ReferenceCitation: Hartmann K, Gotlib J, Akin C, Hermine O, Awan FT, Hexner E, Mauro MJ, Menssen HD, Redhu S, Knoll S, Sotlar K, George TI, Horny HP, Valent P, Reiter A, Kluin-Nelemans HC. Midostaurin improves quality of life and mediator-related symptoms in advanced systemic mastocytosis. J Allergy Clin Immunol. 2020 Aug;146(2):356-366.e4. doi: 10.1016/j.jaci.2020.03.044. Epub 2020 May 11.

family: Hartmann

full_name: Karin Hartmann

given: Karin

family: Gotlib

full_name: Jason Gotlib

given: Jason

family: Akin

full_name: Cem Akin

given: Cem

family: Hermine

full_name: Olivier Hermine

given: Olivier

family: Awan

full_name: Farrukh T. Awan

given: Farrukh T.

family: Hexner

full_name: Elizabeth Hexner

given: Elizabeth

family: Mauro

full_name: Michael J. Mauro

given: Michael J.

family: Menssen

full_name: Hans D. Menssen

given: Hans D.

family: Redhu

full_name: Suman Redhu

given: Suman

family: Knoll

full_name: Stefanie Knoll

given: Stefanie

family: Sotlar

full_name: Karl Sotlar

given: Karl

family: George

full_name: Tracy I. George

given: Tracy I.

family: Horny

full_name: Hans-Peter Horny

given: Hans-Peter

family: Valent

full_name: Peter Valent

given: Peter

family: Reiter

full_name: Andreas Reiter

given: Andreas

family: Kluin-Nelemans

full_name: Hanneke C. Kluin-Nelemans

given: Hanneke C.

journal_name: Journal of Allergy and Clinical Immunology

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

ReferenceCitation: Gotlib J, Kluin-Nelemans HC, George TI, Akin C, Sotlar K, Hermine O, Awan FT, Hexner E, Mauro MJ, Sternberg DW, Villeneuve M, Huntsman Labed A, Stanek EJ, Hartmann K, Horny HP, Valent P, Reiter A. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016 Jun 30;374(26):2530-41. doi: 10.1056/NEJMoa1513098.

location_country:
Austria
United States
Netherlands
Germany
France
Poland
Norway
Australia
Belgium
Turkey
Canada
United Kingdom

location_facility: University of Michigan Comprehensive Cancer Center DeptofMichiganCancerCenter(3)

location_facility: Stanford University Medical Center Stanford University 2

location_facility: Oregon Health and Science University Dept. Hematologic Malignancies

location_facility: Memorial Sloan Kettering Cancer Center Dept. of MSKCC (2)

location_facility: Georgia Health Sciences University Dept.ofMedicalCollegeOfGeorgia

location_facility: Dana Farber Cancer Institute Hematology / Oncology

location_facility: University of California at Los Angeles Dept. of Hematology Clinic

title: A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of 100mg Twice Daily Oral Dosing of Midostaurin Administered to Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease

summary: The purpose of this study was to determine the efficacy and safety of twice daily (bid) oral midostaurin in patients with Aggressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) with or without an Associated Hematological clonal Non-Mast cell lineage Disease (AHNMD).


title: Phase I/II/III Pharmacokinetic and Outcome Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

summary:
Primary Objective:

To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to control of hemorrhaging in both the prophylaxis and on demand groups of IB1001 in subjects with hemophilia B.

Key Secondary Objectives:

To evaluate the ability of IB1001 to provide coverage against bleeding under surgical circumstances; To evaluate the long-term safety and efficacy of IB1001

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Supportive Care

enrollment_type: Actual

ReferenceType: result

ReferenceCitation: Collins PW, Quon DVK, Makris M, Chowdary P, Kempton CL, Apte SJ, Ramanan MV, Hay CRM, Drobic B, Hua Y, Babinchak TJ, Gomperts ED. Pharmacokinetics, safety and efficacy of a recombinant factor IX product, trenonacog alfa in previously treated haemophilia B patients. Haemophilia. 2018 Jan;24(1):104-112. doi: 10.1111/hae.13324. Epub 2017 Aug 17.

name: Arthur Bloom Haemophilia Centre; School of Medicine Cardiff University; Cardiff UK

family: Collins

full_name: P. W. Collins

given: P. W.

sequence: first

name: Hemophilia Treatment Center; Orthopaedic Hospital; Los Angeles CA USA

family: Quon

full_name: D. V. K. Quon

given: D. V. K.

sequence: additional

name: Department of Haematology; Royal Hallamshire Hospital; Sheffield UK

family: Makris

full_name: M. Makris

given: M.

name: Katharine Dormandy Haemophilia Care and Thrombosis Unit; Royal Free Hospital NHS Trust; London UK

family: Chowdary

full_name: P. Chowdary

given: P.

name: Department of Hematology and Medical Oncology; Emory University; Atlanta GA USA

family: Kempton

full_name: C. L. Kempton

given: C. L.

family: Apte

full_name: S. J. Apte

given: S. J.

name: Jehangir Clinical Development Centre; Jehangir Hospital; Pune India

family: Ramanan

full_name: M. V. Ramanan

given: M. V.

name: Manchester Haemophilia Comprehensive Care Centre; Manchester UK

family: Hay

full_name: C. R. M. Hay

given: C. R. M.

name: Clinical Research; Emergent BioSolutions Canada Inc.; Winnipeg MB Canada

family: Drobic

full_name: B. Drobic

given: B.

family: Hua

full_name: Y. Hua

given: Y.

name: Clinical Development and Medical Affairs; Emergent BioSolutions Inc.; Berwyn IL USA

family: Babinchak

full_name: T. J. Babinchak

given: T. J.

name: Consultant for Emergent BioSolutions Inc.; Montrose CO USA

family: Gomperts

full_name: E. D. Gomperts

given: E. D.

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Haemophilia

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via crossref license)

host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

version: publishedVersion

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

host_type: repository

version: acceptedVersion

repository_institution: Cardiff University - ORCA Online Research @Cardiff

repository_normalized: https

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceType: derived

ReferenceCitation: Martinowitz U, Shapiro A, Quon DV, Escobar M, Kempton C, Collins PW, Chowdary P, Makris M, Mannucci PM, Morfini M, Valentino LA, Gomperts E, Lee M. Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis. Haemophilia. 2012 Nov;18(6):881-7. doi: 10.1111/j.1365-2516.2012.02897.x. Epub 2012 Jul 5.

name: Institute of Thrombosis and Hemostasis and the National Hemophilia Center; Sheba Medical Center; Tel Hashomer; Israel

family: Martinowitz

full_name: U. Martinowitz

given: U.

name: Indiana Hemophilia and Thrombosis Center; Indianapolis; IN; USA

family: Shapiro

full_name: A. Shapiro

given: A.

name: The Hemophilia Treatment Center at Los Angeles Orthopaedic Hospital; Los Angeles; CA; USA

full_name: D. V. Quon

given: D. V.

name: University of Texas Houston Health Sciences Center; Houston; TX; USA

family: Escobar

full_name: M. Escobar

name: Aflac Cancer Center and Blood Disorders Service; Emory University School of Medicine; Atlanta; GA; USA

name: Arthur Bloom Haemophilia Centre; University Hospital of Wales School of Medicine; Cardiff University; Cardiff; UK

name: KD Haemophilia Centre and Thrombosis Unit; Royal Free Hospital; London; UK

name: Sheffield Haemophilia and Thrombosis Centre; Royal Hallamshire Hospital; Sheffield; UK

name: Scientific Direction and A. Bianchi Bonomi Hemophilia and Thrombosis Center; IRCCS Cà Granda Foundation Maggiore Policlinico Hospital; Milan; Italy

name: Agency for Haemophilia; Azienda Ospedaliero-Universitaria Careggi; Florence; Italy

name: Rush Hemophilia and Thrombophilia Center; Rush University Medical Center; Chicago; IL; USA

name: Inspiration Biopharmaceuticals; Cambridge; MA; USA

location_country:
Italy
India
France
Poland
Israel
United States
United Kingdom

location_facility: Hemophilia Treatment Center of Las Vegas

location_facility: Rush University Medical Center-Pediatric Hematology Oncology

location_facility: University Hospital of Wales Health Park

location_facility: Sahyadri Specialty Hospital, Deccan Gymkhana

location_facility: University of Texas Health Science Center-Houston, Gulf States Hemophilia & Thrombophilia Center

location_facility: Emory University School of Medicine Pediatric Hematology

location_facility: Centre for Haemostasis and Thrombosis, Basingstoke and North Hampshire Foundation Trust

location_facility: Manchester Haemophilia Comprehensive Care Manchester Royal Infirmary

location_facility: Centre Regional de Traitement de l 'Hemophilie

location_facility: The Hemophilia Treatment Center of Orthopaedic Hospital

location_facility: University of Minnesota Center for Bleeding and Clotting Disorder


ReferenceCitation: Markham A. Brivaracetam: First Global Approval. Drugs. 2016 Mar;76(4):517-22. doi: 10.1007/s40265-016-0555-6. Review.

family: Markham

full_name: Anthony Markham

given: Anthony

journal_name: Drugs

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

title: Brivaracetam: First Global Approval

intervention_type: Drug

lead_sponsor: UCB Pharma

primary_purpose: Treatment

location_country:
Italy
United States
Sweden
Germany
France
Czechia
Hungary
Belgium
Australia
Spain
Canada

type: EudraCT Number

source: euctr

title: An Open-label, Multinational, Multicenter, Follow-up Study to Evaluate the Long-term Safety and Efficacy of Brivaracetam Used at a Flexible Dose up to a Maximum of 200 mg/Day in Subjects Aged 16 Years or Older Suffering From Epilepsy.

summary: The Sponsor wishes to develop brivaracetam as an anti-epileptic treatment in subjects 16 years and older with epilepsy. This study permits continued access to treatment for subjects who participated in a previous epilepsy study. The study will explore the long-term safety and efficacy of brivaracetam.

lead_sponsor_type: industriel

status_simplified: Completed


ReferenceCitation: Tausch E, Beck P, Schlenk RF, Jebaraj BJ, Dolnik A, Yosifov DY, Hillmen P, Offner F, Janssens A, Babu GK, Grosicki S, Mayer J, Panagiotidis P, McKeown A, Gupta IV, Skorupa A, Pallaud C, Bullinger L, Mertens D, Döhner H, Stilgenbauer S. Prognostic and predictive role of gene mutations in chronic lymphocytic leukemia: results from the pivotal phase III study COMPLEMENT1. Haematologica. 2020 Oct 1;105(10):2440-2447. doi: 10.3324/haematol.2019.229161.

family: Eugen Tausch

full_name: Eugen Tausch

family: Philipp Beck

full_name: Philipp Beck

family: Richard F. Schlenk

full_name: Richard F. Schlenk

family: Billy J. Jebaraj

full_name: Billy J. Jebaraj

family: Anna Dolnik

full_name: Anna Dolnik

family: Deyan Y. Yosifov

full_name: Deyan Y. Yosifov

family: Peter Hillmen

full_name: Peter Hillmen

family: Fritz Offner

full_name: Fritz Offner

family: Ann Janssens

full_name: Ann Janssens

family: K. Govind Babu

full_name: K. Govind Babu

family: Sebastian Grosicki

full_name: Sebastian Grosicki

family: Jiri Mayer

full_name: Jiri Mayer

family: Panagiotis Panagiotidis

full_name: Panagiotis Panagiotidis

family: Astrid McKeown

full_name: Astrid McKeown

family: Ira V. Gupta

full_name: Ira V. Gupta

family: Alexandra Skorupa

full_name: Alexandra Skorupa

family: Celine Pallaud

full_name: Celine Pallaud

family: Lars Bullinger

full_name: Lars Bullinger

family: Daniel Mertens

full_name: Daniel Mertens

family: Hartmut Döhner

full_name: Hartmut Döhner

family: Stephan Stilgenbauer

full_name: Stephan Stilgenbauer

currency_apc_doaj: EUR

journal_name: Haematologica

oa_colors: green gold

evidence: oa journal (via doaj)

license: publisher-specific license

license_normalized: publisher-specific

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: Ghent University - Ghent University Academic Bibliography

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repository_normalized: PubMed Central

repository_institution: Max-Delbrueck-Center for Molecular Medicine - MDC Repository

publisher: Ferrata Storti Foundation (Haematologica)

ReferenceCitation: Hillmen P, Robak T, Janssens A, Babu KG, Kloczko J, Grosicki S, Doubek M, Panagiotidis P, Kimby E, Schuh A, Pettitt AR, Boyd T, Montillo M, Gupta IV, Wright O, Dixon I, Carey JL, Chang CN, Lisby S, McKeown A, Offner F; COMPLEMENT 1 Study Investigators. Chlorambucil plus ofatumumab versus chlorambucil alone in previously untreated patients with chronic lymphocytic leukaemia (COMPLEMENT 1): a randomised, multicentre, open-label phase 3 trial. Lancet. 2015 May 9;385(9980):1873-83. doi: 10.1016/S0140-6736(15)60027-7. Epub 2015 Apr 14.

ReferenceCitation: Jewell RC, Laubscher K, Lewis E, Fang L, Gafoor Z, Carey J, McKeown A, West S, Wright O, Sedoti D, Dixon I, Hottenstein CS, Chan G. Assessment of the effect of ofatumumab on cardiac repolarization. J Clin Pharmacol. 2015 Jan;55(1):114-21. doi: 10.1002/jcph.376. Epub 2014 Aug 21.

name: GlaxoSmithKline; Research Triangle Park NC USA

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
Brazil
United States
Sweden
India
Netherlands
Germany
France
Czechia
Greece
Poland
Ireland
Belgium
Russian Federation
Spain
Canada
United Kingdom

title: A Phase III, Open Label, Randomized, Multicenter Trial of Ofatumumab Added to Chlorambucil Versus Chlorambucil Monotherapy in Previously Untreated Patients With Chronic Lymphocytic Leukemia

summary: The purpose of this study was to evaluate the safety and efficacy of ofatumumab added to chlorambucil in patients with untreated Chronic Lymphocytic Leukemia.


title: A Prospective, Double Blind Randomised Controlled Trial to Evaluate the Immunological Benefits and Clinical Effects of an Elimination Diet Using an Amino Acid Based Formula (AAF)

summary: The purpose of this study is to evaluate whether a new version of an amino acid based formula improves tolerance to milk in cow's milk allergic infants/young children.

status: Terminated

ReferenceCitation: Amari S, Shahrook S, Namba F, Ota E, Mori R. Branched-chain amino acid supplementation for improving growth and development in term and preterm neonates. Cochrane Database Syst Rev. 2020 Oct 2;10:CD012273. doi: 10.1002/14651858.CD012273.pub2.

name: Neonatology; National Center for Child Health and Development; Tokyo Japan

family: Amari

full_name: Shoichiro Amari

given: Shoichiro

name: Hamilton Health Sciences; Hamilton Canada

family: Shahrook

full_name: Sadequa Shahrook

given: Sadequa

name: Department of Pediatrics; Saitama Medical Center, Saitama Medical University; Kawagoe Japan

family: Namba

full_name: Fumihiko Namba

given: Fumihiko

name: Global Health Nursing, Graduate School of Nursing Science; St. Luke's International University; Tokyo Japan

family: Ota

full_name: Erika Ota

given: Erika

name: Graduate School of Medicine; Kyoto University; Kyoto Japan

family: Mori

full_name: Rintaro Mori

given: Rintaro

journal_name: Cochrane Database of Systematic Reviews

oa_host_type: repository

ipd_sharing: No

lead_sponsor: Nutricia Research

location_country:
Germany
United Kingdom
France

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

location_facility: David Hyde Allergy Clinic - St. Mary's hospital

intervention_type: Other

status_simplified: Unknown


title: An International, Randomised, Double Blinded, Multi-centre, Active- and Placebo-controlled Dose Response Trial to Evaluate the Efficacy and Safety of SABER-Bupivacaine for Postoperative Pain Control in Patients Undergoing Primary, Elective, Open, Abdominal Hysterectomy

summary: The objective is to identify the optimal dose of SABER-Bupivacaine for postoperative pain control in patients undergoing hysterectomy on the basis of pharmacokinetics, efficacy and safety evaluations. The study duration consists of a screening period up to 14 days and a treatment period 14 days with a long term follow up visit at 6 months. The study will provide further data on the efficacy and safety of the product.

lead_sponsor: Durect

location_country:
Sweden
United Kingdom
France
Hungary
Germany
Latvia


title: A Phase 2, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics and Activity of GS 9450 in Adults With Non-Alcoholic Steatohepatitis (NASH)

summary: The overall purpose of this study is to examine the safety, tolerability, pharmacokinetics (how the body processes a drug), and activity of GS-9450 in preventing liver damage due to scarring, or fibrosis, caused by Non-Alcoholic Steatohepatitis (also known as NASH).

lead_sponsor: Gilead Sciences

location_country:
United States
France


intervention_type: Procedure

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

primary_purpose: Other

location_country: Italy Netherlands France Spain Turkey Germany Ukraine

location_facility: Istanbul Universitesi Cerrahpasa Tip Fakultesi

location_facility: Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii

location_facility: Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite

location_facility: Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik

ipd_sharing: Yes

type: Other Identifier

title: AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS

summary: Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.


title: Phase II Efficacy and Safety Study of Dasatinib in Patients With Chronic and Accelerated Phase Chronic Myeloid Leukemia Relapsing After Allogeneic Blood or Bone Marrow Transplantation

acronym: Dasatinib

summary:
This is a phase II efficacy (indicates the capacity for beneficial change or therapeutic effect) and safety study of Dasatinib in patients with relapsed Chronic Myeloid Leukemia (CML) following a Stem Cell Transplant (SCT) and who are not benefiting from other treatment, such as imatinib therapy.

A relapse is when an illness that has seemed to be getting better, or to have been cured, comes back or gets worse again.

A total of 50 patients ≥18 years of age will be registered on the trial.

design_allocation: Non-Randomized

enrollment_type: Anticipated

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country:
Germany
United Kingdom
France
Switzerland

location_facility:
Hopitaux Universitaires de Geneve
E Herriot Hospital
University Hospital
Hammersmith Hospital
Stiftung Deutsche Klinik für Diagnostik
Uniklinik Leipzig


title: A Randomized, Open-label, Controlled, Multi-center Phase II Study to Evaluate the Efficacy and Safety of Adecatumumab Alone or Sequentially to FOLFOX Relative to FOLFOX After R0 Resection of Colorectal Liver Metastases

summary: The purpose of this study is to determine the effect of adecatumumab alone or following FOLFOX in patients with R0 resected liver metastases from CRC (colorectal carcinoma) and to compare the effect to FOLFOX alone.

lead_sponsor: Amgen Research (Munich) GmbH

location_country: Germany France

location_facility: Klinikum Mannheim GmbH Universitätsklinikum

location_facility: Klinikum am Bruderwald, Sozialstiftung Bamberg

location_facility: Universitätsklinikum der LMU Grosshadern

location_facility: Klinikum der Johannes-Gutenberg Universität


ReferenceCitation: Finn RS, Rugo HS, Gelmon KA, Cristofanilli M, Colleoni M, Loi S, Schnell P, Lu DR, Theall KP, Mori A, Gauthier E, Bananis E, Turner NC, Diéras V. Long-Term Pooled Safety Analysis of Palbociclib in Combination with Endocrine Therapy for Hormone Receptor-Positive/Human Epidermal Growth Factor Receptor 2-Negative Advanced Breast Cancer: Updated Analysis with up to 5 Years of Follow-Up. Oncologist. 2021 May;26(5):e749-e755. doi: 10.1002/onco.13684. Epub 2021 Mar 10.

name: David Geffen School of Medicine at the University of California Los Angeles  Santa Monica California USA

family: Finn

full_name: Richard S. Finn

given: Richard S.

name: University of California San Francisco Comprehensive Cancer Center  San Francisco California USA

family: Rugo

full_name: Hope S. Rugo

given: Hope S.

name: British Columbia Cancer Agency  Vancouver British Columbia Canada

family: Gelmon

full_name: Karen A. Gelmon

given: Karen A.

name: Robert H. Lurie Comprehensive Cancer Center, Feinberg School of Medicine  Chicago Illinois USA

family: Cristofanilli

full_name: Massimo Cristofanilli

given: Massimo

name: IEO European Institute of Oncology, IRCCS  Milan Italy

family: Colleoni

full_name: Marco Colleoni

given: Marco

name: Peter MacCallum Cancer Centre, University of Melbourne  Melbourne Victoria Australia

family: Loi

full_name: Sherene Loi

given: Sherene

family: Schnell

full_name: Patrick Schnell

given: Patrick

family: Lu

full_name: Dongrui R. Lu

given: Dongrui R.

name: Pfizer Oncology  Cambridge Massachusetts USA

family: Theall

full_name: Kathy Puyana Theall

given: Kathy Puyana

full_name: Ave Mori

given: Ave

name: Pfizer Inc.  San Francisco California USA

family: Gauthier

full_name: Eric Gauthier

given: Eric

family: Bananis

full_name: Eustratios Bananis

given: Eustratios

name: Royal Marsden Hospital and Institute of Cancer Research  London United Kingdom

family: Turner

full_name: Nicholas C. Turner

given: Nicholas C.

family: Diéras

full_name: Véronique Diéras

given: Véronique

journal_name: The Oncologist

repository_institution: University of Melbourne - Minerva Access

repository_normalized: University of Melbourne - Minerva Access

repositories: University of Melbourne - Minerva Access

repositories_institution: University of Melbourne - Minerva Access

title: Long‐Term Pooled Safety Analysis of Palbociclib in Combination with Endocrine Therapy for Hormone Receptor‐Positive/Human Epidermal Growth Factor Receptor 2‐Negative Advanced Breast Cancer: Updated Analysis with up to 5 Years of Follow‐Up

ReferenceCitation: Finn RS, Boer K, Bondarenko I, Patel R, Pinter T, Schmidt M, Shparyk YV, Thummala A, Voitko N, Bananis E, McRoy L, Wilner K, Huang X, Kim S, Slamon DJ, Ettl J. Overall survival results from the randomized phase 2 study of palbociclib in combination with letrozole versus letrozole alone for first-line treatment of ER+/HER2- advanced breast cancer (PALOMA-1, TRIO-18). Breast Cancer Res Treat. 2020 Sep;183(2):419-428. doi: 10.1007/s10549-020-05755-7. Epub 2020 Jul 18.

family: Boer

full_name: Katalin Boer

given: Katalin

family: Bondarenko

full_name: Igor Bondarenko

given: Igor

family: Patel

full_name: Ravindranath Patel

given: Ravindranath

repository_institution: Technical University Munich - mediaTUM – the media and publications repository of the Technical University Munich

title: Overall survival results from the randomized phase 2 study of palbociclib in combination with letrozole versus letrozole alone for first-line treatment of ER+/HER2− advanced breast cancer (PALOMA-1, TRIO-18)

ReferenceCitation: Ettl J, Im SA, Ro J, Masuda N, Colleoni M, Schnell P, Bananis E, Lu DR, Cristofanilli M, Rugo HS, Finn RS. Hematologic adverse events following palbociclib dose reduction in patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative advanced breast cancer: pooled analysis from randomized phase 2 and 3 studies. Breast Cancer Res. 2020 Mar 12;22(1):27. doi: 10.1186/s13058-020-01263-0.

title: Hematologic adverse events following palbociclib dose reduction in patients with hormone receptor–positive/human epidermal growth factor receptor 2–negative advanced breast cancer: pooled analysis from randomized phase 2 and 3 studies

ReferenceCitation: Rugo HS, Turner NC, Finn RS, Joy AA, Verma S, Harbeck N, Masuda N, Im SA, Huang X, Kim S, Sun W, Iyer S, Schnell P, Bartlett CH, Johnston S. Palbociclib plus endocrine therapy in older women with HR+/HER2- advanced breast cancer: a pooled analysis of randomised PALOMA clinical studies. Eur J Cancer. 2018 Sep;101:123-133. doi: 10.1016/j.ejca.2018.05.017. Epub 2018 Jul 25.

title: Palbociclib plus endocrine therapy in older women with HR+/HER2– advanced breast cancer: a pooled analysis of randomised PALOMA clinical studies

ReferenceCitation: Diéras V, Rugo HS, Schnell P, Gelmon K, Cristofanilli M, Loi S, Colleoni M, Lu DR, Mori A, Gauthier E, Huang Bartlett C, Slamon DJ, Turner NC, Finn RS. Long-term Pooled Safety Analysis of Palbociclib in Combination With Endocrine Therapy for HR+/HER2- Advanced Breast Cancer. J Natl Cancer Inst. 2019 Apr 1;111(4):419-430. doi: 10.1093/jnci/djy109.

name: University of California San Francisco Helen Diller Family Comprehensive Cancer Center, San Francisco, CA (HSR)

name: British Columbia Cancer Agency, Vancouver, BC, Canada (KG)

name: Robert H. Lurie Comprehensive Cancer Center, Feinberg School of Medicine, Chicago, IL (MC)

name: Peter MacCallum Cancer Centre, University of Melbourne, Melbourne, VIC, Australia (SL)

name: Istituto Europeo di Oncologia, Milan, Italy (MC)

name: David Geffen School of Medicine, Los Angeles, CA (DJS, RSF)

name: Institute of Cancer Research and Royal Marsden Hospital, London, UK (NCT)

journal_name: JNCI: Journal of the National Cancer Institute

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

repository_institution: California Digital Library - eScholarship

repository_normalized: California Digital Library - eScholarship

repositories: California Digital Library - eScholarship

repositories_institution: California Digital Library - eScholarship

ReferenceCitation: Wedam SB, Beaver JA, Amiri-Kordestani L, Bloomquist E, Tang S, Goldberg KB, Sridhara R, Ibrahim A, Kim G, Kluetz P, McKee A, Pazdur R. US Food and Drug Administration Pooled Analysis to Assess the Impact of Bone-Only Metastatic Breast Cancer on Clinical Trial Outcomes and Radiographic Assessments. J Clin Oncol. 2018 Apr 20;36(12):1225-1231. doi: 10.1200/JCO.2017.74.6917. Epub 2018 Mar 9.

name: All authors: Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.

publisher: American Society of Clinical Oncology (ASCO)

publisher_dissemination: American Society of Clinical Oncology (ASCO)

publisher_group: American Society of Clinical Oncology (ASCO)

publisher_normalized: American Society of Clinical Oncology (ASCO)

ReferenceCitation: Bell T, Crown JP, Lang I, Bhattacharyya H, Zanotti G, Randolph S, Kim S, Huang X, Huang Bartlett C, Finn RS, Slamon D. Impact of palbociclib plus letrozole on pain severity and pain interference with daily activities in patients with estrogen receptor-positive/human epidermal growth factor receptor 2-negative advanced breast cancer as first-line treatment. Curr Med Res Opin. 2016 May;32(5):959-65. doi: 10.1185/03007995.2016.1157060. Epub 2016 Mar 2.

name: St. Vincent’s University Hospital, Dublin, Ireland;

name: Orszagos Onkologiai Intezet, Kemoterapia B, Budapest, Hungary;

name: David Geffen School of Medicine at UCLA, Los Angeles, CA, USA

ReferenceCitation: Finn RS, Crown JP, Lang I, Boer K, Bondarenko IM, Kulyk SO, Ettl J, Patel R, Pinter T, Schmidt M, Shparyk Y, Thummala AR, Voytko NL, Fowst C, Huang X, Kim ST, Randolph S, Slamon DJ. The cyclin-dependent kinase 4/6 inhibitor palbociclib in combination with letrozole versus letrozole alone as first-line treatment of oestrogen receptor-positive, HER2-negative, advanced breast cancer (PALOMA-1/TRIO-18): a randomised phase 2 study. Lancet Oncol. 2015 Jan;16(1):25-35. doi: 10.1016/S1470-2045(14)71159-3. Epub 2014 Dec 16.

location_country:
Italy
United States
South Africa
France
Korea, Republic of
Hungary
Ireland
Russian Federation
Spain
Germany
Canada
Ukraine

location_facility: Federal State Budgetary Scientific Institution

location_facility: Department of Oncology and Medical Radiology, SI "DMA of MOH, Ukraine, "MI "Dnipropetrovsk City

location_facility: State Budgetary Healthcare Institution "Samara Regional Clinical Oncology Dispensary"

location_facility: Borsod-Abauj-Zemplen Megyei Korhaz és Egyetemi Oktato Korhaz, Onkologia

location_facility: Municipal Treatment-and-Prophylactic Institution 'Donetsk City Oncological Dispensary' Radiology dep

location_facility: Gemeinschaftspraxis, Onkologischer Schwerpunkt am Oskar-Helene-Heim

location_facility: North Shore Oncology-Hematology Associates

location_facility: Hospital General Universitario Vall D'Hebron

location_facility: CSSS Champlain-Charles-Le Moyne Local HS-0054

location_facility: Gwinnett Hospital System Inc.-d/b/a-The Center for Cancer Care

location_facility: Sansum Santa Barbara Medical Foundation Clinic

location_facility: Orszagos Onkologiai Intezet, Kemoterapia B

location_facility: Klinik und Poliklinik fuer Frauenheilkunde und Geburtshilfe Universitaetsklinik Mainz

location_facility: Universitaetsklinik und Poliklinik fuer Gynaekologie, Martin-Luther-Universitaet Halle-Wittenberg

location_facility: National Cancer Center, Center for Breast Cancer

location_facility: TORI Central Administration (Regulatory Managment Only)

location_facility: Torrance Health Association, DBA Torrance Memorial Physician Network/Cancer Care Associates

location_facility: Texas Oncology-Dallas Presbyterian Hospital

location_facility: Petz Aladar Megyei Oktato Korhaz, Onkoradiologia

location_facility: Frauenaerzte Pruener Gang Abts & Partner

location_facility: Department of Oncotherapy, National Hospital

location_facility: UCLA West Medical Pharmacy (Drug Management Only)

location_facility: TORI -US Central Administration (Regulatory Management)

location_facility: Central Hematology Oncology Medical Group, Inc

location_facility: UCLA Hematology/Oncology - Santa Clarita

location_facility: Republican Clinical Oncology Dispensary of the Ministry of Health of Bashkortostan Republic

location_facility: TORI Central Administration (Regulatory Management)

location_facility: US Oncology Research and Clinical Pharmacy

location_facility: Lviv State Oncologic Regional Treatment and Diagnostic Centre

location_facility: Unita' Operativa di Oncologia, Ospedale Civile di Lugo

location_facility: Divisione Oncologia Medica Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, IRST

location_facility: Nationales Centrum fuer Tumorerkrankungen

location_facility: Central Hematology Oncology Medical group Inc.

location_facility: Frauenklinik und Poliklinik Klinikum rechts der Isar, Technische Universitaet Muenchen

location_facility: Fovarosi Onkormanyzat Uzsoki Utcai Korhaz

location_facility: Municipal clinical treatment-and-propyilactic institution "Donetsk regional oncology center',

location_facility: Santa Monica-UCLA Medical Center and Orthopaedic Hospital

location_facility: Ospedale Civile di Faenza Centro Oncologico

location_facility: Drug Management Only: UCLA West Medical Pharmacy

location_facility: Regulatory Office: Comprehensive Cancer Centers of Nevada

location_facility: Central Coast Medical Oncology Corporation

location_facility: Westwood Bowyer Clinic, Peter Morton Medical Building

location_facility: M.O. di Oncologia - Azienda USL di Rimini - Ospedale "Cervesi"

location_facility: Texas Oncology-Baylor Charles A. Sammons Cancer Center

location_facility: Markusovszky Egyetemi Oktatokorhaz, Onkoradiologiai Osztaly

location_facility: CBCC Global Research, Inc. at Comprehensive Blood and Cancer Center

location_facility: Mutterhaus der Borromaeerinnen, Innere Medizin I

location_facility: Szabolcs-Szatmar-Bereg Megyei Korhazak es

location_facility: Azienda Unita Sanitaria Locale di Rimini, U.O. di Oncologia ed Oncoematologia Ospedale degli Infermi

title: PHASE 1/2, OPEN-LABEL, RANDOMIZED STUDY OF THE SAFETY, EFFICACY, AND PHARMACOKINETICS OF LETROZOLE PLUS PD 0332991 (ORAL CDK 4/6 INHIBITOR) AND LETROZOLE SINGLE AGENT FOR THE FIRST-LINE TREATMENT OF ER POSITIVE, HER2 NEGATIVE ADVANCED BREAST CANCER IN POSTMENOPAUSAL WOMEN

summary: The study is aimed to confirm that letrozole + PD 0332991 is safe and tolerable and to assess the effect of the combination on advanced breast cancer


intervention_type: Biological

lead_sponsor: Genzyme, a Sanofi Company

location_country:
Germany
United Kingdom
France
Netherlands

acronym: RISE

location_facility:
King's College Hospital
UMC St Radboud Centraal
St. Johannes-Hospital Duisburg
Royal Bournemouth Hospital
Hopital Avicenne/University
St. James Hospital
Medizinische Hochschule Hannover

title: A Phase II Study of the Efficacy of Rabbit Anti-thymocyte Globulin (rATG) in Patients With Low and Intermediate-1 Risk Myelodysplastic Syndrome

summary: This is a Phase II, single-arm, open-label, multinational, multicenter study of rATG in patients with low or intermediate-1 risk MDS who have either failed 1 prior treatment with growth factor(s), hypomethylating agents (5-azacitidine or decitabine), or the antiangiogenic agents lenalidomide or thalidomide, or who have never been treated for MDS (i.e., treatment-naïve patients).


ReferenceCitation: Kuemmerle-Deschner JB, Ramos E, Blank N, Roesler J, Felix SD, Jung T, Stricker K, Chakraborty A, Tannenbaum S, Wright AM, Rordorf C. Canakinumab (ACZ885, a fully human IgG1 anti-IL-1β mAb) induces sustained remission in pediatric patients with cryopyrin-associated periodic syndrome (CAPS). Arthritis Res Ther. 2011 Feb 28;13(1):R34. doi: 10.1186/ar3266.

family: Kuemmerle-Deschner

full_name: Jasmin B Kuemmerle-Deschner

given: Jasmin B

family: Ramos

full_name: Eduardo Ramos

given: Eduardo

family: Blank

full_name: Norbert Blank

given: Norbert

family: Roesler

full_name: Joachim Roesler

given: Joachim

family: Felix

full_name: Sandra D Felix

given: Sandra D

family: Jung

full_name: Thomas Jung

given: Thomas

family: Stricker

full_name: Kirstin Stricker

given: Kirstin

family: Chakraborty

full_name: Abhijit Chakraborty

given: Abhijit

family: Tannenbaum

full_name: Stacey Tannenbaum

given: Stacey

family: Wright

full_name: Andrew M Wright

given: Andrew M

family: Rordorf

full_name: Christiane Rordorf

given: Christiane

currency_apc_doaj: GBP

journal_name: Arthritis Research & Therapy

license: cc-by

license_normalized: cc-by

repository_institution: PubMed Central - Europe PMC

publisher_dissemination: Springer

publisher_group: Springer

lead_sponsor: Novartis Pharmaceuticals

location_country:
India
France
Spain
Germany
United Kingdom

location_facility:
Novartis Investigative site
Novartis Investigator Site
Novartis investigative site

title: An Open-Label, Phase II Dose Titration Study of ACZ885 (Human Anti-IL-1β Monoclonal Antibody) to Assess the Clinical Efficacy, Safety, Pharmacokinetics and Pharmacodynamics in Patients With NALP3 Mutations

summary: This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are either untreated or insufficiently treated and require medical intervention.


title: A Multicenter, Open, Single Sequence Crossover Study to Assess the Safety and Efficacy of a Tacrolimus Modified Release, FK506E (MR4), Based Immunosuppressive Regimen in Stable Kidney Transplant Patients Converted From a Prograf® Based Immunosuppressive Regimen

summary: Assessment of the safety and the efficacy of a tacrolimus modified release (MR4) based immunosuppressive regimen in stable kidney transplant subjects converted on a 1:1 (mg:mg) basis from a Prograf® based immunosuppressive regimen.

lead_sponsor: Astellas Pharma Inc

location_country:
Netherlands
Poland
France
Spain
Germany
United Kingdom


title: Phase II Study of the Combination of Cisplatin + Temozolomide in Malignant Glial Tumours in Children and Adolescents at Diagnosis or in Relapse

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

PURPOSE: This phase II trial is studying how well giving cisplatin together with temozolomide works in treating young patients with malignant glioma.

status: Unknown status

lead_sponsor: Children's Cancer and Leukaemia Group

location_country:
Ireland
France
United Kingdom

location_facility: Southampton University Hospital NHS Trust

location_facility: Great Ormond Street Hospital for Children NHS Trust

location_facility: Sir James Spence Institute of Child Health

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Central Manchester and Manchester Children's University Hospitals NHS Trust

location_facility: Addenbrooke's Hospital at Cambridge University Hospitals NHS Foundation Trust

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Our Lady's Hospital for Sick Children Crumlin

location_facility: Royal Marsden NHS Foundation Trust - Surrey


title: An Open International Multicentre Long-Term Follow Up Study to Evaluate Safety of Prolonged Release OROS Methlyphenidate in Adults With Attention Deficit Hyperactivity Disorder

summary:
Trial 42603ATT3004 is an open-label extension study to clinical trial 42603ATT3002 (NCT00246220). In trial 42603ATT3002 the efficacy and safety of OROS methylphenidate was assessed in adult subjects with Attention Deficit Hyperactivity Disease (ADHD). ADHD is a developmental disorder beginning in childhood and characterized by developmentally inappropriate inattention, hyperactivity and impulsiveness. Data on the number of adult patients with ADHD is limited, but it is estimated that approximately 50% of children with ADHD will have symptoms also in adhulthood. The drug tested in this trial is OROS methylphenidate. The active ingredient is methylphenidate and the tablet is designed to release the active ingredient gradually to ensure an effect, which lasts up to 12 hours. Trial 42603ATT3002 consisted of a 5-week period, where subjects were assigned to either receive placebo (empty drug) or one out of three different dosages of OROS methylphenidate. This 5-week period was followed by a 7-week period, where patients received OROS methylphenidate at their optimal dose. In study 42603ATT3004, subjects who complete 42603ATT3002 are followed for a period of at least 52 weeks to evaluate safety and tolerability of OROS methylphenidate in patients who are treated with OROS methylphenidate over a long period of time.

Amendment: At the end of the open-label period of the present study 42603ATT3004, patients are enrolled into a double-blind placebo-controlled period, which lasts an additional 4 weeks. The purpose of this double-blind placebo-controlled period is to evaluate the maintenance of effect under continued treatment with OROS methlyphenidate in comparison to treatment cessation in those patients, who are randomized into the placebo-group.

ReferenceCitation: Buitelaar JK, Trott GE, Hofecker M, Waechter S, Berwaerts J, Dejonkheere J, Schäuble B. Long-term efficacy and safety outcomes with OROS-MPH in adults with ADHD. Int J Neuropsychopharmacol. 2012 Feb;15(1):1-13. doi: 10.1017/S1461145711001131. Epub 2011 Jul 29.

family: Buitelaar

full_name: Jan K. Buitelaar

given: Jan K.

family: Trott

full_name: Götz-Erik Trott

given: Götz-Erik

family: Hofecker

full_name: Maria Hofecker

given: Maria

family: Waechter

full_name: Sandra Waechter

given: Sandra

family: Berwaerts

full_name: Joris Berwaerts

given: Joris

family: Dejonkheere

full_name: Joachim Dejonkheere

family: Schäuble

full_name: Barbara Schäuble

given: Barbara

journal_name: The International Journal of Neuropsychopharmacology

evidence: open (via page says Open Access)

license: implied-oa

license_normalized: implied-oa

license: cc-by-nc-sa

license_normalized: cc-by-nc-sa

repository_normalized: CiteSeerX

version: submittedVersion

evidence: oa repository (semantic scholar lookup)

repositories:
CiteSeerX
PubMed Central
pdfs.semanticscholar.org

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

lead_sponsor: Janssen-Cilag International NV

location_country: Switzerland Netherlands Spain France Norway Portugal Germany


title: A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease

summary: The purpose of this study is to determine whether the response to treatment for extensive chronic Graft versus Host Disease (cGvHD)is improved with the addition of myfortic alongside cyclosporine A and prednisone, compared to the reference treatment of cyclosporine A and prednisone alone.

location_country: Italy Sweden Switzerland Netherlands France Spain Turkey Germany

location_facility:
University Faculty of Medicine
Hospital Clínico Universitario
Ospedale San Martino
University Hospital
Karolinska University Hospital
University Regensburg
Hopital St. Louis


title: Efficacy and Safety of Oral Budesonide-MMX™ (CB-01-02) 6 mg and 9 mg Extended Release Tablets in Patients With Mild or Moderate Active Ulcerative Colitis. A Multicentre, Randomised, Double-Blind, Double-Dummy, Comparative Study Versus Placebo With an Additional Reference Arm Evaluating Entocort®EC

summary:
This will be a multicentre, randomised, double-blind, double-dummy, parallel group comparative study in patients with mild or moderate, active ulcerative colitis. The study will compare budesonide-MMX™ 6 mg and budesonide-MMX™ 9 mg tablets to placebo and to Entocort® 3 x 3 mg capsules, in four parallel groups of patients over an 8 week treatment period.

After the screening visit, patients will enter a washout period of 2 days, then they will be randomised to the following four treatment groups: budesonide-MMX™ tablets (6 mg), budesonide-MMX™ tablets (9 mg), Entocort® capsules (3 x 3 mg) and placebo (tablets and capsules), all administered once a day after breakfast. Hence, each patient will receive, in the morning after breakfast, either one budesonide-MMX™ 6 mg or budesonide MMX™ 9 mg tablet and 3 placebo Entocort® matching capsules, or three Entocort® 3 mg capsules and one placebo budesonide-MMX™ matching tablet, or one placebo budesonide-MMX™ matching tablet and three placebo Entocort® matching capsules.

ReferenceCitation: Travis SP, Danese S, Kupcinskas L, Alexeeva O, D'Haens G, Gibson PR, Moro L, Jones R, Ballard ED, Masure J, Rossini M, Sandborn WJ. Once-daily budesonide MMX in active, mild-to-moderate ulcerative colitis: results from the randomised CORE II study. Gut. 2014 Mar;63(3):433-41. doi: 10.1136/gutjnl-2012-304258. Epub 2013 Feb 22.

family: Travis

full_name: Simon P L Travis

given: Simon P L

family: Danese

full_name: Silvio Danese

given: Silvio

family: Kupcinskas

full_name: Limas Kupcinskas

given: Limas

family: Alexeeva

full_name: Olga Alexeeva

given: Olga

family: D'Haens

full_name: Geert D'Haens

given: Geert

family: Gibson

full_name: Peter R Gibson

given: Peter R

family: Moro

full_name: Luigi Moro

given: Luigi

family: Jones

full_name: Richard Jones

given: Richard

family: Ballard

full_name: E David Ballard

given: E David

family: Masure

full_name: Johan Masure

given: Johan

family: Rossini

full_name: Matteo Rossini

given: Matteo

family: Sandborn

full_name: William J Sandborn

given: William J

journal_name: Gut

evidence: open (via page says license)

repository_institution: University of Oxford - Oxford Academic Notes & Queries

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

lead_sponsor: Bausch Health Americas, Inc.

location_country:
Italy
Sweden
United Kingdom
France
Poland
Lithuania
Israel
Slovakia
Romania
Belgium
Australia
Russian Federation
Estonia
Latvia
Ukraine

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej VIVAMED

location_facility: Paula Stradina Clinical University Hospital

location_facility: Box Hill Hospital, Department of Gastroenterology Clive Ward Centre,

location_facility: Lviv National Medical University after name Danylo Halytsky based on Communal Clinical City hospital No 5, Department of Propedeutic of Internal Disease

location_facility: CRO - IRCCS - Struttura Operativa Complessa di Gastroenterologia Oncologica

location_facility: Uzhgorod State Medical University, chair of therapy and family medicine, district clinical hospitalof station "Uzhgorod"

location_facility: University Hospital of Coventry and Warwickshire

location_facility: Dipartimento di Medicina Interna e Specialità Mediche (DIMI)

location_facility: Niepubliczny Zaklad Opieki Zdrowotnej POLIMEDICA

location_facility: NZOZ Centrum Leczenia Chorob Cywilizacyjnych, oddzial Gdynia, filia Fikakw

location_facility: Odessa city Polyclinic #20, Therapeutic Dept. 6

location_facility: State Scientific Centre of Coloproctology of the Federal Agency for High-Technology Medical Care

location_facility: FNsP Bratislava, Nemocnica Ruzinov V. Interna klinika, Gastroenterohepatologicke oddelenie Ruzinovska

location_facility: Centrul de Gastroenterologie Dr. Goldis Adrian

location_facility: GU research educational medical centre of the administration of the affairs of the president of Russian Federation on the basis of State Healthcare Institution "State Clinical Hospital # 51"

location_facility: St. Petersburg State Medical Academy n.a. I.I. Mechnikov

location_facility: Divisione di Gastroenterologia - Istituto Clinico Humanitas IRCCS in Gastroenterologia

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Gastroenterologické a Hepatologické centrum

location_facility: FGU North-West DIstrict Medical Center of Roszdrav

location_facility: City Clinical Emergency Hospital named after O.I.Meschaninov,

location_facility: Federal State Institution ?National Medical Surgical Center

location_facility: Uzhgorod National University, Hospital surgery chair on the base of Uzhgorod Regional Clinical Hospital

location_facility: Saint-Petersburg GUZ City polyclinic #38 28

location_facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej, Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Kliniki Gastroenterologii, Hepatologii i Chorob Zakaznych,

location_facility: Spitalul Clinic Colentina Sectia Gastroenterologie

location_facility: GUZ of Moscow "City Clinical Hospital #24"

location_facility: Gastromed S.C.Maciej Kralisz, Andrzej Penpicki, Jacek Romatowski, Gabinet, Gastrologiczny i Pracownia Endoskopowa

location_facility: Chair of Gastroenterology and therapy of Dnipropetrovsk State Medical Academy based on Institute of gastroenterology

location_facility: Szpital Uniwersytecki w Krakowie,Oddział Kliniczny Kliniki Gastroenterologii Hematologii i Chorób Zakaźnych

location_facility: Indywidualna Specjalistyczna Praktyka Lekarska

location_facility: FNsP Bratislava, Nemocnica Stare Mesto 1st Internal Clinic Mickiewiczova


title: Intensified Pre-Operative Chemotherapy And Radical Surgery For High Risk Hepatoblastoma

summary:
RATIONALE: Drugs used in chemotherapy work in different ways to stop tumor cells from dividing so they stop growing or die. Giving chemotherapy drugs before surgery may shrink the tumor so that it can be removed.

PURPOSE: This phase II trial is studying how well neoadjuvant chemotherapy works in treating young patients who are undergoing surgical resection for high-risk hepatoblastoma.

ReferenceCitation: Zsiros J, Brugieres L, Brock P, Roebuck D, Maibach R, Zimmermann A, Childs M, Pariente D, Laithier V, Otte JB, Branchereau S, Aronson D, Rangaswami A, Ronghe M, Casanova M, Sullivan M, Morland B, Czauderna P, Perilongo G; International Childhood Liver Tumours Strategy Group (SIOPEL). Dose-dense cisplatin-based chemotherapy and surgery for children with high-risk hepatoblastoma (SIOPEL-4): a prospective, single-arm, feasibility study. Lancet Oncol. 2013 Aug;14(9):834-42. doi: 10.1016/S1470-2045(13)70272-9. Epub 2013 Jul 4.

family: Zsiros

full_name: József Zsiros

given: József

family: Brugieres

full_name: Laurence Brugieres

given: Laurence

family: Brock

full_name: Penelope Brock

given: Penelope

family: Roebuck

full_name: Derek Roebuck

given: Derek

family: Maibach

full_name: Rudolf Maibach

given: Rudolf

family: Zimmermann

full_name: Arthur Zimmermann

given: Arthur

family: Childs

full_name: Margaret Childs

given: Margaret

family: Pariente

full_name: Daniele Pariente

given: Daniele

family: Laithier

full_name: Veronique Laithier

given: Veronique

family: Otte

full_name: Jean-Bernard Otte

given: Jean-Bernard

family: Branchereau

full_name: Sophie Branchereau

given: Sophie

family: Aronson

full_name: Daniel Aronson

given: Daniel

family: Rangaswami

full_name: Arun Rangaswami

given: Arun

family: Ronghe

full_name: Milind Ronghe

given: Milind

family: Casanova

full_name: Michela Casanova

given: Michela

family: Sullivan

full_name: Michael Sullivan

given: Michael

family: Morland

full_name: Bruce Morland

given: Bruce

family: Czauderna

full_name: Piotr Czauderna

given: Piotr

family: Perilongo

full_name: Giorgio Perilongo

given: Giorgio

journal_name: The Lancet Oncology

publisher: Elsevier BV

ReferenceCitation: Weeda VB, Murawski M, McCabe AJ, Maibach R, Brugières L, Roebuck D, Fabre M, Zimmermann A, Otte JB, Sullivan M, Perilongo G, Childs M, Brock P, Zsíros J, Plaschkes J, Czauderna P, Aronson DC. Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma--results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience. Eur J Cancer. 2013 Aug;49(12):2698-704. doi: 10.1016/j.ejca.2013.04.012. Epub 2013 May 15.

title: Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma – Results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience

location_country:
Ireland
France
United Kingdom
Netherlands

location_facility: Leeds Cancer Centre at St. James's University Hospital

location_facility: Royal Liverpool Children's Hospital, Alder Hey

location_facility: Sir James Spence Institute of Child Health at Royal Victoria Infirmary

location_facility: Institute of Child Health at University of Bristol

location_facility: Royal Belfast Hospital for Sick Children

location_facility: Great Ormond Street Hospital for Children

location_facility: Our Lady's Hospital for Sick Children Crumlin


title: Safety of Sports for Patients With Implantable Cardioverter-Defibrillators: A Multi-Center Registry

summary: The registry will obtain information to determine the safety of sports participation for patients with defibrillators (ICDs).

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Saarel EV, Law I, Berul CI, Ackerman MJ, Kanter RJ, Sanatani S, Cohen MI, Berger S, Fischbach PS, Burton DA, Dziura J, Brandt C, Simone L, Li F, Olshansky B, Cannom DS, Lampert RJ. Safety of Sports for Young Patients With Implantable Cardioverter-Defibrillators: Long-Term Results of the Multinational ICD Sports Registry. Circ Arrhythm Electrophysiol. 2018 Nov;11(11):e006305. doi: 10.1161/CIRCEP.118.006305.

name: Pediatric Cardiology, Cleveland Clinic Children’s & Electrophysiology, Cleveland Clinic, OH (E.V.S.).

family: Saarel

full_name: Elizabeth Vickers Saarel

given: Elizabeth Vickers

name: University of Iowa Stead Family Children’s Hospital (I.L.).

family: Law

full_name: Ian Law

given: Ian

name: Children’s National Medical Center, Washington, DC (C.I.B.).

family: Berul

full_name: Charles I. Berul

given: Charles I.

name: Departments of Cardiovascular Medicine, Pediatric & Adolescent Medicine, and Molecular Pharmacology & Experimental Therapeutics, Mayo Clinic, Rochester, MN (M.J.A.)

family: Ackerman

full_name: Michael J. Ackerman

given: Michael J.

name: Nicklaus Children’s Hospital, Miami, FL (R.J.K.).

family: Kanter

full_name: Ronald J. Kanter

given: Ronald J.

name: Division of Cardiology, Department of Pediatrics, University of British Columbia, Vancouver, Canada (S.S.).

family: Sanatani

full_name: Shubhayan Sanatani

given: Shubhayan

name: Inova Children’s Hospital, Falls Church, VA (M.I.C.).

family: Cohen

full_name: Mitchell I. Cohen

given: Mitchell I.

name: Ann and Robert H. Lurie Children’s Hospital, Chicago, IL (S.B.).

family: Berger

full_name: Stuart Berger

given: Stuart

family: Fischbach

full_name: Peter S. Fischbach

given: Peter S.

name: Children’s Hospital Minnesota, Minneapolis (D.A.B.).

family: Burton

full_name: David A. Burton

given: David A.

name: Yale University School of Medicine, New Haven, CT (J.D., C.B., L.S., F.L., R.J.L.).

family: Dziura

full_name: James Dziura

given: James

family: Brandt

full_name: Cynthia Brandt

given: Cynthia

family: Simone

full_name: Laura Simone

given: Laura

family: Li

full_name: Fangyong Li

given: Fangyong

family: Olshansky

full_name: Brian Olshansky

given: Brian

name: Good Samaritan Hospital, Cedars-Sinai Heart Institute, Los Angeles, CA (D.S.C.).

family: Cannom

full_name: David S. Cannom

given: David S.

family: Lampert

full_name: Rachel J. Lampert

given: Rachel J.

journal_name: Circulation: Arrhythmia and Electrophysiology

oa_host_type: publisher

evidence: open (via free pdf)

publisher: Ovid Technologies (Wolters Kluwer Health)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

lead_sponsor: Yale University

location_country:
United States
Switzerland
Netherlands
United Kingdom
Germany
France
Poland
Israel
Norway
Belgium
Australia
Spain
Canada
Czech Republic

location_facility: Children's Hospital and Clinics of Minnesota

location_facility: Midatlantic Cardiovascular Associates, PA

location_facility: The Ohio State University Research Foundation

location_facility: Legacy Cllinical Research & Tech Center (Good Samaritan Hospital_

location_facility: Foundation for the Advancement of Cardiovascular Therapy

location_facility: Arizona Pediatric Cardiology Consultants

location_facility: London Health Sciences Centre University Hospital

lead_sponsor_type: academique


title: An International Field Study Of The Reliability And Validity Of The EORTC QLQ-C30 And A Disease-Specific Questionnaire Module (The QLQ-STO22) In Assessing The Quality Of Life Of Patients With Gastric Cancer

summary:
RATIONALE: Assessing quality of life in patients who are undergoing cancer treatment may help determine the effect of treatment on these patients.

PURPOSE: Clinical trial to study the effectiveness of a quality of life assessment in patients who are receiving treatment for stomach cancer.

ReferenceCitation: Oñate-Ocaña LF, Alcántara-Pilar A, Vilar-Compte D, García-Hubard G, Rojas-Castillo E, Alvarado-Aguilar S, Carrillo JF, Blazeby JM, Aiello-Crocifoglio V. Validation of the Mexican Spanish version of the EORTC C30 and STO22 questionnaires for the evaluation of health-related quality of life in patients with gastric cancer. Ann Surg Oncol. 2009 Jan;16(1):88-95. doi: 10.1245/s10434-008-0175-9. Epub 2008 Nov 1.

family: Oñate-Ocaña

full_name: Luis F. Oñate-Ocaña

given: Luis F.

family: Alcántara-Pilar

full_name: Alberto Alcántara-Pilar

given: Alberto

family: Vilar-Compte

full_name: Diana Vilar-Compte

given: Diana

family: García-Hubard

full_name: Gabriela García-Hubard

given: Gabriela

family: Rojas-Castillo

full_name: Edith Rojas-Castillo

given: Edith

family: Alvarado-Aguilar

full_name: Salvador Alvarado-Aguilar

given: Salvador

family: Carrillo

full_name: José F. Carrillo

given: José F.

family: Blazeby

full_name: Jane M. Blazeby

given: Jane M.

family: Aiello-Crocifoglio

full_name: Vincenzo Aiello-Crocifoglio

given: Vincenzo

journal_name: Annals of Surgical Oncology

title: Validation of the Mexican Spanish Version of the EORTC C30 and STO22 Questionnaires for the Evaluation of Health-Related Quality of Life in Patients with Gastric Cancer

ReferenceCitation: Sadighi S, Montazeri A, Sedighi Z, Mohagheghi MA, Froutan H. Quality of life in patients with gastric cancer: translation and psychometric evaluation of the Iranian version of EORTC QLQ-STO22. BMC Cancer. 2009 Aug 28;9:305. doi: 10.1186/1471-2407-9-305.

family: Sadighi

full_name: Sanamber Sadighi

given: Sanamber

family: Montazeri

full_name: Ali Montazeri

given: Ali

family: Sedighi

full_name: Zahra Sedighi

given: Zahra

family: Mohagheghi

full_name: Mohammad Ali Mohagheghi

given: Mohammad Ali

family: Froutan

full_name: Hossein Froutan

given: Hossein

journal_name: BMC Cancer

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

location_country:
Sweden
France
Spain
Germany
United Kingdom

location_facility:
Royal Infirmary
Centre Alexis Vautrin
University of Marburg
Hospital De Navarra
Bristol Haematology and Oncology Centre
Universitaetsklinikum Charite
Sahlgrenska University Hospital


title: The Atrial High Rate Episodes (A-HIRATE) in Pacemaker Patients

summary: Prospective study assessing the incidence of atrial high rate episodes (AHRE) in pacemaker patients with and without previously diagnosed AT.

ReferenceType: background

ReferenceCitation: Ghali JK, Orlov MV, Araghi-Niknam M, Sherfesee L, Hettrick DA. The influence of symptoms and device detected atrial tachyarrhythmias on medical management: insights from A-HIRATE. Pacing Clin Electrophysiol. 2007 Jul;30(7):850-7.

ReferenceCitation: Orlov MV, Ghali JK, Araghi-Niknam M, Sherfesee L, Sahr D, Hettrick DA; Atrial High Rate Trial Investigators. Asymptomatic atrial fibrillation in pacemaker recipients: incidence, progression, and determinants based on the atrial high rate trial. Pacing Clin Electrophysiol. 2007 Mar;30(3):404-11.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
United States
Australia
France


ReferenceCitation: Thalange N, Bereket A, Jensen LB, Hiort LC, Peterkova V. Development of Insulin Detemir/Insulin Aspart Cross-Reacting Antibodies Following Treatment with Insulin Detemir: 104-week Study in Children and Adolescents with Type 1 Diabetes Aged 2-16 Years. Diabetes Ther. 2016 Dec;7(4):713-724. Epub 2016 Sep 6.

lead_sponsor: Novo Nordisk A/S

location_country: Macedonia, The Former Yugoslav Republic of

title: A 52-Week, Multinational, Multi-Centre, Open-Labelled Extension Trial of Insulin Detemir in Children and Adolescents 3-17 Years With Type 1 Diabetes on a Basal-Bolus Regimen With Insulin Aspart as Bolus Insulin

summary: This trial is conducted in Europe. The aim of this research is to assess the safety of continuous treatment with insulin detemir following participation in trial NN304-1689 (NCT00435019) on antibody development.


title: Two-year Study to Determine the Efficacy and Safety of Risedronate Therapy Administered 35 mg Once A Week in Men With Osteoporosis for 2 Years Followed by a 2 Year Open Label Study

summary: Two year study to determine the safety and efficacy of weekly 35 mg Risedronate doses in men with osteoporosis followed by a two year follow-up study.

ReferenceCitation: Boonen S, Lorenc RS, Wenderoth D, Stoner KJ, Eusebio R, Orwoll ES. Evidence for safety and efficacy of risedronate in men with osteoporosis over 4 years of treatment: Results from the 2-year, open-label, extension study of a 2-year, randomized, double-blind, placebo-controlled study. Bone. 2012 Sep;51(3):383-8. doi: 10.1016/j.bone.2012.06.016. Epub 2012 Jun 30.

family: Boonen

full_name: Steven Boonen

given: Steven

family: Lorenc

full_name: Roman S. Lorenc

given: Roman S.

family: Wenderoth

full_name: Dietrich Wenderoth

given: Dietrich

family: Stoner

full_name: Karen J. Stoner

given: Karen J.

family: Eusebio

full_name: Rachelle Eusebio

given: Rachelle

family: Orwoll

full_name: Eric S. Orwoll

given: Eric S.

journal_name: Bone

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Evidence for safety and efficacy of risedronate in men with osteoporosis over 4years of treatment: Results from the 2-year, open-label, extension study of a 2-year, randomized, double-blind, placebo-controlled study

lead_sponsor: Warner Chilcott

location_country:
United Kingdom
Lebanon
Netherlands
France
Poland
Hungary
Belgium
Australia
United States
Czech Republic

location_facility:
Research Facility
Research Site


title: Central Nervous System Disorders Following Hematopoietic Stem Cell Transplantation: a Prospective Observational Trial From the Infectious Diseases Working Party and the Transplant Complications Working Party of the European Society for Blood and Marrow Transplantation

summary:
All patients undergoing allogeneic or autologous HSCT at the participating centres will be observed. Once a diagnosis of CNS disorder is made, additional data will be reported for these patients.

We will identify clinical and diagnostic characteristics such as cerebrospinal fluid (CSF) and neuroimaging patterns, risk factors, response to treatment (including novel antifungal agents such as isavuconazole) and outcome. In addition, risk factors for CNS disorders after allogeneic and autologous HSCT will be analyzed using a prospectively assessed matched control group. In the future, this study might be the basis for an interventional trial (e.g. using a prophylactic approach).

status: Recruiting

study_completion_date_type: Anticipated

ipd_sharing: Undecided

location_country:
Italy
France
Poland
Tunisia
Hungary
Australia
Russian Federation
Spain
Germany

location_facility: University Hospital, Collegium Medicum UMK

location_facility: University Children's Hospital in Krakow

location_facility: Raisa Gorbacheva Research Institute for Oncology

status_simplified: Ongoing


title: Open Label, Parallel Group, Multicenter Study of Two Intravenous (IV) Ibandronate Dose Regimens (2 mg Every 2 Months and 3 mg Every 3 Months) in Women With Postmenopausal Osteoporosis Who Completed Trial BM16550

summary: This 2-arm study was designed to assess the long-term safety and tolerability of intravenous (IV) treatment with 2 mg or 3 mg Bonviva in women with post-menopausal osteoporosis who had previously completed Bonviva study BM16550 (DIVA study; NCT00048074). Patients received Bonviva either 2 mg IV every 2 months, or 3 mg IV every 3 months. Patients also received daily supplementation with vitamin D and calcium. The anticipated time on study treatment was 2+ years, and the target sample size was 500+ individuals.

lead_sponsor: Hoffmann-La Roche

location_country:
Italy
South Africa
United Kingdom
Mexico
Germany
France
Poland
Norway
Hungary
Belgium
Australia
Spain
Denmark
United States
Czech Republic


title: A Multi-center, Open-label, follow-on Trial to Assess the Long-term Safety and Efficacy of Lacosamide in Subjects With Painful Distal Diabetic Neuropathy Including a Double-blind, Randomized Time Point Withdrawal Subtrial.

summary: SP746 (NCT00546351) is a multi-center, open-label, follow-on trial. The purpose of this trial is to assess safety and tolerability of long-term exposure of lacosamide (previously referred to as SPM 927) in subjects with painful distal diabetic neuropathy.

lead_sponsor: UCB Pharma

location_country:
Italy
Austria
Serbia
Finland
France
Czechia
Bulgaria
Poland
Romania
Hungary
Belgium
Russian Federation
Spain
Germany
United Kingdom


title: A TWINSS Extension Trial to Evaluate the Safety and Tolerability of CFZ533 (Iscalimab) at Two Dose Levels Administered Subcutaneously in Patients With Sjögren's Syndrome

acronym: TWINSS Extn

summary: This study will evaluate the safety and tolerability of iscalimab at two dose levels in patients with Sjögren's Syndrome, who participated in the TWINSS core study, CCFZ533B2201(NCT03905525). Additionally, this Extension study will further explore the pharmacokinetics (PK) and efficacy of iscalimab at two dose level.

ipd_sharing_description:
Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect 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:
Austria
Netherlands
France
Japan
Greece
Israel
Hungary
Russian Federation
Portugal
Turkey
Germany


title: Comprehensive Long-term Follow up of Adults With Arterial Switch Operation - European Collaboration for Prospective Outcome Research in Congenital Heart Disease

acronym: EPOCH-ASO

summary:
Background: Long-term outcomes in adults with prior arterial switch operation (ASO) have not yet been well defined. The aim of this study is to elucidate incidence and predictors of adverse cardiac outcomes in a prospectively followed cohort of adults after their ASO.

Methods: The comprehensive long-term follow up of adults with ASO is a project within the European collaboration for prospective outcome research in congenital heart disease (EPOCH). It is designed as a prospective, international multicenter cohort study. Consecutive patients (aged 16 years or more) with prior ASO will be included at 11 European tertiary care centers. Participants will be followed according to a standardized protocol following international recommendations, including standardized protocols for imaging and for exercise testing. Main outcome measures are all-cause and cardiac-related mortality, rate of cardiac re-intervention, neo-aortic dissection, myocardial infarction, stroke, infective endocarditis, sustained atrial and ventricular arrhythmias, new-onset or worsening pulmonary hypertension and new-onset heart failure. Secondary endpoints are frequency and progression of right ventricular outflow tract stenosis, neo-aortic root dilatation, neo-aortic valve regurgitation and ventricular dysfunction. The impact of demographic, anatomic (e.g. coronary artery anatomy) and functional variables on the above-mentioned outcomes, as well as quality of life and incidence of pregnancy related complications will also be assessed.

Aim: The prospective, international, multicenter EPOCH-ASO study will provide a better understanding of adverse outcomes and their predictors in adults after ASO. The results of the EPOCH-ASO study may help to optimize future care of this novel patient cohort in adult cardiology.

study_start_date_type: Anticipated

lead_sponsor: University of Zurich

location_country: Austria Switzerland Netherlands France Spain

location_facility: Hospital Universitario y Politécnico La Fe Valencia

location_facility: CHU Paris IdF Ouest - HEGP Hôpital Européen Georges Pompidou


title: A Double Blind, Randomized, Placebo Controlled, Parallel Group Dose-range Exploration Study of Sativex® in Relieving Pain in Patients With Advanced Cancer, Who Experience Inadequate Analgesia During Optimized Chronic Opioid Therapy.

acronym: SPRAY

summary: The purpose of this study is to determine the effective dose range and to demonstrate a non-effective dose range of Sativex in patients with advanced cancer, who experience inadequate pain relief even though they are on optimized chronic opioid therapy.

ReferenceCitation: Portenoy RK, Ganae-Motan ED, Allende S, Yanagihara R, Shaiova L, Weinstein S, McQuade R, Wright S, Fallon MT. Nabiximols for opioid-treated cancer patients with poorly-controlled chronic pain: a randomized, placebo-controlled, graded-dose trial. J Pain. 2012 May;13(5):438-49. doi: 10.1016/j.jpain.2012.01.003. Epub 2012 Apr 5.

family: Portenoy

full_name: Russell K. Portenoy

given: Russell K.

family: Ganae-Motan

full_name: Elena Doina Ganae-Motan

given: Elena Doina

family: Allende

full_name: Silvia Allende

given: Silvia

family: Yanagihara

full_name: Ronald Yanagihara

given: Ronald

family: Shaiova

full_name: Lauren Shaiova

given: Lauren

family: Weinstein

full_name: Sharon Weinstein

given: Sharon

family: McQuade

full_name: Robert McQuade

given: Robert

family: Wright

full_name: Stephen Wright

given: Stephen

family: Fallon

full_name: Marie T. Fallon

given: Marie T.

journal_name: The Journal of Pain

title: Nabiximols for Opioid-Treated Cancer Patients With Poorly-Controlled Chronic Pain: A Randomized, Placebo-Controlled, Graded-Dose Trial

lead_sponsor: GW Pharmaceuticals Ltd.

location_country:
Italy
United States
Chile
South Africa
India
United Kingdom
Mexico
Germany
Finland
France
Poland
Romania
Belgium
Spain
Canada
Czech Republic

location_facility: Meenakshi Mission Hospital & Research Centre

location_facility: Trialtech Research - Embassy Drive Medical Centre

location_facility: Beskidzkie Centrum Onkologii im. Jana Pawla

location_facility: Jehangir Clinical Development Centre Pvt. Ltd.

location_facility: Centrum Onkologii - Instytut im. M. Sklodowskiej - Curie

location_facility: FN Hradec Králové - Klinika onkologie a radioterapie

location_facility: Pacific Coast Hematology/Oncology Medical Group, Inc.

location_facility: Spitalul Judetean de Urgenta "Constantin Opris"

location_facility: Indo-American Cancer Institute and Research Center

location_facility: Capital Comprehensive Cancer Care Clinic

location_facility: CHU Charleroi (Hôpital civil de Charleroi)

location_facility: UZ Leuven - Algologisch Centrum Anesthesiologie

location_facility: Dir. S.C.D.U. Psicologia Clinica ed Oncologica

location_facility: Wojewodzki Szpital Specjalistyczny im. M. Kopernika

location_facility: Bhagwaan Mahaveer Cancer Hospital and Research Centre

location_facility: FN a LF UP Olomouc - Ambulance pro lécbu bolesti

location_facility: Deenanath Mangeshkar Hospital and Research Center

location_facility: International Observatory on End of Life Care

location_facility: AR klinika FN Plzen -Ambulance pro lécbu bolesti

location_facility: Spitalul Judetean de Urgenta "Sf. Ioan cel Nou"

location_facility: Basingstoke & North Hampshire NHS Foundation Trust

location_facility: Instituto Radio-oncológico Santiago (INRAD)

location_facility: Spitalul Clinic Judetean Sibiu Oncologie

location_facility: The Center for Clinical Research - Washington County Hospital

location_facility: ARO, Krajská zdravotni, K.Z. a.s, Nemocnice

location_facility: Center of Hope for Cancer and Blood Disorders

location_facility: District Hospital Dr. Alexandru Simionescu

location_facility: S.C. IanuliMed S.R.L. Oncologie Medicala


title: A Prospective, Randomized, Double-dummy, Double-blind, Multicenter Trial Comparing the Safety and Efficacy of Intravenous Moxifloxacin 400 mg IV QD 24 Hours to That of Ertapenem 1.0 g IV QD 24 Hours for 5 to 14 Days for the Treatment of Subjects With Complicated Intra-abdominal Infections (PROMISE Study)

summary: A study to compare the safety and efficacy of moxifloxacin to ertapenem in patients with intra-abdominal infections.

ReferenceCitation: De Waele JJ, Tellado JM, Alder J, Reimnitz P, Jensen M, Hampel B, Arvis P. Randomised clinical trial of moxifloxacin versus ertapenem in complicated intra-abdominal infections: results of the PROMISE study. Int J Antimicrob Agents. 2013 Jan;41(1):57-64. doi: 10.1016/j.ijantimicag.2012.08.013. Epub 2012 Nov 13.

family: De Waele

full_name: J.J. De Waele

family: Tellado

full_name: J.M. Tellado

family: Alder

full_name: J. Alder

given: J.

family: Reimnitz

full_name: P. Reimnitz

given: P.

family: Jensen

full_name: M. Jensen

given: M.

family: Hampel

full_name: B. Hampel

given: B.

family: Arvis

full_name: P. Arvis

journal_name: International Journal of Antimicrobial Agents

lead_sponsor: Bayer

location_country:
South Africa
France
Bulgaria
Greece
Israel
Lithuania
Romania
Belgium
Argentina
Russian Federation
Spain
Estonia
Germany
Latvia


title: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

acronym: NICOFA

summary: Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia. It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene (FXN) and results in decreased levels of FXN protein. FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty. Patients gradually lose coordination, become dysarthric and are frequently wheel-chair bound as adolescents. There is no disease modifying therapy and many patients die prematurely of cardiomyopathy. It was subsequently found that the FXN gene is silenced at the chromatin level by the formation of heterochromatin and that this heterochromatin formation can be antagonized by histone deacetylase inhibitors (HDACi) (Chan et al., 2013). A recent proof-of-concept clinical study on ten patients with Friedreich ataxia demonstrated that FXN levels can be restored to those seen in asymptomatic carriers using the class III HDACi nicotinamide at a dose that is well tolerated by patients (Libri et al., 2014). Since carriers are asymptomatic, this degree of restoration of FXN expression might be expected to halt disease progression. Nicotinamide readily crosses the blood brain barrier and has previously been given at high doses for long periods to normal individuals without serious adverse effects (Gale et al., 2004; Knip et al., 2000). This study will be the first to provide clinical evidence for the efficacy and safety of nicotinamide in patients with Friedreich´s ataxia.

status: Not yet recruiting

ReferenceCitation: Reetz K, Hilgers RD, Isfort S, Dohmen M, Didszun C, Fedosov K, Kistermann J, Mariotti C, Durr A, Boesch S, Klopstock T, Rodríguez de Rivera Garrido FJ, Schöls L, Klockgether T, Pandolfo M, Korinthenberg R, Lavin P, Molenberghs G, Libri V, Giunti P, Festenstein R, Schulz JB; EFACTS or NICOFA study group. Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA). Neurol Res Pract. 2019 Oct 15;1:33. doi: 10.1186/s42466-019-0038-9. eCollection 2019.

apc_source: doaj

family: Reetz

full_name: Kathrin Reetz

given: Kathrin

family: Hilgers

full_name: Ralf-Dieter Hilgers

given: Ralf-Dieter

family: Isfort

full_name: Susanne Isfort

given: Susanne

family: Dohmen

full_name: Marc Dohmen

given: Marc

family: Didszun

full_name: Claire Didszun

given: Claire

family: Fedosov

full_name: Kathrin Fedosov

family: Kistermann

full_name: Jennifer Kistermann

given: Jennifer

family: Mariotti

full_name: Caterina Mariotti

given: Caterina

family: Durr

full_name: Alexandra Durr

given: Alexandra

family: Boesch

full_name: Sylvia Boesch

given: Sylvia

family: Klopstock

full_name: Thomas Klopstock

given: Thomas

family: Rodríguez de Rivera Garrido

full_name: Francisco Javier Rodríguez de Rivera Garrido

given: Francisco Javier

family: Schöls

full_name: Ludger Schöls

given: Ludger

family: Klockgether

full_name: Thomas Klockgether

family: Pandolfo

full_name: Massimo Pandolfo

given: Massimo

family: Korinthenberg

full_name: Rudolf Korinthenberg

given: Rudolf

family: Lavin

full_name: Philip Lavin

given: Philip

family: Molenberghs

full_name: Geert Molenberghs

given: Geert

family: Libri

full_name: Vincenzo Libri

given: Vincenzo

family: Giunti

full_name: Paola Giunti

given: Paola

family: Festenstein

full_name: Richard Festenstein

given: Richard

family: Schulz

full_name: Jörg B. Schulz

given: Jörg B.

repository_institution: University College London - UCL Discovery

repositories:
LIRIAS (KU Leuven)
PubMed Central
UCL Discovery
spiral.imperial.ac.uk

ipd_sharing_description: The study results will be published in appropriate international scientific journals, and publishing details will be given in the clinical study agreement. The study will be registered and study results will be disclosed by the principal investigator in one or more public clinical study registry(ies), according to national/international use.

location_country:
Italy
Austria
France
Spain
Germany
United Kingdom

location_facility: Service de génétique médicale - Hôpital La Pitié Salpetrière

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta


title: Prognostic Imaging Biomarkers for Diabetic Kidney Disease

acronym: iBEAt

summary:
Diabetic kidney disease (DKD) is a common complication of diabetes, and is now the most common form of chronic kidney disease. DKD is the leading cause of kidney disease requiring dialysis or kidney transplantation, and its global incidence and prevalence have reached epidemic levels. While the risk of developing DKD can be ameliorated by tight blood glucose and blood pressure control, it is not fully preventable and once established DKD cannot be cured. Therefore many patients are left with poor and worsening health and with increased mortality risk. Developing new ways to treat DKD requires healthcare professionals to be able to identify those patients most in need of treatment.

One promising approach for identifying patients that are at risk is the use of imaging measurements (called "biomarkers") derived from Magnetic Resonance Imaging (MRI) and Ultrasound (US) of the kidneys. Evidence from early studies shows that such imaging biomarkers can identify underlying problems in DKD such as blood supply, oxygen supply, kidney scarring and kidney function, in ways that are better than those currently available.

The investigators think that imaging biomarkers will improve the identification of patients who are likely to decline from DKD in the short term. The changes found by imaging may even happen before effects on the blood and urine.

The investigators plan to test this hypothesis by performing a study observing 500 patients with early stage DKD, recruited in 5 sites across Europe. All patients will have detailed assessment at the start of their involvement, including clinical assessment, blood and urine samples, and MRI and US scans. The investigators will look at whether imaging biomarkers are associated with other measures that predict progression in DKD, and follow patients every year for 3 years (4 years total study participation) to see if the imaging biomarkers predict worsening DKD.

time_perspective: Cross-Sectional

ReferenceCitation: Grenier N, Merville P, Combe C. Radiologic imaging of the renal parenchyma structure and function. Nat Rev Nephrol. 2016 Jun;12(6):348-59. doi: 10.1038/nrneph.2016.44. Epub 2016 Apr 12. Review.

ReferenceCitation: Gooding KM, Lienczewski C, Papale M, Koivuviita N, Maziarz M, Dutius Andersson AM, Sharma K, Pontrelli P, Garcia Hernandez A, Bailey J, Tobin K, Saunavaara V, Zetterqvist A, Shelley D, Teh I, Ball C, Puppala S, Ibberson M, Karihaloo A, Metsärinne K, Banks RE, Gilmour PS, Mansfield M, Gilchrist M, de Zeeuw D, Heerspink HJL, Nuutila P, Kretzler M, Welberry Smith M, Gesualdo L, Andress D, Grenier N, Shore AC, Gomez MF, Sourbron S; BEAt-DKD consortium. Prognostic imaging biomarkers for diabetic kidney disease (iBEAt): study protocol. BMC Nephrol. 2020 Jun 29;21(1):242. doi: 10.1186/s12882-020-01901-x.

family: Gooding

full_name: Kim M. Gooding

given: Kim M.

family: Lienczewski

full_name: Chrysta Lienczewski

given: Chrysta

family: Papale

full_name: Massimo Papale

family: Koivuviita

full_name: Niina Koivuviita

given: Niina

family: Maziarz

full_name: Marlena Maziarz

given: Marlena

family: Dutius Andersson

full_name: Anna-Maria Dutius Andersson

given: Anna-Maria

family: Sharma

full_name: Kanishka Sharma

given: Kanishka

family: Pontrelli

full_name: Paola Pontrelli

family: Garcia Hernandez

full_name: Alberto Garcia Hernandez

given: Alberto

family: Bailey

full_name: Julie Bailey

given: Julie

family: Tobin

full_name: Kay Tobin

given: Kay

family: Saunavaara

full_name: Virva Saunavaara

given: Virva

family: Zetterqvist

full_name: Anna Zetterqvist

given: Anna

family: Shelley

full_name: David Shelley

given: David

family: Teh

full_name: Irvin Teh

given: Irvin

family: Ball

full_name: Claire Ball

family: Puppala

full_name: Sapna Puppala

given: Sapna

family: Ibberson

full_name: Mark Ibberson

given: Mark

family: Karihaloo

full_name: Anil Karihaloo

given: Anil

family: Metsärinne

full_name: Kaj Metsärinne

given: Kaj

family: Banks

full_name: Rosamonde E. Banks

given: Rosamonde E.

family: Gilmour

full_name: Peter S. Gilmour

given: Peter S.

family: Mansfield

full_name: Michael Mansfield

given: Michael

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

repository_institution: Royal Devon and Exeter - Royal Devon and Exeter Research Repository

location_country:
Italy
Sweden
Switzerland
Finland
France
United Kingdom

location_facility: Centre Hospitalier Universitaire de Bordeaux

location_facility: Università degli Studi di Bari Aldo Moro


title: Post-Market Clinical Follow-up Study to Monitor Device Performance and Outcomes of the CENTERA Transcatheter Heart Valve Sytem

summary: A post market clinical trial to monitor and review the performance and outcomes of the Edwards CENTERA Transcatheter Heart Valve (THV) in patients with severe, symptomatic aortic valve stenosis who are indicated for aortic valve replacement.

status: Active, not recruiting

lead_sponsor: Edwards Lifesciences

location_country:
Netherlands
France
Spain
Denmark
Germany
United Kingdom

location_facility: Hospital Universitario Central de Asturias

location_facility: Vall d'Hebron University Hospital - Barcelona

intervention_type: Device


title: HEPANOVA: A Phase II Trial of Tumor Treating Fields (TTFields, 150kHz) Concomitant With Sorafenib For Advanced Hepatocellular Carcinoma (HCC)

summary: The study is a prospective, phase II trial single arm, historical control aimed to test the efficacy and safety of TTFields, using the NovoTTF-100L(P) System, in combination with sorafenib in patient with advanced HCC. The device is an experimental, portable, battery operated device for chronic administration of alternating electric fields (termed TTFields or TTF) to the region of the malignant tumor, by means of surface, insulated electrode arrays.

ReferenceCitation: Kirson ED, Gurvich Z, Schneiderman R, Dekel E, Itzhaki A, Wasserman Y, Schatzberger R, Palti Y. Disruption of cancer cell replication by alternating electric fields. Cancer Res. 2004 May 1;64(9):3288-95.

ReferenceCitation: Kirson ED, Dbalý V, Tovarys F, Vymazal J, Soustiel JF, Itzhaki A, Mordechovich D, Steinberg-Shapira S, Gurvich Z, Schneiderman R, Wasserman Y, Salzberg M, Ryffel B, Goldsher D, Dekel E, Palti Y. Alternating electric fields arrest cell proliferation in animal tumor models and human brain tumors. Proc Natl Acad Sci U S A. 2007 Jun 12;104(24):10152-7. Epub 2007 Jun 5.

ReferenceCitation: Stupp R, Wong ET, Kanner AA, Steinberg D, Engelhard H, Heidecke V, Kirson ED, Taillibert S, Liebermann F, Dbalý V, Ram Z, Villano JL, Rainov N, Weinberg U, Schiff D, Kunschner L, Raizer J, Honnorat J, Sloan A, Malkin M, Landolfi JC, Payer F, Mehdorn M, Weil RJ, Pannullo SC, Westphal M, Smrcka M, Chin L, Kostron H, Hofer S, Bruce J, Cosgrove R, Paleologous N, Palti Y, Gutin PH. NovoTTF-100A versus physician's choice chemotherapy in recurrent glioblastoma: a randomised phase III trial of a novel treatment modality. Eur J Cancer. 2012 Sep;48(14):2192-202. doi: 10.1016/j.ejca.2012.04.011. Epub 2012 May 18.

family: Stupp

full_name: Roger Stupp

given: Roger

family: Wong

full_name: Eric T. Wong

given: Eric T.

family: Kanner

full_name: Andrew A. Kanner

given: Andrew A.

family: Steinberg

full_name: David Steinberg

family: Engelhard

full_name: Herbert Engelhard

given: Herbert

family: Heidecke

full_name: Volkmar Heidecke

given: Volkmar

family: Kirson

full_name: Eilon D. Kirson

given: Eilon D.

family: Taillibert

full_name: Sophie Taillibert

given: Sophie

family: Liebermann

full_name: Frank Liebermann

given: Frank

family: Dbalý

full_name: Vladimir Dbalý

given: Vladimir

family: Ram

full_name: Zvi Ram

given: Zvi

family: Villano

full_name: J. Lee Villano

given: J. Lee

family: Rainov

full_name: Nikolai Rainov

given: Nikolai

family: Weinberg

full_name: Uri Weinberg

given: Uri

family: Schiff

full_name: David Schiff

family: Kunschner

full_name: Lara Kunschner

given: Lara

family: Raizer

full_name: Jeffrey Raizer

given: Jeffrey

family: Honnorat

full_name: Jerome Honnorat

given: Jerome

family: Sloan

full_name: Andrew Sloan

given: Andrew

family: Malkin

full_name: Mark Malkin

family: Landolfi

full_name: Joseph C. Landolfi

given: Joseph C.

family: Payer

full_name: Franz Payer

given: Franz

family: Mehdorn

full_name: Maximilian Mehdorn

given: Maximilian

title: NovoTTF-100A versus physician’s choice chemotherapy in recurrent glioblastoma: A randomised phase III trial of a novel treatment modality

ReferenceCitation: Kirson ED, Giladi M, Gurvich Z, Itzhaki A, Mordechovich D, Schneiderman RS, Wasserman Y, Ryffel B, Goldsher D, Palti Y. Alternating electric fields (TTFields) inhibit metastatic spread of solid tumors to the lungs. Clin Exp Metastasis. 2009;26(7):633-40. doi: 10.1007/s10585-009-9262-y. Epub 2009 Apr 23.

ReferenceCitation: Giladi M, Schneiderman RS, Voloshin T, Porat Y, Munster M, Blat R, Sherbo S, Bomzon Z, Urman N, Itzhaki A, Cahal S, Shteingauz A, Chaudhry A, Kirson ED, Weinberg U, Palti Y. Mitotic Spindle Disruption by Alternating Electric Fields Leads to Improper Chromosome Segregation and Mitotic Catastrophe in Cancer Cells. Sci Rep. 2015 Dec 11;5:18046. doi: 10.1038/srep18046.

ReferenceCitation: Stupp R, Taillibert S, Kanner AA, Kesari S, Steinberg DM, Toms SA, Taylor LP, Lieberman F, Silvani A, Fink KL, Barnett GH, Zhu JJ, Henson JW, Engelhard HH, Chen TC, Tran DD, Sroubek J, Tran ND, Hottinger AF, Landolfi J, Desai R, Caroli M, Kew Y, Honnorat J, Idbaih A, Kirson ED, Weinberg U, Palti Y, Hegi ME, Ram Z. Maintenance Therapy With Tumor-Treating Fields Plus Temozolomide vs Temozolomide Alone for Glioblastoma: A Randomized Clinical Trial. JAMA. 2015 Dec 15;314(23):2535-43. doi: 10.1001/jama.2015.16669.

name: University Hospital Zurich and University of Zurich, Zurich, Switzerland2Lausanne University Hospital (CHUV), Lausanne, Switzerland

name: Assistance Publique des Hôpitaux de Paris, La Pitié-Salpétrière-University Hospital, Pierre and Marie Curie University, Paris, France

name: Tel Aviv Sourasky Medical Center, Tel Aviv University, Tel Aviv, Israel

name: Geisinger Health System, Danville, Pennsylvania

name: Tufts Medical Center, Boston, Massachusetts

name: University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania

name: Istituto Nazionale Neurologico Carlo Besta, Milan, Italy

name: Cleveland Clinic Foundation, Cleveland, Ohio

name: Baylor University Medical Center, Dallas, Texas13University of Texas Health Science Center, Houston

name: Swedish Neuroscience Institute, Seattle, Washington

name: University of Southern California, Los Angeles

name: Washington University Barnes-Jewish Hospital, St Louis, Missouri

name: Na Homolce Hospital, Prague, Czech Republic

name: New Jersey Neuroscience Institute, Edison

name: Fondazione Ospedale Maggiore Policlinico, Milan, Italy

name: Houston Methodist Hospital, Houston, Texas

name: Hospices Civils de Lyon, University Claude Bernard Lyon 1, Lyon, France

ReferenceCitation: Stupp R, Taillibert S, Kanner A, Read W, Steinberg D, Lhermitte B, Toms S, Idbaih A, Ahluwalia MS, Fink K, Di Meco F, Lieberman F, Zhu JJ, Stragliotto G, Tran D, Brem S, Hottinger A, Kirson ED, Lavy-Shahaf G, Weinberg U, Kim CY, Paek SH, Nicholas G, Bruna J, Hirte H, Weller M, Palti Y, Hegi ME, Ram Z. Effect of Tumor-Treating Fields Plus Maintenance Temozolomide vs Maintenance Temozolomide Alone on Survival in Patients With Glioblastoma: A Randomized Clinical Trial. JAMA. 2017 Dec 19;318(23):2306-2316. doi: 10.1001/jama.2017.18718. Erratum in: JAMA. 2018 May 1;319(17):1824.

name: Lou and Jean MalnatiBrain Tumor Institute of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University Feinberg School of Medicine, Chicago, Illinois

name: Centre Hospitalier Universitaire Vaudois and University of Lausanne, Lausanne, Switzerland

name: Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau et de la Moelle épinière, ICM, F-75013, Paris, France

name: Tel Aviv Medical Center, Tel Aviv, Israel

name: Baylor University Medical Center, Houston, Texas

name: University of Texas Health Sciences Center at Houston

name: University of Pennsylvania, Philadelphia

name: Seoul National University Bundang Hospital, Seoul National University College of Medicine, Bundang, Korea

name: Ottawa Hospital Research Institute, Ottawa, Ontario, Canada

name: Hospital Universitario de Bellvitge, Barcelona, Spain

name: Juravinski Cancer Centre, Hamilton, Ontario, Canada

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

repository_institution: University of Zurich - Zurich Open Repository and Archive

location_country: Italy France Czechia Poland Spain Germany

location_facility: University Hospital Fakultni Nemocnice Olomouc, Oncology Clinic

location_facility: Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu

location_facility: Vall d'Hebron Institute of Oncology (VHIO)

location_facility: HM Hospitales - Centro Integral Oncológico Clara Campal

location_facility: University Medical Center - University of Freiburg


title: REspectful CAring for the AGitated Elderly. How to Best Meet the Needs of People With Dementia With Severe Behavioural Disturbances. Toward a Respectful and Cost-effective Model

acronym: RECAGE

summary: The treatment of Behavioural and Psychological Symptoms of Dementia (BPSD) continue to raise difficult problems. Both the pharmacologic and non-pharmacologic therapies often fail to achieve the expected results. The aim of this study is to assess the short- and long-term efficacy of an intervention, the Special Care Unit for patients with BPSD (SCU-B), already implemented in some countries but not validated so far, as well as its cost-effectiveness

ReferenceCitation: Koskas P, Belqadi S, Mazouzi S, Daraux J, Drunat O. [Behavioral and psychological symptoms of dementia in a pilot psychogeriatric unit: management and outcomes]. Rev Neurol (Paris). 2011 Mar;167(3):254-9. doi: 10.1016/j.neurol.2010.07.030. Epub 2010 Oct 14. French.

ReferenceCitation: Delphin-Combe F, Roubaud C, Martin-Gaujard G, Fortin ME, Rouch I, Krolak-Salmon P. [Effectiveness of a cognitive-behavioral unit on behavioral and psychological symptoms of dementia]. Rev Neurol (Paris). 2013 Jun-Jul;169(6-7):490-4. doi: 10.1016/j.neurol.2012.10.017. Epub 2013 Mar 21. French.

ReferenceCitation: Bianchetti A, Benvenuti P, Ghisla KM, Frisoni GB, Trabucchi M. An Italian model of dementia special care unit: results of a pilot study. Alzheimer Dis Assoc Disord. 1997 Mar;11(1):53-6.

ipd_sharing_description: De-identified participant data both for primary and secondary outcome measures will be made available

lead_sponsor: Fondazione Europea di Ricerca Biomedica Ferb Onlus

location_country: Italy Switzerland France Greece Norway Belgium Germany

location_facility: Zentralinstitut für Seelische Gesundheit

location_facility: Azienda Socio Sanitaria Territoriale di Mantova

location_facility: Azienda Unità Sanitaria Locale di Modena

location_facility: Aristotelio Panepistimio Thessalonikis (Greece)

location_facility: Centre Hospitalier Universitaire St Pierre


title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients

acronym: IMPALA-X

summary:
Clinical trial for subjects with autoimmune pulmonary alveolar proteinosis (aPAP) who have completed the IMPALA trial (NCT02702180).

At the Baseline visit, eligible subjects may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.

Subject will be treated with inhaled molgramostim for up to 36 months.

During the trial, whole lung lavage will be applied as rescue therapy.

lead_sponsor: Savara Inc.

location_country:
Italy
Netherlands
France
Greece
Israel
Russian Federation
Turkey
Denmark
Germany
United Kingdom

location_facility: Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin

location_facility: Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital

location_facility: Pavlov first Saint Petersburg State Medical Univerisity

location_facility: S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo

location_facility: Asklepios Fachkliniken München - Gauting Klinik für Pneumologie

location_facility: Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens

location_facility: Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie

location_facility: Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH

location_facility: Rabin Medical Center Institute of Pulomonary Medicine

location_facility: Dept. Of Intensive Care Unit Royal Brompton Hospital London

location_facility: CHU Rennes Hospital Pontchaillou, Service de Pneumologie


title: A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use)

summary: This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH). Once safety has been established in HV, PH patients with a confirmed diagnosis of PH1 and PH2 will be enrolled across multiple dosing cohorts. The study design will allow enrollment of PH patient cohorts at a given dose level once safety has been demonstrated in HV at that dose level. The study will be conducted in two parts: Part A: Single ascending dose (SAD) in HV; Part B: SAD in patients with PH1 and PH2 (lagging Part A by 1 dose level cohort).

lead_sponsor: Dicerna Pharmaceuticals, Inc.

location_country:
United States
Netherlands
France
Germany
United Kingdom

location_facility: Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie

location_facility: Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon

location_facility: Birmingham Children's Hospital NHS Trust