title: Evaluation of Methods for Extrapolating or Estimating the Size of Children in Pediatric Intensive Care Compared With the Reference Method of the World Health Organization (WHO)

acronym: EvTaReaP

summary:
Size is a key data used daily by dietary teams; the paramedical team, nurse and diet are in charge of its measures.

In pediatric intensive care unit, a reliable size of the child must be obtained. It allows to realize:

a nutritional assessment based on the World Health Organization (WHO) nutritional indices such as the Body Mass Index (BMI), growth chart monitoring and other nutritional indices. Nutritional status should be assessed and followed in pediatric intensive care as it is correlated with the prognosis of children.
an estimate of the energy needs by calculation of the rest energy expenditure.
a calculation of the body surface, useful for drug prescription, evaluation of burn scores, calculation of water and energy requirements and indexing of hemodynamic and ventilatory data.

An error in size measurement results in an error in BMI, calculation of energy requirements, and body surface area.

The WHO has defined "gold standard" criteria for measuring height in children, distinguishing the less than two years in whom the size is measured strictly lengthened, using a metric, and the more than two years in which height is measured standing with a stadiometer. In the context of pediatric resuscitation, the criteria for WHO size measurement are difficult to meet (coma, sedation, respiratory assistance, catheter, monitoring, proclive position, etc.) compromising standing or rectitude required for measurements.

The child is a growing organism. Health book sizes and declarative sizes are not always up-to-date.

It is therefore important to overcome these difficulties by using estimating or extrapolation methods that are applicable and safe in pediatric intensive care unit.

Currently, in pediatric intensive care units, the size evaluation, by direct measurement, estimation or extrapolation of segmental measurement, is not systematic because of the complexity of the measurement; To date, no method is used as a reference method in pediatric intensive care.

Among Children in pediatric intensive care unit (which does not usually meet the criteria of the WHO Gold Standard for Measurement of Height), to determine the optimal method for size measurement, by comparing different methods of estimating / extrapolating the size, gold standard WHO (achievable after the stay in intensive care).

lead_sponsor: Hospices Civils de Lyon

location_country: Belgium France Switzerland Lebanon

location_facility: Service de réanimation et de surveillance continue pédiatrique CHRU de Lille

location_facility: Service de réanimation pédiatrique Centre hospitalier universitaire de Bordeaux

location_facility: Service de réanimation pédiatrique Hôpital Reine Fabiola Hôpital Publique

location_facility: Service de réanimation pédiatrique Rue Du Morvan (CHRU NANCY - HOPITAUX DE BRABOIS)

location_facility: Service de réanimation pédiatrique Hôpital de l'hôtel dieu de France

location_facility: Service de réanimation pédiatrique CHU La Timone AP-HM

location_facility: Service de réanimation pédiatrique Centre hospitalier universitaire de Nantes

lead_sponsor_type: academique


title: International Breast Cancer Intervention Study II (IBIS-II) (DCIS)

acronym: IBIS-II DCIS

summary:
RATIONALE: Estrogen can stimulate the growth of breast cancer cells. Hormone therapy using either tamoxifen or anastrozole may fight breast cancer by blocking the use of estrogen. It is not yet known whether tamoxifen is more effective than anastrozole in preventing breast cancer after surgery for ductal carcinoma in situ.

PURPOSE: This randomized phase III trial is studying how well adjuvant tamoxifen works compared to anastrozole in treating postmenopausal women who have undergone surgery to remove ductal carcinoma in situ.

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: background

ReferenceCitation: Juraskova I, Butow P, Lopez A, Seccombe M, Coates A, Boyle F, McCarthy N, Reaby L, Forbes JF. Improving informed consent: pilot of a decision aid for women invited to participate in a breast cancer prevention trial (IBIS-II DCIS). Health Expect. 2008 Sep;11(3):252-62. doi: 10.1111/j.1369-7625.2008.00498.x.

ReferenceCitation: Forbes JF, Sestak I, Howell A, Bonanni B, Bundred N, Levy C, von Minckwitz G, Eiermann W, Neven P, Stierer M, Holcombe C, Coleman RE, Jones L, Ellis I, Cuzick J; IBIS-II investigators. Anastrozole versus tamoxifen for the prevention of locoregional and contralateral breast cancer in postmenopausal women with locally excised ductal carcinoma in situ (IBIS-II DCIS): a double-blind, randomised controlled trial. Lancet. 2016 Feb 27;387(10021):866-73. doi: 10.1016/S0140-6736(15)01129-0. Epub 2015 Dec 11.

ReferenceCitation: Sestak I, Smith SG, Howell A, Forbes JF, Cuzick J. Early participant-reported symptoms as predictors of adherence to anastrozole in the International Breast Cancer Intervention Studies II. Ann Oncol. 2018 Feb 1;29(2):504-509. doi: 10.1093/annonc/mdx713.

lead_sponsor: Queen Mary University of London

location_country:
Italy
Austria
Chile
Sweden
Switzerland
France
Malta
Hungary
Australia
Belgium
Ireland
Turkey
Germany
United Kingdom

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

location_facility: Centre de Lutte Contre le Cancer Georges-Francois Leclerc

location_facility: Austrian Breast & Colorectal Cancer Study Group

location_facility: Centre Hospitalier Universitaire Vaudois

location_facility: Centre for Cancer Research and Cell Biology at Queen's University Belfast

location_facility: Royal Infirmary of Edinburgh at Little France

location_facility: Hopital Cantonal Universitaire de Geneve

location_facility: Centre Hospital Regional Universitaire de Limoges

intervention_type: Drug

status_simplified: Completed


title: Pre- and Post-Operative Chemotherapy With Oxaliplatin 5FU/LV Versus Surgery Alone in Resectable Liver Metastases From Colorectal Origin - Phase III Study

summary:
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and combining chemotherapy with surgery may kill more tumor cells. It is not yet known if surgery is more effective with or without chemotherapy for liver metastases.

PURPOSE: Randomized phase III trial to compare the effectiveness of surgery with or without combination chemotherapy in treating patients who have liver metastases from colorectal cancer.

status: Unknown status

ReferenceType: result

ReferenceCitation: Sorbye H, Mauer M, Gruenberger T, et al.: Predictive factors for the effect of perioperative FOLFOX for resectable liver metastasis in colorectal cancer patients (EORTC phase III study 40983). [Abstract] J Clin Oncol 28 (Suppl 15): A-3544, 2010.

ReferenceCitation: Sorbye H, Mauer M, Gruenberger T, et al.: Evaluation of carcinoembryonic antigen (CEA) as a predictive baseline factor for the benefit of perioperative FOLFOX in resectable liver metastasis from colorectal cancer (EORTC study 40983). [Abstract] American Society of Clinical Oncology 2010 Gastrointestinal Cancers Symposium, 22-24 January 2010, Orlando, Florida. A-407, 2010.

ReferenceCitation: Benoist S, Nordlinger B. The role of preoperative chemotherapy in patients with resectable colorectal liver metastases. Ann Surg Oncol. 2009 Sep;16(9):2385-90. doi: 10.1245/s10434-009-0492-7. Epub 2009 Jun 25. Review.

family: Benoist

full_name: Stéphane Benoist

given: Stéphane

sequence: first

family: Nordlinger

full_name: Bernard Nordlinger

given: Bernard

sequence: additional

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Annals of Surgical Oncology

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer

publisher_group: Springer

publisher_normalized: Springer

title: The Role of Preoperative Chemotherapy in Patients with Resectable Colorectal Liver Metastases

ReferenceCitation: Nordlinger B, Sorbye H, Glimelius B, Poston GJ, Schlag PM, Rougier P, Bechstein WO, Primrose JN, Walpole ET, Finch-Jones M, Jaeck D, Mirza D, Parks RW, Collette L, Praet M, Bethe U, Van Cutsem E, Scheithauer W, Gruenberger T; EORTC Gastro-Intestinal Tract Cancer Group; Cancer Research UK; Arbeitsgruppe Lebermetastasen und-tumoren in der Chirurgischen Arbeitsgemeinschaft Onkologie (ALM-CAO); Australasian Gastro-Intestinal Trials Group (AGITG); Fédération Francophone de Cancérologie Digestive (FFCD). Perioperative chemotherapy with FOLFOX4 and surgery versus surgery alone for resectable liver metastases from colorectal cancer (EORTC Intergroup trial 40983): a randomised controlled trial. Lancet. 2008 Mar 22;371(9617):1007-16. doi: 10.1016/S0140-6736(08)60455-9.

family: Sorbye

full_name: Halfdan Sorbye

given: Halfdan

family: Glimelius

full_name: Bengt Glimelius

given: Bengt

family: Poston

full_name: Graeme J Poston

given: Graeme J

family: Schlag

full_name: Peter M Schlag

given: Peter M

family: Rougier

full_name: Philippe Rougier

given: Philippe

family: Bechstein

full_name: Wolf O Bechstein

given: Wolf O

family: Primrose

full_name: John N Primrose

given: John N

family: Walpole

full_name: Euan T Walpole

given: Euan T

family: Finch-Jones

full_name: Meg Finch-Jones

given: Meg

family: Jaeck

full_name: Daniel Jaeck

given: Daniel

family: Mirza

full_name: Darius Mirza

given: Darius

family: Parks

full_name: Rowan W Parks

given: Rowan W

family: Collette

full_name: Laurence Collette

given: Laurence

family: Praet

full_name: Michel Praet

given: Michel

family: Bethe

full_name: Ullrich Bethe

given: Ullrich

family: Van Cutsem

full_name: Eric Van Cutsem

given: Eric

family: Scheithauer

full_name: Werner Scheithauer

given: Werner

family: Gruenberger

full_name: Thomas Gruenberger

given: Thomas

repository_institution: University of Edinburgh - Edinburgh Research Explorer

ReferenceCitation: Julie C, Lutz MP, Aust D, et al.: Pathological analysis of hepatic injury after oxaliplatin-based neoadjuvant chemotherapy of colorectal cancer liver metastases: results of the EORTC Intergroup phase III study 40983. [Abstract] American Society of Clinical Oncology 2007 Gastrointestinal Cancers Symposium, 19 -21 January 2007, Orlando, Florida A-241, 2007.

ReferenceCitation: Nordlinger B, Sorbye H, Collette L, et al.: Final results of the EORTC Intergroup randomized phase III study 40983 [EPOC] evaluating the benefit of peri-operative FOLFOX4 chemotherapy for patients with potentially resectable colorectal cancer liver metastases. [Abstract] J Clin Oncol 25 (Suppl 18): A-LBA5, 2007.

ReferenceCitation: Gruenberger T, Sorbye H, Debois M, et al.: Tumor response to pre-operative chemotherapy (CT) with FOLFOX-4 for resectable colorectal cancer liver metastases (LM). Interim results of EORTC Intergroup randomized phase III study 40983. [Abstract] J Clin Oncol 24 (Suppl 18): A-3500, 2006.

ReferenceCitation: Nordlinger B, Sorbye H, Debois M, et al.: Feasibility and risks of pre-operative chemotherapy (CT) with Folfox 4 and surgery for resectable colorectal cancer liver metastases (LM). Interim results of the EORTC Intergroup randomized phase III study 40983. [Abstract] J Clin Oncol 23 (Suppl 16): A-3528, 253s, 2005.

ReferenceCitation: Tanis E, Julié C, Emile JF, Mauer M, Nordlinger B, Aust D, Roth A, Lutz MP, Gruenberger T, Wrba F, Sorbye H, Bechstein W, Schlag P, Fisseler A, Ruers T. Prognostic impact of immune response in resectable colorectal liver metastases treated by surgery alone or surgery with perioperative FOLFOX in the randomised EORTC study 40983. Eur J Cancer. 2015 Nov;51(17):2708-17. doi: 10.1016/j.ejca.2015.08.014. Epub 2015 Sep 2.

ReferenceCitation: Nordlinger B, Sorbye H, Glimelius B, Poston GJ, Schlag PM, Rougier P, Bechstein WO, Primrose JN, Walpole ET, Finch-Jones M, Jaeck D, Mirza D, Parks RW, Mauer M, Tanis E, Van Cutsem E, Scheithauer W, Gruenberger T; EORTC Gastro-Intestinal Tract Cancer Group; Cancer Research UK; Arbeitsgruppe Lebermetastasen und-tumoren in der Chirurgischen Arbeitsgemeinschaft Onkologie (ALM-CAO); Australasian Gastro-Intestinal Trials Group (AGITG); Fédération Francophone de Cancérologie Digestive (FFCD). Perioperative FOLFOX4 chemotherapy and surgery versus surgery alone for resectable liver metastases from colorectal cancer (EORTC 40983): long-term results of a randomised, controlled, phase 3 trial. Lancet Oncol. 2013 Nov;14(12):1208-15. doi: 10.1016/S1470-2045(13)70447-9. Epub 2013 Oct 11.

ReferenceCitation: Sorbye H, Mauer M, Gruenberger T, Glimelius B, Poston GJ, Schlag PM, Rougier P, Bechstein WO, Primrose JN, Walpole ET, Finch-Jones M, Jaeck D, Mirza D, Parks RW, Collette L, Van Cutsem E, Scheithauer W, Lutz MP, Nordlinger B; EORTC Gastro-Intestinal Tract Cancer Group; Cancer Research UK (CRUK); Arbeitsgruppe Lebermetastasen und-tumoren in der Chirurgischen Arbeitsgemeinschaft Onkologie (ALM-CAO); Australasian Gastro-Intestinal Trials Group (AGITG); Fédération Francophone de Cancérologie Digestive (FFCD). Predictive factors for the benefit of perioperative FOLFOX for resectable liver metastasis in colorectal cancer patients (EORTC Intergroup Trial 40983). Ann Surg. 2012 Mar;255(3):534-9. doi: 10.1097/SLA.0b013e3182456aa2.

publisher: Ovid Technologies (Wolters Kluwer Health)

title: Predictive Factors for the Benefit of Perioperative FOLFOX for Resectable Liver Metastasis in Colorectal Cancer Patients (EORTC Intergroup Trial 40983)

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

location_country:
Italy
Austria
Sweden
Hong Kong
Netherlands
France
Norway
Australia
Belgium
New Zealand
Portugal
Germany
United Kingdom

location_facility: Universitaetsklinkum Magdeburg der Otto-von-Guericke-Universitaet Magdeburg

location_facility: Klinikum der Friedrich-Schiller Universitaet Jena

location_facility: Ospedale San Martino/Cliniche Universitarie Convenzionate

location_facility: Robert Roessle Klinik at Charite - Campus Berlin Buch im Helios Klinikum Berlin

location_facility: Clatterbridge Centre for Oncology NHS Trust

location_facility: Royal Liverpool and Broadgreen Hospitals

location_facility: Centre Hospitalier Regional et Universitaire d'Angers

location_facility: Centre Regional de Lutte Contre le Cancer - Centre Val d'Aurelle

location_facility: Cookridge Hospital at Leeds Teaching Hospital NHS Trust

location_facility: Academisch Ziekenhuis der Vrije Universiteit Brussel

location_facility: Institute of Oncology at Prince of Wales Hospital

location_facility: Klinik der Justus - Leibig - Universitaet Giessen

location_facility: Queen Elizabeth Hospital at University of Birmingham

location_facility: Centre Hospitalier Universitaire Henri Mondor

location_facility: Instituto Portugues de Oncologia Centro do Porto, SA

location_facility: Klinikum Rechts Der Isar - Technische Universitaet Muenchen

location_facility: Haukeland Hospital - University of Bergen


status: Recruiting

study_start_date_type: Actual

lead_sponsor: Merck Sharp & Dohme Corp.

enrollment_type: Anticipated

location_country:
Italy
United States
Chile
Peru
South Africa
Spain
Germany
France
Japan
Australia
Puerto Rico
Russian Federation
Portugal
Canada
United Kingdom
Korea, Republic of
Colombia
Ukraine

acronym: DOR/ISL in heavily treatment-experienced participants

location_facility: Saint Petersburg Center for Prophylactic of AIDS and Inf. Diseases ( Site 5101)

location_facility: Holdsworth House Medical Practice ( Site 5300)

location_facility: McGill University Health Center - Research Institute-CVIS Clinical Research Unit ( Site 4102)

location_facility: ASST Fatebenefratelli-Ospedale Sacco ( Site 5000)

location_facility: Azienda Ospedaliero Universitaria di Modena Policlinico ( Site 5004)

location_facility: Hospital Universitario La Paz ( Site 5604)

location_facility: Gbuz Samarskiy Oblastnoy Klinicheskiy Tsentr Profilaktiki I Bor'by So Spid ( Site 5113)

location_facility: Republican Clinical Hospital of Infectious Diseases n. a. A.F.Agafonov ( Site 5104)

location_facility: Center Hospital of the National Center for Global Health and Medicine ( Site 5401)

location_facility: Regional Center for Prevent. and Control of AIDS and Inf. Diseases ( Site 5106)

location_facility: Centro Cardiovascular Cardiosur ( Site 4407)

location_facility: A.P.H. Paris, Hopital Saint Louis ( Site 4714)

location_facility: Hospital Dr. Hernan Henriquez Aravena ( Site 4405)

location_facility: Smolensk Center On Aids And Infectious Diseases Prophylaxis ( Site 5115)

location_facility: The University of Mississippi Medical Center ( Site 4036)

location_facility: Universita' Vita Salute. Ospedale San Raffaele ( Site 5002)

location_facility: Holdsworth House Medical Practice - Brisbane ( Site 5312)

location_facility: Saint Hope Foundation, Inc. ( Site 4034)

location_facility: CHU de Montpellier - Hopital Saint-Eloi ( Site 4721)

location_facility: Centre Hospitalier de Tourcoing ( Site 4700)

location_facility: Policlinico Universidad Nacional Mayor de San Marcos ( Site 4501)

location_facility: North Texas Infectious Diseases Consultants, PA ( Site 4005)

location_facility: Hospital Clinic i Provincial ( Site 5601)

location_facility: Saint Michael's Medical Center-Research - Infectious Disease ( Site 4035)

location_facility: University of Alabama at Birmingham 1917 Research Clinic ( Site 4031)

location_facility: FGU Republican Clinical Infectious Hospital of Roszdrav ( Site 5100)

location_facility: Universitaetsklinikum Essen ( Site 4607)

location_facility: Georgetown University Hospital ( Site 4015)

location_facility: Centro Hospitalar de Sao Joao. EPE - Hospital de Sao Joao ( Site 4907)

location_facility: Mykolaiv center of paliative assistance and integrated services ( Site 5621)

location_facility: Hospital Dr. Fernando Fonseca, EPE - Amadora/Sintra ( Site 4902)

location_facility: Vancouver ID Research and Care Centre Society ( Site 4100)

location_facility: MNE Odesa Regional Center of Socially Significant Diseases ( Site 5611)

location_facility: Triple O Research Institute, P.A. ( Site 4020)

location_facility: Ottawa Hospital Research Institute ( Site 4111)

location_facility: Centro Hospitalar de Lisboa Norte Hospital de Santa Maria ( Site 4913)

location_facility: Ospedale San Gerardo ASST Monza ( Site 5012)

location_facility: Toronto General Hospital - University Health Network ( Site 4105)

location_facility: Wits Clinical HIV Research Unit ( Site 4804)

location_facility: Istituto Nazionale per Le Malattie Infettive Lazzaro Spallanzani ( Site 5005)

location_facility: Hospital Geral de Santo Antonio ( Site 4908)

location_facility: CHU de Nice Hopital Archet 1 ( Site 4703)

location_facility: Palmtree Clinical Research, Inc. ( Site 4016)

location_facility: ZIBP-Zentrum fur Infektiologie Berlin Prenzlauer Berg GmbH ( Site 4603)

location_facility: ICH Study Center GmbH & Co.KG ( Site 4609)

location_facility: Institute of Epidemiology and Infect Diseases of the NAMS of Ukraine ( Site 5615)

location_facility: MI Vinnytsia Regional Center of AIDS Prevention and Care ( Site 5618)

location_facility: Kyiv City Clinical Hospital 5 ( Site 5616)

location_facility: Icahn School of Medicine at Mount Sinai ( Site 4000)

location_facility: Dnipropetrovsk Regional Center of Socially Significant Diseases ( Site 5619)

location_facility: Hospital de Nossa Senhora da Oliveira- EPE ( Site 4905)

location_facility: Policlinico Gemelli Instituto di Clinica Chirurgica ( Site 5006)

location_facility: Hospital Universitari Germans Trias i Pujol ( Site 5600)

location_facility: A.P.H. Paris. Hopital Bichat Claude Bernard ( Site 4724)

location_facility: Regional Clinical Infectious Hospital ( Site 5614)

location_facility: Pusan National University Hospital ( Site 5503)

location_facility: Hopital Hotel Dieu [Paris, France] ( Site 4723)

location_facility: Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico ( Site 5001)

location_facility: Chatham County Health Department ( Site 4029)

location_facility: CHU de Bordeaux- Hopital Saint Andre ( Site 4715)

location_facility: Kherson City Clinical Hospital n.a. Y.Y. Karabelesh ( Site 5620)

location_facility: IRCCS Policlinico San Matteo ( Site 5010)

location_facility: The Catholic University of Korea. Seoul St. Mary s Hospital ( Site 5502)

location_facility: Infectious Clinical Hospital #2 ( Site 5114)

location_facility: Severance Hospital Yonsei University Health System ( Site 5500)

location_facility: Monash Health-Monash Medical Centre ( Site 5313)

location_facility: Clinica Colsanitas S.A. Sede Clinica Universitaria Colombia ( Site 4306)

location_facility: Medizinische Hochschule Hannover ( Site 4612)

location_facility: Azienda Ospedaliera San Paolo ( Site 5003)

location_facility: University of North Carolina at Chapel Hill ( Site 4026)

ipd_sharing: Yes

study_completion_date_type: Anticipated

type: Other Identifier

type: EudraCT Number

type: Registry Identifier

source: euctr

title: A Phase 3, Randomized, Clinical Study in HIV-1-Infected Heavily Treatment-Experienced Participants Evaluating the Antiretroviral Activity of Blinded Islatravir (ISL), Doravirine (DOR), and Doravirine/Islatravir (DOR/ISL), Each Compared to Placebo, and the Antiretroviral Activity, Safety, and Tolerability of Open-Label DOR/ISL

summary: This is a 2-part, phase 3 clinical study evaluating the antiretroviral activity and safety/tolerability of islatravir (ISL), doravirine (DOR), and a fixed dose combination (FDC) of DOR/ISL (also known as MK-8591A) in heavily treatment-experienced (HTE) participants with human immunodeficiency virus type 1 (HIV-1) infection. It is hypothesized that the percentage of participants receiving DOR/ISL to achieve ≥0.5 log10 decrease in HIV-1 ribonucleic acid (RNA) from study baseline (Day 1) to Day 8 is superior to placebo, each given in combination with failing antiretroviral therapy (ART).

lead_sponsor_type: industriel

status_simplified: Ongoing


title: A Phase 2/3 Trial of the Efficacy and Safety of Bardoxolone Methyl in Patients With Alport Syndrome

acronym: CARDINAL

summary: This international, multi-center, Phase 2/3 trial will study the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with Alport syndrome. The Phase 2 portion of the trial will be open-label and enroll up to 30 patients. The Phase 3 portion of the trial will be double-blind, randomized, placebo-controlled and will enroll up to 180 patients.

ReferenceType: derived

ReferenceCitation: Chertow GM, Appel GB, Andreoli S, Bangalore S, Block GA, Chapman AB, Chin MP, Gibson KL, Goldsberry A, Iijima K, Inker LA, Knebelmann B, Mariani LH, Meyer CJ, Nozu K, O'Grady M, Silva AL, Stenvinkel P, Torra R, Warady BA, Pergola PE. Study Design and Baseline Characteristics of the CARDINAL Trial: A Phase 3 Study of Bardoxolone Methyl in Patients with Alport Syndrome. Am J Nephrol. 2021;52(3):180-189. doi: 10.1159/000513777. Epub 2021 Mar 31.

family: Chertow

full_name: Glenn M. Chertow

given: Glenn M.

family: Appel

full_name: Gerald B. Appel

given: Gerald B.

family: Andreoli

full_name: Sharon Andreoli

given: Sharon

family: Bangalore

full_name: Sripal Bangalore

given: Sripal

family: Block

full_name: Geoffrey A. Block

given: Geoffrey A.

family: Chapman

full_name: Arlene B. Chapman

given: Arlene B.

family: Chin

full_name: Melanie P. Chin

given: Melanie P.

family: Gibson

full_name: Keisha L. Gibson

given: Keisha L.

family: Goldsberry

full_name: Angie Goldsberry

given: Angie

family: Iijima

full_name: Kazumoto Iijima

given: Kazumoto

family: Inker

full_name: Lesley A. Inker

given: Lesley A.

family: Knebelmann

full_name: Bertrand Knebelmann

given: Bertrand

family: Mariani

full_name: Laura H. Mariani

given: Laura H.

family: Meyer

full_name: Colin J. Meyer

given: Colin J.

family: Nozu

full_name: Kandai Nozu

given: Kandai

family: O’Grady

full_name: Megan O’Grady

given: Megan

family: Silva

full_name: Arnold L. Silva

given: Arnold L.

family: Stenvinkel

full_name: Peter Stenvinkel

given: Peter

family: Torra

full_name: Roser Torra

given: Roser

family: Warady

full_name: Bradley A. Warady

given: Bradley A.

family: Pergola

full_name: Pablo E. Pergola

given: Pablo E.

journal_name: American Journal of Nephrology

oa_colors: green other

oa_host_type: publisher;repository

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

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

ReferenceCitation: Kashtan CE, Gross O. Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults-an update for 2020. Pediatr Nephrol. 2021 Mar;36(3):711-719. doi: 10.1007/s00467-020-04819-6. Epub 2020 Nov 6. Review. Erratum in: Pediatr Nephrol. 2021 Jan 12;:.

title: Clinical practice recommendations for the diagnosis and management of Alport syndrome in children, adolescents, and young adults–an update for 2020

location_country:
United States
France
Japan
Australia
Puerto Rico
Spain
Germany
United Kingdom

location_facility: Children's Research Institute - The Children's Mercy Hospital

location_facility: Arizona Kidney Disease and Hypertension Research Services, PLLC

location_facility: The Warren Alpert Medical School of Brown University

location_facility: Children's Hospital of Philadelphia (CHOP)

location_facility: Washington University School of Medicine

location_facility: Brookview Hills Research Associates, PLLC

location_facility: Emory University School of Medicine and Children's Healthcare of Atlanta

location_facility: Hopital Necker-Universite Paris Descartes

location_facility: University of Minnesota - Division of Pediatric Nephrology

location_facility: University of Pittsburgh School of Medicine - Division of Pediatric Nephrology

location_facility: University of California San Francisco - Children's Renal Center

location_facility: Tokyo Metropolitan Children's Medical Center

location_facility: General Clinical Research Center - Parnassus

location_facility: University Children's Hospital Heidelberg

location_facility: Puerto Rico Clinical & Translational Research Center

location_facility: South Carolina Nephrology & Hypertension Center, Inc

location_facility: Japanese Red Cross Nagoya Daini Hospital


intervention_type: Other

lead_sponsor: Mirum Pharmaceuticals, Inc.

location_country:
Italy
Austria
Brazil
Lebanon
Mexico
Germany
France
Poland
Singapore
Hungary
Argentina
Belgium
Turkey
United States
United Kingdom
Canada
Colombia

acronym: MARCH-PFIC

location_facility: Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon

location_facility: Instytut Pomnik Centrum, Zdrowia Dziecka

location_facility: VUMC (Vanderbilt University School of Medicine)

location_facility: University of Texas, Health Science Center San Antonio

location_facility: University of Alberta, Edmonton Clinic Health Academy

location_facility: Michigan Medicine (University of Michigan)

location_facility: Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK)

location_facility: Sociedade Beneficente de Senhoras Hospital Sírio-Libanês

location_facility: Koc University Research Center for Translational Medicine (KUTTAM)

location_facility: Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde

location_facility: Consultario de Joshue David Covarrubias Esquer

location_facility: University of Texas Southwestern Medical Center

location_facility: Hospital Papa Giovanni XXIII / Unità di Pediatria

location_facility: Columbia University, Irving Medical Center

ipd_sharing: No

title: MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC

summary: The purpose of this study is to determine if the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).


ReferenceCitation: Bines J, Clark E, Barton C, Restuccia E, Procter M, Sonnenblick A, Fumagalli D, Parlier D, Arahmani A, Baselga J, Viale G, Reaby LL, Frank E, Gelber RD, Piccart M, Jackisch C, Petersen JA. Patient-reported function, health-related quality of life, and symptoms in APHINITY: pertuzumab plus trastuzumab and chemotherapy in HER2-positive early breast cancer. Br J Cancer. 2021 Jul;125(1):38-47. doi: 10.1038/s41416-021-01323-y. Epub 2021 Apr 7.

family: Bines

full_name: José Bines

given: José

family: Clark

full_name: Emma Clark

given: Emma

family: Barton

full_name: Claire Barton

given: Claire

family: Restuccia

full_name: Eleonora Restuccia

given: Eleonora

family: Procter

full_name: Marion Procter

given: Marion

family: Sonnenblick

full_name: Amir Sonnenblick

given: Amir

family: Fumagalli

full_name: Debora Fumagalli

given: Debora

family: Parlier

full_name: Damien Parlier

given: Damien

family: Arahmani

full_name: Amal Arahmani

given: Amal

family: Baselga

full_name: José Baselga

family: Viale

full_name: Giuseppe Viale

given: Giuseppe

family: Reaby

full_name: Linda L. Reaby

given: Linda L.

family: Frank

full_name: Elizabeth Frank

given: Elizabeth

family: Gelber

full_name: Richard D. Gelber

given: Richard D.

family: Piccart

full_name: Martine Piccart

given: Martine

family: Jackisch

full_name: Christian Jackisch

given: Christian

family: Petersen

full_name: Jennifer A. Petersen

given: Jennifer A.

journal_name: British Journal of Cancer

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

ReferenceCitation: Piccart M, Procter M, Fumagalli D, de Azambuja E, Clark E, Ewer MS, Restuccia E, Jerusalem G, Dent S, Reaby L, Bonnefoi H, Krop I, Liu TW, Pieńkowski T, Toi M, Wilcken N, Andersson M, Im YH, Tseng LM, Lueck HJ, Colleoni M, Monturus E, Sicoe M, Guillaume S, Bines J, Gelber RD, Viale G, Thomssen C; APHINITY Steering Committee and Investigators. Adjuvant Pertuzumab and Trastuzumab in Early HER2-Positive Breast Cancer in the APHINITY Trial: 6 Years' Follow-Up. J Clin Oncol. 2021 May 1;39(13):1448-1457. doi: 10.1200/JCO.20.01204. Epub 2021 Feb 4.

name: Institut Jules Bordet and L'Université Libre de Bruxelles (ULB), Brussels, Belgium

name: Frontier Science Scotland Ltd, Kincraig, Kingussie, United Kingdom

name: Breast International Group (BIG), Brussels, Belgium

family: de Azambuja

full_name: Evandro de Azambuja

given: Evandro

name: Roche Products Limited, Welwyn Garden City, United Kingdom

name: University of Texas, MD Anderson Cancer Center, Huston, TX

family: Ewer

name: Hoffmann-La Roche Ltd, Basel, Switzerland

name: CHU Liege and Liege University, Liege, Belgium

name: Duke Cancer Institute, Duke University, Durham, NC

name: Inaugural Chair—Consumer Advisory Panel, Breast Cancer Trials Group, Newcastle, Australia

name: Consumer Advisor to Breast Researchers, Garvan Institute of Research, Sydney, Australia

name: Institute Bergonié, UNICANCER, University of Bordeaux, Bordeaux, France

name: Dana-Farber Cancer Institute, Boston, MA

name: National Health Research Institutes, Taipei, Taiwan

name: Oncological Department, Postgraduate Medical Education Center, Warsaw, Poland

name: Breast Cancer Unit, Kyoto University Hospital, Kyoto, Japan

name: Director of Medical Oncology, Westmead Hospital, Sydney

name: Associate Professor of Medicine, University of Sydney, Sydney, Australia

name: Department of Oncology, Rigshospitalet, University Hospital, Copenhagen, Denmark

name: Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea

name: Comprehensive Breast Health Center, Experimental Surgery, Department of Surgery, Taipei-Veterans General Hospital, Taipei, Taiwan

name: Gynäkologisch-Onkologische Praxis Hannover, Hannover, Germany

name: Division of Medical Senology, IEO, European Institute of Oncology, IRCCS, Milan, Italy

name: Instituto Nacional de Câncer, Rio de Janeiro, Brazil

name: Dana-Farber Cancer Institute, Harvard Medical School, Harvard TH Chan School of Public Health, Frontier Science Foundation, Boston, MA

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

name: Martin-Luther-University Halle-Wittenberg, Halle (Saale), Germany

name: for the APHINITY Steering Committee and Investigators

repository_institution: University of Liège - Open Repository and Bibliography

repositories_institution: University of Liège - Open Repository and Bibliography

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: Bines J, Procter M, Restuccia E, Viale G, Zardavas D, Suter T, Arahmani A, Van Dooren V, Baselga J, Clark E, Eng-Wong J, Gelber RD, Piccart M, Mobus V, de Azambuja E; APHINITY Steering Committee and Investigators. Incidence and Management of Diarrhea With Adjuvant Pertuzumab and Trastuzumab in Patients With Human Epidermal Growth Factor Receptor 2-Positive Breast Cancer. Clin Breast Cancer. 2020 Apr;20(2):174-181.e3. doi: 10.1016/j.clbc.2019.06.016. Epub 2019 Sep 5.

ReferenceCitation: Luo Y, Li W, Jiang Z, Zhang Q, Wang L, Mao Y, Tjan-Heijnen VCG, Im SA, McConnell R, Bejarano S, Fumagalli D, Bines J, Wang B, Garg A, Kirschbrown WP, Xu B. Pharmacokinetics of pertuzumab administered concurrently with trastuzumab in Chinese patients with HER2-positive early breast cancer. Anticancer Drugs. 2019 Sep;30(8):866-872. doi: 10.1097/CAD.0000000000000808.

ReferenceCitation: Kirschbrown WP, Kågedal M, Wang B, Lindbom L, Knott A, Mack R, Monemi S, Nijem I, Girish S, Freeman C, Fumagalli D, McConnell R, Jerusalem G, Twelves C, Baselga J, von Minckwitz G, Bines J, Garg A. Pharmacokinetic and exploratory exposure-response analysis of pertuzumab in patients with operable HER2-positive early breast cancer in the APHINITY study. Cancer Chemother Pharmacol. 2019 Jun;83(6):1147-1158. doi: 10.1007/s00280-019-03826-1. Epub 2019 Apr 11.

repository_institution: Université Libre de Bruxelles - Dépôt institutionnel de l'Université libre de Bruxelles

title: Pharmacokinetic and exploratory exposure–response analysis of pertuzumab in patients with operable HER2-positive early breast cancer in the APHINITY study

ReferenceCitation: von Minckwitz G, Procter M, de Azambuja E, Zardavas D, Benyunes M, Viale G, Suter T, Arahmani A, Rouchet N, Clark E, Knott A, Lang I, Levy C, Yardley DA, Bines J, Gelber RD, Piccart M, Baselga J; APHINITY Steering Committee and Investigators. Adjuvant Pertuzumab and Trastuzumab in Early HER2-Positive Breast Cancer. N Engl J Med. 2017 Jul 13;377(2):122-131. doi: 10.1056/NEJMoa1703643. Epub 2017 Jun 5. Erratum in: N Engl J Med. 2017 Aug 17;377(7):702. Erratum in: N Engl J Med. 2018 Oct 18;379(16):1585.

name: From the German Breast Group, Neu-Isenburg, Germany (G.M.); Frontier Science (Scotland), Kincraig, United Kingdom (M. Procter); the Breast European Adjuvant Study Team (BrEAST) Data Center (E.A., N.R.), the Breast International Group (D.Z., A.A.), and Institut Jules Bordet, Université Libre de Bruxelles (M. Piccart) — all in Brussels; Roche Pharma (M.B., E.C., A.K.) and the Department of Cardiology, Cardio-Oncology, Bern University Hospital (T.S.) — both in Bern, Switzerland; the Department of Pathology,...

title: Adjuvant Pertuzumab and Trastuzumab in Early HER2-Positive Breast Cancer

location_country:
Peru
Guatemala
Romania
Australia
Belgium
Russian Federation
Denmark
Taiwan
Ukraine
Croatia
France
Japan
Panama
Poland
Thailand
Argentina
El Salvador
Canada
Slovenia
Korea, Republic of
Colombia
South Africa
Chile
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Hong Kong
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Philippines
Switzerland
Bulgaria
Hungary
Ireland
China
Germany
United States
United Kingdom
Italy
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Mexico
Czechia
Israel
New Zealand
Spain

location_facility: The Affiliated Hospital of Military Medical Sciences(The 307th Hospital of Chinese PLA)

location_facility: Uni Hospital Linkoeping; Dept. of Oncology

location_facility: Gesundheitszentrum St. Marien GmbH; Med. II, Hämatologie/Onkologie

location_facility: Lvov State Regional Oncology Medical & Diagnostic Center

location_facility: Arcispedale Santa Maria Nuova; Oncologia

location_facility: Corporacio Sanitaria Parc Tauli; Servicio de Oncologia

location_facility: Schwarzwald-Baar Klinikum Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Orszagos Onkologiai Intezet; B Belgyogyaszati Osztaly

location_facility: Complejo Hospitalario Universitario de Santiago (CHUS) ; Servicio de Oncologia

location_facility: Centro Oncologico MD Anderson Internacional; Servicio de Oncologia

location_facility: Az. Osp. Di Busto P.O. Di Saronno; U.O. Di Oncologia Medica

location_facility: Chaim Sheba Medical Center; Oncology Dept

location_facility: Hospital Universitari Germans Trias i Pujol; Servicio de Oncologia

location_facility: St James Uni Hospital; Icrf Cancer Medicine Research Unit

location_facility: Universitätsklinikum Ulm Am Michelsberg; Frauenklinik

location_facility: Niigata Cancer Ctr Hospital; Breast Surgery

location_facility: Irccs Istituto Europeo Di Oncologia (IEO); Ricerca Di Senologia Medica

location_facility: Centro Estatal De Cancerologia De Durango; Oncology

location_facility: Petz Aladar Megyei Oktato Korhaz; Oncoradiologia

location_facility: Fakultni nemocnice Olomouc; Onkologicka klinika

location_facility: IRCCS Istituto Clinico Humanitas; Oncologia

location_facility: Instituto Valenciano Oncologia; Oncologia Medica

location_facility: Saitama Medical University International Medical Center; Breast Oncology

location_facility: University of North Carolina-Chapel Hill

location_facility: Krankenhaus Der Stadt Wien-Hietzing; Abt. Für Gynäkologie U. Geburtshilfe

location_facility: Cancer Centers of the Carolina; Eastside Medical Center

location_facility: Mount Vernon Hospital; Centre For Cancer Treatment

location_facility: Tiroler Landeskrankenanstalten Ges.M.B.H.; Abt. Für Gynäkologie

location_facility: SANA Klinikum Hameln-Pyrmont; Frauenklinik / Brustzentrum

location_facility: Institut Paoli Calmettes; Oncologie Medicale

location_facility: Ospedale della Misericordia e Dolce; Radiologia

location_facility: Narodowy Inst.Onkologii im.Sklodowskiej-Curie Panstw.Inst.Bad; Klinika Nowtw.Piersi i Chir.Rekonstr

location_facility: Cancer Center of Acadiana at Lafayette General

location_facility: Cancer Care Centers of South Texas-HOAST - San Antonio

location_facility: Clinical Oncology Dispensary of Ministry of Health of Tatarstan

location_facility: Aichi Cancer Center Hospital, Breast Oncology

location_facility: Texas Oncology-Baylor Sammons Cancer Center

location_facility: Kyunghee University Hospital; Endocrinology

location_facility: National Cancer Center Hospital East; Breast and Medical Oncology

location_facility: Hospital Provincial de Castellon; Servicio de Oncologia

location_facility: Queen Alexandra Hospital; Haematology and Oncology Centre

location_facility: Christie Hospital; Breast Cancer Research Office

location_facility: Mater Misericordiae Hospital; Chemotherapy Cottage

location_facility: Northeastern Ontario; Regional Cancer Centre

location_facility: Fondazione Del Piemonte Per L'oncologia Ircc Di Candiolo; Dipartimento Oncologico

location_facility: Galway University Hospital; Clinical Trials Department

location_facility: Hospital General Universitario de Elche; Servicio de Oncologia

location_facility: SBI of Healthcare Samara Regional Clinical Oncology Dispensary

location_facility: Uni Hospital of North Staffordshire; Staffordshire Oncology Centre

location_facility: A. Ö. Krankenhaus Der Barmherzigen Brüder; Interne Abt.

location_facility: Westmead Hospital; Medical Oncology and Pallative Care

location_facility: Klinikum Kreuzschwestern Wels; Iii. Interne Abt.

location_facility: Klinikum der Universität München; Frauenklinik - Onkologie II

location_facility: Hospital Universitario Reina Sofia; Servicio de Oncologia

location_facility: Azienda Ospedaliera S. Maria - Terni; Oncologia

location_facility: MULTISCAN, s.r.o., Radiologicke centrum Pardubice

location_facility: University of Missouri-Columbia; Ellis Fischel Cancer Center

location_facility: Städtische Kliniken Frankfurt am Main Höchst

location_facility: Sankt Elisabeth Krankenhaus; Gynaekology

location_facility: Universitätsmedizin Mainz; Klinik u. Poliklinik f. Geburtshilfe u. Frauenheilkunde

location_facility: RoMed Klinikum Rosenheim; Klinik für Gynäkologie und Geburtshilfe

location_facility: Luisenkrankenhaus GmbH & Co. KG., Brustzentrum

location_facility: Tri-Service General Hospital, Division of General Surgery

location_facility: Hospital General de México; Unidad de Oncologia

location_facility: HELIOS Klinikum Berlin-Buch; Klinik für Gynäkologie und Geburtshilfe

location_facility: Evangelisches Krankenhaus; Abt. Gynäkologie und Geburtshilfe

location_facility: Mount Sinai Beth Israel Comprehensive Cancer Center

location_facility: Natl Hosp Org Shikoku; Cancer Ctr, Surgery

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

location_facility: Carolina Oncology Specialists, PA - Hickory

location_facility: Karolinska University Hospital; Department of General Oncology

location_facility: Hospital Universitario Miguel Servet; Servicio Oncologia

location_facility: Northwest Georgia Oncology Centers PC - Marietta

location_facility: Ist. Uni Federico Ii; Divisione Di Oncologia Medica - Dpt. Di Medicina Interna

location_facility: Centre Georges-François Lecler; Ctr de Lutte Contre le Canc

location_facility: Sir Charles Gairdner Hospital; Medical Oncology

location_facility: Institut Gustave Roussy; Oncologie Medicale

location_facility: Complejo Hospitalario de Jaen-Hospital Universitario Medico Quirurgico; Servicio de Oncologia

location_facility: Praxisgemeinschaft; Frauenärzte am Bahnhofsplatz

location_facility: St. Josefs-Hospital Wiesbaden GmbH; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Tom Baker Cancer Centre; Dept of Medicine

location_facility: Hematology Oncology Associates of the Treasure Coast

location_facility: Hopital Augustin Morvan; Federation De Cancerologie

location_facility: Beaumont Hospital; Cancer Clinical Trials Unit

location_facility: Changhua Christian Hospital; Dept of Surgery

location_facility: Polyclinique De Courlancy; Centre Radiotherapie Oncologie

location_facility: National Cancer Center Hospital; Breast and Medical Oncology

location_facility: IRCCS Fondazione Maugeri; Oncologia Medica I

location_facility: S.I. Russian Oncological Research Center n.a. N.N. Blokhin

location_facility: Shanghai Jiao Tong University School of Medicine (SJTUSM) - Ruijin Hospital (GuangCi Hospital)

location_facility: Royal Devon & Exeter Hospital; Oncology Centre

location_facility: Centre Alexis Vautrin; Oncologie Medicale

location_facility: Frauenarzt-Zentrum Zehlendorf an der Teltower Eiche

location_facility: The Mark H. Zangmeister Ctr; Mid Ohio Onc/Hem Inc.

location_facility: Lkh Vöcklabruck; I. Abt. Für Innere Medizin

location_facility: Tokyo Metropolitan; Komagome Hospital, Surgery

location_facility: Universitätsfrauen- und Poliklinik am Klinikum Suedstadt

location_facility: Bacs-Kiskun Megyei Korhaz, SZTE AOK Oktato Korhaza, Onkoradiologiai Kozpont

location_facility: Centre Leon Berard; Departement Oncologie Medicale

location_facility: Hospital Universitari Arnau de Vilanova de Lleida; Servicio de Oncologia

location_facility: UMHAT Tsaritsa Yoanna - ISUL; Clinic of Oncotherapy

location_facility: Complejo Hospitalario de Toledo- H. Virgen de la Salud; Servicio de Oncologia

location_facility: Hämatologisch-Onkologische Schwerpunktpraxis; Dres. Schlag & Schöttker

location_facility: Albert Einstein Healthcare Network ; Cancer Center

location_facility: Universitätsklinikum Halle (Saale); Universitätsklinik Und Poliklinik Für Gynäkologie

location_facility: National Cheng Kung Uni Hospital; Surgery

location_facility: Klinikum Esslingen; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Sjællands Universitetshospital, Næstved; Onkologisk Afdeling

location_facility: Praxisklinik Krebsheilkunde für Frauen / Brustzentrum (Dres. Kittel/Klare)

location_facility: Shizuoka General Hospital; Breast Surgery

location_facility: The Cancer Inst. Hosp. of JFCR; Breast Oncology Center

location_facility: Chum Hopital Notre Dame; Centre D'Oncologie

location_facility: Credit Valley Hospital/Carlo Fidani Peel Regional Cancer Centre

location_facility: Isis Centro Especializado de Luces; Oncology

location_facility: SHATOD Dr. Marko Antonov Markov-Varna, EOOD

location_facility: Nordsjællands Hospital, Hillerød, Onkologisk Afdeling

location_facility: St. Elisabeth Krankenhaus Köln GmbH; Gynäkologie und Geburtshilfe

location_facility: Sydvestjysk Sygehus Esbjerg; Onkologisk afdeling

location_facility: Palmerston North Hospital; Regional Cancer Treatment Service

location_facility: Washington University School of Medicine

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

location_facility: Cancer Specialists; North Florida ;Jacksonville (AC Skinner Pkwy)

location_facility: Northwest Cancer Specialists - Portland (NE Hoyt St)

location_facility: Opolskie Centrum Onkologii;Oddzial Onkologii Klinicznej

location_facility: Royal Cornwall Hospital; Dept of Clinical Oncology

location_facility: Medizinisches Versorgungszentrum am Klinikum Oldenburg GmbH

location_facility: University Of Santo Tomas; Oncology; Benavides Cancer Institute

location_facility: Veterans Memorial Medical Ctr; Cancer Research Centre

location_facility: Universitätsklinikum Greifswald; Klinik für Frauenheilkunde und Brustzentrum

location_facility: Centre Henri Becquerel; Oncologie Medicale

location_facility: Florida Cancer Specialists, Research Department

location_facility: South Carolina Oncology Associates - SCRI

location_facility: MVZ für Hämatologie, Onkologie, Strahlentherapie und Palliativmedizin -; Klinik Dr. Hancken

location_facility: SBI of Healthcare of Stavropol region Stavropol Regional Clinical Oncology Dispensary

location_facility: Sygehus Syd Roskilde; Onkologisk/haematologisk ambulatorium

location_facility: Hospital de la Santa Creu i Sant Pau; Servicio de Oncologia

location_facility: Dres. Arnd Nusch Naser Ali-Mohammad Kalhori und Werner Langer

location_facility: Universitätsklinikum des Saarlandes; Klinik f. Frauenheilkunden und Geburtshilfe

location_facility: Hospital General Universitario J.M Morales Meseguer; Servicio de Oncologia

location_facility: UNI-Klinikum Campus Kiel Klinik für Gynäkologie und Geburtshilfe

location_facility: Quincy Medical Group; Canc Ctr at Blessing Hosp

location_facility: St. Bartholomew'S Hospital; Dept of Medical Oncology

location_facility: Hamilton Health Sciences - Juravinski Cancer Centre

location_facility: Campus Universitario S.Venuta; Centro Oncologico T.Campanella

location_facility: Fujian Medical University Union Hospital

location_facility: Centro Oncológico Gallego José Antonio Quiroga y Piñeiro, Servicio de Oncologia

location_facility: Cheltenham General Hospital; Gloucestershire Oncology Centre

location_facility: Inst. Nacional de Cancerologia; Clinica de Seno

location_facility: Universitätsklinikum Münster; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Weston Park Hospital; Cancer Clinical Trials Centre

location_facility: Odense Universitetshospital, Onkologisk Afdeling R

location_facility: Aalborg Universitetshospital, Klinik Kirurgi-Kræft, Onkologisk afd.

location_facility: Western General Hospital; Edinburgh Breast Unit

location_facility: Yonsei University Severance Hospital; Medical Oncology

location_facility: Korea University Anam Hospital; Oncology Haemotology

location_facility: Kaohsiung Medical Uni Chung-Ho Hospital; Dept of Surgery

location_facility: Hospital Angeles Metropolitano; Room 220

location_facility: Gunma University Hospital; Department of Thoracic and Visceral Organ Surgery

location_facility: Hebei Medical University Fourth Hospital;(Tumor Hospital of Hebei Province)

location_facility: Newcastle Mater Misericordiae Hospital; Oncology

location_facility: Centro de Diagnóstico y Tratamiento Integral de Mama, Hospital San José Tec de Monterrey

location_facility: Hospital Univ. Nuestra Señora de Valme; Servicio de Oncologia

location_facility: Kantonsspital St. Gallen; Onkologie/Hämatologie

location_facility: Bialostockie Ctr Onkologii; Oddzial Chemioterapii Dziennej

location_facility: The Clatterbridge Cancer Ctr For Oncolgy

location_facility: Wesley Medical Centre; Clinic For Haematology and Oncology

location_facility: Centre Francois Baclesse; Recherche Clinique

location_facility: Nationales Centrum für Tumorerkrankungen (NCT) ; Gyn. Onk. Frauenklinik; Uniklinikum Heidelberg

location_facility: Centro Regional de Enfermedades Oncológicas, S.A.

location_facility: Ospedale S. Croce Di Fano; Servizio Oncologia

location_facility: Lismore Base Hospital; Cancer Care & Haematology Unit

location_facility: Luzerner Kantonsspital; Medizinische Onkologie

location_facility: Institut Claudius Regaud; Departement Oncologie Medicale

location_facility: Hospital Universitario Virgen Macarena; Servicio de Oncologia

location_facility: Cancer Centre of Southeastern Ontario; Kingston General Hospital

location_facility: Taichung Veterans General Hospital; Dept of Surgery

location_facility: Wits Donald Gordon Clinical Trial Centre; Medical Oncology

location_facility: ViDia Christliche Kliniken Karlsruhe, Vincentius-Diakonissen-Kliniken gAG; Frauenklinik

location_facility: Ospedale Mater Salutis; Dept of Oncology

location_facility: Hämatologisch/Onkologische Praxis Dr. Herbrick - Zipp/Prof. Dr. Decker, Studienzentrum

location_facility: Tokai University Hospital, Breast and Endocrine Surgery

location_facility: Buddhachinaraj Phitsanulok Hospital; Chemotherapy Unit ; Department of Medicine

location_facility: Herlev Hospital; Afdeling for Kræftbehandling

location_facility: Klinikum Chemnitz gGmbH; Frauen- und Kinderklinik

location_facility: Marien-Hospital Witten; Frauenklinik Brustzentrum

location_facility: Songklanagarind Hospital; Department of Surgery

location_facility: Hopital Hautepierre; Hematologie Oncologie

location_facility: Brustzentrum Rhein-Ruhr Servicegesellschaft mbH

location_facility: Klinik Johann Wolfgang von Goethe Uni; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Institut d'oncologie de l'Orangerie; Chimiotherapie

location_facility: Klinikum Kassel GmbH; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Hospital Ramon y Cajal; Servicio de Oncologia

location_facility: Cancer Hospital Chinese Academy of Medical Sciences.

location_facility: Shizuoka Cancer Center; Female Internal Medicine

location_facility: Seoul National University Bundang Hospital

location_facility: Klinikum rechts der Isar der TU München; Klinik und Poliklinik für Frauenheilkunde

location_facility: Providence St. Mary Regional Cancer Center

location_facility: Universitätsklinikum Schleswig-Holstein / Campus Lübeck; Klinik für Frauenheilkunde und Geburtshilfe

location_facility: Mater Misericordiae Uni Hospital; Oncology

location_facility: Kumamoto City Hospital, Breast and Endocrine Surgery

location_facility: Royal Berkshire Hospital; Berkshire Cancer Centre

location_facility: Centre Hospitalier Departemental Les Oudairies

location_facility: Hospital Arnau de Vilanova (Valencia) Servicio de Oncologia

location_facility: Uni Hospital Split; Oncology & Radiotherapy

location_facility: The 1st Affiliated Hospital of Nanchang Unversity

location_facility: Policlinico di Monza; Istituto di Oncologia

location_facility: IInstituto Oncologico de San Sebastian, Oncologikoa; Servicio de Oncologia

location_facility: Beth Israel Deac Med Ctr; East Campus Rsch Pharmacy

location_facility: Harbin Medical University Cancer Hospital

location_facility: DONAU ISAR Klinikum Deggendorf; Frauenklinik

location_facility: Waikato Hospital; Dept of Medical Oncology

location_facility: Xiehe Hospital, Tongji Medical College Huazhong University of Science & Technology

location_facility: Irccs Centro Di Riferimento Oncologico (CRO); Dipartimento Di Oncologia Medica

location_facility: Hospital Universitario Virgen del Rocio; Servicio de Oncologia

location_facility: Osaka International Cancer Institute; Breast and Endocrine Surgery

location_facility: Magyar Honvedseg Egeszsegugyi Kozpont; Fázis I-es Klinikai Farmakológiai Vizsgálóhely

location_facility: Seoul National Uni Hospital; Dept. of Internal Medicine/Hematology/Oncology

location_facility: Szegedi Tudomanyegyetem, AOK, Szent-Gyorgyi Albert Klinikai Kozpont, Onkoterapias Klinika

location_facility: Chiba Cancer Center; Breast Surgical Oncology

location_facility: St. Marianna University School of Medicine Hospital, Breast and Endocrine Surgery

location_facility: Uniklinik Köln, Klinik und Poliklinik und Geburtshilfe; Brustzentrum Köln

location_facility: National Hospital Organization Kyushu Cancer Center;Breast Oncology

location_facility: Hospital Clínic i Provincial; Servicio de Hematología y Oncología

location_facility: Hospital Clinico Universitario Virgen de la Victoria; Servicio de Oncologia

location_facility: Vanderbilt Breast Center at One Hundred Oaks

location_facility: Medizinische Universität Wien; Univ.Klinik für Frauenheilkunde - Klinik für Gynäkologie

location_facility: Ospedale Antonio Perrino; Oncologia Medica

location_facility: HOP Prive Arras Les Bonnettes; Chimiotherapie

location_facility: Abramson Cancer Center; Univ of Pennsylvania

location_facility: Hospital Clinico Universitario de Salamanca; Servicio de Oncologia

location_facility: Peter Maccallum Cancer Institute; Medical Oncology

location_facility: The Center for Cancer and Blood Disorders - Fort Worth

location_facility: Azienda Ospedaliero-Universitaria S.Orsola-Malpighi; Unità Operativa Oncologia Medica

location_facility: Instituto Nacional de Enfermedades Neoplasicas

location_facility: Lkh Salzburg - Univ. Klinikum Salzburg; Iii. Medizinische Abt.

location_facility: Tokyo Medical Uni. Hospital; Breast Oncology

location_facility: Centro Oncológico Sixtino / Centro Oncológico SA

location_facility: Hospital de Donostia; Servicio de Oncologia Medica

location_facility: Texas Oncology-El Paso Cancer Treatment Center Grandview

location_facility: Geelong Hospital; Andrew Love Cancer Centre

location_facility: Fuzhou General Hospital, PLA Nanjing Military Area Command

location_facility: Hiroshima City Hiroshima Citizens Hospital; Breast Surgery

location_facility: Békés Megyei Pándy Kálmán Kórház; Onkologiai tanszek

location_facility: Academish Ziekenhuis Maastricht (Azm); Inwendige Geneeskunde

location_facility: Churchill Hospital; Oxford Cancer and Haematology Centre

location_facility: Sourasky / Ichilov Hospital; Oncology Department

location_facility: Universitätsklinikum "Carl Gustav Carus"; Frauenheilkunde und Geburtshilfe

location_facility: Eastern Ct Hema/Onco Assoc; Dept of Oncology

location_facility: IRCCS Istituto Nazionale Tumori Fondazione Pascale; Oncologia Medica A

location_facility: ASST DEGLI SPEDALI CIVILI DI BRESCIA; Oncologia Medica

location_facility: Ordensklinikum Linz Barmherzige Schwestern; Abt. fur Innere Medizin 1

location_facility: Hospital General Universitario Gregorio Marañon; Servicio de Oncologia

location_facility: Az. Osp. Uni Ria San Martino; Cliniche Uni Rie Convenzionate U.O. Oncologia Medical

location_facility: Dana Farber Cancer Institute at Faulkner Hospital

location_facility: Prof. Dr. I. Chiricuta Institute of Oncology

location_facility: Hospital Universitario de Canarias;servicio de Oncologia

location_facility: Chiang Rai Prachanukraw Hospital; Department of Pediatrics, Faculty of Medicine

location_facility: Ente Ospedaliero Ospedali Galliera; S.C. Oncologia Medica

location_facility: Norrlands Universitetssjukhus, Umeå, Cancercentrum; Dept of Oncology

location_facility: St Vincent'S Uni Hospital; Medical Oncology

location_facility: VETERANS GENERAL HOSPITAL; Department of General Surgery

location_facility: Hospital Juan Ramon Jimenez;Servicio de Oncologia

location_facility: Hospital Clinico Universitario de Valencia; Servicio de Onco-hematologia

location_facility: Centre Rene Huguenin; CONSULT SPECIALISEES

location_facility: Universitätsklinikum Bonn; Zentrum für Geburtshilfe und Frauenheilkunde

location_facility: National Hospital Organization Osaka National Hospital; Breast Surgery

location_facility: Kaiser Permanente; Oncology Clinical Trials

location_facility: Kumamoto Shinto General Hospital; Breast Cancer Center

location_facility: Lopburi Cancer Hospital; Chemotherpy Unit; Chemotherapy

location_facility: Klinikum Mutterhaus der Borromaeerinnen gGmbH; Haematologie/Onkologie

location_facility: Clinique Armoricaine Radiologie; Hopital de Jour

location_facility: HonorHealth Research Institute - Bisgrove

location_facility: Clinica Anglo Americana - Centro de Investigacion Oncologia CAA

location_facility: Hospital Universitario 12 de Octubre; Servicio de Oncologia

location_facility: Praxis für Interdisziplinäre Onkologie und Hämatologie GbR

location_facility: Peterborough City Hospital, Edith Cavell Campus; Oncology Department

location_facility: Uniwersyteckie Centrum Kliniczne, Klinika Onkologii i Radioterapii

location_facility: State Budget Institution of Healthcare of Stavropol region Pyatigorsk Oncology Dispensary

location_facility: IRCCS Istituto Nazionale Per La Ricerca Sul Cancro (IST); Oncologia Medica A

location_facility: Queen Elizabeth II Health Sciences Centre; Oncology

location_facility: Debreceni Egyetem, Klinikai Kozpont, Onkologiai Klinika

location_facility: Centre Oscar Lambret; Cancerologie Gynecologique

location_facility: Kaiser Permanente Sacramento Medical Center

location_facility: Bcca - Vancouver Island Cancer Centre; Oncology

location_facility: Fakultni Nemocnice Hradec Kralove; Dept of Radiotherapy & Oncology

location_facility: US oncology research at Minnesota Oncology

location_facility: Emergency University Bucharest Hospital; Oncology Department

location_facility: Polyclinique Francheville; MED CHIMIOTHERAPIE RADIOTHERAPIE

location_facility: Azienda Ospedaliera Di Perugia Ospedale s. Maria Della Misericordia; Oncologia Medica

location_facility: Sana Klinikum Offenbach GmbH; Klinik für Gynäkologie & Geburtshilfe

location_facility: Onkologische Schwerpunktpraxis Bielefeld; Haemotologie & Internistische onkologie

location_facility: Texas Oncology - Dallas Presbyterian Hospital

location_facility: Hospital Universitario Marques de Valdecilla; Servicio de Oncologia

location_facility: Gynaekologisch-Onkologische Schwerpunktpraxis Prof. Dr. med. Lueck, Dr. Schrader und Dr. Noeding

location_facility: National Taiwan Uni Hospital; General Surgery

location_facility: Hospital Universitario Clínico San Carlos; Servicio de Oncologia

location_facility: Kyoto University Hospital; Breast Surgery

location_facility: Samsung Medical Centre; Division of Hematology/Oncology

location_facility: Ivano-Frankivsk Regional Oncology Center

location_facility: Hämatologisch-Onkologische Gemeinschaftspraxis am Bethanien-Krankenhaus

location_facility: Medizinische Universität Wien; Univ.Klinik für Innere Medizin I - Abt. für Onkologie

location_facility: Niagara Health Systems - St. Catherines General Site; Niagara Health System-St. Catharines Site

location_facility: Kyiv City Clinical Oncological Center, Day Hospital Department for Oncological patients

location_facility: Centre Antoine Lacassagne; Hopital De Jour A2

location_facility: Tongji Hosp, Tongji Med. Col, Huazhong Univ. of Sci. & Tech

location_facility: Lkh-Univ. Klinikum Graz; Klinik Für Innere Medizin I

location_facility: Atlantic Health Science Corporation; Saint John Regional Hospital Facility

location_facility: Rabin MC; Davidof Center - Oncology Institute

location_facility: Centrum Onkologii;Im. Franciszka Lukaszczyka;Onkologii

location_facility: NZOZ Mazowiecki Szpital Onkologiczny Uczelni Warszawskiej im. M. Skłodowskiej-Curie

location_facility: Hospital Son Llatzer; Servicio de Oncologia

location_facility: Heartland CCOP/Missouri Baptist Medical Center

location_facility: Hospital Universitario la Fe; Servicio de Oncologia

location_facility: Royal Marsden Hosp NHS Fnd; Medicine - Breast Unit

location_facility: University Hospital coventry; Oncology Department

location_facility: Complejo Hospitalario Universitario A Coruña (CHUAC, Materno Infantil), Oncología

location_facility: Washington Cancer Institute at MedStar Washington Hospital Center.

location_facility: Ch Pitie Salpetriere; Cmrr Ile De France Salpetriere

location_facility: Florida Cancer Specialists; Saint Petersburg

location_facility: The First Affiliated Hospital of College of Medicine, Zhejiang University

title: A Randomized Multicenter, Double-Blind, Placebo-Controlled Comparison of Chemotherapy Plus Trastuzumab Plus Placebo Versus Chemotherapy Plus Trastuzumab Plus Pertuzumab as Adjuvant Therapy in Patients With Operable HER2-Positive Primary Breast Cancer

summary: This randomized, double-blind, placebo-controlled, two-arm study will assess the safety and efficacy of pertuzumab in addition to chemotherapy plus trastuzumab as adjuvant therapy in participants with operable HER2-positive primary breast cancer. This study will be carried out in collaboration with the Breast International Group (BIG).


title: Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Two Year Study to Evaluate the Effect of Subcutaneous RO4909832 on Cognition and Function in Prodromal Alzheimer's Disease With Option for up to an Additional Two Years of Treatment and an Open-Label Extension With Active Study Treatment

summary:
This multi-center, randomized, double-blind, placebo-controlled parallel-group study will evaluate the effect of gantenerumab (RO4909832) on cognition and functioning and the safety and pharmacokinetics in participants with prodromal Alzheimer's Disease. Participants will be randomized to receive subcutaneous (SC) injections of either gantenerumab or placebo. Participants who consent to be part of the sub study will undergo positron emission tomography (PET) scanning to assess brain amyloid. The anticipated time on study treatment is 104 weeks in Part 1, with an option for an additional up to 2 years of treatment in Part 2, followed by an open-label extension (Part 3) until July 2020.

The dosing for Parts 1 and 2 was stopped after a planned futility interim analysis showed a low probability of meeting the primary outcome measure with the doses studied. The study has converted to open-label to investigate higher gantenerumab doses.

ReferenceCitation: Klein G, Delmar P, Voyle N, Rehal S, Hofmann C, Abi-Saab D, Andjelkovic M, Ristic S, Wang G, Bateman R, Kerchner GA, Baudler M, Fontoura P, Doody R. Gantenerumab reduces amyloid-β plaques in patients with prodromal to moderate Alzheimer's disease: a PET substudy interim analysis. Alzheimers Res Ther. 2019 Dec 12;11(1):101. doi: 10.1186/s13195-019-0559-z.

family: Klein

full_name: Gregory Klein

given: Gregory

family: Delmar

full_name: Paul Delmar

given: Paul

family: Voyle

full_name: Nicola Voyle

given: Nicola

family: Rehal

full_name: Sunita Rehal

given: Sunita

family: Hofmann

full_name: Carsten Hofmann

given: Carsten

family: Abi-Saab

full_name: Danielle Abi-Saab

given: Danielle

family: Andjelkovic

full_name: Mirjana Andjelkovic

given: Mirjana

family: Ristic

full_name: Smiljana Ristic

given: Smiljana

family: Wang

full_name: Guoqiao Wang

given: Guoqiao

family: Bateman

full_name: Randall Bateman

given: Randall

family: Kerchner

full_name: Geoffrey A. Kerchner

given: Geoffrey A.

family: Baudler

full_name: Monika Baudler

given: Monika

family: Fontoura

full_name: Paulo Fontoura

given: Paulo

family: Doody

full_name: Rachelle Doody

given: Rachelle

currency_apc_doaj: GBP

journal_name: Alzheimer's Research & Therapy

oa_colors: green gold

evidence: oa journal (via doaj)

host_type: publisher

license: cc-by

license_normalized: cc-by

version: publishedVersion

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

evidence: oa repository (via pmcid lookup)

license_normalized: no license

title: Gantenerumab reduces amyloid-β plaques in patients with prodromal to moderate Alzheimer’s disease: a PET substudy interim analysis

ReferenceCitation: Ostrowitzki S, Lasser RA, Dorflinger E, Scheltens P, Barkhof F, Nikolcheva T, Ashford E, Retout S, Hofmann C, Delmar P, Klein G, Andjelkovic M, Dubois B, Boada M, Blennow K, Santarelli L, Fontoura P; SCarlet RoAD Investigators. A phase III randomized trial of gantenerumab in prodromal Alzheimer's disease. Alzheimers Res Ther. 2017 Dec 8;9(1):95. doi: 10.1186/s13195-017-0318-y. Erratum in: Alzheimers Res Ther. 2018 Sep 27;10(1):99.

family: Ostrowitzki

full_name: Susanne Ostrowitzki

given: Susanne

repository_institution: University College London - UCL Discovery

repositories_institution: University College London - UCL Discovery

title: A phase III randomized trial of gantenerumab in prodromal Alzheimer’s disease

ReferenceCitation: Delrieu J, Ousset PJ, Vellas B. Gantenerumab for the treatment of Alzheimer's disease. Expert Opin Biol Ther. 2012 Aug;12(8):1077-86. doi: 10.1517/14712598.2012.688022. Epub 2012 May 15. Review.

location_country:
Finland
Belgium
Australia
Russian Federation
Denmark
France
Poland
Argentina
Canada
Korea, Republic of
Chile
Sweden
Switzerland
Netherlands
Portugal
Germany
United States
United Kingdom
Italy
Brazil
Mexico
Czechia
Spain
Turkey

location_facility: PANAKEIA - Arzneimittelforschung Leipzig GmbH

location_facility: Hospital das Clinicas - UFPR; Ciencias da Saude

location_facility: Klinikum Joh.Wolfg.Goethe-UNI Zentrum d. Psychiatrie Klinik f. Psychiatrie Psychosomatik

location_facility: Istanbul University Istanbul School of Medicine; Neurology

location_facility: Hospital de Santa Maria; Servico de Neurologia

location_facility: ALPI-Inst. de Rehabilitacion Marcelo Fitte

location_facility: PALLMED Sp. z o.o. prowadząca NZOZ DOM SUE RYDER

location_facility: McGill University; Sir Mortimer B Davis Jewish General Hospital; Neurological and Psychiatric

location_facility: Universidade Federal de Sao Paulo - UNIFESPX; Neurologia

location_facility: Klinikum rechts der Isar der TU München; Klinik für Psychiatrie und Psychotherapie

location_facility: Saint Petersburg State Institution of Healthcare City Geriatric Medico-Social Center

location_facility: Zentralinstitut für Seelische Gesundheit Abt.Gerontopsychiatrie

location_facility: St. Anne´s University Hospital; Clinical Trials Department

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

location_facility: Irccs Multimedica Santa Maria; Unita' Di Neurologia

location_facility: Universitätsklinikum Bonn; Medizinische Klinik und Poliklinik I; Allgemeine Innere Medizin

location_facility: Campus for Ageing & Vitality; Clincal Ageing Research Unit

location_facility: Western Michigan University Homer Stryker M.D. School of Medicine Center for Clinical Research

location_facility: Llandough Hospital; Llandough Hospital Memory Team 3rd Floor Academic Building

location_facility: Victoria Centre; Kingshill Research Centre

location_facility: Ondokuz Mayis University School of Medicine; Neurology

location_facility: Hospital das Clinicas - FMUSP; Psiquiatria

location_facility: City Clinical Hospital # 2 n.a. V.I. Razumovsky

location_facility: Estimulacion Magnetica Trnscraneal de Mexico SC.

location_facility: Hospital de Cruces; Servicio de Neurologia

location_facility: Prince of Wales Hospital, Academic Department for Old Age Psychiatry

location_facility: Australian Alzheimer's Research Foundation

location_facility: Sverdlovsk Regional Clinical Psychoneurological War Veteran Hospital

location_facility: Hospital del Mar; Servicio de Neurologia

location_facility: Akdeniz University School of Medicine, Neurology Department

location_facility: CHU De Caen; Service De Neurologie Dejerine

location_facility: Heidelberg Repatriation Hospital; Medical and Cognitive Research Centre

location_facility: Clinical Neuroscience Research Associates, Inc.

location_facility: Fondazione San Raffaele Del Monte Tabor; Dipartimento Di Neurologia

location_facility: Instituto De Neurología Cognitiva - INECO

location_facility: Hopital Pierre Wertheimer; Laboratoire De Neuro Psychologie

location_facility: Hospital Prof. Dr. Fernando Fonseca; Servico de Neurologia

location_facility: HUG; Département de santé mentale et de psychiatrie Unité de psychiatrie gériatrique

location_facility: Seoul National University Bundang Hospital; Neurology Department

location_facility: Universita' Di Parma Istituto Neurologia

location_facility: IRCCS "Centro S. Giovanni di Dio" Fatebenefratelli -UO Alzheimer

location_facility: Hospital Ramon y Cajal; Servicio de Neurologia

location_facility: CHU de Rouen Hopital; Service de Neurologie

location_facility: Charing Cross Hospital; Dept of Neurosciences

location_facility: Hospital Universitario La Paz; Servicio de Neurologia

location_facility: Universitätsklinikum Rostock Zentrum für Nervenheilkunde

location_facility: Kawartha Centre - Redefining Healthy Aging

location_facility: Azienda Ospedaliera Spedali Civili; Scienze Neurologiche

location_facility: Hospital Clinic i Provincial; Servicio de Neurologia

location_facility: Uni Di Firenze Dip. Scienze Neurol Psic Sod Neurologia 1

location_facility: University of Rochester Medical Center; Monroe Community Hospital

location_facility: Hospital Mutua De Terrasa; Servicio de Neurologia

location_facility: Hornsby Ku-ring-gai Hospital; Division of Rehabilitation & Aged Care

location_facility: Hospital Angeles de Culiacán, Neurociencias Estudios Clínicos SC

location_facility: Hopital Hautepierre; Centre dInvestigation Clinique

location_facility: Russian Medical Military Academy n.a. S.M.Kirov; Neurology Department

location_facility: Universitätsklinikum Ulm; Klinik für Neurologie

location_facility: Royal Adelaide Hospital; Memory Trials Centre

location_facility: Univ Berlin; Klin fur Psychi & Psycho Charite

location_facility: Jeroen Bosch Ziekenhuis; Polikliniek Geriatrie

location_facility: Rhode Island Mood & Memory Research Institute

location_facility: Aarhus Universitetshospital, Neurologisk Afdeling F, Demensklinikken

location_facility: Unidad de Investigacion en Enfermedades Cronico-Degenerativa; Reumatologia

location_facility: State autonomous institution of healthcare Inter-regional clinical and diagnostic center

location_facility: Hospital Universitario 12 de Octubre; Servicio de Neurologia

location_facility: Oregon Health and Science University, Layton Aging and Alzheimer's Disease Center

location_facility: Hospital Universitario Dr. Peset; Servicio de Neurologia

location_facility: IME - Instituto Médico Especializado; Ensayos Clínicos

location_facility: Hospital de la Santa Creu i Sant Pau; Servicio de Neurologia

location_facility: Hop Guillaume Et Rene Laennec; Cmrr St Herblain

location_facility: Moorgreen Hospital; Memory Assessment & Rsch Ctr

location_facility: Universitäres Zentrum für Altersmedizin und Rehabilitation

location_facility: Office of Dr Klaus Steinwachs Neurology & Psychiatry

location_facility: Turku Hospital; PET (Positron Emission Tomography) Center

location_facility: Nuovo Ospedale Civile S. Agostino-Estense; Clinica Neurologica - Dipartimento di Neuroscienze

location_facility: Skånes Universitetssjukhus Malmö, Minneskliniken


title: Precision Event Monitoring for Patients With Heart Failure Using HeartLogic: PREEMPT-HF

acronym: PREEMPT-HF

summary:
The goal of the PREEMPT-HF study is to collect device and clinical event data to evaluate extended applications of the HeartLogic Heart Failure Diagnostic (HeartLogic) in a broad spectrum of heart failure patients with an implantable cardioverter defibrillator or cardiac resynchronization therapy defibrillator. There are no primary safety and/or efficacy endpoints for this study.

Heart failure is a complex clinical syndrome with high morbidity, mortality, and economic burden. Chronic Heart Failure is persistent, gradually progressive, and punctuated by episodes of acute worsening leading to hospitalizations. Therefore, there remains an unmet clinical need to slow the progression of Heart Failure and prevent hospitalizations. HeartLogic, available in Boston Scientific cardiac resynchronization therapy devices and defibrillators, combines novel sensor parameters such as heart sounds and respiration with other measurements like thoracic impedance, heart rate, and activity into a HeartLogic Index for the early detection of worsening Heart Failure. However, there is limited data on the association of HeartLogic with the risk of Hear Failure readmissions and tachyarrhythmias, or for phenotyping the broad spectrum of Heart Failure patients.

status: Active, not recruiting

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Benjamin EJ, Blaha MJ, Chiuve SE, Cushman M, Das SR, Deo R, de Ferranti SD, Floyd J, Fornage M, Gillespie C, Isasi CR, Jiménez MC, Jordan LC, Judd SE, Lackland D, Lichtman JH, Lisabeth L, Liu S, Longenecker CT, Mackey RH, Matsushita K, Mozaffarian D, Mussolino ME, Nasir K, Neumar RW, Palaniappan L, Pandey DK, Thiagarajan RR, Reeves MJ, Ritchey M, Rodriguez CJ, Roth GA, Rosamond WD, Sasson C, Towfighi A, Tsao CW, Turner MB, Virani SS, Voeks JH, Willey JZ, Wilkins JT, Wu JH, Alger HM, Wong SS, Muntner P; American Heart Association Statistics Committee and Stroke Statistics Subcommittee. Heart Disease and Stroke Statistics-2017 Update: A Report From the American Heart Association. Circulation. 2017 Mar 7;135(10):e146-e603. doi: 10.1161/CIR.0000000000000485. Epub 2017 Jan 25. Review. Erratum in: Circulation. 2017 Mar 7;135(10 ):e646. Circulation. 2017 Sep 5;136(10 ):e196.

ReferenceCitation: Ambrosy AP, Fonarow GC, Butler J, Chioncel O, Greene SJ, Vaduganathan M, Nodari S, Lam CSP, Sato N, Shah AN, Gheorghiade M. The global health and economic burden of hospitalizations for heart failure: lessons learned from hospitalized heart failure registries. J Am Coll Cardiol. 2014 Apr 1;63(12):1123-1133. doi: 10.1016/j.jacc.2013.11.053. Epub 2014 Feb 5.

ReferenceCitation: Gheorghiade M, Pang PS. Acute heart failure syndromes. J Am Coll Cardiol. 2009 Feb 17;53(7):557-573. doi: 10.1016/j.jacc.2008.10.041. Review.

ReferenceCitation: Boehmer JP, Hariharan R, Devecchi FG, Smith AL, Molon G, Capucci A, An Q, Averina V, Stolen CM, Thakur PH, Thompson JA, Wariar R, Zhang Y, Singh JP. A Multisensor Algorithm Predicts Heart Failure Events in Patients With Implanted Devices: Results From the MultiSENSE Study. JACC Heart Fail. 2017 Mar;5(3):216-225. doi: 10.1016/j.jchf.2016.12.011.

ReferenceCitation: Whellan DJ, Lindenfeld J. Easy to Predict, Difficult to Prevent. JACC Heart Fail. 2017 Mar;5(3):226-228. doi: 10.1016/j.jchf.2017.01.006.

ReferenceCitation: Desai AS. The three-phase terrain of heart failure readmissions. Circ Heart Fail. 2012 Jul 1;5(4):398-400. doi: 10.1161/CIRCHEARTFAILURE.112.968735.

name: From the Advanced Heart Disease Section, Cardiovascular Division, Brigham and Women’s Hospital, Boston, MA.

family: Desai

full_name: Akshay S. Desai

given: Akshay S.

journal_name: Circulation: Heart Failure

oa_host_type: publisher

evidence: open (via free pdf)

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

title: The Three-Phase Terrain of Heart Failure Readmissions

ReferenceCitation: Dharmarajan K, Wang Y, Lin Z, Normand ST, Ross JS, Horwitz LI, Desai NR, Suter LG, Drye EE, Bernheim SM, Krumholz HM. Association of Changing Hospital Readmission Rates With Mortality Rates After Hospital Discharge. JAMA. 2017 Jul 18;318(3):270-278. doi: 10.1001/jama.2017.8444.

ReferenceCitation: Shah SJ, Katz DH, Selvaraj S, Burke MA, Yancy CW, Gheorghiade M, Bonow RO, Huang CC, Deo RC. Phenomapping for novel classification of heart failure with preserved ejection fraction. Circulation. 2015 Jan 20;131(3):269-79. doi: 10.1161/CIRCULATIONAHA.114.010637. Epub 2014 Nov 14.

ipd_sharing: Undecided

lead_sponsor: Boston Scientific Corporation

location_country:
Italy
United States
Hong Kong
Netherlands
Germany
France
Ireland
Belgium
Australia
Spain
Denmark
Canada
United Kingdom

location_facility: University of Iowa Hospitals and Clinics

location_facility: Baptist Health Medical Center (Little Rock)

location_facility: Hospital Universitario Virgen De La Victoria

location_facility: The University of Texas, Southwestern Medical Center

location_facility: Augusta Kranken Anstalt GmbH - Klinik für Kardiologie und Angiologie

location_facility: Strong Memorial Hospital of the University of Rochester

location_facility: Cardiology Associates of Northeast Arkansas

location_facility: South Manchester University Hospital Trust

location_facility: Christus Trinity Mother Frances Health System

location_facility: St. Francis Health System - St. Francis Hospital

location_facility: Providence Regional Medical Center Everett

intervention_type: Device


title: A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

summary: To evaluate the efficacy and safety of eplontersen after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398). For more information, please visit http://www.neuro-ttransform.com/.

ReferenceCitation: Coelho T, Ando Y, Benson MD, Berk JL, Waddington-Cruz M, Dyck PJ, Gillmore JD, Khella SL, Litchy WJ, Obici L, Monteiro C, Tai LJ, Viney NJ, Buchele G, Brambatti M, Jung SW, St L O'Dea L, Tsimikas S, Schneider E, Geary RS, Monia BP, Gertz M. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L(Rx) (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Neurol Ther. 2021 Jun;10(1):375-389. doi: 10.1007/s40120-021-00235-6. Epub 2021 Feb 26.

family: Coelho

full_name: Teresa Coelho

given: Teresa

family: Ando

full_name: Yukio Ando

given: Yukio

family: Benson

full_name: Merrill D. Benson

given: Merrill D.

family: Berk

full_name: John L. Berk

given: John L.

family: Waddington-Cruz

full_name: Márcia Waddington-Cruz

given: Márcia

family: Dyck

full_name: Peter J. Dyck

given: Peter J.

family: Gillmore

full_name: Julian D. Gillmore

given: Julian D.

family: Khella

full_name: Sami L. Khella

given: Sami L.

family: Litchy

full_name: William J. Litchy

given: William J.

family: Obici

full_name: Laura Obici

given: Laura

family: Monteiro

full_name: Cecilia Monteiro

given: Cecilia

family: Tai

full_name: Li-Jung Tai

given: Li-Jung

family: Viney

full_name: Nicholas J. Viney

given: Nicholas J.

family: Buchele

full_name: Gustavo Buchele

given: Gustavo

family: Brambatti

full_name: Michela Brambatti

given: Michela

family: Jung

full_name: Shiangtung W. Jung

given: Shiangtung W.

family: St. L. O’Dea

full_name: Louis St. L. O’Dea

given: Louis

family: Tsimikas

full_name: Sotirios Tsimikas

given: Sotirios

family: Schneider

full_name: Eugene Schneider

given: Eugene

family: Geary

full_name: Richard S. Geary

given: Richard S.

family: Monia

full_name: Brett P. Monia

given: Brett P.

family: Gertz

full_name: Morie Gertz

given: Morie

title: Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

location_country:
Italy
Brazil
United States
Sweden
Spain
Germany
Cyprus
France
Greece
Argentina
Australia
New Zealand
Portugal
Turkey
Canada
Taiwan

location_facility: Hospital Universitário Clementino Fraga Filho

location_facility: Chang Gung Memorial Hospital - Linkou Branch

location_facility: Centre Hospitalier Universitaire de Toulouse

location_facility: Fondazione IRCCS Istituto Neurologico Carlo Besta

location_facility: İstanbul Üniversitesi - Istanbul Tıp Fakültesi

location_facility: Johns Hopkins University Neurology Research Office

location_facility: Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio

location_facility: Fondazione Policlinico Universitario Agostino Gemelli

location_facility: University General Hospital of Heraklion (PAGNI)

location_facility: Azienda Ospedaliera Universitaria Policlinico Gaetano Martino

location_facility: Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

location_facility: Indiana University School of Medicine - Indianapolis

location_facility: Perron Institute for Neurological and Translational Science

location_facility: Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria

location_facility: University of North Carolina Hospitals - Neurology Clinic

location_facility: Associação de Assistência à Criança Deficiente - Unidade Lar Escola

location_facility: The Cyprus Institute of Neurology and Genetics


ipd_sharing_description: AbbVie is committed to responsible data sharing regarding the clinical trials we sponsor. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information (e.g., protocols and clinical study reports), as long as the trials are not part of an ongoing or planned regulatory submission. This includes requests for clinical trial data for unlicensed products and indications.

lead_sponsor: AbbVie

location_country:
Italy
United States
Netherlands
France
Korea, Republic of
Hungary
Spain
Germany
United Kingdom
Canada

location_facility: Hospital Santa Creu i Sant Pau /ID# 213259

location_facility: Kyungpook National Univ Hosp /ID# 209912

location_facility: Queen Elizabeth University Hos /ID# 206574

location_facility: Chu de Nice-Hopital L'Archet Ii /Id# 208131

location_facility: Universitaetsklinikum Schleswig-Holstein /ID# 207571

location_facility: Hospital Universitario La Paz /ID# 210065

location_facility: Banner University Medical Cent /ID# 208392

location_facility: Complesso integrato Columbus /ID# 204549

location_facility: Debreceni Egyetem Klinikai Kozpont /ID# 221952

location_facility: Meridian Investigator Network /ID# 204646

location_facility: Meridian Investigator Network /ID# 218568

location_facility: Charite Universitaetsmedizin Berlin /ID# 207570

location_facility: Universita di Catanzaro Magna Graecia /ID# 204546

location_facility: Elisabeth Tweesteden Ziekenhuis /ID# 206272

location_facility: Maastricht Universitair Medisch Centrum /ID# 204428

location_facility: Clinical Associates in Researc /ID# 204689

location_facility: CHU NANCY - Hopital Brabois Adultes /ID# 208133

location_facility: Hospital General Universitario Gregorio Maranon /ID# 204504

location_facility: Penn Presbyterian Medical Center /ID# 206826

location_facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico /ID# 208504

location_facility: Orange County Institute of Gastroenterology and Endoscopy /ID# 207405

location_facility: Universitaetsklinikum Frankfurt /ID# 207569

title: A Multicenter, Single Arm, Open-label Study to Investigate the Efficacy and Safety of Ravagalimab (ABBV-323) in Subjects With Moderate to Severe Ulcerative Colitis Who Failed Prior Therapy

summary: Study M15-722 is a Phase 2a study to investigate the efficacy and safety of Ravagalimab (ABBV-323) in participants with moderate to severe UC who failed prior therapy.


title: A Phase 2, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of IPI-926 in Patients With Metastatic or Locally Advanced (Unresectable) Chondrosarcoma

summary: IPI-926 is an inhibitor of the hedgehog pathway. IPI-926 may improve therapeutic outcomes in patients with Chondrosarcoma.

lead_sponsor: Infinity Pharmaceuticals, Inc.

location_country:
Italy
Austria
United States
Sweden
Netherlands
Germany
France
Poland
Norway
Australia
Russian Federation
Canada
United Kingdom

location_facility: Fondazione IRCCS Istituto Nazionale dei Tumori

location_facility: University of Washington - Seattle Cancer Care Alliance

location_facility: University of Miami - Sylvester Comprehensive Cancer Center

location_facility: Centre Léon Bérard, Service d'Oncologie Médicale

location_facility: Centrum Onkologii Instytut im. Marii Sklodowskiej-Curie w Warszawie

location_facility: Pennsylvania Oncology Hematology Associates

location_facility: Moscow n.a. P.A Herzen Oncology Research Institute of Rosmedtechnologies

location_facility: (TGen) Translational Genomics Research Institute


ReferenceCitation: Banaschewski T, Johnson M, Lecendreux M, Zuddas A, Adeyi B, Hodgkins P, Squires LA, Coghill DR. Health-related quality of life and functional outcomes from a randomized-withdrawal study of long-term lisdexamfetamine dimesylate treatment in children and adolescents with attention-deficit/hyperactivity disorder. CNS Drugs. 2014 Dec;28(12):1191-203. doi: 10.1007/s40263-014-0193-z.

family: Banaschewski

full_name: Tobias Banaschewski

given: Tobias

family: Johnson

full_name: Mats Johnson

given: Mats

family: Lecendreux

full_name: Michel Lecendreux

given: Michel

family: Zuddas

full_name: Alessandro Zuddas

given: Alessandro

family: Adeyi

full_name: Ben Adeyi

given: Ben

family: Hodgkins

full_name: Paul Hodgkins

given: Paul

family: Squires

full_name: Liza A. Squires

given: Liza A.

family: Coghill

full_name: David R. Coghill

given: David R.

journal_name: CNS Drugs

oa_colors: green hybrid

evidence: open (via page says license)

license: cc-by-nc

license_normalized: cc-by-nc

license: implied-oa

license_normalized: implied-oa

title: Health-Related Quality of Life and Functional Outcomes from a Randomized-Withdrawal Study of Long-Term Lisdexamfetamine Dimesylate Treatment in Children and Adolescents with Attention-Deficit/Hyperactivity Disorder

ReferenceCitation: Coghill DR, Banaschewski T, Lecendreux M, Johnson M, Zuddas A, Anderson CS, Civil R, Dauphin M, Higgins N, Lyne A, Gasior M, Squires LA. Maintenance of efficacy of lisdexamfetamine dimesylate in children and adolescents with attention-deficit/hyperactivity disorder: randomized-withdrawal study design. J Am Acad Child Adolesc Psychiatry. 2014 Jun;53(6):647-657.e1. doi: 10.1016/j.jaac.2014.01.017. Epub 2014 Mar 4.

family: Anderson

full_name: Colleen S. Anderson

given: Colleen S.

family: Civil

full_name: Richard Civil

given: Richard

family: Dauphin

full_name: Matthew Dauphin

given: Matthew

family: Higgins

full_name: Nicholas Higgins

given: Nicholas

family: Lyne

full_name: Andrew Lyne

journal_name: Journal of the American Academy of Child & Adolescent Psychiatry

repository_institution: University of Dundee - Discovery Research Portal

title: Maintenance of Efficacy of Lisdexamfetamine Dimesylate in Children and Adolescents With Attention-Deficit/Hyperactivity Disorder: Randomized-Withdrawal Study Design

location_country:
Italy
United States
Sweden
France
Poland
Hungary
Belgium
Germany
United Kingdom

location_facility: Barn och Ungdomsmedicin klinik Mölnlycke

location_facility: Azienda Ospedaliera Universitaria Policlinico G. Martino

location_facility: Klinikum der Johannes Gutenberg-Universität Mainz

location_facility: East Kent NHS and Social Care Partnership Trust

location_facility: Szpital Uniwersytecki im. dr. Antoniego Jurasza w Bydgoszczy

location_facility: Schwerpunktpraxis für Entwicklung und Lernen

location_facility: Astrid Lindgren Children's Hospital, Karolinska University Hospital

location_facility: Universitair Ziekenhuis Gent, Kinder - en Jeugdpsychiatrie

location_facility: Basildon Hospital - Child Development Centre,

location_facility: Samodzielny Publiczny Dzieciecy Szpital Kliniczny, Poradnia Psychiatryczna, ul. Marszalkowska 24,

location_facility: Universität zu Köln, Klinik und Poliklinik für Psychiatrie und Psychotherapie des Kindes und Jugendalters

location_facility: ZNA Antwerpen, Commandant Weynsstraat 165

location_facility: Universitätsklinikum Gießen und Marburg GmbH, Universitätsklinikum Gießen und Marburg GmbH, Hans-Sachs-Straße 4,

location_facility: Drottning Silvias Barnsjukhus, Otterhällegatan 12A

location_facility: Zentralinstitut für Seelische Gesundheit Mannheim

location_facility: Gyermek és Ifjúságpszichiátriai Szakrendelés és Gondozó

location_facility: Azienda Ospedaliera della 2? Universita di Napoli

location_facility: Azienda Ospedaliera Policlinico Consorziale

location_facility: Praxis Dr med. Friedrich Kaiser und Dr. med. Ingrid Marinesse

location_facility: Wojewódzki Osrodek Lecznictwa Psychiatrycznego

location_facility: Northampton Child and Adolescent Mental Health Services

title: A Phase III, Double-blind, Placebo-controlled, Randomised Withdrawal, Multicentre, Extension, Safety and Efficacy Study of Lisdexamfetamine Dimesylate (LDX) in Children and Adolescents Aged 6-17 With Attention- Deficit/Hyperactivity Disorder (ADHD)

summary: The main aim of this study is to evaluate the long-term maintenance of efficacy of LDX after administered to children and adolescents aged 6-17 with ADHD for at least 6 months


title: Validation Trial for a Multi-parameter Diagnostic Blood Test for the Diagnosis of Alzheimer's Disease

acronym: RH-VAL

summary: The purpose of this study is to validate a diagnostic test that combines different blood markers to identify and correctly classify patients with Alzheimer's disease (AD) compared to individuals with behavioural variant frontotemporal dementia (bvFTD, patient control) versus cognitively healthy individuals (healthy control).

lead_sponsor: Raman Health Technologies, S.L.

location_country: Belgium France Spain

location_facility: Cognitive and Behavioral Disease Center and Alzheimer's Institute

location_facility: Centre Mémoire paris Nord Ile de France GH Saint-Louis Lariboisière Fernand Widal


ReferenceCitation: Valipour A, Jäger M, Wu P, Schmitt J, Bunch C, Weberschock T. Interventions for mycosis fungoides. Cochrane Database Syst Rev. 2020 Jul 7;7:CD008946. doi: 10.1002/14651858.CD008946.pub3.

name: Department of Dermatology, Venereology and Allergology; Johann Wolfgang Goethe-University Hospital; Frankfurt am Main Germany

name: Evidence-Based Medicine Frankfurt, Institute of General Practice; Goethe University; Frankfurt Germany

family: Valipour

full_name: Arash Valipour

given: Arash

name: Hautklinik; Städtisches Klinikum Karlsruhe; Karlsruhe Germany

family: Jäger

full_name: Manuel Jäger

given: Manuel

name: Department of Dermatology; University of California Davis; Sacramento CA USA

family: Wu

full_name: Peggy Wu

given: Peggy

name: Center for Evidence-Based Healthcare; Faculty of Medicine Carl Gustav Carus, Technischen Universität (TU) Dresden; Dresden Germany

family: Schmitt

full_name: Jochen Schmitt

given: Jochen

name: c/o Cochrane Skin Group; The University of Nottingham; Nottingham UK

family: Bunch

full_name: Charles Bunch

given: Charles

family: Weberschock

full_name: Tobias Weberschock

journal_name: Cochrane Database of Systematic Reviews

oa_host_type: repository

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

title: Interventions for mycosis fungoides

ipd_sharing_description: No intent to share information.

lead_sponsor: Galderma R&D

location_country:
Germany
United States
France

acronym: CTCL

title: A Randomized, Double-blind, Multi-centre, Placebo-controlled, Parallel-arm Phase 2 Trial to Assess Safety, Efficacy and Pharmacokinetics of CD11301 0.03% and 0.06% Gel in the Treatment of Cutaneous T-Cell Lymphoma (CTCL), Stages IA, IB and IIA

summary: To assess the efficacy, safety and pharmacokinetics in participants treated with CD11301 gel vs. placebo for early stage CTCL (IA, IB, or IIA).


title: An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

summary: To collect and evaluate long-term safety and effectiveness data in HPP patients who have/ are receiving treatment with asfotase alfa.

status: Enrolling by invitation

ReferenceCitation: Seefried L, Dahir K, Petryk A, Högler W, Linglart A, Martos-Moreno GÁ, Ozono K, Fang S, Rockman-Greenberg C, Kishnani PS. Burden of Illness in Adults With Hypophosphatasia: Data From the Global Hypophosphatasia Patient Registry. J Bone Miner Res. 2020 Nov;35(11):2171-2178. doi: 10.1002/jbmr.4130. Epub 2020 Aug 10.

name: Orthopedic Department University of Würzburg  Würzburg Germany

family: Seefried

full_name: Lothar Seefried

given: Lothar

name: Vanderbilt University Medical Center  Nashville TN USA

family: Dahir

full_name: Kathryn Dahir

given: Kathryn

name: Alexion Pharmaceuticals, Inc.  Boston MA USA

family: Petryk

full_name: Anna Petryk

given: Anna

name: Department of Pediatrics and Adolescent Medicine Johannes Kepler University Linz  Linz Austria

family: Högler

full_name: Wolfgang Högler

given: Wolfgang

name: AP‐HP Paris‐Saclay, Endocrinology and Diabetes for Children Reference Center for Rare Disorders of Calcium and Phosphate Metabolism, Filière OSCAR  Paris France

family: Linglart

full_name: Agnès Linglart

given: Agnès

name: Department of Endocrinology Hospital Infantil Universitario Niño Jesús, IIS La Princesa  Madrid Spain

name: Department of Pediatrics Universidad Autónoma de Madrid  Madrid Spain

family: Martos‐Moreno

full_name: Gabriel Ángel Martos‐Moreno

given: Gabriel Ángel

name: Department of Pediatrics, Graduate School of Medicine Osaka University  Osaka Japan

family: Ozono

full_name: Keiichi Ozono

given: Keiichi

family: Fang

full_name: Shona Fang

given: Shona

name: Department of Pediatrics and Child Health University of Manitoba  Winnipeg Canada

family: Rockman‐Greenberg

full_name: Cheryl Rockman‐Greenberg

given: Cheryl

name: Division of Medical Genetics, Department of Pediatrics Duke University Medical Center  Durham NC USA

family: Kishnani

full_name: Priya S Kishnani

given: Priya S

journal_name: Journal of Bone and Mineral Research

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

repository_institution: Würzburg University - Online Publication Service of Würzburg University

ReferenceCitation: Högler W, Langman C, Gomes da Silva H, Fang S, Linglart A, Ozono K, Petryk A, Rockman-Greenberg C, Seefried L, Kishnani PS. Diagnostic delay is common among patients with hypophosphatasia: initial findings from a longitudinal, prospective, global registry. BMC Musculoskelet Disord. 2019 Feb 14;20(1):80. doi: 10.1186/s12891-019-2420-8.

family: Langman

full_name: Craig Langman

given: Craig

family: Gomes da Silva

full_name: Hugo Gomes da Silva

given: Hugo

family: Rockman-Greenberg

full_name: Cheryl Rockman-Greenberg

repositories_institution:
PubMed Central - Europe PMC
University of Manitoba - Mspace

location_country:
Italy
United States
Saudi Arabia
Spain
Germany
France
Ireland
Australia
Russian Federation
Portugal
Canada
United Kingdom


lead_sponsor: UCB Pharma

location_country:
Mexico
Poland
France
Hungary
United States

title: A MULTI-CENTER, OPEN-LABEL, EXPLORATORY STUDY TO INVESTIGATE THE SAFETY AND EFFICACY OF LACOSAMIDE AS ADJUNCTIVE THERAPY IN SUBJECTS ≥1 MONTH TO <18 YEARS WITH EPILEPSY SYNDROMES ASSOCIATED WITH GENERALIZED SEIZURES.

summary: SP0966 is an exploratory study to investigate safety and efficacy of Lacosamide (LCM) in children with epilepsy syndromes associated with generalized seizures. LCM will be added to current antiepileptic treatment.


location_country:
Italy
Austria
Netherlands
Germany
Finland
France
Australia
United States
United Kingdom

location_facility: Medizinische Universitaet Wien /ID# 208497

location_facility: The Royal Marsden NHS Foundation Trust /ID# 211263

location_facility: Oxford University Hospitals NHS Foundation Trust /ID# 211264

location_facility: Helsinki University Hospital /ID# 208108

location_facility: Dana-Farber Cancer Institute /ID# 207728

location_facility: Universitair Medisch Centrum Groningen /ID# 207990

location_facility: Azienda Sanitaria Universitaria Giuliano Isontina /ID# 211487

location_facility: University of Texas MD Anderson Cancer Center /ID# 207746

location_facility: CHRU Lille - Hopital Claude Huriez /ID# 208726

title: A Prospective, Open-Label, Single-Arm, Phase 2, Multicenter Study Evaluating the Efficacy of Venetoclax Plus Ibrutinib in Subjects With T-Cell Prolymphocytic Leukemia

summary:
A study to evaluate the safety and efficacy of venetoclax plus ibrutinib for participants with T-cell Prolymphocytic Leukemia (T-PLL) and follows a 2-stage design as follows:

Stage 1: Enroll 14 participants with relapsed or refractory (R/R) T-PLL and move to Stage 2 if 4 or more participants meet protocol-specified response criteria. Response assessment will be performed on a continued basis until all 14 participants have enrolled into Stage 1 and have completed the Week 24 disease assessment.

Stage 2: Enroll up to an additional 23 participants.