title: Safety and Performance of the Motorized Spiral Endoscope (PowerSpiral) in Subjects Indicated for Small-bowel Enteroscopy: A PMCF Registry

acronym: SAMISEN

summary: The objective of the SAMISEN study is to assess the performance and safety of diagnostic and therapeutic procedures with the newly designed Olympus Motorized Spiral Enteroscope (PowerSpiral).

ReferenceCitation: Beyna T, Moreels T, Arvanitakis M, Pioche M, Saurin JC, May A, Knabe M, Agnholt JS, Bjerregaard NC, Puustinen L, Schlag C, Aabakken L, Paulsen V, Schneider M, Neurath MF, Rath T, Devière J, Neuhaus H. Motorized spiral enteroscopy: results of an international multicenter prospective observational clinical study in patients with normal and altered gastrointestinal anatomy. Endoscopy. 2022 Apr 21. doi: 10.1055/a-1831-6215. [Epub ahead of print]

lead_sponsor: Olympus Europe SE & Co. KG

location_country: Denmark Finland France Belgium Norway Germany

location_facility: Hospices Civils de Lyon, Hôpital Edouard Herriot


ipd_sharing: No

status: Completed

primary_purpose: Treatment

intervention_type: Drug

title: An Open-label, Multicenter Rollover Study to Provide Continued Treatment With Anetumab Ravtansine for Participants With Solid Tumors Who Were Enrolled in Previous Bayer-sponsored Studies

study_completion_date_type: Actual

lead_sponsor: Bayer

type: EudraCT Number

source: euctr

study_start_date_type: Actual

study_type: Interventional

ipd_sharing_description:
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

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

enrollment_type: Actual

location_country:
Poland
United States
Italy
France

location_facility: ASST Grande Ospedale Metropolitano Niguarda

location_facility: Szpital Wojewodzki W Szczecinie Zdunowie

summary: The purpose of this study is to enable patients with solid tumors, who received anetumab ravtansine in a Bayer-sponsored clinical trial, to continue treatment after their respective study has been closed. The patients will be observed to collect information on how safe and efficient the drug is.

lead_sponsor_type: industriel

status_simplified: Completed


title: A Phase 3 Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) vs VELCADE, Lenalidomide, and Dexamethasone (VRd) in Subjects With Previously Untreated Multiple Myeloma Who Are Eligible for High-dose Therapy

acronym: Perseus

summary: Background of the study: The combination of daratumumab with VRd is anticipated to further improve response rates in patients and may lead to improved long-term outcomes in newly diagnosed patients with multiple myeloma. Given this potential, and based upon the initial safety and efficacy observed in the ongoing Phase 2 Study MMY2004, as well as continued positive results with daratumumab in various disease settings and combination regimens, this Phase 3 study is designed to demonstrate improved outcomes for patients treated with daratumumab+VRd. The Phase 3 study will utilize the subcutaneous (SC) formulation of daratumumab instead of the IV formulation utilized in the Phase 2 study, which may limit additional toxicity to patients treated with the quadruplet regimen.

status: Active, not recruiting

study_completion_date_type: Anticipated

design_allocation: Randomized

ReferenceType: derived

ReferenceCitation: Swan D, Henderson R, McEllistrim C, Naicker SD, Quinn J, Cahill MR, Mykytiv V, Lenihan E, Mulvaney E, Nolan M, Parker I, Natoni A, Lynch K, Ryan AE, Szegezdi E, Krawczyk J, Murphy P, O'Dwyer M. CyBorD-DARA in Newly Diagnosed Transplant-Eligible Multiple Myeloma: Results from the 16-BCNI-001/CTRIAL-IE 16-02 Study Show High Rates of MRD Negativity at End of Treatment. Clin Lymphoma Myeloma Leuk. 2022 Nov;22(11):847-852. doi: 10.1016/j.clml.2022.07.011. Epub 2022 Jul 21.

lead_sponsor: European Myeloma Network

location_country: Denmark Poland Spain Greece Italy Australia Switzerland France Turkey Belgium Norway Netherlands Czechia

location_facility: Hospital Clinic I Provincial de Barcelona

location_facility: Azienda Ospedaliero-Universitaria Ospedali Riuniti Umberto I - G.M. Lancisi - G. Salesi Di Ancona

location_facility: Uniwersytet Jagiellonski Collegium Medicum

status_simplified: Ongoing


title: A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia

summary: This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.

status: Terminated

ReferenceCitation: White PC. Emerging treatment for congenital adrenal hyperplasia. Curr Opin Endocrinol Diabetes Obes. 2022 Jun 1;29(3):271-276. doi: 10.1097/MED.0000000000000723. Review.

lead_sponsor: Millendo Therapeutics US, Inc.

location_country: Brazil Spain France Israel Czechia

location_facility: Hospital General Universitario Gregorio Marañón

location_facility: University of Ribeirão Preto, Medicine Faculty (Faculdade de Medicina) of Ribeirão Preto

location_facility: Universidade Federal de São Paulo, Escola Paulista de Medicina

location_facility: Hospital das Clínicas da FMUSP - Prédio do Instituto Central

status_simplified: Unknown


title: ImpleMentAll - Towards Evidence-based Tailored Implementation Strategies for eHealth

acronym: ImpleMentAll

summary:
The ImpleMentAll (IMA) project aims to examine the effectiveness of tailored implementation compared to usual implementation of Internet-based Cognitive Behavioural Therapy (iCBT) for patients suffering from common mental disorders in routine practice. Common mental health disorders account for an alarming proportion of the global burden of disease. Being regarded as an evidence-based psychotherapeutic eHealth intervention, Internet- based Cognitive Behavioural Therapy (iCBT), has the potential to answer to this societal challenge by providing an efficacious and efficient treatment from which more people can benefit. ImpleMentAll will develop, apply, and evaluate tailored implementation strategies in the context of on-going eHealth implementation initiatives in the EU and beyond. The objectives are:

To develop a generic Integrated Theory-based Framework for Intervention Tailoring Strategies (the ItFits-toolkit) for data-driven tailored implementation of evidence-based eHealth services.
To demonstrate the impact of the ItFits toolkit on the implementation of eHealth for common mental disorders.
To disseminate the validated toolkit in various healthcare contexts across Europe.

Following a stepped-wedge trial design, the ItFits-toolkit will be introduced in twelve implementation sites in nine countries, and evaluated for its effectiveness in obtaining implementation success. An in-depth process evaluation using a realist evaluation methodology will provide information about the particularities of tailored implementation and the application of the ItFits-toolkit in real implementation work. The resulting ItFits-toolkit will enable data driven evaluation of eHealth implementation projects and its methods, materials, and strategies will provide concrete guidance on tuning implementation interventions to local determinant of practice across a variety of health care systems.

primary_purpose: Health Services Research

ReferenceCitation: Doukani A, Cerga Pashoja A, Fanaj N, Qirjako G, Meksi A, Mustafa S, Vis C, Hug J. Organizational Readiness for Implementing an Internet-Based Cognitive Behavioral Therapy Intervention for Depression Across Community Mental Health Services in Albania and Kosovo: Directed Qualitative Content Analysis. JMIR Form Res. 2021 Nov 1;5(11):e29280. doi: 10.2196/29280.

ReferenceCitation: Bührmann L, Schuurmans J, Ruwaard J, Fleuren M, Etzelmüller A, Piera-Jiménez J, Finch T, Rapley T, Potthoff S, Aouizerate B, Batterham PJ, Calear A, Christensen H, Pedersen CD, Ebert DD, Van der Eycken E, Fanaj N, van Genugten C, Hanssen D, Hegerl U, Hug J, Kleiboer A, Mathiasen K, May C, Mustafa S, Oehler C, Cerga-Pashoja A, Pope C, Qirjako G, Rosmalen J, Sacco Y, Samalin L, Skjøth MM, Tarp K, Titzler I, Zanalda E, Zbukvic I, Smit JH, Riper H, Vis C; ImpleMentAll consortium. Tailored implementation of internet-based cognitive behavioural therapy in the multinational context of the ImpleMentAll project: a study protocol for a stepped wedge cluster randomized trial. Trials. 2020 Oct 28;21(1):893. doi: 10.1186/s13063-020-04686-4.

ipd_sharing: Yes

ipd_sharing_description: IPD will be available on request following a standardised data accession form on relevant participant data. Note this data does not contain clinical outcomes.

lead_sponsor: VU University of Amsterdam

location_country: Denmark Albania Spain Italy Australia Netherlands France Germany Kosovo

location_facility:
MHCPPriz
BDI
FDM
Internet Psychiatrien
CMHTIR
UMCG
GGZ InGeest
BSA
ASLTO3
ANU
Get.On
DF

intervention_type: Other

lead_sponsor_type: academique


title: Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

acronym: AAVance

summary: MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Tardieu M, Zérah M, Husson B, de Bournonville S, Deiva K, Adamsbaum C, Vincent F, Hocquemiller M, Broissand C, Furlan V, Ballabio A, Fraldi A, Crystal RG, Baugnon T, Roujeau T, Heard JM, Danos O. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Hum Gene Ther. 2014 Jun;25(6):506-16. doi: 10.1089/hum.2013.238. Epub 2014 May 5.

ipd_sharing: Undecided

lead_sponsor: LYSOGENE

location_country:
United Kingdom
Netherlands
United States
France
Germany

location_facility: University Medical Center Hamburg-Eppendorf

location_facility: Baylor college of medicine / Texas children's hospital


title: A Multicenter, Open Label, Sponsor Blinded, Randomized, Active Controlled, Parallel Group, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of Murepavadin Given With Ertapenem Versus an Anti-pseudomonal-β-lactam-based Antibiotic in Adult Subjects With Nosocomial Pneumonia Suspected or Confirmed to be Due to Pseudomonas Aeruginosa

acronym: PRISM-UDR

summary: This is a phase 3, multicenter, open-label, sponsor blinded, randomized active-controlled, parallel group to investigate the efficacy, safety and tolerability of intravenous murepavadin given with ertapenem versus an anti-pseudomonal β-lactam based antibiotic in the treatment of nosocomial pneumonia in adult subjects

lead_sponsor: Polyphor Ltd.

location_country:
United States
France
Israel
Czechia

location_facility:
Research site
Research Site


type: Other Identifier

title: Open Label, Two Cohort (With and Without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients With Gaucher Disease Type 1 and Type 3

acronym: ELIKIDS

summary:
Primary Objective:

Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to <18 years old).

Secondary Objective:

Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to <18 years old).

design_allocation: Non-Randomized

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

lead_sponsor: Sanofi

location_country:
Spain
Italy
Russian Federation
United Kingdom
Argentina
Canada
France
Turkey
Japan
Sweden

location_facility:
Investigational Site Number :6430001
Investigational Site Number :6430005
Investigational Site Number :7920004
Investigational Site Number :7920003
Investigational Site Number :7520002
Investigational Site Number :3800001
Investigational Site Number :6430004
Investigational Site Number :7240003
Investigational Site Number :7240002
Investigational Site Number :2500002
Investigational Site Number :1240001
Investigational Site Number :7920002
Investigational Site Number :3920001
Investigational Site Number :3800002
Investigational Site Number :7240001
Investigational Site Number :1240002
Investigational Site Number :1240003
Investigational Site Number :3920002
Investigational Site Number :8260002
Investigational Site Number :0320001
Investigational Site Number :6430002
Investigational Site Number :7520001


title: Natural History Study of CEP290-Related Retinal Degeneration

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

study_type: Observational

time_perspective: Prospective

lead_sponsor: Editas Medicine, Inc.

location_country:
Germany
United States
Netherlands
France

location_facility: Universitaetsklinikum Giessen and Marburg GmbH


title: Zimmer® Maxera™ Acetabular System in Total Hip Arthroplasty. A Multicenter, Prospective, Non-controlled Post Market Clinical Follow-up Study

summary: This study is a multicenter, prospective, non-controlled post market clinical follow-up study. The objectives of this study are to confirm the safety and performance of the commercially available Zimmer® Maxera™ Acetabular System in Total Hip Arthroplasty.

lead_sponsor: Zimmer Biomet

location_country:
Spain
United Kingdom
Australia
Finland
Canada
France
Belgium
Netherlands

location_facility: Royal Liverpool and Broadgreen University Teaching Hospitals


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

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

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

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

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

lead_sponsor: Amgen

location_country:
Spain
Australia
Netherlands
United States
France
Germany

location_facility: Hospital Universitari Germans Trias i Pujol

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


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

acronym: TICON

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

lead_sponsor: Merck KGaA, Darmstadt, Germany

location_country: Denmark Portugal Spain Italy Canada France Norway

location_facility: Hospital Universitario Virgen de las Nieves

location_facility: Fertility Clinic Skive Regional Hospital

location_facility: St. Olavs Hospital Hf, Universitetssykehuset i Trondheim

location_facility: Centro Hospitalar e Universitario de Coimbra

location_facility: Sykehuset Telemark HF Fertilitetsavdeliningen Sor

intervention_type: Device


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

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

status: Unknown status

primary_purpose: Diagnostic

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

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

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

lead_sponsor: Czech Technical University in Prague

location_country:
Italy
Austria
Canada
United States
France
Germany
Czechia

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


title: Phase III Study Assessing the "Best of" Radiotherapy Compared to the "Best of" Surgery (Trans-oral Surgery (TOS)) in Patients With T1-T2, N0-N1 Oropharyngeal, Supraglottic Carcinoma and With T1, N0 Hypopharyngeal Carcinoma

acronym: Best Of

summary:
Oropharyngeal Squamous Cell Carcinoma (OPSCC) arises in the soft palate, tonsils, base of tongue, pharyngeal wall, and the vallecula. Most of the patients with early stage OPSCC are usually cured. Treatment of early stage OPSCC can be successfully achieved with primary surgery including neck dissection, as indicated, or with definitive radiotherapy. The current standard treatment for OPSCC is therefore based on either surgery and/or radiotherapy, both associated with comparable, high tumor control rates but with different side effects profiles and technical constraints.

In order to decrease the potential morbidity of surgery, transoral approaches have been developed within the last decades, including transoral robotic surgery (TORS), transoral laser microsurgery (TLM) or conventional transoral techniques. On the other hand, patients with head and neck cancer treated with IMRT experienced significant improvements in cause specific survival (CSS) compared with patients treated with non-IMRT techniques thus suggesting that IMRT may be beneficial in terms of patient's outcomes and toxicity profile. It is as yet unclear however, which one of the new techniques is superior to the other in terms of function preservation. Given that the functional outcome of most importance is swallowing function, the preservation of swallowing is thus of major importance.

The main objective of the study is to assess and compare the patient-reported swallowing function over the first year after randomization to either IMRT or TOS among patients with early stage OPSCC, SGSCC, and HPSCC.

status: Recruiting

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

location_country:
Poland
Spain
Italy
United Kingdom
Switzerland
France
Belgium
Germany

location_facility: Klinikum Rechts der isar Der Technische Universitaet Muenchen

location_facility: Guy's and St Thomas' NHS Foundation trust - Guy s and St Thomas' NHS - Guy's Hospital

location_facility: Hopitaux Universitaires de Strasbourg - Hautepierre

location_facility: Cardiff and Vale University Health Board - University Hospital of Wales

location_facility: Imperial College Healthcare NHS Trust - Charing Cross Hospital

location_facility: Universitaets Krankenhaus Eppendorf - UKE - University Cancer Center

location_facility: Institut Jules Bordet-Hopital Universitaire ULB

location_facility: Hospital Universitario Central De Asturias

location_facility: Universitaetsklinikum Tuebingen- Crona Kliniken

location_facility: Cambridge University Hospital NHS - Addenbrookes Hospital

location_facility: University Hospitals Bristol NHS Foundation Trust - Bristol Haematology And Oncology Centre

location_facility: South Tees Hospitals NHS Foundation Trust - The James Cook University Hospital

location_facility: UniversitaetsSpital Zurich - Klinik fur Ohren, Hals und Gesichtschirurgie

location_facility: Hull and East Yorkshire Hospitals NHS Trust - Castle Hill Hospital

location_facility: Staedtisches Klinikum Leipzig - Klinikum St Georg

location_facility: Centre Hospitalier Universitaire Vaudois - Lausanne

location_facility: Universitaetklinikum Halle - Martin Luther Universitaet

intervention_type: Radiation


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

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

ReferenceCitation: Liistro F, Weinberg I, Almonacid Popma A, Shishehbor MH, Deckers S, Micari A. Paclitaxel-coated balloons versus percutaneous transluminal angioplasty for infrapopliteal chronic total occlusions: the IN.PACT BTK randomised trial. EuroIntervention. 2022 Apr 1;17(17):e1445-e1454. doi: 10.4244/EIJ-D-21-00444.

lead_sponsor: Medtronic Endovascular

location_country: Italy Greece Switzerland France Belgium

location_facility: Hopital Guillaume et Rene Laennec - Centre Hospitalier Universitaire


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

lead_sponsor: Ultragenyx Pharmaceutical Inc

location_country:
Spain
Italy
United Kingdom
United States
France
Germany

location_facility: IRCCS Fondazione Istituto Neurologico Nazionale C. Mondino

location_facility: Sheffield Children's NHS Foundation Trust

location_facility: Leonard Wolfson Experimental Neurology Centre

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


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

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

time_perspective: Other

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

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

location_facility: National Center of Neurology and Psychiatry


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

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

lead_sponsor: Hoffmann-La Roche

location_country:
Denmark
Spain
Canada
United States
France

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

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

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

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: The Ottawa Hospital Cancer Centre; Oncology


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

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

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

lead_sponsor: Fondazione Italiana Linfomi ONLUS

location_country: Brazil Portugal Italy Austria Ukraine France

location_facility: Ematologia IRCCS Policlinico S. Matteo di Pavia

location_facility: Oncologia Medica A - Centro di Riferimento Oncologico

location_facility: Divisione di Ematologia - Azienda Ospedaliera Bianchi Melacrino Morelli

location_facility: SC. Ematologia. Osp. Riuniti Papardo Piemonte

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

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

location_facility: Clinica Ematologica AO San Gerardo di Monza

location_facility: Dipartimento di Ematologia Ospedale Civile Spirito Santo Pescara

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

location_facility: SC Oncoematologia con autotrapianto AO S. Maria Terni

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

location_facility: UO Ematologia con Trapianto Policlinico Consorziale

location_facility: Divisione di Ematologia, Ospedale San Bortolo

location_facility: Ematologia Ospedale Madonna delle Grazie

location_facility: Dipartimento di Oncoematologia Ospedale San Raffaele

location_facility: Unità Operativa Complessa di Ematologia - AO di Cosenza

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

location_facility: Dipartimento di Oncologia Medica ed Ematologia Istituto Humanitas

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

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

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

location_facility: SC Ematologia Arcispedale Santa Maria Nuova

location_facility: USC Ematologia Ospedali Riuniti di Bergamo

location_facility: National Cancer Institute of Health Ukraine

location_facility: Ematologia e CTMO Ospedale Maggiore di Parma


title: BIO|MASTER.Ilivia Family / Plexa

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

primary_purpose: Other

lead_sponsor: Biotronik SE & Co. KG

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

location_facility: Hospital Universitario Virgen de Macarena

location_facility: Pauls Stradins Clinical University Hospital


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

acronym: COSIMO

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

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

lead_sponsor: Bayer

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


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

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

ReferenceType: result

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

lead_sponsor: Novartis Pharmaceuticals

location_country:
Italy
United States
France
Singapore
Belgium
Japan
Germany

location_facility: Massachusetts General Hospital Massachusetts General Hospital

location_facility: MD Anderson Cancer Center SC - LSZ102X2101


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

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

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

lead_sponsor: Eli Lilly and Company

location_country:
Spain
Italy
Korea, Republic of
United States
France

location_facility: Ospedale Policlinico Giambattista Rossi, Borgo Roma


title: Randomised, Double-blind, Placebo-controlled Study of APD421 (Amisulpride for IV Injection) as Treatment of Established Post-operative Nausea and Vomiting, in Patients Who Have Had Prior Prophylaxis

summary: Double-blind, randomised, parallel-group, placebo-controlled, adaptive, seamless, dose-selecting study to compare the efficacy of APD421 to placebo as treatment of established PONV, in patients who have had prior PONV prophylaxis.

lead_sponsor: Acacia Pharma Ltd

location_country:
Germany
United States
France

location_facility: Wake Forest University School of Medicine


title: A Three Arms Prospective, Randomized Phase II Study to Evaluate the Best Sequential Approach With Combo Immunotherapy (Ipilimumab/Nivolumab) and Combo Target Therapy (LGX818/MEK162) in Patients With Metastatic Melanoma and BRAF Mutation

acronym: SECOMBIT

summary: To evaluate the best sequencing approach with the combination of target agents (LGX818 plus MEK162) and the combination of immunomodulatory antibodies (ipilimumab plus nivolumab) in patients with metastatic melanoma and BRAF V600 mutation.

ReferenceCitation: Ascierto PA, Mandalà M, Ferrucci PF, Guidoboni M, Rutkowski P, Ferraresi V, Arance A, Guida M, Maiello E, Gogas H, Richtig E, Fierro MT, Lebbè C, Helgadottir H, Queirolo P, Spagnolo F, Tucci M, Del Vecchio M, Gonzales Cao M, Minisini AM, De Placido S, Sanmamed MF, Mallardo D, Curvietto M, Melero I, Palmieri G, Grimaldi AM, Giannarelli D, Dummer R, Chiarion Sileni V. Sequencing of Ipilimumab Plus Nivolumab and Encorafenib Plus Binimetinib for Untreated BRAF-Mutated Metastatic Melanoma (SECOMBIT): A Randomized, Three-Arm, Open-Label Phase II Trial. J Clin Oncol. 2022 Sep 1:JCO2102961. doi: 10.1200/JCO.21.02961. [Epub ahead of print]

lead_sponsor: Fondazione Melanoma Onlus

location_country:
Poland
Spain
Italy
Austria
Greece
United Kingdom
Switzerland
Sweden
France
Germany

location_facility: Azienda Ospedaliera Universitaria Integrata di Udine

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

location_facility: IRCCS - Istituto Scientifico Romagnolo per la Cura e lo Studio dei Tumori (I.R.S.T) S.r.l.

location_facility: Maria Sklodowska-Curie Institute - Oncology Center

location_facility: Fondazione I.R.C.C.S. Istituto Nazionale dei Tumori

location_facility: Istituto Dermopatico dell'Immacolata - IDI - IRCCS

location_facility: Azienda Ospedaliero Universitaria Federico II

location_facility: IEO - Istituto Europeo di Oncologia - IRCCS

location_facility: Karl Landsteiner University of Health Sciencies - University Clinic

location_facility: Università degli Studi di Bari Aldo Moro

location_facility: Istituto Nazionale per lo Studio e la Cura dei Tumori "Fondazione Giovanni Pascale"


title: Non-vitamin K Antagonist Oral Anticoagulants in Patients With Atrial High Rate Episodes - An Investigator-driven, Prospective, Randomised, Double-blind, Multi-centre Trial Initiated by the European Society of Cardiology and AFNET

acronym: NOAH

summary: NOAH is an investigator-initiated, prospective, parallel-group, double-blind, randomised, multi-centre trial. The objective of the trial is to demonstrate that oral anticoagulation using the NOAC edoxaban is superior to current therapy to pre-vent stroke, systemic embolism, or cardiovascular death in patients with AHRE and at least two stroke risk factors but without AF. The trial will be conducted in several European countries.

primary_purpose: Prevention

ReferenceCitation: Bertaglia E, Blank B, Blomström-Lundqvist C, Brandes A, Cabanelas N, Dan GA, Dichtl W, Goette A, de Groot JR, Lubinski A, Marijon E, Merkely B, Mont L, Piorkowski C, Sarkozy A, Sulke N, Vardas P, Velchev V, Wichterle D, Kirchhof P. Atrial high-rate episodes: prevalence, stroke risk, implications for management, and clinical gaps in evidence. Europace. 2019 Oct 1;21(10):1459-1467. doi: 10.1093/europace/euz172. Review.

ReferenceCitation: Kirchhof P, Blank BF, Calvert M, Camm AJ, Chlouverakis G, Diener HC, Goette A, Huening A, Lip GYH, Simantirakis E, Vardas P. Probing oral anticoagulation in patients with atrial high rate episodes: Rationale and design of the Non-vitamin K antagonist Oral anticoagulants in patients with Atrial High rate episodes (NOAH-AFNET 6) trial. Am Heart J. 2017 Aug;190:12-18. doi: 10.1016/j.ahj.2017.04.015. Epub 2017 May 3.

lead_sponsor: Atrial Fibrillation Network

location_country:
Poland
Portugal
Romania
Spain
Greece
Austria
Italy
Netherlands
United Kingdom
Ukraine
Sweden
Hungary
France
Belgium
Germany
Bulgaria
Czechia

location_facility:
Several Sites
Several


title: A Phase I, Multicenter, Open-Label Safety, Pharmacokinetic and Preliminary Efficacy Study of Wild-type Sparing EGFR Inhibitor, AC0010MA, in Adult Patients With Previously Treated EGFRmut and Acquired T790M Mutation Non-Small Cell Lung Cancer (NSCLC)

summary: AC0010MA is a new, irreversible, Epidermal Growth Factor Receptor (EGFR) mutation selective Tyrosine Kinase Inhibitor. Aim at local advanced or metastatic non-small cell lung cancer patients with EGFR mutation or T790M drug-resistant mutation. The molecular mechanism: by irreversible combining the EGFR-RTKs ATP binding site of cell, selectively suppress the activities of EGFR tyrosine kinase phosphorylation, block the signal transduction pathway of EGFR and inhibit the function of ras/raf/MAPK downstream, thus block the tumor cell growth by EGFR induction, and promotes apoptosis. AC0010MA Maleate Capsules has three characters: 1. Irreversible binding to EGFR; 2. Effectively suppresses the tumor cell with EGFR mutant while has no suppression to EGFR wild-type cell; 3. Efficient suppress the tumor cell with EGFR T790M drug-resistant mutation.

lead_sponsor: ACEA Therapeutics, Inc.

location_country:
United States
Spain
France

location_facility: City of Hope Comprehensive Cancer Center


title: A Prospective, Multi-Center, Post-Market Surveillance Study to Assess the Clinical Efficacy and Fusion Rates of the Zimmer TM Ardis Interbody Fusion System

summary: This study is a post-market clinical follow-up study to fulfill the post-market surveillance obligations according to Medical Device Directive and MEDDEV 2.12-2_Rev2_January 2012. The data collected from this study will serve the purpose of confirming safety and performance of the TM Ardis implant. The goal of this study is to demonstrate that implant is effective in reducing patient disability.

lead_sponsor: Zimmer Biomet

location_country: Spain Canada Sweden France Belgium Germany

location_facility: Sahlgrenska University Hospital Department of Orthopedics, Spine Divison

location_facility: Fundación Jiménez Díaz - Servicio de Cirugía Ortopédica y Traumatología -Avda. Reyes Católicos,


title: A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

summary: Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. This 3-year, non-interventional, two-part, natural history study is designed to gain insight into total body HO, FOP disease progression, the impact of FOP on subjects' physical functioning, and clinical features and biomarkers that may be useful in the diagnosis and monitoring of disease progression. This natural history study will also provide important information to inform the design of subsequent interventional trials.

ReferenceCitation: Connor JM, Evans DA. Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J Bone Joint Surg Br. 1982;64(1):76-83.

ReferenceCitation: Zhang W, Zhang K, Song L, Pang J, Ma H, Shore EM, Kaplan FS, Wang P. The phenotype and genotype of fibrodysplasia ossificans progressiva in China: a report of 72 cases. Bone. 2013 Dec;57(2):386-91. doi: 10.1016/j.bone.2013.09.002. Epub 2013 Sep 17.

ReferenceCitation: Cohen RB, Hahn GV, Tabas JA, Peeper J, Levitz CL, Sando A, Sando N, Zasloff M, Kaplan FS. The natural history of heterotopic ossification in patients who have fibrodysplasia ossificans progressiva. A study of forty-four patients. J Bone Joint Surg Am. 1993 Feb;75(2):215-9.

ReferenceCitation: Warner SE, Kaplan FS, Pignolo RJ, Smith SE, Hsiao EC, De Cunto C, Di Rocco M, Harnett K, Grogan D, Genant HK. Whole-body Computed Tomography Versus Dual Energy X-ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva. Calcif Tissue Int. 2021 Dec;109(6):615-625. doi: 10.1007/s00223-021-00877-6. Epub 2021 Jul 31.

ReferenceCitation: Kou S, De Cunto C, Baujat G, Wentworth KL, Grogan DR, Brown MA, Di Rocco M, Keen R, Al Mukaddam M, le Quan Sang KH, Masharani U, Kaplan FS, Pignolo RJ, Hsiao EC. Patients with ACVR1(R206H) mutations have an increased prevalence of cardiac conduction abnormalities on electrocardiogram in a natural history study of Fibrodysplasia Ossificans Progressiva. Orphanet J Rare Dis. 2020 Jul 29;15(1):193. doi: 10.1186/s13023-020-01465-x.

ReferenceCitation: Towler OW, Shore EM, Kaplan FS. Skeletal malformations and developmental arthropathy in individuals who have fibrodysplasia ossificans progressiva. Bone. 2020 Jan;130:115116. doi: 10.1016/j.bone.2019.115116. Epub 2019 Oct 23.

ReferenceCitation: Pignolo RJ, Baujat G, Brown MA, De Cunto C, Di Rocco M, Hsiao EC, Keen R, Al Mukaddam M, Sang KLQ, Wilson A, White B, Grogan DR, Kaplan FS. Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes. Orphanet J Rare Dis. 2019 May 3;14(1):98. doi: 10.1186/s13023-019-1068-7. Erratum in: Orphanet J Rare Dis. 2019 May 23;14(1):113.

lead_sponsor: Clementia Pharmaceuticals Inc.

location_country:
United Kingdom
Italy
Australia
Argentina
United States
France

location_facility: University of Pennsylvania, Center for FOP & Related Bone Disorders

location_facility: The Royal National Orthopaedic Hospital, Brockley Hill

location_facility: Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)

location_facility: Hôpital Necker-Enfants Malades, Department of Genetics

location_facility: University of California San Francisco, Division of Endocrinology and Metabolism

location_facility: Hospital Italiano de Buenos Aires, Department of Pediatrics

location_facility: Gaslini Institute, Unit of Rare Diseases, Department of Pediatrics


title: Mitral Valve Repair Clinical Trial (MAVERIC Trial)

acronym: MAVERIC

summary: The objective of the study is to evaluate the safety and performance of the ARTO System in patients with mitral valve regurgitation (MR) associated with congestive heart failure (CHF).

ReferenceCitation: Erglis A, Narbute I, Poupineau M, Hovasse T, Kamzola G, Zvaigzne L, Erglis M, Erglis K, Greene S, Rogers JH. Treatment of Secondary Mitral Regurgitation in Chronic Heart Failure. J Am Coll Cardiol. 2017 Dec 5;70(22):2834-2835. doi: 10.1016/j.jacc.2017.09.1110.

ReferenceCitation: Rogers JH, Thomas M, Morice MC, Narbute I, Zabunova M, Hovasse T, Poupineau M, Rudzitis A, Kamzola G, Zvaigzne L, Greene S, Erglis A. Treatment of Heart Failure With Associated Functional Mitral Regurgitation Using the ARTO System: Initial Results of the First-in-Human MAVERIC Trial (Mitral Valve Repair Clinical Trial). JACC Cardiovasc Interv. 2015 Jul;8(8):1095-1104. doi: 10.1016/j.jcin.2015.04.012. Epub 2015 Jun 24.

lead_sponsor: Mvrx, Inc.

location_country:
United Kingdom
Latvia
Italy
Australia
France

location_facility: Brighton and Sussex University Hospitals

location_facility: Pauls Stradins Clinical University Hospital


intervention_type: Biological

title: A Randomized, Open-Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the Anti-FGF23 Antibody, KRN23, in Pediatric Patients With X-linked Hypophosphatemia (XLH)

ReferenceCitation: Linglart A, Imel EA, Whyte MP, Portale AA, Högler W, Boot AM, Padidela R, Van't Hoff W, Gottesman GS, Chen A, Skrinar A, Scott Roberts M, Carpenter TO. Sustained Efficacy and Safety of Burosumab, a Monoclonal Antibody to FGF23, in Children With X-Linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Feb 17;107(3):813-824. doi: 10.1210/clinem/dgab729.

ReferenceCitation: Mao M, Carpenter TO, Whyte MP, Skrinar A, Chen CY, San Martin J, Rogol AD. Growth Curves for Children with X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2020 Oct 1;105(10). pii: dgaa495. doi: 10.1210/clinem/dgaa495.

ReferenceCitation: Carpenter TO, Whyte MP, Imel EA, Boot AM, Högler W, Linglart A, Padidela R, Van't Hoff W, Mao M, Chen CY, Skrinar A, Kakkis E, San Martin J, Portale AA. Burosumab Therapy in Children with X-Linked Hypophosphatemia. N Engl J Med. 2018 May 24;378(21):1987-1998. doi: 10.1056/NEJMoa1714641.

location_country:
Netherlands
United States
United Kingdom
France

location_facility:
Shriners Hospital for Children
Great Ormond Street Hospital
University of California San Francisco
University Medical Center Groningen
Royal Manchester Hospital
Yale University School of Medicine
Indiana University Hospital
Bicetre Hospital
Birmingham Children's University

summary:
The objectives of the study are to:

Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants
Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile
Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients
Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients
Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function
Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients
Evaluate the long-term safety and efficacy of burosumab


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

lead_sponsor: Shire

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

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

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

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

location_facility: University of Minnesota Department of Pediatrics

location_facility: Hospital Universitario Austral A Unidad de Investigacion

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

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


title: Correlate - Safety and Effectiveness of Regorafenib in Routine Clinical Practice Settings

acronym: Correlate

summary: This study is a prospective observational cohort study. The study will be conducted in routine clinical practice settings. It is planned to enroll 1000 patients with metastatic Colorectal Cancer (mCRC) who have been previously treated with other approved treatments for metastatic disease and for whom a decision has been made by the physician to treat with regorafenib according to local health authority approved label. It is the aim of this observational cohort study to further characterize safety and effectiveness of regorafenib in routine clinical practice setting. Healthcare resource utilization in the routine provision of care is becoming increasingly important from a health economics and outcomes research perspective. Therefore, another aim of this observational cohort study is to capture healthcare resource associated with the management of treatment emergent adverse events in the real world setting. The primary objective of this study is to further characterize safety of regorafenib use in routine clinical practice settings. The secondary objective of this study is to assess the effectiveness of regorafenib in routine clinical practice settings as measured by Overall Survival (OS), Progression Free Survival (PFS) and Disease control rate (DCR). Health Related Quality of Life (HRQoL) Data will be collected in applicable countries.

ReferenceCitation: Yeh KH, Yang TS, Hsu TC, Tzu-Liang Chen W, Chen HH, Teng HW, Lin BW, Kuan FC, Chiang FF, Duann CW, Li YS, Lin MT, Fiala-Buskies S, Ducreux M, Wang JY. Real-world evidence of the safety and effectiveness of regorafenib in Taiwanese patients with metastatic colorectal cancer: CORRELATE Taiwan. J Formos Med Assoc. 2021 Nov;120(11):2023-2031. doi: 10.1016/j.jfma.2020.12.015. Epub 2021 Jan 7.

ReferenceCitation: Ducreux M, Petersen LN, Öhler L, Bergamo F, Metges JP, de Groot JW, Wang JY, García Paredes B, Dochy E, Fiala-Buskies S, Cervantes A, O'Connor JM, Falcone A; CORRELATE Investigators. Safety and effectiveness of regorafenib in patients with metastatic colorectal cancer in routine clinical practice in the prospective, observational CORRELATE study. Eur J Cancer. 2019 Dec;123:146-154. doi: 10.1016/j.ejca.2019.09.015. Epub 2019 Nov 4.

location_country: Denmark Taiwan Luxembourg Spain Italy Austria Argentina Switzerland France Singapore Mexico Netherlands Czechia


title: Safety of Oral Anticoagulation Therapy Withdrawal After Successful Cardiac Ablation in Patients With Atrial Fibrillation and Associated High Risk Factors for Embolic Events (OAT Pilot Study)

acronym: OAT

summary: The objective of this study is to determine the safety of discontinuing oral anticoagulation therapy in high risk patients who have had a successful cardiac ablation and remain AF recurrence free for 3 months post ablation.

lead_sponsor: Biosense Webster, Inc.

location_country:
Germany
United States
Italy
France

location_facility: Texas Cardiac Arrhythmia Research Foundation


title: A Phase Ib/II, Open-label, Multi-center, Dose Escalation Study of MEK162 in Combination With Panitumumab in Adult Patients With Mutant RAS or Wild-type RAS Metastatic Colorectal Cancer

lead_sponsor: Pfizer

ipd_sharing_description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

location_country:
Spain
Italy
Canada
United States
France
Belgium
Netherlands

location_facility: Memorial Sloan Kettering Cancer Center Oncology Dept

location_facility: University of California at Los Angeles Dept of Onc

summary: The primary purpose of the phase Ib is to estimate the MTD/RPD2 and of the phase II is to assess the anti-tumor activity of MEK162 in combination with panitumumab.


title: A Phase I, Multi-center, Non-randomized, Open-label, Dose Escalation Design Study to Characterize Safety, Tolerability, Pharmacokinetics and Maximum Tolerated Dose of BAY 1125976 in Subjects With Advanced Solid Tumors

summary:
This is the first study where BAY1125976 is given to humans. Patients (all comers) will receive the study drug treatment in a dose-escalation scheme (no placebo group) to determine the safety, tolerability and maximum tolerated dose (MTD) of BAY1125976. The relative bioavailability of liquid service formulation and tablets will be determined.

After the MTD is defined breast cancer patients with and without AKT1 mutation will be treated.

The study will also assess the pharmacokinetics, biomarker status, pharmacodynamic parameters and tumor response of BAY1125976.

BAY1125976 will be given daily as single oral application. Treatment will be stopped if the tumor continues to grow, if side effects, which the patient cannot tolerate, occur or if the patient decides to exit treatment.

location_country:
Germany
United States
Switzerland
France


acronym: PENELOPE-B

title: Phase III Study Evaluating Palbociclib (PD-0332991), a Cyclin-Dependent Kinase (CDK) 4/6 Inhibitor in Patients With Hormone-receptor-positive, HER2-normal Primary Breast Cancer With High Relapse Risk After Neoadjuvant Chemotherapy "PENELOPEB"

lead_sponsor: German Breast Group

ReferenceCitation: Mittendorf EA, Jeruss JS, Tucker SL, Kolli A, Newman LA, Gonzalez-Angulo AM, Buchholz TA, Sahin AA, Cormier JN, Buzdar AU, Hortobagyi GN, Hunt KK. Validation of a novel staging system for disease-specific survival in patients with breast cancer treated with neoadjuvant chemotherapy. J Clin Oncol. 2011 May 20;29(15):1956-62. doi: 10.1200/JCO.2010.31.8469. Epub 2011 Apr 11.

ReferenceCitation: Musgrove EA, Caldon CE, Barraclough J, Stone A, Sutherland RL. Cyclin D as a therapeutic target in cancer. Nat Rev Cancer. 2011 Jul 7;11(8):558-72. doi: 10.1038/nrc3090. Review.

ReferenceCitation: ENCODE Project Consortium. An integrated encyclopedia of DNA elements in the human genome. Nature. 2012 Sep 6;489(7414):57-74. doi: 10.1038/nature11247.

ReferenceCitation: Finn RS, Dering J, Conklin D, Kalous O, Cohen DJ, Desai AJ, Ginther C, Atefi M, Chen I, Fowst C, Los G, Slamon DJ. PD 0332991, a selective cyclin D kinase 4/6 inhibitor, preferentially inhibits proliferation of luminal estrogen receptor-positive human breast cancer cell lines in vitro. Breast Cancer Res. 2009;11(5):R77. doi: 10.1186/bcr2419.

ReferenceCitation: Symmans WF, Peintinger F, Hatzis C, Rajan R, Kuerer H, Valero V, Assad L, Poniecka A, Hennessy B, Green M, Buzdar AU, Singletary SE, Hortobagyi GN, Pusztai L. Measurement of residual breast cancer burden to predict survival after neoadjuvant chemotherapy. J Clin Oncol. 2007 Oct 1;25(28):4414-22. Epub 2007 Sep 4.

ReferenceCitation: Mauri D, Pavlidis N, Ioannidis JP. Neoadjuvant versus adjuvant systemic treatment in breast cancer: a meta-analysis. J Natl Cancer Inst. 2005 Feb 2;97(3):188-94.

ReferenceCitation: von Minckwitz G, Untch M, Blohmer JU, Costa SD, Eidtmann H, Fasching PA, Gerber B, Eiermann W, Hilfrich J, Huober J, Jackisch C, Kaufmann M, Konecny GE, Denkert C, Nekljudova V, Mehta K, Loibl S. Definition and impact of pathologic complete response on prognosis after neoadjuvant chemotherapy in various intrinsic breast cancer subtypes. J Clin Oncol. 2012 May 20;30(15):1796-804. doi: 10.1200/JCO.2011.38.8595. Epub 2012 Apr 16.

ReferenceCitation: Loibl S, Marmé F, Martin M, Untch M, Bonnefoi H, Kim SB, Bear H, McCarthy N, Melé Olivé M, Gelmon K, García-Sáenz J, Kelly CM, Reimer T, Toi M, Rugo HS, Denkert C, Gnant M, Makris A, Koehler M, Huang-Bartelett C, Lechuga Frean MJ, Colleoni M, Werutsky G, Seiler S, Burchardi N, Nekljudova V, von Minckwitz G. Palbociclib for Residual High-Risk Invasive HR-Positive and HER2-Negative Early Breast Cancer-The Penelope-B Trial. J Clin Oncol. 2021 May 10;39(14):1518-1530. doi: 10.1200/JCO.20.03639. Epub 2021 Apr 1.

location_country:
Spain
Ireland
Austria
Australia
United Kingdom
Canada
Korea, Republic of
United States
France
Japan
Germany

location_facility: Contact: Japan Breast Cancer Research Group

location_facility: Contact: Austrian Breast & Colorectal Cancer Study Group

location_facility: Contact: Australia and New Zealand Breast Cancer Trials Group

summary: The PENELOPEB study is designed to demonstrate that in the background of standard anti-hormonal therapy palbociclib provides superior invasive disease-free survival (iDFS) compared to placebo in pre- and postmenopausal women with HR-positive/HER2-normal early breast cancer at high risk of relapse after showing less than pathological complete response to neoadjuvant taxane- containing chemotherapy. Considering the high risk of recurrence in patients after neoadjuvant chemotherapy and a high CPS-EG score, palbociclib appears to be an attractive option with a favourable safety profile for these patients.


title: A Randomised, Double-blind, Placebo-controlled, Multi-national, Phase IIIb Study to Assess the Sustained Clinical Effect and Safety of Sublingual Immunotherapy Administered as Birch Pollen Extract Solution at a Dose of 300 IR Once Daily to Patients Suffering From Birch Pollen-induced Rhinoconjunctivitis

summary: The purpose of this 2-year study is to assess the sustained clinical efficacy and safety of 300 IR/day of a sublingual solution of birch pollen allergen extract starting 4 months prior to the birch pollen season and continuing over the birch pollen season compared with placebo for reduction of rhinoconjunctivitis-related symptoms and anti-allergy medication usage.

ReferenceCitation: Worm M, Rak S, de Blay F, Malling HJ, Melac M, Cadic V, Zeldin RK. Sustained efficacy and safety of a 300IR daily dose of a sublingual solution of birch pollen allergen extract in adults with allergic rhinoconjunctivitis: results of a double-blind, placebo-controlled study. Clin Transl Allergy. 2014 Feb 11;4(1):7. doi: 10.1186/2045-7022-4-7.

lead_sponsor: Stallergenes Greer

location_country:
Denmark
Poland
Latvia
Estonia
Finland
Czech Republic
Lithuania
Sweden
France
Germany
Slovakia

location_facility: Centre of Investigations and Treatment of Allergic Diseases

location_facility: SPZOZ Uniwersytecki Szpital Kliniczny Nr 1im.N.Barlickiego w Uniwersystetu Medycznego w Łodzi

location_facility: Allergic Diseases Diagnostics and Treatment Centre

location_facility: Sahlgrenska Universitetssjukhuset - Avd för Lungmedicin och Allergi

location_facility: NHC, Hôpitaux Universitaires de Strasbourg

location_facility: Universitätsmedizin Berlin - Allergie-Centrum-Charité

location_facility: National University Hospital - Allergy Unit 4222


title: A Phase III Single Arm Study to Evaluate the Efficacy, Safety and Local Tolerability of a Subcutaneous 3-month Formulation of Triptorelin Pamoate (11.25 mg) in Patients With Locally Advanced or Metastatic Prostate Cancer

acronym: DKP 3M SC

summary: Assess the efficacy and safety of Triptorelin pamoate 3M formulation (11.25mg) when administered by subcutaneous route.

lead_sponsor: Ipsen

location_country: Poland Latvia France Romania Bulgaria


title: A Multicenter, Open-label, Dose Escalation, Phase I Study of LJM716 Administered Intravenously in Combination With Trastuzumab in Patients With HER2 Overexpressing Metastatic Breast Cancer or Gastric Cancer

summary: This is a multicenter, open-label, dose escalation, phase I study to estimate the Maximum Tolerated Dose (MTD) or a lower Recommended Dose for Expansion (RDE) of LJM716 in combination with trastuzumab in patients with Human Epidermal growth factor Receptor 2 (HER2) overexpressing Metastatic Breast Cancer (MBC) or gastric cancer (MGC). The study consists of a dose escalation part and a dose expansion part. LJM716 will be administered intravenously once weekly unless a less frequent dosing regimen such as every 2 weeks or once every 4 weeks is introduced. Patients will continue on their trastuzumab dosing, administered intravenously once weekly at 2mg/kg. During dose escalation, a minimum of 15 patients are anticipated to be treated in successive cohorts. The dose escalation will continue until the MTD/RDE is declared. The RDE dose selected will either be the MTD or a dose below the MTD based on safety and Pharmacokinetic/Pharmacodynamic (PK/PD) considerations. Following the MTD/RDE declaration, approximately 20 MBC and 20 MGC patients will be enrolled in separate arms in the dose expansion part and treated at the MTD/RDE to further assess the safety, tolerability, and anti-tumor activity of the combination.

location_country:
Taiwan
Spain
Italy
United Kingdom
Korea, Republic of
United States
France
Belgium
Netherlands


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

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

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

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

lead_sponsor: Allergan

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


title: A Multi-centre, Multinational, Cross-sectional, Incident Case-control Study on Factors Associated With the Development of Endometrioma and Deep Infiltrating Endometriosis

acronym: FEELING

summary: This study will aim to identify genetic, medical, dietary and environmental factors associated with the development of endometrioma and/or deep infiltrating endometriosis in order to move towards early diagnosis and implementation of potential preventative measures.

time_perspective: Cross-Sectional

ReferenceCitation: Dai Y, Zhou Y, Zhang X, Xue M, Sun P, Leng J, Chapron C. Factors associated with deep infiltrating endometriosis versus ovarian endometrioma in China: a subgroup analysis from the FEELING study. BMC Womens Health. 2018 Dec 22;18(1):205. doi: 10.1186/s12905-018-0697-7.

ReferenceCitation: Chapron C, Lang JH, Leng JH, Zhou Y, Zhang X, Xue M, Popov A, Romanov V, Maisonobe P, Cabri P. Factors and Regional Differences Associated with Endometriosis: A Multi-Country, Case-Control Study. Adv Ther. 2016 Aug;33(8):1385-407. doi: 10.1007/s12325-016-0366-x. Epub 2016 Jun 24.

location_country:
Russian Federation
France
China

location_facility: Women's Hospital School of Medicine, Zhejiang University

location_facility: Moscow Regional Scientific Research Institute of Obstetrics and Gynaecology

location_facility: The Third Xiangya Hospital of Central South University

location_facility: Research Institute of Maternity and Childhood named after V.N. Gorodkov


title: Prospective, Multicenter, Single Arm Feasibility and Safety Study of the Endologix Fenestrated Stent Graft System for the Endovascular Repair of Juxtarenal/Pararenal (JAA/PAA) Aneurysms

summary: The aim of this study is to evaluate the safety and feasibility of the Endologix Fenestrated Stent Graft System for the endovascular repair of juxtarenal or pararenal aortic aneurysms.

ReferenceCitation: Mertens R, Bergoeing M, Mariné L, Valdés F, Krämer A, Vergara J. Ventana fenestrated stent-graft system for endovascular repair of juxtarenal aortic aneurysms. J Endovasc Ther. 2012 Apr;19(2):173-8. doi: 10.1583/11-3706.1.

lead_sponsor: Endologix

location_country:
United Kingdom
New Zealand
United States
Chile
France
Netherlands

location_facility:
Auckland City Hospital
Rijnstate Hospital
Hospital Universidad Catolica
Bureau de Recherche Clinique
Cleveland Clinic Foundation
St. George's Vascular Institute
University of Indiana
UCLA


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

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

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

lead_sponsor: Alexion Pharmaceuticals

location_country:
United States
France
United Kingdom
Czechia


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

acronym: TOTAL

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

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

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

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

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

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

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

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

ReferenceCitation: Jani J, Keller RL, Benachi A, Nicolaides KH, Favre R, Gratacos E, Laudy J, Eisenberg V, Eggink A, Vaast P, Deprest J; Antenatal-CDH-Registry Group. Prenatal prediction of survival in isolated left-sided diaphragmatic hernia. Ultrasound Obstet Gynecol. 2006 Jan;27(1):18-22. doi: 10.1002/uog.2688.

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

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

ReferenceCitation: Deprest J, Flake A. How should fetal surgery for congenital diaphragmatic hernia be implemented in the post-TOTAL trial era: A discussion. Prenat Diagn. 2022 Mar;42(3):301-309. doi: 10.1002/pd.6091. Epub 2022 Jan 22. Review.

ReferenceCitation: Van Calster B, Benachi A, Nicolaides KH, Gratacos E, Berg C, Persico N, Gardener GJ, Belfort M, Ville Y, Ryan G, Johnson A, Sago H, Kosinski P, Bagolan P, Van Mieghem T, DeKoninck PLJ, Russo FM, Hooper SB, Deprest JA. The randomized Tracheal Occlusion To Accelerate Lung growth (TOTAL)-trials on fetal surgery for congenital diaphragmatic hernia: reanalysis using pooled data. Am J Obstet Gynecol. 2022 Apr;226(4):560.e1-560.e24. doi: 10.1016/j.ajog.2021.11.1351. Epub 2021 Nov 19.

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

lead_sponsor: University Hospital, Gasthuisberg

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

location_facility: University of Texas Health Science Center

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

location_facility: National Center for Child Health and Development


acronym: GLOW

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

lead_sponsor: AstraZeneca

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

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


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

acronym: GARDIAN

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

lead_sponsor: Bayer

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


summary: This study will evaluate the biodegradation of the brimonidine tartrate posterior segment drug delivery system.

location_country:
Portugal
United Kingdom
Italy
Australia
Czech Republic
Korea, Republic of
United States
France
India
Israel
Philippines
Germany


acronym: SHIELD

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

lead_sponsor: Novartis Pharmaceuticals

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

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

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


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

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

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

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

location_country:
Hong Kong
Spain
Australia
United States
France
Belgium


title: Treatment of Patients With Acute Sinusitis in Daily Practice

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

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

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


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

acronym: EUS-EG

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

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

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

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

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

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

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

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

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

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

lead_sponsor: University of Medicine and Pharmacy Craiova

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

location_facility: HELIOS Klinikum Wuppertal, University of Witten/Herdecke


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

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


title: A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System (BVS EECSS) in the Treatment of Patients With de Novo Native Coronary Artery Lesions.

acronym: ABSORB B

summary:
The purpose of this study is to assess the safety and performance of the BVS Everolimus Eluting Coronary Stent System (EECSS) in the treatment of patients with a maximum of two de novo native coronary artery lesions located in two different major epicardial vessels.

Currently in development at Abbott Vascular. Not available for sale in the United States.

ReferenceCitation: Zeng Y, Cavalcante R, Collet C, Tenekecioglu E, Sotomi Y, Miyazaki Y, Katagiri Y, Asano T, Abdelghani M, Nie S, Bourantas CV, Bruining N, Onuma Y, Serruys PW. Coronary calcification as a mechanism of plaque/media shrinkage in vessels treated with bioresorbable vascular scaffold: A multimodality intracoronary imaging study. Atherosclerosis. 2018 Feb;269:6-13. doi: 10.1016/j.atherosclerosis.2017.11.002. Epub 2017 Dec 2.

ReferenceCitation: Onuma Y, Grundeken MJ, Nakatani S, Asano T, Sotomi Y, Foin N, Ng J, Okamura T, Wykrzykowska JJ, de Winter RJ, van Geuns RJ, Koolen J, Christiansen EH, Whitbourn R, McClean D, Smits P, Windecker S, Ormiston JA, Serruys PW. Serial 5-Year Evaluation of Side Branches Jailed by Bioresorbable Vascular Scaffolds Using 3-Dimensional Optical Coherence Tomography: Insights From the ABSORB Cohort B Trial (A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System in the Treatment of Patients With De Novo Native Coronary Artery Lesions). Circ Cardiovasc Interv. 2017 Sep;10(9). pii: e004393. doi: 10.1161/CIRCINTERVENTIONS.116.004393. Erratum in: Circ Cardiovasc Interv. 2018 Jan;11(1):e000031. Christiansen, Evald [corrected to Christiansen, Evald H].

ReferenceCitation: Zeng Y, Tateishi H, Cavalcante R, Tenekecioglu E, Suwannasom P, Sotomi Y, Collet C, Nie S, Jonker H, Dijkstra J, Radu MD, Räber L, McClean DR, van Geuns RJ, Christiansen EH, Fahrni T, Koolen J, Onuma Y, Bruining N, Serruys PW. Serial Assessment of Tissue Precursors and Progression of Coronary Calcification Analyzed by Fusion of IVUS and OCT: 5-Year Follow-Up of Scaffolded and Nonscaffolded Arteries. JACC Cardiovasc Imaging. 2017 Oct;10(10 Pt A):1151-1161. doi: 10.1016/j.jcmg.2016.11.016. Epub 2017 Mar 15.

ReferenceCitation: Onuma Y, Collet C, van Geuns RJ, de Bruyne B, Christiansen E, Koolen J, Smits P, Chevalier B, McClean D, Dudek D, Windecker S, Meredith I, Nieman K, Veldhof S, Ormiston J, Serruys PW; ABSORB Investigators. Long-term serial non-invasive multislice computed tomography angiography with functional evaluation after coronary implantation of a bioresorbable everolimus-eluting scaffold: the ABSORB cohort B MSCT substudy. Eur Heart J Cardiovasc Imaging. 2017 May 1;18(8):870-879. doi: 10.1093/ehjci/jex022.

ReferenceCitation: Serruys PW, Ormiston J, van Geuns RJ, de Bruyne B, Dudek D, Christiansen E, Chevalier B, Smits P, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Wasungu L, Ediebah D, Veldhof S, Onuma Y. A Polylactide Bioresorbable Scaffold Eluting Everolimus for Treatment of Coronary Stenosis: 5-Year Follow-Up. J Am Coll Cardiol. 2016 Feb 23;67(7):766-76. doi: 10.1016/j.jacc.2015.11.060.

ReferenceCitation: Ishibashi Y, Nakatani S, Sotomi Y, Suwannasom P, Grundeken MJ, Garcia-Garcia HM, Bartorelli AL, Whitbourn R, Chevalier B, Abizaid A, Ormiston JA, Rapoza RJ, Veldhof S, Onuma Y, Serruys PW. Relation Between Bioresorbable Scaffold Sizing Using QCA-Dmax and Clinical Outcomes at 1 Year in 1,232 Patients From 3 Study Cohorts (ABSORB Cohort B, ABSORB EXTEND, and ABSORB II). JACC Cardiovasc Interv. 2015 Nov;8(13):1715-26. doi: 10.1016/j.jcin.2015.07.026.

ReferenceCitation: Karanasos A, Garcia-Garcia HM, van Geuns RJ, Regar E. Fate of side-branch jailing and a malapposed platinum marker after resorption of an everolimus-eluting bioresorbable vascular scaffold: serial optical coherence tomography observations. JACC Cardiovasc Interv. 2015 Mar;8(3):e53-e54. doi: 10.1016/j.jcin.2014.10.020.

ReferenceCitation: Onuma Y, Serruys PW, Muramatsu T, Nakatani S, van Geuns RJ, de Bruyne B, Dudek D, Christiansen E, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Garcia-Garcia HM, Veldhof S, Rapoza R, Ormiston JA. Incidence and imaging outcomes of acute scaffold disruption and late structural discontinuity after implantation of the absorb Everolimus-Eluting fully bioresorbable vascular scaffold: optical coherence tomography assessment in the ABSORB cohort B Trial (A Clinical Evaluation of the Bioabsorbable Everolimus Eluting Coronary Stent System in the Treatment of Patients With De Novo Native Coronary Artery Lesions). JACC Cardiovasc Interv. 2014 Dec;7(12):1400-11. doi: 10.1016/j.jcin.2014.06.016.

ReferenceCitation: Zhang YJ, Iqbal J, Nakatani S, Bourantas CV, Campos CM, Ishibashi Y, Cho YK, Veldhof S, Wang J, Onuma Y, Garcia-Garcia HM, Dudek D, van Geuns RJ, Serruys PW; ABSORB Cohort B Study Investigators. Scaffold and edge vascular response following implantation of everolimus-eluting bioresorbable vascular scaffold: a 3-year serial optical coherence tomography study. JACC Cardiovasc Interv. 2014 Dec;7(12):1361-9. doi: 10.1016/j.jcin.2014.06.025. Epub 2014 Nov 12.

ReferenceCitation: Muramatsu T, Onuma Y, van Geuns RJ, Chevalier B, Patel TM, Seth A, Diletti R, García-García HM, Dorange CC, Veldhof S, Cheong WF, Ozaki Y, Whitbourn R, Bartorelli A, Stone GW, Abizaid A, Serruys PW; ABSORB Cohort B Investigators; ABSORB EXTEND Investigators; SPIRIT FIRST Investigators; SPIRIT II Investigators; SPIRIT III Investigators; SPIRIT IV Investigators. 1-year clinical outcomes of diabetic patients treated with everolimus-eluting bioresorbable vascular scaffolds: a pooled analysis of the ABSORB and the SPIRIT trials. JACC Cardiovasc Interv. 2014 May;7(5):482-93. doi: 10.1016/j.jcin.2014.01.155. Epub 2014 Apr 16.

ReferenceCitation: Serruys PW, Onuma Y, Garcia-Garcia HM, Muramatsu T, van Geuns RJ, de Bruyne B, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Rapoza R, Ormiston JA. Dynamics of vessel wall changes following the implantation of the absorb everolimus-eluting bioresorbable vascular scaffold: a multi-imaging modality study at 6, 12, 24 and 36 months. EuroIntervention. 2014 Mar 20;9(11):1271-84. doi: 10.4244/EIJV9I11A217.

ReferenceCitation: Ormiston JA, Serruys PW, Onuma Y, van Geuns RJ, de Bruyne B, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Rapoza R, Garcia-Garcia HM. First serial assessment at 6 months and 2 years of the second generation of absorb everolimus-eluting bioresorbable vascular scaffold: a multi-imaging modality study. Circ Cardiovasc Interv. 2012 Oct;5(5):620-32. doi: 10.1161/CIRCINTERVENTIONS.112.971549. Epub 2012 Oct 9.

ReferenceCitation: Gutiérrez-Chico JL, Gijsen F, Regar E, Wentzel J, de Bruyne B, Thuesen L, Ormiston J, McClean DR, Windecker S, Chevalier B, Dudek D, Whitbourn R, Brugaletta S, Onuma Y, Serruys PW. Differences in neointimal thickness between the adluminal and the abluminal sides of malapposed and side-branch struts in a polylactide bioresorbable scaffold: evidence in vivo about the abluminal healing process. JACC Cardiovasc Interv. 2012 Apr;5(4):428-35. doi: 10.1016/j.jcin.2011.12.015.

ReferenceCitation: Brugaletta S, Radu MD, Garcia-Garcia HM, Heo JH, Farooq V, Girasis C, van Geuns RJ, Thuesen L, McClean D, Chevalier B, Windecker S, Koolen J, Rapoza R, Miquel-Hebert K, Ormiston J, Serruys PW. Circumferential evaluation of the neointima by optical coherence tomography after ABSORB bioresorbable vascular scaffold implantation: can the scaffold cap the plaque? Atherosclerosis. 2012 Mar;221(1):106-12. doi: 10.1016/j.atherosclerosis.2011.12.008. Epub 2011 Dec 13.

ReferenceCitation: Gutiérrez-Chico JL, Radu MD, Diletti R, Sheehy A, Kossuth MB, Oberhauser JP, Glauser T, Harrington J, Rapoza RJ, Onuma Y, Serruys PW. Spatial distribution and temporal evolution of scattering centers by optical coherence tomography in the poly(L-lactide) backbone of a bioresorbable vascular scaffold. Circ J. 2012;76(2):342-50. Epub 2011 Nov 19.

ReferenceCitation: Serruys PW, Onuma Y, Dudek D, Smits PC, Koolen J, Chevalier B, de Bruyne B, Thuesen L, McClean D, van Geuns RJ, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Hebert KM, Sudhir K, Garcia-Garcia HM, Ormiston JA. Evaluation of the second generation of a bioresorbable everolimus-eluting vascular scaffold for the treatment of de novo coronary artery stenosis: 12-month clinical and imaging outcomes. J Am Coll Cardiol. 2011 Oct 4;58(15):1578-88. doi: 10.1016/j.jacc.2011.05.050.

ReferenceCitation: Gomez-Lara J, Radu M, Brugaletta S, Farooq V, Diletti R, Onuma Y, Windecker S, Thuesen L, McClean D, Koolen J, Whitbourn R, Dudek D, Smits PC, Regar E, Veldhof S, Rapoza R, Ormiston JA, Garcia-Garcia HM, Serruys PW. Serial analysis of the malapposed and uncovered struts of the new generation of everolimus-eluting bioresorbable scaffold with optical coherence tomography. JACC Cardiovasc Interv. 2011 Sep;4(9):992-1001. doi: 10.1016/j.jcin.2011.03.020.

ReferenceCitation: Gomez-Lara J, Brugaletta S, Farooq V, van Geuns RJ, De Bruyne B, Windecker S, McClean D, Thuesen L, Dudek D, Koolen J, Whitbourn R, Smits PC, Chevalier B, Morel MA, Dorange C, Veldhof S, Rapoza R, Garcia-Garcia HM, Ormiston JA, Serruys PW. Angiographic geometric changes of the lumen arterial wall after bioresorbable vascular scaffolds and metallic platform stents at 1-year follow-up. JACC Cardiovasc Interv. 2011 Jul;4(7):789-99. doi: 10.1016/j.jcin.2011.04.009.

ReferenceCitation: Gutiérrez-Chico JL, Serruys PW, Girasis C, Garg S, Onuma Y, Brugaletta S, García-García H, van Es GA, Regar E. Quantitative multi-modality imaging analysis of a fully bioresorbable stent: a head-to-head comparison between QCA, IVUS and OCT. Int J Cardiovasc Imaging. 2012 Mar;28(3):467-78. doi: 10.1007/s10554-011-9829-y. Epub 2011 Feb 26.

ReferenceCitation: Serruys PW, Onuma Y, Ormiston JA, de Bruyne B, Regar E, Dudek D, Thuesen L, Smits PC, Chevalier B, McClean D, Koolen J, Windecker S, Whitbourn R, Meredith I, Dorange C, Veldhof S, Miquel-Hebert K, Rapoza R, García-García HM. Evaluation of the second generation of a bioresorbable everolimus drug-eluting vascular scaffold for treatment of de novo coronary artery stenosis: six-month clinical and imaging outcomes. Circulation. 2010 Nov 30;122(22):2301-12. doi: 10.1161/CIRCULATIONAHA.110.970772. Epub 2010 Nov 15.

ReferenceCitation: Gomez-Lara J, Garcia-Garcia HM, Onuma Y, Garg S, Regar E, De Bruyne B, Windecker S, McClean D, Thuesen L, Dudek D, Koolen J, Whitbourn R, Smits PC, Chevalier B, Dorange C, Veldhof S, Morel MA, de Vries T, Ormiston JA, Serruys PW. A comparison of the conformability of everolimus-eluting bioresorbable vascular scaffolds to metal platform coronary stents. JACC Cardiovasc Interv. 2010 Nov;3(11):1190-8. doi: 10.1016/j.jcin.2010.07.016.

ReferenceCitation: Okamura T, Onuma Y, García-García HM, Regar E, Wykrzykowska JJ, Koolen J, Thuesen L, Windecker S, Whitbourn R, McClean DR, Ormiston JA, Serruys PW; ABSORB Cohort B Investigators. 3-Dimensional optical coherence tomography assessment of jailed side branches by bioresorbable vascular scaffolds: a proposal for classification. JACC Cardiovasc Interv. 2010 Aug;3(8):836-44. doi: 10.1016/j.jcin.2010.05.011.

lead_sponsor: Abbott Medical Devices

location_country:
Denmark
Poland
Australia
New Zealand
Switzerland
France
Belgium
Netherlands

location_facility:
Onze-Lieve VrouweZiekenhuis
St. Vincent's Hospital
Institut Hospitalier Jacques Cartier
Maasstad Ziekenhuis
Auckland City Hospital
Jagiellonian University
Catharina ZH Eindhoven
Skejby Sygehus
Monash Heart
Inselspital Bern, Kardiologie
Erasmus Medical Center
Christchurch Hospital


title: Clofarabine in Combination With a Standard Remission Induction Regimen (AraC and Idarubicin) in Patients 18-60 Years Old With Previously Untreated Intermediate and Bad Risk Acute Myelogenous Leukemia (AML) or High Risk Myelodysplasia (MDS) : a Phase I-II Study of the EORTC-LG and GIMEMA (AML-14A Trial)

summary:
RATIONALE: Drugs used in chemotherapy, such as clofarabine, cytarabine, and idarubicin, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells.

PURPOSE: This randomized phase I/II trial is studying the side effects and best dose of clofarabine and to see how well it works when given together with cytarabine and idarubicin in treating patients with intermediate-risk or high-risk acute myeloid leukemia or high-risk myelodysplasia.

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

location_country: Croatia Italy France Belgium Netherlands

location_facility: Azienda Ospedallera Universitaria - Policlinico Tor Vergata

location_facility: Radboud University Nijmegen Medical Center


title: A Double-Blind, Multicenter Study Evaluating the Efficacy and Safety of One Fixed Dose of SR58611A (700 mg/Day) Versus Placebo and Paroxetine (20 mg/Day) in Patients With a Recurrent Major Depressive Episode.

summary:
Primary objective :

To demonstrate the antidepressant efficacy on the Hamilton Depression Rating Scale of SR58611A 700mg/day compared with placebo in the treatment of patients with a recurrent major depressive episode.

Secondary objectives:

To assess the safety profile of SR58611A 700 mg/d in comparison to placebo and to assess plasma concentrations of the active metabolite.

lead_sponsor: Sanofi

location_country:
Croatia
Montenegro
Serbia
Estonia
Czech Republic
France
Netherlands
Bulgaria


title: A Prospective Observational Registry on the Use of NovoSeven® (Activated Recombinant Human Factor VIIa) for on Demand Treatment of Mild to Moderate Bleeds in Haemophilia A and B Patients With Inhibitors

acronym: ONE

summary: This study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.

lead_sponsor: Novo Nordisk A/S

location_country:
Poland
Portugal
Saudi Arabia
Italy
Austria
Netherlands
United Kingdom
Czech Republic
Sweden
Venezuela
Algeria
France
South Africa
Turkey
Belgium
Germany