title: Prospective Randomized Study of the Incidence and Outcome of Veno-Occlusive Disease (VOD) With the Prophylactic Use of Defibrotide (DF) in Pediatric Stem Cell Transplantation

acronym: VOD-DF

summary: The aim of this trial is to evaluate whether the prophylactic use of Defibrotide (DF) in pediatric patients (age less than 18 years) undergoing stem cell transplantation and who are at high risk of developing hepatic Veno-occlusive Disease (VOD) will have an impact on the incidence and severity of the disease. Patients will be randomly assigned to one of two treatment arms: Those allocated to the Prophylactic Arm will receive the study drug (Defibrotide) from the day of conditioning onwards. Patients allocated to the Control Arm will receive the study drug (Defibrotide) from the day that VOD is diagnosed.

ReferenceCitation: Corbacioglu S, Cesaro S, Faraci M, Valteau-Couanet D, Gruhn B, Rovelli A, Boelens JJ, Hewitt A, Schrum J, Schulz AS, Müller I, Stein J, Wynn R, Greil J, Sykora KW, Matthes-Martin S, Führer M, O'Meara A, Toporski J, Sedlacek P, Schlegel PG, Ehlert K, Fasth A, Winiarski J, Arvidson J, Mauz-Körholz C, Ozsahin H, Schrauder A, Bader P, Massaro J, D'Agostino R, Hoyle M, Iacobelli M, Debatin KM, Peters C, Dini G. Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial. Lancet. 2012 Apr 7;379(9823):1301-9. doi: 10.1016/S0140-6736(11)61938-7. Epub 2012 Feb 23.

family: Corbacioglu

full_name: Selim Corbacioglu

given: Selim

family: Cesaro

full_name: Simone Cesaro

given: Simone

family: Faraci

full_name: Maura Faraci

given: Maura

family: Valteau-Couanet

full_name: Dominique Valteau-Couanet

given: Dominique

family: Gruhn

full_name: Bernd Gruhn

given: Bernd

family: Rovelli

full_name: Attilio Rovelli

given: Attilio

family: Boelens

full_name: Jaap J Boelens

given: Jaap J

family: Hewitt

full_name: Annette Hewitt

given: Annette

family: Schrum

full_name: Johanna Schrum

given: Johanna

family: Schulz

full_name: Ansgar S Schulz

given: Ansgar S

family: Müller

full_name: Ingo Müller

given: Ingo

family: Stein

full_name: Jerry Stein

given: Jerry

family: Wynn

full_name: Robert Wynn

given: Robert

family: Greil

full_name: Johann Greil

given: Johann

family: Sykora

full_name: Karl-Walter Sykora

given: Karl-Walter

family: Matthes-Martin

full_name: Susanne Matthes-Martin

given: Susanne

family: Führer

full_name: Monika Führer

given: Monika

family: O'Meara

full_name: Anne O'Meara

given: Anne

family: Toporski

full_name: Jacek Toporski

given: Jacek

family: Sedlacek

full_name: Petr Sedlacek

given: Petr

family: Schlegel

full_name: Paul G Schlegel

given: Paul G

family: Ehlert

full_name: Karoline Ehlert

given: Karoline

family: Fasth

full_name: Anders Fasth

given: Anders

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Italy
Austria
Sweden
Switzerland
Netherlands
France
Israel
Ireland
Germany
United Kingdom

location_facility:
Institute G. Gaslini
University Children Hospital
Great Ormond Street Hospital
Klinik Kinder-Onkologie
Children's Hospital
Our Lady's Hospital for Sick Children
Schneider Children's MC
Universitätsspital
Poliklinik Kinderheilkunde
St Anna Kinderspital
Medical School
Ospedale S. Gerardo
Kinderklinik
Hopital Cantonal Universitaire
Johann-Wolfgang Goethe Universität
Christian-Albrecht -University
University Hospital
Inst. Gustave Roussy
Sahlgrenska University Hospital
Clinica di Oncoematologia Pediatrica
Kinderpoliklinik Uni
Universitätsspital Eppendorf
Inselspital


title: A Multi-centre, Randomised, Double-blind, Placebocontrolled, Parallel-group Trial Investigating the Efficacy and Safety of Norditropin® SimpleXx® in Tibia Fractures

summary: This trial is conducted in Africa, Europe and Middle East. This trial investigates the efficacy and safety of three dose levels of Norditropin® (growth hormone) as compared to placebo in the treatment of tibia fractures. The trial will be conducted in two parts: in the first part, the patients will be evaluated with regard to efficacy (fracture healing) and safety at short time intervals until week 24 post-surgery. In the second part, long-term safety and fracture healing up to 12 months post-surgery will be evaluated.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: result

ReferenceCitation: Raschke M, Rasmussen MH, Govender S, Segal D, Suntum M, Christiansen JS. Effects of growth hormone in patients with tibial fracture: a randomised, double-blind, placebo-controlled clinical trial. Eur J Endocrinol. 2007 Mar;156(3):341-51.

ReferenceType: derived

ReferenceCitation: Krusenstjerna-Hafstrøm T, Rasmussen MH, Raschke M, Govender S, Madsen J, Christiansen JS. Biochemical markers of bone turnover in tibia fracture patients randomly assigned to growth hormone (GH) or placebo injections: Implications for detection of GH abuse. Growth Horm IGF Res. 2011 Dec;21(6):331-5. doi: 10.1016/j.ghir.2011.08.003. Epub 2011 Oct 1.

family: Krusenstjerna-Hafstrøm

full_name: Thomas Krusenstjerna-Hafstrøm

given: Thomas

sequence: first

family: Rasmussen

full_name: Michael Højby Rasmussen

given: Michael Højby

sequence: additional

family: Raschke

full_name: Michael Raschke

given: Michael

family: Govender

full_name: Shunmugam Govender

given: Shunmugam

family: Madsen

full_name: Jesper Madsen

given: Jesper

family: Christiansen

full_name: Jens Sandahl Christiansen

given: Jens Sandahl

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Growth Hormone & IGF Research

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Novo Nordisk A/S

location_country:
South Africa
Finland
France
Poland
Israel
Norway
Hungary
Spain
Germany

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: Natural History Study of CEP290-Related Retinal Degeneration

summary: A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related retinal degeneration caused by the common intron 26 mutation.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Editas Medicine, Inc.

location_country:
Germany
United States
France
Netherlands

location_facility: Universitaetsklinikum Giessen and Marburg GmbH

status_simplified: Ongoing


title: Calcipotriol Plus Betamethasone Dipropionate Gel Compared to Betamethasone Dipropionate in the Gel Vehicle, Calcipotriol in the Gel Vehicle, and the Gel Vehicle Alone in Scalp Psoriasis

summary:
The purpose of this study is to evaluate whether once daily treatment for up to 8 weeks of calcipotriol 50 mcg/g plus betamethasone 0.5 mg/g (as dipropionate) gel is safe and more effective than betamethasone 0.5 mg/g (as dipropionate) in the gel vehicle, calcipotriol 50 mcg/g in the gel vehicle or the gel vehicle used alone in patients with scalp psoriasis.

The primary outcome is the proportion of patients with absence of disease or very mild disease after 8 weeks of treatment.

lead_sponsor: LEO Pharma

location_country:
Sweden
Spain
France
Norway
Portugal
Denmark
Canada
United Kingdom

location_facility: Hôpital de l'Archet, Service de Dermatologie

location_facility: Western Infirmary, Dermatology Department

location_facility: Roskilde Hospital, Division of Dermatology

location_facility: Hospital Virgen de la Macarena, Servicio de Dermatología

location_facility: Hospitais da Universidade de Coimbra, Servico de dermatologia

location_facility: FRCPC, Centre de Recherche Dermatologique du Quebec Metropolitain


type: EudraCT Number

title: Phase 1/1b Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AMG 596 as Monotherapy and in Combination With AMG 404 in Subjects With Glioblastoma or Malignant Glioma Expressing Mutant Epidermal Growth Factor Receptor Variant III (EGFRvIII)

summary:
This is a Phase 1/1b Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AMG 596 monotherapy or in combination with AMG 404 in Subjects with Glioblastoma or Malignant Glioma Expressing Mutant Epidermal Growth Factor Receptor Variant III (EGFRvIII).

This is a first in human (FIH), open-label, sequential-dose-escalation study in subjects with EGFRvIII-positive glioblastoma or malignant glioma. This study will enroll 2 groups of subjects according to disease stage, recurrent disease (Group 1) and maintenance treatment after SoC in newly diagnosed disease (Group 2).

design_allocation: Non-Randomized

ReferenceType: background

ReferenceCitation: Sternjak A, Lee F, Thomas O, Balazs M, Wahl J, Lorenczewski G, Ullrich I, Muenz M, Rattel B, Bailis JM, Friedrich M. Preclinical Assessment of AMG 596, a Bispecific T-cell Engager (BiTE) Immunotherapy Targeting the Tumor-specific Antigen EGFRvIII. Mol Cancer Ther. 2021 May;20(5):925-933. doi: 10.1158/1535-7163.MCT-20-0508. Epub 2021 Feb 25.

ipd_sharing: Yes

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

lead_sponsor: Amgen

location_country:
United States
Netherlands
France
Australia
Spain
Germany

location_facility: Hospital Universitari Germans Trias i Pujol

location_facility: UniversitÃ¤tsklinikum Carl Gustav Carus der Technischen UniversitÃ¤t Dresden


title: A Phase IV, Single-blinded, Prospective, Randomized, Controlled, Multi-center Study to Compare the Clinical Outcomes of Genea Embryo Review Instrument Plus (GERI+) Time Lapse System With a Conventional Embryo Culture and Assessment System

acronym: TICON

summary: The main purpose of the study was to evaluate the overall clinical value of GERI+ as an integrated embryo culture and assessment system, providing an undisturbed culture environment, continuous monitoring of embryo development and automated scoring using a predictive algorithm.

status: Terminated

lead_sponsor: Merck KGaA, Darmstadt, Germany

location_country: Italy Spain France Norway Portugal Denmark Canada

location_facility: Sykehuset Telemark HF Fertilitetsavdeliningen Sor

location_facility: St. Olavs Hospital Hf, Universitetssykehuset i Trondheim

location_facility: Fertility Clinic Skive Regional Hospital

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Centro Hospitalar e Universitario de Coimbra

intervention_type: Device

status_simplified: Unknown


title: Automatic Acoustic Speech Analysis and REM Sleep Behaviour Disorder for Detecting Subjects at High Risk for Parkinson's Disease and Other Alpha-synucleinopathies

summary: Speech is an important indicator of motor function and movement coordination and can be extremely sensitive to involvement in the course of neurologic diseases. The aim of this project is to discover for the first time using simple speech recording and high end pattern analysis preclinical stages of disabling central nervous system disorders including Parkinson's disease and other alpha-synucleinopathies in "at high risk" patients with REM sleep behavior disorder and thus provide one essential prerequisite for trials on REM sleep behavior disorder with preventive therapy.

status: Unknown status

primary_purpose: Diagnostic

ReferenceCitation: Rusz J, Hlavnička J, Tykalová T, Bušková J, Ulmanová O, Růžička E, Šonka K. Quantitative assessment of motor speech abnormalities in idiopathic rapid eye movement sleep behaviour disorder. Sleep Med. 2016 Mar;19:141-7. doi: 10.1016/j.sleep.2015.07.030. Epub 2015 Sep 14.

ReferenceCitation: Postuma RB, Lang AE, Gagnon JF, Pelletier A, Montplaisir JY. How does parkinsonism start? Prodromal parkinsonism motor changes in idiopathic REM sleep behaviour disorder. Brain. 2012 Jun;135(Pt 6):1860-70. doi: 10.1093/brain/aws093. Epub 2012 May 4.

ipd_sharing_description: De-identified clinical data (speech recordings and associated clinical scales) may be shared with the Michael J. Fox Foundation (the study funder). This data may be kept for storage at a central repository either hosted by the Michael J. Fox Foundation, its collaborators, or consultants.

lead_sponsor: Czech Technical University in Prague

location_country:
Italy
Austria
Germany
France
Czechia
United States
Canada

location_facility:
Gui-de-Chauliac Hospital
San Raffaele Hospital
University of Marburg
Mayo Clinic
McGill University Health Centre
Medical University of Innsbruck
General University Hospital

intervention_type: Other

lead_sponsor_type: academique


title: Randomized Study of IN.PACT 014 Paclitaxel-Coated Percutaneous Transluminal Angioplasty Balloon Catheter vs. Standard Percutaneous Transluminal Angioplasty for the Treatment of Chronic Total Occlusions in the Infrapopliteal Arteries

summary: To assess the safety and efficacy of the paclitaxel drug-eluting balloon IN.PACT 014 versus conventional percutaneous transluminal angioplasty (PTA) for the treatment of patients with chronic total occlusions in the infrapopliteal arteries.

status: Active, not recruiting

ReferenceCitation: Liistro F, Weinberg I, Almonacid Popma A, Shishehbor MH, Deckers S, Micari A. Paclitaxel-coated balloons versus percutaneous transluminal angioplasty for infrapopliteal chronic total occlusions: the IN.PACT BTK randomised trial. EuroIntervention. 2021 Oct 5. pii: EIJ-D-21-00444. doi: 10.4244/EIJ-D-21-00444. [Epub ahead of print]

lead_sponsor: Medtronic Endovascular

location_country: Italy Switzerland France Greece Belgium

location_facility: Hopital Guillaume et Rene Laennec - Centre Hospitalier Universitaire


lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Italy
United States
France
Spain
Germany
United Kingdom

location_facility: IRCCS Fondazione Istituto Neurologico Nazionale C. Mondino

location_facility: Sheffield Children's NHS Foundation Trust

location_facility: Leonard Wolfson Experimental Neurology Centre

source: euctr

title: A Phase 3, Randomized, Double-blind, Placebo-controlled, Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

summary: The primary objective of the study was to evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS.


title: International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases

summary: This study aims to understand the state of onset of NLSD(neutral lipid storage disease) / TGCV(triglyceride deposit cardiovasculopathy) worldwide, background information of affected patients, and natural history of the disease, as well as exploring the prognostic factors and assessing the efficacy of disease-specific treatment.

time_perspective: Other

lead_sponsor: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

location_country:
Italy
Austria
Netherlands
Germany
France
Japan
China
United States
United Kingdom

location_facility: National Center of Neurology and Psychiatry


title: A Multi-Center, Open Label, Phase I, Dose Finding and Expansion Study of RO7051790 Administered Orally in Patients With Relapsed, Extensive-Stage Disease Small Cell Lung Cancer (ED SCLC)

summary: This is a Phase I, open-label, multicenter study designed to assess the safety and tolerability of RO7051790 in participants with relapsed ED SCLC. This dose escalation and expansion study plans to determine the maximum tolerated dose and/or optimal biological dose as a recommended Phase 2 dose for RO7051790, based on the safety, tolerability, pharmacokinetic and pharmacodynamic profiles observed after oral administration of RO7051790.

lead_sponsor: Hoffmann-La Roche

location_country:
France
Spain
Denmark
United States
Canada

location_facility: Hospital Univ Vall d'Hebron; Servicio de Oncologia

location_facility: Centro Integral Oncologico Clara Campal (CIOCC); Dirección Médica

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: The Ottawa Hospital Cancer Centre; Oncology

location_facility: University Health Network; Princess Margaret Hospital; Medical Oncology Dept


title: Indolent Non Follicular Lymphomas Prognostic Project - Prospective Collection of Data of Possible Prognostic Relevance in Patients With Indolent Non-follicular B-CELL Lymphomas

summary: Prospective collection of data of possible prognostic relevance in patients with indolent non - follicular B-CELL Lymphomas.

ReferenceCitation: Luminari S, Merli M, Rattotti S, Tarantino V, Marcheselli L, Cavallo F, Varettoni M, Bianchi B, Merli F, Tedeschi A, Cabras G, Re F, Visco C, Torresan Delamain M, Cencini E, Spina M, Ferrero S, Ferrari A, Deodato M, Mannina D, Annibali O, Rago A, Orsucci L, Defrancesco I, Frigeni M, Cesaretti M, Arcaini L. Early progression as a predictor of survival in marginal zone lymphomas: an analysis from the FIL-NF10 study. Blood. 2019 Sep 5;134(10):798-801. doi: 10.1182/blood.2019001088. Epub 2019 Jul 10.

name: Division of Hematology, Azienda Unità Sanitaria Locale Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Reggio Emilia, Reggio Emilia, Italy;

name: Surgical, Medical and Dental Department of Morphological Sciences related to the Transplant, Oncology and Regenerative Medicine Department, University of Modena and Reggio Emilia, Modena, Italy;

family: Luminari

full_name: Stefano Luminari

given: Stefano

name: Division of Hematology, University Hospital Ospedale di Circolo e Fondazione Macchi–Azienda Socio-Sanitaria Territoriale Sette Laghi, University of Insubria, Varese, Italy;

family: Merli

full_name: Michele Merli

given: Michele

name: Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;

family: Rattotti

full_name: Sara Rattotti

given: Sara

name: PhD Program in Clinical and Experimental Medicine, University of Modena and Reggio Emilia, Modena, Italy;

name: Lymphoma Unit, Department of Onco-hematology, IRCCS San Raffaele Scientific Institute, Milan, Italy;

family: Tarantino

full_name: Vittoria Tarantino

given: Vittoria

name: Fondazione Italiana Linfomi, Modena, Italy;

family: Marcheselli

full_name: Luigi Marcheselli

given: Luigi

name: Hematology Division, Department of Molecular Biotechnologies and Health Sciences, University of Turin, Turin, Italy;

name: Division of Hematology 1, Azienda Ospedaliera Universitaria (AOU) Città della Salute e della Scienza di Torino, Turin, Italy;

family: Cavallo

full_name: Federica Cavallo

given: Federica

family: Varettoni

full_name: Marzia Varettoni

given: Marzia

family: Bianchi

full_name: Benedetta Bianchi

given: Benedetta

full_name: Francesco Merli

given: Francesco

name: Department of Hematology, Niguarda Cancer Center, Niguarda Hospital, Milan, Italy;

family: Tedeschi

full_name: Alessandra Tedeschi

given: Alessandra

name: Division of Hematology, Ospedale Oncologico Armando Businco, Cagliari, Italy;

family: Cabras

full_name: Giuseppina Cabras

given: Giuseppina

name: Division of Hematology, Azienda Ospedaliero–Universitaria di Parma, Parma, Italy;

family: Re

full_name: Francesca Re

given: Francesca

name: Section of Hematology, Department of Medicine, University of Verona, Verona, Italy;

family: Visco

full_name: Carlo Visco

given: Carlo

name: Center of Hematology and Hemotherapy, Department of Internal Medicine, Faculty of Medicine, State University of Campinas, Campinas, Brazil;

family: Torresan Delamain

full_name: Marcia Torresan Delamain

given: Marcia

name: Division of Hematology, Department of Oncology, Azienda Ospedaliera Universitaria Senese and University of Siena, Siena, Italy;

family: Cencini

full_name: Emanuele Cencini

given: Emanuele

name: Division of Medical Oncology A, National Cancer Institute, Aviano, Italy;

family: Spina

full_name: Michele Spina

family: Ferrero

full_name: Simone Ferrero

given: Simone

family: Ferrari

full_name: Angela Ferrari

given: Angela

family: Deodato

full_name: Marina Deodato

given: Marina

name: Department of Hematology, Azienda Ospedaliera Papardo, Messina, Italy;

family: Mannina

full_name: Donato Mannina

given: Donato

name: Unit of Hematology, Stem Cell Transplantation, Transfusion Medicine and Cellular Therapy, University Campus Bio-Medico, Rome, Italy;

family: Annibali

full_name: Ombretta Annibali

given: Ombretta

name: Department of Cellular Biotechnology and Hematology, University La Sapienza, Rome, Italy;

family: Rago

full_name: Angela Rago

name: Division of Hematology 2, AOU Città della Salute e della Scienza di Torino, Turin, Italy; and

family: Orsucci

full_name: Lorella Orsucci

given: Lorella

family: Defrancesco

name: Department of Molecular Medicine, University of Pavia, Pavia, Italy

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

location_country: Italy Austria Brazil France Portugal Ukraine

location_facility: Clinica Ematologica AO San Gerardo di Monza

location_facility: UO Ematologia con Trapianto Policlinico Consorziale

location_facility: SC. Ematologia. Osp. Riuniti Papardo Piemonte

location_facility: Ematologia IRCCS Policlinico S. Matteo di Pavia

location_facility: Dipartimento di Oncoematologia Ospedale San Raffaele

location_facility: Dipartimento di Oncologia Medica ed Ematologia Istituto Humanitas

location_facility: Unità Operativa Complessa di Ematologia - AO di Cosenza

location_facility: UOC Ematologia 1/CTMO, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

location_facility: SC Oncoematologia con autotrapianto AO S. Maria Terni

location_facility: USC Ematologia Ospedali Riuniti di Bergamo

location_facility: SC Ematologia Arcispedale Santa Maria Nuova

location_facility: SC Ematologia Universitaria, AO Città della Salute e della Scienza

location_facility: UOC di Ematologia con trapianto, Ospedale S. Maria Goretti

location_facility: SC Ematologia, AO Città della Salute e della Scienza

location_facility: Divisione di Ematologia, Ospedale San Bortolo

location_facility: Ematologia Ospedale Madonna delle Grazie

location_facility: Ematologia e CTMO Ospedale Maggiore di Parma

location_facility: UOC Ematologia, Azienda Ospedaliera Garibaldi P.O. Nesima

location_facility: National Cancer Institute of Health Ukraine

location_facility: Dipartimento di Oncologia ed Ematologia Università di Modena e Reggio Emilia

location_facility: Dipartimento di Ematologia Ospedale Civile Spirito Santo Pescara

location_facility: UO Ematologia e Trapianto Cellule Staminali, IRCCS, Centro di riferimento Oncologico di Basilicata

location_facility: Oncologia Medica A - Centro di Riferimento Oncologico

location_facility: Center of Hematology and Hemotherapy, UNICAMP, University of Campinas

location_facility: Divisione di Ematologia - Azienda Ospedaliera Bianchi Melacrino Morelli

location_facility: Unità operativa Complessa Ematologia - Azienda Ospedaliera Ospedale di Circolo e Fondazione Macchi


title: BIO|MASTER.Ilivia Family / Plexa

summary: Post-Market Clinical Follow-up of the new Ilivia ICD Family and the new Plexa right ventricular lead to fulfill requirements by the notified body and to support regulatory approval outside of the CE region

primary_purpose: Other

lead_sponsor: Biotronik SE & Co. KG

location_country: Austria Switzerland France Israel Singapore Australia Spain Denmark Germany Latvia

location_facility: Pauls Stradins Clinical University Hospital

location_facility: Hospital Universitario Virgen de Macarena


type: Other Identifier

title: COSIMO Cancer Associated Thrombosis - Patient Reported Outcomes With Rivaroxaban. A Non-interventional Study on Patients Changing to Xarelto® for Treatment of Venous Thromboembolism (VTE) and Prevention of Recurrent VTE in Patients With Active Cancer.

acronym: COSIMO

summary: This study aims to collect patient reported outcomes and assess treatment satisfaction in active cancer patients treated with rivaroxaban for VTE (venous thromboembolism).

ReferenceCitation: Cohen AT, Maraveyas A, Beyer-Westendorf J, Lee AYY, Mantovani LG, Bach M; COSIMO Investigators. COSIMO - patients with active cancer changing to rivaroxaban for the treatment and prevention of recurrent venous thromboembolism: a non-interventional study. Thromb J. 2018 Sep 4;16:21. doi: 10.1186/s12959-018-0176-2. eCollection 2018.

family: Cohen

full_name: Alexander T. Cohen

given: Alexander T.

family: Maraveyas

full_name: Anthony Maraveyas

given: Anthony

family: Beyer-Westendorf

full_name: Jan Beyer-Westendorf

given: Jan

family: Lee

full_name: Agnes Y. Y. Lee

given: Agnes Y. Y.

family: Mantovani

full_name: Lorenzo G. Mantovani

given: Lorenzo G.

family: Bach

full_name: Miriam Bach

given: Miriam

currency_apc_doaj: GBP

journal_name: Thrombosis Journal

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: Università degli Studi di Milano - Institutional Research Information System

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

evidence: oa repository (via pmcid lookup)

license_normalized: no license

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: COSIMO – patients with active cancer changing to rivaroxaban for the treatment and prevention of recurrent venous thromboembolism: a non-interventional study

lead_sponsor: Bayer

location_country:
Italy
Netherlands
Germany
France
Belgium
Australia
Spain
Denmark
Canada
United Kingdom


title: A Phase I/Ib, Open Label Study of LSZ102 Single Agent and LSZ102 in Combination With Either LEE011 (LSZ102 + LEE011) or BYL719 (LSZ102 + BYL719) in Patients With Advanced or Metastatic ER+ Breast Cancer Who Have Progressed After Endocrine Therapy

summary: To characterize the safety and tolerability, identify recommended doses and regimens for future studies, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of LSZ102 as a single agent and in combination with either LEE011 or BYL719 in adult patients with locally advanced or metastatic ER+ breast cancer who have progressed after endocrine therapy.

ReferenceCitation: Jhaveri K, Juric D, Yap YS, Cresta S, Layman RM, Duhoux FP, Terret C, Takahashi S, Huober J, Kundamal N, Sheng Q, Balbin A, Ji Y, He W, Crystal A, De Vita S, Curigliano G. A Phase I Study of LSZ102, an Oral Selective Estrogen Receptor Degrader, with or without Ribociclib or Alpelisib, in Patients with Estrogen Receptor-Positive Breast Cancer. Clin Cancer Res. 2021 Nov 1;27(21):5760-5770. doi: 10.1158/1078-0432.CCR-21-1095. Epub 2021 Aug 25.

family: Jhaveri

full_name: Komal Jhaveri

given: Komal

family: Juric

full_name: Dejan Juric

given: Dejan

family: Yap

full_name: Yoon-Sim Yap

given: Yoon-Sim

family: Cresta

full_name: Sara Cresta

given: Sara

family: Layman

full_name: Rachel M Layman

given: Rachel M

family: Duhoux

full_name: Francois P Duhoux

given: Francois P

family: Terret

full_name: Catherine Terret

given: Catherine

family: Takahashi

full_name: Shunji Takahashi

given: Shunji

family: Huober

full_name: Jens Huober

given: Jens

family: Kundamal

full_name: Nicole Kundamal

given: Nicole

family: Sheng

full_name: Qing Sheng

given: Qing

family: Balbin

full_name: Alejandro Balbin

given: Alejandro

family: Ji

full_name: Yan Ji

given: Yan

family: He

full_name: Wei He

given: Wei

family: Crystal

full_name: Adam Crystal

given: Adam

family: De Vita

full_name: Serena De Vita

given: Serena

family: Curigliano

full_name: Giuseppe Curigliano

given: Giuseppe

journal_name: Clinical Cancer Research

oa_colors: green hybrid

evidence: open (via free pdf)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: Universita Degli Studi Di Milano - Archivio Istituzionale della Ricerca

publisher: American Association for Cancer Research (AACR)

title: A Phase I Study of LSZ102, an Oral Selective Estrogen Receptor Degrader, With or Without Ribociclib or Alpelisib, in Patients with Estrogen Receptor-Positive Breast Cancer

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United States
France
Japan
Singapore
Belgium
Germany

location_facility: MD Anderson Cancer Center SC - LSZ102X2101

location_facility: Massachusetts General Hospital Massachusetts General Hospital


title: A Phase 1b Dose-Escalation and Cohort-Expansion Study of the Safety, Tolerability, and Efficacy of a Novel Transforming Growth Factor-β Receptor I Kinase Inhibitor (Galunisertib) Administered in Combination With the Anti-PD-L1 Antibody Durvalumab (MEDI4736) in Recurrent or Refractory Metastatic Pancreatic Cancer

summary: The main purpose of this study is to evaluate the safety and efficacy of the study drug known as galunisertib administered in combination with the anti-programmed cell death-ligand 1 (PD-L1) antibody durvalumab in participants with refractory metastatic pancreatic cancer.

ReferenceCitation: Melisi D, Oh DY, Hollebecque A, Calvo E, Varghese A, Borazanci E, Macarulla T, Merz V, Zecchetto C, Zhao Y, Gueorguieva I, Man M, Gandhi L, Estrem ST, Benhadji KA, Lanasa MC, Avsar E, Guba SC, Garcia-Carbonero R. Safety and activity of the TGFβ receptor I kinase inhibitor galunisertib plus the anti-PD-L1 antibody durvalumab in metastatic pancreatic cancer. J Immunother Cancer. 2021 Mar;9(3). pii: e002068. doi: 10.1136/jitc-2020-002068.

family: Melisi

full_name: Davide Melisi

given: Davide

family: Oh

full_name: Do-Youn Oh

given: Do-Youn

family: Hollebecque

full_name: Antoine Hollebecque

given: Antoine

family: Calvo

full_name: Emiliano Calvo

given: Emiliano

family: Varghese

full_name: Anna Varghese

given: Anna

family: Borazanci

full_name: Erkut Borazanci

given: Erkut

family: Macarulla

full_name: Teresa Macarulla

given: Teresa

family: Merz

full_name: Valeria Merz

given: Valeria

family: Zecchetto

full_name: Camilla Zecchetto

given: Camilla

family: Zhao

full_name: Yumin Zhao

given: Yumin

family: Gueorguieva

full_name: Ivelina Gueorguieva

given: Ivelina

family: Man

full_name: Michael Man

given: Michael

family: Gandhi

full_name: Leena Gandhi

given: Leena

family: Estrem

full_name: Shawn T Estrem

given: Shawn T

family: Benhadji

full_name: Karim A Benhadji

given: Karim A

family: Lanasa

full_name: Mark C Lanasa

given: Mark C

family: Avsar

full_name: Emin Avsar

given: Emin

family: Guba

full_name: Susan C Guba

given: Susan C

family: Garcia-Carbonero

full_name: Rocio Garcia-Carbonero

given: Rocio

currency_apc_doaj: USD

journal_name: Journal for ImmunoTherapy of Cancer

license: cc-by-nc

license_normalized: cc-by-nc

location_country:
Italy
France
Spain
United States
Korea, Republic of

location_facility: Ospedale Policlinico Giambattista Rossi, Borgo Roma


title: International Protocol for the Treatment of Childhood Anaplastic Large Cell Lymphoma

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. It is not yet known which combination chemotherapy regimen is more effective for treating anaplastic large cell lymphoma.

PURPOSE: This randomized phase III trial is studying several different regimens of combination chemotherapy to compare how well they work in treating children with anaplastic large cell lymphoma.

ReferenceCitation: Attarbaschi A, Mann G, Rosolen A, Williams D, Uyttebroeck A, Marky I, Lamant L, Horibe K, Wrobel G, Beishuizen A, Wössmann W, Reiter A, Mauguen A, Le Deley MC, Brugières L; European Intergroup for Childhood Non-Hodgkin Lymphoma (EICNHL) ALCL99 Trial. Limited stage I disease is not necessarily indicative of an excellent prognosis in childhood anaplastic large cell lymphoma. Blood. 2011 May 26;117(21):5616-9. doi: 10.1182/blood-2010-12-324012. Epub 2011 Mar 28.

name: Paediatric Hematology and Oncology, St Anna Children's Hospital, Vienna, Austria;

family: Attarbaschi

full_name: Andishe Attarbaschi

given: Andishe

family: Mann

full_name: Georg Mann

given: Georg

name: Paediatric Hematology and Oncology, University of Padova, Padova, Italy;

family: Rosolen

full_name: Angelo Rosolen

given: Angelo

name: Paediatric Hematology and Oncology, Cambridge University Hospitals Foundation Trust, Addenbrooke's Hospital, Cambridge, United Kingdom;

family: Williams

full_name: Denise Williams

given: Denise

name: Paediatric Hematology and Oncology, University of Leuven, Leuven, Belgium;

family: Uyttebroeck

full_name: Anne Uyttebroeck

given: Anne

name: Paediatric Hematology and Oncology, University of Goteborg, Goteborg, Sweden;

family: Marky

full_name: Ildiko Marky

given: Ildiko

name: Pathology, Hôpital Purpan, Toulouse, France;

family: Lamant

full_name: Laurence Lamant

given: Laurence

name: National Hospital Organization Nagoya Medical Center, Aichi, Japan;

family: Horibe

full_name: Keizo Horibe

given: Keizo

name: Department of Bone Marrow Transplantation and Childhood Oncology and Hematology, Medical University of Wroclaw, Wroclaw, Poland;

family: Wrobel

full_name: Grazyna Wrobel

given: Grazyna

name: Paediatric Hematology and Oncology, Erasmus MC–Sophia Children's Hospital, Rotterdam, The Netherlands;

family: Beishuizen

full_name: Auke Beishuizen

given: Auke

name: NHL-BFM Study Centre, Paediatric Hematology and Oncology, Justus Liebig University, Giessen, Germany;

family: Wössmann

full_name: Wilhelm Wössmann

given: Wilhelm

family: Reiter

full_name: Alfred Reiter

given: Alfred

name: Biostatistics and Epidemiology Unit, Institute Gustave-Roussy, Villejuif, France;

family: Mauguen

full_name: Audrey Mauguen

given: Audrey

name: Université Paris-Sud 11, Paris, France; and

family: Le Deley

full_name: Marie-Cecile Le Deley

given: Marie-Cecile

name: Paediatric Hematology and Oncology, Institute Gustave-Roussy, Villejuif, France

family: Brugières

full_name: Laurence Brugières

name: for the European Intergroup for Childhood Non-Hodgkin Lymphoma (EICNHL) ALCL99 Trial

journal_name: Blood

publisher: American Society of Hematology

publisher_dissemination: American Society of Hematology

publisher_group: American Society of Hematology

publisher_normalized: American Society of Hematology

ReferenceCitation: Wrobel G, Mauguen A, Rosolen A, Reiter A, Williams D, Horibe K, Brugières L, Le Deley MC; European Inter-Group for Childhood, Non-Hodgkin Lymphoma (EICNHL). Safety assessment of intensive induction therapy in childhood anaplastic large cell lymphoma: report of the ALCL99 randomised trial. Pediatr Blood Cancer. 2011 Jul 1;56(7):1071-7. doi: 10.1002/pbc.22940. Epub 2011 Jan 28.

name: on behalf of European Inter-Group for Childhood, Non-Hodgkin Lymphoma (EICNHL)

title: Safety assessment of intensive induction therapy in childhood anaplastic large cell lymphoma: Report of the ALCL99 randomised trial

ReferenceCitation: Le Deley MC, Rosolen A, Williams DM, Horibe K, Wrobel G, Attarbaschi A, Zsiros J, Uyttebroeck A, Marky IM, Lamant L, Woessmann W, Pillon M, Hobson R, Mauguen A, Reiter A, Brugières L. Vinblastine in children and adolescents with high-risk anaplastic large-cell lymphoma: results of the randomized ALCL99-vinblastine trial. J Clin Oncol. 2010 Sep 1;28(25):3987-93. doi: 10.1200/JCO.2010.28.5999. Epub 2010 Aug 2.

name: From the Institut Gustave-Roussy, Villejuif; Université Paris-Sud, Le Kremlin-Bicêtre; Centre Hospitalier Universitaire Toulouse, Hôpital Purpan; L'Institut National de la Santé et de la Recherche Médicale U563, Centre de Physiopathologie de Toulouse Purpan, Toulouse, France; University Hospital, Padova, Italy; Cambridge University Hospital National Health Service Trust, Cambridge; Children's Cancer and Leukaemia Group Data Centre, Leicester, United Kingdom; Clinical Research Center, National Hospital...

publisher: American Society of Clinical Oncology (ASCO)

title: Vinblastine in Children and Adolescents With High-Risk Anaplastic Large-Cell Lymphoma: Results of the Randomized ALCL99-Vinblastine Trial

lead_sponsor: Gustave Roussy, Cancer Campus, Grand Paris

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

location_facility: Karolinska University Hospital - Huddinge

location_facility: Hospital Clinico Universitario de Valencia


title: Postoperative Treatment of Glioblastoma With BNCT at the Petten Irradiation Facility

summary:
RATIONALE: Boron neutron capture therapy may selectively kill tumor cells without harming normal tissue.

PURPOSE: This phase I trial is studying the side effects and best dose of boron neutron capture therapy following surgery in treating patients with glioblastoma multiforme removed during surgery.

ReferenceCitation: Wittig A, Moss RL, Stecher-Rasmussen F, Appelman K, Rassow J, Roca A, Sauerwein W. Neutron activation of patients following boron neutron capture therapy of brain tumors at the high flux reactor (HFR) Petten (EORTC Trials 11961 and 11011). Strahlenther Onkol. 2005 Dec;181(12):774-82.

ReferenceCitation: Verbakel WF, Hideghety K, Morrissey J, Sauerwein W, Stecher-Rasmussen F. Towards in vivo monitoring of neutron distributions for quality control of BNCT. Phys Med Biol. 2002 Apr 7;47(7):1059-72.

ReferenceCitation: Hüsing J, Sauerwein W, Hideghéty K, Jöckel KH. A scheme for a dose-escalation study when the event is lagged. Stat Med. 2001 Nov 30;20(22):3323-34.

ReferenceCitation: Rassow J, Stecher-Rasmussen F, Voorbraak W, Moss R, Vroegindeweij C, Hideghéty K, Sauerwein W. Comparison of quality assurance for performance and safety characteristics of the facility for Boron Neutron Capture therapy in Petten/NL with medical electron accelerators. Radiother Oncol. 2001 Apr;59(1):99-108. Review.

ReferenceCitation: Sauerwein W, Moss R, Rassow J, Stecher-Rasmussen F, Hideghéty K, Wolbers JG, Sack H. Organisation and management of the first clinical trial of BNCT in Europe (EORTC protocol 11961).EORTC BNCT study group. Strahlenther Onkol. 1999 Jun;175 Suppl 2:108-11.

ReferenceCitation: Gabel D, Philipp KH, Wheeler FJ, Huiskamp R. The compound factor of the 10B(n,alpha)7Li reaction from borocaptate sodium and the relative biological effectiveness of recoil protons for induction of brain damage in boron neutron capture therapy. Radiat Res. 1998 Apr;149(4):378-86.

ReferenceCitation: Pignol JP, Oudart H, Chauvel P, Sauerwein W, Gabel D, Prevot G. Selective delivery of 10B to soft tissue sarcoma using 10B-L-borophenylalanine for boron neutron capture therapy. Br J Radiol. 1998 Mar;71(843):320-3.

ReferenceCitation: Verbakel WF, Sauerwein W, Hideghety K, Stecher-Rasmussen F. Boron concentrations in brain during boron neutron capture therapy: in vivo measurements from the phase I trial EORTC 11961 using a gamma-ray telescope. Int J Radiat Oncol Biol Phys. 2003 Mar 1;55(3):743-56.

ReferenceCitation: Hideghéty K, Sauerwein W, Haselsberger K, Grochulla F, Fankhauser H, Moss R, Huiskamp R, Gabel D, de Vries M. Postoperative treatment of glioblastoma with BNCT at the petten irradiation facility (EORTC protocol 11,961). Strahlenther Onkol. 1999 Jun;175 Suppl 2:111-4.

ReferenceCitation: Gabel D, Touw D, Stecher-Rasmussen F, et al.: Quality control of Na2B12H11SH, a drug boron neutron capture therapy in EORTC trial 11961. [Abstract] Ann Oncol 9(suppl 2): 129, 1998.

ReferenceCitation: Hideghéty K, Sauerwein W, Wittig A, Götz C, Paquis P, Grochulla F, Haselsberger K, Wolbers J, Moss R, Huiskamp R, Fankhauser H, de Vries M, Gabel D. Tissue uptake of BSH in patients with glioblastoma in the EORTC 11961 phase I BNCT trial. J Neurooncol. 2003 Mar-Apr;62(1-2):145-56.

ReferenceCitation: Hideghety W, Sauerwein W, DeVries M, et al.: Post-operative treatment of glioblastoma with boron neutron capture therapy at the European High Flux Reactor Petten (EORTC protocol 11961). [Abstract] Ann Oncol 9(suppl 2): 129, 1998.

ReferenceCitation: Sauerwein W, Hideghety K, De Vries M, et al.: Boron neutron capture therapy (BNCT) for the treatment of glioblastoma (EORTC protocol 11961). [Abstract] Radiother Oncol 48(suppl 1): s157, 1998.

ReferenceCitation: Sauerwein W, Hideghety K, De Vries M, et al.: Conducting phase I clinical trial in binary treatment modality: methodical questions for the evaluation of boron neutron capture therapy. [Abstract] Ann Oncol 9(suppl 2): 129, 1998.

ReferenceCitation: Vos MJ, Turowski B, Zanella FE, Paquis P, Siefert A, Hideghéty K, Haselsberger K, Grochulla F, Postma TJ, Wittig A, Heimans JJ, Slotman BJ, Vandertop WP, Sauerwein W. Radiologic findings in patients treated with boron neutron capture therapy for glioblastoma multiforme within EORTC trial 11961. Int J Radiat Oncol Biol Phys. 2005 Feb 1;61(2):392-9.

ReferenceCitation: Haselsberger K, Pendl G, Sauerwein W, et al.: BNCT for glioblastoma in Europe: design of the EORTC protocol 11961 and clinical course of the first patient. [Abstract] J Neurooncol 39 (2): A-P155, 147, 1998.

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

location_country: Italy Austria Netherlands Germany France Canada

location_facility: Klinikum der Universitaet Muenchen - Grosshadern Campus

location_facility: Toronto Sunnybrook Regional Cancer Centre at Sunnybrook Health Sciences Centre

location_facility: EC Joint Research Centre - Institute for Energy

intervention_type: Procedure


ReferenceCitation: Wijburg FA, Whitley CB, Muenzer J, Gasperini S, Del Toro M, Muschol N, Cleary M, Sevin C, Shapiro E, Bhargava P, Kerr D, Alexanderian D. Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial. Mol Genet Metab. 2019 Feb;126(2):121-130. doi: 10.1016/j.ymgme.2018.10.006. Epub 2018 Oct 24.

family: Wijburg

full_name: Frits A. Wijburg

given: Frits A.

family: Whitley

full_name: Chester B. Whitley

given: Chester B.

family: Muenzer

full_name: Joseph Muenzer

given: Joseph

family: Gasperini

full_name: Serena Gasperini

given: Serena

family: del Toro

full_name: Mireia del Toro

given: Mireia

family: Muschol

full_name: Nicole Muschol

given: Nicole

family: Cleary

full_name: Maureen Cleary

given: Maureen

family: Sevin

full_name: Caroline Sevin

given: Caroline

family: Shapiro

full_name: Elsa Shapiro

given: Elsa

family: Bhargava

full_name: Parul Bhargava

given: Parul

family: Kerr

full_name: Douglas Kerr

given: Douglas

family: Alexanderian

full_name: David Alexanderian

given: David

journal_name: Molecular Genetics and Metabolism

evidence: open (via crossref license)

repository_institution: INFN Catania - INFM-OAR

repository_normalized: Zenodo

version: acceptedVersion

ipd_sharing_description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

lead_sponsor: Shire

location_country:
Italy
United States
Netherlands
France
Argentina
Spain
Germany
United Kingdom

location_facility: University of Minnesota Department of Pediatrics

location_facility: U.O.S Malattie Metaboliche Rare Clinical Pediatrica

location_facility: Hospital Universitario Austral A Unidad de Investigacion

title: A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of rhHNS (Recombinant Human Heparan N Sulfatase) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Early Stage Mucopolysaccharidosis Type IIIA Disease

summary:
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase). In the absence of this enzyme, there is an accumulation of the glycosaminoglycan, heparan sulfate, resulting in progressive neurodegeneration. Symptoms are usually first noted in the 1st or 2nd year of life, although definitive diagnosis is often delayed, with an average age of diagnosis of 4.5 years. The disease is characterized by developmental delays initially, followed by neurological developmental arrest, then regression. These developmental deficits are typically associated with severe behavioral disturbances. Patients have a significantly reduced lifespan, with few surviving beyond the 2nd or 3rd decade.

The purpose of this study is to evaluate the safety and efficacy of recombinant human heparan-N-sulfatase (rhHNS) in pediatric patients with Early Stage Mucopolysaccharidosis Type III A Disease.


title: Multicenter, Open-label, Extension Study to Characterize the Long-term Efficacy and Safety of Early Versus Delayed Treatment With Venglustat (GZ/SAR402671) in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

acronym: STAGED-PKD-EXT

summary:
Primary Objective:

-To determine the effect of early versus delayed treatment with venglustat on the total kidney volume (TKV) in participants at risk of rapidly progressive ADPKD.

Secondary Objective:

To determine the effect of early versus delayed treatment with venglustat on the renal function (estimated glomerular filtration rate [eGFR]).
To characterize the safety profile of venglustat.
To evaluate the effect of venglustat on the lens by ophthalmological examination.
To evaluate the effect of venglustat on mood using Beck Depression Inventory-II (BDI-II).

ipd_sharing_description: Qualified researchers may request access to patient 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. Patient 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: Sanofi

location_country:
Netherlands
Germany
France
Japan
Romania
Belgium
Australia
Spain
United States
Korea, Republic of

location_facility:
Investigational Site Number 8400019
Investigational Site Number 0560002
Investigational Site Number 0360001
Investigational Site Number 3920007
Investigational Site Number 4100002
Investigational Site Number 2760001
Investigational Site Number 5280002
Investigational Site Number 2500002
Investigational Site Number 3920002
Investigational Site Number 3920005
Investigational Site Number 6420002
Investigational Site Number 3920001
Investigational Site Number 4100001
Investigational Site Number 3920004
Investigational Site Number 7240003
Investigational Site Number 6420001
Investigational Site Number 7240002


title: SUrgical Versus PERcutaneous AXillary Artery International Registry A Physician-initiated, International, Multicentric, Retrospective, Observational, Voluntary Registry - SUPER AXA International Registry

summary: The purpose of the Study is to compare the outcomes of the surgical and the percutaneous approach to the upper extremity access (axillary or brachial artery) during endovascular procedures on the aortic valve, the aorta, and its side branches.

time_perspective: Retrospective

ReferenceCitation: Mirza AK, Oderich GS, Sandri GA, Tenorio ER, Davila VJ, Kärkkäinen JM, Hofer J, Cha S. Outcomes of upper extremity access during fenestrated-branched endovascular aortic repair. J Vasc Surg. 2019 Mar;69(3):635-643. doi: 10.1016/j.jvs.2018.05.214. Epub 2018 Oct 24.

ReferenceCitation: Branzan D, Steiner S, Haensig M, Scheinert D, Schmidt A. Percutaneous Axillary Artery Access for Endovascular Treatment of Complex Thoraco-abdominal Aortic Aneurysms. Eur J Vasc Endovasc Surg. 2019 Sep;58(3):344-349. doi: 10.1016/j.ejvs.2019.01.011. Epub 2019 Jul 30.

ReferenceCitation: Malgor RD, Marques de Marino P, Verhoeven E, Katsargyris A. A systematic review of outcomes of upper extremity access for fenestrated and branched endovascular aortic repair. J Vasc Surg. 2020 May;71(5):1763-1770.e2. doi: 10.1016/j.jvs.2019.09.028. Epub 2019 Nov 15.

ReferenceCitation: Deuschl F, Schofer N, Seiffert M, Hakmi S, Mizote I, Schaefer A, Schirmer J, Reichenspurner H, Blankenberg S, Conradi L, Schäfer U. Direct percutaneous transaxillary implantation of a novel self-expandable transcatheter heart valve for aortic stenosis. Catheter Cardiovasc Interv. 2017 Dec 1;90(7):1167-1174. doi: 10.1002/ccd.26986. Epub 2017 Mar 15.

ReferenceCitation: Schäfer U, Deuschl F, Schofer N, Frerker C, Schmidt T, Kuck KH, Kreidel F, Schirmer J, Mizote I, Reichenspurner H, Blankenberg S, Treede H, Conradi L. Safety and efficacy of the percutaneous transaxillary access for transcatheter aortic valve implantation using various transcatheter heart valves in 100 consecutive patients. Int J Cardiol. 2017 Apr 1;232:247-254. doi: 10.1016/j.ijcard.2017.01.010. Epub 2017 Jan 7.

ReferenceCitation: Fröhlich GM, Baxter PD, Malkin CJ, Scott DJ, Moat NE, Hildick-Smith D, Cunningham D, MacCarthy PA, Trivedi U, de Belder MA, Ludman PF, Blackman DJ; National Institute for Cardiovascular Outcomes Research. Comparative survival after transapical, direct aortic, and subclavian transcatheter aortic valve implantation (data from the UK TAVI registry). Am J Cardiol. 2015 Nov 15;116(10):1555-9. doi: 10.1016/j.amjcard.2015.08.035. Epub 2015 Sep 3.

ReferenceCitation: Arnett DM, Lee JC, Harms MA, Kearney KE, Ramos M, Smith BM, Anderson EC, Tayal R, McCabe JM. Caliber and fitness of the axillary artery as a conduit for large-bore cardiovascular procedures. Catheter Cardiovasc Interv. 2018 Jan 1;91(1):150-156. doi: 10.1002/ccd.27416. Epub 2017 Nov 11.

ReferenceCitation: Kappetein AP, Head SJ, Généreux P, Piazza N, van Mieghem NM, Blackstone EH, Brott TG, Cohen DJ, Cutlip DE, van Es GA, Hahn RT, Kirtane AJ, Krucoff MW, Kodali S, Mack MJ, Mehran R, Rodés-Cabau J, Vranckx P, Webb JG, Windecker S, Serruys PW, Leon MB; Valve Academic Research Consortium (VARC)-2. Updated standardized endpoint definitions for transcatheter aortic valve implantation: the Valve Academic Research Consortium-2 consensus document (VARC-2). Eur J Cardiothorac Surg. 2012 Nov;42(5):S45-60. doi: 10.1093/ejcts/ezs533. Epub 2012 Oct 1.

ReferenceCitation: Bertoglio L, Grandi A, Melloni A, Kahlberg A, Melissano G, Chiesa R. Percutaneous AXillary Artery (PAXA) Access at the First Segment During Fenestrated and Branched Endovascular Aortic Procedures. Eur J Vasc Endovasc Surg. 2020 Jun;59(6):929-938. doi: 10.1016/j.ejvs.2020.01.027. Epub 2020 Feb 20.

ReferenceCitation: Schaefer A, Schirmer J, Schofer N, Schneeberger Y, Deuschl F, Blankenberg S, Reichenspurner H, Conradi L, Schäfer U. Transaxillary transcatheter aortic valve implantation utilizing a novel vascular closure device with resorbable collagen material: a feasibility study. Clin Res Cardiol. 2019 Jul;108(7):779-786. doi: 10.1007/s00392-018-1407-z. Epub 2018 Dec 17.

ReferenceCitation: De Palma R, Rück A, Settergren M, Saleh N. Percutaneous axillary arteriotomy closure during transcatheter aortic valve replacement using the MANTA device. Catheter Cardiovasc Interv. 2018 Nov 1;92(5):998-1001. doi: 10.1002/ccd.27383. Epub 2017 Oct 25.

ReferenceCitation: Puippe GD, Kobe A, Rancic Z, Pfiffner R, Lachat M, Pfammatter T. Safety of percutaneous axillary artery access with a suture-mediated closing device for parallel endograft aortic procedures - a retrospective pilot study. Vasa. 2018 Jun;47(4):311-317. doi: 10.1024/0301-1526/a000702. Epub 2018 Mar 27.

name: Department of Diagnostic and                     Interventional Radiology, University Hospital Zurich, Zurich, Switzerland

family: Puippe

full_name: Gilbert Dominique Puippe

given: Gilbert Dominique

family: Kobe

full_name: Adrian Kobe

given: Adrian

name: Division of Cardiac and Vascular Surgery,                     University Hospital Zurich, Zurich, Switzerland

family: Rancic

full_name: Zoran Rancic

given: Zoran

family: Pfiffner

full_name: Roger Pfiffner

given: Roger

family: Lachat

full_name: Mario Lachat

given: Mario

family: Pfammatter

full_name: Thomas Pfammatter

given: Thomas

journal_name: Vasa

publisher: Hogrefe Publishing Group

publisher_dissemination: Hogrefe Publishing Group

publisher_group: Hogrefe Publishing Group

publisher_normalized: Hogrefe Publishing Group

title: Safety of percutaneous axillary artery access with a suture-mediated closing device for parallel endograft aortic procedures – a retrospective pilot study

ipd_sharing: Undecided

lead_sponsor: IRCCS San Raffaele

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

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: Service de Chirurgie Thoracique et Cardiovasculaire

location_facility: Ascension St John Hospital and Medical Center

location_facility: Nuffield Dept. of Clinical Neurosciences, University of Oxford

location_facility: University Heart and Vascular Center Hamburg


lead_sponsor: Sumitomo Dainippon Pharma Oncology, Inc

location_country:
Taiwan
United States
France
Japan

location_facility: Winship Cancer Institute, Emory University

title: A Rollover Study to Provide Continued Access to Napabucasin for Patients Enrolled in Boston Biomedical-sponsored Napabucasin Protocols

summary: This is an open-label, multi-center, multi-national, non-randomized rollover study designed to allow continued access to napabucasin for patients who have participated in a Boston Biomedical-sponsored study and are being treated with napabucasin (monotherapy or combination) and who are deriving continued clinical benefit in the parent study at the time of closure.


title: SYMBIOSIS Post-Market Registry

acronym: SYMBIOSIS

summary: The purpose of this observational, multi-center prospective, post-market registry is to confirm real-world device safety and performance, to ensure the continued acceptability of identified risks, and detect emerging risks.

lead_sponsor: Medtronic Cardiovascular

location_country: Austria Netherlands France Greece Norway Belgium Germany

location_facility:
UZ Leuven
Ziekenhuis Oost-Limburg
Catharina Ziekenhuis
CHU d'Angers
Onassis Cardiac Surgery Center
Haukeland Universitetssjukehus
St. Antonius Ziekenhuis
AKH Wien
Herz- und Diabeteszentrum NRW


title: Observational Study, for Quality Assessment, of Sarcoma as a Model to Improve Diagnosis and Clinical Care of Rare Tumors Through a European and Latin American Multidisciplinary NETWORK

acronym: (SELNET)

summary:
Post-authorisation, multicentric, observational, retrospective and prospective study to assess quality of care of sarcoma patients in expert and non-expert centers by analysing correlation of quality items and outcomes such as relapse free survival, overall survival, percentage of amputation, etc.

Expert pathology peer review will be performed to detect differences between expert and non-expert centers as well as differences in treatment and patient prognosis.

Tumor samples of 4 types of sarcoma would also be included in translational research to detect biomarkers and produce preclinical models.

ipd_sharing_description: Data could be shared receiving an application to the Study Main contact indicating reason and objectives.

lead_sponsor: Grupo Espanol de Investigacion en Sarcomas

location_country:
Italy
Brazil
Peru
Costa Rica
Mexico
France
Argentina
Spain

location_facility: Instituo Nacional de enfermedades Neoplásicas


title: Prospective Registry for Patients Undergoing Artificial Urinary Sphincter (AUS) Surgery for Female Stress Urinary Incontinence (SUI) Due to Intrinsic Sphincter Deficiency (ISD)

acronym: VENUS

summary: Prospective collection of pre-defined parameters of AUS Surgery for Female Stress Urinary Incontinence due to Intrinsic Sphincter Deficiency

ipd_sharing_description: Investigators may share pseudonymised individual data with residents/urologist interested to analyze and publish the results of the registry

lead_sponsor: European Association of Urology Research Foundation

location_country:
France
Belgium
Spain
Germany
United Kingdom

location_facility: University of Rennes, Department of Urology


intervention_type: Biological

lead_sponsor: Baxalta now part of Shire

primary_purpose: Prevention

location_country:
Italy
Netherlands
France
Russian Federation
Spain
Turkey
United States

acronym: rVWF Pediatric and Adult Study

location_facility: Groupement Hospitalier Est- Hôpital Louis Pradel

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Hospital Universitari i Politecnic La Fe

location_facility: SAIH "Kemerovo Regional Clinical Hospital"

location_facility: Hospital General Universitario de Alicante

location_facility: FSBI of Science "Kirov Scientific and Research Institute of Hematology and Blood Transfusion of FMBA

location_facility: Indiana Hemophilia and Thrombosis Center

location_facility: Azienda Ospedaliera Universitaria Careggi

title: A Phase 3b, Prospective, Open-label, Uncontrolled, Multicenter Study on Long-term Safety and Efficacy of rVWF in Pediatric and Adult Subjects With Severe Von Willebrand Disease (VWD)

summary: This study will evaluate the long-term safety and hemostatic efficacy of recombinant von Willebrand factor (rVWF) (vonicog alfa) prophylaxis in adult and pediatric participants (aged 12 to <18 years) with severe von Willebrand disease (VWD) with the option of once weekly dosing, and to further assess the safety and efficacy of rVWF in on-demand (OD) treatment of bleeding episodes, and in perioperative management of surgical bleeding.


intervention_type: Genetic

ipd_sharing_description: Bluebird bio is committed to transparency and appropriately de-identified patient-level datasets and supporting documents may be shared following attainment of applicable marketing approvals associated with this study and consistent with criteria established by bluebird bio and/or industry best practices to maintain the privacy of study participants. For enquiries, please contact us at datasharing@bluebirdbio.com.

lead_sponsor: bluebird bio

location_country:
Italy
United States
Netherlands
France
Germany
United Kingdom

location_facility: Boston Children's Hospital/Massachusetts General Hospital

title: A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

summary:
Study ALD-104 is an international, non-randomized, open-label, multi-site study in male participants (<or=17 years of age at enrollment) with cerebral adrenoleukodystrophy (CALD). Approximately 35 participants will be infused with Lenti-D Drug Product after myeloablative conditioning with busulfan and fludarabine.

This trial will evaluate the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of CALD. A subject's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the subject following myeloablative conditioning.


title: A Phase I First Time in Human Open Label Study of GSK3745417 Administered With and Without Anticancer Agents in Participants With Advanced Solid Tumors

summary: This study aims to evaluate the safety, tolerability, and preliminary clinical activity and establish a recommended dose of GSK3745417 administered alone (Part 1A) or co-administered (Part 2A) with dostarlimab in participants with refractory/relapsed solid tumors. Both parts will consist of a dose escalation phase.

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

lead_sponsor: GlaxoSmithKline

location_country:
United States
Netherlands
France
Australia
Spain
Canada
Korea, Republic of


title: Evaluation of the Xpert® Bladder Cancer Monitor Assay Compared to Cystoscopy for the Follow-up of Patients With History of Low or Intermediate Risk Non-muscle-invasive Bladder Cancer (NMIBC): an Observational Prospective International Multicenter Study

acronym: ANTICIPATE X

summary: Bladder cancer is the 5th most common cancer in Europe, with more than 151,000 new cases diagnosed in 2012 (4% of the total). Bladder cancer has the highest recurrence rate of any malignancy, often as high as 70% within 5 years of successful treatment. This high recurrence rate requires diligent and accurate monitoring as a means for early diagnosis and treatment. Considering the burden associated to repeated invasive cystoscopies, there is a need for robust but accurate tests for surveillance. In that prospect, urinary molecular tests have been developed although none were deemed adequate in the European clinical guidelines to replace cystoscopies. The Xpert Bladder Cancer Monitor Assay is a qualitative in vitro diagnostic test designed to monitor for the recurrence of bladder cancer in patients previously diagnosed with this cancer. The test provides a fast and accurate result, is non-invasive and easy to perform. The aim of this study is to assess the non-inferiority of the Xpert Bladder Cancer Monitor assay in detecting recurrences in comparison to cystoscopy in the follow-up of patients with low or intermediate risk non-muscle-invasive bladder cancer (NMIBC).

lead_sponsor: Cepheid

location_country:
Italy
Austria
Sweden
Netherlands
France
Czechia
Spain
Germany
United Kingdom

location_facility: CHU Toulouse / Institut Universitaire du Cancer Toulouse Oncopole

location_facility: Humanitas University - Gradenigo Hospital of Turin

location_facility: University Clinic for Urology and Andrology

intervention_type: Diagnostic Test


title: Persistence Of Major Molecular Remission In Chronic Myeloid Leukemia After A Second Stop Of Tki Treatment In Patients Who Failed An Initial Stop Attempt: A Multicenter Prospective Trial

summary: This study will enroll CML patients who have failed a first TKI stopping attempt. After failure and at least a year of TKI treatment, patients will proceed to dasatinib treatment for another 2 years. If MR4 or better is re-achieved and maintained for at least one year, patients will be eligible for a second stop. After verification of MR4, TKI treatment will be stopped and patients followed in the same manner as after first stop. If MMR is lost (BCR-ABL >0.1% (IS)), TKI treatment will once again be restarted.

ipd_sharing_description: Study results will be published as soon as possible. Steering committee will decide on sharing IPD. This is still pending.

lead_sponsor: VU University Medical Center

location_country: Sweden Netherlands Finland France Norway Denmark Germany

location_facility: St Olavs Hospital-Trondheim University Hospital

location_facility: Uppsala University Hospital (Akademiska)


title: The Effect of Low Protein, Extensively Hydrolyzed Infant Formula on Allergy Prevention in At-risk Infants up to 1 Year of Age: a Randomized, Double-blind, Controlled Intervention Study and the Long-term Effect on Allergy Prevention of Early Nutrition Given in the First 120 Days of Life in At-risk Infants Until the Child is 6 Years of Age

summary: A multi-center, prospective, randomized, controlled, doubleblind intervention clinical Trial is performed to proof the efficiency of hydrolyzed infant formula showing a risk reduction of developing an allergy.

lead_sponsor: HiPP GmbH & Co. Vertrieb KG

location_country: Italy Serbia Spain Finland France Czechia Bulgaria Poland Portugal Germany

location_facility: Clinical Hospital Center "Dr Dragisa Misovic-Dedinje"

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Klinik für Kinder- und Jugendmedizin Marienhospital gGmbH Wesel

location_facility: Studienambulanz der pädiatrische Pneumologie und AllergologieKlinik für Kinder- und Jugendmedizin (KKJM)

location_facility: Second specialized obstetric and gynecological hospital for active treatment Sheinovo EAD

location_facility: Klinik für Kinder- und Jugendmedizin der Ruhruniversität Bochum im St. Josef Hospital


summary: This study will compare the safety and efficacy of the 700 ug dexamethasone intravitreal implant with ranibizumab 0.5 mg intravitreal injections in patients with diabetic macular edema (DME).

ReferenceCitation: Callanan DG, Loewenstein A, Patel SS, Massin P, Corcóstegui B, Li XY, Jiao J, Hashad Y, Whitcup SM. A multicenter, 12-month randomized study comparing dexamethasone intravitreal implant with ranibizumab in patients with diabetic macular edema. Graefes Arch Clin Exp Ophthalmol. 2017 Mar;255(3):463-473. doi: 10.1007/s00417-016-3472-1. Epub 2016 Sep 8.

ReferenceCitation: Rittiphairoj T, Mir TA, Li T, Virgili G. Intravitreal steroids for macular edema in diabetes. Cochrane Database Syst Rev. 2020 Nov 17;11:CD005656. doi: 10.1002/14651858.CD005656.pub3.

name: Department of Epidemiology; Johns Hopkins Bloomberg School of Public Health; Baltimore Maryland USA

family: Rittiphairoj

full_name: Thanitsara Rittiphairoj

given: Thanitsara

name: Wilmer Eye Institute; Johns Hopkins University School of Medicine; Baltimore Maryland USA

family: Mir

full_name: Tahreem A Mir

given: Tahreem A

name: Department of Ophthalmology; University of Colorado Denver Anschutz Medical Campus; Aurora CO USA

family: Li

full_name: Tianjing Li

given: Tianjing

name: Department of Neurosciences, Psychology, Drug Research and Child Health (NEUROFARBA); University of Florence; Florence Italy

name: Centre for Public Health; Queen's University Belfast; Belfast UK

family: Virgili

full_name: Gianni Virgili

given: Gianni

journal_name: Cochrane Database of Systematic Reviews

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Chakravarthy H, Devanathan V. Molecular Mechanisms Mediating Diabetic Retinal Neurodegeneration: Potential Research Avenues and Therapeutic Targets. J Mol Neurosci. 2018 Nov;66(3):445-461. doi: 10.1007/s12031-018-1188-x. Epub 2018 Oct 6. Review.

family: Chakravarthy

full_name: Harshini Chakravarthy

given: Harshini

family: Devanathan

full_name: Vasudharani Devanathan

given: Vasudharani

journal_name: Journal of Molecular Neuroscience

lead_sponsor: Allergan

location_country:
Italy
South Africa
Netherlands
Spain
Germany
France
Israel
Belgium
Portugal
Denmark
United States
United Kingdom


title: Use of the VisuMax Femtosecond Laser Lenticule Removal Procedure for the Correction of Hyperopia With or Without Astigmatism

summary: The correction of farsightedness using ReLEx SMILE for hyperopia is focus of this investigation. The objective is to evaluate the safety and effectiveness.

lead_sponsor: Carl Zeiss Meditec AG

location_country:
India
France
Czechia
China
Germany
United Kingdom

location_facility: University Medical Center Universitätsklinikum Gießen Marburg

location_facility: Medical Center Nethradhama Superspeciality Eye Hospital


ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison. Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

family: Newman

full_name: Nancy J. Newman

given: Nancy J.

family: Yu-Wai-Man

full_name: Patrick Yu-Wai-Man

given: Patrick

family: Carelli

full_name: Valerio Carelli

given: Valerio

family: Biousse

full_name: Valerie Biousse

given: Valerie

family: Moster

full_name: Mark L. Moster

given: Mark L.

family: Vignal-Clermont

full_name: Catherine Vignal-Clermont

given: Catherine

family: Sergott

full_name: Robert C. Sergott

given: Robert C.

family: Klopstock

full_name: Thomas Klopstock

family: Sadun

full_name: Alfredo A. Sadun

given: Alfredo A.

family: Girmens

full_name: Jean-François Girmens

given: Jean-François

family: La Morgia

full_name: Chiara La Morgia

given: Chiara

family: DeBusk

full_name: Adam A. DeBusk

given: Adam A.

family: Jurkute

full_name: Neringa Jurkute

given: Neringa

family: Priglinger

full_name: Claudia Priglinger

given: Claudia

family: Karanjia

full_name: Rustum Karanjia

given: Rustum

family: Josse

full_name: Constant Josse

given: Constant

family: Salzmann

full_name: Julie Salzmann

given: Julie

family: Montestruc

full_name: François Montestruc

given: François

family: Roux

full_name: Michel Roux

given: Michel

family: Taiel

full_name: Magali Taiel

given: Magali

family: Sahel

full_name: José-Alain Sahel

given: José-Alain

journal_name: Frontiers in Neurology

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

location_country:
Italy
United States
France
Germany
United Kingdom

location_facility: LMU Klinikum der Universität München / Friedrich-Baur-Institut

title: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials

summary: The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.


title: An Open-Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Participants With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency

summary: This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL Deficiency and was designed to examine 3 doses of sebelipase alfa. The targeted number for this study was 9 evaluable participants.

ReferenceCitation: Balwani M, Breen C, Enns GM, Deegan PB, Honzík T, Jones S, Kane JP, Malinova V, Sharma R, Stock EO, Valayannopoulos V, Wraith JE, Burg J, Eckert S, Schneider E, Quinn AG. Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease. Hepatology. 2013 Sep;58(3):950-7. doi: 10.1002/hep.26289. Epub 2013 Mar 28.

name: Department of Genetics and Genomic SciencesThe Mount Sinai School of MedicineNew YorkNY

family: Balwani

full_name: Manisha Balwani

given: Manisha

name: Manchester Academic Health Sciences CentreGenetic MedicineSt. Mary's HospitalManchesterUK

family: Breen

full_name: Catherine Breen

name: Department of PediatricsLucile Packard Children's HospitalStanford UniversityStanfordCA

family: Enns

full_name: Gregory M. Enns

given: Gregory M.

name: Department of MedicineAddenbrooke's HospitalCambridgeUK

family: Deegan

full_name: Patrick B. Deegan

given: Patrick B.

name: Department of Pediatrics and Adolescent MedicineFirst Faculty of MedicineCharles University and General University HospitalPragueCzech Republic

family: Honzík

full_name: Tomas Honzík

given: Tomas

family: Jones

full_name: Simon Jones

given: Simon

name: Divisions of Endocrinology & Metabolism (JPK) and Cardiology (EOS)University of CaliforniaSan FranciscoCA

family: Kane

full_name: John P. Kane

given: John P.

family: Malinova

full_name: Vera Malinova

given: Vera

name: Salford Royal Hospital Foundation TrustSalfordUK

family: Sharma

full_name: Reena Sharma

given: Reena

family: Stock

full_name: Eveline O. Stock

given: Eveline O.

name: Inherited Metabolic DisordersHôpital Necker‐Enfants MaladesParisFrance

family: Valayannopoulos

full_name: Vassili Valayannopoulos

given: Vassili

family: Wraith

full_name: J. Edmond Wraith

given: J. Edmond

family: Burg

full_name: Jennifer Burg

given: Jennifer

family: Eckert

full_name: Stephen Eckert

given: Stephen

family: Schneider

full_name: Eugene Schneider

given: Eugene

family: Quinn

full_name: Anthony G. Quinn

given: Anthony G.

journal_name: Hepatology

oa_host_type: repository

title: Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients With cholesteryl ester storage disease

lead_sponsor: Alexion Pharmaceuticals

location_country:
United States
United Kingdom
France
Czechia


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

acronym: TOTAL

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

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

ReferenceCitation: Deprest J, Breysem L, Gratacos E, Nicolaides K, Claus F, Debeer A, Smet MH, Proesmans M, Fayoux P, Storme L. Tracheal side effects following fetal endoscopic tracheal occlusion for severe congenital diaphragmatic hernia. Pediatr Radiol. 2010 May;40(5):670-3. doi: 10.1007/s00247-010-1579-9. Epub 2010 Mar 30.

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

ReferenceCitation: Deprest JA, Hyett JA, Flake AW, Nicolaides K, Gratacos E. Current controversies in prenatal diagnosis 4: Should fetal surgery be done in all cases of severe diaphragmatic hernia? Prenat Diagn. 2009 Jan;29(1):15-9. doi: 10.1002/pd.2108.

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

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

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

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

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

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

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

name: From the Department of Obstetrics and Gynecology, KU Leuven (J.A.D., F.M.R.) and Academic Department of Development and Regeneration, Biomedical Sciences, University Hospitals KU Leuven, Leuven, Belgium (J.A.D., B.V.C., F.M.R.); King’s College Hospital (K.H.N.) and the Institute for Women’s Health, University College London Hospital (J.A.D.) — both in London; Hospital Antoine–Béclère, Université Paris–Saclay, Clamart, France (A.B.); Hospital Clinic and Sant Joan de Déu, Barcelona (E.G.); Mount Sinai...

family: Deprest

full_name: Jan A. Deprest

given: Jan A.

family: Nicolaides

full_name: Kypros H. Nicolaides

given: Kypros H.

family: Benachi

full_name: Alexandra Benachi

given: Alexandra

family: Gratacos

full_name: Eduard Gratacos

given: Eduard

family: Ryan

full_name: Greg Ryan

given: Greg

family: Persico

full_name: Nicola Persico

given: Nicola

family: Sago

full_name: Haruhiko Sago

given: Haruhiko

family: Johnson

full_name: Anthony Johnson

given: Anthony

family: Wielgoś

full_name: Mirosław Wielgoś

given: Mirosław

family: Berg

full_name: Christoph Berg

given: Christoph

family: Van Calster

full_name: Ben Van Calster

given: Ben

family: Russo

full_name: Francesca M. Russo

given: Francesca M.

journal_name: New England Journal of Medicine

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

lead_sponsor: University Hospital, Gasthuisberg

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

location_facility: University of Texas Health Science Center

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

location_facility: National Center for Child Health and Development


lead_sponsor: AstraZeneca

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

acronym: GLOW

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

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


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

acronym: GARDIAN

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

lead_sponsor: Bayer

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


lead_sponsor: H. Lundbeck A/S

location_country:
Italy
South Africa
Serbia
United Kingdom
France
Bulgaria
Poland
Hungary
Russian Federation
Spain
Estonia
Germany
Latvia

location_facility: Unidad de Salud Mental Infanto-Juvenil (USMI-J) Edificio de Consultas Externas. Hospital MarAtimo

location_facility: SPECTRUM Centrum Neurologii i Psychiatrii

location_facility: Filip Rybakowski Specjalistyczna Praktyka Lekarska

location_facility: Medicorehabilitation Research Center "Phoenix"

location_facility: Scientific Institute Fondazione Stella Maris

location_facility: Guz "Saratov Regional Psychiatric Hospital St. Sofii"

location_facility: Clinic of Neurology and Psychiatry for Children and Adolescents

location_facility: Specialized Clinical Psychiatric Hospital 1 of the Ministry of Health

location_facility: Clinical Center of Vojvodina - Clinic of Psychiatry

location_facility: Prywatne Gabinety Lekarskie ¿Promedicus¿ Anna Agnieszka Tomczak

location_facility: State Budgetary Healthcare Institution of Sverdlovsk Region

location_facility: Nizhny Novgorod Region State Institution Of Healthcare

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Universita degli Studi di Messina - Facolta di Medicina e Chirurgia

location_facility: Rheinhessen-Fachklinik Mainz, Kinder und Jugendpsychiatri

location_facility: Przychodnia Syntonia Poradnia Zdrowia Psychicznego

location_facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej (SPZOZ) - Centrum Neuropsychiatrii "NEUROMED"

location_facility: Lipetsk Regional Psychoneurological Hospital

location_facility: Centrum Badan Klinicznych PI-House Sp. z o.o.

location_facility: Bekes Megyei Kozponti Korhaz Pandy Kalman Tagkorhaza

location_facility: Rostov State Medical University of the Minzdravsotsrazvitiya of Russia

location_facility: Marienthal Center of Psychiatry and Psychology

title: Long-term, Open-label, Flexible-dose, Continuation Extension Study With Vortioxetine in Child and Adolescent Patients With Major Depressive Disorder (MDD) From 7 to 17 Years of Age

summary: Evaluation of the long-term safety and tolerability of vortioxetine in child and adolescent patients with a Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5™) diagnosis of MDD


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

family: Dick

full_name: Andrew D. Dick

given: Andrew D.

family: Tugal-Tutkun

full_name: Ilknur Tugal-Tutkun

given: Ilknur

family: Foster

full_name: Stephen Foster

family: Zierhut

full_name: Manfred Zierhut

given: Manfred

family: Melissa Liew

full_name: S.H. Melissa Liew

family: Bezlyak

full_name: Vladimir Bezlyak

given: Vladimir

family: Androudi

full_name: Sofia Androudi

given: Sofia

journal_name: Ophthalmology

title: Secukinumab in the Treatment of Noninfectious Uveitis: Results of Three Randomized, Controlled Clinical Trials

lead_sponsor: Novartis Pharmaceuticals

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

acronym: SHIELD

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

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


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

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

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

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

family: Isakoff

full_name: S J Isakoff

given: S J

family: Wang

full_name: D Wang

given: D

family: Campone

full_name: M Campone

given: M

family: Calles

full_name: A Calles

given: A

family: Leip

full_name: E Leip

given: E

family: Turnbull

full_name: K Turnbull

given: K

family: Bardy-Bouxin

full_name: N Bardy-Bouxin

given: N

family: Duvillié

full_name: L Duvillié

given: L

family: Calvo

full_name: E Calvo

journal_name: British Journal of Cancer

evidence: open (via page says license)

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

publisher_dissemination: Springer

publisher_group: Springer

lead_sponsor: Pfizer

location_country:
Hong Kong
France
Belgium
Australia
Spain
United States


title: Treatment of Patients With Acute Sinusitis in Daily Practice

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

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

family: Mösges

full_name: Ralph Mösges

given: Ralph

family: Desrosiers

full_name: Martin Desrosiers

given: Martin

family: Arvis

full_name: Pierre Arvis

given: Pierre

family: Heldner

full_name: Stephanie Heldner

given: Stephanie

journal_name: PLoS ONE

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

publisher: Public Library of Science (PLoS)

publisher_dissemination: Public Library of Science

publisher_group: Public Library of Science

publisher_normalized: Public Library of Science

title: Characterisation of Patients Receiving Moxifloxacin for Acute Bacterial Rhinosinusitis in Clinical Practice: Results from an International, Observational Cohort Study

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


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

acronym: EUS-EG

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

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

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

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

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

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

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

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

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

family: Săftoiu

full_name: Adrian Săftoiu

given: Adrian

family: Vilmann

full_name: Peter Vilmann

given: Peter

family: Gorunescu

full_name: Florin Gorunescu

given: Florin

family: Janssen

full_name: Jan Janssen

given: Jan

family: Hocke

full_name: Michael Hocke

given: Michael

family: Larsen

full_name: Michael Larsen

family: Iglesias–Garcia

full_name: Julio Iglesias–Garcia

given: Julio

family: Arcidiacono

full_name: Paolo Arcidiacono

given: Paolo

family: Will

full_name: Uwe Will

given: Uwe

family: Giovannini

full_name: Marc Giovannini

given: Marc

family: Dietrich

full_name: Cristoph F. Dietrich

given: Cristoph F.

family: Havre

full_name: Roald Havre

given: Roald

family: Gheorghe

full_name: Cristian Gheorghe

given: Cristian

family: McKay

full_name: Colin McKay

given: Colin

family: Gheonea

full_name: Dan Ionuţ Gheonea

given: Dan Ionuţ

family: Ciurea

full_name: Tudorel Ciurea

given: Tudorel

journal_name: Clinical Gastroenterology and Hepatology

title: Efficacy of an Artificial Neural Network–Based Approach to Endoscopic Ultrasound Elastography in Diagnosis of Focal Pancreatic Masses

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

full_name: A. Săftoiu

given: A.

full_name: P. Vilmann

given: P.

full_name: F. Gorunescu

given: F.

lead_sponsor: University of Medicine and Pharmacy Craiova

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

location_facility: HELIOS Klinikum Wuppertal, University of Witten/Herdecke


summary: This study evaluates the efficacy and safety of Cyclosporine 0.010% eye drops in the treatment of Atopic Keratoconjunctivitis (chronic and severe inflammation of the eye). The study consists of a double-masked phase, and open-labeled phase, and an open-labeled maintenance phase. For the first 3 months of the study, patients will receive either masked Cyclosporine 0.010% eye drops or vehicle four times daily; for the next 6 months, patients may receive open-labeled Cyclosporine 0.010% eye drops four times daily. At month 9, patients who are in remission, will be re-randomized to receive either open-labeled Cyclosporine 0.010% eye drops four times daily or twice daily.

location_country:
Italy
United States
India
United Kingdom
Germany
France
Israel
Australia
New Zealand
Spain
Canada
Czech Republic