title: Evaluation of the Clinical Utility and Cost Effectiveness Ratio of Generalized Neonatal Screening for Severe Combined Immunodeficiencies (SCID) by Quantification of TRECs on Guthrie Cards

acronym: DEPISTREC

summary:
Severe combined Immunodeficiencies ( SCID ) are a group of inherited diseases of the immune system by characterised profound abnormalities of T cell development . Infants with SCID require prompt clinical response to Prevent life -threatening infection and studies show significantly improved survival in babies Diagnosed at birth as a result of previous family history . SCID follows criteria for population -based newborn screening since it is asymptomatic at birth and fatal within the first year of life, the confirmation of the disease is easy, there is a curative treatment , and it is known that early stem cell transplantation improves survival . Quantification of TRECs (T- cell receptor excision circles ) in DNA extracted from Guthrie samples is a sensitive screening test for Specific and SCID .

The investigators propose in this study to perform a neonatal screening of SCID , in a population of 200,000 babies over a period of two years .

The investigators propose to study the clinical utility and cost effectiveness ratio, and SCID screening to demonstrate that could result in a broad benefit to Individuals detected , making screening relatively cost-effective in spite of the low incidence of the disease .

primary_purpose: Screening

ReferenceCitation: Thomas C, Durand-Zaleski I, Frenkiel J, Mirallié S, Léger A, Cheillan D, Picard C, Mahlaoui N, Riche VP, Roussey M, Sébille V, Rabetrano H, Dert C, Fischer A, Audrain M. Clinical and economic aspects of newborn screening for severe combined immunodeficiency: DEPISTREC study results. Clin Immunol. 2019 May;202:33-39. doi: 10.1016/j.clim.2019.03.012. Epub 2019 Apr 1.

family: Thomas

full_name: Caroline Thomas

given: Caroline

family: Durand-Zaleski

full_name: Isabelle Durand-Zaleski

given: Isabelle

family: Frenkiel

full_name: Jérôme Frenkiel

given: Jérôme

family: Mirallié

full_name: Sophie Mirallié

given: Sophie

family: Léger

full_name: Alexandra Léger

given: Alexandra

family: Cheillan

full_name: David Cheillan

given: David

family: Picard

full_name: Capucine Picard

given: Capucine

family: Mahlaoui

full_name: Nizar Mahlaoui

given: Nizar

family: Riche

full_name: Valéry-Pierre Riche

given: Valéry-Pierre

family: Roussey

full_name: Michel Roussey

given: Michel

family: Sébille

full_name: Véronique Sébille

given: Véronique

family: Rabetrano

full_name: Hasina Rabetrano

given: Hasina

family: Dert

full_name: Cécile Dert

given: Cécile

family: Fischer

full_name: Alain Fischer

given: Alain

family: Audrain

full_name: Marie Audrain

given: Marie

journal_name: Clinical Immunology

ReferenceCitation: Audrain MAP, Léger AJC, Hémont CAF, Mirallié SM, Cheillan D, Rimbert MGM, Le Thuaut AM, Sébille-Rivain VA, Prat A, Pinel EMQ, Divry E, Dert CGL, Fournier MAG, Thomas CJC. Newborn Screening for Severe Combined Immunodeficiency: Analytic and Clinical Performance of the T Cell Receptor Excision Circle Assay in France (DEPISTREC Study). J Clin Immunol. 2018 Oct;38(7):778-786. doi: 10.1007/s10875-018-0550-7. Epub 2018 Sep 24.

full_name: Marie A. P. Audrain

given: Marie A. P.

full_name: Alexandra J. C. Léger

given: Alexandra J. C.

family: Hémont

full_name: Caroline A. F. Hémont

given: Caroline A. F.

full_name: Sophie M. Mirallié

given: Sophie M.

family: Rimbert

location_facility: Saint-Herblain Private Hospital Polyclinique de l'Atlantique

location_facility: Paris University Hospital Armand-Trousseau

location_facility: Bordeaux Maison de Santé Protestante Bordeaux Bagatelle

location_facility: Angers Private Hospital Clinique de l'Anjou

location_facility: Paris University Hospital La Pitié Salpétrière

location_facility: Nantes Private Hospital Clinique Jules Verne

location_facility: Saint-Martin-d'Hère Private Hospital Clinique Belledonne

location_facility: Strasbourg Private Hospital Clinique Adassa

location_facility: Toulouse University Hospital P. DE VIGUIER

location_facility: Poissy Hospital CHI Poissy-Saint-Germain

location_facility: Aulnay-sous-Bois Hospital CHI Robert Ballanger

location_facility: Toulouse Private Hospital Clinique Sarrus Teinturiers

location_facility: Bordeaux Private Hospital Polyclinique Bordeuax Nord Aquitaine

location_facility: Marseille University Hospital La Conception

location_facility: Rennes Private Hospital Clinique Mutualiste La Sagesse

location_facility: Corbeil Essonnes Hospital Sud Francilien


title: Randomized, Multicenter, Phase II/III Study, Evaluating Fractionated Cisplatin Chemotherapy/Gemcitabine Versus Carboplatin/Gemcitabine in the Treatment of Advanced or Metastatic Urothelial Cancer With Impaired Renal Function.

acronym: VEFORA

summary:
This is a phase II/III, multicenter, randomized study which includes 420 patients on six years + 3 years follow up. 92 patients will be included during the phase II ; additional 328 patients will be included.

Patients with an advanced or metastatic urothelial cancer with impaired renal function will be randomized in one of the two following chemotherapy arm:

Fractionated Cisplatin + Gemcitabine.
Carboplatin + Gemcitabine.

The main objective of the part II study will be to evaluate the efficacy and the safety of a chemotherapy with a doublet platinum salt compound/Gemcitabine with fractionated Cisplatin or Carboplatin in this population.

The main objective of the part III study will be to compare the efficacy in terms of overall survival of a chemotherapy with a doublet platinum salt/Gemcitabine with fractionated Cisplatin or Carboplatin in this population.

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Institut Claudius Regaud

location_facility: Institut de Cancerologie Lucien Neuwirth

location_facility: INSTITUT DE CANCEROLOGIE DE L'OUEST - Site Paul Papin

location_facility: INSTITUT DE CANCEROLOGIE DE L'OUEST - Site René Gauducheau

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: Evaluation of the Efficacy and Safety of an Infant Formula Containing Synbiotics and Its Effects on the Incidence of Infectious Diseases in the Infant Gut : a Double-blind, Randomized, Controlled Interventional Study

acronym: GOLFIII

summary: The primary objective of this study is to demonstrate that a synbiotic formula, fed for the duration of the first year of life (infant and follow-on formula) reduces the incidence rate of episodes of infectious diarrhea in infants during the first year of life compared to a standard infant formula.

status: Unknown status

study_completion_date_type: Anticipated

primary_purpose: Prevention

lead_sponsor: HiPP GmbH & Co. Vertrieb KG

location_facility: C.I.C pédiatrique - C.H.U. de Nantes - Hôpital Mère-Enfant

location_facility: C.I.C Pédiatrique - C.H.U. de Grenoble - Hôpital Couple-Enfant

intervention_type: Dietary Supplement

status_simplified: Unknown


ReferenceType: derived

ReferenceCitation: Tourbah A, Gout O, Vighetto A, Deburghgraeve V, Pelletier J, Papeix C, Lebrun-Frenay C, Labauge P, Brassat D, Toosy A, Laplaud DA, Outteryck O, Moreau T, Debouverie M, Clavelou P, Heinzlef O, De Sèze J, Defer G, Sedel F, Arndt C. MD1003 (High-Dose Pharmaceutical-Grade Biotin) for the Treatment of Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis: A Randomized, Double-Blind, Placebo-Controlled Study. CNS Drugs. 2018 Jul;32(7):661-672. doi: 10.1007/s40263-018-0528-2.

family: Tourbah

full_name: Ayman Tourbah

given: Ayman

sequence: first

family: Gout

full_name: Olivier Gout

given: Olivier

sequence: additional

family: Vighetto

full_name: Alain Vighetto

family: Deburghgraeve

full_name: Véronique Deburghgraeve

family: Pelletier

full_name: Jean Pelletier

given: Jean

family: Papeix

full_name: Caroline Papeix

given: Caroline

family: Lebrun-Frenay

full_name: Christine Lebrun-Frenay

given: Christine

family: Labauge

full_name: Pierre Labauge

given: Pierre

family: Brassat

full_name: David Brassat

family: Toosy

full_name: Ahmed Toosy

given: Ahmed

family: Laplaud

full_name: David-Axel Laplaud

given: David-Axel

family: Outteryck

full_name: Olivier Outteryck

family: Moreau

full_name: Thibault Moreau

given: Thibault

family: Debouverie

full_name: Marc Debouverie

given: Marc

family: Clavelou

full_name: Pierre Clavelou

family: Heinzlef

full_name: Olivier Heinzlef

family: De Sèze

full_name: Jérôme De Sèze

given: Jérôme

family: Defer

full_name: Gilles Defer

given: Gilles

family: Sedel

full_name: Frédéric Sedel

given: Frédéric

family: Arndt

full_name: Carl Arndt

given: Carl

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: CNS Drugs

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via page says license)

host_type: publisher

license: cc-by-nc

license_normalized: cc-by-nc

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Aix-Marseille Université

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

repository_institution: University College London - UCL Discovery

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

location_facility: Centre hospitalier national d'ophtalmologie des Quinze Vingts

location_facility: Centre hospitalier Intercommunal Poissy/Saint-Germain-en-Laye

title: Effect of MD1003 in Chronic Visual Loss Related to Optic Neuritis in Multiple Sclerosis: a Pivotal Randomized Double Masked Placebo Controlled Study

summary: The purpose of this study is to demonstrate the superiority of MD1003 over placebo in the visual improvement of patients suffering from chronic visual loss resulting from multiple sclerosis related optic neuritis.


title: A Randomized Phase III Study of Decitabine (DAC) With or Without Hydroxyurea (HY) Versus HY in Patients With Advanced Proliferative Chronic Myelomonocytic Leukemia (CMML)

acronym: GFM-DAC-CMML

summary:
This is a phase III, two-arm, randomized, stratified, multicenter, open-label study with individual therapeutic benefit aim:

Decitabine (DAC) with or without Hydroxyurea (HY) versus HY in patients with advanced proliferative Chronic Myelomonocytic Leukemia (CMML)

The primary objective of the study is to compare between the two arms Event-free Survival (EFS).

Secondary objectives are to compare between both arms:

Overall Survival (OS) Cumulative incidence of AML Overall and Complete Response Rates at 3 and 6 cycles according to IWG 2006 criteria modified for CMML Response duration Toxicity (hematological and non hematological) Prognostic factors

ReferenceCitation: Pleyer L, Leisch M, Kourakli A, Padron E, Maciejewski JP, Xicoy Cirici B, Kaivers J, Ungerstedt J, Heibl S, Patiou P, Hunter AM, Mora E, Geissler K, Dimou M, Jimenez Lorenzo MJ, Melchardt T, Egle A, Viniou AN, Patel BJ, Arnan M, Valent P, Roubakis C, Bernal Del Castillo T, Galanopoulos A, Calabuig Muñoz M, Bonadies N, Medina de Almeida A, Cermak J, Jerez A, Montoro MJ, Cortés A, Avendaño Pita A, Lopez Andrade B, Hellstroem-Lindberg E, Germing U, Sekeres MA, List AF, Symeonidis A, Sanz GF, Larcher-Senn J, Greil R. Outcomes of patients with chronic myelomonocytic leukaemia treated with non-curative therapies: a retrospective cohort study. Lancet Haematol. 2021 Feb;8(2):e135-e148. doi: 10.1016/S2352-3026(20)30374-4.

family: Pleyer

full_name: Lisa Pleyer

given: Lisa

family: Leisch

full_name: Michael Leisch

given: Michael

family: Kourakli

full_name: Alexandra Kourakli

given: Alexandra

family: Padron

full_name: Eric Padron

given: Eric

family: Maciejewski

full_name: Jaroslaw Pawel Maciejewski

given: Jaroslaw Pawel

family: Xicoy Cirici

full_name: Blanca Xicoy Cirici

given: Blanca

family: Kaivers

full_name: Jennifer Kaivers

given: Jennifer

family: Ungerstedt

full_name: Johanna Ungerstedt

given: Johanna

family: Heibl

full_name: Sonja Heibl

given: Sonja

family: Patiou

full_name: Peristera Patiou

given: Peristera

family: Hunter

full_name: Anthony Michael Hunter

given: Anthony Michael

family: Mora

full_name: Elvira Mora

given: Elvira

family: Geissler

full_name: Klaus Geissler

given: Klaus

family: Dimou

full_name: Maria Dimou

given: Maria

family: Jimenez Lorenzo

full_name: Maria-José Jimenez Lorenzo

given: Maria-José

family: Melchardt

full_name: Thomas Melchardt

given: Thomas

family: Egle

full_name: Alexander Egle

given: Alexander

family: Viniou

full_name: Athina-Nora Viniou

given: Athina-Nora

family: Patel

full_name: Bhumika Jayantibhai Patel

given: Bhumika Jayantibhai

family: Arnan

full_name: Montserrat Arnan

given: Montserrat

family: Valent

full_name: Peter Valent

given: Peter

family: Roubakis

full_name: Christoforos Roubakis

given: Christoforos

family: Bernal del Castillo

full_name: Teresa Bernal del Castillo

given: Teresa

location_facility: IUCT Oncopole - Département d'hématologie


ReferenceCitation: Imdad A, Mackoff SP, Urciuoli DM, Syed T, Tanner-Smith EE, Huang D, Gomez-Duarte OG. Interventions for preventing diarrhoea-associated haemolytic uraemic syndrome. Cochrane Database Syst Rev. 2021 Jul 5;7:CD012997. doi: 10.1002/14651858.CD012997.pub2.

name: Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition; SUNY Upstate Medical University; Syracuse NY USA

family: Imdad

full_name: Aamer Imdad

given: Aamer

name: College of Medicine; SUNY Upstate Medical University; Syracuse NY USA

family: Mackoff

full_name: Samuel P Mackoff

given: Samuel P

family: Urciuoli

full_name: David M Urciuoli

given: David M

name: Meharry Medical College; Nashville Tennessee USA

family: Syed

full_name: Tamkeenat Syed

given: Tamkeenat

name: Counseling Psychology and Human Services; University of Oregon; Eugene Oregon USA

family: Tanner-Smith

full_name: Emily E Tanner-Smith

given: Emily E

name: Department of Pediatrics, Division of Pediatric Nephrology; SUNY Upstate Medical University; Syracuse NY USA

family: Huang

full_name: Dongmei Huang

given: Dongmei

name: Division of Pediatric Infectious Diseases, Department of Pediatrics; University at Buffalo, State University of New York; Buffalo NY USA

family: Gomez-Duarte

full_name: Oscar G Gomez-Duarte

given: Oscar G

journal_name: Cochrane Database of Systematic Reviews

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: Matrat L, Bacchetta J, Ranchin B, Tanné C, Sellier-Leclerc AL. Pediatric atypical hemolytic-uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil? Pediatr Nephrol. 2021 Jun;36(6):1647-1650. doi: 10.1007/s00467-021-05025-8. Epub 2021 Mar 28.

family: Matrat

full_name: Lucie Matrat

given: Lucie

family: Bacchetta

full_name: Justine Bacchetta

given: Justine

family: Ranchin

full_name: Bruno Ranchin

given: Bruno

family: Tanné

full_name: Corentin Tanné

given: Corentin

family: Sellier-Leclerc

full_name: Anne-Laure Sellier-Leclerc

given: Anne-Laure

journal_name: Pediatric Nephrology

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: Pediatric atypical hemolytic–uremic syndrome due to auto-antibodies against factor H: is there an interest to combine eculizumab and mycophenolate mofetil?

ReferenceCitation: Muff-Luett M, Sanderson KR, Engen RM, Zahr RS, Wenderfer SE, Tran CL, Sharma S, Cai Y, Ingraham S, Winnicki E, Weaver DJ, Hunley TE, Kiessling SG, Seamon M, Woroniecki R, Miyashita Y, Xiao N, Omoloja AA, Kizilbash SJ, Mansuri A, Kallash M, Yu Y, Sherman AK, Srivastava T, Nester CM. Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes-a Pediatric Nephrology Research Consortium study. Pediatr Nephrol. 2021 Aug;36(8):2349-2360. doi: 10.1007/s00467-021-04965-5. Epub 2021 Mar 10.

family: Muff-Luett

full_name: Melissa Muff-Luett

given: Melissa

family: Sanderson

full_name: Keia R. Sanderson

given: Keia R.

family: Engen

full_name: Rachel M. Engen

given: Rachel M.

family: Zahr

full_name: Rima S. Zahr

given: Rima S.

family: Wenderfer

full_name: Scott E. Wenderfer

given: Scott E.

family: Tran

full_name: Cheryl L. Tran

given: Cheryl L.

family: Sharma

full_name: Sheena Sharma

given: Sheena

family: Cai

full_name: Yi Cai

given: Yi

repository_institution: University of North Carolina at Chapel Hill - Carolina Digital Repository

title: Eculizumab exposure in children and young adults: indications, practice patterns, and outcomes—a Pediatric Nephrology Research Consortium study

location_facility:
Women, Mother and Child Hospital
Morvan Hospital
Pellegrin Hospital
Necker Hospital
University Hospital
Anne de Bretagne University Hospital
Trousseau Hospital
Jeanne de Flandre Hospital
La Timone Hospital
Purpan Children Hospital
Mother and Child Hospital
Robert Debré Hospital
Clocheville Hospital

title: Early Treatment With the Monoclonal C5 Antibody Eculizumab in Pediatric Patients Affected by Shiga-toxin Related Hemolytic and Uremic Syndrome: A Phase III Prospective Randomized Controlled Therapeutic Trial Versus Placebo

summary:
The investigators aim to perform the first controlled randomized prospective study using ECZ in pediatric STEC-HUS. This is of great interest as there is still no efficient specific therapy in that potentially devastating disease.

Furthermore, published data concerning the use of ECZ in STEC-HUS are controversial, reflecting statistical bias in retrospective or uncontrolled studies, thus emphasizing the need for prospective studies.


type: Other Identifier

title: Etude du Dosage Des d-dimères sériques Pour le Diagnostic d'Exclusion Des Thrombi Intra-atriaux Avant Ablation Endocavitaire de Fibrillation Atriale

summary: The purpose of this study is to determine whether D-dimer dosage allow to exclude risk of intraatrial thrombus before performing an ablation of atrial fibrillation

primary_purpose: Diagnostic

lead_sponsor: Groupe Hospitalier de la Rochelle Ré Aunis

location_facility: Service de cardiologie, Institut Jacques Cartier

location_facility: Service de cardiologie, AP Hôpital la Pitié Salpêtrière

location_facility: Service de cardiologie, Centre Cardiologique du Nord

location_facility: Service de cardiologie, CHU Toulouse Hôtel Dieu St Jacques

location_facility: Service de cardiologie, CHI Aix Pertuis CHPA-CHIAP

location_facility: Service de cardiologie, Polyclinique Vauban

location_facility: Service de cardiologie, Institut Mutualiste Montsouris

location_facility: Service de cardiologie, AP Hôpital Henri Mondor

location_facility: Service de cardiologie, CHU Clermont Ferrand

location_facility: Service de cardiologie, Nouvelles Cliniques Nantaises

location_facility: Service de cardiologie, Groupe Hospitalier Bichat Claude Bernard

location_facility: Service de cardiologie, Clinique Saint Gatien

location_facility: Service de cardiologie, CHR Metz Thionville

location_facility: Service de cardiologie, Clinique Saint Joseph

location_facility: Service de cardiologie, Hôpital Saint Joseph

location_facility: Service de cardiologie, HP Les Franciscaines

location_facility: Service de cardiologie, CHU Saint Etienne

location_facility: Service de cardiologie, HCL Groupement Est

location_facility: Service de cardiologie, APM Hôpital de La Timone

location_facility: Service de cardiologie, CHI Le Raincy Montfermeil

location_facility: Service de cardiologie, CH de la Région d'Annecy

intervention_type: Other

lead_sponsor_type: academique


ReferenceType: result

ReferenceCitation: McCormack FX, Inoue Y, Moss J, Singer LG, Strange C, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, Brown KK, Lynch JP 3rd, Goldberg HJ, Young LR, Kinder BW, Downey GP, Sullivan EJ, Colby TV, McKay RT, Cohen MM, Korbee L, Taveira-DaSilva AM, Lee HS, Krischer JP, Trapnell BC; National Institutes of Health Rare Lung Diseases Consortium; MILES Trial Group. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med. 2011 Apr 28;364(17):1595-606. doi: 10.1056/NEJMoa1100391. Epub 2011 Mar 16.

family: McCormack

full_name: Francis X. McCormack

given: Francis X.

family: Inoue

full_name: Yoshikazu Inoue

given: Yoshikazu

family: Moss

full_name: Joel Moss

given: Joel

family: Singer

full_name: Lianne G. Singer

given: Lianne G.

family: Strange

full_name: Charlie Strange

given: Charlie

family: Nakata

full_name: Koh Nakata

given: Koh

family: Barker

full_name: Alan F. Barker

given: Alan F.

family: Chapman

full_name: Jeffrey T. Chapman

given: Jeffrey T.

family: Brantly

full_name: Mark L. Brantly

given: Mark L.

family: Stocks

full_name: James M. Stocks

given: James M.

family: Brown

full_name: Kevin K. Brown

given: Kevin K.

family: Lynch

full_name: Joseph P. Lynch

given: Joseph P.

suffix: III

family: Goldberg

full_name: Hilary J. Goldberg

given: Hilary J.

family: Young

full_name: Lisa R. Young

given: Lisa R.

family: Kinder

full_name: Brent W. Kinder

given: Brent W.

family: Downey

full_name: Gregory P. Downey

given: Gregory P.

family: Sullivan

full_name: Eugene J. Sullivan

given: Eugene J.

family: Colby

full_name: Thomas V. Colby

given: Thomas V.

family: McKay

full_name: Roy T. McKay

given: Roy T.

family: Cohen

full_name: Marsha M. Cohen

given: Marsha M.

family: Korbee

full_name: Leslie Korbee

given: Leslie

family: Taveira-DaSilva

full_name: Angelo M. Taveira-DaSilva

given: Angelo M.

family: Lee

full_name: Hye-Seung Lee

given: Hye-Seung

family: Krischer

full_name: Jeffrey P. Krischer

given: Jeffrey P.

family: Trapnell

full_name: Bruce C. Trapnell

title: Efficacy and Safety of Sirolimus in Lymphangioleiomyomatosis

ReferenceCitation: Goldberg HJ, Harari S, Cottin V, Rosas IO, Peters E, Biswal S, Cheng Y, Khindri S, Kovarik JM, Ma S, McCormack FX, Henske EP. Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study. Eur Respir J. 2015 Sep;46(3):783-94. doi: 10.1183/09031936.00210714. Epub 2015 Jun 25.

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

location_facility: University of Cincinnati, Department of Internal Medicine, Pulmonary, Critical Care & Sleep Medicine,

location_facility: Center for LAM Research and Clinical Care

title: An Exploratory, Open-label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis

summary: This was an exploratory study to determine whether escalating doses of RAD001 (everolimus) were safe and effective in patients with Lymphangioleiomyomatosis


title: Thelin (Sitaxentan Sodium) Patient Safety Registry A Non-Interventional, Patient Registry For The Collection Of Pre-Defined Safety Data In Patients Prescribed Thelin

acronym: PROSE

summary: The Thelin Patient Safety Registry is a post-marketing program in the European Union (EU) that is designed to supplement the reporting of spontaneous adverse events (AE) and better characterize known and potential safety signals for Thelin. The registry is a secure, restricted access, electronic system which collects anonymous, pre-defined, patient-level data on demographic variables, safety monitoring measurements (i.e. liver function tests, haemoglobin and international normalized ratio (INR) measurements), concomitant medications, information on AEs and Thelin drug discontinuation. Regular review of the data is conducted to assess the frequency of identified safety risks and to monitor for the emergence of new safety signals at monthly pharmacovigilance meetings, quarterly signal detection meetings, and for each Periodic Safety Update Report (PSUR).

status: Terminated

study_type: Observational

time_perspective: Prospective

lead_sponsor: Pfizer

location_country: Germany Belgium France


title: A Phase II Trial of SGI-110 in Patients With IPSS High and Int 2 Myelodysplastic Syndrome, Acute Myeloid Leukemia With 20-30% Marrow Blasts or Chronic Myelomonocytic Leukemia Type 2 Not Responding to Azacitidine or Decitabine After at Least 6 Courses or Relapsing After a Response

summary: Treatment of patients with WHO defined IPSS int 2 and high risk MDS , AML with 20-30% marrow blasts and CMML type 2, after failure of azacitidine or decitabine exposure for at least 6 courses, or relapse after initial response.

lead_sponsor: Groupe Francophone des Myelodysplasies

location_facility: Hopital Purpan Service d'Hématologie Clinique

location_facility: Centre Hospitalier de la région d'Annecy


title: A Randomized, Double-blind, Two Arm, Parallel, Placebo Controlled Study of Rimonabant 20 mg Effect on High Density Lipoprotein Kinetics in Patients With Abdominal Obesity and Additional Cardiometabolic Risk Factors

summary:
The objective of the study is to evaluate the effect of Rimonabant 20mg in comparison to placebo, on HDL and VLDL lipoprotein kinetics, over a 12 months period.

Primary objectives:

To assess effect of Rimonabant on HDL ApoA-I fractional catabolic rate (FCR).

Secondary objectives:

To assess effect of Rimonabant on HDL ApoA-I production rate (PR) and on other lipoprotein kinetics.
To assess effect of Rimonabant on lipids, glycemic and inflammatory parameters
To assess effect of Rimonabant on body composition
To assess safety of Rimonabant

ReferenceCitation: Vergès B, Adiels M, Boren J, Barrett PH, Watts GF, Chan D, Duvillard L, Söderlund S, Matikainen N, Kahri J, Robin I, Taskinen MR. Interrelationships between the kinetics of VLDL subspecies and HDL catabolism in abdominal obesity: a multicenter tracer kinetic study. J Clin Endocrinol Metab. 2014 Nov;99(11):4281-90. doi: 10.1210/jc.2014-2365. Epub 2014 Jul 31.

family: Vergès

full_name: Bruno Vergès

given: Bruno

family: Adiels

full_name: Martin Adiels

given: Martin

family: Boren

full_name: Jan Boren

given: Jan

family: Barrett

full_name: Peter Hugh Barrett

given: Peter Hugh

family: Watts

full_name: Gerald F. Watts

given: Gerald F.

family: Chan

full_name: Dick Chan

given: Dick

family: Duvillard

full_name: Laurence Duvillard

given: Laurence

family: Söderlund

full_name: Sanni Söderlund

given: Sanni

family: Matikainen

full_name: Niina Matikainen

given: Niina

family: Kahri

full_name: Juhani Kahri

given: Juhani

family: Robin

full_name: Isabelle Robin

given: Isabelle

family: Taskinen

full_name: Marja-Riitta Taskinen

given: Marja-Riitta

journal_name: The Journal of Clinical Endocrinology & Metabolism

evidence: open (via free pdf)

license_normalized: no license

repository_institution: Chalmers University of Technology - Chalmers Research

publisher: The Endocrine Society

publisher_dissemination: The Endocrine Society

publisher_group: The Endocrine Society

publisher_normalized: The Endocrine Society

title: Interrelationships Between the Kinetics of VLDL Subspecies and HDL Catabolism in Abdominal Obesity: A Multicenter Tracer Kinetic Study

lead_sponsor: Sanofi

location_country:
Finland
Australia
France
United Kingdom


title: Non Interventional Study Dealing With the Use of Trabectedin (Yondelis®)-Pegylated Liposomal Doxorubicin (Caelyx®) in Patient With Platinum-sensitive Relapse

summary: The French cooperative group GINECO proposes to implement an observational study to describe a real situation, in daily practice tolerance and methods of administration of trabectedin (Yondelis®)-Pegylated liposomal doxorubicin(Caelyx)®, in ovarian platinum sensitive cancer relapse.

ReferenceCitation: Selle F, Heudel PE, Hardy-Bessard AC, Pozet A, Meunier J, Gladieff L, Lotz JP, Provansal M, Augereau P, Berton D, Bonichon-Lamichhane N, Orfeuvre H, Pautier P, Kalbacher E, Tazi Y, Spaeth D. GINECO Prospective Non-interventional PROSPECTYON Study: Trabectedin Plus Pegylated Liposomal Doxorubicin for Platinum-sensitive Recurrent Ovarian Cancer. Anticancer Res. 2020 Jul;40(7):3939-3945. doi: 10.21873/anticanres.14385.

family: SELLE

full_name: FRÉDÉRIC SELLE

given: FRÉDÉRIC

family: HEUDEL

full_name: PIERRE-ETIENNE HEUDEL

given: PIERRE-ETIENNE

family: HARDY-BESSARD

full_name: ANNE-CLAIRE HARDY-BESSARD

given: ANNE-CLAIRE

family: POZET

full_name: ASTRID POZET

given: ASTRID

family: MEUNIER

full_name: JEROME MEUNIER

given: JEROME

family: GLADIEFF

full_name: LAURENCE GLADIEFF

given: LAURENCE

family: LOTZ

full_name: JEAN-PIERRE LOTZ

given: JEAN-PIERRE

family: PROVANSAL

full_name: MAGALI PROVANSAL

given: MAGALI

family: AUGEREAU

full_name: PAULE AUGEREAU

given: PAULE

family: BERTON

full_name: DOMINIQUE BERTON

given: DOMINIQUE

family: BONICHON-LAMICHHANE

full_name: NATHALIE BONICHON-LAMICHHANE

given: NATHALIE

family: ORFEUVRE

full_name: HUBERT ORFEUVRE

given: HUBERT

family: PAUTIER

full_name: PATRICIA PAUTIER

given: PATRICIA

family: KALBACHER

full_name: ELSA KALBACHER

given: ELSA

family: TAZI

full_name: YOUSEF TAZI

given: YOUSEF

family: SPAETH

full_name: DOMINIQUE SPAETH

journal_name: Anticancer Research

publisher: Anticancer Research USA Inc.

publisher_dissemination: Anticancer Research USA Inc.

publisher_group: Anticancer Research USA Inc.

publisher_normalized: Anticancer Research USA Inc.

lead_sponsor: ARCAGY/ GINECO GROUP

location_facility: Centre Hospitalier Universitaire Dupuytren

location_facility: Hôpital Privé Drôme Ardèche - Clinique Pasteur

location_facility: Cabinet d'Oncologie - Hôpital Privé Sainte Marie

location_facility: Centre de Radiothérapie - Clinique Sainte-Anne

location_facility: Hôpital Morvan - Centre Hospitalier Universitaire

location_facility: Hôpital de la Milétrie - Centre Hospitalier Universitaire de Poitiers - Pôle Régional de Cancérologie


title: Multicentric, Randomized, Phase III Trial Comparing 2 Strategies in Patients With Non-squamous Non-small Cell Lung Cancer With Asymptomatic Brain Metastases

summary:
The patients carrying a complicated primary lung cancer brain metastases die in less than 3 months of delay disease in the absence of treatment. The median survival of these patients is approximately six months when the treatment associated with radiotherapy chemotherapy based on cisplatin is now the standard treatment. In most studies the patients die of their brain disease in one case only two, so it is likely that some patients do not require brain irradiation (prognosis in this case is linked to extra-cerebral disease ). The benefits for patients in group B (without systematic irradiation) are not to suffer the side effects of this radiation. The risks are in the same group to see brain metastases become symptomatic.

The role of cerebral radiotherapy in the patients treated with chemotherapy is unclear: should all patients be irradiated systematically (since the "reference" treatment is involved and with the aim of obtaining better control of the brain lesions and maintaining a better neurological status) or should only the patients showing cerebral progression be irradiated (avoidance of possibly useless brain radiotherapy and its side effects). The aim of this study is to better determine the position of cerebral radiotherapy in this context.

Main objective:

determine whether there is a difference in terms of progression-free survival between a therapeutic strategy with initial systematic brain radiotherapy followed by chemotherapy cis-platine/alimta + / - Bevacizumab and strategy with an initial chemotherapy cis-platine/alimta + / - Bevacizumab associated with brain radiotherapy only in cases of cerebral progression in patients with NSCLC with asymptomatic brain metastases

primary_purpose: Other

ReferenceCitation: Monnet I, Vergnenègre A, Robinet G, Berard H, Lamy R, Falchero L, Vieillot S, Schott R, Ricordel C, Chouabe S, Thomas P, Gervais R, Madroszyk A, Abdiche S, Chiappa AM, Greillier L, Decroisette C, Auliac JB, Chouaïd C; GFPC 02-13 (METAL2) investigators. Phase III randomized study of carboplatin pemetrexed with or without bevacizumab with initial versus "at progression" cerebral radiotherapy in advanced non squamous non-small cell lung cancer with asymptomatic brain metastasis. Ther Adv Med Oncol. 2021 Apr 16;13:17588359211006983. doi: 10.1177/17588359211006983. eCollection 2021.

apc_source: doaj

name: Service de Pneumologie, CHI Créteil, Créteil, France

family: Monnet

full_name: Isabelle Monnet

given: Isabelle

name: Service de Pneumologie, CHU Limoges, Limoges, France

family: Vergnenègre

full_name: Alain Vergnenègre

given: Alain

name: Service de Pneumologie, CHU Brest, Brest, France

family: Robinet

full_name: Gilles Robinet

given: Gilles

name: Service de Pneumologie, Hôpital d’instruction des armées Sainte-Anne, Toulon, France

family: Berard

full_name: Henri Berard

given: Henri

name: Service de Pneumologie, CH Bretagne Sud, Lorient, France

family: Lamy

full_name: Regine Lamy

given: Regine

name: Service de Pneumologie, Centre Hospitalier de Villefranche de Rouergue, Villefranche, France

family: Falchero

full_name: Lionel Falchero

given: Lionel

name: Service d’oncologie, Centre Saint Pierre, Perpignan, France

family: Vieillot

full_name: Sabine Vieillot

given: Sabine

name: Service d’Oncologie, Centre Paul Strauss, Strasbourg, France

family: Schott

full_name: Roland Schott

given: Roland

name: Service de Pneumologie, CHU Rennes, Rennes, France

family: Ricordel

full_name: Charles Ricordel

given: Charles

name: Service de Pneumologie, CH Charleville Mézière, Charleville Mézière, France

family: Chouabe

full_name: Stephane Chouabe

given: Stephane

name: Service de Pneumologie, CH de gap, Gap, France

family: Thomas

full_name: Pascal Thomas

given: Pascal

name: Service d’Oncologie, Centre François Baclesse, Caen, France

family: Gervais

full_name: Radj Gervais

given: Radj

name: Service d’Oncologie, Institut Paoli-Calmettes, Marseille, France

family: Madroszyk

full_name: Anne Madroszyk

given: Anne

name: Service d’Oncologie, CH Libourne, Libourne

family: Abdiche

full_name: Samir Abdiche

given: Samir

name: Service de Pneumologie, CH de Quimper, Quimper, France

family: Chiappa

full_name: Anne Marie Chiappa

given: Anne Marie

name: Department of Multidisciplinary Oncology and Therapeutic Innovations, APHM, Hôpital Nord, Marseille, France

family: Greillier

full_name: Laurent Greillier

given: Laurent

name: Service de Pneumologie, CH d’Annecy, Annecy, France

family: Decroisette

full_name: Chantal Decroisette

given: Chantal

name: Service de Pneumologie, CH de Mantes la Jolie, Mantes la Jolie, France

family: Auliac

full_name: Jean Bernard. Auliac

given: Jean Bernard.

name: Service de Pneumologie, CHI Créteil, 40 avenue de Verdun, Créteil, 94010, France

name: Institut Mondor de Recherche Biomédicale, U955 Inserm—Université Paris Est Créteil, Créteil, France

family: Chouaïd

full_name: Christos Chouaïd

given: Christos

currency_apc_doaj: USD

journal_name: Therapeutic Advances in Medical Oncology

oa_colors: green gold

evidence: oa journal (via doaj)

evidence: oa repository (via pmcid lookup)

title: Phase III randomized study of carboplatin pemetrexed with or without bevacizumab with initial versus “at progression” cerebral radiotherapy in advanced non squamous non-small cell lung cancer with asymptomatic brain metastasis

lead_sponsor: Centre Hospitalier Intercommunal Creteil

location_facility: Centre Hospitalier Intercommunal de Cornouaille

location_facility: Institut de Cancérologie de la Loire (I.C.L)

location_facility: Centre d'Oncologie et de Radiothérapie du Pays Basque

location_facility: Centre Hospitalier de la Région d'Annecy (CHRA)

location_facility: Hopital d'Instruction des Armées Sainte Anne


title: Pharmaco-epidemiological Study on the Effect of Retacrit® on Chemotherapy Induced Anaemia in Standard Oncology and Haematology Practice: Impact of Concomitant Iron Supplementation

acronym: SYNERGY

summary: The aim of this study is to describe, in a real-life context, the impact of an epoetin alpha biosimilar, Retacrit®, on anaemia in patients receiving chemotherapy, according to concomitant iron supplementation.

lead_sponsor: Hospira, now a wholly owned subsidiary of Pfizer

location_facility:
Chu Rennes
H.I.A Saint Anne
Ch Duffaut
Chu St Eloi
Clinique des Bonnettes
Ico Angers
Clinique St Faron
Chu Gabriel Montpied
Pole Sante Republique
CHG Cahors
Saint Antoine
Ch Chambery
Hopital Troyes
TENON
Ch Le Mans
Ch St Malo
CH Boulogne-sur-Mer
CH Sainte Musse
Hopital Foch
MEDIPOLE
Clinique Belvédère
Victor Hugo
HARTMANN
Inst. Claudius Regaud
Clinique Pasteur
CHU SUD
CHR
CHU Purpan
Clinique des Dentellieres
Clinique Ste Marie
Hopital A.Pare
Chu Timone
Hopital Des Courses
Clinique St Laurent
Chp St Greg
Cac Jean Godinot
Chu Grenoble
CHU du BOCAGE
Saint Louis
CAC Paul Strauss
CHG
Hopital Louis Pasteur
BEAUJON
Hopital Mignot
CHG William MOREY
Chi Frejus - St Raphael
Chu Estaing
Ch Du Pays D'Aix
IPC
Fondation Saint Joseph
Hopital St Quentin
CHRA
CH Jacques Monod
Hopital Franco Britannique
Centre Oscar Lambret
Ch Valence
Victor Dubos
Hpt Rene Dubos
Clinique de l'Ormeau
CH Emile Muller
Clinique Rambot
Chu Hopital Civil
Clinique CONVERT
CHU Caremeau
Clinique Vitalia Desertines
Icl St Etienne
CHG St Gaudens


title: Pilot Evaluation Of CORACTO® (Rapamycin-Eluting Coronary Stent Delivery System) in The Treatment Of Patients With Coronary Artery Disease

acronym: PILOT-SECRET

summary: The purpose of this study is to evaluate the safety and effectiveness of the CORACTO® (Rapamycin®-Eluting coronary stent delivery system) for the treatment of up to two de novo lesions or restenotic post-PTCA (non-stented) lesions located in up to two epicardial native coronary arteries (maximum one lesion per vessel) suitable for treatment with stents from 2.5 to 4.0 mm in diameter < than 15 mm suitable for treatment with a single CORACTO® stent in a population of 100 patients.

enrollment_type: Anticipated

lead_sponsor: Alvimedica

location_country: Turkey Belgium France Spain

location_facility: Groupe Hospitalier Mutualiste de Grenoble

intervention_type: Device


title: Adjuvant Gemcitabine Versus NEOadjuvant Gemcitabine/Oxaliplatin Plus Adjuvant Gemcitabine in Resectable PAncreatic Cancer: a Randomized Multicenter Phase III Study (NEOPAC Study)

summary:
The outcome of patients with resected pancreatic cancer has significantly been improved by adjuvant chemotherapy. However, a large proportion of patients cannot receive adjuvant chemotherapy due to surgical complications. Neoadjuvant chemotherapy has been shown to be safe and effective and can be applied to all patients. This study should test neoadjuvant chemotherapy in a randomized manner.

Patients with resectable cytologically proven adenocarinoma of the pancreatic head are randomized to arm A or B.

Patients randomized to arm A receive an 8-week neoadjuvant chemotherapy with gemcitabine/oxaliplatin followed by surgery. Thereafter, all patients receive adjuvant gemcitabine for six months.

Patients randomized to arm B undergo surgery and receive the same adjuvant treatment as in arm A.

The primary study-endpoint is the recurrence-free survival. Tumor recurrence are determined by computed tomography in a defined protocol.

Trial with medicinal product

ReferenceCitation: Heinrich S, Pestalozzi B, Lesurtel M, Berrevoet F, Laurent S, Delpero JR, Raoul JL, Bachellier P, Dufour P, Moehler M, Weber A, Lang H, Rogiers X, Clavien PA. Adjuvant gemcitabine versus NEOadjuvant gemcitabine/oxaliplatin plus adjuvant gemcitabine in resectable pancreatic cancer: a randomized multicenter phase III study (NEOPAC study). BMC Cancer. 2011 Aug 10;11:346. doi: 10.1186/1471-2407-11-346.

family: Heinrich

full_name: Stefan Heinrich

given: Stefan

family: Pestalozzi

full_name: Bernhard Pestalozzi

given: Bernhard

family: Lesurtel

full_name: Mickael Lesurtel

given: Mickael

family: Berrevoet

full_name: Frederik Berrevoet

given: Frederik

family: Laurent

full_name: Stéphanie Laurent

given: Stéphanie

family: Delpero

full_name: Jean-Robert Delpero

given: Jean-Robert

family: Raoul

full_name: Jean-Luc Raoul

given: Jean-Luc

family: Bachellier

full_name: Phillippe Bachellier

given: Phillippe

family: Dufour

full_name: Patrick Dufour

given: Patrick

family: Moehler

full_name: Markus Moehler

given: Markus

family: Weber

full_name: Achim Weber

given: Achim

family: Lang

full_name: Hauke Lang

given: Hauke

family: Rogiers

full_name: Xavier Rogiers

given: Xavier

family: Clavien

full_name: Pierre-Alain Clavien

given: Pierre-Alain

currency_apc_doaj: GBP

journal_name: BMC Cancer

license: cc-by

license_normalized: cc-by

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: University of Zurich - Zurich Open Repository and Archive

location_facility:
University Hospital of Marseille
University Hospital of Zurich
University Hospital Mainz
University Hospital of Gent
University Hospital of Strasbourg


title: A Multicenter Open Label Phase II Study of to Assess the Efficacy and Safety of APO866 in the Treatment of Patients With Refractory or Relapsed Cutaneous T-cell Lymphoma

summary: This phase II study is designed to determine the efficacy and safety of APO866 for the treatment of patients with advanced forms of cutaneous T-cell lymphoma (CTCL). APO866 has shown to induce growth inhibition in cultures of human CTCL cells as well as in animal models with subcutaneously implanted human CTCL tumors. APO866 was considered to be safe and well-tolerated in a phase I study that treated 24 patients with advanced cancer. APO866 is administered by intravenous infusion continuously for 96 hours and that is repeated every 4 weeks. Patients will receive 3 cycles of treatment and the primary efficacy endpoint will be assessed at Week 16. patients will be followed up for 12 months

design_allocation: Non-Randomized

lead_sponsor: Onxeo

location_country: Austria Germany France Switzerland

location_facility: University Clinic for Dermatology, Medical Faculty of Mannheim of the Heidelberg University

location_facility: Department of Dermatology, University Hospital of Zürich

location_facility: Department of Dermatology, Medical University Graz


title: Clinical and Molecular Description of PKD1 and PKD2 Mutation Negative Carriers in Autosomal Dominant Polycystic Kidney Disease (ADPKD): The GeneQuest Study

acronym: GeneQuest

summary:
The aim of this study is to identify families with ADPKD , characterize the phenotype and screen for mutations in known genes (PKD1 and PKD2, and then HNF1b and UMOD in PKD1 PKD2 negative carriers).

Genome wide analysis will be performed in families without mutations identified.

ipd_sharing: Yes

ipd_sharing_description: All collected data that underlie results in a publication

lead_sponsor: University Hospital, Brest

location_facility: Centre de néphrologie et de dialyse d'Armorique


title: Pilot Study to Assess Efficacy and Safety of a Quadruple Therapy With Asunaprevir, Daclatasvir, Ribavirin and Pegylated Interferon Alpha-2a in HCV Genotype 4-infected Patients Non-responders to Pegylated Interferon-Ribavirin Regimen

summary:
Success rates, after retreatment with Peg-Interferon/Ribavirin bitherapy, in patients infected with HCV (hepatitis C virus) genotype 4 and non-responders to a first standard treatment, are disappointing. The association of Asunaprevir and Daclatasvir in combination with the standard-of-care bitherapy has been shown to increase the efficacy of the treatment in non-responders genotype 1-infected patients.

Given the absence of current solutions and urgent therapeutic needs for HCV genotype 4-infected patients previously treated with pegylated Interferon/Ribavirin, this pilot study aims to evaluate the efficacy and safety of a quadritherapy associating Asunaprevir, Daclatasvir, pegylated Interferon alpha-2a and Ribavirin, in this very difficult to treat population.

60 subjects will be enrolled.

The primary endpoint will be the rate of sustained virological response (SVR), defined by an undetectable HCV RNA, at Week 36 (12 weeks after the end of a 24 weeks quadritherapy).

lead_sponsor: ANRS, Emerging Infectious Diseases

location_facility:
Hôpital Albert Michallon
Hôpital Henri Mondor
Hôpital Purpan
Hôpital de Brabois
Hôpital Tenon
Institut Arnault Tzank
Hôpital Pitié Salpêtrière
Hôpital Beaujon
Hôpital de l'Hôtel Dieu
Hôpital de l'Archet
Hôpital Saint Antoine
Hôpital de Haut Lévêque
Hôpital Claude Huriez
Hôpital Paul Brousse
Hôpital Jean Verdier
Hôpital de la Croix Rousse
Centre Hospitalier Intercommunal
Hôpital Saint Eloi
Hôpital Charles Nicolle
Hôpital de La Source
Hôpital Cochin
Hôpital AVICENNE
Hôpital Dupuytren
Hôpital Pontchaillou
Fondation Hôpital Saint Joseph


title: Automated Assessment of Pulmonary Vein Isolation (PVI) Using a Novel EP Recording System (CathVision Cube® System)

acronym: PVISION

summary: A prospective, multi-center study with the CathVision Cube® system and the PVI Analyzer software in radiofrequency (RF) and cryo balloon procedures.

status: Recruiting

ipd_sharing: No

lead_sponsor: CathVision ApS

location_country: Belgium France

location_facility:
Universitair Ziekenhuis Brussel
Ghent University Hospital
AZ Sint-Jan Hospital
Clinique Pasteur Toulouse,

status_simplified: Ongoing


title: Combined Application of Remote and Intra-Coronary Ischemic Conditioning in Acute Myocardial Infarction: A Multicenter, Randomized, Controlled Clinical Trial (CARIOCA Study)

acronym: CARIOCA

summary: Infarct size is a major determinant of prognosis after AMI. Evidence indicates that the combination of intracoronary ischemic conditioning (ICIC) and remote ischemic conditioning (RIC) can significantly reduce infarct size in STEMI patients. Whether the combination of these two interventions may improve clinical outcome after STEMI remains unknown. The objective of the present study is to determine whether combination of ICIC and RIC can improve STEMI patients clinical outcome at 6 months.

lead_sponsor: Hospices Civils de Lyon

location_facility:
Hopital Louis Pradel
Universitair Ziekenhuis Antwerpen
CHU de Charleroi
Algemeen Ziekenhuis Sint Jan


title: Compare the Performance of Targeted Biopsy Versus Aspiration Biopsy With a Pipette for the Diagnosis of Endometrial Cancer

summary:
Endometrial cancer is the most common pelvic gynecological cancer in so-called developed countries, with 320,000 new cases annually, including more than 1,500 in Belgium. It preferentially affects postmenopausal women. Overall survival at 5 years is 76% but is 95% for early forms, which represent more than 70% of diagnoses. The main risk factors are obesity, diabetes and tamoxifen intake for breast cancer, which explains the increasing incidence. Half a million new annual cases are expected in 2035.

The main symptom is postmenopausal metrorrhagia.Among these women, the prevalence of the disease is estimated between 10 and 15%. Currently the recommendations are to make an evaluation by endovaginal ultrasound followed by an endometrial biopsy. The histological type is the main predictor of the severity of the disease and acts as guideline for the treatment.It is therefore essential to have precise biopsy results before starting therapeutic management.

The most widely used technique is the blind biopsy by aspiration using a pipette because it is inexpensive, easy, without any specific equipment. However, recent studies showed that this technique has a poor sensitivity in the target population of postmenopausal women. Other studies have shown that targeted biopsies under hysteroscopic control could have a much higher sensitivity.

The main objective of this study is thus to compare the performance of the targeted biopsy under hysteroscopy to the performance of the biopsy by aspiration with a pipette, for the diagnosis of endometrial cancer.

status: Withdrawn

lead_sponsor: Brugmann University Hospital

location_facility:
Hôpitaux Iris Sud - Site Ixelles
CHR Citadelle
Hôpital Bicêtre
CHU Brugmann

intervention_type: Procedure


title: Multicenter, Open-label, Phase 1, Dose-escalation, Cohort-expansion, First-in-Human Study of KHK2455 Administered as Monotherapy and in Combination With Mogamulizumab in Adult Subjects With Locally Advanced or Metastatic Solid Tumors

summary: The purpose of this study is to characterize the safety, tolerability, and determine the maximum tolerated dose (MTD) or the highest protocol-defined dose, in the absence of exceeding the MTD, of KHK2455 administered orally in combination with mogamulizumab to subjects with locally advanced or metastatic solid tumors.

lead_sponsor: Kyowa Kirin, Inc.

location_country:
United States
France


title: Advanced Cardiac Therapeutics DiamondTemp TempeRAture-Controlled and Contact Sensing RF Ablation Clinical Trial for Atrial Fibrillation

acronym: TRAC-AF

summary: The primary objective of this study is to establish the safety and performance effectiveness of the DiamondTemp System for the treatment of drug refractory, recurrent, symptomatic paroxysmal atrial fibrillation (PAF).

ReferenceCitation: Starek Z, Lehar F, Jez J, Pesl M, Neuzil P, Sediva L, Petru J, Dujka L, Funasako M, Kautzner J, Peichl P, Aldhoon B, Albenque JP, Combes S, Boveda S, Dukkipati SR, Reddy VY. Efficacy and safety of novel temperature-controlled radiofrequency ablation system during pulmonary vein isolation in patients with paroxysmal atrial fibrillation: TRAC-AF study. J Interv Card Electrophysiol. 2021 Jun 5. doi: 10.1007/s10840-021-00986-0. [Epub ahead of print]

family: Starek

full_name: Zdenek Starek

given: Zdenek

family: Lehar

full_name: Frantisek Lehar

given: Frantisek

family: Jez

full_name: Jiri Jez

given: Jiri

family: Pesl

full_name: Martin Pesl

given: Martin

family: Neuzil

full_name: Petr Neuzil

given: Petr

family: Sediva

full_name: Lucie Sediva

given: Lucie

family: Petru

full_name: Jan Petru

given: Jan

family: Dujka

full_name: Libor Dujka

given: Libor

family: Funasako

full_name: Moritoshi Funasako

given: Moritoshi

family: Kautzner

full_name: Josef Kautzner

given: Josef

family: Peichl

full_name: Petr Peichl

family: Aldhoon

full_name: Bashar Aldhoon

given: Bashar

family: Albenque

full_name: Jean-Paul Albenque

given: Jean-Paul

family: Combes

full_name: Stephane Combes

given: Stephane

family: Boveda

full_name: Serge Boveda

given: Serge

family: Dukkipati

full_name: Srinivas R. Dukkipati

given: Srinivas R.

family: Reddy

full_name: Vivek Y. Reddy

given: Vivek Y.

journal_name: Journal of Interventional Cardiac Electrophysiology

oa_host_type: repository

repository_institution: Authorea - Authorea

repository_normalized: Authorea - Authorea

version: submittedVersion

ReferenceCitation: Iwasawa J, Koruth JS, Petru J, Dujka L, Kralovec S, Mzourkova K, Dukkipati SR, Neuzil P, Reddy VY. Temperature-Controlled Radiofrequency Ablation for Pulmonary Vein Isolation in Patients With Atrial Fibrillation. J Am Coll Cardiol. 2017 Aug 1;70(5):542-553. doi: 10.1016/j.jacc.2017.06.008.

family: Iwasawa

full_name: Jin Iwasawa

given: Jin

family: Koruth

full_name: Jacob S. Koruth

given: Jacob S.

journal_name: Journal of the American College of Cardiology

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_facility: Institut Klinické a Experimentální Medicíny (IKEM)


type: EudraCT Number

title: An Open-Label, Phase I, Dose-Escalation Study Evaluating the Safety, Tolerability, and Pharmacokinetics of GDC-0994 in Patients With Locally Advanced or Metastatic Solid Tumors

summary: This is an open-label, multicenter, dose-escalation study to assess the safety, tolerability, and pharmacokinetics of GDC-0994 in patients with locally advanced or metastatic solid tumors. Patients will be enrolled in one of two stages: a dose-escalation stage (Stage I) or the subsequent expansion stage (Stage II). Stage I will evaluate the safety, tolerability, and pharmacokinetics of increasing doses of GDC-0994 administered daily. Stage II will gather additional data on safety, tolerability, and pharmacokinetics of the recommended dose of GDC-0994 determined in Stage I.

lead_sponsor: Genentech, Inc.

location_facility: Institut Gustave Roussy; Departement Oncologie Medicale


title: A Phase 1 Study of MM-141 in Patients With Advanced Solid Tumors

summary: This study is a Phase 1 and pharmacologic open-labeled dose-escalation trial using a "3+3" design, evaluating MM-141 at varying dose levels and frequencies.

lead_sponsor: Merrimack Pharmaceuticals


title: An Observational Clinical Feasibility Study of The Magnetic Anal Sphincter

summary: The purpose of this feasibility study is to obtain preliminary safety and effectiveness information and develop procedural optimization for the Magnetic Anal Sphincter device (FENIX System) in reinforcement of anal sphincter function to treat severe fecal incontinence (FI).

lead_sponsor: Torax Medical Incorporated

location_country:
Denmark
United States
France

location_facility:
University of Medicine
Colon and Rectal Surgery Associates
Arhus Universitetshospital
Providence Medical Research Center


title: Cryoablation for the Palliation of Painful Bone Metastases

summary: This study will evaluate the safety and efficacy of cryoablation therapy for relief of pain associated with metastatic bone tumors.

primary_purpose: Supportive Care

ReferenceType: background

ReferenceCitation: Callstrom MR, Atwell TD, Charboneau JW, Farrell MA, Goetz MP, Rubin J, Sloan JA, Novotny PJ, Welch TJ, Maus TP, Wong GY, Brown KJ. Painful metastases involving bone: percutaneous image-guided cryoablation--prospective trial interim analysis. Radiology. 2006 Nov;241(2):572-80.

ReferenceCitation: Coleman RE. Management of bone metastases. Oncologist. 2000;5(6):463-70. Review.

ReferenceCitation: Goetz MP, Callstrom MR, Charboneau JW, Farrell MA, Maus TP, Welch TJ, Wong GY, Sloan JA, Novotny PJ, Petersen IA, Beres RA, Regge D, Capanna R, Saker MB, Grönemeyer DH, Gevargez A, Ahrar K, Choti MA, de Baere TJ, Rubin J. Percutaneous image-guided radiofrequency ablation of painful metastases involving bone: a multicenter study. J Clin Oncol. 2004 Jan 15;22(2):300-6.

ReferenceCitation: Callstrom MR, Charboneau JW, Goetz MP, Rubin J, Wong GY, Sloan JA, Novotny PJ, Lewis BD, Welch TJ, Farrell MA, Maus TP, Lee RA, Reading CC, Petersen IA, Pickett DD. Painful metastases involving bone: feasibility of percutaneous CT- and US-guided radio-frequency ablation. Radiology. 2002 Jul;224(1):87-97.

ReferenceCitation: Simon CJ, Dupuy DE. Percutaneous minimally invasive therapies in the treatment of bone tumors: thermal ablation. Semin Musculoskelet Radiol. 2006 Jun;10(2):137-44. Epub 2006 Apr 5. Review.

ReferenceCitation: Ullrick SR, Hebert JJ, Davis KW. Cryoablation in the musculoskeletal system. Curr Probl Diagn Radiol. 2008 Jan-Feb;37(1):39-48. Review.

lead_sponsor: Boston Scientific Corporation

location_country:
United States
Canada
France

location_facility: University of Texas M.D. Anderson Cancer Center


title: Evaluating Cryoablation of Metastatic Lung/Pleura Tumors in Patients - Safety and Efficacy

acronym: ECLIPSE

summary: ECLIPSE will evaluate the safety and efficacy of cryoablation therapy used to treat tumors less than or equal to 3.5 centimeters (cm) in participants with pulmonary metastatic disease.

ReferenceCitation: Gillams A. Lung tumour ablation - where are we now? Cancer Imaging. 2008 Apr 22;8:116-7. doi: 10.1102/1470-7330.2008.0015.

ReferenceCitation: Ahmed A, Littrup P. Percutaneous cryotherapy of the thorax: safety considerations for complex cases. AJR Am J Roentgenol. 2006 Jun;186(6):1703-6.

ReferenceCitation: Asimakopoulos G, Beeson J, Evans J, Maiwand MO. Cryosurgery for malignant endobronchial tumors: analysis of outcome. Chest. 2005 Jun;127(6):2007-14.

ReferenceCitation: Kawamura M, Izumi Y, Tsukada N, Asakura K, Sugiura H, Yashiro H, Nakano K, Nakatsuka S, Kuribayashi S, Kobayashi K. Percutaneous cryoablation of small pulmonary malignant tumors under computed tomographic guidance with local anesthesia for nonsurgical candidates. J Thorac Cardiovasc Surg. 2006 May;131(5):1007-13.

ReferenceCitation: Wang H, Littrup PJ, Duan Y, Zhang Y, Feng H, Nie Z. Thoracic masses treated with percutaneous cryotherapy: initial experience with more than 200 procedures. Radiology. 2005 Apr;235(1):289-98.

ReferenceCitation: Blazeby JM, Avery K, Sprangers M, Pikhart H, Fayers P, Donovan J. Health-related quality of life measurement in randomized clinical trials in surgical oncology. J Clin Oncol. 2006 Jul 1;24(19):3178-86. Review.

ReferenceCitation: Ware J Jr, Kosinski M, Keller SD. A 12-Item Short-Form Health Survey: construction of scales and preliminary tests of reliability and validity. Med Care. 1996 Mar;34(3):220-33.

location_facility:
Institut Gustave Roussy
Ronald Regan UCLA Medical Center
Karmanos Cancer Institute
Mayo Clinic Rochester


title: A Phase IIa, Safety and Preliminary Effects Study of WST11 (Stakel®) Mediated Vascular-Targeted Photodynamic (VTP) Therapy in Subjects With Choroidal Neovascularization (CNV) Associated With Age-Related Macular Degeneration (AMD)

summary:
The objectives of this study are to evaluate the safety(first objective) and efficacy(second objective)of an experimental drug product,Stakel®, in the treatment of neovascular Age related Macular Degeneration (AMD). The drug product is activated in patients by exposure to light at a specific wavelength ("Vascular Targeted Photodynamic therapy", "VTP"). The exploratory objective is to assess whether it is possible to delay or reduce the requirement for anti Vascular Endothelium Growth Factor (anti VEGF) intravitreal therapy in the first 12 weeks after VTP.

All subjects will have a 52 weeks safety follow up telephone call (Not for Adverse Events (AEs) collection).

ipd_sharing_description: The data are available in case report form for each patient

lead_sponsor: Steba Biotech S.A.

location_facility:
Palmetto Retina Center
Valley Retina Institute
Johns Hopkins,Wilmer Eye Institute
Hotel Dieu de Paris Hospital


title: A Phase 1/2 Multiple Ascending Dose Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of BMS-863233 in Subjects With Advanced and/or Metastatic Solid Tumors

summary: The purpose of this study is to determine safety, tolerability and maximum tolerated dose of BMS-863233 in subjects advanced and/or Metastatic solid tumors.

lead_sponsor: Bristol-Myers Squibb

location_facility:
Dana-Farber Cancer Institute
Karmanos Cancer Institute
Local Institution


title: Long-Term Outcome in Offspring and Mothers of Dexamethasone-Treated Pregnancies at Risk for Classical Congenital Adrenal Hyperplasia Owing to 21-Hydroxylase Deficiency

summary: Congenital adrenal hyperplasia (CAH) is a genetic disorder that affects the amount of steroids that the body forms. The most common form of CAH is 21-hydroxylase deficiency (21OHD), which leads to cortisol deficiency and causes the development of mature masculine characteristics in newborn, prepubescent, and grown females, and prepubescent males. Prenatal treatment with dexamethasone, a corticosteroid, has been shown to reduce the masculinization of genitalia. However, the long-term effects of dexamethasone on the children who received it as fetuses and on mothers who were exposed to it while they were pregnant have not been determined. This study will investigate potential long-term adverse side effects of prenatal dexamethasone treatment in children and young adults who received dexamethasone as fetuses and their mothers who were exposed to it during pregnancy.

lead_sponsor: Office of Rare Diseases (ORD)

location_country:
Brazil
United States
France

location_facility: University of Texas Southwestern Medical Center


type: U.S. NIH Grant/Contract

title: Apparent Mineralocorticoid Excess Syndrome Natural History Clinical Protocol

summary: Apparent mineralocorticoid excess (AME) is a rare inherited disease that can cause severe high blood pressure and low blood potassium in children and adults. It is caused by abnormal hormone metabolism and can be fatal. This study will focus on the genetic basis, natural history, disease progression, and survival of people with AME.

ReferenceCitation: New MI, Geller DS, Fallo F, Wilson RC. Monogenic low renin hypertension. Trends Endocrinol Metab. 2005 Apr;16(3):92-7. Review.

ReferenceCitation: Palermo M, Quinkler M, Stewart PM. Apparent mineralocorticoid excess syndrome: an overview. Arq Bras Endocrinol Metabol. 2004 Oct;48(5):687-96. Epub 2005 Mar 7. Review.

ReferenceCitation: Quinkler M, Bappal B, Draper N, Atterbury AJ, Lavery GG, Walker EA, DeSilva V, Taylor NF, Hala S, Rajendra N, Stewart PM. Molecular basis for the apparent mineralocorticoid excess syndrome in the Oman population. Mol Cell Endocrinol. 2004 Mar 31;217(1-2):143-9.

lead_sponsor: Icahn School of Medicine at Mount Sinai


title: A Phase IIb Extension Study of LdT (Telbivudine), Lamivudine or LdT Plus Lamivudine in Patients With Chronic Hepatitis B Who Have Completed Study NV-02B-003

summary: This is an extension study for patients who have previously completed Idenix Study NV-02B-003. This study is being conducted to compare the safety and effectiveness of treatment beyond 1 year of telbivudine and telbivudine combined with lamivudine, a drug currently approved for the treatment of hepatitis B.

lead_sponsor: Novartis

location_country:
United States
Canada
France
China


title: IMPACT: International Post Market Product Surveillance Study of IntrACranial Aneurysms Treated With an Endovascular Approach

acronym: IMPACT

summary: IMPACT is an observational, post market study designed to provide an ongoing safety and performance evaluation of Stryker Neurovascular devices used for the treatment of intracranial aneurysms with an endovascular approach.

ipd_sharing_description: After completion of the 12-month primary endpoint report, and under the guidance of a physician lead publication committee, a multi-center abstract reporting the results will be prepared and may be presented at a major meeting(s). A multi-center publication may also be prepared for publication in a peer reviewed scientific journal.

lead_sponsor: Stryker Neurovascular

location_country: Italy Austria Finland France

location_facility:
Gemelli Hospital
CHU Tours
Uniklinik Salzburg
Turku University Hospital
CHU Brest


title: HepaStem Long-Term Safety Registry - Registry for Patients Who Have Been Administered HepaStem

acronym: PROLONGSTEM

summary: All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences

status: Enrolling by invitation

time_perspective: Other

lead_sponsor: Promethera Biosciences

location_country: Belgium France Spain

location_facility:
UZ Leuven
CHU Toulouse
Hospital de Badajoz
UCL
CHU Lille


title: BIOTRONIK 4French for AMBulatory Peripheral Intervention. A Multicenter, Controlled Trial Comparing 4French Versus 6French Femoral Access for Endovascular Treatment of Lower-extremity Peripheral Artery Disease in an Ambulatory Setting: BIO4AMB

summary: BIOTRONIK 4French for AMBulatory peripheral intervention. A multicenter, controlled trial comparing 4French versus 6French femoral access for endovascular treatment of lower-extremity peripheral artery disease in an ambulatory setting: BIO4AMB

lead_sponsor: Biotronik AG

location_country: Austria Belgium France Switzerland

location_facility:
Medizinische Universitaet Graz
Clinique Saint Joseph
A.Z.Sint-Blasius
CHU du Bocage
Osepedale Regionale di Lugano


title: Clinical Evaluation of the Carmat Total Artificial Heart for Patients With Advanced Heart Failure

acronym: ADVANCEHF

summary:
The objective of this clinical investigation is to evaluate the safety and performance of the Carmat Total Artificial Heart (TAH) in subjects with advanced heart failure requiring biventricular support.

Each subject receiving the Carmat TAH will be evaluated at 6 months (180 days) for primary and secondary endpoints with further follow-up assessments up to 2 years.

The results of the study will be used to support a CE mark application.

ReferenceCitation: Netuka I, Pya Y, Bekbossynova M, Ivak P, Konarik M, Gustafsson F, Smadja DM, Jansen P, Latrémouille C. Initial bridge to transplant experience with a bioprosthetic autoregulated artificial heart. J Heart Lung Transplant. 2020 Dec;39(12):1491-1493. doi: 10.1016/j.healun.2020.07.004. Epub 2020 Jul 14.

family: Netuka

full_name: Ivan Netuka

given: Ivan

family: Pya

full_name: Yuriy Pya

given: Yuriy

family: Bekbossynova

full_name: Makhabbat Bekbossynova

given: Makhabbat

family: Ivak

full_name: Peter Ivak

given: Peter

family: Konarik

full_name: Miroslav Konarik

given: Miroslav

family: Gustafsson

full_name: Finn Gustafsson

given: Finn

family: Smadja

full_name: David M. Smadja

given: David M.

family: Jansen

full_name: Piet Jansen

given: Piet

family: Latrémouille

full_name: Christian Latrémouille

given: Christian

journal_name: The Journal of Heart and Lung Transplantation

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

ReferenceCitation: Bizouarn P, Roussel JC, Trochu JN, Perlès JC, Latrémouille C. Effects of pre-load variations on hemodynamic parameters with a pulsatile autoregulated artificial heart during the early post-operative period. J Heart Lung Transplant. 2018 Jan;37(1):161-163. doi: 10.1016/j.healun.2017.06.018. Epub 2017 Jul 8.

family: Bizouarn

full_name: Philippe Bizouarn

given: Philippe

family: Roussel

full_name: Jean-Christian Roussel

given: Jean-Christian

family: Trochu

full_name: Jean-Noël Trochu

given: Jean-Noël

family: Perlès

full_name: Jean-Christophe Perlès

given: Jean-Christophe

lead_sponsor: Carmat SA

location_country: Denmark France Czechia Kazakhstan

location_facility: Institute for Clinical and Experimental Medicine

location_facility: CHU Pontchaillou Centre Cardio-Pneumologie

location_facility: "National Research Cardiac Surgery Center"

location_facility: Service de Chirurgie Cardiaque - Institut Cœur Poumon


title: An Open-Label, Phase 1a/1b Study of Ramucirumab in Combination With Other Targeted Agents in Advanced Cancers

summary: The main purpose of this study is to evaluate the safety of ramucirumab in combination with other targeted agents in participants with advanced cancers.

ReferenceCitation: Saleh M, Cassier PA, Eberst L, Naik G, Morris VK, Pant S, Terret C, Gao L, Long A, Mao H, McNeely S, Wagner EK, Carlesi RM, Fu S. Phase I Study of Ramucirumab Plus Merestinib in Previously Treated Metastatic Colorectal Cancer: Safety, Preliminary Efficacy, and Pharmacokinetic Findings. Oncologist. 2020 Nov;25(11):e1628-e1639. doi: 10.1634/theoncologist.2020-0520. Epub 2020 Jul 17.

name: O'Neal Comprehensive Cancer Center of University of Alabama at Birmingham  Birmingham Alabama USA

family: Saleh

full_name: Mansoor Saleh

given: Mansoor

name: Centre Léon Bérard, Department of Medical Oncology  Lyon France

family: Cassier

full_name: Philippe A. Cassier

given: Philippe A.

name: Université Claude Bernard Lyon 1  Lyon France

family: Eberst

full_name: Lauriane Eberst

given: Lauriane

family: Naik

full_name: Gurudatta Naik

given: Gurudatta

name: University of Texas MD Anderson Cancer Center  Houston Texas USA

family: Morris

full_name: Van K. Morris

given: Van K.

family: Pant

full_name: Shubham Pant

given: Shubham

family: Terret

full_name: Catherine Terret

given: Catherine

name: Eli Lilly and Company  Indianapolis Indiana USA

family: Gao

full_name: Ling Gao

given: Ling

family: Long

full_name: Amanda Long

given: Amanda

family: Mao

full_name: Huzhang Mao

given: Huzhang

family: McNeely

full_name: Samuel McNeely

given: Samuel

name: BioStat Solutions, Inc.  Frederick Maryland USA

family: Wagner

full_name: Erin K. Wagner

given: Erin K.

name: Eli Lilly Italia  Sesto Fiorentino Florence Italy

family: Carlesi

full_name: Roberto M. Carlesi

given: Roberto M.

family: Fu

full_name: Siqing Fu

given: Siqing

journal_name: The Oncologist

lead_sponsor: Eli Lilly and Company

location_country:
United States
United Kingdom
France

location_facility: For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 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: A Multicenter, Randomized, Open Label, Single and Multiple Dose Study of the Pharmacokinetics and Pharmacodynamics of 2 Dose Levels of Pantoprazole Sodium Enteric-Coated Spheroid Suspension in Infants Aged 1 Through 11 Months With Presumed GERD

summary: The purpose of this study is to characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profiles to determine the safety and tolerability of single and multiple doses of pantoprazole in infants aged 1 through 11 months.

ReferenceCitation: Tammara BK, Sullivan JE, Adcock KG, Kierkus J, Giblin J, Rath N, Meng X, Maguire MK, Comer GM, Ward RM. Randomized, open-label, multicentre pharmacokinetic studies of two dose levels of pantoprazole granules in infants and children aged 1 month through <6 years with gastro-oesophageal reflux disease. Clin Pharmacokinet. 2011 Aug;50(8):541-50. doi: 10.2165/11591900-000000000-00000.

family: Tammara

full_name: Brinda K. Tammara

given: Brinda K.

family: Sullivan

full_name: Janice E. Sullivan

given: Janice E.

family: Adcock

full_name: Kim G. Adcock

given: Kim G.

family: Kierkus

full_name: Jaroslaw Kierkus

given: Jaroslaw

family: Giblin

full_name: John Giblin

given: John

family: Rath

full_name: Natalie Rath

given: Natalie

family: Meng

full_name: Xu Meng

given: Xu

family: Maguire

full_name: Mary K. Maguire

given: Mary K.

family: Comer

full_name: Gail M. Comer

given: Gail M.

family: Ward

full_name: Robert M. Ward

given: Robert M.

journal_name: Clinical Pharmacokinetics

publisher_dissemination: Springer

publisher_group: Springer

title: Randomized, Open-Label, Multicentre Pharmacokinetic Studies of Two Dose Levels of Pantoprazole Granules in Infants and Children Aged 1 Month through <6 Years with Gastro-Oesophageal Reflux Disease

lead_sponsor: Wyeth is now a wholly owned subsidiary of Pfizer

location_country:
Italy
Switzerland
Germany
France
Poland
Belgium
Australia
United States


title: An Open-label Phase 1b/2 Study Assessing the Safety and Efficacy of Mitazalimab in Combination With Chemotherapy in Patients With Metastatic Pancreatic Ductal Adenocarcinoma

summary: Phase 1b/2 study to assess the safety and efficacy of mitazalimab in combination with chemotherapy in patients with metastatic pancreatic ductal adenocarcinoma.

lead_sponsor: Alligator Bioscience AB

location_facility:
Centre Lyon Berard
Cliniques Universitaires St-Luc
Institute de Cancérologie de Lorraine
UZA Antwerp
Hospital Erasme

intervention_type: Biological


title: An Open-Label Multicenter Phase Ib Study of AN0025 in Combination With Pembrolizumab in Patients With Advanced Solid Tumors

summary: This is an open-label, multicenter, phase Ib study to evaluate the safety and preliminary efficacy of AN0025 in combination with pembrolizumab in patients with locally advanced/metastatic tumors. It will include a dose-limiting toxicity observation phase followed by an expansion phase. All enrolled patients will be treated with AN0025 and Pembrolizumab until the patient experiences disease progression, unacceptable toxicity or withdraws consent, or for a maximum of 35 cycles (approximately 2 years). The dose of pembrolizumab will remain constant at 200 mg every 3 weeks (Q3W) for each dose level of AN0025 and in each cohort.

lead_sponsor: Adlai Nortye Biopharma Co., Ltd.

location_facility: University of Utah School of Medicine Huntsman Cancer Institute


title: Safety and Pharmacokinetics of ODM-209 in Patients With Metastatic Castration-resistant Prostate Cancer or Estrogen Receptor-positive, Human Epidermal Growth Factor Receptor 2-negative Advanced Breast Cancer

acronym: STESIDES

summary: The purpose of this first-in-human study is to evaluate safety and tolerability of ODM-209 and find the dose of ODM-209.

lead_sponsor: Orion Corporation, Orion Pharma

location_country: Denmark Finland France

location_facility: Rigshospitalet, University Hospital of Copenhagen


title: Biomarker Research Study for PF-03084014 in cHEmoresistant Triple-negative Breast cAncer

acronym: RHEA

summary: This is a phase II open label Biomarker Research Study off PF-03084014 in non-metastatic triple-negative breast cancer patients with residual disease (cHEmoresistant) after completion of standard neoadjuvant chemotherapy.

lead_sponsor: Jules Bordet Institute

location_facility:
CMSE
Institut de Cancérologie Gustave Roussy
Institut Jules Bordet
Institut Curie
Grand Hopital de Charleroi


summary: This observational study will evaluate patients' perception of treatment with erythropoiesis-stimulating agents in patients with chronic kidney disease not on dialysis. Eligible patients initiated on treatment with Mircera (methoxy polyethylene glycol-epoetin beta) will be followed for 6 months.

lead_sponsor: Hoffmann-La Roche


title: International Validation Study of Sentinel Node Biopsy in Early Cervical Cancer

acronym: SENTICOLIII

summary:
SENTICOL III is large prospective multicenter international randomized study designed to validate the Sentinel Lymph Node (SLN) mapping technique in early cervical cancer. This "validation study" will compare the outcome of patients with negative SLN (experimental arm) vs patients with negative SLN + Pelvic Lymph Node dissection (PLN)(reference arm).

There will be a "quality assurance" program which will be developed in participating centers with detailed requirements in terms of surgeons' qualifications, pathology qualification, SLN ultrastaging, standardization of the procedure, etc. as well as respect of the "safety algorithm".

ReferenceCitation: Lecuru FR, McCormack M, Hillemanns P, Anota A, Leitao M, Mathevet P, Zweemer R, Fujiwara K, Zanagnolo V, Zahl Eriksson AG, Hudson E, Ferron G, Plante M. SENTICOL III: an international validation study of sentinel node biopsy in early cervical cancer. A GINECO, ENGOT, GCIG and multicenter study. Int J Gynecol Cancer. 2019 May;29(4):829-834. doi: 10.1136/ijgc-2019-000332. Epub 2019 Mar 20.

family: Lecuru

full_name: Fabrice R Lecuru

given: Fabrice R

family: McCormack

full_name: Mary McCormack

given: Mary

family: Hillemanns

full_name: Peter Hillemanns

given: Peter

family: Anota

full_name: Amelie Anota

given: Amelie

family: Leitao

full_name: Mario Leitao

given: Mario

family: Mathevet

full_name: Patrice Mathevet

given: Patrice

family: Zweemer

full_name: Ronald Zweemer

given: Ronald

family: Fujiwara

full_name: Keiichi Fujiwara

given: Keiichi

family: Zanagnolo

full_name: Vanna Zanagnolo

given: Vanna

family: Zahl Eriksson

full_name: Ane Gerda Zahl Eriksson

given: Ane Gerda

family: Hudson

full_name: Emma Hudson

given: Emma

family: Ferron

full_name: Gwenael Ferron

given: Gwenael

family: Plante

full_name: Marie Plante

given: Marie

journal_name: International Journal of Gynecologic Cancer

version: acceptedVersion

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

lead_sponsor: Centre Hospitalier Universitaire de Besancon

location_facility: Institut de Cancérologie de l'Ouest - Centre René Gauducheau

location_facility: Institut de Cancérologie de Montpellier Val d'Aurelle

location_facility: Centre de Lutte Contre le Cancer Antoine Lacassagne

location_facility: CHRU de Lille - Hôpital Jeanne de Flandre

location_facility: CHU de Poitiers - Hôpital de la Milétrie

location_facility: Centre de Lutte Contre le Cancer Jean Perrin

location_facility: Institut Universitaire du Cancer de Toulouse

location_facility: CHU de Clermont-Ferrand - Hôpital d'Estaing

location_facility: Centre de Lutte Contre le Cancer Gustave Roussy

location_facility: Centre Hospitalier Bichat - Claude Bernard

location_facility: Groupement Hospitalier Est - Hôpital Femme Mère Enfant

location_facility: Center de Lutte Contre le Cancer François Baclesse

location_facility: Groupe Hospitalier Diaconesses Croix Saint Simon


title: Randomized, Double-blind, Phase 3 Study of Tucatinib or Placebo in Combination With Ado-trastuzumab Emtansine (T-DM1) for Subjects With Unresectable Locally-advanced or Metastatic HER2+ Breast Cancer (HER2CLIMB-02)

summary:
This study is being done to see if tucatinib with ado-trastuzumab emtansine (T-DM1) works better than T-DM1 alone to help patients who have a specific type of breast cancer called HER2 positive breast carcinoma. The breast cancer in this study is either metastatic (spread into other parts of the body) or cannot be removed completely with surgery.

Patients in this study will be randomly assigned to get either tucatinib or placebo (a pill with no medicine). This is a blinded study, so neither patients nor their doctors will know whether a patient gets tucatinib or placebo. All patients in the study will get T-DM1, a drug that is often used to treat this cancer.

Each treatment cycle lasts 21 days. Patients will swallow tucatinib pills or placebo pills two times every day. Patients will get T-DM1 injections from the study site staff on the first day of every cycle.

lead_sponsor: Seagen Inc.

location_country:
Italy
Austria
United States
Sweden
Switzerland
Netherlands
Germany
France
Japan
Israel
Singapore
Australia
Belgium
Spain
Denmark
Canada
United Kingdom
Korea, Republic of

location_facility: SCL Health Good Samaritan Medical Center Cancer Centers of Colorado

location_facility: Klinikum Rechts der Isar der Technischen Universitat Munchen

location_facility: The Royal Marsden NHS Foundation Trust (RM)

location_facility: LKH Salzburg, Universitatsklinikum der PMU

location_facility: Ft Wayne Medical Oncology and Hematology, Inc TRIO

location_facility: National Hospital Organization Kyushu Cancer Center

location_facility: Texas Oncology - San Antonio Medical Center

location_facility: Huntsman Cancer Institute/University of Utah

location_facility: Grand Hôpital de Charleroi - Saint-Joseph

location_facility: InVO- Institut fUr Versorgungsforschung in der onkologie GbR

location_facility: Ironwood Cancer & Research Centers - Chandler

location_facility: CHI Saint Joseph Medical Group Cancer Care Center

location_facility: Oncology and Hematology Assoc of SW VA DBA Blue Ridge Cancer Care

location_facility: Seconda Università degli Studi di Napoli, AOU

location_facility: Cancer Centre of Southeastern Ontario At Kingston General Hospital

location_facility: Chao Family Comprehensive Cancer Center University of California Irvine

location_facility: Kaiser Permanente Moanalua Medical Center

location_facility: Centre Leon Berard - Centre regional de lutte contre le cancer Rhone-Alpes

location_facility: Florida Cancer Specialists - North Region

location_facility: Hospital General Universitario Gregorio Marañon

location_facility: University of Colorado Hospital / University of Colorado

location_facility: Illinois Cancer Specialists - Arlington Heights

location_facility: UNC Lineberger Comprehensive Cancer Center / University of North Carolina

location_facility: University Health Network, Princess Margaret Hospital

location_facility: Queen Elizabeth II Health Sciences Centre

location_facility: Texas Oncology - Methodist Dallas Cancer Center

location_facility: London Health Sciences Centre - Victoria Hospital

location_facility: Helen F. Graham Cancer Center / Christiana Care Health Systems

location_facility: Kliniken Essen-Mitte - Evang. Huyssens-Stiftung

location_facility: MD Anderson Cancer Center / University of Texas

location_facility: Hospital Clinico Universitario de Valencia

location_facility: University of South Alabama - Mitchell Cancer Institute

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

location_facility: Severance Hospital, Yonsei University Health System

location_facility: H. Lee Moffitt Cancer Center and Research Institute

location_facility: California Cancer Associates for Research and Excellence Inc (cCARE)

location_facility: Florida Cancer Specialists - South Region

location_facility: Hakuaikai Sagara Hospital/Breast Surgery

location_facility: Lombardi Cancer Center / Georgetown University Medical Center

location_facility: University Hospital Basel - Brustzentrum

location_facility: Cancer Treatment Centers of America / Western Regional Medical Center

location_facility: Torrance Memorial Physician Network - TRIO

location_facility: Skanes University Hospital - Universitetssjukhus

location_facility: Hopital du Saint-Sacrement, CHU de Quebec-Universite Laval

location_facility: Centre de Lutte contre le Cancer - François Baclesse

location_facility: Centre de Recherche du Centre Hospitalier de l'Universite de Montreal

location_facility: Saint Barnabas Medical Center Cancer Center

location_facility: Institute Curie - Centre de Lutte Contre Le Cancer CLCC de Paris

location_facility: University of Alberta / Cross Cancer Institute

location_facility: Cancer Treatment Centers of America / Eastern Regional Medical Center

location_facility: University of Pennsylvania / Perelman Center for Advanced Medicine

location_facility: University of Michigan Comprehensive Cancer Center

location_facility: University of Ottawa / Ottawa General Hospital

location_facility: Hospital Center Regional University Morvan De Brest

location_facility: Winship Cancer Institute / Emory University School of Medicine

location_facility: North Mississippi Medical Center Hematology Oncology - Tupelo

location_facility: UCLA Medical Center / David Geffen School of Medicine

location_facility: Seoul National University Bundang Hospital

location_facility: Hiroshima City Hiroshima Citizens Hospital

location_facility: Fondazione del Piemonte per l'Oncologia - Istituto di Candiolo FPO - IRCCS

location_facility: Centre Henri Becquerel / Centre Regional de Lutte Contre le Cancer

location_facility: University Hospitals Birmingham NHS Foundation Trust

location_facility: Hopital Saint-Louis / Service d'Hematologie

location_facility: Goteborgs Universitet - Sahlgrenska Akademin - Institutionen For Kliniska Vetenskaper (Institute Of Clinical Sciences)

location_facility: Miami Cancer Institute at Baptist Health, Inc.

location_facility: Hospital Universitario Virgen de la Victoria

location_facility: Universitatsmedizin der Johannes Gutenberg-Universitat Mainz

location_facility: Florida Cancer Specialists - East West Palm Beach, FL (SCRI)

location_facility: Texas Oncology - Baylor Sammons Cancer Center

location_facility: Saint Francis Hospital / Bon Secours - Virginia

location_facility: Institute of Oncology of Southern Switzerland

location_facility: University of Colorado Health Memorial Hospital

location_facility: Brig Center for Cancer Care and Survivorship

location_facility: Policlinico Universitario Agostino Gemelli

location_facility: Kaiser Permanente Medical Center Northern California

location_facility: Kapi'olani Medical Center for Women and Children

location_facility: The Center for Cancer and Blood Disorders: Fortworth

location_facility: University of California Irvine - Newport

location_facility: University of California at San Francisco

location_facility: University of Texas Southwestern Medical Center

location_facility: National University Cancer Institute, Singapore

location_facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust


title: Study of Thrombin Generation and Thrombus Degradation in Cerebral Venous Thrombosis : Correlation With Clinical and Radiological Evolution

acronym: PHRC-TVC

summary:
Cerebral venous thrombosis is considered as a rare type of stroke with an annual incidence of 3 to 4 per million people. It occurs generally in young patients (mean age of occurrence = 40 years) and principally in young females (75%) generally in pregnancy or oral contraceptive use situations.

The onset may be acute (less than 2 days), subacute (between 2 and 30 days) or chronic (more than 30 days). The clinical presentation is highly variable and includes patients with only a mild headache, others with focal neurological deficits and a few with a dramatic syndrome and a coma. Moreover the evolution can be very different with unpredictable outcome: more often it is favorable with a low mortality rate, but in some cases it can be a worse course. The aim of this study is to evaluate the correlation of some biological markers: thrombin generation test and D-Dimers (marker of fibrin generation and degradation) with the type of onset or the wide spectrum of clinical presentations or the different modes of evolution.

All patients over 16 years ago may be included in the program when CVT diagnosis is proved by magnetic resonance angiography (MRA). For each included patient, there are four blood assays: the first just at the time of diagnosis and before the beginning of treatment, the second before the beginning of the oral anticoagulant treatment. The third assay is done in the third month at the time of a MRA. The last assay is made one month after the end of the anticoagulant treatment or in 12th month after the beginning of the disease if the treatment goes on.

For each sample, the investigators perform a thrombin generation test and a D-Dimers measurement.

ReferenceCitation: Triquenot Bagan A, Crassard I, Drouet L, Barbieux-Guillot M, Marlu R, Robinet-Borgomino E, Morange PE, Wolff V, Grunebaum L, Klapczynski F, André-Kerneis E, Pico F, Martin-Bastenaire B, Ellie E, Menard F, Rouanet F, Freyburger G, Godenèche G, Allano HA, Moulin T, Mourey G, Derex L, Berruyer M, Runavot G, Trichet C, Viader F, Le Querrec A, Husein TT, Cluet-Dennetiere S, Macian-Montoro F, Donnard M, Guillon B, Ternisien C, Zuber M, Laplanche S, Tassan P, Peeltier JY, Canaple S, Roussel B, Gaillard N, Scavazza E, Le Cam Duchez V. Cerebral Venous Thrombosis: Clinical, Radiological, Biological, and Etiological Characteristics of a French Prospective Cohort (FPCCVT)-Comparison With ISCVT Cohort. Front Neurol. 2021 Nov 8;12:753110. doi: 10.3389/fneur.2021.753110. eCollection 2021.

family: Triquenot Bagan

full_name: Aude Triquenot Bagan

given: Aude

family: Crassard

full_name: Isabelle Crassard

given: Isabelle

family: Drouet

full_name: Ludovic Drouet

given: Ludovic

family: Barbieux-Guillot

full_name: Marianne Barbieux-Guillot

given: Marianne

family: Marlu

full_name: Raphaël Marlu

given: Raphaël

family: Robinet-Borgomino

full_name: Emmanuelle Robinet-Borgomino

given: Emmanuelle

family: Morange

full_name: Pierre-Emmanuel Morange

given: Pierre-Emmanuel

family: Wolff

full_name: Valérie Wolff

given: Valérie

family: Grunebaum

full_name: Lelia Grunebaum

given: Lelia

family: Klapczynski

full_name: Frédéric Klapczynski

given: Frédéric

family: André-Kerneis

full_name: Elisabeth André-Kerneis

given: Elisabeth

family: Pico

full_name: Fernando Pico

given: Fernando

family: Martin-Bastenaire

full_name: Brigitte Martin-Bastenaire

given: Brigitte

family: Ellie

full_name: Emmanuel Ellie

given: Emmanuel

family: Menard

full_name: Fanny Menard

given: Fanny

family: Rouanet

full_name: François Rouanet

given: François

family: Freyburger

full_name: Geneviève Freyburger

given: Geneviève

family: Godenèche

full_name: Gaëlle Godenèche

given: Gaëlle

family: Allano

full_name: Hong-An Allano

given: Hong-An

family: Moulin

full_name: Thierry Moulin

given: Thierry

family: Mourey

full_name: Guillaume Mourey

given: Guillaume

family: Derex

full_name: Laurent Derex

given: Laurent

family: Berruyer

full_name: Micheline Berruyer

given: Micheline

family: Runavot

title: Cerebral Venous Thrombosis: Clinical, Radiological, Biological, and Etiological Characteristics of a French Prospective Cohort (FPCCVT)—Comparison With ISCVT Cohort

location_facility:
Hôpital Henri Mondor
C.H.U de Strasbourg
Hôpital Neurologique de Lyon
CH Côte Basque
GH Pitié-Salpêtrière
Hôpital Jean Minjioz
C.H. de Versailles
Hôpital Nord de Laënnec
C.H. de Perpignan - Hôpital Saint-Jean
Hôpital Pellegrin - CHU Bordeaux
CHI de Poissy- site de Poissy
CHU Estaing
Hôpital de la Timone
CHU hopitaux de rouen
C.H.I. Eure-Seine
Hôpital Guy de Chauliac
C.H. Saint-Denis
Hôpital de la Cavale Blanche
C.H. Yves Le Foll
Ch Victor Dupouy
Hôpital Côte de Nacre
Chu D' Angers
C.H.U. Limoges
C.H. de Meaux
G.H. Paris Saint-Joseph
C.H.U. de Grenoble
C.H. de La Rochelle
Hôpital J. Monod
CHRU Bretonneau
C.H. François Quesnay
Hôpitaux Civils de Colmar
G.H.U. Carémeau
Chu Amiens
C.H. de Compiègne
CHU-Hôpital Général
C.H.U de Poitiers


title: Description of Sign-and-symptom Associations at Acromegaly Diagnosis.

acronym: ACRO-POLIS

summary: The purpose of this study is to describe most characteristic association of signs and symptoms present at the time of acromegaly diagnosis.

time_perspective: Retrospective

ReferenceCitation: Caron P, Brue T, Raverot G, Tabarin A, Cailleux A, Delemer B, Renoult PP, Houchard A, Elaraki F, Chanson P. Signs and symptoms of acromegaly at diagnosis: the physician's and the patient's perspectives in the ACRO-POLIS study. Endocrine. 2019 Jan;63(1):120-129. doi: 10.1007/s12020-018-1764-4. Epub 2018 Sep 29. Erratum in: Endocrine. 2018 Oct 31;:.

family: Caron

full_name: Philippe Caron

given: Philippe

family: Brue

full_name: Thierry Brue

family: Raverot

full_name: Gérald Raverot

given: Gérald

family: Tabarin

full_name: Antoine Tabarin

given: Antoine

family: Cailleux

full_name: Anne Cailleux

given: Anne

family: Delemer

full_name: Brigitte Delemer

family: Renoult

full_name: Peggy Pierre Renoult

given: Peggy Pierre

family: Houchard

full_name: Aude Houchard

given: Aude

family: Elaraki

full_name: Fatine Elaraki

given: Fatine

family: Chanson

full_name: Philippe Chanson

journal_name: Endocrine

title: Signs and symptoms of acromegaly at diagnosis: the physician’s and the patient’s perspectives in the ACRO-POLIS study

lead_sponsor: Ipsen

location_facility: Chu de Grenoble Hôpital Albert Michallon