title: A Phase 1/2 Dose-Escalation Study of XL147 (SAR245408) or XL765 (SAR245409) in Combination With Letrozole in Subjects With Hormone Receptor-Positive and HER2-Negative Breast Cancer Refractory to a Nonsteroidal Aromatase Inhibitor

summary:
Phase 1 of this study will evaluate the maximum tolerated dose (MTD) of XL147 when given in combination with letrozole (Femara) and of XL765 when given in combination with letrozole. After the MTD is established for each combination (Phase 2), subjects will be enrolled to evaluate the preliminary efficacy and safety of these combinations in subjects with breast cancer refractory to a non-steroidal aromatase inhibitor that is ER+/PGR+ and HER2-. Letrozole is used in the treatment of different types of breast cancer, but patients can develop resistance.

Upregulation of PI3K activity is one of the most common characteristics of human cancer cells, including breast tumor cells. Activation of PI3K results in stimulation of AKT and mTOR kinases, resulting in the promotion of tumor cell proliferation and survival. Preclinical and retrospective clinical data suggest that aberrant activation of the PI3K pathway may play a role in aromatase inhibitor resistance in patients with ER+, HER2- breast cancer. XL147 is a potent inhibitor of PI3K, and XL765 is a potent dual inhibitor of PI3K and mTOR; therefore either of these compounds in combination with letrozole warrants clinical investigation.

lead_sponsor: Sanofi

location_country:
United States
France
Spain

location_facility:
Investigational Site Number 3321
Investigational Site Number 1331
Investigational Site Number 3324
Investigational Site Number 1330
Investigational Site Number 3415
Investigational Site Number 1441
Investigational Site Number 3420
Investigational Site Number 1601
Investigational Site Number 1252
Investigational Site Number 5246
Investigational Site Number 3413
Investigational Site Number 1238
Investigational Site Number 1214
Investigational Site Number 1138
Investigational Site Number 1537
Investigational Site Number 5201
Investigational Site Number 3419


title: Medtronic Adapta/Versa/Sensia Long Term Reliability Study

summary: The primary purpose of this study is to assess the long term reliability of the Medtronic Adapta®/Sensia™/Versa™ platform of devices. This study is required by FDA as a condition of approval of nEw3 devices. Patients will be followed for 5 years after implant. This study utilizes data collected from the System Longevity Study (SLS).

status: Completed

study_completion_date_type: Actual

study_type: Observational

time_perspective: Prospective

enrollment_type: Actual

lead_sponsor: Medtronic

location_country:
Italy
Austria
United States
Sweden
Serbia
Switzerland
Netherlands
Germany
France
Belgium
Denmark
Canada

lead_sponsor_type: industriel

status_simplified: Completed


type: EudraCT Number

type: Other Identifier

title: A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638

summary: The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

ReferenceType: background

ReferenceCitation: McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. Epub 2007 May 16.

ReferenceCitation: Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.

ReferenceCitation: Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. Erratum in: Blood. 2011 May 19;117(20):5551.

ReferenceType: derived

ReferenceCitation: Charrow J, Fraga C, Gu X, Ida H, Longo N, Lukina E, Nonino A, Gaemers SJM, Jouvin MH, Li J, Wu Y, Xue Y, Peterschmitt MJ. Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial. Mol Genet Metab. 2018 Mar;123(3):347-356. doi: 10.1016/j.ymgme.2017.12.001. Epub 2018 Jan 4.

family: Charrow

full_name: Joel Charrow

given: Joel

sequence: first

family: Fraga

full_name: Cristina Fraga

given: Cristina

sequence: additional

family: Gu

full_name: Xuefan Gu

given: Xuefan

family: Ida

full_name: Hiroyuki Ida

given: Hiroyuki

family: Longo

full_name: Nicola Longo

given: Nicola

family: Lukina

full_name: Elena Lukina

given: Elena

family: Nonino

full_name: Alexandre Nonino

given: Alexandre

family: Gaemers

full_name: Sebastiaan J.M. Gaemers

given: Sebastiaan J.M.

family: Jouvin

full_name: Marie-Helene Jouvin

given: Marie-Helene

family: Li

full_name: Jing Li

given: Jing

family: Wu

full_name: Yaoshi Wu

given: Yaoshi

family: Xue

full_name: Yong Xue

given: Yong

family: Peterschmitt

full_name: M. Judith Peterschmitt

given: M. Judith

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Molecular Genetics and Metabolism

oa_host_type: publisher

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Austria
United States
Brazil
India
Serbia
Sweden
Netherlands
Croatia
France
Greece
Japan
Romania
Australia
Russian Federation
China
Canada
Portugal

location_facility: University of California, San Diego Medical Center

location_facility: St. Petersburg State Medical Pavlov University

location_facility: Instituto Tropical de Medicina Reprodutiva e Menopausa - INTRO

location_facility: State Medical and Prophylactic Healthcare Institution; Chelyabinsk Regional Clinical Hospital

location_facility: Cettro - Centro de Tratamento de Oncologia e Hematologia

location_facility: Hospital de Clínicas da Universidade Federal do Parana

location_facility: General Hospital of Athens "G. Gennimatas"

location_facility: IGEIM - Institute of Genetic and Inborn Erros of Metabolism

location_facility: Hemocentro de Ribeirão Preto Núcleo de Hemoterapia de Franca

location_facility: Hospital Universitario Walter Cantidio - HUWC

location_facility: Hematology Research Center of Russian Academy of Medical Sciences

location_facility: Shanghai Xinhua Hospital Shanghai Xinhua Hospital

location_facility: Hôpital Femme Mère Enfant Centre de référence des maladies Héréditaires du métabolisme

intervention_type: Drug


title: A pRospective, Multicenter, Single-arm Study EvaLuating the Safety and feasIbility of Targeted Lung Denervation (TLD) for the trEatment oF Severe Asthma

summary: Prospective, multicenter, single-arm (non-randomized) study of Targeted Lung Denervation (TLD) Therapy in subjects with severe asthma.

study_start_date_type: Actual

status: Unknown status

study_completion_date_type: Anticipated

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Nuvaira, Inc.

location_country:
Germany
United Kingdom
France
Netherlands

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: Royal Brompton & Harefield NHS Foundation Trust

location_facility: Chelsea & Westminster Hospital NHS Foundation Trust

intervention_type: Device

status_simplified: Unknown


title: Interventional, Randomised, Open Label, Multi-centre, Parallel-group, Controlled Study Investigating the Effects of Using the PReDicT Test to Guide the Antidepressant Treatment of Depressed Patients

summary:
Depression is a very common, serious and in some cases life-threatening condition, affecting around 350 million people globally. Approximately 11% of citizens in the European Union suffer from depression at some point in their lives. Depression is associated with significant socio-economic costs and has been predicted to become the greatest cause of disability worldwide by 2030 . In 2010 it was estimated that there were approximately 30 million patients with depression in Europe, with aggregated economic costs of approximately €92 billion . Improvements in managing the treatment of depression are urgently needed to improve patient outcomes, contain rising healthcare costs, improve workplace productivity and help to address global economic and societal challenges.

While a range of effective antidepressant medications are available to treat depression, it takes 4-6 weeks after starting antidepressant treatment before a physician can detect whether the treatment is working. However, surprisingly, more than 50% of patients fail to respond to the first antidepressant treatment they are prescribed. Therefore, it often takes several months to identify an effective antidepressant treatment for the majority of patients with depression. During this time a patient's ability to work and function socially is severely impaired. Individuals may be absent from work for many weeks or months and this places a substantial burden on the economy and on healthcare resources.

ReferenceType: result

ReferenceCitation: World Health Organization, The Global Burden of Disease: 2004 update. nDeneva, World Health Organization, 2008.

ReferenceCitation: Wittchen HU, Jacobi F, Rehm J, Gustavsson A, Svensson M, Jönsson B, Olesen J, Allgulander C, Alonso J, Faravelli C, Fratiglioni L, Jennum P, Lieb R, Maercker A, van Os J, Preisig M, Salvador-Carulla L, Simon R, Steinhausen HC. The size and burden of mental disorders and other disorders of the brain in Europe 2010. Eur Neuropsychopharmacol. 2011 Sep;21(9):655-79. doi: 10.1016/j.euroneuro.2011.07.018. Review.

family: Wittchen

full_name: H.U. Wittchen

family: Jacobi

full_name: F. Jacobi

given: F.

family: Rehm

full_name: J. Rehm

given: J.

family: Gustavsson

full_name: A. Gustavsson

given: A.

family: Svensson

full_name: M. Svensson

given: M.

family: Jönsson

full_name: B. Jönsson

given: B.

family: Olesen

full_name: J. Olesen

family: Allgulander

full_name: C. Allgulander

given: C.

family: Alonso

full_name: J. Alonso

family: Faravelli

full_name: C. Faravelli

family: Fratiglioni

full_name: L. Fratiglioni

given: L.

family: Jennum

full_name: P. Jennum

given: P.

family: Lieb

full_name: R. Lieb

given: R.

family: Maercker

full_name: A. Maercker

family: van Os

full_name: J. van Os

family: Preisig

full_name: M. Preisig

family: Salvador-Carulla

full_name: L. Salvador-Carulla

family: Simon

full_name: R. Simon

family: Steinhausen

full_name: H.-C. Steinhausen

journal_name: European Neuropsychopharmacology

oa_host_type: repository

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

host_type: repository

license_normalized: no license

repository_normalized: qucosa

version: submittedVersion

ReferenceCitation: Rush AJ. STAR*D: what have we learned? Am J Psychiatry. 2007 Feb;164(2):201-4.

ReferenceCitation: Kingslake J, Dias R, Dawson GR, Simon J, Goodwin GM, Harmer CJ, Morriss R, Brown S, Guo B, Dourish CT, Ruhé HG, Lever AG, Veltman DJ, van Schaik A, Deckert J, Reif A, Stäblein M, Menke A, Gorwood P, Voegeli G, Pérez V, Browning M. The effects of using the PReDicT Test to guide the antidepressant treatment of depressed patients: study protocol for a randomised controlled trial. Trials. 2017 Nov 23;18(1):558. doi: 10.1186/s13063-017-2247-2.

repository_institution: University of Nottingham - Repository@Nottingham

repository_institution: Würzburg University - Online Publication Service of Würzburg University

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

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

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

repository_institution: Goethe University Frankfurt - Publication Server of Goethe University Frankfurt am Main

location_country:
Netherlands
France
Spain
Germany
United Kingdom

location_facility: Dept of Psychiatry, Outpatient Clinic, University Hospital

location_facility: Rothwell and Desborough Healthcare Group

location_facility: University of Nottingham Health Service - Cripps Health Centre


title: REPAIR - transcatheteR rEPair of mitrAl Insufficiency With caRdioband System

acronym: REPAIR

summary:
The Cardioband Transcatheter System (Cardioband) is indicated for the treatment of secondary (functional) mitral regurgitation (FMR). The Cardioband is a transcatheter system, deployed on the beating heart through a transseptal approach. The Cardioband is deployed along the posterior annulus of the mitral valve (MV) and is adjusted under trans-esophageal guidance on the beating heart. A CE mark study with 30 subjects has been completed and documented reduction of severity of mitral regurgitation (MR) and improvement in 6- minute walk test in subjects with moderate to severe MR.

Study objectives are to test the efficacy of the Cardioband in improving MR and heart failure symptoms in patients with symptomatic (New York Heart Association (NYHA) Class III-IVa), severe MR in the post-marketing setting, And to evaluate the safety of the Cardioband system in the post-marketing setting.

status: Terminated

ipd_sharing: Undecided

lead_sponsor: Edwards Lifesciences

location_country: Italy Germany France

location_facility:
Bonn University
Universitäres Herzzentrum Hamburg GmbH
Asklepios Klinik, St. Georg
Universitatsmedizin Mainz
Bichat hospital
Hospital san raffaele
Bad Nauheim, Kerckhoff-Klinik
University Hospital Halle (Saale)
Universitätsklinikum Gießen und Marburg
Koln, Universitätsklinikum


title: A Randomized Phase III Study to Compare Arsenic Trioxide (ATO) Combined to ATRA and Idarubicin Versus Standard ATRA and Anthracyclines-based Chemotherapy (AIDA Regimen) for Patients With Newly Diagnosed, High-risk Acute Promyelocytic Leukemia

summary:
Acute promyelocytic leukemia (APL) is a rare subtype of acute myeloid leukemia (AML) characterized by consistent clinical, morphologic, and genetic features. According to the FAB classification APL is designated as"M3 leukemia" and assigned to the WHO defined type of AML with recurrent cytogenetic abnormalities, "acute promyelocytic leukemia with t(15;17)(q22;q12), (PML/RARα) and variants".

Despite the dramatic progress achieved in frontline therapy of APL with ATRA plus anthracycline-based regimens, relapses still occur in approximately 20% of patients. Moreover, these regimens are associated with significant toxicities due to severe myelosuppression frequently associated with life-threatening infections and potentially serious late effects including development of secondary MDS/AML. In a recent randomized clinical trial in low/intermediate-risk APL (WBC ≤ 10 GPt/l APL0406 trial) a combination of arsenic trioxide (ATO) and ATRA has been shown to result into better survival with significantly lower toxicity rates compared to the standard ATRA + idarubicin (AIDA) therapy. Inspired by the results of this trial the investigators intend to perform a randomized study in high-risk APL (WBC at diagnosis > 10 GPt/l) comparing standard AIDA-based treatment with ATO/ATRA combination including low-doses idarubicin during induction. The investigators propose a modified ATO/ATRA protocol with the addition of two doses of IDA (50% compared to standard AIDA induction) for induction because of the anticipated need of adding anthracyclines to control hyperleukocytosis and to achieve long-term disease control in this high-risk APL population. This is followed by 4 cycles of ATO/ATRA consolidation therapy. As in the APL0406 study for low/intermediate-risk patients the investigators expect less severe hematologic toxicity and treatment-related mortality resulting in an improved outcome for patients in the experimental arm. Furthermore, from the start of consolidation, these patients (in contrast to the standard arm) can be treated on an outpatient basis, which is also considered to be associated with an improved quality of life. The study will be conducted as a European intergroup study.

status: Recruiting

lead_sponsor: Technische Universität Dresden

location_country: Italy Netherlands France Spain Germany

location_facility:
SAL study group
AML-SG study group
HOVON study group
OSHO study group
PETHEMA study group
French-Belgian-Swiss APL study group
GIMEMA study group
AML-CG study group

lead_sponsor_type: academique

status_simplified: Ongoing


title: Non-interventional Study to Investigate the Short and Long Term Real-life Safety, Effectiveness, and Adherence of Velphoro® in Patients With Hyperphosphataemia Undergoing Haemodialysis or Peritoneal Dialysis

acronym: VERIFIE

summary:
An oral highly potent P binder Velphoro is a mixture of polynuclear iron(III) oxyhydroxide, sucrose, and starches. It was well tolerated in the clinical development program. The approved indication in the European Union (EU) is to control serums phosphorus (sP) levels in adult CKD (Chronic kidney disease) patients on HD (Haemodialysis) or PD (Peritoneal dialysis).

It is of major interest to observe the drug in daily use outside of controlled trial settings. The Marketing Authorisation Holder wishes to obtain further systematic data within a non-interventional study to investigate short and long-term safety.

Effectiveness and Treatment adherence during real-life use will be evaluated.

lead_sponsor: Vifor Fresenius Medical Care Renal Pharma

location_country:
Italy
Netherlands
France
Greece
Spain
Germany
United Kingdom

location_facility: Hospital Universitario Marqués de Valdecilla


title: Multi-center, Prospective, Post-market Study

summary: The purpose of this multi-center, non-interventional, prospective, post-market clinical study is to collect real world data on patient outcomes and evaluate the procedural success and performance of the Lifetech CeraFlex™ occluders for patients with secundum type Atrial Septum Defect (ASD), Patent Foramen Ovale (PFO) or Patent Ductus Arteriosus (PDA).

lead_sponsor: Lifetech Scientific (Shenzhen) Co., Ltd.

location_country: Italy Switzerland France Ireland Germany

location_facility: AP Hospital Europeen G Pompidou and Hospital Necker


ReferenceCitation: Brændvik SM, Roeleveld K, Andersen GL, Raftemo AE, Ramstad K, Majkic-Tajsic J, Lamvik T, Lund B, Follestad T, Vik T. The WE-Study: does botulinum toxin A make walking easier in children with cerebral palsy?: Study protocol for a randomized controlled trial. Trials. 2017 Feb 6;18(1):58. doi: 10.1186/s13063-016-1772-8.

lead_sponsor: St. Olavs Hospital

location_country: Norway France Poland

acronym: WE

location_facility: Department of Orthopaedic Surgery, St. Olavs University Hospital

location_facility: Mazowieckie Centrum Neuropsychiatrii, Zagorze

source: euctr

title: Does Botulinum Toxin A Make Walking Easier in Children With Cerebral Palsy?

summary: In Norway, about 60% of all children with cerebral palsy (CP) are being treated with botulinum toxin A (BoNT-A) at 6 years of age, mainly in the legs. Despite this widespread use of the drug, the evidence for a positive effect on walking is insufficient. Moreover, large variation in effect is seen by clinicians. The main objective of the present study is to investigate whether injections with BoNT-A in the calf muscles make walking easier in children with spastic CP within 6 months, reflected by reduced energy cost during walking.


title: Open Non-randomized Clinical Investigation to Evaluate the Safety and Effectiveness of the Nerve Capping Device to Prevent Neuroma Formation After Traumatic Nerve Section

summary: This study is conducted to clinically assess safety and performance of the Polyganics nerve capping device for the treatment of symptomatic neuroma. There is sufficient clinical experience with regard to the safety of the commercially available nerve guide, NEUROLAC®. This new nerve capping device is identical in material and manufacturing. The exception is in design, where NEUROLAC® has two open ends, the nerve capping device has one closed (sealed) end. This study will be conducted to obtain data on the clinical performance of the capping device's ability to isolate the nerve end, resulting in a reduction of pain of experienced from the symptomatic neuroma and prevention of the reoccurrence of a symptomatic neuroma.

lead_sponsor: Polyganics BV

location_country: France Netherlands

location_facility:
Strasbourg University Hospitals
UMCG
Martini Hospital Groningen
MUMC
Haga Hospital
MC Groep
Albert Schweitzer Hospital


title: An Open-label, Multicenter Extension Study of Onartuzumab in Patients With Solid Tumors on Study Treatment Previously Enrolled in an F.Hoffmann-la Roche- and/or Genentech- Sponsored Study

summary: This extension study will provide continued onartuzumab and/or parent trial (P-trial) designated control treatments to participants with cancer who were previously enrolled in a company-sponsored onartuzumab P-trial and who derived benefit, as assessed by the responsible investigator, from the therapy administered in the P-trial. The study will also collect safety data with regard to administration of continued onartuzumab therapy.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
South Africa
Serbia
France
Japan
Russian Federation
Spain
Latvia

location_facility: HOSPITAL DE MADRID NORTE SANCHINARRO- CENTRO INTEGRAL ONCOLOGICO CLARA CAMPAL; Servicio de Oncologia

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

location_facility: Clin Hospital Center - Kragujevac; Pulmonary Diseases

location_facility: Azienda Ospedaliero Universitaria San Giovanni Battista Di Torino

location_facility: Rigas Austrumu Kliniska Universitates slimnica, Latvijas Onkologijas centrs

location_facility: University of the Witwatersrand Research

location_facility: National Hospital Organization Shikoku Cancer Center


title: Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety Study With PA101 in Patients With Indolent Systemic Mastocytosis

summary:
This is a randomized, placebo- and active-controlled, 2-period crossover, 2 cohort study in adult patients with indolent systemic mastocytosis (ISM).

The purpose of the study is to determine the efficacy and safety profile of PA101 delivered via eFlow high efficiency nebulizer in patients with ISM who are symptomatic despite using standard treatments.

lead_sponsor: Patara Pharma

location_facility:
University Medical Center of Groningen
Klinikum Darmstadt
Hospital Universitario de Fuenlabrada
Charité - Universitätsmedizin Berlin
University of Salerno
University Medical Center Mainz
Hopital Necker - Enfants Malades
Technical University München


ReferenceCitation: Wang X, Hruska M, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 1: Modeling optimal treatment duration and sustained virologic response rates. J Clin Pharmacol. 2015 Jan;55(1):63-72. doi: 10.1002/jcph.363. Epub 2014 Jul 24.

family: Wang

full_name: Xiaodong Wang

given: Xiaodong

name: Bristol-Myers Squibb Research and Development at Hopewell; New Jersey USA

family: Hruska

full_name: Matthew Hruska

given: Matthew

family: Chan

full_name: Phyllis Chan

given: Phyllis

name: Pfizer Inc.; Cambridge Massachusetts USA

family: Ahmad

full_name: Alaa Ahmad

given: Alaa

family: Freeman

full_name: Jeremy Freeman

given: Jeremy

name: Roche Pharmaceuticals; Greater New York City area New York USA

family: Horga

full_name: Maria Arantxa Horga

given: Maria Arantxa

name: Bristol-Myers Squibb/ZymoGenetics; Seattle Washington USA

family: Hillson

full_name: Jan Hillson

given: Jan

family: Kansra

full_name: Vikram Kansra

given: Vikram

family: Lopez-Talavera

full_name: Juan-Carlos Lopez-Talavera

given: Juan-Carlos

journal_name: The Journal of Clinical Pharmacology

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Hruska M, Wang X, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 2: Exposure-response analyses for efficacy and safety variables. J Clin Pharmacol. 2015 Jan;55(1):73-80. doi: 10.1002/jcph.361. Epub 2014 Jul 24.

name: Bristol-Myers Squibb Research and Development at Hopewell; Hopewell NJ USA

name: Roche Pharmaceuticals; Greater New York City Area NY USA

name: Bristol-Myers Squibb/ZymoGenetics; Seattle WA USA

lead_sponsor: ZymoGenetics

location_country:
Italy
Austria
United States
Germany
France
Poland
Romania
Australia
Puerto Rico
Spain
Canada

acronym: EMERGE

title: Randomized, Controlled Phase 2a/b Study of the Efficacy and Safety of PEG-rIL-29 Administered in Combination With Ribavirin to Treatment-Naive Subjects With Chronic Hepatitis C Virus Infection

summary: Interleukin 29 (IL-29) is a substance that is produced in the body to help fight viral infections. The purpose of this study is to evaluate the safety and antiviral effects of several different doses of PEG-rIL-29 (a man-made form of IL-29) when it is given in combination with daily oral doses of ribavirin (an antiviral drug) to subjects with hepatitis C infection who have received no prior treatment for this disease.


lead_sponsor: Ipsen

location_country:
Brazil
Sweden
Mexico
Netherlands
France
Czechia
Lithuania
Poland
Romania
Belgium
United States
Latvia
Ukraine

acronym: TULIPIA

location_facility: Hospital Universitário Clementino Fraga Filho

location_facility: Massachusetts General Hospital / Neuroendocrine Unit

location_facility: UIM Endocrinología Experimental, Hospital de Especialidades

location_facility: Insituto Nacional de Neurologia y Neurocirugia

location_facility: Academy of Medical Science of Ukraine, Department of Clinical Endocrinology

location_facility: Vilnius University Hospital "Santariskiu Klinikos"

location_facility: Centre Hospitalier Universitaire de Liège

location_facility: Oregon University, Dept. of Endocrinology and Neurosurgery

location_facility: "C.I. Parhon" National Institute of Endocrinology

location_facility: Administration of Medical Services and Rehabilitation of "ARTEM"

location_facility: P. Stradins Clinical University Hospital

location_facility: National Medical University n.a. M.I.Pirogov

location_facility: General University Hospital, Clinic of Internal Medicine,

location_facility: University Hospital Olomouc, Clinic of Internal Medicine

title: Phase II, Open, Randomised, Parallel Group, Noncomparative Multicentre Study to Assess the Efficacy and Safety of Repeated Subcutaneous (S.C.) Administration of Different Doses of BIM 23A760 in Acromegalic Patients

summary: The purpose of this study is to assess the efficacy of repeated subcutaneous (under the skin) injections at different doses of BIM 23A760 on growth hormone and insulin-like growth factor-1 levels in patients with acromegaly after 6 months of treatment.


title: Phase I/Prospective Randomized Phase II Trial Of the Safety and Efficacy of Tamoxifen in Combination With GDC-0032 Compared With Tamoxifen alONe.

acronym: Poseidon

summary:
This study is designed as a phase 1 dose escalation study followed by a randomised phase II study. The study will be performed in three different centres: Addenbrooke & Cambridge university (Cambridge, UK), Netherlands Cancer Institute Amsterdam), and Vall d'Hebron Hospital (Barcelona, Spain).

Three to six patients will be followed for one completed cycle of therapy (28 days) and subsequent enrolment of new cohorts will be based on the safety assessment in that first cycle and the documentation of dose limiting toxicities. To determine the safety and efficacy of tamoxifen in combination with the isoform selective Pi3K inhibitor GDC-0032 compared with tamoxifen alone.

status: Suspended

lead_sponsor: The Netherlands Cancer Institute

location_country:
Spain
United Kingdom
France
Netherlands

location_facility:
Hospital Germans Trias i Pujol
Haaglanden Medisch Centrum
Hospital ICO-Hospitalet (Bellvitge)
Hospital Clínic de Barcelona
Antoni van Leeuwenhoek
Hospital Arnau de Vilanova
MUMC
University of Cambridge
Deventer Ziekenhuis
Vall d'Hebron University Hospital/VHIO
Reinier de Graaf Gasthuis
Hospital Universitari Sant Joan de Reus
Ziekenhuis Groep Twente
Gustave Roussy


ReferenceCitation: Paz-Ares L, Mezger J, Ciuleanu TE, Fischer JR, von Pawel J, Provencio M, Kazarnowicz A, Losonczy G, de Castro G Jr, Szczesna A, Crino L, Reck M, Ramlau R, Ulsperger E, Schumann C, Miziara JE, Lessa ÁE, Dediu M, Bálint B, Depenbrock H, Soldatenkova V, Kurek R, Hirsch FR, Thatcher N, Socinski MA; INSPIRE investigators. Necitumumab plus pemetrexed and cisplatin as first-line therapy in patients with stage IV non-squamous non-small-cell lung cancer (INSPIRE): an open-label, randomised, controlled phase 3 study. Lancet Oncol. 2015 Mar;16(3):328-37. doi: 10.1016/S1470-2045(15)70046-X. Epub 2015 Feb 18.

family: Paz-Ares

full_name: Luis Paz-Ares

given: Luis

family: Mezger

full_name: Jörg Mezger

given: Jörg

family: Ciuleanu

full_name: Tudor E Ciuleanu

given: Tudor E

family: Fischer

full_name: Jürgen R Fischer

given: Jürgen R

family: von Pawel

full_name: Joachim von Pawel

given: Joachim

family: Provencio

full_name: Mariano Provencio

given: Mariano

family: Kazarnowicz

full_name: Andrzej Kazarnowicz

given: Andrzej

family: Losonczy

full_name: György Losonczy

given: György

family: de Castro

full_name: Gilberto de Castro

given: Gilberto

suffix: Jr

family: Szczesna

full_name: Aleksandra Szczesna

given: Aleksandra

family: Crino

full_name: Lucio Crino

given: Lucio

family: Reck

full_name: Martin Reck

given: Martin

family: Ramlau

full_name: Rodryg Ramlau

given: Rodryg

family: Ulsperger

full_name: Ernst Ulsperger

given: Ernst

family: Schumann

full_name: Christian Schumann

given: Christian

family: Miziara

full_name: Jose Elias A Miziara

given: Jose Elias A

family: Lessa

full_name: Álvaro E Lessa

given: Álvaro E

family: Dediu

full_name: Mircea Dediu

given: Mircea

family: Bálint

full_name: Beatrix Bálint

given: Beatrix

family: Depenbrock

full_name: Henrik Depenbrock

given: Henrik

family: Soldatenkova

full_name: Victoria Soldatenkova

given: Victoria

family: Kurek

full_name: Raffael Kurek

given: Raffael

family: Hirsch

full_name: Fred R Hirsch

given: Fred R

family: Thatcher

full_name: Nick Thatcher

given: Nick

family: Socinski

full_name: Mark A Socinski

location_country:
Italy
Austria
Brazil
South Africa
Spain
Germany
Croatia
France
Greece
Poland
Slovakia
Romania
Hungary
Australia
Belgium
Russian Federation
Portugal
United States
United Kingdom
Canada

location_facility:
Imclone Investigational Site
ImClone Investigational Site

title: A Randomized, Multicenter, Open-Label Phase 3 Study of Pemetrexed-Cisplatin Chemotherapy Plus Necitumumab (IMC-11F8) Versus Pemetrexed-Cisplatin Chemotherapy Alone in the First-Line Treatment of Patients With Stage IV Nonsquamous Non-Small Cell Lung Cancer (NSCLC)

summary: The research study is testing the investigational drug necitumumab in the treatment of advanced non-small cell lung cancer. The aim of this study is to determine if necitumumab, given together with a standard chemotherapy combination consisting of cisplatin and pemetrexed will be more effective in improving participant disease than the standard chemotherapy combination alone.


status: Active, not recruiting

lead_sponsor: Janssen Sciences Ireland UC

location_country:
Brazil
United States
South Africa
France
Panama
Thailand
Romania
Argentina
Puerto Rico
Spain
Canada

title: Continued Access to Etravirine (ETR) in Treatment Experienced HIV-1 Infected Subjects

summary: The purpose of this study is to provide etravirine (ETR) through this trial until participants can be switched to locally available ETR-based treatment regimens (that is, commercially available and reimbursed, or accessible through another source [example, access program or government program]), or local standard of care, as appropriate.


title: Mid to Long-term Quality of Life Effects Of imatiNIb Versus DASatinib in Chronic Myeloid Leukemia Patients (LEONIDAS)

summary: The broad goal of this study is to investigate if differences exist (and in which areas and of what magnitude) in QoL and symptoms of patients with CML being treated with first line therapy with dasatinib versus those receiving first line therapy with imatinib. Also, an additional objective is to characterize medication-taking behavior associated with imatinib or dasatinib.

time_perspective: Cross-Sectional

lead_sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country: Italy Germany France Spain

location_facility: Clinica Ematologica ed Unità di Terapie Cellulari Carlo Melzi - A.O. Universitaria

location_facility: Clinica di Ematologia - Policlinico Umberto I- Università degli Studi "Sapienza"

location_facility: U.O.C. Ematologia e Trapianto cellule staminali - Pad Cesalpino- A.O. San Camillo Forlanini

location_facility: Divisione Ematologia - Dipartimento di Medicina Interna -Policlinico Universitario di Messina

location_facility: U.O. Ematologia con trapianto - Azienda Ospedaliero-Universitaria Policlinico di Bari

location_facility: U.O. di Ematologia - Ente Ospedaliero San Giovanni Addolorata

location_facility: U.O.C. Ematologia - A.O. SS Annunziata - P.O.S.G. Moscati

location_facility: Dipartimento di Scienze Cliniche e Biologiche - Ospedale S. Luigi Gonzaga

location_facility: U.O. di Ematologia - Casa Sollievo della Sofferenza

location_facility: Ematologia Policlinico - Università degli Studi di Roma Tor Vergata (PTV)

location_facility: Onco-Ematologia - Ospedale S. Andrea- Vercelli

location_facility: XIX Divisione di Ematologia con trapianto - A.O.R.N. "A. Cardarelli"

location_facility: Istituto di Ematologia "L. e A. Seragnoli" - Università degli Studi di Bologna - Policlinico S. Orsola - Malpighi

location_facility: Clinica Ematologica - Dipartimento di Medicina Interna - IRCCS San Martino - IST

location_facility: Unità Operativa Ematologia e Centro Trapianti - Dipartimento di Oncologia ed Ematologia - AUSL Ospedale di Piacenza

location_facility: Divisione di Ematologia - A.O. Ospedali Riuniti di Reggio Calabria "Bianchi-Melacrino-Morelli"

location_facility: S. C. di Ematologia - Azienda Ospedaliera - SS. Antonio e Biagio e Cesare Arrigo

location_facility: Azienda Ospedaliera - Arcispedale S. Anna Sezione di Ematologia e Fisiopatologia delle Emostasi

location_facility: Dipartimento di Med. Clinica e Sperimentale- Area di Ematologia - Facoltà di Medicina e Chirurgia - Università degli Studi di Napoli "Federico II" -

location_facility: Ematologia e CTMO - A.O.U. Università degli Studi di Parma

location_facility: Department of Hematology, Hospital Universitario de la Princesa

location_facility: Divisione di Ematologia - Policlinico Careggi

location_facility: Divisione clinicizzata di Ematologia - Dipartimento di Scienze Mediche - Osp. Ferrarotto

location_facility: S.C. di Oncoematologia - A.O. "S. Maria"

location_facility: S.C.D.O. Ematologia II - A.O.U.S. San Giovanni Battista di Torino (Molinette)

location_facility: Divisione di Ematologia - Ospedale S.Eugenio

location_facility: U.O.C. Ematologia - A.O. Ospedali Riuniti "Villa Sofia-Cervello"

location_facility: Clinica di Ematologia - Azienda Ospedaliera Regionale di Torrette

location_facility: Ematologia ed Immunologia Clinica - Università degli studi di Padova

location_facility: Department of Hematology and Oncology, Hôpital Mignot, Université Versailles Saint-Quentin-en-Yvelines

location_facility: Divisione di Ematologia con trapianto di midollo - A.O.U. Policlinico "Paolo Giaccone"

location_facility: Universität Heidelberg - III. Medizinische Klinik - Universitätsmedizin Mannheim

location_facility: Department of Onco-Hematology - IRCCS; Centro di Riferimento Oncologico della Basilicata

location_facility: Divisione di Ematologia - Dip. Di Medicina Clinica e Sperimentale & BRMA - Università Piemonte Orientale "Amedeo Avogato"

location_facility: Divisione di Ematologia - Azienda Ospedaliera Ospedali Riuniti "Papardo Piemonte"

location_facility: U.O. Ematologia Brindisi- Ospedale A. Perrino ASL BR

location_facility: U.O. Ematologia 1 - Centro Trapianti di Midollo - Ospedale Maggiore Milano

location_facility: U.O di Ematologia d. U. - Azienda Ospedaliera Universitaria Integrata Verona

location_facility: U.O. Ematologia - Azienda USLL12 Veneziana - Ospedale dell'Angelo e Ospedale civile S. Giovanni e Paolo

location_facility: USD - Centro Trapianti Midollo Osseo Adulti - Cattedra di Ematologia - Azienda Spedali Civili - Brescia

intervention_type: Other


title: Proof of Concept Study of Eurartesim® in Patients With Imported Uncomplicated Plasmodium Vivax Malaria

summary: The aim of the present study is to investigate the efficacy, safety and tolerability of a therapeutic course of Eurartesim® in travellers who contracted malaria due to infection by P. vivax in endemic countries.

ReferenceCitation: Hasugian AR, Purba HL, Kenangalem E, Wuwung RM, Ebsworth EP, Maristela R, Penttinen PM, Laihad F, Anstey NM, Tjitra E, Price RN. Dihydroartemisinin-piperaquine versus artesunate-amodiaquine: superior efficacy and posttreatment prophylaxis against multidrug-resistant Plasmodium falciparum and Plasmodium vivax malaria. Clin Infect Dis. 2007 Apr 15;44(8):1067-74. Epub 2007 Mar 5.

ReferenceCitation: Ratcliff A, Siswantoro H, Kenangalem E, Maristela R, Wuwung RM, Laihad F, Ebsworth EP, Anstey NM, Tjitra E, Price RN. Two fixed-dose artemisinin combinations for drug-resistant falciparum and vivax malaria in Papua, Indonesia: an open-label randomised comparison. Lancet. 2007 Mar 3;369(9563):757-765. doi: 10.1016/S0140-6736(07)60160-3.

ReferenceCitation: Sinclair D, Gogtay N, Brand F, Olliaro P. Artemisinin-based combination therapy for treating uncomplicated Plasmodium vivax malaria. Cochrane Database Syst Rev. 2011 Jul 6;(7):CD008492. doi: 10.1002/14651858.CD008492.pub2. Review. Update in: Cochrane Database Syst Rev. 2013;10:CD008492.

lead_sponsor: Alfasigma S.p.A.

location_country: Italy Switzerland Netherlands France Israel Spain Germany

location_facility: Hôpital St André-CHU, Médecine interne et Maladies tropicales

location_facility: 15. The Center for Geographic Medicine and Tropical Diseases, Department of Medicine C - The Chaim Sheba Medical Center

location_facility: Medical and Diagnostic Service Department, Swiss Tropical and Public Health Institute

location_facility: Dep. Infectious Disease, Section Travel Medicine, Leiden University Medical Centre

location_facility: Clinica di Malattie Infettive e Tropicali, Universitá di Brescia

location_facility: Department of Infectious Diseases & Tropical Medicine, University of Munich

location_facility: Centro di Malattie Tropicali - INMI Spallanzani

location_facility: Azienda Ospedaliera Arcispedale S. Maria Nuova IRCCS - Dip. Medicina Interna e Spec. Mediche

location_facility: Medizinische Klinik mit Schwerpunkt Infektiologie, Charite/Campus Virchow-Klinikum


title: Phase I Study of Plitidepsin (Aplidin®) in Combination With Bortezomib and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma

summary: Study of Plitidepsin (Aplidin®) to determine the recommended dose (RD) of plitidepsin in Combination with Bortezomib and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma.

primary_purpose: Other

lead_sponsor: PharmaMar

location_country: France Spain

location_facility: Hospital Universitario Germans Trias I Pujol

location_facility: Hospital Universitari i Politècnic la Fe


title: A Prospective, Multicenter Clinical Study to Evaluate the Clinical Outcomes of Targeted Radiofrequency Ablation and Vertebral Augmentation to Treat Painful Metastatic Vertebral Body Tumor(s)

acronym: EU-STAR

summary: To evaluate the clinical outcomes of minimally invasive targeted radiofrequency tumor ablation (t-RFA) and kyphoplasty (also known as vertebral augmentation) in painful metastatic lesions in the spine.

lead_sponsor: DFINE Inc.

location_facility:
Nouvel Höpital Civil
Goethe-Universität Frankfurt am Main
Cardarelli Hospital Naples, Italy
Universität zu Köln
Otto-von-Guericke-Universität Magdeburg
Universitätsklinikum Bonn
Ospedale di Treviso Ca' Foncello
Policlinico Tor Vergata


lead_sponsor: PCI Biotech AS

location_country:
Norway
Germany
United Kingdom
France

location_facility: Klinikum der Johann Wolfgang Goethe-Universität

location_facility: Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden

location_facility: Klinikum rechts der Isar, Technische Universität München

title: A Phase I/II Dose Escalation Study to Assess the Safety, Tolerability and Efficacy of Amphinex®-Induced Photochemical Internalisation (PCI) of Gemcitabine in Patients With Advanced Inoperable Cholangiocarcinomas

summary: This is a Phase I Dose Escalation Study in which the safety, tolerability and efficacy of Amphinex®--induced Photochemical Internalisation (PCI) of Gemcitabine followed by Gemcitabine/Cisplatin Chemotherapy will be assessed in patients with advanced inoperable cholangiocarcinomas.


design_allocation: Non-Randomized

lead_sponsor: UCB Pharma

location_country:
Italy
Austria
Switzerland
Mexico
Finland
France
Czechia
Bulgaria
Greece
Romania
Russian Federation
Spain
Turkey
Denmark
United States

title: An Open-Label, Multicenter, Multinational Study of Lacosamide as First Add-On Anti-epileptic Drug (AED) Treatment in Subjects With Partial-Onset Seizures

summary: To evaluate the efficacy and safety of oral Lacosamide as first add on treatment in subjects with uncontrolled partial-onset seizures after prior treatment with a monotherapy Antiepileptic Drug (AED) regimen compared to subjects who have received treatment with at least 2 AEDs.


title: Cardioband Adjustable Annuloplasty System For Transcatheter Repair of Mitral Valve Regurgitation

summary: To evaluate the performance and safety of the Cardioband Adjustable Annuloplasty System for repair of functional mitral regurgitation.

ReferenceCitation: Maisano F, La Canna G, Latib A, Denti P, Taramasso M, Kuck KH, Colombo A, Alfieri O, Guidotti A, Messika-Zeitoun D, Vahanian A. First-in-man transseptal implantation of a "surgical-like" mitral valve annuloplasty device for functional mitral regurgitation. JACC Cardiovasc Interv. 2014 Nov;7(11):1326-8. doi: 10.1016/j.jcin.2014.08.003. Epub 2014 Oct 15.

ReferenceCitation: Maisano F, Taramasso M, Nickenig G, Hammerstingl C, Vahanian A, Messika-Zeitoun D, Baldus S, Huntgeburth M, Alfieri O, Colombo A, La Canna G, Agricola E, Zuber M, Tanner FC, Topilsky Y, Kreidel F, Kuck KH. Cardioband, a transcatheter surgical-like direct mitral valve annuloplasty system: early results of the feasibility trial. Eur Heart J. 2016 Mar 7;37(10):817-25. doi: 10.1093/eurheartj/ehv603. Epub 2015 Nov 18.

ReferenceCitation: Messika-Zeitoun D, Nickenig G, Latib A, Kuck KH, Baldus S, Schueler R, La Canna G, Agricola E, Kreidel F, Huntgeburth M, Zuber M, Verta P, Grayburn P, Vahanian A, Maisano F. Transcatheter mitral valve repair for functional mitral regurgitation using the Cardioband system: 1 year outcomes. Eur Heart J. 2019 Feb 1;40(5):466-472. doi: 10.1093/eurheartj/ehy424.

name: Department of Cardiology, Assistance Publique – Hôpitaux de Paris (AP-HP), Bichat Hospital, Paris, France

name: INSERM U1148, Bichat Hospital, Paris, France

name: Université Paris Diderot, Sorbonne Paris Cité, Paris, France

name: Division of Cardiology, University of Ottawa Heart Institute, Ottawa, Canada

family: Messika-Zeitoun

full_name: David Messika-Zeitoun

given: David

name: Department of Cardiology, Heart Center Bonn, University Hospital Bonn, Bonn, Germany

family: Nickenig

full_name: Georg Nickenig

given: Georg

name: Department of Cardiology, San Raffaele Scientific Institute, Milan, Italy

family: Latib

full_name: Azeem Latib

given: Azeem

name: Department of Cardiology, Asklepios Klinik St. Georg Hospital, Hamburg, Germany

family: Kuck

full_name: Karl-Heinz Kuck

given: Karl-Heinz

name: Department of Cardiology, Heart Center, University of Cologne, Cologne, Germany

family: Baldus

full_name: Stephan Baldus

given: Stephan

family: Schueler

full_name: Robert Schueler

given: Robert

family: La Canna

full_name: Giovanni La Canna

given: Giovanni

family: Agricola

full_name: Eustachio Agricola

given: Eustachio

family: Kreidel

full_name: Felix Kreidel

given: Felix

family: Huntgeburth

full_name: Michael Huntgeburth

given: Michael

name: Valve Clinic, University Heart Center, University Hospital Zürich, Zürich, Switzerland

family: Zuber

full_name: Michel Zuber

given: Michel

family: Verta

full_name: Patrick Verta

given: Patrick

name: Department of Cardiology, Baylor Heart and Vascular Institute, Baylor University Medical Center, Dallas, TX, USA

family: Grayburn

full_name: Paul Grayburn

given: Paul

family: Vahanian

full_name: Alec Vahanian

given: Alec

family: Maisano

full_name: Francesco Maisano

given: Francesco

journal_name: European Heart Journal

evidence: open (via free pdf)

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

ReferenceCitation: Nickenig G, Hammerstingl C, Schueler R, Topilsky Y, Grayburn PA, Vahanian A, Messika-Zeitoun D, Urena Alcazar M, Baldus S, Volker R, Huntgeburth M, Alfieri O, Latib A, La Canna G, Agricola E, Colombo A, Kuck KH, Kreidel F, Frerker C, Tanner FC, Ben-Yehuda O, Maisano F. Transcatheter Mitral Annuloplasty in Chronic Functional Mitral Regurgitation: 6-Month Results With the Cardioband Percutaneous Mitral Repair System. JACC Cardiovasc Interv. 2016 Oct 10;9(19):2039-2047. doi: 10.1016/j.jcin.2016.07.005.

family: Hammerstingl

full_name: Christoph Hammerstingl

given: Christoph

family: Topilsky

full_name: Yan Topilsky

given: Yan

location_country: Italy Switzerland Netherlands France Israel Germany

location_facility: Universitätskliniken zu Köln Klinik III Innere Medizin

location_facility: Azienda Ospedaliero - Universitaria "Policlinico - Vittorio Emanuele"

location_facility: Zentrum für Kardiologie, Universitätsmedizin Mainz

location_facility: Universitätsklinikum Bonn, Medizinische Klinik und Poliklinik II


location_country:
Italy
United States
Mexico
France
Japan
Poland
Hungary
Belgium
China
Germany
Ukraine

title: An Open-Label Study To Determine Safety, Tolerability And Efficacy Of Long -Term Oral Lacosamide (LCM) As Adjunctive Therapy In Children With Epilepsy

summary: SP848 is an open-label study to evaluate long-term safety, tolerability, and efficacy in children with epilepsy treated with Lacosamide (LCM) oral solution (syrup) or LCM tablets as adjunctive therapy.


summary:
Systemic sclerosis is an orphan, multiorgan disease affecting the connective tissue of the skin and several internal organs.

Digital ulcers are frequent and have a major impact on the quality of life in patients with systemic sclerosis. The etiology of digital ulcers is complex and multifactorial and the principal mechanisms underlying the digital ulcers formation are ischemic, mechanic and inflammatory, alone or in combination, on the basis of the systemic sclerosis vasculopathy. Consequently, there are at least three types of DU: (i) those localized at the tips of the fingers and toes, mainly resulting from an ischemic process, (ii) those localized on the dorsal aspect of the fingers where the skin retraction due to fibrosis over bony prominences seems to be the main cause, and (iii) those evolving on a pitting scar or subcutaneous calcinosis due to a combined irritative-inflammatory mechanism. An early therapy to prevent or rapidly heal digital ulcers is today considered a mandatory approach to maintain quality of life and spare the enormous costs due to conventional digital ulcer management.

This observational trial is part of the collaborative project "DeSScipher", one out of five observational trials to decipher the optimal management of systemic sclerosis.

Aim of this observational trial is:

To identify the best treatment combination for prevention of digital ulcers in patients with fulfilment of the new ACR/EULAR SSc criteria or the preliminary VEDOSS criteria for very early diagnosis of systemic sclerosis
To identify the best treatment associated with improved healing of digital ulcers in patients with fulfilment of the new ACR/EULAR SSc criteria

Thus, the observational trial consist of a prevention arm and a healing arm.

ReferenceCitation: Blagojevic J, Bellando-Randone S, Abignano G, Avouac J, Cometi L, Czirják L, Denton CP, Distler O, Frerix M, Guiducci S, Huscher D, Jaeger VK, Lóránd V, Maurer B, Nihtyanova S, Riemekasten G, Siegert E, Tarner IH, Vettori S, Walker UA, Allanore Y, Müller-Ladner U, Del Galdo F, Matucci-Cerinic M; EUSTAR co-workers. Classification, categorization and essential items for digital ulcer evaluation in systemic sclerosis: a DeSScipher/European Scleroderma Trials and Research group (EUSTAR) survey. Arthritis Res Ther. 2019 Jan 24;21(1):35. doi: 10.1186/s13075-019-1822-1.

family: Blagojevic

full_name: J. Blagojevic

given: J.

family: Bellando-Randone

full_name: S. Bellando-Randone

given: S.

family: Abignano

full_name: G. Abignano

given: G.

family: Avouac

full_name: J. Avouac

family: Cometi

full_name: L. Cometi

given: L.

family: Czirják

full_name: L. Czirják

family: Denton

full_name: C. P. Denton

given: C. P.

family: Distler

full_name: O. Distler

given: O.

family: Frerix

full_name: M. Frerix

given: M.

family: Guiducci

full_name: S. Guiducci

family: Huscher

full_name: D. Huscher

given: D.

family: Jaeger

full_name: V. K. Jaeger

given: V. K.

family: Lóránd

full_name: V. Lóránd

given: V.

family: Maurer

full_name: B. Maurer

given: B.

family: Nihtyanova

full_name: S. Nihtyanova

family: Riemekasten

full_name: G. Riemekasten

family: Siegert

full_name: E. Siegert

given: E.

family: Tarner

full_name: I. H. Tarner

given: I. H.

family: Vettori

full_name: S. Vettori

family: Walker

full_name: U. A. Walker

given: U. A.

family: Allanore

full_name: Y. Allanore

given: Y.

family: Müller-Ladner

full_name: U. Müller-Ladner

given: U.

family: Del Galdo

full_name: F. Del Galdo

given: F.

family: Matucci-Cerinic

repository_institution: CERN European Organization for Nuclear Research - Zenodo

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

repository_institution: University College London - UCL Discovery

repository_institution: University of Zurich - Zurich Open Repository and Archive

location_country:
Italy
Switzerland
France
Hungary
Germany
United Kingdom

location_facility: Felix-Platter Spital, University of Basel

location_facility: The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital

location_facility: Royal Free Hospital, University College London

location_facility: Pecsi Tudomanyegyetem - University of Pecs

location_facility: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016

location_facility: Centre for Pediatric Rheumatology, Klinikum Eilbek

location_facility: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology

location_facility: Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie

location_facility: University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine

location_facility: University of Zurich, Department of Rheumatology


title: A Two-part, Single- and Two Arm Randomized, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy (in Part 2 Only) of KRP203 in Patients Undergoing Stem Cell Transplant for Hematological Malignancies

summary: Two part study to evaluate the safety, tolerability, pharmacokinetics, and efficacy (in Part 2 only) of KRP203 in patients undergoing allogeneic hemopoietic stem cell transplant for hematological malignancies

primary_purpose: Prevention

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

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

lead_sponsor: Novartis Pharmaceuticals

location_country: Germany France Switzerland


summary:
Systemic sclerosis is an orphan, multiorgan disease affecting the connective tissue of the skin and all internal organs. Cardiac involvement, mainly characterised by small intramyocardial coronary artery involvement and myocardial fibrosis, can cause the development of impaired diastolic ventricular filling, cardiac blocks and ventricular arrhythmias, and can ensue in congestive heart failure and sudden death. Until now, no drug has been proven to have a therapeutic effect on SSc myocardial disease on an evidence-based level. Short-term trials and retrospective studies have suggested a favourable and protective effect of calcium channel blockers and angiotensin converting enzyme inhibitors in patients with myocardial involvement. However, no data are presently available on the prevention and treatment of severe heart disease.

This observational trial is part of the collaborative project "DeSScipher", one out of five observational trials to decipher the optimal management of systemic sclerosis. Aim of this observational trial is to assess the efficacy and safety of calcium channel blockers and angiotensin converting enzyme inhibitors in asymptomatic SSc patients with cardiac involvement.

lead_sponsor: Gabriele Valentini


title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel, Dose-Ranging Study to Evaluate The Efficacy, Safety, Tolerability, and Pharmacodynamics of NBI-6024 In Adult and Adolescent Patients With New Onset Type 1 Diabetes Mellitus

summary:
This was a study designed to evaluate the efficacy of multiple doses of an investigational drug, NBI-6024, in adult (18 to 35 years of age) and adolescent (10 to 17 years of age) patients with new onset type 1 diabetes mellitus, on endogenous insulin production.

A total of 188 patients were enrolled in the study. The study was divided into three periods: screening, treatment (comprising an induction phase and maintenance phase), and follow-up.

NBI-6024 was generally well tolerated and exhibits a benign safety profile, as there were no significant safety issues with NBI-6024 treatment. In summary, NBI-6024 did not demonstrate statistically significant efficacy compared with placebo.

primary_purpose: Basic Science

ReferenceCitation: Walter M, Philotheou A, Bonnici F, Ziegler AG, Jimenez R; NBI-6024 Study Group. No effect of the altered peptide ligand NBI-6024 on beta-cell residual function and insulin needs in new-onset type 1 diabetes. Diabetes Care. 2009 Nov;32(11):2036-40. doi: 10.2337/dc09-0449. Epub 2009 Aug 18.

family: Walter

full_name: M. Walter

family: Philotheou

full_name: A. Philotheou

given: A.

family: Bonnici

full_name: F. Bonnici

family: Ziegler

full_name: A.-G. Ziegler

family: Jimenez

full_name: R. Jimenez

given: R.

journal_name: Diabetes Care

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via page says license)

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

publisher: American Diabetes Association

publisher_dissemination: American Diabetes Association

publisher_group: American Diabetes Association

publisher_normalized: American Diabetes Association

title: No Effect of the Altered Peptide Ligand NBI-6024 on -Cell Residual Function and Insulin Needs in New-Onset Type 1 Diabetes

lead_sponsor: Neurocrine Biosciences

location_country:
South Africa
United Kingdom
Finland
France
Spain
Germany
Czech Republic
Canada

location_facility: Diabetes Center for Children and Adolescents

location_facility: University Hospital and School of Medicine

location_facility: Center Hospitalier Universitaire de Sherbrooke


title: A 24-Week, Flexible-Dose, Open-Label Extension Study of Lurasidone for the Treatment of Bipolar I Depression

summary: This clinical study is designed to test the hypothesis that lurasidone is effective, tolerable, and safe for the treatment of patients with bipolar I depression

ReferenceCitation: Forester BP, Sajatovic M, Tsai J, Pikalov A, Cucchiaro J, Loebel A. Safety and Effectiveness of Long-Term Treatment with Lurasidone in Older Adults with Bipolar Depression: Post-Hoc Analysis of a 6-Month, Open-Label Study. Am J Geriatr Psychiatry. 2018 Feb;26(2):150-159. doi: 10.1016/j.jagp.2017.08.013. Epub 2017 Oct 10.

family: Forester

full_name: Brent P. Forester

given: Brent P.

family: Sajatovic

full_name: Martha Sajatovic

given: Martha

family: Tsai

full_name: Joyce Tsai

given: Joyce

family: Pikalov

full_name: Andrei Pikalov

given: Andrei

family: Cucchiaro

full_name: Josephine Cucchiaro

given: Josephine

family: Loebel

full_name: Antony Loebel

given: Antony

journal_name: The American Journal of Geriatric Psychiatry

lead_sponsor: Sunovion

location_country:
South Africa
Peru
India
France
Japan
Lithuania
Poland
Slovakia
Romania
Russian Federation
United States
Czech Republic
Canada
Colombia
Ukraine

location_facility: Univerzitna nemocnica Bratislava, Nemocnica Ruzinov Psychiatricka klinika

location_facility: MCB Clinical Research Centers-Colorado Springs

location_facility: Brain Mind Behaviour Neurosciences Research Institute

location_facility: Kaunas Hospital Division Marui Hospital, Public Institution

location_facility: R.K. Yadav Memorial Mental Health & De-Addiction Hospital Psychiatry

location_facility: Artemis Institute for Clinical Research LLC

location_facility: Seskines Outpatient Clinic, Public Institution

location_facility: Nemocnica s poliklinikou Liptovsky Mikulas Psychiatricke oddelenie

location_facility: Seth K M School of P G Medicine & Research

location_facility: LLC International Medical Centre "SOGAZ"

location_facility: Pro mente sana s.r.o. Psychiaricka ambulancia

location_facility: Silutes Mental Health and Psychotherapy Center, JSC

location_facility: Crimean republican Clinical Psychiatric Hospital

location_facility: Russian State Medical University of RosZdrav

location_facility: St.In."Inst. of Neurol., Psych. and Narcol. of the AMSU"

location_facility: Odesa SMU, based on Odessa Reg. Psychiatric Hosp.#1

location_facility: Cosmos Hospitals-Delhi Psychiatry Center-Dept. of Psychiatry

location_facility: Spitalul Clinic de Neuropsihiatrie Craiova

location_facility: PsychoLine s.r.o. Psychiatricka ambulancia

location_facility: Reg. Psych. Hosp.n.a. O.Yuschenko, Dept. #121 VNMI

location_facility: Osaka Institute of Clinical Psychiatry Shin-abuyama Hospital

location_facility: Spitalul Clinic Judetean de Urgenta Cluj

location_facility: Kohnodai Hp., National Center for Global Health and Medicine

location_facility: Zirmunai Mental Health Center, Public Institution

location_facility: Centro de Investigaciones y Proyectos en Neurociencias CIPNA Investigations

location_facility: Spitalul Clinic de Neurologie si Psihiatrie Oradea

location_facility: City Psychiatric Hospital #2 of St. Nikolay Chudotvorets

location_facility: University of South Florida Institute - Tampa

location_facility: Kyiv City Psychoneurological Hospital #2

location_facility: Vijayawada Institute of Mental Health and Neurosciences, Psychiatry

location_facility: Reg. Clin. Psychiatric Hosp. DSMU n.af.M.Gorkiy

location_facility: Centro de Investigaciones del Sistema Nerviosa Limitada-Gr

location_facility: Hopital Caremeau, Service de Psychiatrie A

location_facility: Mahatma Gandhi Institute of Medical Sciences

location_facility: Brij Psychiatry Hospital & Muskaan Research Centre

location_facility: Spitalul Clinic de Urgenta Militar Central

location_facility: Saint Anne, s.r.o., Psychiatricke oddeleni

location_facility: Zapor Reg. Cl. Hosp., Zaporizhzhia SMU, ENT Dept.

location_facility: Moscow Medical Academy name I.M. Sechenov

location_facility: Centro de Investigacion y Atencion para la Salud Mental

location_facility: Spitalul Clinic de Psihaiatrie Prof. Dr. Alexandru Obregia

location_facility: Nemocnica s poliklinikou sv Barbory Roznava a.s. Psychiatricke oddelenie

location_facility: University of California at Irvine Medical Center

location_facility: California Neuropsychopharmacology Clinical Research Instit

location_facility: Lviv Reg.St.Cl.Psych.Hosp.Dept.#20, LNMU n.a.D. Halytskiy Chair of Psychiatry, Psychology and Sexololgy

location_facility: Centrul de Recuperare si Reabilitare Gura Ocnitei

location_facility: Mental Illness Treatment Rehabilitation Foundation (MITR)

location_facility: National Center of Neurology and Psychiatry

location_facility: Bekhterev Scientific Research Psychoneurological Institute

location_facility: Reg. Ci.Ps.H.n.a.O.F.Maltsev, Fem.Ac.Gen.Ps. D.5B,HESIU UM

location_facility: Com. Inst. "Dnipropetrovsk Reg. Cl. Psych. Hosp." DSMA

location_facility: Nemocnica s poliklinikou Prievidza so sidlom v Bojniciach Psychiatricke oddelenie

location_facility: Centrum zdravia R. B.K. s.r.o. Psychiatricka ambulancia

location_facility: Ternopil Regional Municipal Dispensary, Narcology Dept., Ch. of Neurol. Psych. Narcol. & Med. Psychol.


title: Randomized ph. II Study to Explore Efficacy and Feasibility of Upfront Rotations Between SUNitinib and Everolimus vs Sequential Treatment of 1st lIne Sunitinib & 2nd Line EverolimuS Until Progression in Pats Met. Clear Cell Renal Cancer

acronym: SUNRISES

summary: The objective of this study is to assess the progression-free survival, of patients who receive rotations of sunitinib and everolimus versus patients who receive sunitinib as a first line treatment followed by everolimus when progression occurs.

ReferenceCitation: Rodriguez-Vida A, Bamias A, Esteban E, Saez MI, Lopez-Brea M, Castellano D, Caballero C, Gonzalez-Larriba JL, Calvo E, Macia S, Ravaud A, Bellmunt J. Randomised Phase II study comparing alternating cycles of sunitinib and everolimus vs standard sequential administration in first-line metastatic renal carcinoma (SUNRISES study). BJU Int. 2020 Nov;126(5):559-567. doi: 10.1111/bju.15165. Epub 2020 Aug 2.

name: Hospital del Mar-Institut Hospital del Mar d'Investigacions Mèdiques; Barcelona Spain

family: Rodriguez-Vida

full_name: Alejo Rodriguez-Vida

given: Alejo

name: Department of Clinical Therapeutics; Alexandra General Hospital; National and Kapodistrian University of Athens; Athens Greece

family: Bamias

full_name: Aristotelis Bamias

given: Aristotelis

name: Hospital Central de Asturias; Oviedo Spain

family: Esteban

full_name: Emilio Esteban

given: Emilio

name: UGCI of Medical Oncology; Hospitales Regional and Universitario Virgen de la Victoria; Málaga Spain

name: Institute of Biomedical Research (IBIMA); Málaga Spain

family: Saez

full_name: Maria Isabel Saez

given: Maria Isabel

name: Hospital Marqués de Valdecilla; Santander Spain

family: Lopez-Brea

full_name: Marta Lopez-Brea

given: Marta

family: Castellano

full_name: Daniel Castellano

given: Daniel

name: Hospital General Universitario de Valencia; Valencia Spain

family: Caballero

full_name: Cristina Caballero

given: Cristina

name: Hospital Clínico San Carlos; Madrid Spain

family: Gonzalez-Larriba

full_name: Jose Luis Gonzalez-Larriba

given: Jose Luis

name: START Madrid-CIOCC; Centro Integral Oncológico Clara Campal; Madrid Spain

family: Calvo

full_name: Emiliano Calvo

given: Emiliano

family: Macia

full_name: Sonia Macia

given: Sonia

name: Hôpital Saint André; Bordeaux University Hospital; Bordeaux France

family: Ravaud

full_name: Alain Ravaud

given: Alain

name: Beth Israel Deaconess Medical Center and PSMAR_IMIM Research Lab; Harvard Medical School; Boston MA USA

family: Bellmunt

full_name: Joaquim Bellmunt

given: Joaquim

journal_name: BJU International

lead_sponsor: Associació per a la Recerca Oncologica, Spain

location_country: Spain France Greece

location_facility: Hospital Universitario Central de Asturias

location_facility: Hospital Universitario Virgen de la Victoria


title: Post-Market Surveillance Registry for the NeoChord DS1000

summary: To monitor the long-term performance of the CE Marked NeoChord Artificial Chordae Delivery System

lead_sponsor: NeoChord

location_country: Italy Switzerland France Lithuania Germany

location_facility: Vilniaus Universiteto ligonines Santariskiu

location_facility: Bordeaux Heart University Hospital (CHU)


title: POLARIS: A Non-interventional Study in Patients With Diabetic Macular Edema (DME) With Central Involvement to Assess the Effectiveness of Existing Anti-vascular Endothelial Growth Factor (Anti-VEGF) Treatment Regimens

acronym: POLARIS

summary: POLARIS : A non-interventional study in patients with diabetic macular edema (DME) with central involvement to assess the effectiveness of existing anti-vascular endothelial growth factor (anti-VEGF) treatment regimens.

lead_sponsor: Bayer

location_country:
Spain
France
Greece
Slovakia
Russian Federation
Portugal
Germany
United Kingdom


summary: This is a prospective, randomised, open label, controlled pilot study to evaluate the safety and preliminary effectiveness of the ReCell Autologous Cell Harvesting Device (ReCell) for the management of chronic leg ulcers associated with venous insufficiency. Outcome will be compared between study participants receiving standard care (debridement, compression therapy) and participants receiving ReCell in addition to standard care.

lead_sponsor: Avita Medical

location_country:
United Kingdom
France

location_facility: Cambridge University Hospitals - Addenbrooke's Hospital

location_facility: University Hospital of South Manchester - Wythenshawe Hospital

location_facility: The Leeds Teaching Hospitals - James's University Hospital


title: Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.

summary: The purpose of this study is to verify that pediatric patients, especially those who are not old enough to swallow capsules, accept the taste and palatability of a new suspension.

lead_sponsor: Swedish Orphan Biovitrum

location_country:
Germany
United Kingdom
France

location_facility: Evelina Children's Hospital, St Thomas' Hospital


type: Other Grant/Funding Number

title: International Validation and Testing of a Supplementary Questionnaire Module for Assessment of Oral and Dental Health in Cancer; the QLQ-OH17

summary:
Assessment of oral and dental problems is seldom routine in clinical oncology, despite the potential negative impact of these problems on nutritional status, social function and quality of life (QoL). A brief, assessment tool for oral/dental health and related QoL-issues to improve symptom management has been requested. The present study will be conducted on behalf of and with support from the European Organisation for Research and Treatment of Cancer - (EORTC) Quality of Life Group (QLG). The study represents phase IV, the final step, in the development of an international, symptom specific questionnaire module, focusing on oral and dental problems in relation to cancer and its treatment. Phase I-III of this stepwise development process was conducted from 2008 to 2011, as an international collaboration and conducted according to the guidelines for module development set forth by the EORTC QLG. The resulting module, the QLQ-OH17, is now subject to an international field testing and validation study as described in this project description.

The present version of the QLQ-OH17 consists of 17 items conceptualized into four multi-item scales (pain/discomfort, xerostomia, eating and information) and three single items related to use of dentures and future worries. The aim of the present study is to conduct phase IV; an international field study to confirm the psychometric properties of the QLQ-OH17

ReferenceCitation: Hjermstad MJ, Bergenmar M, Bjordal K, Fisher SE, Hofmeister D, Montel S, Nicolatou-Galitis O, Pinto M, Raber-Durlacher J, Singer S, Tomaszewska IM, Tomaszewski KA, Verdonck-de Leeuw I, Yarom N, Winstanley JB, Herlofson BB; EORTC QoL Group. International field testing of the psychometric properties of an EORTC quality of life module for oral health: the EORTC QLQ-OH15. Support Care Cancer. 2016 Sep;24(9):3915-24. doi: 10.1007/s00520-016-3216-0. Epub 2016 Apr 25.

ReferenceCitation: Hjermstad MJ, Bergenmar M, Fisher SE, Montel S, Nicolatou-Galitis O, Raber-Durlacher J, Singer S, Verdonck-de Leeuw I, Weis J, Yarom N, Herlofson BB. The EORTC QLQ-OH17: a supplementary module to the EORTC QLQ-C30 for assessment of oral health and quality of life in cancer patients. Eur J Cancer. 2012 Sep;48(14):2203-11. doi: 10.1016/j.ejca.2012.04.003. Epub 2012 May 8.

family: Hjermstad

full_name: Marianne Jensen Hjermstad

given: Marianne Jensen

family: Bergenmar

full_name: Mia Bergenmar

given: Mia

family: Fisher

full_name: Sheila E. Fisher

given: Sheila E.

family: Montel

full_name: Sébastien Montel

given: Sébastien

family: Nicolatou-Galitis

full_name: Ourania Nicolatou-Galitis

given: Ourania

family: Raber-Durlacher

full_name: Judith Raber-Durlacher

given: Judith

family: Singer

full_name: Susanne Singer

given: Susanne

family: Verdonck-de Leeuw

full_name: Irma Verdonck-de Leeuw

given: Irma

family: Weis

full_name: Joachim Weis

given: Joachim

family: Yarom

full_name: Noam Yarom

given: Noam

family: Herlofson

full_name: Bente B. Herlofson

given: Bente B.

journal_name: European Journal of Cancer

title: The EORTC QLQ-OH17: A supplementary module to the EORTC QLQ-C30 for assessment of oral health and quality of life in cancer patients

ReferenceCitation: Johns SA, Brown LF, Beck-Coon K, Talib TL, Monahan PO, Giesler RB, Tong Y, Wilhelm L, Carpenter JS, Von Ah D, Wagner CD, de Groot M, Schmidt K, Monceski D, Danh M, Alyea JM, Miller KD, Kroenke K. Randomized controlled pilot trial of mindfulness-based stress reduction compared to psychoeducational support for persistently fatigued breast and colorectal cancer survivors. Support Care Cancer. 2016 Oct;24(10):4085-96. doi: 10.1007/s00520-016-3220-4. Epub 2016 May 17.

family: Johns

full_name: Shelley A. Johns

given: Shelley A.

family: Brown

full_name: Linda F. Brown

given: Linda F.

family: Beck-Coon

full_name: Kathleen Beck-Coon

given: Kathleen

family: Talib

full_name: Tasneem L. Talib

given: Tasneem L.

family: Monahan

full_name: Patrick O. Monahan

given: Patrick O.

family: Giesler

full_name: R. Brian Giesler

given: R. Brian

family: Tong

full_name: Yan Tong

given: Yan

family: Wilhelm

full_name: Laura Wilhelm

given: Laura

family: Carpenter

full_name: Janet S. Carpenter

given: Janet S.

family: Von Ah

full_name: Diane Von Ah

repositories_institution:
Indiana University - IUScholarWorks
PubMed Central - Europe PMC

ipd_sharing_description: Phase III and Phase IV reports approved by the EORTC Quality of Life Group Executive Committee. First publication in Eur J Cancer. 2012 Sep;48(14):2203-11. doi: 10.1016/j.ejca.2012.04.003. Second publication accepted for publication in Supp Care Cancer, April 2016

location_country:
Italy
Sweden
Netherlands
France
Poland
Greece
Israel
Norway
Germany
United Kingdom

location_facility: Istituto Nazionale Tumori "Fondazione G. Pascale"- IRCCS,


title: Influence of the Pre-existing Cognitive Status on the Outcome in Patients Treated by Thrombolytic Therapy for Acute Cerebral Ischaemia

acronym: OPHELIE-COG

summary:
At the acute stage of cerebral ischaemia, the only effective drug that increases the proportion of patients who survive without dependency is thrombolytic therapy by intravenous (i.v.) tissue-plasminogen activator (t-PA). This treatment is entered into routine practice with similar results than in trials, in various places of the world including Europe and Japan.

Stroke and dementia are closely related. About one patient in ten has dementia before a first-ever stroke, and more than one in three has dementia after a recurrent stroke. Pre-existing dementia is associated with a worse outcome of stroke, and pre-existing cognitive impairment without dementia is associated with a higher rate of institutionalisation within 3 years. In many patients cognitive impairment is due to the summation of the effects of vascular and Alzheimer lesions of the brain.

More and more patients nowadays who are eligible for rt-PA are already known as demented at admission. A retrospective study conducted in a cohort of patients with dementia who had an ischaemic stroke and were treated by rtPA suggested that there is no increased risk of cerebral bleeding and death as compared with non demented patients. However, pre-existing cognitive impairment is possibly associated with (i) an increased risk of bleeding in patients with cognitive impairment, and (ii) a higher sensitivity to the neurotoxic effect of rt-PA on the brain tissue.

Japanese patients differ from European patients by a higher risk of spontaneous intracranial haemorrhage, and a higher proportion of patients with small-vessel diseases.

The primary objective of the OPHELIE-COG study is to determine whether ischaemic stroke patients who are treated with i.v. rt-PA are more likely to have a poor outcome (defined as a modified Rankin scale 2 to 6 at month 3) in the presence of pre-existing cognitive impairment or dementia. The secondary objectives are to determine whether (i) they have an increased risk of symptomatic intracerebral haemorrhages, (ii) the proportion of patients who have a poor outcome is lower than expected from the placebo group of randomised trials for patients with a similar range of baseline severity, and (iii) the influence of the cognitive state on outcome differs between Japanese and European patients.

lead_sponsor: University Hospital, Lille

location_country: France Japan

location_facility:
Fukuoka-Higashi Medical center
Fukuoka Redcross Hospital
Lille University Hospital
Kawasaki Medical University
Kyushu Rosai Hospital
St. Mary's Hospital
Kyushu University
Kyushu Medical Center
Steel Memorial Yawata Hospital


title: A Phase II, Open-label, Single-arm, Exploratory Pharmacogenomic Study of Single Agent Eribulin (HALAVEN®) as Neoadjuvant Treatment for Operable Stage I-II HER2 Non-overexpressing Breast Cancer.

acronym: NeoEribulin

summary: This is a prospective, non-randomized, open-label, multicenter, single-arm exploratory pharmacogenomic study of single agent eribulin as neoadjuvant therapy in patients with operable Stage III HER2 non-overexpressing breast cancer.

ReferenceCitation: Prat P, Llombart A, de la Peña L, Di Cosimo S, Oliveira M, Ortega V, Rubio I, Muñoz E, Harbeck N, Cortés J. NeoEribulin: A Phase II, non-randomized, open-label, single-arm, multicenter, exploratory pharmacogenomic study of single agent eribulin as neoadjuvant treatment for operable Stage I-II HER2 non-overexpressing breast cancer. Poster session presented at: 35th Annual San Antonio Breast Cancer Symposium (SABCS); 2012 December 4th-8th; San Antonio, Texas, United States.

lead_sponsor: SOLTI Breast Cancer Research Group

location_country: Spain Germany France Portugal

location_facility: Klinikum des Landkreises Deggendorf Frauenklinik Mammazentrum

location_facility: Instituto Portugues de Oncologia de Coimbra Francisco Gentil, EPE

location_facility: Hospital Universitario Virgen de la Arrixaca

location_facility: Instituto Portugues de Oncologia de Porto Francisco Gentil, EPE

location_facility: Hospital Universitari Arnau de Vilanova de Lleida

location_facility: Hospital Clinico Universitario de Valencia

location_facility: Complejo Hospitalario Universitario de Santiago

location_facility: Hospital Universitario Puerta de Hierro de Majadahonda

location_facility: Complejo Hospitalario San Pedro de Alcántara

location_facility: Klinikum Südstadt Rostock, Universitätsfrauenklinik und Poliklinik

location_facility: Hospital Universitario Clínico San Carlos

location_facility: Brustzentrum im Krankenhaus Köln-Holweide Priv. Doz.


title: An Open-label, Multicenter Phase II Extension of Study 28063 (ATAMS) to Obtain Long-term Follow-up Data in Subjects With Relapsing Multiple Sclerosis Treated With Atacicept for up to 5 Years (ATAMS-Extension)

acronym: ATAMS ext

summary:
This study (28851) is a long-term follow-up study of subjects enrolled in ATAMS study 28063 (NCT00642902). The aim of this study is to monitor the safety and tolerability of atacicept administered for up to 5 years to subjects with relapsing multiple sclerosis (RMS).

This extension study consists of two parts. Part A will be double blind and Part B will be open label. During Part A, subjects initially randomized to atacicept will continue to receive the atacicept dose to which they have been randomized in study 28063 (ATAMS) once a week subcutaneously (under the skin). Subjects randomized to placebo in ATAMS will receive atacicept at 150 mg once a week subcutaneously during Part A. Once the results of ATAMS are available and the atacicept dose with the best benefit / risk ratio has been identified, all subjects will be switched to this dose and will continue the extension study open-label (Part B). Throughout the study, subjects and investigators will remain blinded with respect to initial and part A treatment allocation/dose.

ReferenceCitation: Kappos L, Hartung HP, Freedman MS, Boyko A, Radü EW, Mikol DD, Lamarine M, Hyvert Y, Freudensprung U, Plitz T, van Beek J; ATAMS Study Group. Atacicept in multiple sclerosis (ATAMS): a randomised, placebo-controlled, double-blind, phase 2 trial. Lancet Neurol. 2014 Apr;13(4):353-63. doi: 10.1016/S1474-4422(14)70028-6. Epub 2014 Mar 6.

family: Kappos

full_name: Ludwig Kappos

given: Ludwig

family: Hartung

full_name: Hans-Peter Hartung

given: Hans-Peter

family: Freedman

full_name: Mark S Freedman

given: Mark S

family: Boyko

full_name: Alexey Boyko

given: Alexey

family: Radü

full_name: Ernst Wilhelm Radü

given: Ernst Wilhelm

family: Mikol

full_name: Daniel D Mikol

given: Daniel D

family: Lamarine

full_name: Marc Lamarine

given: Marc

family: Hyvert

full_name: Yann Hyvert

given: Yann

family: Freudensprung

full_name: Ulrich Freudensprung

given: Ulrich

family: Plitz

full_name: Thomas Plitz

given: Thomas

family: van Beek

full_name: Johan van Beek

given: Johan

journal_name: The Lancet Neurology

lead_sponsor: EMD Serono

location_country:
United States
Sweden
Switzerland
Lebanon
Netherlands
Germany
New Caledonia
United Kingdom
France
Lithuania
Belgium
Australia
Russian Federation
Spain
Canada
Czech Republic
Ukraine


title: Model 4396 Left Ventricular Lead Study

summary: This study is designed to show that a new lead, which paces the left bottom chamber (left ventricle) of the heart, is safe and effective. Using a lead on the left side of the heart has been shown to potentially improve heart failure symptoms. The shape and size of this new lead may make it a good choice for patients or physicians.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Italy
Austria
United States
Saudi Arabia
France
Australia
Denmark
Canada


lead_sponsor: Ineos Healthcare Limited

location_country:
Brazil
United States
South Africa
Germany
France
Poland
Malta
Australia
New Zealand
Spain
Canada

location_facility: NZOZ Miedzynarodowe Centrum Dializ Poznan Odz. Rawicz

location_facility: Long Island Hypertension and Nephrology PLLC

location_facility: Faculdade de Ciencias Medicas de Sorocaba - Hosp Santa Lucin

location_facility: Hospital Universitario da Univ Federal de Juiz de Fora

location_facility: Western Nephrology & Metabolic Bone Disease PC

location_facility: Arizona Kidney Disease and Hypertension Center

location_facility: Scott and White Memorial Hospital and Clinic

location_facility: Nephrology Educational Services and Research

location_facility: London Health Science Centre - University Campus Site

location_facility: Universidade Federal de Sao Paulo - UNIFESP

location_facility: Nephrology Associates of Northern Virginia

location_facility: Clinical Research Associates of Tidewater

location_facility: University of Texas Health Science Center at San Antonio

location_facility: Kansas Nephrology Research Institute LLC

location_facility: Internal Medicine Kidney and Hypertension Center

location_facility: SC Nephrology & Hypertension Center Inc.

location_facility: Brookdale Physicians Dialysis Associates

title: An Open, Randomized, Controlled, Parallel Group, Phase III Study to Investigate the Safety and Efficacy of Fermagate and Lanthanum Carbonate Together With a Randomized Placebo Controlled Double Blind Fermagate Comparison in Hemodialysis Patients With Hyperphosphatemia

summary:
Magnesium iron hydroxycarbonate is a phosphate binder that absorbs phosphate from food, reducing the amount that the body can absorb.

The purpose of this study is to assess the efficacy of magnesium iron hydroxycarbonate in subjects requiring hemodialysis, compared with a marketed phosphate binder, lanthanum carbonate and placebo.


title: Abiliti™ Treatment in Obese Subjects: A Randomized Post-Market Surveillance Multicenter Study

summary: The main objective of this post-market surveillance study is to compare the subject's weight loss and quality of life, and device safety performance of the abiliti System and the adjustable gastric banding therapies in obese to morbidly obese subjects.

ReferenceCitation: Morales-Conde S, Alarcón Del Agua I, Busetto L, Favretti F, Anselmino M, Rovera GM, Socas-Macias M, Barranco-Moreno A, Province-Azalde R, Torres AJ. Implanted Closed-Loop Gastric Electrical Stimulation (CLGES) System with Sensor-Based Feedback Safely Limits Weight Regain at 24 Months. Obes Surg. 2018 Jun;28(6):1766-1774. doi: 10.1007/s11695-017-3093-8.

family: Morales-Conde

full_name: S. Morales-Conde

given: S.

family: Alarcón del Agua

full_name: I. Alarcón del Agua

given: I.

family: Busetto

full_name: L. Busetto

given: L.

family: Favretti

full_name: F. Favretti

given: F.

family: Anselmino

full_name: M. Anselmino

given: M.

family: Rovera

full_name: G. M. Rovera

given: G. M.

family: Socas-Macias

full_name: M. Socas-Macias

family: Barranco-Moreno

full_name: A. Barranco-Moreno

given: A.

family: Province-Azalde

full_name: R. Province-Azalde

given: R.

family: Torres

full_name: A. J. Torres

given: A. J.

journal_name: Obesity Surgery

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

ReferenceCitation: Busetto L, Torres AJ, Morales-Conde S, Alarcón Del Agua I, Moretto C, Fierabracci P, Rovera G, Segato G, Rubio MA, Favretti F. Impact of the feedback provided by a gastric electrical stimulation system on eating behavior and physical activity levels. Obesity (Silver Spring). 2017 Mar;25(3):514-521. doi: 10.1002/oby.21760. Epub 2017 Feb 6.

name: University of Padua, Department of Medicine; Padua Italy

full_name: Luca Busetto

given: Luca

name: Complutense University of Madrid, Hospital Clinico “San Carlos,” Hospital Universitario Madrid Monteprincipe; Madrid Spain

full_name: Antonio J. Torres

given: Antonio J.

name: Hospital Virgen del Rocio; Seville Spain

full_name: Salvador Morales-Conde

given: Salvador

full_name: Isaias Alarcón del Agua

given: Isaias

family: Moretto

full_name: Carlo Moretto

given: Carlo

family: Fierabracci

full_name: Paola Fierabracci

given: Paola

full_name: Giuseppe Rovera

given: Giuseppe

name: Vicenza Regional Hospital; Vicenza Italy

family: Segato

full_name: Gianni Segato

given: Gianni

family: Rubio

full_name: Miguel A. Rubio

given: Miguel A.

full_name: Franco Favretti

given: Franco

ReferenceCitation: Alarcón Del Agua I, Socas-Macias M, Busetto L, Torres-Garcia A, Barranco-Moreno A, Garcia de Luna PP, Morales-Conde S. Post-implant Analysis of Epidemiologic and Eating Behavior Data Related to Weight Loss Effectiveness in Obese Patients Treated with Gastric Electrical Stimulation. Obes Surg. 2017 Jun;27(6):1573-1580. doi: 10.1007/s11695-016-2495-3.

location_country:
Italy
Switzerland
France
Spain
Germany
United Kingdom

location_facility: Polyclinique de Rillieux, Clinique Lyon-Nord

location_facility: Complutense University of Madrid Hospital Clinico "San Carlos"


ReferenceCitation: Comi G, De Stefano N, Freedman MS, Barkhof F, Uitdehaag BM, de Vos M, Marhardt K, Chen L, Issard D, Kappos L. Subcutaneous interferon β-1a in the treatment of clinically isolated syndromes: 3-year and 5-year results of the phase III dosing frequency-blind multicentre REFLEXION study. J Neurol Neurosurg Psychiatry. 2017 Apr;88(4):285-294. doi: 10.1136/jnnp-2016-314843. Epub 2016 Dec 30.

family: Comi

full_name: Giancarlo Comi

given: Giancarlo

family: De Stefano

full_name: Nicola De Stefano

given: Nicola

family: Freedman

full_name: Mark S Freedman

given: Mark S

family: Barkhof

full_name: Frederik Barkhof

given: Frederik

family: Uitdehaag

full_name: Bernard M J Uitdehaag

given: Bernard M J

family: de Vos

full_name: Marlieke de Vos

given: Marlieke

family: Marhardt

full_name: Kurt Marhardt

given: Kurt

family: Chen

full_name: Liang Chen

given: Liang

family: Issard

full_name: Delphine Issard

given: Delphine

family: Kappos

full_name: Ludwig Kappos

given: Ludwig

journal_name: Journal of Neurology, Neurosurgery & Psychiatry

oa_colors: green hybrid

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: University of Oxford - Oxford Academic Notes & Queries

repository_normalized: UCL Discovery

repositories_institution: University College London - UCL Discovery

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

lead_sponsor: Merck KGaA, Darmstadt, Germany

location_country:
Finland
Greece
Romania
Belgium
Russian Federation
Czech Republic
Morocco
Croatia
France
Poland
Slovakia
Argentina
Canada
Bulgaria
Portugal
Germany
Italy
Austria
Serbia
Lebanon
Israel
Spain
Estonia
Latvia

acronym: REFLEXION

location_facility:
Reserch Site
Research Site

title: Double-blind Extension of the Study 27025 (REFLEX) to Obtain Long-term Follow-up Data in Patients With Clinically Definite MS and Patients With a First Demyelinating Event at High Risk of Converting to MS, Treated With Rebif® New Formulation (REFLEXION)

summary: REFLEXION is a double blind extension of the study 27025 (NCT00404352) (REFLEX). The purpose of the study is to obtain long-term follow-up data in subjects with clinically definite multiple sclerosis (MS) and subjects with a first demyelinating event at high risk of converting to MS, treated with fetal bovine serum [FBS]-free/human serum albumin [HSA]-free formulation of interferon [IFN]-beta-1a (RNF).


title: Prognostic Significance and Longitudinal Assessment of Patient-reported Quality of Life and Symptoms in Myelodysplastic Syndromes. A Large-scale International, Observational Study: PROMYS Study

acronym: PROMYS

summary:
RATIONALE: Gathering information about quality of life, fatigue, and other symptoms from patients with myelodysplastic syndromes may help doctors learn more about the disease and may help plan treatment.

PURPOSE: This clinical trial is studying quality of life and symptoms in patients with newly diagnosed myelodysplastic syndromes.

ReferenceCitation: Efficace F, Gaidano G, Sprangers M, Cottone F, Breccia M, Voso MT, Caocci G, Stauder R, Di Tucci AA, Sanpaolo G, Selleslag D, Angelucci E, Platzbecker U, Mandelli F. Preference for involvement in treatment decisions and request for prognostic information in newly diagnosed patients with higher-risk myelodysplastic syndromes. Ann Oncol. 2014 Feb;25(2):447-54. doi: 10.1093/annonc/mdt557.

ReferenceCitation: Caocci G, Voso MT, Angelucci E, Stauder R, Cottone F, Abel G, Nguyen K, Platzbecker U, Beyne-Rauzy O, Gaidano G, Invernizzi R, Molica S, Criscuolo M, Breccia M, Lübbert M, Sanpaolo G, Buccisano F, Ricco A, Palumbo GA, Niscola P, Zhang H, Fenu S, La Nasa G, Mandelli F, Efficace F. Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes. Leuk Res. 2015 Aug;39(8):859-65. doi: 10.1016/j.leukres.2015.05.012. Epub 2015 Jun 12.

ReferenceCitation: Efficace F, Gaidano G, Breccia M, Criscuolo M, Cottone F, Caocci G, Bowen D, Lübbert M, Angelucci E, Stauder R, Selleslag D, Platzbecker U, Sanpaolo G, Jonasova A, Buccisano F, Specchia G, Palumbo GA, Niscola P, Wan C, Zhang H, Fenu S, Klimek V, Beyne-Rauzy O, Nguyen K, Mandelli F. Prevalence, severity and correlates of fatigue in newly diagnosed patients with myelodysplastic syndromes. Br J Haematol. 2015 Feb;168(3):361-70. doi: 10.1111/bjh.13138. Epub 2014 Oct 1.

ReferenceCitation: Efficace F, Gaidano G, Breccia M, Voso MT, Cottone F, Angelucci E, Caocci G, Stauder R, Selleslag D, Sprangers M, Platzbecker U, Ricco A, Sanpaolo G, Beyne-Rauzy O, Buccisano F, Palumbo GA, Bowen D, Nguyen K, Niscola P, Vignetti M, Mandelli F. Prognostic value of self-reported fatigue on overall survival in patients with myelodysplastic syndromes: a multicentre, prospective, observational, cohort study. Lancet Oncol. 2015 Nov;16(15):1506-1514. doi: 10.1016/S1470-2045(15)00206-5. Epub 2015 Sep 21.

ReferenceCitation: Efficace F, Cottone F, Abel G, Niscola P, Gaidano G, Bonnetain F, Anota A, Caocci G, Cronin A, Fianchi L, Breccia M, Stauder R, Platzbecker U, Palumbo GA, Luppi M, Invernizzi R, Bergamaschi M, Borin L, Di Tucci AA, Zhang H, Sprangers M, Vignetti M, Mandelli F. Patient-reported outcomes enhance the survival prediction of traditional disease risk classifications: An international study in patients with myelodysplastic syndromes. Cancer. 2018 Mar 15;124(6):1251-1259. doi: 10.1002/cncr.31193. Epub 2017 Dec 12.

ReferenceCitation: Efficace F, Cottone F, Oswald LB, Cella D, Patriarca A, Niscola P, Breccia M, Platzbecker U, Palumbo GA, Caocci G, Stauder R, Ricco A, Petranovic D, Caers J, Luppi M, Fianchi L, Frairia C, Capodanno I, Follini E, Sarlo C, Fazi P, Vignetti M. The IPSS-R more accurately captures fatigue severity of newly diagnosed patients with myelodysplastic syndromes compared with the IPSS index. Leukemia. 2020 Sep;34(9):2451-2459. doi: 10.1038/s41375-020-0746-8. Epub 2020 Feb 21.

lead_sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

location_country:
Italy
Austria
Brazil
Germany
Croatia
Czechia
France
Belgium
China
United States
United Kingdom
Taiwan

location_facility: AZ Sint-Jan AV, Department of Hematology

location_facility: Curitiba Unidade de Hematologia - Hemoterapia e Oncologia. Setor de Transplante de Medula Ossea. Complexo Hospital de Clinicas da Universidade Federal do Parana (CHC-UFPR)

location_facility: Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova - Irccs - Sc Ematologia

location_facility: Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST

location_facility: Complesso Ospedaliero S. Giovanni Addolorata

location_facility: Asl Di Piacenza - Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti

location_facility: Ematologia - Dipartimento di Medicina Clinica e Sperimentale

location_facility: Divisione Ematologia - Università Campus Bio-Medico

location_facility: Divisione Ematologia - Azienda Ospedaliera Universitaria "San Martino""

location_facility: U.O. Ematologia e Trapianto di Midollo - Ist. Scientifico Ospedale San Raffaele

location_facility: SOS Dipartimento di Oncoematologia Osp. Rovigo

location_facility: ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia

location_facility: Department of Hematology - Universitatsklinikum Carl Gustav Carus

location_facility: Aou Di Parma - Sc Ematologia E Centro Trapianti Midollo Osseo

location_facility: U.O.A. di Medicina Trasfusionale e DH di Ematologia - ASL TO 4 Ospedale di Ivrea

location_facility: Aou Città Della Salute E Della Scienza - Ospedale S. Giovanni Battista Molinette

location_facility: Saint - Cloud Medecin des CLCC Hematologie Service E Hopital ReneHuguenin - Institut Curie

location_facility: Aou Arcispedale Sant'Anna - Uoc Ematologia E Fisiopatologia Della Coagulazione

location_facility: UO Ematologia - AOU Policlinico di Modena

location_facility: Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza

location_facility: Università Cattolica del Sacro Cuore - Policlinico A. Gemelli

location_facility: Asst Di Monza - Ospedale S. Eugenio - Uo Ematologia E Cta

location_facility: ASL N.8 - Ospedale "A. Businco" - Unità Operativa di Ematologia e Trapianto di Midollo

location_facility: Unità Operativa Ematologia 1 - Università degli Studi di Bari

location_facility: Università degli Studi "La Sapienza" - Dip. Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia

location_facility: Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo

location_facility: Purpan Hospital - Internal Medicine Department

location_facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro

location_facility: Affiliated Hospital of Liaoning University of Traditional Chinese Medicine

location_facility: The University of Texas, MD Anderson Cancer Center

location_facility: Hematology - Clinical Hospital Centre Rijeka - Faculty of Medicine - University of Rijeka

location_facility: Divisione di Ematologia Ospedale "Santa Maria Goretti"

location_facility: Memorial Sloan-Kettering Cancer Center (MSKCC)

location_facility: Asl Di Asti - Ospedali Riuniti - Presidio Ospedaliero Cardinal G. Massaia - Sc Oncologia

location_facility: Università degli Studi - Policlinico di Tor Vergata


title: A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

summary: This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

ReferenceCitation: Martell LA, Cunico RL, Ohh J, Fulkerson W, Furneaux R, Foehr ED. Validation of an LC-MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome. Bioanalysis. 2011 Aug;3(16):1855-66. doi: 10.4155/bio.11.172.

name: BioMarin Pharmaceutical Inc., Novato CA 94949, USA

family: Martell

full_name: Lisa Argento Martell

given: Lisa Argento

name: Bay Bioanalytical Laboratory Inc., Hercules, CA 94547, USA

family: Cunico

full_name: Robert L Cunico

given: Robert L

family: Ohh

full_name: Jayoung Ohh

given: Jayoung

family: Fulkerson

full_name: Wendy Fulkerson

given: Wendy

name: Industrial Research Limited, Lower Hutt 5040, New Zealand

family: Furneaux

full_name: Richard Furneaux

given: Richard

family: Foehr

full_name: Erik D Foehr

given: Erik D

journal_name: Bioanalysis

publisher: Future Science Ltd

publisher_dissemination: Future Science Ltd

publisher_group: Future Science Ltd

publisher_normalized: Future Science Ltd

title: Validation of an LC–MS/MS assay for detecting relevant disaccharides from keratan sulfate as a biomarker for Morquio A syndrome

lead_sponsor: BioMarin Pharmaceutical

location_country:
Italy
Brazil
United States
Netherlands
Germany
France
Argentina
Canada
United Kingdom
Taiwan