title: Duloxetine Hydrochloride 60 mg or 120 mg Once Daily Compared With Placebo in Patients With Generalized Anxiety Disorder.

summary: This is a clinical trial assessing duloxetine compared with placebo in patients who have generalized anxiety disorder.

ReferenceCitation: Sheehan DV, Harnett-Sheehan K, Spann ME, Thompson HF, Prakash A. Assessing remission in major depressive disorder and generalized anxiety disorder clinical trials with the discan metric of the Sheehan disability scale. Int Clin Psychopharmacol. 2011 Mar;26(2):75-83. doi: 10.1097/YIC.0b013e328341bb5f.

family: Sheehan

full_name: David V. Sheehan

given: David V.

family: Harnett-Sheehan

full_name: Kathy Harnett-Sheehan

given: Kathy

family: Spann

full_name: Melissa E. Spann

given: Melissa E.

family: Thompson

full_name: Harry F. Thompson

given: Harry F.

family: Prakash

full_name: Apurva Prakash

given: Apurva

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

ReferenceCitation: Sheehan DV, Meyers AL, Prakash A, Robinson MJ, Swindle RW, Russell JM, Mallinckrodt CH. The relationship between functional outcomes and the treatment of anxious and painful somatic symptoms in patients with generalized anxiety disorder. Curr Med Res Opin. 2008 Sep;24(9):2457-66. doi: 10.1185/03007990802293643 . Epub 2008 Jul 24.

name: University of South Florida College of Medicine, Tampa, FL, USA

name: Lilly Research Laboratories, Eli Lilly and Company, Indianapolis, IN, USA

family: Meyers

full_name: Adam L. Meyers

given: Adam L.

family: Robinson

full_name: Michael J. Robinson

given: Michael J.

family: Swindle

full_name: Ralph W. Swindle

given: Ralph W.

family: Russell

full_name: James M. Russell

given: James M.

family: Mallinckrodt

full_name: Craig H. Mallinckrodt

given: Craig H.

journal_name: Current Medical Research and Opinion

publisher: Informa Healthcare

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

ReferenceCitation: Hartford JT, Endicott J, Kornstein SG, Allgulander C, Wohlreich MM, Russell JM, Perahia DG, Erickson JS. Implications of pain in generalized anxiety disorder: efficacy of duloxetine. Prim Care Companion J Clin Psychiatry. 2008;10(3):197-204.

ReferenceCitation: Koponen H, Allgulander C, Erickson J, Dunayevich E, Pritchett Y, Detke MJ, Ball SG, Russell JM. Efficacy of duloxetine for the treatment of generalized anxiety disorder: implications for primary care physicians. Prim Care Companion J Clin Psychiatry. 2007;9(2):100-7.

ReferenceCitation: Allgulander C, Hartford J, Russell J, Ball S, Erickson J, Raskin J, Rynn M. Pharmacotherapy of generalized anxiety disorder: results of duloxetine treatment from a pooled analysis of three clinical trials. Curr Med Res Opin. 2007 Jun;23(6):1245-52. Epub 2007 Apr 25.

ReferenceCitation: Endicott J, Russell JM, Raskin J, Detke MJ, Erickson J, Ball SG, Marciniak M, Swindle RW. Duloxetine treatment for role functioning improvement in generalized anxiety disorder: three independent studies. J Clin Psychiatry. 2007 Apr;68(4):518-24. Erratum in: J Clin Psychiatry. 2007 Apr;68(4):518. J Clin Psychiatry. 2007 May;68(5):806.

location_country:
United States
South Africa
Sweden
Finland
France
Spain
Germany

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


title: Phase II Clinical Trial On Taxol As Single Agent In Locally Advanced And/Or Metastatic Or Recurrent Vulva Cancer Not Amenable For Surgery And/Or Radiotherapy

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

PURPOSE: Phase II trial to study the effectiveness of paclitaxel in treating patients who have locally advanced, metastatic, or recurrent cancer of the vulva.

status: Completed

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: result

ReferenceCitation: Witteveen PO, van der Velden J, Vergote I, Guerra C, Scarabeli C, Coens C, Demonty G, Reed N. Phase II study on paclitaxel in patients with recurrent, metastatic or locally advanced vulvar cancer not amenable to surgery or radiotherapy: a study of the EORTC-GCG (European Organisation for Research and Treatment of Cancer--Gynaecological Cancer Group). Ann Oncol. 2009 Sep;20(9):1511-1516. doi: 10.1093/annonc/mdp043. Epub 2009 Jun 1.

family: Witteveen

full_name: P.O. Witteveen

sequence: first

family: van der Velden

full_name: J. van der Velden

given: J.

sequence: additional

family: Vergote

full_name: I. Vergote

given: I.

family: Guerra

full_name: C. Guerra

given: C.

family: Scarabeli

full_name: C. Scarabeli

family: Coens

full_name: C. Coens

family: Demonty

full_name: G. Demonty

given: G.

family: Reed

full_name: N. Reed

given: N.

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Annals of Oncology

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: elsevier-specific: oa user license

license_normalized: elsevier-specific

version: publishedVersion

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

host_type: repository

license_normalized: no license

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

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

repository_institution: RCAAP Project by FCT - Portuguese National Funding Agency for Science, Research and Technology - RCAAP Search Portal

version: submittedVersion

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Phase II study on paclitaxel in patients with recurrent, metastatic or locally advanced vulvar cancer not amenable to surgery or radiotherapy: a study of the EORTC-GCG (European Organisation for Research and Treatment of Cancer—Gynaecological Cancer Group)

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

location_country:
Italy
Netherlands
France
Belgium
Portugal
United Kingdom

location_facility: Clatterbridge Centre for Oncology NHS Trust

location_facility: Hospitais da Universidade de Coimbra (HUC)

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


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.

study_start_date_type: Actual

study_completion_date_type: Actual

design_allocation: Randomized

ipd_sharing: No

lead_sponsor: Acacia Pharma Ltd

location_country:
Germany
United States
France

location_facility: Wake Forest University School of Medicine


type: EudraCT Number

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.

status: Unknown status

study_completion_date_type: Anticipated

lead_sponsor: Fondazione Melanoma Onlus

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

location_facility: Karl Landsteiner University of Health Sciencies - University Clinic

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

location_facility: Maria Sklodowska-Curie Institute - Oncology Center

location_facility: Università degli Studi di Bari Aldo Moro

location_facility: Azienda Ospedaliera Universitaria Integrata di Udine

location_facility: Azienda Ospedaliero Universitaria Federico II

location_facility: Istituto Dermopatico dell'Immacolata - IDI - IRCCS

location_facility: IEO - Istituto Europeo di Oncologia - IRCCS

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

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

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

status_simplified: Unknown


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.

status: Recruiting

primary_purpose: Prevention

enrollment_type: Anticipated

ReferenceType: background

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

location_facility:
Several Sites
Several

status_simplified: Ongoing


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.

status: Terminated

design_allocation: Non-Randomized

lead_sponsor: ACEA Therapeutics, Inc.

location_country:
United States
France
Spain

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.

ipd_sharing: Undecided

lead_sponsor: Zimmer Biomet

location_country: Sweden France Belgium Spain Germany Canada

location_facility: Sahlgrenska University Hospital Department of Orthopedics, Spine Divison

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

intervention_type: Device


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.

study_type: Observational

time_perspective: Prospective

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.

ReferenceType: derived

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.

family: Warner

full_name: Sarah E. Warner

given: Sarah E.

family: Kaplan

full_name: Frederick S. Kaplan

given: Frederick S.

family: Pignolo

full_name: Robert J. Pignolo

given: Robert J.

family: Smith

full_name: Stacy E. Smith

given: Stacy E.

family: Hsiao

full_name: Edward C. Hsiao

given: Edward C.

family: De Cunto

full_name: Carmen De Cunto

given: Carmen

family: Di Rocco

full_name: Maja Di Rocco

given: Maja

family: Harnett

full_name: Kathleen Harnett

given: Kathleen

family: Grogan

full_name: Donna Grogan

given: Donna

family: Genant

full_name: Harry K. Genant

given: Harry K.

journal_name: Calcified Tissue International

license: cc-by

license_normalized: cc-by

repository_institution: University of California - eScholarship University of California

repository_normalized: California Digital Library - eScholarship

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: Whole-body Computed Tomography Versus Dual Energy X‑ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva

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.

family: Kou

full_name: Samuel Kou

given: Samuel

family: Baujat

full_name: Geneviève Baujat

given: Geneviève

family: Wentworth

full_name: Kelly L. Wentworth

given: Kelly L.

full_name: Donna R. Grogan

given: Donna R.

family: Brown

full_name: Matthew A. Brown

given: Matthew A.

family: Keen

full_name: Richard Keen

title: Patients with ACVR1R206H mutations have an increased prevalence of cardiac conduction abnormalities on electrocardiogram in a natural history study of Fibrodysplasia Ossificans Progressiva

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.

repositories_institution:
King's College London - Research Portal
PubMed Central - Europe PMC

location_country:
Italy
France
Argentina
Australia
United States
United Kingdom

location_facility: The Royal National Orthopaedic Hospital, Brockley Hill

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

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

location_facility: Hospital Italiano de Buenos Aires, Department of Pediatrics

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

location_facility: Hôpital Necker-Enfants Malades, Department of Genetics

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


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).

status: Active, not recruiting

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.

family: Erglis

full_name: Andrejs Erglis

given: Andrejs

family: Narbute

full_name: Inga Narbute

given: Inga

family: Poupineau

full_name: Mathieu Poupineau

given: Mathieu

family: Hovasse

full_name: Thomas Hovasse

given: Thomas

family: Kamzola

full_name: Ginta Kamzola

given: Ginta

family: Zvaigzne

full_name: Ligita Zvaigzne

given: Ligita

full_name: Martins Erglis

given: Martins

full_name: Kristaps Erglis

given: Kristaps

family: Greene

full_name: Samantha Greene

given: Samantha

family: Rogers

full_name: Jason H. Rogers

given: Jason H.

journal_name: Journal of the American College of Cardiology

oa_host_type: publisher

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.

family: Thomas

full_name: Martyn Thomas

given: Martyn

family: Morice

full_name: Marie-Claude Morice

given: Marie-Claude

family: Zabunova

full_name: Milana Zabunova

given: Milana

family: Rudzitis

full_name: Ainars Rudzitis

given: Ainars

location_country:
Italy
United Kingdom
France
Australia
Latvia

location_facility: Brighton and Sussex University Hospitals

location_facility: Pauls Stradins Clinical University Hospital


title: A Phase III Trial of Risk-stratified, Reduced Intensity Adjuvant Treatment in Patients Undergoing Transoral Surgery for Human Papillomavirus (HPV)-Positive Oropharyngeal Cancer

acronym: PATHOS

summary:
The main objectives of the PATHOS study are:

To assess whether swallowing function can be improved following transoral resection of HPV-positive OPSCC, by reducing the intensity of adjuvant treatment protocols. The aim is to personalise treatment, based on disease biology (HPV status and pathology findings), to optimise patient outcomes.

To demonstrate the non-inferiority of reducing the intensity of adjuvant treatment protocols in terms of overall survival in the reduced intensity treatment arms.

ReferenceCitation: Owadally W, Hurt C, Timmins H, Parsons E, Townsend S, Patterson J, Hutcheson K, Powell N, Beasley M, Palaniappan N, Robinson M, Jones TM, Evans M. PATHOS: a phase II/III trial of risk-stratified, reduced intensity adjuvant treatment in patients undergoing transoral surgery for Human papillomavirus (HPV) positive oropharyngeal cancer. BMC Cancer. 2015 Aug 27;15:602. doi: 10.1186/s12885-015-1598-x.

apc_source: openAPC

family: Owadally

full_name: Waheeda Owadally

given: Waheeda

family: Hurt

full_name: Chris Hurt

given: Chris

family: Timmins

full_name: Hayley Timmins

given: Hayley

family: Parsons

full_name: Emma Parsons

given: Emma

family: Townsend

full_name: Sarah Townsend

given: Sarah

family: Patterson

full_name: Joanne Patterson

given: Joanne

family: Hutcheson

full_name: Katherine Hutcheson

given: Katherine

family: Powell

full_name: Ned Powell

given: Ned

family: Beasley

full_name: Matthew Beasley

given: Matthew

family: Palaniappan

full_name: Nachi Palaniappan

given: Nachi

family: Robinson

full_name: Max Robinson

given: Max

family: Jones

full_name: Terence M. Jones

given: Terence M.

family: Evans

full_name: Mererid Evans

given: Mererid

currency_apc_doaj: GBP

journal_name: BMC Cancer

oa_colors: green gold

evidence: oa journal (via doaj)

repository_institution: Newcastle Univesity - Newcastle University ePrints

repository_institution: Cardiff University - ORCA Online Research @Cardiff

repository_normalized: https

version: acceptedVersion

repositories:
PubMed Central
eprint.ncl.ac.uk
https

location_country:
France
Belgium
Australia
United States
United Kingdom

location_facility: European Organisation for Research and Treatment of Cancer

location_facility: Cambridge University Hospitals NHS Foundation Trust

location_facility: Derby Teaching Hospitals NHS Foundation Trust

location_facility: Royal United Hospitals Bath NHS Foundation Trust

location_facility: Aintree University Hospitals NHS Foundation Trust

location_facility: HPV Research Group Section of Pathology Cardiff University ,School of Medicine

location_facility: Guys and St Thomas's NHS Foundation Trust

location_facility: Cardiff and Vale University Local Health Board

location_facility: Newcastle upon Tyne Hospitals NHS Foundation Trust

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: Central Manchester University Hospital NHS Foundation Trust

location_facility: Oxford University Hospitals NHS Foundation Trust

location_facility: Abertawe Bro Morgannwg University Health Board


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.

name: UBiometrics, Ultragenyx Pharmaceutical Inc., Novato, California

family: Mao

full_name: Meng Mao

given: Meng

name: Department of Pediatrics, Department of Orthopaedics and Rehabilitation, Yale University School of Medicine, New Haven, Connecticut

family: Carpenter

full_name: Thomas O Carpenter

given: Thomas O

name: Division of Bone and Mineral Diseases, Department of Internal Medicine, Washington University School of Medicine, St. Louis, Missouri

name: Center for Metabolic Bone Disease and Molecular Research, Shriners Hospitals for Children - St Louis, St Louis, Missouri

family: Whyte

full_name: Michael P Whyte

given: Michael P

name: Clinical Outcomes, Research and Evaluation, Ultragenyx Pharmaceutical Inc., Novato, California

family: Skrinar

full_name: Alison Skrinar

given: Alison

family: Chen

full_name: Chao-Yin Chen

given: Chao-Yin

name: Clinical Development, Ultragenyx Pharmaceutical Inc., Novato, California

family: San Martin

full_name: Javier San Martin

given: Javier

name: Pediatrics, University of Virginia School of Medicine, Charlottesville, Virginia

family: Rogol

full_name: Alan D Rogol

given: Alan D

journal_name: The Journal of Clinical Endocrinology & Metabolism

publisher: The Endocrine Society

publisher_dissemination: The Endocrine Society

publisher_group: The Endocrine Society

publisher_normalized: The Endocrine Society

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.

name: From Yale University School of Medicine, New Haven, CT (T.O.C.); Shriners Hospital for Children and Division of Bone and Mineral Diseases, Department of Internal Medicine, Washington University School of Medicine, St. Louis (M.P.W.); Indiana University School of Medicine, Indianapolis (E.A.I.); University of Groningen, Groningen, the Netherlands (A.M.B.); Birmingham Children’s Hospital, Birmingham (W. Högler), Royal Manchester Children’s Hospital, Manchester (R.P.), and Great Ormond Street Hospital,...

full_name: Thomas O. Carpenter

given: Thomas O.

full_name: Michael P. Whyte

given: Michael P.

family: Imel

full_name: Erik A. Imel

given: Erik A.

family: Boot

full_name: Annemieke M. Boot

given: Annemieke M.

family: Högler

full_name: Wolfgang Högler

given: Wolfgang

family: Linglart

full_name: Agnès Linglart

given: Agnès

family: Padidela

full_name: Raja Padidela

given: Raja

family: van’t Hoff

full_name: William van’t Hoff

given: William

family: Kakkis

full_name: Emil Kakkis

given: Emil

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

location_country:
United States
United Kingdom
France
Netherlands

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

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)

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 Phase III Study Comparing Induction Chemotherapy to Daily Low Dose Cisplatin Both Combined With High Dose Radiotherapy Using Concomitant Boost Technique in Patients With Inoperable Non-Small Cell Lung Cancer Stage I, II, and Low Volume Stage III

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Radiation therapy used high-energy x-rays to damage tumor cells. It is not yet know whether chemotherapy followed by radiation therapy is more effective than chemotherapy given with radiation therapy for non-small cell lung cancer.

PURPOSE: Randomized phase III trial to compare the effectiveness of two different regimens of chemotherapy and radiation therapy in treating patients who have unresectable stage I, stage II, or stage III non-small cell lung cancer.

ReferenceCitation: Belderbos J, Uitterhoeve L, van Zandwijk N, Belderbos H, Rodrigus P, van de Vaart P, Price A, van Walree N, Legrand C, Dussenne S, Bartelink H, Giaccone G, Koning C; EORTC LCG and RT Group. Randomised trial of sequential versus concurrent chemo-radiotherapy in patients with inoperable non-small cell lung cancer (EORTC 08972-22973). Eur J Cancer. 2007 Jan;43(1):114-21. Epub 2006 Nov 3.

location_country:
Netherlands
France
Belgium
Germany
United Kingdom

location_facility: Twee Steden Ziekenhuis Vestiging Tilburg

intervention_type: Radiation


title: PMCF Study - Verification of Activa IM-Nail™ Safety and Clinical Performance

acronym: BRINFF

summary: The Activa IM-Nail™ is used in the fixation of forearm fractures with cast to achieve a level of reduction and stabilisation that is appropriate to the age of the child. The post-market clinical follow-up study will be performed to identify the residual risk related to re-fracture and to determine its impact to the risk/benefit ratio of Activa IM-Nail™.

ReferenceCitation: Korhonen L, Perhomaa M, Kyrö A, Pokka T, Serlo W, Merikanto J, Sinikumpu JJ. Intramedullary nailing of forearm shaft fractures by biodegradable compared with titanium nails: Results of a prospective randomized trial in children with at least two years of follow-up. Biomaterials. 2018 Dec;185:383-392. doi: 10.1016/j.biomaterials.2018.09.011. Epub 2018 Sep 11.

lead_sponsor: Thomas Klestil

location_country: Austria Switzerland France Hungary Portugal Denmark Germany

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Péterfy Hospital, National Trauma Center, Department of Pediatric Trauma Surgery

location_facility: University Medical Center Schleswig-Holstein, Campus Lübeck Department of Pediatric Surgery

location_facility: Lille University Center, Jeanne de Flandre Hospital

location_facility: Herlev and Gentofte University Hospital Department of Orthopedic Surgery

location_facility: Pécs University Hospital, Department of Pediatrics

intervention_type: Other


lead_sponsor: Associazione Italiana Ematologia Oncologia Pediatrica

location_country: Italy Sweden Netherlands Spain France Czechia Israel Ireland Belgium Portugal Denmark Germany

acronym: ICC APL Study 02

location_facility: Pediatrics and Adolescent Medicine Aarhus University Hospital

location_facility: Policlinico Umberto I Università "LA Sapienza" - Dip. Biotecnologie cellulari ed ematologia UOS Ematologia Pediatrica

location_facility: Univerità degli Studi della Campania- Luigi Vanvitelli - Sevizio di Oncologia Pediatrica

location_facility: Ospedale santa Chiara - AOU Pisana, UO Oncoematologia Pediatrica

location_facility: Rappaport Children'S Hospital, Rambam Health Care Campus

location_facility: Valencia University Medical School University Hospital La Fe

location_facility: AOU Policlinico Vittorio Emanuele - UOC Ematologia ed Oncologia Pediatrica con TNO

location_facility: Childrens hematology and oncology Uppsala University

location_facility: IRCCS Istituto Gannina Gaslini - Dipartimento di Oncoematologia

location_facility: Universitätsklinikum Essen (AöR) Zentrum für Kinder-und Jugendmedizin Klinik für Kinderheilkunde III

location_facility: Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE

location_facility: A.O. Universitaria Meyer - DAI Oncoematologia Pediatrica

location_facility: ARNAS Civico di Cristina e Benfratelli - UOC Oncoematologia Pediatrica

location_facility: Fondazione Monza e Brianza per il Bambino e la sua Mamma (MBBM) - Ospedale San Gerardo

location_facility: Ospedale "Casa Sollievo della Sofferenza" - UO Oncoematologia Pediatrica

location_facility: Instituto Português de Oncologia do Porto Francisco Gentil, E. P. E.

location_facility: AOU Policlinico Dipartimento di Pediatria

location_facility: Ospedale Papa Giovanni XXIII - USS Oncoematologia Pediatrica

location_facility: AOU Policlinico Sant'Orsola-Malpighi - Oncologia ed Ematologia Pediatrica

location_facility: AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita

location_facility: Hôpital Universitaire des Enfants Reine Fabiola (Huderf)

location_facility: Centro Hospitalar Universitário de Coimbra - Hospital Pediátrico de Coimbra

location_facility: Ospedale Pediatrico Microcitemico "A.Cau", Az.Ospedaliera Brotzu - SC Oncoematologia Ped. e Patologia della coagulazione

location_facility: Azienda Ospedaliera di Padova - Oncoematologia Pediatrica

location_facility: Fondazione IRCCS Policlinico San Matteo - Oncoematologia Pediatrica

location_facility: Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Ospedale Pediatrico "Bambino Gesù"

source: euctr

title: Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia

summary: The trial is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR-positive for the PML-RARα transcript and less than 18 years of age.


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

lead_sponsor: UCB Biopharma SRL

location_country:
United States
Mexico
France
Japan
Poland
Hungary
Belgium
Spain
Germany

ipd_sharing: Yes

title: Open-Label, Single-Arm, Multicenter Study to Evaluate Long-Term Safety and Tolerability of Brivaracetam Used as Adjunctive Treatment in Pediatric Study Participants With Epilepsy

summary: The purpose of the study is to evaluate the long-term safety and tolerability of brivaracetam.


type: Other Identifier

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.

family: Yeh

full_name: Kun-Huei Yeh

given: Kun-Huei

family: Yang

full_name: Tsai-Sheng Yang

given: Tsai-Sheng

family: Hsu

full_name: Tzu-Chi Hsu

given: Tzu-Chi

family: Tzu-Liang Chen

full_name: William Tzu-Liang Chen

full_name: Hong-Hwa Chen

given: Hong-Hwa

family: Teng

full_name: Hao-Wei Teng

given: Hao-Wei

family: Lin

full_name: Bo-Wen Lin

given: Bo-Wen

family: Kuan

full_name: Feng-Che Kuan

given: Feng-Che

family: Chiang

full_name: Feng-Fan Chiang

given: Feng-Fan

family: Duann

full_name: Chi-Wei Duann

given: Chi-Wei

family: Li

full_name: Ying-Shiuan Li

given: Ying-Shiuan

full_name: Meng-Ting Lin

given: Meng-Ting

family: Fiala-Buskies

full_name: Sabine Fiala-Buskies

given: Sabine

family: Ducreux

full_name: Michel Ducreux

given: Michel

family: Wang

full_name: Jaw-Yuan Wang

given: Jaw-Yuan

currency_apc_doaj: USD

journal_name: Journal of the Formosan Medical Association

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

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.

family: Petersen

full_name: Lone Nørgård Petersen

given: Lone Nørgård

family: Öhler

full_name: Leopold Öhler

given: Leopold

family: Bergamo

full_name: Francesca Bergamo

given: Francesca

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


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.

primary_purpose: Other

lead_sponsor: Biosense Webster, Inc.

location_country:
Italy
Germany
United States
France

location_facility: Texas Cardiac Arrhythmia Research Foundation


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.

lead_sponsor: Pfizer

location_country:
Italy
United States
Netherlands
France
Belgium
Spain
Canada

location_facility: University of California at Los Angeles Dept of Onc

location_facility: Memorial Sloan Kettering Cancer Center Oncology Dept

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

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.

lead_sponsor: Bayer

location_country:
Germany
United States
France
Switzerland


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.

name: German Breast Group, Neu-Isenburg, Germany

name: Center for Hematology and Oncology Bethanien, Frankfurt, Germany

family: Loibl

full_name: Sibylle Loibl

given: Sibylle

name: Department of Gynaecology and Obstetrics, University Hospital Mannheim, Mannheim, Germany

family: Marmé

full_name: Frederik Marmé

given: Frederik

name: Instituto de Investigacion Sanitaria Gregorio Marañon, CIBERONC, Universidad Complutense, Madrid, Spain

family: Martin

full_name: Miguel Martin

given: Miguel

name: Department of Gynaecology and Obstetrics, Breast Cancer Center, HELIOS Klinikum Berlin Buch, Berlin, Germany

family: Untch

full_name: Michael Untch

given: Michael

name: UCBG (Unicancer Breast Cancer Group) and Institut Bergonié, Université de Bordeaux, Bordeaux, France

family: Bonnefoi

full_name: Hervé Bonnefoi

given: Hervé

name: Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea, and KCSG (Korean Cancer Study Group), Korea

family: Kim

full_name: Sung-Bae Kim

given: Sung-Bae

name: Division of Surgical Oncology, Massey Cancer Center, Virginia Commonwealth University, VCU Health, Richmond, VA

family: Bear

full_name: Harry Bear

given: Harry

name: Breast Cancer Trials Australia and New Zealand, Newcastle, Australia

family: McCarthy

full_name: Nicole McCarthy

given: Nicole

name: Oncology Research Group, Hospital Universitario Sant Joan de Reus, Reus, Spain

family: Melé Olivé

full_name: Mireia Melé Olivé

given: Mireia

name: BC Cancer Agency, Vancouver, British Columbia, Canada

family: Gelmon

full_name: Karen Gelmon

given: Karen

name: Instituto de Investigación Sanitaria Hospital Clinico San Carlos (IdISSC), Madrid, Spain

family: García-Sáenz

full_name: José García-Sáenz

given: José

name: Mater Misericordiae University Hospital and Breast Group, Cancer Trials, Dublin, Ireland

family: Kelly

full_name: Catherine M. Kelly

given: Catherine M.

name: Department of Obstetrics and Gynecology, University of Rostock, Rostock, Germany

family: Reimer

full_name: Toralf Reimer

given: Toralf

name: Breast Surgery, Graduate School of Medicine, Kyoto University, Kyoto, Japan

family: Toi

full_name: Masakazu Toi

given: Masakazu

name: Breast Department, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA

family: Rugo

full_name: Hope S. Rugo

given: Hope S.

name: Institute of Pathology, University Hospital Marburg and Philipps-Universität Marburg, Germany

family: Denkert

full_name: Carsten Denkert

given: Carsten

name: Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria

family: Gnant

full_name: Michael Gnant

name: Mount Vernon Cancer Centre, Northwood, United Kingdom

family: Makris

full_name: Andreas Makris

given: Andreas

family: Koehler

full_name: Maria Koehler

given: Maria

family: Huang-Bartelett

full_name: Cynthia Huang-Bartelett

given: Cynthia

family: Lechuga Frean

full_name: Maria Jose Lechuga Frean

given: Maria Jose

name: IEO, European Institute of Oncology, IRCCS, Milan, Italy

family: Colleoni

full_name: Marco Colleoni

given: Marco

name: Latin American Cooperative Oncology Group (LACOG), Porto Alegre, Brazil

family: Werutsky

full_name: Gustavo Werutsky

given: Gustavo

publisher: American Society of Clinical Oncology (ASCO)

title: Palbociclib for Residual High-Risk Invasive HR-Positive and HER2-Negative Early Breast Cancer—The Penelope-B Trial

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

location_facility: Contact: Japan Breast Cancer Research Group

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

location_facility: Contact: Austrian Breast & Colorectal Cancer Study Group

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"

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.

name: <idGroup xmlns="http://www.wiley.com/namespaces/wiley"><id type="grid" value="grid.6363.0"></id><id type="isni" value="0000000122184662"></id></idGroup>Allergy‐Centre‐CharitéCharité ‐ Universitätsmedizin Berlin10117BerlinGermany

family: Worm

full_name: Margitta Worm

given: Margitta

name: <idGroup xmlns="http://www.wiley.com/namespaces/wiley"><id type="grid" value="grid.1649.a"></id><id type="isni" value="000000009445082X"></id></idGroup>Asthma and Allergy Research GroupDepartment of Respiratory Medicine and AllergologySahlgrenska University HospitalGöteborgSweden

family: Rak

full_name: Sabina Rak

given: Sabina

name: <idGroup xmlns="http://www.wiley.com/namespaces/wiley"><id type="grid" value="grid.412220.7"></id><id type="isni" value="000000012177138X"></id></idGroup>Chest Diseases DepartmentStrasbourg University HospitalFederation of Translational MedicineUniversity of StrasbourgStrasbourgFrance

family: Blay

full_name: Frédéric Blay

given: Frédéric

name: <idGroup xmlns="http://www.wiley.com/namespaces/wiley"><id type="grid" value="grid.411646.0"></id><id type="isni" value="0000000406467402"></id></idGroup>Allergy ClinicGentofte University HospitalCopenhagenDenmark

family: Malling

full_name: Hans‐Jorgen Malling

given: Hans‐Jorgen

name: <idGroup xmlns="http://www.wiley.com/namespaces/wiley"><id type="grid" value="grid.420215.0"></id><id type="isni" value="0000000405112052"></id></idGroup>Stallergenes S.AAntonyFrance

family: Melac

full_name: Michel Melac

given: Michel

family: Cadic

full_name: Véronique Cadic

given: Véronique

family: Zeldin

full_name: Robert K Zeldin

given: Robert K

journal_name: Clinical and Translational Allergy

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: 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

lead_sponsor: Stallergenes Greer

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

location_facility: Centre of Investigations and Treatment of Allergic Diseases

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

location_facility: National University Hospital - Allergy Unit 4222

location_facility: Allergic Diseases Diagnostics and Treatment Centre

intervention_type: Biological


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: France Bulgaria Poland Romania Latvia


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.

lead_sponsor: Novartis Pharmaceuticals

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


title: Incidence of Dyssynchronous Spontaneous Breathing Effort, Breath-stacking and Reverse Triggering in Early ARDS

acronym: BEARDS

summary:
Patients sedated under mechanical ventilation with acute hypoxemic respiratory failure with a PaO2/FiO2 equal or less than 200mmHg (Acute Respiratory Distress Syndrome, ARDS and non-ARDS) will be included in the study early in the course of the disease (first week of mechanical ventilation). At enrollment, data on the clinical condition of the patient will be recorded together with ventilation settings: ventilation mode, the fraction of inspired oxygen (FiO2), PEEP, tidal volume, set pressure, respiratory rate, time of the respiratory cycle, recent blood gas parameters.

Airway pressure, flow, and esophageal pressure (or alternatively electrical activity of the diaphragm, Eadi) will be recorded 3 times a day for 7 days:

Period 1 (morning): duration 20-30 minutes
Period 2 (afternoon): duration 20-30 minutes
Period 3 (evening / night): duration 20-30 minutes

Registration will be ended at extubation, death or at eight days from the first recording.

Monitoring of vital parameters (hemodynamic and respiratory) will be continuous throughout the duration of the study, as per normal clinical practice. All drugs used during the day of the measurements will be recorded. The patient will then be followed until discharge from the ICU and after 60 days of discharge to evaluate mortality.

As an ancillary study, in a subgroup of patients continuous simplified measurement of respiratory recordings together with hourly clinical data on sedation and extended simplified polysomnography recordings will be performed within the first 7 days from inclusion.

The analysis of the recorded waveforms will be performed in a single center by a centralized system that will quantify dyssynchrony and its intensity, calculate pressure time product, collect clinical and physiological data and outcome, and investigate possible correlations.

ReferenceCitation: Pham T, Montanya J, Telias I, Piraino T, Magrans R, Coudroy R, Damiani LF, Mellado Artigas R, Madorno M, Blanch L, Brochard L; BEARDS study investigators. Automated detection and quantification of reverse triggering effort under mechanical ventilation. Crit Care. 2021 Feb 15;25(1):60. doi: 10.1186/s13054-020-03387-3.

family: Pham

full_name: Tài Pham

given: Tài

family: Montanya

full_name: Jaume Montanya

given: Jaume

family: Telias

full_name: Irene Telias

given: Irene

family: Piraino

full_name: Thomas Piraino

given: Thomas

family: Magrans

full_name: Rudys Magrans

given: Rudys

family: Coudroy

full_name: Rémi Coudroy

given: Rémi

family: Damiani

full_name: L. Felipe Damiani

given: L. Felipe

family: Mellado Artigas

full_name: Ricard Mellado Artigas

given: Ricard

family: Madorno

full_name: Matías Madorno

given: Matías

family: Blanch

full_name: Lluis Blanch

given: Lluis

family: Brochard

full_name: Laurent Brochard

given: Laurent

family: Mellado Artigas

full_name: Ricard Mellado Artigas

family: Santis

full_name: Cesar Santis

given: Cesar

ipd_sharing_description: We plan to share physiologic tracings with no participant identification.

location_country: Italy Netherlands Spain France Thailand Greece China Germany Canada Taiwan

location_facility: Centre Hospitalier Universitaire - CHU Angers

location_facility: Azienda Ospedaliero - Universitaria OORR Ospedali Riuniti di Foggia

location_facility: National Cheng-Kung University and Hospital

location_facility: Beijing Tiantan Hospital, Capital Medical University

location_facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico


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.

family: Dai

full_name: Yi Dai

given: Yi

family: Zhou

full_name: Yingfang Zhou

given: Yingfang

family: Zhang

full_name: Xinmei Zhang

given: Xinmei

family: Xue

full_name: Min Xue

given: Min

family: Sun

full_name: Pengran Sun

given: Pengran

family: Leng

full_name: Jinhua Leng

given: Jinhua

family: Chapron

full_name: Charles Chapron

given: Charles

journal_name: BMC Women's Health

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.

family: Lang

full_name: Jing-He Lang

given: Jing-He

full_name: Jin-Hua Leng

given: Jin-Hua

family: Popov

full_name: Alexander Popov

given: Alexander

family: Romanov

full_name: Vladimir Romanov

given: Vladimir

family: Maisonobe

full_name: Pascal Maisonobe

given: Pascal

title: Factors and Regional Differences Associated with Endometriosis: A Multi-Country, Case–Control Study

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

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

location_facility: Moscow Regional Scientific Research Institute of Obstetrics and Gynaecology

location_facility: The Third Xiangya Hospital of Central South University


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.

family: Mertens

full_name: Renato Mertens

given: Renato

family: Bergoeing

full_name: Michel Bergoeing

given: Michel

family: Mariné

full_name: Leopoldo Mariné

given: Leopoldo

family: Valdés

full_name: Francisco Valdés

given: Francisco

family: Krämer

full_name: Albrecht Krämer

given: Albrecht

family: Vergara

full_name: Jeanette Vergara

given: Jeanette

journal_name: Journal of Endovascular Therapy

publisher: International Society of Endovascular Specialists

title: Ventana Fenestrated Stent-Graft System for Endovascular Repair of Juxtarenal Aortic Aneurysms

lead_sponsor: Endologix

location_country:
Chile
Netherlands
France
New Zealand
United States
United Kingdom

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


title: International Multicentre Observational Trial of Perioperative Ventilatory Management in Cardiac Surgery

acronym: VENICE

summary: The purpose of this study is to evaluate prospectively ventilatory practices in the perioperative cardiac surgery period.

study_start_date_type: Anticipated

ReferenceCitation: Futier E, Constantin JM, Paugam-Burtz C, Pascal J, Eurin M, Neuschwander A, Marret E, Beaussier M, Gutton C, Lefrant JY, Allaouchiche B, Verzilli D, Leone M, De Jong A, Bazin JE, Pereira B, Jaber S; IMPROVE Study Group. A trial of intraoperative low-tidal-volume ventilation in abdominal surgery. N Engl J Med. 2013 Aug 1;369(5):428-37. doi: 10.1056/NEJMoa1301082.

family: Futier

full_name: Emmanuel Futier

given: Emmanuel

family: Constantin

full_name: Jean-Michel Constantin

given: Jean-Michel

family: Paugam-Burtz

full_name: Catherine Paugam-Burtz

given: Catherine

family: Pascal

full_name: Julien Pascal

given: Julien

family: Eurin

full_name: Mathilde Eurin

given: Mathilde

family: Neuschwander

full_name: Arthur Neuschwander

given: Arthur

family: Marret

full_name: Emmanuel Marret

family: Beaussier

full_name: Marc Beaussier

given: Marc

family: Gutton

full_name: Christophe Gutton

given: Christophe

family: Lefrant

full_name: Jean-Yves Lefrant

given: Jean-Yves

family: Allaouchiche

full_name: Bernard Allaouchiche

given: Bernard

family: Verzilli

full_name: Daniel Verzilli

given: Daniel

family: Leone

full_name: Marc Leone

family: De Jong

full_name: Audrey De Jong

given: Audrey

family: Bazin

full_name: Jean-Etienne Bazin

given: Jean-Etienne

family: Pereira

full_name: Bruno Pereira

given: Bruno

family: Jaber

full_name: Samir Jaber

given: Samir

journal_name: New England Journal of Medicine

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

title: A Trial of Intraoperative Low-Tidal-Volume Ventilation in Abdominal Surgery

ReferenceCitation: Lellouche F, Delorme M, Bussières J, Ouattara A. Perioperative ventilatory strategies in cardiac surgery. Best Pract Res Clin Anaesthesiol. 2015 Sep;29(3):381-95. doi: 10.1016/j.bpa.2015.08.006. Epub 2015 Sep 4. Review.

ReferenceCitation: Güldner A, Kiss T, Serpa Neto A, Hemmes SN, Canet J, Spieth PM, Rocco PR, Schultz MJ, Pelosi P, Gama de Abreu M. Intraoperative protective mechanical ventilation for prevention of postoperative pulmonary complications: a comprehensive review of the role of tidal volume, positive end-expiratory pressure, and lung recruitment maneuvers. Anesthesiology. 2015 Sep;123(3):692-713. doi: 10.1097/ALN.0000000000000754. Review.

lead_sponsor: University Hospital, Caen

location_country: Italy Brazil Netherlands France Czechia Japan Poland Norway Belgium Germany

location_facility: Osaka city University Graduate school of Medecine

location_facility: CUB Erasm, Université libre de Bruxelles

location_facility: Klinik für Anästhesiologie und Intensivmedizin

location_facility: Universidade Federal de São Paulo (UNIFESP)

lead_sponsor_type: academique


title: A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy

acronym: Advance

summary: This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).

lead_sponsor: Minoryx Therapeutics, S.L.

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

location_facility: Institute of Genomic Medicine and Rare Disorders

location_facility: National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)

location_facility: Universitat Leipzig Klinik and Poliklinik für Neurologie


title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

summary: This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

lead_sponsor: bluebird bio

location_country:
Italy
United States
France
Greece
Germany
United Kingdom

location_facility: UCSF Benioff Children's Hospital Oakland

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

location_facility: General Hospital of Thessaloniki 'G.Papanikolaou'

intervention_type: Genetic


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

lead_sponsor: Allergan

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


title: An Open-label, Multi-center, Global, Rollover Study for Patients Who Have Previously Been Treated With Capmatinib (INC280) as Monotherapy or in Combination in a Novartis Sponsored Trial.

summary: The rollover study will provide continued treatment and assessment of long-term safety follow-up in patients receiving capmatinib (INC280) as a single agent or in combination with EGF816 or Gefitinib in a Novartis sponsored study and in the opinion of the Investigator would benefit from continued treatment.

location_country:
Italy
Germany
France
Korea, Republic of
Singapore
Spain
Denmark
United States
Canada

location_facility: Massachusetts General Hospital MGH Cancer Center


title: A Randomised, Double-blind, Parallel Group, Equivalence, Multicentre Phase III Trial to Compare the Efficacy, Safety and Pharmacokinetics of HD201 to Herceptin® in Patients With HER2+ Early Breast Cancer

acronym: TROIKA

summary: In the TROIKA study, the proposed biosimilar HD201 will be compared to its reference product Herceptin®. The aim of the study is to demonstrate equivalence of HD201 and Herceptin® in terms of efficacy, safety and pharmacokinetics.

lead_sponsor: Prestige Biopharma Limited

location_country:
Italy
Spain
France
Bulgaria
Malaysia
Poland
Georgia
Thailand
Hungary
Russian Federation
Belarus
Estonia
Ukraine

location_facility: GI "V. T. Zaycev Institute General and Urgent Surgery of NAMS of Ukraine "


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.

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

location_country:
Italy
Switzerland
France
Poland
Belgium
Spain
Germany
United Kingdom

location_facility: Hopitaux Universitaires de Strasbourg - Hautepierre

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

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

location_facility: Cambridge University Hospital NHS - Addenbrookes Hospital

location_facility: Imperial College Healthcare NHS Trust - Charing Cross Hospital

location_facility: Centre Hospitalier Universitaire Vaudois - Lausanne

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

location_facility: Universitaets Krankenhaus Eppendorf - UKE - University Cancer Center

location_facility: Staedtisches Klinikum Leipzig - Klinikum St Georg

location_facility: Institut Jules Bordet-Hopital Universitaire ULB

location_facility: Hospital Universitario Central De Asturias

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

location_facility: Universitaetsklinikum Tuebingen- Crona Kliniken


title: A Phase I, Open-label, Multi-center Dose Escalation Study of FAZ053 as Single Agent and in Combination With PDR001 in Adult Patients With Advanced Malignancies

summary:
The purpose of this "first-in-human" study of FAZ053 is to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of FAZ053 administered Intravenously (i.v.)as a single agent or in combination with PDR001 in adult patients with advanced solid tumors.

By blocking the interaction between Programmed Death Ligand-1 (PD-L1) and its receptors, Programmed Death-1 (PD-1) and B7.1, FAZ053 inhibits the PD-L1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells.

This study has been designed as a Phase I, open-label, multi-center study with a dose escalation part of FAZ053 as single agent and in combination with PDR001, followed by a dose expansion part of FAZ053 as single agent.

FAZ053 will initially be dosed every three weeks. A less frequent dosing regimen such as every 6 weeks may be evaluated in parallel.

A patient may continue treatment with FAZ053 single agent or in combination with PDR001 until the patient experiences unacceptable toxicity, confirmed disease progression per immune related Response Criteria and/or treatment is discontinued at the discretion of the investigator or the patient.

location_country:
Italy
France
Japan
Israel
Singapore
Spain
United States
Canada
Taiwan

location_facility:
Memorial Sloan Kettering Cancer Center
Novartis Investigative Site
UT MD Anderson Cancer Center


title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

summary: This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

ReferenceCitation: Hamed EM, Meabed MH, Aly UF, Hussein RRS. Recent Progress in Gene Therapy and Other Targeted Therapeutic Approaches for Beta Thalassemia. Curr Drug Targets. 2019;20(16):1603-1623. doi: 10.2174/1389450120666190726155733. Review.

name: Department of Pharmaceutics and Clinical Pharmacy; Faculty of Pharmacy; Nahda University, Nahda, Egypt

family: Hamed

full_name: Eman M. Hamed

given: Eman M.

name: Professor of Haematology; Faculty of Medicine; Beni-Suef University, Beni Suef, Egypt

family: Meabed

full_name: Mohamed Hussein Meabed

given: Mohamed Hussein

name: Asso. Professor of Pharmaceutics; Faculty of Pharmacy; Minia University, Minya, Egypt

family: Aly

full_name: Usama Farghaly Aly

given: Usama Farghaly

name: Lecturer of Clinical Pharmacy; Faculty of Pharmacy; Beni- Suef University, Egypt

family: Hussein

full_name: Raghda R.S. Hussein

given: Raghda R.S.

journal_name: Current Drug Targets

publisher: Bentham Science Publishers Ltd.

publisher_dissemination: Bentham Science Publishers Ltd.

publisher_group: Bentham Science Publishers Ltd.

publisher_normalized: Bentham Science Publishers Ltd.

location_country:
Italy
United States
Thailand
France
Germany
United Kingdom


title: An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of RM-493, a MC4R Agonist, in Early Onset POMC Deficiency Obesity Due to Bi-Allelic Loss-of-Function POMC or PCSK1 Genetic Mutation

summary: The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight and other factors in patients with pro-opiomelanocortin (POMC) deficiency obesity due to rare bi-allelic loss-of function POMC or PCSK1 genetic mutations.

ReferenceCitation: Clément K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C, Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kühnen P; Setmelanotide POMC and LEPR Phase 3 Trial Investigators. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. Lancet Diabetes Endocrinol. 2020 Dec;8(12):960-970. doi: 10.1016/S2213-8587(20)30364-8. Epub 2020 Oct 30.

family: Clément

full_name: Karine Clément

given: Karine

family: van den Akker

full_name: Erica van den Akker

given: Erica

family: Argente

full_name: Jesús Argente

given: Jesús

family: Bahm

full_name: Allison Bahm

given: Allison

family: Chung

full_name: Wendy K Chung

given: Wendy K

family: Connors

full_name: Hillori Connors

given: Hillori

family: De Waele

full_name: Kathleen De Waele

given: Kathleen

family: Farooqi

full_name: I Sadaf Farooqi

given: I Sadaf

family: Gonneau-Lejeune

full_name: Julie Gonneau-Lejeune

given: Julie

family: Gordon

full_name: Gregory Gordon

given: Gregory

family: Kohlsdorf

full_name: Katja Kohlsdorf

given: Katja

family: Poitou

full_name: Christine Poitou

given: Christine

family: Puder

full_name: Lia Puder

given: Lia

family: Swain

full_name: James Swain

given: James

family: Stewart

full_name: Murray Stewart

given: Murray

family: Yuan

full_name: Guojun Yuan

given: Guojun

family: Wabitsch

full_name: Martin Wabitsch

given: Martin

family: Kühnen

full_name: Peter Kühnen

given: Peter

family: Pigeon-Kherchiche

full_name: Patricia Pigeon-Kherchiche

given: Patricia

family: Flaus-Furmaniuk

full_name: Anna Flaus-Furmaniuk

given: Anna

family: Bald

full_name: Martin Bald

family: Denzer

full_name: Christian Denzer

given: Christian

family: von Schnurbein

full_name: Julia von Schnurbein

given: Julia

location_country:
United States
Germany
France
Belgium
Spain
Canada
United Kingdom

location_facility: Institute of Cardiometabolism and Nutrition / Hopital de la Pitié-Salpêtrière

location_facility: University of Cambridge Metabolic Research Laboratories

location_facility: Charité Campus Virchow-Klinikum / Institute for Experimental Pediatric Endocrinology


title: Molecular Reclassification to Find Clinically Useful Biomarkers for Systemic Autoimmune Diseases: Cross Sectional Cohort

acronym: PRECISESADS

summary: Connective tissue diseases (CTD) or systemic autoimmune diseases (SADs) as they are known today are a group of chronic inflammatory conditions with autoimmune aetiology with few treatment options and difficult diagnosis.Brest team contribute to perform a new classification of the following systemic autoimmune diseases in a European Union's Seventh Framework Programme. The aim of this research is to reclassify the individuals affected by SADs into molecular clusters instead of clinical entities through the determination of molecular profiles using several "Omics" techniques.

lead_sponsor: Fundación Pública Andaluza Progreso y Salud

location_country: Italy Austria Switzerland Spain France Hungary Belgium Portugal Germany

location_facility: Hospital Clinic I Provicia- Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS)

location_facility: Hospital Regional de Málaga Servicio Andaluz de Salud

location_facility: UZ Leuven - KU Leuven, Department of Rheumatology (KU LEUVEN)

location_facility: Hospital Universitario Reina Sofía Andaluz de Salud

location_facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico (IRCCS)

location_facility: Université catholique de Louvain - Cliniques Universitaires Saint-Luc

location_facility: Deutsches Rheuma-Forschungszentrum Berlin (DRFZ)

location_facility: Hospital Universitario San Cecilio Servicio Andaluz de Salud

location_facility: Hospital Universitario Marqués de Valdecilla, Servicio Cántabro de Salud


lead_sponsor: Grifols Therapeutics LLC

location_country:
Sweden
France
Czechia
Poland
Hungary
Australia
Spain
Germany
United Kingdom

location_facility: The Children's Memorial Health Institute

location_facility: Children's Hospital at Municipal Hospital St. Georg

title: A Multi-Center, Open-Label, Single-Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency

summary: Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%) who complete the entire study. This study will include 3 study stages: Screening/Previous Regimen Phase, IGSC 20% Treatment Stage 1 (13 IGSC 20% weekly doses), and IGSC 20% Treatment Stage 2 (39 IGSC 20% weekly doses). A total of 52 doses of IGSC 20% will be administered with a final follow-up visit 1 week after the last dose at Week 53. Subjects/caregivers will be trained on self-administration of IGSC 20% by the clinical site personnel.


title: Pipeline Flex With SHield Technology Embolization - An International MulticEnter ObservationaL Post Market StuDy of Treated Intra Cranial Aneurysms (SHIELD)

acronym: SHIELD

summary: This is a prospective, single-arm, multi-center post-market observational study assessing the performance of the Pipeline™ Flex Embolization Device with Shield Technology™ in subjects undergoing treatment for intracranial aneurysms in a large real-world, post-market setting.

ReferenceCitation: Nelson PK, Lylyk P, Szikora I, Wetzel SG, Wanke I, Fiorella D. The pipeline embolization device for the intracranial treatment of aneurysms trial. AJNR Am J Neuroradiol. 2011 Jan;32(1):34-40. doi: 10.3174/ajnr.A2421. Epub 2010 Dec 9.

ReferenceCitation: Becske T, Kallmes DF, Saatci I, McDougall CG, Szikora I, Lanzino G, Moran CJ, Woo HH, Lopes DK, Berez AL, Cher DJ, Siddiqui AH, Levy EI, Albuquerque FC, Fiorella DJ, Berentei Z, Marosfoi M, Cekirge SH, Nelson PK. Pipeline for uncoilable or failed aneurysms: results from a multicenter clinical trial. Radiology. 2013 Jun;267(3):858-68. doi: 10.1148/radiol.13120099. Epub 2013 Feb 15.

ReferenceCitation: Kallmes DF, Hanel R, Lopes D, Boccardi E, Bonafé A, Cekirge S, Fiorella D, Jabbour P, Levy E, McDougall C, Siddiqui A, Szikora I, Woo H, Albuquerque F, Bozorgchami H, Dashti SR, Delgado Almandoz JE, Kelly ME, Turner R 4th, Woodward BK, Brinjikji W, Lanzino G, Lylyk P. International retrospective study of the pipeline embolization device: a multicenter aneurysm treatment study. AJNR Am J Neuroradiol. 2015 Jan;36(1):108-15. doi: 10.3174/ajnr.A4111. Epub 2014 Oct 29. Erratum in: AJNR Am J Neuroradiol. 2015 May;36(5):E39-40.

ReferenceCitation: Chitale R, Gonzalez LF, Randazzo C, Dumont AS, Tjoumakaris S, Rosenwasser R, Chalouhi N, Gordon D, Jabbour P. Single center experience with pipeline stent: feasibility, technique, and complications. Neurosurgery. 2012 Sep;71(3):679-91; discussion 691. doi: 10.1227/NEU.0b013e318260fe86.

ReferenceCitation: Saatci I, Yavuz K, Ozer C, Geyik S, Cekirge HS. Treatment of intracranial aneurysms using the pipeline flow-diverter embolization device: a single-center experience with long-term follow-up results. AJNR Am J Neuroradiol. 2012 Sep;33(8):1436-46. doi: 10.3174/ajnr.A3246. Epub 2012 Jul 19.

ReferenceCitation: Kan P, Siddiqui AH, Veznedaroglu E, Liebman KM, Binning MJ, Dumont TM, Ogilvy CS, Gaughen JR Jr, Mocco J, Velat GJ, Ringer AJ, Welch BG, Horowitz MB, Snyder KV, Hopkins LN, Levy EI. Early postmarket results after treatment of intracranial aneurysms with the pipeline embolization device: a U.S. multicenter experience. Neurosurgery. 2012 Dec;71(6):1080-7; discussion 1087-8. doi: 10.1227/NEU.0b013e31827060d9.

ReferenceCitation: Yu SC, Kwok CK, Cheng PW, Chan KY, Lau SS, Lui WM, Leung KM, Lee R, Cheng HK, Cheung YL, Chan CM, Wong GK, Hui JW, Wong YC, Tan CB, Poon WL, Pang KY, Wong AK, Fung KH. Intracranial aneurysms: midterm outcome of pipeline embolization device--a prospective study in 143 patients with 178 aneurysms. Radiology. 2012 Dec;265(3):893-901. doi: 10.1148/radiol.12120422. Epub 2012 Sep 20.

ReferenceCitation: McAuliffe W, Wycoco V, Rice H, Phatouros C, Singh TJ, Wenderoth J. Immediate and midterm results following treatment of unruptured intracranial aneurysms with the pipeline embolization device. AJNR Am J Neuroradiol. 2012 Jan;33(1):164-70. doi: 10.3174/ajnr.A2727. Epub 2011 Oct 6.

ReferenceCitation: Skukalek SL, Winkler AM, Kang J, Dion JE, Cawley CM, Webb A, Dannenbaum MJ, Schuette AJ, Asbury B, Tong FC. Effect of antiplatelet therapy and platelet function testing on hemorrhagic and thrombotic complications in patients with cerebral aneurysms treated with the pipeline embolization device: a review and meta-analysis. J Neurointerv Surg. 2016 Jan;8(1):58-65. doi: 10.1136/neurintsurg-2014-011145. Epub 2014 Nov 10. Review.

ReferenceCitation: Rice H, Martínez Galdámez M, Holtmannspötter M, Spelle L, Lagios K, Ruggiero M, Vega P, Sonwalkar H, Chapot R, Lamin S. Periprocedural to 1-year safety and efficacy outcomes with the Pipeline Embolization Device with Shield technology for intracranial aneurysms: a prospective, post-market, multi-center study. J Neurointerv Surg. 2020 Nov;12(11):1107-1112. doi: 10.1136/neurintsurg-2020-015943. Epub 2020 Jun 1.

family: Rice

full_name: Hal Rice

given: Hal

family: Martínez Galdámez

full_name: Mario Martínez Galdámez

given: Mario

family: Holtmannspötter

full_name: Markus Holtmannspötter

given: Markus

family: Spelle

full_name: Laurent Spelle

given: Laurent

family: Lagios

full_name: Konstantinos Lagios

given: Konstantinos

family: Ruggiero

full_name: Maria Ruggiero

given: Maria

family: Vega

full_name: Pedro Vega

given: Pedro

family: Sonwalkar

full_name: Hemant Sonwalkar

given: Hemant

family: Chapot

full_name: René Chapot

given: René

family: Lamin

full_name: Saleh Lamin

given: Saleh

journal_name: Journal of NeuroInterventional Surgery

evidence: open (via page says license)

license: cc-by-nc

license_normalized: cc-by-nc

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

ipd_sharing_description: No individual participant data will be available

lead_sponsor: Medtronic Neurovascular Clinical Affairs

location_country:
Italy
Finland
France
Greece
Israel
Hungary
Australia
Spain
Denmark
Germany
United Kingdom

location_facility: Hospital Universitario Central de Asturias


title: EnSite™ HD Grid Catheter Mapping System for Advanced High Density Three-Dimensional Mapping in Non-Paroxysmal Atrial Fibrillation and Atrial Tachycardia

summary: In this clinical investigation, the safety, feasibility and performance of the novel EnSite™ HD Grid Catheter mapping system for advanced high-density three-dimensional mapping will be studied in patients undergoing catheter ablation procedures for the treatment of non-paroxysmal atrial fibrillation (AF) or left atrial tachycardia (AT).

primary_purpose: Diagnostic

lead_sponsor: Abbott Medical Devices

location_country:
Italy
Hong Kong
France
Australia
Germany

location_facility: Herzzentrum Dresden GmbH Universitatsklinik


title: Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene

acronym: REVERSE

summary: The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for more than six months and up to one year.

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

family: Newman

full_name: Nancy J. Newman

given: Nancy J.

family: Yu-Wai-Man

full_name: Patrick Yu-Wai-Man

given: Patrick

family: Carelli

full_name: Valerio Carelli

given: Valerio

family: Biousse

full_name: Valerie Biousse

given: Valerie

family: Moster

full_name: Mark L. Moster

given: Mark L.

family: Vignal-Clermont

full_name: Catherine Vignal-Clermont

given: Catherine

family: Sergott

full_name: Robert C. Sergott

given: Robert C.

family: Klopstock

full_name: Thomas Klopstock

given: Thomas

family: Sadun

full_name: Alfredo A. Sadun

given: Alfredo A.

family: Girmens

full_name: Jean-François Girmens

given: Jean-François

family: La Morgia

full_name: Chiara La Morgia

given: Chiara

family: DeBusk

full_name: Adam A. DeBusk

given: Adam A.

family: Jurkute

full_name: Neringa Jurkute

given: Neringa

family: Priglinger

full_name: Claudia Priglinger

given: Claudia

family: Karanjia

full_name: Rustum Karanjia

given: Rustum

family: Josse

full_name: Constant Josse

given: Constant

family: Salzmann

full_name: Julie Salzmann

given: Julie

family: Montestruc

full_name: François Montestruc

given: François

family: Roux

full_name: Michel Roux

given: Michel

family: Taiel

full_name: Magali Taiel

given: Magali

family: Sahel

full_name: José-Alain Sahel

given: José-Alain

journal_name: Frontiers in Neurology

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

location_country:
Italy
United States
France
Germany
United Kingdom

location_facility: Moorfields Eye Hospital NHS Foundation Trust

location_facility: Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

location_facility: Department of Neurology, University of Munich, Friedrich-Baur-Institute

location_facility: Doheny Eye Center, University of California, Los Angeles

location_facility: Centre National Hospitalier d'Ophtalmologie des Quinze-Vingt

location_facility: IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

location_facility: Department of Ophthalmology, Emory University School of Medicine


title: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene

acronym: RESCUE

summary: The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less.

family: Newman

full_name: Nancy J. Newman

given: Nancy J.

family: Yu-Wai-Man

full_name: Patrick Yu-Wai-Man

given: Patrick

family: Carelli

full_name: Valerio Carelli

given: Valerio

family: Biousse

full_name: Valerie Biousse

given: Valerie

family: Moster

full_name: Mark L. Moster

given: Mark L.

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

title: Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison

ReferenceCitation: Newman NJ, Yu-Wai-Man P, Carelli V, Moster ML, Biousse V, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Barboni P, DeBusk AA, Girmens JF, Rudolph G, Karanjia R, Taiel M, Blouin L, Smits G, Katz B, Sahel JA; LHON Study Group. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 May;128(5):649-660. doi: 10.1016/j.ophtha.2020.12.012. Epub 2021 Jan 12.

repository_institution: University College London - UCL Discovery

repositories:
HAL
UCL Discovery
www.repository.cam.ac.uk

location_facility: Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt


title: A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

summary: To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A

lead_sponsor: Origin Biosciences

location_country:
France
Malaysia
Israel
Norway
Spain
Turkey
United States
United Kingdom

location_facility: Cincinnati Children's Hospital Medical Center


lead_sponsor: Genzyme, a Sanofi Company

location_country:
France
Poland
Russian Federation
United States
United Kingdom

location_facility:
Investigational Site Number 826002
Investigational Site Number 840001
Investigational Site Number 840003
Investigational Site Number 616001
Investigational Site Number 840002
Investigational Site Number 250001
Investigational Site Number 643002

title: An Open-label, Multicenter, Multinational Extension Study of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Adult Male Patients Diagnosed With Fabry Disease

summary:
Primary Objective:

To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).

Secondary Objective:

To assess the long-term effect of GZ/SAR402671 on pharmacodynamic and exploratory efficacy endpoints in adult male participants with Fabry disease who previously completed study ACT13739.


ReferenceCitation: Jia Y, Juarez J, Li J, Manuia M, Niederst MJ, Tompkins C, Timple N, Vaillancourt MT, Pferdekamper AC, Lockerman EL, Li C, Anderson J, Costa C, Liao D, Murphy E, DiDonato M, Bursulaya B, Lelais G, Barretina J, McNeill M, Epple R, Marsilje TH, Pathan N, Engelman JA, Michellys PY, McNamara P, Harris J, Bender S, Kasibhatla S. EGF816 Exerts Anticancer Effects in Non-Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor. Cancer Res. 2016 Mar 15;76(6):1591-602. doi: 10.1158/0008-5472.CAN-15-2581. Epub 2016 Jan 29.

family: Jia

full_name: Yong Jia

given: Yong

family: Juarez

full_name: Jose Juarez

given: Jose

family: Li

full_name: Jie Li

given: Jie

family: Manuia

full_name: Mari Manuia

given: Mari

family: Niederst

full_name: Matthew J. Niederst

given: Matthew J.

family: Tompkins

full_name: Celin Tompkins

given: Celin

family: Timple

full_name: Noelito Timple

given: Noelito

family: Vaillancourt

full_name: Mei-Ting Vaillancourt

given: Mei-Ting

family: Pferdekamper

full_name: AnneMarie Culazzo Pferdekamper

given: AnneMarie Culazzo

family: Lockerman

full_name: Elizabeth L. Lockerman

given: Elizabeth L.

full_name: Chun Li

given: Chun

family: Anderson

full_name: Jennifer Anderson

given: Jennifer

family: Costa

full_name: Carlotta Costa

given: Carlotta

family: Liao

full_name: Debbie Liao

given: Debbie

family: Murphy

full_name: Eric Murphy

given: Eric

family: DiDonato

full_name: Michael DiDonato

given: Michael

family: Bursulaya

full_name: Badry Bursulaya

given: Badry

family: Lelais

full_name: Gerald Lelais

given: Gerald

family: Barretina

full_name: Jordi Barretina

given: Jordi

family: McNeill

full_name: Matthew McNeill

given: Matthew

family: Epple

full_name: Robert Epple

given: Robert

family: Marsilje

full_name: Thomas H. Marsilje

given: Thomas H.

family: Pathan

full_name: Nuzhat Pathan

given: Nuzhat

family: Engelman

full_name: Jeffrey A. Engelman

given: Jeffrey A.

family: Michellys

full_name: Pierre-Yves Michellys

given: Pierre-Yves

publisher: American Association for Cancer Research (AACR)

title: EGF816 Exerts Anticancer Effects in Non–Small Cell Lung Cancer by Irreversibly and Selectively Targeting Primary and Acquired Activating Mutations in the EGF Receptor

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

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

location_country:
Italy
United States
Germany
France
Norway
Singapore
Australia
Spain
Canada
Korea, Republic of
Taiwan

location_facility: Massachusetts General Hospital Mass General

title: A Phase Ib/II, Multicenter, Open-label Study of EGF816 in Combination With INC280 in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer.

summary: The study is designed to determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of EGF816 in combination with INC280 and to estimate the preliminary anti-tumor activity of EGF816 in combination with INC280 in patients with advanced non-small cell lung cancer (NSCLC) with documented EGFR mutation.


title: A Phase II, Multicenter, Open-label Study of EGF816 in Combination With Nivolumab in Adult Patients With EGFR Mutated Non-small Cell Lung Cancer and of INC280 in Combination With Nivolumab in Adult Patients With cMet Positive Non-small Cell Lung Cancer

summary: To determine the efficacy and safety of Nivolumab in combination with EGF816 and of Nivolumab in combination with INC280 in previously treated NSCLC patients

location_country:
Italy
United States
Switzerland
Netherlands
France
Singapore
Australia
Spain
Germany

location_facility: University of Texas MD Anderson Cancer Center Thoractic Head/Neck Med.Onc(2)

location_facility: NKI-AVL, Department of Thoracic-Oncology