guidelines for the management of aneurysmal subarachnoid hemorrhage: a guideline for healthcare professionals from the American Heart Association/american Stroke Association. Stroke. 2012 Jun;43(6):1711-37. doi: 10.1161/STR.0b013e3182587839. Epub 2012 May 3.

ReferenceCitation: Steiner T, Juvela S, Unterberg A, Jung C, Forsting M, Rinkel G; European Stroke Organization. European Stroke Organization guidelines for the management of intracranial aneurysms and subarachnoid haemorrhage. Cerebrovasc Dis. 2013;35(2):93-112. doi: 10.1159/000346087. Epub 2013 Feb 7. Review.

ReferenceCitation: Keedy A. An overview of intracranial aneurysms. Mcgill J Med. 2006 Jul;9(2):141-6.

ipd_sharing_description: No plan to share IPD.

lead_sponsor: Corindus Inc.

location_country: Canada Australia France Spain

location_facility: University Clinical Hospital of Valladolid


type: EudraCT Number

title: A Multicentre, Open-Label Phase I/II Study to Evaluate the Safety, Tolerability, Biodistribution and Anti Tumour Activity of 177LU-OPS201 With Companion Imaging 68Ga OPS202 PET/CT in Previously Treated Subjects With Locally Advanced or Metastatic Cancers Expressing Somatostatin Receptor 2 (SSTR2)

summary: This study consists of two phases. The phase I study is designed to investigate the safety and tolerability of Satoreotide tetraxetan following fractionated i.v. administrations in pre-treated subjects with locally advanced or metastatic cancers expressing sstr2 as identified by Satoreotide trizoxetan Positron Emission Tomography (PET/CT) scans. This phase will encompass both radioactivity escalation and peptide mass dose evaluation. Phase II will assess the efficacy of Satoreotide tetraxetan in subjects in selected indications, in a basket design.

study_start_date_type: Actual

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Other

enrollment_type: Actual

ipd_sharing: Yes

ipd_sharing_description: Where patient data can be anonymised, Ipsen will share all individual participant data that underlie the results reported in the published journal article with qualified researchers who provide a valid research question. Study documents, such as the study protocol and clinical study report, are not always available.

lead_sponsor: Ipsen

location_country:
Austria
Switzerland
Germany
France
Belgium
United States
United Kingdom

location_facility:
University Hospital (UZ) Leuven
MD Anderson Cancer Center
Medical University of Innsbruck
Universitaetsklinikum Essen
Royal Marsden Hospital - Surrey
CHU de Bordeaux - Hôpital Haut Lévêque
CHU de Marseille - Hôpital la Timone
University Hospital Basel

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: A Prospective Multicenter Clinical Study to Evaluate the Safety and Effectiveness of Subepithelial Implantation of the Transform Corneal Allograft (TCA) for Improving Near Vision in Presbyopic Subjects

summary: The objective of this clinical study is to evaluate the safety and effectiveness of subepithelial implantation of the Allotex TransForm corneal allograft (TCA) for improving near vision in presbyopic subjects.

study_start_date_type: Anticipated

status: Withdrawn

study_completion_date_type: Anticipated

primary_purpose: Treatment

lead_sponsor: Allotex, Inc.

location_country:
Austria
Switzerland
France
Ireland
Belgium
United Kingdom

location_facility: Corneo Plastic Unit and Eye Bank Queen Victoria Hospital

location_facility: Institute Laser Vision Noemie de Rothschild, Fondation Ophthalmolique Adolphe de Rothschild

intervention_type: Other


title: A Prospective Multicenter Clinical Study to Evaluate the Safety and Effectiveness of Intrastromal Implant of the Transform Corneal Allograft (TCA) for Providing Near Vision in Presbyopic Subjects

summary: The objective of this clinical study is to evaluate the safety and effectiveness of intrastromal implantation of the Allotex TransForm corneal allograft (TCA) for improving near vision in presbyopic subjects.

status: Active, not recruiting

status_simplified: Ongoing


title: A Prospective Multi Center Clinical Study to Evaluate the Safety And Effectiveness of the Transform™ Corneal Allograft (TCA) for Treatment of Hyperopia

summary:
The objective of this clinical study is to evaluate the safety and effectiveness of intrastromal implantation of the Allotex TCA for improving distance vision in hyperopic subjects.

The overall objective with respect to visual outcome is to provide improved vision without the requirement of additional visual aids.


title: TransCatheter Aortic Valve Implantation and Fractional Flow Reserve-Guided Percutaneous Coronary Intervention Versus Conventional Surgical Aortic Valve Replacement and Coronary By-Pass Grafts for Treatment of Patients With Coronary MultiVessel Disease and Aortic Valve Stenosis

acronym: TCW

summary: The trial objective is to investigate whether Fractional Flow Reserve (FFR)-Guided Percutaneous Coronary Intervention (PCI) and TransCatheter Aortic Valve Implantation (TAVI) strategy for treatment of multivessel disease and aortic stenosis will be non-inferior to Coronary Artery By-pass Grafting (CABG) and Surgical Aortic Valve Replacement (SAVR) for a composite primary endpoint of all-cause mortality, stroke, myocardial infarction, coronary or valve re-intervention and life-threatening or disabling bleeding at one year.

status: Recruiting

design_allocation: Randomized

enrollment_type: Anticipated

ipd_sharing: Undecided

lead_sponsor: Maatschap Cardiologie Zwolle

location_country: Austria Netherlands Spain France Greece Slovakia Portugal Denmark Germany

location_facility: University Hospital Frankfurt - Goethe University

location_facility: Universitäres Herz- und Gefäßzentrum UKE Hamburg GmbH

location_facility: Rigshospitalet, Copenhagen University Hospital

location_facility: Hospital Clinico Universitario San Carlos

intervention_type: Device


type: Other Identifier

title: A Multi-centre, Randomised Controlled Study, to Evaluate the Safety and Performance of The DIALIVE Liver Dialysis Device (LDD) in Patients With Acute on Chronic Liver Failure (ACLF) Versus Standard of Care (SOC)

acronym: DIALIVE _ACLF

summary: The First-In-Man study is a multi-centre, randomised, controlled, study to generate data for the evaluation of safety and performance of DIALIVE Liver Dialysis Device in 24 evaluable patients with Acute on Chronic Liver Failure (ACLF) versus standard of care (SOC).

status: Completed

primary_purpose: Device Feasibility

ReferenceType: background

ReferenceCitation: Lee KC, Baker LA, Stanzani G, Alibhai H, Chang YM, Jimenez Palacios C, Leckie PJ, Giordano P, Priestnall SL, Antoine DJ, Jenkins RE, Goldring CE, Park BK, Andreola F, Agarwal B, Mookerjee RP, Davies NA, Jalan R. Extracorporeal liver assist device to exchange albumin and remove endotoxin in acute liver failure: Results of a pivotal pre-clinical study. J Hepatol. 2015 Sep;63(3):634-42. doi: 10.1016/j.jhep.2015.04.020. Epub 2015 May 1.

ipd_sharing: No

ipd_sharing_description: Only anonymized clinical data can be shared out of central database according to the H2020 project plan.

lead_sponsor: Yaqrit Ltd

location_country:
Austria
France
Romania
Belgium
Spain
Denmark
Germany
United Kingdom

location_facility:
university Hospital Graz
university hospital BASILDON
University Hospital of Rostock
University Hospital
Paul Brousse Hospital
Fundeni Clinical Institute
University Hospital Gregorio Maragnon
Royal Free Hospital London NHS
Queens Medical Centre
Hospital Clinic Barcelona
University Hospital Erasmus
Hôpital Beaujon

status_simplified: Completed


ipd_sharing_description: Qualified researchers may request access to participant level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Participant level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://www.clinicalstudydatarequest.com/.

lead_sponsor: Genzyme, a Sanofi Company

location_country:
France
Japan
United States
United Kingdom
Taiwan

acronym: Mini-COMET

location_facility:
Investigational Site Number 2500001
Investigational Site Number 2500003
Investigational Site Number 8260001
Investigational Site Number 8260002
Investigational Site Number 1580001
Investigational Site Number 3920002
Investigational Site Number 8400005
Investigational Site Number 3920001
Investigational Site Number 8400001
Investigational Site Number 8400002

source: euctr

title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response

summary:
Primary Objective:

To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset Pompe disease previously treated with alglucosidase alfa.

Secondary Objective:

To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.


ReferenceType: derived

ReferenceCitation: Steff AM, Cadieux-Dion C, de Lannoy G, Prato MK, Czeszak X, André B, Ingels DC, Louckx M, Dewé W, Picciolato M, Maleux K, Fissette L, Dieussaert I. Hamster neogenin, a host-cell protein contained in a respiratory syncytial virus candidate vaccine, induces antibody responses in rabbits but not in clinical trial participants. Hum Vaccin Immunother. 2020 Jun 2;16(6):1327-1337. doi: 10.1080/21645515.2019.1693749. Epub 2020 Jan 17.

family: Steff

full_name: Ann-Muriel Steff

given: Ann-Muriel

sequence: first

family: Cadieux-Dion

full_name: Chanel Cadieux-Dion

given: Chanel

sequence: additional

family: de Lannoy

full_name: Gaël de Lannoy

given: Gaël

family: Prato

full_name: Maria Key Prato

given: Maria Key

family: Czeszak

full_name: Xavier Czeszak

given: Xavier

family: André

full_name: Bruno André

given: Bruno

family: Ingels

full_name: Dominique C Ingels

given: Dominique C

family: Louckx

full_name: Marc Louckx

given: Marc

family: Dewé

full_name: Walthère Dewé

given: Walthère

family: Picciolato

full_name: Marta Picciolato

given: Marta

family: Maleux

full_name: Koen Maleux

given: Koen

family: Fissette

full_name: Laurence Fissette

given: Laurence

family: Dieussaert

full_name: Ilse Dieussaert

given: Ilse

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Human Vaccines & Immunotherapeutics

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

evidence: oa repository (via pmcid lookup)

host_type: repository

license_normalized: no license

repository_normalized: PubMed Central

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

ReferenceCitation: Schwarz TF, McPhee RA, Launay O, Leroux-Roels G, Talli J, Picciolato M, Gao F, Cai R, Nguyen TL, Dieussaert I, Miller JM, Schmidt AC. Immunogenicity and Safety of 3 Formulations of a Respiratory Syncytial Virus Candidate Vaccine in Nonpregnant Women: A Phase 2, Randomized Trial. J Infect Dis. 2019 Oct 22;220(11):1816-1825. doi: 10.1093/infdis/jiz395.

name: Institute of Laboratory Medicine and Vaccination Centre, Klinikum Würzburg Mitte, Würzburg, Germany

family: Schwarz

full_name: Tino F Schwarz

given: Tino F

name: GlaxoSmithKline (GSK), Rockville, Maryland

family: McPhee

full_name: Roderick A McPhee

given: Roderick A

name: Université de Paris, Inserm, clinical investigation center 1417, Assistance Publique–Hôpitaux de Paris, Hôpital Cochin, Paris, France

family: Launay

full_name: Odile Launay

given: Odile

name: Center for Vaccinology, Ghent University and University Hospital, Belgium

family: Leroux-Roels

full_name: Geert Leroux-Roels

given: Geert

name: Ravi-ja Uuringukeskus Innomedica OÜ, Tallinn, Estonia

family: Talli

full_name: Jaak Talli

given: Jaak

family: Gao

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

title: An Observer-blind Study to Rank Different Formulations of GSK Biologicals' Investigational RSV Vaccine (GSK3003891A) Administered to Healthy Women

summary: The purpose of this study is to rank different RSV vaccine dosages of antigen (or formulations) based on safety/reactogenicity and immune response data. The formulations eliciting strong immune responses while maintaining an acceptable safety profile will be considered for further evaluation, including in studies vaccinating pregnant women.


title: Assessing Clinical Outcomes Using the EDWARDS INTUITY Elite Valve System in Isolated AVR Using Minimally InvaSive Surgery In a EurOpean Multi-ceNter, Active, Post-market Registry

acronym: MISSION

summary: The purpose of this active, observational, open-label, non-randomized, post-market surveillance study is to confirm that EDWARDS INTUITY Elite reduces cross clamp time (XCT) in MIS setting when compared to published data with a conventional valve within the MIS setting. The published dataset will used as a control group. Then to describe short term (30 days) and long term (6 months) clinical safety, to assess and compare hemodynamic data with EDWARDS INTUITY Elite to a conventional valve at discharge and at 6 months post AVR, to assess Quality of Life at baseline, and at 6 months post AVR to assess NYHA functional class at baseline, discharge, 1 month and at 6 months post AVR to assess Fitness for hospital discharge.

study_type: Observational

time_perspective: Other

lead_sponsor: Edwards Lifesciences

location_country:
Italy
Austria
Luxembourg
Netherlands
France
Spain
Denmark
Germany
United Kingdom

location_facility: Herz- und Gefäß-Klinik GmbH Bad Neustadt

location_facility: G. Pasquinucci Heart Hospital - "G.Monasterio" Foundation

location_facility: Klinisshe Abteiluing Für Herz-thoraxchirurgie

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia"

location_facility: Università Cattolica del Sacro Cuore Policlinico

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: Universitätsklinikum der Ruhr-Universität Bochum Klinik für Herz- und Thoraxchirurgie

location_facility: Complexo Hospitalario Universitario A Coruna

location_facility: Universitätsklinik für Herzchirurgie Medizinische Universität Innsbruck

location_facility: Hôpital Cardiologique CHU Bordeaux Haut Leveque

location_facility: Institut National de Chirurgie Cardiaque et Cardiologie Interventionnelle (INCCI)


title: The SPACER Trial - Repair of Tricuspid Valve Regurgitation Using the Edwards TricuSPid TrAnsCatheter REpaiR System

acronym: SPACER

summary: The purpose of the study is to assess the safety and device performance of the Edwards Tricuspid Transcatheter Repair System in patients with clinically significant, symptomatic, tricuspid regurgitation who are at high surgical risk for standard tricuspid repair/replacement.

status: Unknown status

location_country: Switzerland France Greece Germany Canada

location_facility: Institut Universitaire de Cardiologie et de Pneumologie de Quebec-Universite Laval

location_facility: Munich University Clinic, Ludwig-Maximilian University

location_facility: Universitäts-Herzzentrum Freiburg - Bad Krozingen

location_facility: St. Paul's Hospital, Providence Health Care Research Institute


ReferenceType: result

ReferenceCitation: Simpson EL, Bieber T, Guttman-Yassky E, Beck LA, Blauvelt A, Cork MJ, Silverberg JI, Deleuran M, Kataoka Y, Lacour JP, Kingo K, Worm M, Poulin Y, Wollenberg A, Soo Y, Graham NM, Pirozzi G, Akinlade B, Staudinger H, Mastey V, Eckert L, Gadkari A, Stahl N, Yancopoulos GD, Ardeleanu M; SOLO 1 and SOLO 2 Investigators. Two Phase 3 Trials of Dupilumab versus Placebo in Atopic Dermatitis. N Engl J Med. 2016 Dec 15;375(24):2335-2348. Epub 2016 Sep 30.

ReferenceCitation: Griffiths C, de Bruin-Weller M, Deleuran M, Fargnoli MC, Staumont-Sallé D, Hong CH, Sánchez-Carazo J, Foley P, Seo SJ, Msihid J, Chen Z, Cyr SL, Rossi AB. Dupilumab in Adults with Moderate-to-Severe Atopic Dermatitis and Prior Use of Systemic Non-Steroidal Immunosuppressants: Analysis of Four Phase 3 Trials. Dermatol Ther (Heidelb). 2021 Aug;11(4):1357-1372. doi: 10.1007/s13555-021-00558-0. Epub 2021 Jun 18.

family: Griffiths

full_name: Christopher Griffiths

given: Christopher

family: de Bruin-Weller

full_name: Marjolein de Bruin-Weller

given: Marjolein

family: Deleuran

full_name: Mette Deleuran

given: Mette

family: Fargnoli

full_name: Maria Concetta Fargnoli

given: Maria Concetta

family: Staumont-Sallé

full_name: Delphine Staumont-Sallé

given: Delphine

family: Hong

full_name: Chih-ho Hong

given: Chih-ho

family: Sánchez-Carazo

full_name: Jose Sánchez-Carazo

given: Jose

family: Foley

full_name: Peter Foley

given: Peter

family: Seo

full_name: Seong Jun Seo

given: Seong Jun

family: Msihid

full_name: Jérôme Msihid

given: Jérôme

family: Chen

full_name: Zhen Chen

given: Zhen

family: Cyr

full_name: Sonya L. Cyr

given: Sonya L.

family: Rossi

full_name: Ana B. Rossi

given: Ana B.

journal_name: Dermatology and Therapy

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by-nc

license_normalized: cc-by-nc

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

repository_normalized: University of Melbourne - Minerva Access

version: submittedVersion

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Hamilton JD, Harel S, Swanson BN, Brian W, Chen Z, Rice MS, Amin N, Ardeleanu M, Radin A, Shumel B, Ruddy M, Patel N, Pirozzi G, Mannent L, Graham NMH. Dupilumab suppresses type 2 inflammatory biomarkers across multiple atopic, allergic diseases. Clin Exp Allergy. 2021 Jul;51(7):915-931. doi: 10.1111/cea.13954. Epub 2021 Jun 26.

name: Regeneron Pharmaceuticals, Inc Tarrytown NY USA

family: Hamilton

full_name: Jennifer D. Hamilton

given: Jennifer D.

family: Harel

full_name: Sivan Harel

given: Sivan

family: Swanson

full_name: Brian N. Swanson

given: Brian N.

family: Brian

full_name: William Brian

given: William

ReferenceCitation: Boguniewicz M, Beck LA, Sher L, Guttman-Yassky E, Thaçi D, Blauvelt A, Worm M, Corren J, Soong W, Lio P, Rossi AB, Lu Y, Chao J, Eckert L, Gadkari A, Hultsch T, Ruddy M, Mannent LP, Graham NMH, Pirozzi G, Chen Z, Ardeleanu M. Dupilumab Improves Asthma and Sinonasal Outcomes in Adults with Moderate to Severe Atopic Dermatitis. J Allergy Clin Immunol Pract. 2021 Mar;9(3):1212-1223.e6. doi: 10.1016/j.jaip.2020.12.059. Epub 2021 Jan 13.

journal_name: The Journal of Allergy and Clinical Immunology: In Practice

ReferenceCitation: Silverberg JI, Simpson EL, Guttman-Yassky E, Cork MJ, de Bruin-Weller M, Yosipovitch G, Eckert L, Chen Z, Ardeleanu M, Shumel B, Hultsch T, Rossi AB, Hamilton JD, Orengo JM, Ruddy M, Graham NMH, Pirozzi G, Gadkari A. Dupilumab Significantly Modulates Pain and Discomfort in Patients With Atopic Dermatitis: A Post Hoc Analysis of 5 Randomized Clinical Trials. Dermatitis. 2021 Oct 1;32(1S):S81-S91. doi: 10.1097/DER.0000000000000698.

publisher: Ovid Technologies (Wolters Kluwer Health)

ReferenceCitation: Alexis AF, Rendon M, Silverberg JI, Pariser DM, Lockshin B, Griffiths CE, Weisman J, Wollenberg A, Chen Z, Davis JD, Li M, Eckert L, Gadkari A, Shumel B, Rossi AB, Graham NM, Ardeleanu M. Efficacy of Dupilumab in Different Racial Subgroups of Adults With Moderate-to-Severe Atopic Dermatitis in Three Randomized, Placebo-Controlled Phase 3 Trials. J Drugs Dermatol. 2019 Aug 1;18(8):804-813.

ReferenceCitation: Wollenberg A, Beck LA, Blauvelt A, Simpson EL, Chen Z, Chen Q, Shumel B, Khokhar FA, Hultsch T, Rizova E, Rossi AB, Graham NMH, Pirozzi G, Lu Y, Ardeleanu M. Laboratory safety of dupilumab in moderate-to-severe atopic dermatitis: results from three phase III trials (LIBERTY AD SOLO 1, LIBERTY AD SOLO 2, LIBERTY AD CHRONOS). Br J Dermatol. 2020 May;182(5):1120-1135. doi: 10.1111/bjd.18434. Epub 2019 Dec 1.

name: Department of Dermatology and Allergology Ludwig Maximilian University of Munich Frauenlobstraße 9–11 80337 Munich Germany

name: Department of Dermatology University of Rochester Medical Center Rochester NY U.S.A

name: Oregon Medical Research Center Portland OR U.S.A

title: Laboratory safety of dupilumab in moderate‐to‐severe atopic dermatitis: results from three phase III trials (LIBERTY AD SOLO 1, LIBERTY AD SOLO 2, LIBERTY AD CHRONOS)

ReferenceCitation: Silverberg JI, Simpson EL, Ardeleanu M, Thaçi D, Barbarot S, Bagel J, Chen Z, Eckert L, Chao J, Korotzer A, Rizova E, Rossi AB, Lu Y, Graham NMH, Hultsch T, Pirozzi G, Akinlade B. Dupilumab provides important clinical benefits to patients with atopic dermatitis who do not achieve clear or almost clear skin according to the Investigator's Global Assessment: a pooled analysis of data from two phase III trials. Br J Dermatol. 2019 Jul;181(1):80-87. doi: 10.1111/bjd.17791. Epub 2019 Apr 11.

name: Department of Dermatology, Preventive Medicine and Medical Social Sciences Northwestern University Feinberg School of Medicine NMH/Arkes Family Pavilion Suite 1600, 676 N. Saint Clair Chicago IL 60611 U.S.A.

name: Department of Dermatology Oregon Health & Science University Portland OR U.S.A.

name: Regeneron Pharmaceuticals, Inc. Tarrytown NY U.S.A.

name: Service de Dermatologie Centre Hospitalier Universitaire (CHU) de Nantes Nantes France

name: Eczema Treatment Center of Central New Jersey East Windsor NJ U.S.A.

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

ReferenceCitation: Simpson EL. Dupilumab Improves General Health-Related Quality-of-Life in Patients with Moderate-to-Severe Atopic Dermatitis: Pooled Results from Two Randomized, Controlled Phase 3 Clinical Trials. Dermatol Ther (Heidelb). 2017 Jun;7(2):243-248. doi: 10.1007/s13555-017-0181-6. Epub 2017 May 13.

location_country:
Italy
United States
Sweden
Hong Kong
France
Korea, Republic of
Lithuania
Poland
Germany
United Kingdom
Canada

title: A Phase 3 Confirmatory Study Investigating the Efficacy and Safety of Dupilumab Monotherapy Administered to Adult Patients With Moderate-to-Severe Atopic Dermatitis

summary: This is a randomized, double-blind, placebo-controlled, parallel-group study to confirm the efficacy and safety of Dupilumab monotherapy in adults with moderate-to-severe atopic dermatitis (AD).


title: Arimoclomol Prospective Doubleblind, Randomised, Placebo-controlled Study in Patients Diagnosed With NiemannPick Disease Type C

summary:
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC).

The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.

The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including patients aged 6 to <24 months at study enrolment.

ReferenceCitation: Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Harmatz P, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsøe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, Í Dali C. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.

name: SphinCS GmbH, Institute of Clinical Science for LSD  Hochheim Germany

family: Mengel

full_name: Eugen Mengel

given: Eugen

name: Departments of Neurology, Pediatrics and Medical Genetics Mayo Clinic  Rochester MN USA

family: Patterson

full_name: Marc C. Patterson

given: Marc C.

name: Regional Coordination Center for Rare Diseases, Academic Hospital ‘Santa Maria della Misericordia’  Udine Italy

family: Da Riol

full_name: Rosalia M. Da Riol

given: Rosalia M.

name: Pediatric Neurology Department Vall d'Hebron University Hospital  Barcelona Spain

family: Del Toro

full_name: Mireia Del Toro

given: Mireia

name: Division of Metabolism Ospedale Pediatrico Bambino Gesù, IRCCS  Rome Italy

family: Deodato

full_name: Federica Deodato

given: Federica

name: Department of Paediatrics, Division of Endocrinology, Diabetology and Metabolism, and Institute of Clinical Chemistry, Inselspital University Hospital Bern, University of Bern  Bern Switzerland

family: Gautschi

full_name: Matthias Gautschi

given: Matthias

name: Department of Metabolic Medicine Great Ormond Street Hospital, Institute of Child Health, UCL, NIHR Biomedical Research Center  London UK

family: Grunewald

full_name: Stephanie Grunewald

given: Stephanie

name: Centre for Inherited Metabolic Diseases, Copenhagen University Hospital (Rigshospitalet)  Copenhagen Denmark

family: Grønborg

full_name: Sabine Grønborg

given: Sabine

name: Gastroenterology and Hepatology UCSF Benioff Children's Hospital Oakland  Oakland CA USA

family: Harmatz

full_name: Paul Harmatz

given: Paul

name: Department of Pediatric Neurology, Reference Centre for Lysosomal Diseases University Hospital Armand Trousseau  Paris France

family: Héron

full_name: Bénédicte Héron

given: Bénédicte

name: Department of Inborn Errors of Metabolism University of Munich Children's Hospital  Munich Germany

family: Maier

full_name: Esther M. Maier

given: Esther M.

name: Department of Neuropediatrics Centre Hospitalier Universitaire de Montpellier  Montpellier France

family: Roubertie

full_name: Agathe Roubertie

given: Agathe

name: Department of Inherited Metabolic Disorders Birmingham Children's Hospital  Birmingham UK

family: Santra

full_name: Saikat Santra

given: Saikat

name: Department of Paediatrics, Nutrition and Metabolic Diseases The Children's Memorial Institute  Warsaw Poland

family: Tylki‐Szymanska

full_name: Anna Tylki‐Szymanska

given: Anna

name: Biostatistics, Clinical Trials Consulting & Training Limited  Buckingham UK

family: Day

full_name: Simon Day

given: Simon

family: Andreasen

full_name: Anne Katrine Andreasen

given: Anne Katrine

family: Geist

full_name: Marie Aavang Geist

given: Marie Aavang

family: Havnsøe Torp Petersen

full_name: Nikolaj Havnsøe Torp Petersen

given: Nikolaj

family: Ingemann

full_name: Linda Ingemann

given: Linda

family: Hansen

full_name: Thomas Hansen

given: Thomas

family: Blaettler

full_name: Thomas Blaettler

family: Kirkegaard

full_name: Thomas Kirkegaard

family: í Dali

full_name: Christine í Dali

given: Christine

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

title: Efficacy and safety of arimoclomol in Niemann–Pick disease type C: Results from a double‐blind, randomized, placebo‐controlled, multinational phase 2/3 trial of a novel treatment

ReferenceCitation: Patterson MC, Lloyd-Price L, Guldberg C, Doll H, Burbridge C, Chladek M, íDali C, Mengel E, Symonds T. Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale. Orphanet J Rare Dis. 2021 Feb 12;16(1):79. doi: 10.1186/s13023-021-01719-2.

location_country:
Italy
Switzerland
Germany
France
Poland
Spain
Denmark
United States
United Kingdom

location_facility: University Hospital Copenhagen (Rigshospitalet)

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udin

location_facility: Dr. von Haunersches Kinderspital der Universität München

location_facility: Villa Metabolica, Universitätsmedizin Mainz


title: A Phase Ib Dose Escalation Study of BCL201 in Combination With Idelalisib in Patients With Follicular Lymphoma (FL) and Mantle Cell Lymphoma (MCL)

summary:
This is a phase Ib multi-center, open-label study: escalation part followed by expansion part. The primary purpose of the Phase Ib CBCL201X2102C study is to characterize the safety and tolerability of BCL201 combined with idelalisib in patients with FL and MCL.

Approximately 65 patients are to be enrolled.

The primary endpoint for the Phase Ib is frequency, severity and seriousness of AEs, lab abnormalities and other safety parameters such as ECG changes. An adaptive Bayesian logistic regression model (BLRM) will guide the dose escalation to determine the MTD/RDE in phase Ib. In addition Bayesian regression models will be used to estimate the dose-exposure relationships for both BCL201 and idelalisib in order to guide the escalation steps. A Bayesian method for the expansion part will be used for the primary activity objective.

The study data will be analyzed and reported based on all patients' data of the escalation and expansion part.

design_allocation: Non-Randomized

lead_sponsor: Novartis Pharmaceuticals

location_country:
Austria
United States
Germany
France


title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Multi-Center Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of GS-5806 in Hematopoietic Cell Transplant (HCT) Recipients With Respiratory Syncytial Virus (RSV) Infection of the Lower Respiratory Tract

summary: The primary objective of this study is to evaluate the effect of presatovir on respiratory syncytial virus (RSV) viral load in autologous or allogeneic hematopoietic cell transplant (HCT) recipients with an acute RSV lower respiratory tract infection (LRTI).

ReferenceCitation: Marty FM, Chemaly RF, Mullane KM, Lee DG, Hirsch HH, Small CB, Bergeron A, Shoham S, Ljungman P, Waghmare A, Blanchard E, Kim YJ, McKevitt M, Porter DP, Jordan R, Guo Y, German P, Boeckh M, Watkins TR, Chien JW, Dadwal SS. A Phase 2b, Randomized, Double-blind, Placebo-Controlled Multicenter Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of Presatovir in Hematopoietic Cell Transplant Recipients with Respiratory Syncytial Virus Infection of the Lower Respiratory Tract. Clin Infect Dis. 2020 Dec 31;71(11):2787-2795. doi: 10.1093/cid/ciz1167.

name: Division of Infectious Disease, Dana-Farber Cancer Institute, Boston, Massachusetts, USA

family: Marty

full_name: Francisco M Marty

given: Francisco M

name: Department of Infectious Diseases, Infection Control and Employee Health, University of Texas MD Anderson Cancer Center, Houston, Texas, USA

family: Chemaly

full_name: Roy F Chemaly

given: Roy F

name: Section of Infectious Diseases, University of Chicago Medical Center, Chicago, Illinois, USA

family: Mullane

full_name: Kathleen M Mullane

given: Kathleen M

name: Department of Infectious Disease, College of Medicine, The Catholic University of Korea, Seoul, South Korea

family: Lee

full_name: Dong-Gun Lee

given: Dong-Gun

name: Department of Biomedicine, University Hospital Basel, Basel, Switzerland

family: Hirsch

full_name: Hans H Hirsch

given: Hans H

name: Department of Medicine/Division of Infectious Diseases, Weill Cornell Medicine, New York, New York, USA

family: Small

full_name: Catherine B Small

given: Catherine B

name: Service de pneumologie, Univ Paris Diderot, Hôpital Saint Louis, Paris, France

family: Bergeron

full_name: Anne Bergeron

given: Anne

name: Department of Medicine, Division of Infectious Diseases, The Johns Hopkins Hospital, Baltimore, Maryland, USA

family: Shoham

full_name: Shmuel Shoham

given: Shmuel

name: Department of Cellular Therapy and Allogeneic Stem Cell Transplantation, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden

family: Ljungman

full_name: Per Ljungman

given: Per

name: Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA

name: Department of Allergy and Infectious Diseases, University of Washington School of Medicine, Seattle, Washington, USA

family: Waghmare

full_name: Alpana Waghmare

given: Alpana

name: Department of Respiratory Diseases, CHU de Bordeaux, Bordeaux, France

family: Blanchard

full_name: Elodie Blanchard

given: Elodie

name: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

family: Kim

full_name: Yae-Jean Kim

given: Yae-Jean

name: Gilead Sciences, Inc., Foster City, California, USA

family: McKevitt

full_name: Matt McKevitt

given: Matt

family: Porter

full_name: Danielle P Porter

given: Danielle P

family: Jordan

full_name: Robert Jordan

given: Robert

family: Guo

full_name: Ying Guo

given: Ying

family: German

full_name: Polina German

given: Polina

family: Boeckh

full_name: Michael Boeckh

given: Michael

family: Watkins

full_name: Timothy R Watkins

given: Timothy R

family: Chien

full_name: Jason W Chien

given: Jason W

name: Division of Infectious Disease, City of Hope National Medical Center, Duarte, California, USA

family: Dadwal

full_name: Sanjeet S Dadwal

given: Sanjeet S

journal_name: Clinical Infectious Diseases

location_country:
Sweden
Switzerland
France
United States
Korea, Republic of

location_facility:
University Clinical Basel
New York Weill Cornell Medical Center
Hopital Foch
City of Hope
Dana Farber Cancer Institute
MD Anderson Cancer Center
Seoul Saint Mary's Hospital
Hopital Saint-Louis, APHP
University of Chicago
CHU de Bordeaux
Fred Hutchison Cancer Research Center
Karolinska Institutet
University of Minnesota
Duke University
University of Michigan
John Hopkins
Medical College of Wisconsin


title: A Prospective, Non-comparative, Multi-center Clinical Trial to Evaluate the Safety and Efficacy of the STARflo Glaucoma Implant in Patients With Open Angle Glaucoma

summary: Prospective, Non-comparative, Multi-center clinical trial to evaluate the safety and efficacy of the STARflo Glaucoma Implant in patients with refractory open angle glaucoma.

lead_sponsor: iSTAR Medical

location_country: Switzerland France Bulgaria Belgium Germany

location_facility: Universitatsklinik fur Augenheilkunde Inselspital


ReferenceCitation: Yardley DA, Coleman R, Conte P, Cortes J, Brufsky A, Shtivelband M, Young R, Bengala C, Ali H, Eakel J, Schneeweiss A, de la Cruz-Merino L, Wilks S, O'Shaughnessy J, Glück S, Li H, Miller J, Barton D, Harbeck N; tnAcity investigators. nab-Paclitaxel plus carboplatin or gemcitabine versus gemcitabine plus carboplatin as first-line treatment of patients with triple-negative metastatic breast cancer: results from the tnAcity trial. Ann Oncol. 2018 Aug 1;29(8):1763-1770. doi: 10.1093/annonc/mdy201.

ReferenceCitation: Metts JL, Alazraki AL, Clark D, Amankwah EK, Wasilewski-Masker KJ, George BA, Olson TA, Cash T. Gemcitabine/nab-paclitaxel for pediatric relapsed/refractory sarcomas. Pediatr Blood Cancer. 2018 Sep;65(9):e27246. doi: 10.1002/pbc.27246. Epub 2018 May 17.

ReferenceCitation: Kim S, Signorovitch JE, Yang H, Patterson-Lomba O, Xiang CQ, Ung B, Parisi M, Marshall JL. Comparative Effectiveness of nab-Paclitaxel Plus Gemcitabine vs FOLFIRINOX in Metastatic Pancreatic Cancer: A Retrospective Nationwide Chart Review in the United States. Adv Ther. 2018 Oct;35(10):1564-1577. doi: 10.1007/s12325-018-0784-z. Epub 2018 Sep 12.

ReferenceCitation: Weiss J, Gilbert J, Deal AM, Weissler M, Hilliard C, Chera B, Murphy B, Hackman T, Liao JJ, Grilley Olson J, Hayes DN. Induction chemotherapy with carboplatin, nab-paclitaxel and cetuximab for at least N2b nodal status or surgically unresectable squamous cell carcinoma of the head and neck. Oral Oncol. 2018 Sep;84:46-51. doi: 10.1016/j.oraloncology.2018.06.028. Epub 2018 Jul 19.

ReferenceCitation: Cartwright TH, Parisi M, Espirito JL, Wilson TW, Pelletier C, Patel M, Babiker HM. Clinical Outcomes with First-Line Chemotherapy in a Large Retrospective Study of Patients with Metastatic Pancreatic Cancer Treated in a US Community Oncology Setting. Drugs Real World Outcomes. 2018 Sep;5(3):149-159. doi: 10.1007/s40801-018-0137-x.

ReferenceCitation: Li F, Zhang H, He M, Liao J, Chen N, Li Y, Zhou S, Palmisano M, Yu A, Pai MP, Yuan H, Sun D. Different Nanoformulations Alter the Tissue Distribution of Paclitaxel, Which Aligns with Reported Distinct Efficacy and Safety Profiles. Mol Pharm. 2018 Oct 1;15(10):4505-4516. doi: 10.1021/acs.molpharmaceut.8b00527. Epub 2018 Sep 21.

ReferenceCitation: Langer CJ, Kim ES, Anderson EC, Jotte RM, Modiano M, Haggstrom DE, Socoteanu MP, Smith DA, Dakhil C, Konduri K, Berry T, Ong TJ, Sanford A, Amiri K, Goldman JW, Weiss J; ABOUND.70+ Investigators. nab-Paclitaxel-Based Therapy in Underserved Patient Populations: The ABOUND.70+ Study in Elderly Patients With Advanced NSCLC. Front Oncol. 2018 Jul 24;8:262. doi: 10.3389/fonc.2018.00262. eCollection 2018.

ReferenceCitation: Gajra A, Karim NA, Mulford DA, Villaruz LC, Matrana MR, Ali HY, Santos ES, Berry T, Ong TJ, Sanford A, Amiri K, Spigel DR. nab-Paclitaxel-Based Therapy in Underserved Patient Populations: The ABOUND.PS2 Study in Patients With NSCLC and a Performance Status of 2. Front Oncol. 2018 Jul 24;8:253. doi: 10.3389/fonc.2018.00253. eCollection 2018.

ReferenceCitation: Pelzer U, Wislocka L, Jühling A, Striefler J, Klein F, Roemmler-Zehrer J, Sinn M, Denecke T, Bahra M, Riess H. Safety and efficacy of Nab-paclitaxel plus gemcitabine in patients with advanced pancreatic cancer suffering from cholestatic hyperbilirubinaemia-A retrospective analysis. Eur J Cancer. 2018 Sep;100:85-93. doi: 10.1016/j.ejca.2018.06.001. Epub 2018 Jul 4.

ReferenceCitation: Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcón S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.

ReferenceCitation: Topçul M, Çeti N İL, Özbaş Turan S, Kolusayin Ozar MÖ. In vitro cytotoxic effect of PARP inhibitor alone and in combination with nab-paclitaxel on triple-negative and luminal A breast cancer cells. Oncol Rep. 2018 Jul;40(1):527-535. doi: 10.3892/or.2018.6364. Epub 2018 Apr 12.

ReferenceCitation: Young R, Mainwaring P, Clingan P, Parnis FX, Asghari G, Beale P, Aly A, Botteman M, Romano A, Ferrara S, Margunato-Debay S, Harris M. nab-Paclitaxel plus gemcitabine in metastatic pancreatic adenocarcinoma: Australian subset analyses of the phase III MPACT trial. Asia Pac J Clin Oncol. 2018 Oct;14(5):e325-e331. doi: 10.1111/ajco.12999. Epub 2018 Jun 22.

ReferenceCitation: Neumann CCM, von Hörschelmann E, Reutzel-Selke A, Seidel E, Sauer IM, Pratschke J, Bahra M, Schmuck RB. Tumor-stromal cross-talk modulating the therapeutic response in pancreatic cancer. Hepatobiliary Pancreat Dis Int. 2018 Oct;17(5):461-472. doi: 10.1016/j.hbpd.2018.09.004. Epub 2018 Sep 7.

ReferenceCitation: Veenstra VL, Damhofer H, Waasdorp C, van Rijssen LB, van de Vijver MJ, Dijk F, Wilmink HW, Besselink MG, Busch OR, Chang DK, Bailey PJ, Biankin AV, Kocher HM, Medema JP, Li JS, Jiang R, Pierce DW, van Laarhoven HWM, Bijlsma MF. ADAM12 is a circulating marker for stromal activation in pancreatic cancer and predicts response to chemotherapy. Oncogenesis. 2018 Nov 16;7(11):87. doi: 10.1038/s41389-018-0096-9.

ReferenceCitation: Morgensztern D, Cobo M, Ponce Aix S, Postmus PE, Lewanski CR, Bennouna J, Fischer JR, Juan-Vidal O, Stewart DJ, Fasola G, Ardizzoni A, Bhore R, Wolfsteiner M, Talbot DC, Jin Ong T, Govindan R, On Behalf Of The Abound L Investigators. ABOUND.2L+: A randomized phase 2 study of nanoparticle albumin-bound paclitaxel with or without CC-486 as second-line treatment for advanced nonsquamous non-small cell lung cancer (NSCLC). Cancer. 2018 Dec 15;124(24):4667-4675. doi: 10.1002/cncr.31779. Epub 2018 Nov 1.

ReferenceCitation: Marschner N, Salat C, Söling U, Hansen R, Grebhardt S, Harde J, Nusch A, Potthoff K. Final Effectiveness and Safety Results of NABUCCO: Real-World Data From a Noninterventional, Prospective, Multicenter Study in 697 Patients With Metastatic Breast Cancer Treated With nab-Paclitaxel. Clin Breast Cancer. 2018 Dec;18(6):e1323-e1337. doi: 10.1016/j.clbc.2018.07.010. Epub 2018 Aug 10.

ReferenceCitation: Watson S, de la Fouchardière C, Kim S, Cohen R, Bachet JB, Tournigand C, Ferraz JM, Lefevre M, Colin D, Svrcek M, Meurisse A, Louvet C. Oxaliplatin, 5-Fluorouracil and Nab-paclitaxel as perioperative regimen in patients with resectable gastric adenocarcinoma: A GERCOR phase II study (FOXAGAST). Eur J Cancer. 2019 Jan;107:46-52. doi: 10.1016/j.ejca.2018.11.006. Epub 2018 Dec 7. Erratum in: Eur J Cancer. 2019 Sep;118:190.

ReferenceCitation: Li YF, Zhang C, Zhou S, He M, Zhang H, Chen N, Li F, Luan X, Pai M, Yuan H, Sun D, Li Y. Species difference in paclitaxel disposition correlated with poor pharmacological efficacy translation from mice to humans. Clin Pharmacol. 2018 Nov 8;10:165-174. doi: 10.2147/CPAA.S185449. eCollection 2018.

ReferenceCitation: Hurria A, Soto-Perez-de-Celis E, Blanchard S, Burhenn P, Yeon CH, Yuan Y, Li D, Katheria V, Waisman JR, Luu TH, Somlo G, Noonan AM, Lee T, Sudan N, Chung S, Rotter A, Arsenyan A, Levi A, Choi J, Rubalcava A, Morrison R, Mortimer JE. A Phase II Trial of Older Adults With Metastatic Breast Cancer Receiving nab-Paclitaxel: Melding the Fields of Geriatrics and Oncology. Clin Breast Cancer. 2019 Apr;19(2):89-96. doi: 10.1016/j.clbc.2018.10.002. Epub 2018 Oct 16.

ReferenceCitation: Fernández A, Salgado M, García A, Buxò E, Vera R, Adeva J, Jiménez-Fonseca P, Quintero G, Llorca C, Cañabate M, López LJ, Muñoz A, Ramírez P, González P, López C, Reboredo M, Gallardo E, Sanchez-Cánovas M, Gallego J, Guillén C, Ruiz-Miravet N, Navarro-Pérez V, De la Cámara J, Alés-Díaz I, Pazo-Cid RA, Carmona-Bayonas A. Prognostic factors for survival with nab-paclitaxel plus gemcitabine in metastatic pancreatic cancer in real-life practice: the ANICE-PaC study. BMC Cancer. 2018 Nov 29;18(1):1185. doi: 10.1186/s12885-018-5101-3.

ReferenceCitation: Awasthi N, Schwarz MA, Zhang C, Schwarz RE. Augmentation of Nab-Paclitaxel Chemotherapy Response by Mechanistically Diverse Antiangiogenic Agents in Preclinical Gastric Cancer Models. Mol Cancer Ther. 2018 Nov;17(11):2353-2364. doi: 10.1158/1535-7163.MCT-18-0489. Epub 2018 Aug 30.

ReferenceCitation: Cristea MC, Frankel P, Synold T, Rivkin S, Lim D, Chung V, Chao J, Wakabayashi M, Paz B, Han E, Lin P, Leong L, Hakim A, Carroll M, Prakash N, Dellinger T, Park M, Morgan RJ. A phase I trial of intraperitoneal nab-paclitaxel in the treatment of advanced malignancies primarily confined to the peritoneal cavity. Cancer Chemother Pharmacol. 2019 Mar;83(3):589-598. doi: 10.1007/s00280-019-03767-9. Epub 2019 Jan 8.

ReferenceCitation: Hegewisch-Becker S, Aldaoud A, Wolf T, Krammer-Steiner B, Linde H, Scheiner-Sparna R, Hamm D, Jänicke M, Marschner N; TPK-Group (Tumour Registry Pancreatic Cancer). Results from the prospective German TPK clinical cohort study: Treatment algorithms and survival of 1,174 patients with locally advanced, inoperable, or metastatic pancreatic ductal adenocarcinoma. Int J Cancer. 2019 Mar 1;144(5):981-990. doi: 10.1002/ijc.31751. Epub 2018 Oct 3.

ReferenceCitation: Wang Z, Huang C, Yang JJ, Song Y, Cheng Y, Chen GY, Yan HH, Ben XS, Wang BC, Xu CR, Jiang BY, Zhou Q, Chen HJ, Wu YL. A randomised phase II clinical trial of nab-paclitaxel and carboplatin compared with gemcitabine and carboplatin as first-line therapy in advanced squamous cell lung carcinoma (C-TONG1002). Eur J Cancer. 2019 Mar;109:183-191. doi: 10.1016/j.ejca.2019.01.007. Epub 2019 Feb 7.

ReferenceCitation: Barnes JA, Ellis ML, Hwang S, Emarine J, Merwin P, Salinas GD, Musher BL. Identification of Educational Gaps Among Oncologists Who Manage Patients with Pancreatic Cancer. J Gastrointest Cancer. 2019 Mar;50(1):84-90. doi: 10.1007/s12029-017-0033-8.

ReferenceCitation: Woo W, Carey ET, Choi M. Spotlight on liposomal irinotecan for metastatic pancreatic cancer: patient selection and perspectives. Onco Targets Ther. 2019 Feb 21;12:1455-1463. doi: 10.2147/OTT.S167590. eCollection 2019. Review.

ReferenceCitation: Seiwert TY, Foster CC, Blair EA, Karrison TG, Agrawal N, Melotek JM, Portugal L, Brisson RJ, Dekker A, Kochanny S, Gooi Z, Lingen MW, Villaflor VM, Ginat DT, Haraf DJ, Vokes EE. OPTIMA: a phase II dose and volume de-escalation trial for human papillomavirus-positive oropharyngeal cancer. Ann Oncol. 2019 Feb 1;30(2):297-302. doi: 10.1093/annonc/mdy522. Erratum in: Ann Oncol. 2019 Oct 1;30(10):1673.

ReferenceCitation: De Luca R, Profita G, Cicero G. Nab-paclitaxel in pretreated metastatic breast cancer: evaluation of activity, safety, and quality of life. Onco Targets Ther. 2019 Feb 26;12:1621-1627. doi: 10.2147/OTT.S191519. eCollection 2019.

ReferenceCitation: Morgensztern D, Ko A, O'Brien M, Ong TJ, Waqar SN, Socinski MA, Postmus PE, Bhore R. Association between depth of response and survival in patients with advanced-stage non-small cell lung cancer treated with first-line chemotherapy. Cancer. 2019 Jul 15;125(14):2394-2399. doi: 10.1002/cncr.32114. Epub 2019 Apr 1.

ReferenceCitation: Li F, Yuan H, Zhang H, He M, Liao J, Chen N, Li Y, Zhou S, Palmisano M, Yu A, Pai M, Sun D. Neonatal Fc Receptor (FcRn) Enhances Tissue Distribution and Prevents Excretion of nab-Paclitaxel. Mol Pharm. 2019 Jun 3;16(6):2385-2393. doi: 10.1021/acs.molpharmaceut.8b01314. Epub 2019 May 1.

ReferenceCitation: Sonbol MB, Ahn DH, Goldstein D, Okusaka T, Tabernero J, Macarulla T, Reni M, Li CP, O'Neil B, Van Cutsem E, Bekaii-Saab T. CanStem111P trial: a Phase III study of napabucasin plus nab-paclitaxel with gemcitabine. Future Oncol. 2019 Apr;15(12):1295-1302. doi: 10.2217/fon-2018-0903. Epub 2019 Feb 15.

ReferenceCitation: Morgensztern D, Dols MC, Ponce Aix S, Postmus PE, Bennouna J, Fischer JR, Juan-Vidal O, Stewart DJ, Ardizzoni A, Bhore R, Wolfsteiner M, Reck M, Talbot D, Govindan R, Ong TJ. nab-Paclitaxel Plus Durvalumab in Patients With Previously Treated Advanced Stage Non-small Cell Lung Cancer (ABOUND.2L+). Front Oncol. 2021 Feb 11;10:569715. doi: 10.3389/fonc.2020.569715. eCollection 2020.

family: Morgensztern

full_name: Daniel Morgensztern

given: Daniel

family: Dols

full_name: Manuel Cobo Dols

given: Manuel Cobo

family: Ponce Aix

full_name: Santiago Ponce Aix

given: Santiago

family: Postmus

full_name: Pieter E. Postmus

given: Pieter E.

family: Bennouna

full_name: Jaafar Bennouna

given: Jaafar

family: Fischer

full_name: Jürgen R. Fischer

given: Jürgen R.

family: Juan-Vidal

full_name: Oscar Juan-Vidal

given: Oscar

family: Stewart

full_name: David J. Stewart

given: David J.

family: Ardizzoni

full_name: Andrea Ardizzoni

given: Andrea

family: Bhore

full_name: Rafia Bhore

given: Rafia

family: Wolfsteiner

full_name: Marianne Wolfsteiner

given: Marianne

family: Reck

full_name: Martin Reck

given: Martin

family: Talbot

full_name: Denis Talbot

given: Denis

family: Govindan

full_name: Ramaswamy Govindan

given: Ramaswamy

family: Ong

full_name: Teng Jin Ong

given: Teng Jin

currency_apc_doaj: USD

journal_name: Frontiers in Oncology

location_country:
Italy
United States
France
Spain
Germany
United Kingdom
Canada

location_facility: Hospital Universitario Germans Trias i Pujol

location_facility: Azienda Ospedaliero Universitaria Di Bologna - Policlinico S.Orsola Malpighi

location_facility: Hospital of Central Connecticut Gynecologic Oncology

location_facility: Hospital General Universitario de Valencia

location_facility: Centre Rene Gauducheau Centre de Lutte Contre Le Cancer Nantes Atlantique

location_facility: John Theurer Cancer Center at Hackensack University Medical Center

location_facility: Azienda Ospedaliero Universitaria di Parma

location_facility: Hospital Universitari i Politecnic La Fe de Valencia

location_facility: University Hospital of South Manchester NHS Foundation Trust -Wythenshawe Hospital - North West Lung

location_facility: Royal Victoria Hospital McGill Department of Oncology(RVH)

location_facility: Penn State Milton S Hershey Medical Center

location_facility: Azienda Ospedaliero-Universitaria Santa Maria della Misericordia die Udine

location_facility: Weill Cornell Medical College - New York - Presbyterian Hospital

title: A Phase 2, Open-Label, Multi-Center Study to Assess Safety and Efficacy of Second/Third-Line Treatment With NAB®-Paclitaxel (ABI-007) In Combination With Epigenetic Modifying Therapy Of CC-486, Or Immunotherapy of Durvalumab (MEDI4736), Or As Monotherapy In Subjects With Advanced Non-Small Cell Lung Cancer (NSCLC): Abound.2L+

summary: This is a Phase 2, open-label, multicenter study to assess the efficacy and safety of second/third-line treatment with nab-paclitaxel in combination with the epigenetic modifying therapy of CC-486 or immunotherapy of durvalumab, and nab-paclitaxel monotherapy in subjects with advanced non-small cell lung cancer (NSCLC).


title: An Open-Label, Extension Study of the Effects of LMTM in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD)

summary: The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.

lead_sponsor: TauRx Therapeutics Ltd

location_country:
United States
Netherlands
Germany
Finland
Croatia
France
Malaysia
Romania
Singapore
Belgium
Australia
Russian Federation
Spain
Canada
United Kingdom
Korea, Republic of
Taiwan

location_facility: Ruan Neurology Clinic and Research Center

location_facility: State Budgetary Healthcare Institution of Sverdlovsk region "Sverdlovsk Regional Clinical Psychiatric Hospital"

location_facility: Wessex Neurological Centre, Southampton General Hospital

location_facility: True North Clinical Research Halifax Inc

location_facility: Nuffield Department of Clinical Neurosciences

location_facility: University Hospitals Case Medical Center, Neurology Clinical Trials Unit

location_facility: Discipline of Psychiatry, University of Queensland

location_facility: Kingshill Research Centre, Victoria Hospital

location_facility: Neurodegenerative Disorders Research Pty Ltd

location_facility: Neurological Associates of Albany, P. C.

location_facility: Royal Adelaide Hospital Memory Trials Centre

location_facility: St Margaret's Hospital Mental Health Unit

location_facility: Seoul National University Boramae Medical Center

location_facility: Hospital of the University of Pennsylvania, Department of Neurology

location_facility: Psychiatry Hospital "Dr. Gheorghe Preda", Department of Psychiatry III

location_facility: Charité, University Medicine Berlin, CBF, Neurology

location_facility: McGill Centre for Studies in Aging, Alzheimer Disease Research Unit

location_facility: University Hospital Ghent Department of Neurology

location_facility: Institute for Neurodegenerative Disorders

location_facility: Saint Petersburg Psychoneurological Research Institute n. a. V.M. Bekhterev

location_facility: Medical and Cognitive Research Unit, Austin Health Heidelberg Repatriation Hospital

location_facility: Xenoscience, Inc / 21st Century Neurology

location_facility: Alexian Brothers Neurosciences Institute

location_facility: Belfast Health and Social Care Trust (BHSCT)

location_facility: University Kuala Lumpur Royal College of Medicine

location_facility: Non-governmental Healthcare Institution "Central Clinical Hospital #6 of the JSC "Russian Railways"

location_facility: RICE - The Research Institute for the Care of Older People

location_facility: Memory Assessment and Research Centre (MARC)

location_facility: Imperial College Healthcare NHS Trust - Charing Cross Hospital

location_facility: Leonard Wolfson Experimental Neurology Centre

location_facility: Advanced Memory Research Institute of NJ PC

location_facility: Mental Health Research Center of the Russian Academy of Medical Sciences, Gerontopsychiatry department

location_facility: Neurological Research Center - Hattiesburg Clinic

location_facility: Clinical Neuroscience Solutions CNS Healthcare

location_facility: Sussex Partnership NHS Foundation Trust, Cognitive Treatment and Research Unit

location_facility: University Hospital Centre Zagreb, Department of Neurology

location_facility: City Clinical Hospital #34, City Scientific Practical Neurological Center

location_facility: Geriatric Clinical Trials Group, St. Joseph's Health Care, Parkwood Hospital Site

location_facility: True North Clinical Research Kentville Inc

location_facility: McCusker Alzheimer's Research Foundation Inc

location_facility: Research Foundation for Mental Hygiene, Inc.

location_facility: University of Eastern Finland, Brain Research Unit Mediteknia

location_facility: University of British Columbia Hospital, Clinic for Alzheimer Disease and Related Disorders

location_facility: Mayo Clinic, Alzheimer's Disease Research Center

location_facility: Memory Enhancement Center of America, Inc

location_facility: UNC Department of Neurology, Physicians Office Building

location_facility: City Geriatric Medical and Social Center

location_facility: Division of Rehabilitation and Aged Care

location_facility: University of Virginia Adult Neurology, Primary Care Center


title: Phase IIb Study to Assess the Efficacy, Safety and Tolerability of Two Dosing Regimens of AllerT in Comparison to Placebo in Adult Subjects Allergic to Birch Pollen

summary: The main objective of the trial is to demonstrate the efficacy of a two months pre-seasonal treatment with AllerT 100 µg maintenance dose in reducing symptoms of allergic rhinoconjunctivitis during the following birch pollen season

lead_sponsor: Anergis

location_country: Sweden Switzerland Poland France Lithuania Denmark Latvia

location_facility: Paul Stradins Clinical University Hospital - Pulmonology Allergology

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny - Allergology

location_facility: Viktorija Vevere private practice of Allergology

location_facility: Silesian Piasts University of Medicine in Wrocław

location_facility: Center of examination and treatment of allergic diseases

location_facility: Antakalnio affiliation of the Vilnius City Allergy Center

location_facility: Centre Hospitalier Universitaire Vaudois


title: An Open-label Extension of Study AC-066A301 Investigating the Safety and Tolerability of ACT-385781A in Patients With Pulmonary Arterial Hypertension (PAH)

summary: This is an open-label, nonrandomized extension to study AC-066A301. The study will assess safety and tolerability of ACT-385781A while providing a means for continuing treatment after ending participation in study AC-066A301.

lead_sponsor: Actelion

location_country: Italy Netherlands France Belgium Spain Canada

location_facility: Sir Mortimer B Davis Jewish General Hospital


location_facility: University Health Network, Toronto TGH - 10 EN - 220

title: A Multicenter, Single-arm, Open-label, Phase 3b Study to Assess the Effects of Switching From Flolan® to EFI/ACT-385781A in Patients With Pulmonary Arterial Hypertension

summary: This study is investigating the effect of switching from Flolan® to Epoprostenol for Injection (EFI/ACT-385781A) in pulmonary arterial hypertension patients currently treated with Flolan®. For this purpose patients being treated for at least 12 months with Flolan® will be switched from Flolan® to EFI/ACT-385781A and followed-up for 90 days. During these 90 days safety and tolerability of EFI/ACT-385781A will closely be monitored in all treated patients. This 90 follow-up will provide clinical evidence on the safety, tolerability, efficacy and treatment satisfaction of switching from Flolan® to EFI/ACT-385781A in patients with pulmonary arterial hypertension.


title: An Open-label, Phase I/IIa, Dose Escalating Study of 2B3-101 in Patients With Solid Tumors and Brain Metastases or Recurrent Malignant Glioma.

summary: The purpose of this study is to determine the safety, tolerability, and pharmacokinetics (PK) of 2B3-101 both as single agent and in combination with trastuzumab. Furthermore, the study will explore the preliminary antitumor activity of 2B3-101 as single agent in patients with with solid tumors and brain metastases or recurrent malignant glioma as well as in patients with various forms of breast cancer with and in combination with trastuzumab in HER2+ breast cancer patients with brain metastases.

lead_sponsor: BBB-Therapeutics B.V.

location_country:
United States
Belgium
France
Netherlands

location_facility: Vrije Universiteit medisch centrum (Vumc)

location_facility: Leids Universitair Medisch Centrum (LUMC)

location_facility: UNC Lineberger Comprehensive Cancer Center


title: REVOLUTION (WFCC-133): Clinical Workflow Study for the Evaluation of the Multi-Electrode Pulmonary Vein Isolation System for the Treatment of Paroxysmal Atrial Fibrillation (PAF)

acronym: REVOLUTION

summary: The purpose of this study is to assess the safety and effectiveness of the Circular and Crescent Mapping and Ablation catheters and the workflow of the Multi-Electrode Irrigated Pulmonary Vein Isolation System when used for the treatment of drug refractory symptomatic paroxysmal atrial fibrillation (PAF).

lead_sponsor: Biosense Webster, Inc.

location_country: Italy France Czechia Belgium Denmark Germany

location_facility: Institute for Clinical and Experimental Medicine (IKEM)


ReferenceCitation: Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.

family: Ratjen

full_name: Felix Ratjen

given: Felix

family: Moeller

full_name: Alexander Moeller

given: Alexander

family: McKinney

full_name: Martha L. McKinney

given: Martha L.

family: Asherova

full_name: Irina Asherova

given: Irina

family: Alon

full_name: Nipa Alon

given: Nipa

family: Maykut

full_name: Robert Maykut

given: Robert

family: Angyalosi

full_name: Gerhild Angyalosi

given: Gerhild

family: Bede

full_name: Olga Bede

given: Olga

family: Bolbas

full_name: Katalin Bolbas

given: Katalin

family: Bulatov

full_name: Vladimir Bulatov

given: Vladimir

family: Chertok

full_name: Elena Chertok

given: Elena

family: Colombo

full_name: Carla Colombo

given: Carla

family: Fayon

full_name: Michael Fayon

given: Michael

family: Gonczi

full_name: Ferenc Gonczi

given: Ferenc

family: Gouda

full_name: Essam Gouda

given: Essam

family: Hammermann

full_name: Jutta Hammermann

given: Jutta

family: Munck

full_name: Anne Munck

given: Anne

family: Solomon

full_name: Melinda Solomon

given: Melinda

family: Tsanakas

full_name: John Tsanakas

given: John

journal_name: Journal of Cystic Fibrosis

oa_host_type: publisher

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

location_country:
Italy
Switzerland
France
Greece
Romania
Hungary
Russian Federation
Egypt
Germany
Canada

acronym: EARLY

title: A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years

summary: This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.


title: International, Multicenter, Prospective, Randomized, Parallel Group, Open Label Protocol to Evaluate Safety and Efficacy of Isolated Pancreas Transplantation Compared to Intensive Insulin Therapy in Type 1 Diabetic Patients With Overt Diabetic Nephropathy and Mildly Reduced Renal Function

acronym: PANCREAS

summary: Current medical therapies are not able to prevent progression of established macroproteinuira (i.e. diabetic nephropathy) to end-stage renal failure in type 1 (insulin dependent) diabetic patients. In this setting, proteinuria is a major risk factor for mortality. Pancreas transplantation, on the contrary, can revert diabetic nephropathy and thereby prevent end-stage chronic renal failure, with theoretically lower risk of death as compared to current medical therapies.The main objective of this study is to assess superiority of isolated pancreas transplantation versus intensive exogenous insulin therapy in type 1 diabetic patients with overt diabetic nephropathy and mildly reduced renal function. The primary endpoint is a composite efficacy/failure end-point including: patient mortality and renal function impairment during 5 years in patients with badly controlled diabetes and nephropathy resisting to up-to-date nephroprotective therapies.Main secondary objectives are safety and efficacy of both regimens, including proteinuria and renal histology evaluation, metabolic control and quality of life, acute and chronic extrarenal complications of diabetes, pancreas survival and all risks related to the transplant procedure (anaesthesia, surgery and immunosuppression side-effects) and to the intensive insulin therapy management.

lead_sponsor: Nantes University Hospital

location_country:
Italy
Brazil
France
United States
Czech Republic

location_facility: Diabetes Center - Institute for Clinical and Experimental Medicine

location_facility: Centre Hospitalier et Universitaire de Nantes

location_facility: Hôpital Edouard-Herriot - Hospices Civils de Lyon

location_facility: Istituto Scientifico Ospedale San Raffaele

intervention_type: Procedure

lead_sponsor_type: academique


title: The Cardiovascular Morbidity in Children With Chronic Renal Failure Study

summary:
Children and adolescents with chronic kidney disease (CKD) are at high risk for cardiovascular (CV) morbidity and mortality. Recent studies suggest that pediatric patients with even moderately impaired kidney function may be afflicted with significant early cardiac and vascular abnormalities.

The pathogenesis and the natural course of CV comorbidity in pediatric CKD patients is still elusive. In this multicenter, prospective, observational study the prevalence, degree and progression of CV comorbidity in children will be characterized and related to CKD progression. The morphology and function of the heart and vessels will be monitored by sensitive, non-invasive methods and will be compared with aged matched healthy controls. Multiple potential clinical, anthropometric, biochemical, and pharmacological risk factors will be monitored prospectively and will be related to CV status. Genotyping might identify predisposing genetic factors for progression of CV comorbidity and underlying nephropathies.

time_perspective: Prospective

lead_sponsor: Heidelberg University

location_country:
Italy
Austria
Sweden
Serbia
Switzerland
United Kingdom
France
Poland
Lithuania
Hungary
Belgium
Portugal
Turkey
Germany
Czech Republic

location_facility: Center for Pediatrics and Adolescent Medicine

location_facility: Goztepe Educational and Research Hospital

location_facility: Haseki Educational and Research Hospital


title: A 2-Part Trial: a Randomized 6-day Repeat-dose, Parallel-group Study in Subjects With T2DM to Assess the Safety and Tolerability of GSK1614235 Compared to Placebo and Sitagliptin; and a Randomized Single-dose, Food Effect Study in Healthy Volunteers to Assess Safety and Tolerability of GSK1614235

summary:
The purpose of Part A of this study is to test whether repeated doses of the study drug (GSK1614235) are safe and well tolerated (i.e. do not produce unacceptable side effects) and whether we can obtain some preliminary information as to whether it works in lowering blood glucose levels. We will do this by comparing the effect of the study drug with placebo (no drug present) and against a drug (sitagliptin) known to control blood glucose in the treatment of diabetes.

The purpose of Part B of this study is to determine the how the timing of dosing, relative to meals, affects the response to study drug.

ReferenceCitation: Dobbins RL, Greenway FL, Chen L, Liu Y, Breed SL, Andrews SM, Wald JA, Walker A, Smith CD. Selective sodium-dependent glucose transporter 1 inhibitors block glucose absorption and impair glucose-dependent insulinotropic peptide release. Am J Physiol Gastrointest Liver Physiol. 2015 Jun 1;308(11):G946-54. doi: 10.1152/ajpgi.00286.2014. Epub 2015 Mar 12.

name: GlaxoSmithKline, Enteroendocrine Unit, Research Triangle Park, North Carolina; and

family: Dobbins

full_name: Robert L. Dobbins

given: Robert L.

name: Pennington Biomedical Research Center, Louisiana State University, Baton Rouge, Louisiana

family: Greenway

full_name: Frank L. Greenway

given: Frank L.

family: Chen

full_name: Lihong Chen

given: Lihong

family: Liu

full_name: Yaping Liu

given: Yaping

family: Breed

full_name: Sharon L. Breed

given: Sharon L.

family: Andrews

full_name: Susan M. Andrews

given: Susan M.

family: Wald

full_name: Jeffrey A. Wald

given: Jeffrey A.

family: Walker

full_name: Ann Walker

given: Ann

family: Smith

full_name: Chari D. Smith

given: Chari D.

bso_classification: Biology (fond.)

journal_name: American Journal of Physiology-Gastrointestinal and Liver Physiology

publisher: American Physiological Society

publisher_dissemination: American Physiological Society

publisher_group: American Physiological Society

publisher_normalized: American Physiological Society

lead_sponsor: GlaxoSmithKline

location_country:
Germany
United States
United Kingdom
France


location_country:
United States
Netherlands
France
Spain
Germany
Taiwan

title: A Phase I/II Multi-center, Open Label Study of TKI258 Administered Orally on an Intermittent Schedule in Adult Patients With Advanced or Metastatic Renal Cell Cancer (RCC)

summary: This is a phase I/II open-label study to delineate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of TKI258. The eligible subject population consists of subjects who have been diagnosed with advanced or metastatic renal cell cancer that is refractory to standard therapy or for which no curative standard therapy exists.


title: A Randomised, Controlled and Double-blind Clinical Investigation on the Effectiveness and Safety of a Foam Dressing Biatain Ibu Non-adhesive vs. Biatain Non-adhesive, in Painful Chronic Venous Leg Ulcers

summary: The objective of this investigation is to demonstrate the effectiveness and safety of Biatain Ibu non-adhesive foam dressing compared to Biatain non-adhesive foam dressing.

lead_sponsor: Coloplast A/S

location_country: Denmark Germany France Spain

location_facility: C.S. SERGAS Rosalía de Castro - Enfermero

location_facility: Department for Dermatology, University School of Medicine

location_facility: Fachartzpraxis für Dermatologie, Allergologie, Lasermedizin


title: Therapeutic Impact of Albumin Dialysis With the Molecular Adsorbents Recirculating System (MARS®) in Severely Decompensated Chronic Liver Disease

acronym: RELIEF

summary: The objective of this trial is to evaluate the impact of elimination of albumin bound substances during albumin dialysis (MARS®) on mortality and the clinical time course in patients with a recent severe clinical deterioration of chronic liver disease caused by a precipitating (trigger) event within 4 weeks manifested by jaundice, encephalopathy and/or renal failure.

ReferenceCitation: Stange J, Mitzner S, Ramlow W, Gliesche T, Hickstein H, Schmidt R. A new procedure for the removal of protein bound drugs and toxins. ASAIO J. 1993 Jul-Sep;39(3):M621-5.

ReferenceCitation: Stange J, Ramlow W, Mitzner S, Schmidt R, Klinkmann H. Dialysis against a recycled albumin solution enables the removal of albumin-bound toxins. Artif Organs. 1993 Sep;17(9):809-13.

lead_sponsor: Baxter Healthcare Corporation

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

location_facility: Martin Luther Universität Halle-Wittenberg


title: A Phase I/II Open Label Dose Escalation Study to Investigate the Safety and Tolerability of Acadesine in Patients With B-cell Chronic Lymphocytic Leukemia

summary: The main aim of this study is to test the safety of acadesine in patients with B-CLL and see what effects it has on patients and their leukaemia. The study also aims to examine the way acadesine is processed by the body. The study will look at the effects of acadesine in the body and the concentration of the drug and its main by-product (ZMP) in the blood to determine the dose and the frequency of dosing that is likely to be the most effective.

lead_sponsor: Advancell - Advanced In Vitro Cell Technologies, S.A.

location_country: Belgium France Spain

location_facility: Service des maladies du sang Hôpital HURIEZ, CHRU

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Cliniques universitaires Saint-Luc Haematology Dept

location_facility: Hematologia Clinica, Institut Catala d'Oncologia


title: A Multicenter, Phase III, Randomised Study of Photodynamic Therapy With Metvix Cream 160 mg/g in Comparison With Cryotherapy in Patients With Primary Superficial Basal Cell Carcinoma

summary:
The purpose of this study is to compare the efficacy of Photodynamic Therapy (PDT) with methyl aminolevulinate (MAL) cream to cryotherapy, in treatment of patients with primary superficial basal cell carcinoma.

Secondary objectives are to compare cosmetic outcome and tolerability (adverse events) in these patients, 3 months after treatment. In addition the recurrence rates in the two treatment groups will be compared up to five years after treatment.

lead_sponsor: Galderma R&D

location_country:
Italy
Austria
Sweden
Finland
France
Belgium
United Kingdom

location_facility: Service de Dermatologie, C.H.U Saint Louis


title: An Open-labeled Trial With a Dose-escalation Part and a Parallel Group Design(1) Investigating Zalutumumab, an Anti-EGF Receptor Antibody, in Combination With Chemo-Radiation as First Line Treatment of Patients With Cancer of the Head and Neck (1) The Parallel Group Part Was Cancelled

summary: The purpose of this study is to investigate the safety of zalutumumab in combination with chemotherapy and radiotherapy as treatment of patients with head and neck cancer

lead_sponsor: Genmab

location_country:
Sweden
Netherlands
France
Belgium
United States

location_facility: Hopital Bretonneau Clinique d'Oncologie et Radiothérapie

location_facility: Centre Georges-Francois Leclerc Hospital


title: Profile of Depressive Symptoms in Parkinson's Disease (PRODEST-PD)

summary: To explore or establish the relationship between cognitive, mood and motor symptoms in PD to scores on depression rating scales in a naturalistic setting.

lead_sponsor: Boehringer Ingelheim

location_country:
Italy
Austria
Switzerland
Netherlands
France
Spain
Germany
United Kingdom

location_facility: Universitatsklinikum Carl Gustav Carus Dresden

location_facility: Boehringer Ingelheim Investigational Site


title: Enzymatic Debridement in Burns Patients (Children & Adults): A Comparison to Standard of Care (Protocol MW 2004-11-02)

summary:
Burns represent one of the most severe and dreaded traumas. Burned and traumatized tissue is known as eschar. The dead eschar, if not removed, often becomes heavily contaminated and is the source of local and/or systemic infection or sepsis. The local inflammation and infection destroy healthy surrounding tissues and extends the original damage. In order to prevent these complications, and in order to minimize the risk of infection, it is imperative to evaluate the burn and remove all of the offending eschar at the earliest possible opportunity. This removal of dead tissue is termed "debridement".

The most direct debridement method for eschar removal is surgery. Traditional, conservative non-surgical debridement is a lengthy process which often involves many complications.

The objective of this study is to evaluate the safety and enzymatic debriding efficacy of Debrase Gel Dressing (DGD) in hospitalized patients with deep partial thickness and/or full thickness thermal burns and to compare DGD to standard of care (SOC).

ReferenceCitation: Rosenberg L, Lapid O, Bogdanov-Berezovsky A, Glesinger R, Krieger Y, Silberstein E, Sagi A, Judkins K, Singer AJ. Safety and efficacy of a proteolytic enzyme for enzymatic burn debridement: a preliminary report. Burns. 2004 Dec;30(8):843-50.

ReferenceCitation: Rosenberg L, Krieger Y, Bogdanov-Berezovski A, Silberstein E, Shoham Y, Singer AJ. A novel rapid and selective enzymatic debridement agent for burn wound management: a multi-center RCT. Burns. 2014 May;40(3):466-74. doi: 10.1016/j.burns.2013.08.013. Epub 2013 Sep 26.

family: Rosenberg

full_name: Lior Rosenberg

given: Lior

family: Krieger

full_name: Yuval Krieger

given: Yuval

family: Bogdanov-Berezovski

full_name: Alex Bogdanov-Berezovski

given: Alex

family: Silberstein

full_name: Eldad Silberstein

given: Eldad

family: Shoham

full_name: Yaron Shoham

given: Yaron

family: Singer

full_name: Adam J. Singer

given: Adam J.

journal_name: Burns

title: A novel rapid and selective enzymatic debridement agent for burn wound management: A multi-center RCT

lead_sponsor: MediWound Ltd

location_country:
Italy
Brazil
France
Poland
Israel
Slovakia
Romania
Australia
Germany
United Kingdom

location_facility: Clinic of Burns and Reconstructive Surgery Hospital Kosice

location_facility: Center for Burns & Reconstructive Surgery, University Hopsital Bratislava

location_facility: Pronto Socorro para Queimaduras de Goiania

location_facility: Emergency Clinic Hospital "Bagdazar-Arsenie"

location_facility: Centre Hospitalier Regional et Universitaire de Marseille, Service de Chirurgie Plastique Reparatrice et Esthetique

location_facility: Hospital do Servidor Publico do Estado de Sao Paulo

location_facility: Direttore U.O. Chirurgia Plastica e Centro Ustioni Ospedale Civico


type: Registry Identifier

title: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Efficacy and Safety of TAK-475 or Placebo When Co-administered With Current Lipid-lowering Therapy in Subjects With Homozygous Familial Hypercholesterolemia.

summary: The purpose of the study is to determine the efficacy of lapaquistat acetate, once daily (QD), to lower cholesterol in subjects with homozygous familial hypercholesterolemia undergoing lipid-lowering treatment.

ReferenceCitation: Stein EA, Bays H, O'Brien D, Pedicano J, Piper E, Spezzi A. Lapaquistat acetate: development of a squalene synthase inhibitor for the treatment of hypercholesterolemia. Circulation. 2011 May 10;123(18):1974-85. doi: 10.1161/CIRCULATIONAHA.110.975284. Epub 2011 Apr 25.

name: From the Metabolic and Atherosclerosis Research Center, Cincinnati, OH (E.A.S.); Louisville Metabolic and Atherosclerosis Research Center, Louisville, KY (H.B.); and Takeda Pharmaceuticals North America, Inc., Deerfield, IL (D.O., J.P., E.P., A.S.).

family: Stein

full_name: Evan A. Stein

given: Evan A.

family: Bays

full_name: Harold Bays

given: Harold

family: O'Brien

full_name: Dennis O'Brien

given: Dennis

family: Pedicano

full_name: Jim Pedicano

given: Jim

family: Piper

full_name: Edward Piper

given: Edward

family: Spezzi

full_name: Andrea Spezzi

given: Andrea

journal_name: Circulation

evidence: open (via free pdf)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: Lapaquistat Acetate

lead_sponsor: Takeda

location_country:
France
Poland
Israel
United States
United Kingdom
Canada


title: A Comparison of the Inflammatory Control of Asthma Provided by One Inhalation of Symbicort® Turbuhaler® 160/4.5 µg/Inhalation b.i.d. Plus As-needed Versus One Inhalation of Symbicort® Turbuhaler® 320/9 µg/Inhalation b.i.d. + One Inhalation of Pulmicort® Turbuhaler® 400 µg/Dose b.i.d. Plus Terbutaline Turbuhaler® 0.4 mg/Inhalation As-needed. A 12-month, Randomised, Double-blind, Parallel-group, Active Controlled, Multinational, Phase IIIB Study in Adult Patients With Asthma, EOS

summary: The purpose of this study is to determine if a flexible dose of Symbicort has a similar effect on the airways of patients with asthma as a higher fixed-dose of Symbicort with additional glucocorticosteroids added.

lead_sponsor: AstraZeneca

location_country:
Sweden
France
Spain
Denmark
Germany
United Kingdom


title: A Phase IIIb, Randomized, Open Label Study to Compare the Safety, Efficacy and Tolerability of Anagrelide Hydrochloride Versus Hydroxyurea in High-Risk Essential Thrombocythaemia Patients.

summary: Essential thrombocythaemia is a disorder of bone marrow, which causes too many platelets to be produced. Platelets are small cells carried around in the blood, which help form blood clots. When patients have too many platelets, there is a risk of blood clots forming unnecessarily and excessive bleeding. The aim of this study is to gain additional information on the safety profile of Anagrelide (Xagrid(r)) and Hydroxyurea (also known as hydroxycarbamide).

ReferenceCitation: Birgegård G, Folkvaljon F, Garmo H, Holmberg L, Besses C, Griesshammer M, Gugliotta L, Wu J, Achenbach H, Kiladjian JJ, Harrison CN. Leukemic transformation and second cancers in 3649 patients with high-risk essential thrombocythemia in the EXELS study. Leuk Res. 2018 Nov;74:105-109. doi: 10.1016/j.leukres.2018.10.006. Epub 2018 Oct 11.

family: Birgegård

full_name: Gunnar Birgegård

given: Gunnar

family: Folkvaljon

full_name: Folke Folkvaljon

given: Folke

family: Garmo

full_name: Hans Garmo

given: Hans

family: Holmberg

full_name: Lars Holmberg

given: Lars

family: Besses

full_name: Carlos Besses

given: Carlos

family: Griesshammer

full_name: Martin Griesshammer

given: Martin

family: Gugliotta

full_name: Luigi Gugliotta

given: Luigi

family: Wu

full_name: Jingyang Wu

given: Jingyang

family: Achenbach

full_name: Heinrich Achenbach

given: Heinrich

family: Kiladjian

full_name: Jean-Jacques Kiladjian

given: Jean-Jacques

family: Harrison

full_name: Claire N. Harrison

given: Claire N.

journal_name: Leukemia Research

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: King's College London - Research Portal

repository_normalized: Pure (Denmark)

ReferenceCitation: Gotic M, Egyed M, Gercheva L, Warzocha K, Kvasnicka HM, Achenbach H, Wu J. Cardiovascular Safety of Anagrelide Hydrochloride versus Hydroxyurea in Essential Thrombocythaemia. Cardiovasc Toxicol. 2021 Mar;21(3):236-247. doi: 10.1007/s12012-020-09615-0. Epub 2020 Oct 29.

family: Gotic

full_name: Mirjana Gotic

given: Mirjana

family: Egyed

full_name: Miklos Egyed

given: Miklos

family: Gercheva

full_name: Liana Gercheva

given: Liana

family: Warzocha

full_name: Krzysztof Warzocha

given: Krzysztof

family: Kvasnicka

location_country: Serbia France Bulgaria Poland Hungary Portugal

location_facility: University of Debrecen Medical and Health Science Centre

location_facility: Uniwersyteckie Centrum Kliniczne Katedra i Klinika Hematologii i Transplantologii

location_facility: Institute for Haematology of Clinical Centre of Serbia

location_facility: University Multiprofile Hospital for Active Treatment ''Dr Georgi Stranski'' - Pleven

location_facility: Hopital Saint Louis - Centre d'Investigation Clinique

location_facility: University Multiprofile Hospital for active Treatment ''Alexandrovska'' Clinic of Haematology

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1

location_facility: University Multiprofile Hospital for Active Treament ''Sv. Marina'' - Varna Haematology Clinic

location_facility: CHU Angers Services des Maladies du Sang

location_facility: Katedra i Klinika Onkologii i Chorob Wewnetrznych Akademii Medycznej

location_facility: Klinika Hematologii Instytut Hematologii i Transfuzjologi


title: Non-Randomized, Prospective, Multi-center Evaluation of the ABSOLUTE™ .035 Peripheral Self-Expanding Stent System for Occluded or Stenotic Superficial Femoral or Proximal Popliteal Arteries

summary: The purpose of this study is to investigate the performance of the ABSOLUTE™ .035 peripheral self-expanding stent system in preventing restenosis of occluded or stenotic superficial femoral or proximal popliteal arteries.

lead_sponsor: Abbott Medical Devices

location_country: Italy Austria France Greece Belgium Spain Germany

location_facility: Allgemeines Krankenhaus der Stadt Wien (AKH Wien)

location_facility: Universitäres Herz & Gefässzentrum Hamburg


title: An EORTC Randomized, Double Blind, Placebo-Controlled, Phase II Multi-Center Trial Of Anastrozole (Arimidex) In Combination With ZD 1839 (Iressa) Or Placebo In Patients With Advanced Breast Cancer

summary:
RATIONALE: Estrogen can stimulate the growth of breast cancer cells. Hormone therapy using anastrozole may fight breast cancer by reducing the production of estrogen. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining anastrozole with gefitinib may kill more tumor cells.

PURPOSE: Randomized phase II trial to compare the effectiveness of anastrozole with or without gefitinib in treating postmenopausal women who have metastatic or locally recurrent breast cancer.

ReferenceCitation: Mauriac L, Cameron D, Dirix L: Results of randomized phase II trial combining Iressa (gefitinib) and arimidex in women with advanced breast cancer (ABC): EORTC protocol 10021. [Abstract] 31st Annual San Antonio Breast Cancer Symposium, December 10-14, 2008, San Antonio, Texas. A-6133, 2008.

ReferenceCitation: Tryfonidis K, Basaran G, Bogaerts J, Debled M, Dirix L, Thery JC, Tjan-Heijnen VC, Van den Weyngaert D, Cufer T, Piccart M, Cameron D; EORTC-Breast Cancer Group. A European Organisation for Research and Treatment of Cancer randomized, double-blind, placebo-controlled, multicentre phase II trial of anastrozole in combination with gefitinib or placebo in hormone receptor-positive advanced breast cancer (NCT00066378). Eur J Cancer. 2016 Jan;53:144-54. doi: 10.1016/j.ejca.2015.10.012. Epub 2015 Dec 24.

family: Tryfonidis

full_name: Konstantinos Tryfonidis

given: Konstantinos

family: Basaran

full_name: Gul Basaran

given: Gul

family: Bogaerts

full_name: Jan Bogaerts

given: Jan

family: Debled

full_name: Marc Debled

given: Marc

family: Dirix

full_name: Luc Dirix

given: Luc

family: Thery

full_name: Jean-Christophe Thery

given: Jean-Christophe

family: Tjan-Heijnen

full_name: Vivianne C.G. Tjan-Heijnen

given: Vivianne C.G.

family: Van den Weyngaert

full_name: Danielle Van den Weyngaert

given: Danielle

family: Cufer

full_name: Tanja Cufer

given: Tanja

family: Piccart

full_name: Martine Piccart

given: Martine

family: Cameron

full_name: David Cameron

given: David

journal_name: European Journal of Cancer

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

location_country:
United Kingdom
Netherlands
France
Belgium
Slovenia

location_facility: Edinburgh Cancer Centre at Western General Hospital

location_facility: Universitair Medisch Centrum St. Radboud - Nijmegen

location_facility: Netherlands Cancer Institute - Antoni van Leeuwenhoek Hospital


title: Oral Empirical Therapy of Fever in Low-Risk Neutropenic Cancer Patients: A Prospective, Double-Blind, Randomized, Multicenter Trial Comparing Monotherapy (Single Daily Dose Moxifloxacin) With Combination Therapy (Ciprofloxacin Plus Amoxicillin/Clavulanic Acid)

summary:
RATIONALE: Antibiotics such as amoxicillin, ciprofloxacin, and moxifloxacin may be effective in preventing or controlling fever and neutropenia in patients with cancer. It is not yet known whether moxifloxacin alone is more effective than amoxicillin combined with ciprofloxacin in treating neutropenia and fever.

PURPOSE: This randomized clinical trial is studying how well moxifloxacin works and compares it to ciprofloxacin together with amoxicillin in treating neutropenia and fever in patients with cancer.

primary_purpose: Supportive Care

ReferenceCitation: Kern WV, Marchetti O, Drgona L, Akan H, Aoun M, Akova M, de Bock R, Paesmans M, Viscoli C, Calandra T. Oral antibiotics for fever in low-risk neutropenic patients with cancer: a double-blind, randomized, multicenter trial comparing single daily moxifloxacin with twice daily ciprofloxacin plus amoxicillin/clavulanic acid combination therapy--EORTC infectious diseases group trial XV. J Clin Oncol. 2013 Mar 20;31(9):1149-56. doi: 10.1200/JCO.2012.45.8109. Epub 2013 Jan 28.

name: Winfried V. Kern, Universitätsklinikum Freiburg, Freiburg, Germany; Oscar Marchett, Thierry Calandra, Centre Hospitalier Universitaire Vaudois and University of Lausanne, Lausanne, Switzerland; Lubus Drgona, National Cancer Institute and Comenius University, Bratislava, Slovakia; Hamdi Akan, Cebeci Hospital, Ankara University; Murat Akova, Hacettepe University Hospital, Ankara, Turkey; Marianne Paesmans, Institut Jules Bordet, Brussels; Robrecht de Bock, Algemeen Ziekenhuis Middelheim, Antwerpen,...

family: Kern

full_name: Winfried V. Kern

given: Winfried V.

family: Marchetti

full_name: Oscar Marchetti

given: Oscar

family: Drgona

full_name: Lubos Drgona

given: Lubos

family: Akan

full_name: Hamdi Akan

given: Hamdi

family: Aoun

full_name: Mickel Aoun

given: Mickel

family: Akova

full_name: Murat Akova

given: Murat

family: de Bock

full_name: Robrecht de Bock

given: Robrecht

family: Paesmans

full_name: Marianne Paesmans

given: Marianne

family: Viscoli

full_name: Claudio Viscoli

given: Claudio

family: Calandra

full_name: Thierry Calandra

given: Thierry

journal_name: Journal of Clinical Oncology

publisher: American Society of Clinical Oncology (ASCO)

title: Oral Antibiotics for Fever in Low-Risk Neutropenic Patients With Cancer: A Double-Blind, Randomized, Multicenter Trial Comparing Single Daily Moxifloxacin With Twice Daily Ciprofloxacin Plus Amoxicillin/Clavulanic Acid Combination Therapy—EORTC Infectious Diseases Group Trial XV

location_country: Italy Switzerland France Israel Slovakia Belgium Turkey Germany

location_facility: Medizinische Universitaetsklinik I at the University of Cologne

location_facility: Ruprecht - Karls - Universitaet Heidelberg

location_facility: Hacettepe University - Faculty of Medicine

location_facility: Klinikum der Albert - Ludwigs - Universitaet Freiburg

location_facility: Frauenklinik - Universitaetsklinikum Rostock am Klinikum Sudstadt

location_facility: Istituto Nazionale per la Ricerca sul Cancro

location_facility: Charite - Universitaetsmedizin Berlin - Campus Benjamin Franklin


title: A Study of Two Different Doses of ABT-378/Ritonavir in HIV-Infected Patients Who Have Taken Protease Inhibitors and Non-Nucleoside Reverse Transcriptase Inhibitors

summary: The purpose of this study is to determine the safety and effectiveness of a high dose of ABT-378/ritonavir

lead_sponsor: Abbott

location_country:
United States
France
Spain

location_facility: The University of North Carolina at Chapel Hill


title: Pilot Testing of SAGIT in Patients With Acromegaly in Clinical Practice

acronym: SAGIT Pilot

summary:
The purpose of this pilot study is to test SAGIT (Signs and symptoms - Associated comorbidities - GH concentration level - IGF-1 - Tumour). SAGIT is a Clinician-Reported Outcomes (ClinROs) tool developed to describe patients with acromegaly. This study will determine the potential use of a finalised operational version for patient classification in clinical practice and studies.

In addition, this study intends to carry out a qualitative evaluation of the acceptability of SAGIT by the practicing endocrinologist in terms of relevance, ease of use, applicability and usefulness of the tool in practice.

time_perspective: Cross-Sectional

ReferenceCitation: Giustina A, Bevan JS, Bronstein MD, Casanueva FF, Chanson P, Petersenn S, Thanh XM, Sert C, Houchard A, Guillemin I, Melmed S; SAGIT Investigator Group. SAGIT®: clinician-reported outcome instrument for managing acromegaly in clinical practice--development and results from a pilot study. Pituitary. 2016 Feb;19(1):39-49. doi: 10.1007/s11102-015-0681-2.

family: Giustina

full_name: Andrea Giustina

given: Andrea

family: Bevan

full_name: John S. Bevan

given: John S.

family: Bronstein

full_name: Marcello D. Bronstein

given: Marcello D.

family: Casanueva

full_name: Felipe F. Casanueva

given: Felipe F.

family: Chanson

full_name: Philippe Chanson

given: Philippe

family: Petersenn

full_name: Stephan Petersenn

given: Stephan

family: Thanh

full_name: Xuan-Mai Truong Thanh

given: Xuan-Mai Truong

family: Sert

full_name: Caroline Sert

given: Caroline

family: Houchard

full_name: Aude Houchard

given: Aude

family: Guillemin

full_name: Isabelle Guillemin

given: Isabelle

family: Melmed

full_name: Shlomo Melmed

given: Shlomo

journal_name: Pituitary

evidence: open (via page says license)

repository_institution: PubMed Central - Europe PMC

title: SAGIT®: clinician-reported outcome instrument for managing acromegaly in clinical practice—development and results from a pilot study

lead_sponsor: Ipsen

location_country:
Italy
Brazil
Germany
France
Spain
United States
United Kingdom

location_facility: Azienda Ospedaliera Città della Salute e della Scienza

location_facility: Azienda Ospedaliera Spedali Civil di Brescia

location_facility: NW Pituitary Center and Neurological Surgery

location_facility: Universitary Hospital Virgen de la Vitoria

location_facility: Praxisgemeinschaft an der Kaisereiche, Wilhelm-Hauff-Straße 21


ReferenceCitation: Mussai F, Campana D, Bhojwani D, Stetler-Stevenson M, Steinberg SM, Wayne AS, Pastan I. Cytotoxicity of the anti-CD22 immunotoxin HA22 (CAT-8015) against paediatric acute lymphoblastic leukaemia. Br J Haematol. 2010 Aug;150(3):352-8. doi: 10.1111/j.1365-2141.2010.08251.x. Epub 2010 Jun 7.

lead_sponsor: MedImmune LLC

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

title: A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin

summary: The primary objective of this study is to evaluate the efficacy of moxetumomab pasudotox in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.


title: A Combined Phase IIa / IIb Study of the Efficacy, Safety, and Tolerability of Repeated Topical Doses of Regorafenib Eye Drops, in Treatment-naïve Subjects With Neovascular Age Related Macular Degeneration

acronym: DREAM

summary:
Part A (Phase IIa):

Primary objectives:

The study part A is designed to investigate whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate the safety and tolerability of the regorafenib eye drops.

Part B (Phase IIb):

Primary objectives:

The study part B is designed to investigate:

how often the regorafenib eye drops need to be given per day
whether the use of regorafenib eye drops can help patients with neovascular (wet) Age-Related Macular Degeneration (wAMD) to see better after 4 weeks and 12 weeks after inclusion into this study.

Secondary objectives:

The study will also evaluate how the different dosings of regorafenib eye drops affect patients vision, the safety and the tolerability.

lead_sponsor: Bayer

location_country:
Italy
Austria
United States
Chile
Hong Kong
Switzerland
Germany
France
Japan
Israel
Slovakia
Hungary
Australia
Spain
Canada
Czech Republic
Korea, Republic of
Colombia


title: An Open-label, Multicenter, Phase IIIb Study Assessing the Long-term Efficacy and Safety of AOP2014 and Standard First Line Treatment (BAT) in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study

acronym: CONTI-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 show that the study drug AOP2014 (pegylated proline interferon alpha-2b) has the long term efficacy and safety in controlling the disease. A comparison arm is receiving best available therapy as selected by the investigator. Response to the treatment is measured by several blood parameters as well as size of the spleen.

Interferon-alpha has been shown to be effective in controlling the blood parameters by immunologically influencing the blood building cells. This can lead to a suppression of the disease-causing stem cells and help healthy stem cells to proliferate. Through this mechanism it is possible that Interferon-alpha can avoid long-term damaging effects of the disease.

lead_sponsor: AOP Orphan Pharmaceuticals AG

location_country:
Austria
France
Czechia
Bulgaria
Poland
Slovakia
Romania
Hungary
Russian Federation
Spain
Germany
Ukraine

location_facility: Independent Public Teaching Hospital No.1 in Lublin

location_facility: Saint Cyril and Metod University Hospital Bratislava

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

location_facility: Emergency Clinical County Hospital Brasov

location_facility: Multiprofile Hospital for Active Treatment "Sveta Marina", Varna

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

location_facility: University Hospital Bonn, Center for Internal Medicine, Medical Clinic and Outpatient Clinic III

location_facility: Fryderyk Chopin Provincial Specialized Hospital

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

location_facility: O.F. Herbachevskyi Regional Clinical Hospital

location_facility: St Istvan and St Laszlo Hospital of Budapest

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

location_facility: Institute of Blood Pathology and Transfusion Medicine

location_facility: Dnipropetrovsk City Multispecialty Clinical Hospital #4

location_facility: University Multiprofile Hospital for Active Treatment "Sveti Georgi", Plovdiv

location_facility: Samara Kalinin Regional Clinical Hospital

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

location_facility: Nicolaus Copernicus Municipal Specialist Hospital

location_facility: Aachen University Hospital, Medical Clinic IV

location_facility: Institute of Hematology and Transfusion Medicine

location_facility: University Hospital Carl Gustav Carus, Medical Clinic and Polyclinic I

location_facility: Specialized Hospital for Active Treatment of Hematological Diseases

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