title: ACR/EULAR Endorsed Study to Develop New Diagnostic and Classification Criteria for Primary Systemic Vasculitis

acronym: DCVAS

summary:
Vasculitis is group of diseases where inflammation of blood vessels is the common feature. Patients typically present with fever, fatigue, weakness and muscle and joint aches. These symptoms are very common among many different diseases, not just vasculitis. A clustering of other symptoms, physical examination findings, blood tests, radiology and biopsy help make the diagnosis. There are currently no criteria to help doctors make a diagnosis of vasculitis when a patient presents with these non specific symptoms and they are reliant on previous experience and disease definitions. One of the aims of this project is to develop diagnostic criteria for the primary systemic vasculitides (granulomatosis with polyangiitis (Wegener's), microscopic polyangiitis, Churg Strauss syndrome, polyarteritis nodosa, giant cell arteritis, Takayasu arteritis). We, the investigators, will do this by studying a large group of patients with vasculitis and comparing them to a large group of patients that present in a similar way, but do not have vasculitis. By comparing the 2 groups we will create a list of items to differentiate between vasculitis and 'vasculitis mimics'.

We also aim to update the current classification criteria. Classification criteria are used to group patients into different types of vasculitis, once a diagnosis of vasculitis has been made, and are useful for studying patients in clinical trials with similar or identical diseases. The current classification criteria (American college of Rheumatology 1990 criteria) were developed 20 years ago, before the availability of some important diagnostic tests (e.g. antineutrophil cytoplasmic antibodies [ANCA]), and are now not consistent with some of the current disease definitions. Therefore to progress future research in vasculitis, it is important that the classification criteria are updated. We will recruit 260 patients with each of the 6 types of vasculitis and compare them with 1300 controls (patients with the 5 other types of vasculitis), in order to determine the optimal combination of symptoms, signs and investigations that classify each person into the appropriate group.

status: Unknown status

ReferenceCitation: Singh JA, Solomon DH, Dougados M, Felson D, Hawker G, Katz P, Paulus H, Wallace C; Classification and Response Criteria Subcommittee of the Committee on Quality Measures, American College of Rheumatology. Development of classification and response criteria for rheumatic diseases. Arthritis Rheum. 2006 Jun 15;55(3):348-52.

ReferenceCitation: Rao JK, Allen NB, Pincus T. Limitations of the 1990 American College of Rheumatology classification criteria in the diagnosis of vasculitis. Ann Intern Med. 1998 Sep 1;129(5):345-52.

ReferenceCitation: Hunder GG, Arend WP, Bloch DA, Calabrese LH, Fauci AS, Fries JF, Leavitt RY, Lie JT, Lightfoot RW Jr, Masi AT, et al. The American College of Rheumatology 1990 criteria for the classification of vasculitis. Introduction. Arthritis Rheum. 1990 Aug;33(8):1065-7.

ReferenceCitation: Fries JF, Hunder GG, Bloch DA, Michel BA, Arend WP, Calabrese LH, Fauci AS, Leavitt RY, Lie JT, Lightfoot RW Jr, et al. The American College of Rheumatology 1990 criteria for the classification of vasculitis. Summary. Arthritis Rheum. 1990 Aug;33(8):1135-6.

ReferenceCitation: Jennette JC, Falk RJ, Andrassy K, Bacon PA, Churg J, Gross WL, Hagen EC, Hoffman GS, Hunder GG, Kallenberg CG, et al. Nomenclature of systemic vasculitides. Proposal of an international consensus conference. Arthritis Rheum. 1994 Feb;37(2):187-92. Review.

ReferenceCitation: Sørensen SF, Slot O, Tvede N, Petersen J. A prospective study of vasculitis patients collected in a five year period: evaluation of the Chapel Hill nomenclature. Ann Rheum Dis. 2000 Jun;59(6):478-82.

ReferenceCitation: Felson DT, Anderson JJ. Methodological and statistical approaches to criteria development in rheumatic diseases. Baillieres Clin Rheumatol. 1995 May;9(2):253-66. Review.

ReferenceCitation: Yates M, MacGregor AJ, Robson J, Craven A, Merkel PA, Luqmani RA, Watts RA. The association of vascular risk factors with visual loss in giant cell arteritis. Rheumatology (Oxford). 2017 Apr 1;56(4):524-528. doi: 10.1093/rheumatology/kew397.

family: Yates

full_name: Max Yates

given: Max

family: MacGregor

full_name: Alex J. MacGregor

given: Alex J.

family: Robson

full_name: Joanna Robson

given: Joanna

family: Craven

full_name: Anthea Craven

given: Anthea

family: Merkel

full_name: Peter A. Merkel

given: Peter A.

family: Luqmani

full_name: Raashid A. Luqmani

given: Raashid A.

family: Watts

full_name: Richard A. Watts

given: Richard A.

journal_name: Rheumatology

repository_institution: University of East Anglia - UEA Digital Repository

publisher: Oxford University Press (OUP)

publisher_dissemination: Oxford University Press

publisher_group: Oxford University Press

publisher_normalized: Oxford University Press

lead_sponsor: University of Oxford

location_country:
Finland
Australia
Belgium
Russian Federation
Denmark
Czech Republic
India
Spain
France
Japan
Poland
Norway
Argentina
Canada
Slovenia
Korea, Republic of
Sweden
Switzerland
Netherlands
Sri Lanka
Ireland
Hungary
China
Portugal
Germany
United States
United Kingdom
Italy
Austria
Mexico
Egypt
New Zealand
Turkey

location_facility: Imperial College Healthcare NHS Trust, Hammersmith Hospital

location_facility: Peking Union Medical College Hospital, Beijing

location_facility: Santa Maria Nuova Hospital, Reggio Emilia

location_facility: New York University Langone Medical Centre

location_facility: Basildon and Thurrock University Hospitals NHS Foundation Trust

location_facility: Instituto Nacional de Enfermedades Respiratorias

location_facility: St. Vincent's University Hospital, Dublin

location_facility: Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

location_facility: North Cumbria University Hospitals, The Cumberland Infirmary

location_facility: University of Manchester, Manchester Royal Infirmary

location_facility: Dartmouth-Hitchcock Medical Centre, Lebanon, NH

location_facility: Heatherwood & Wexham Park Hospitals NHS Foundation Trust

location_facility: Christian Medical College & Hospital, Vellore

location_facility: Nizam's Institute of Medical Sciences, Hyderabad

location_facility: The University Hospital of Northern Norway, Tromsø

location_facility: Waitemata District Health Board, North Shore Hospital

location_facility: Postgraduate Institute of Medical Education and Research, Chandigarh

location_facility: Ramos Mejia Hospital, University of Buenos Aires

location_facility: Assiut University, Assiut University Hospitals

location_facility: Istanbul University, Cerrahpasa Medical School

location_facility: Oxford University Hospitals NHS Trust (The Churchill Hospital)

location_facility: Chatrapathi Shahuji Maharaj Medical Center, Lucknow (IProcess)

location_facility: Haydarpasa Education and Research Hospital

location_facility: University of Debrecen Medical and Health Science Center

location_facility: Vasculitis Center, Boston University School of Medicine

location_facility: Cochin Hospital, Université Paris-descartes

location_facility: NHS Fife, Whyteman's Brae Hospital, Windygates

location_facility: NHS Greater Glasgow & Clyde, Gartnavel Hospital

location_facility: Istanbul University, Istanbul Medical School

location_facility: Epsom and St Helier University Hospitals NHS Trust

location_facility: Southampton University Hospitals NHS Trust

location_facility: University of Michigan, Internal Medicine

location_facility: Nottingham University Hospitals NHS Trust (QMC)


title: A Single Blind, Multi-centre, Randomised Multinational Phase III Study to Compare the Efficacy and Tolerability of Anagrelide vs Hydroxyurea in Patients With Essential Thrombocythaemia

acronym: ANAHYDRET

summary:
Study AOP 03-007 was designed as a pivotal study to test, if Anagrelide (Thromboreductin®)was not inferior to HU with respect to efficacy in patients with ET. This approach to demonstrate non-inferiority was based on the following decision points:

• ET is a rare disease and recruitment of large patient number (> 1600) to prove superiority was not considered possible.

. It was decided to recruit only treatment naïve high risk patients to avoid pre-treatment bias, which further limited the number of patients eligible for the study.

status: Completed

study_type: Interventional

ReferenceType: derived

ReferenceCitation: Gisslinger H, Gotic M, Holowiecki J, Penka M, Thiele J, Kvasnicka HM, Kralovics R, Petrides PE; ANAHYDRET Study Group. Anagrelide compared with hydroxyurea in WHO-classified essential thrombocythemia: the ANAHYDRET Study, a randomized controlled trial. Blood. 2013 Mar 7;121(10):1720-8. doi: 10.1182/blood-2012-07-443770. Epub 2013 Jan 11.

name: Medical University of Vienna, Department of Internal Medicine I, Division of Hematology and Blood Coagulation, Vienna, Austria;

family: Gisslinger

full_name: Heinz Gisslinger

given: Heinz

sequence: first

name: Institute of Hematology, Clinical Center of Serbia, Belgrade, Serbia;

family: Gotic

full_name: Mirjana Gotic

given: Mirjana

sequence: additional

name: Silesian Medical University, Department of Hematology and BMT, Katowice, Poland;

family: Holowiecki

full_name: Jerzy Holowiecki

given: Jerzy

name: University Hospital Brno, Department of Clinical Hematology, Brno, Czech Republic;

family: Penka

full_name: Miroslav Penka

given: Miroslav

name: Institute of Pathology, University of Cologne, Cologne, Germany;

family: Thiele

full_name: Juergen Thiele

given: Juergen

name: Department of Pathology, University of Frankfurt/Main, Frankfurt/Main, Germany;

family: Kvasnicka

full_name: Hans-Michael Kvasnicka

given: Hans-Michael

name: Center for Molecular Medicine (CeMM), Austrian Academy of Sciences, Vienna, Austria; and

family: Kralovics

full_name: Robert Kralovics

given: Robert

name: Hematology Oncology Center and Ludwig Maximilians University of Munich Medical School, Munich, Germany

family: Petrides

full_name: Petro E. Petrides

given: Petro E.

bso_classification: Medical research

datasource: user

genre: journal-article

genre_raw: journal-article

journal_name: Blood

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

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)

publisher: American Society of Hematology

publisher_dissemination: American Society of Hematology

publisher_group: American Society of Hematology

publisher_normalized: American Society of Hematology

lead_sponsor: AOP Orphan Pharmaceuticals AG

location_country:
Italy
Austria
France
Poland
Lithuania
Slovenia
Singapore
Hungary
Germany
Czech Republic

location_facility:
Center Munich
Center Modena
Center Berlin
Center Ulm
Center Saarbrücken
Center Praha
Center Halle
Center Singapore
Center Warszawa
Center Ljubljana
Center Innsbruck
Center Paris
Center Lodz
Center Brno
Center Pavia
Center Olomouc
Center Budapest
Center Katowice
Center Vilnius
Center Gdansk
Center Lublin
Center AKH
Center Hanusch
Center Krakow

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Completed


title: ADenosine Following Pulmonary Vein Isolation to Target Dormant Conduction Elimination: the ADVICE Trial

acronym: ADVICE

summary: Atrial fibrillation (AF) is the most common heart rhythm disorder, impairs quality of life and increases stroke risk and mortality. Despite advances in medical treatment, AF remains uncontrolled in many patients. In many patients, AF is initiated by abnormal electrical impulses from the pulmonary veins. A catheter ablation procedure called pulmonary vein isolation (PVI) has therefore been developed, using heat to isolate the PV foci from the heart. PVI is very effective, but must be repeated in up to 50% of cases because the foci isolation is not permanent after initial PVI. The intravenous administration of a drug called adenosine during the PVI procedure can unmask residual conduction that would otherwise remain unnoticed, so-called "dormant conduction". In our experience, additional ablation guided by adenosine reduces AF recurrence and the need for a repeat PVI procedure. However, the adenosine-guided approach has not yet been proven as standard therapy. The present study, to be conducted at 15 clinical centres in Canada, Europe and Australia is therefore intended to evaluate the efficacy of adenosine-guided ablation to prevent AF recurrence. Five hundred twenty-six patients will be included in the study, which should be completed within 2 years. In all patients, the presence of dormant conduction will be tested with adenosine during PVI. If dormant conduction is observed, additional ablation will be performed in half of these patients selected randomly. If there is no dormant conduction, randomly selected patients will be followed in a registry. If the adenosine-guided approach is demonstrated to improve the success rate of PVI procedures, it should become the standard approach for a "permanent cure" of AF, and therefore benefit patients by reducing arrhythmia recurrence, hospitalizations and the need for repeat interventions.

study_completion_date_type: Actual

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceCitation: Willems S, Khairy P, Andrade JG, Hoffmann BA, Levesque S, Verma A, Weerasooriya R, Novak P, Arentz T, Deisenhofer I, Rostock T, Steven D, Rivard L, Guerra PG, Dyrda K, Mondesert B, Dubuc M, Thibault B, Talajic M, Roy D, Nattel S, Macle L; ADVICE Trial Investigators*. Redefining the Blanking Period After Catheter Ablation for Paroxysmal Atrial Fibrillation: Insights From the ADVICE (Adenosine Following Pulmonary Vein Isolation to Target Dormant Conduction Elimination) Trial. Circ Arrhythm Electrophysiol. 2016 Aug;9(8). pii: e003909. doi: 10.1161/CIRCEP.115.003909.

name: For the author affiliations, please see the Appendix.

family: Willems

full_name: Stephan Willems

given: Stephan

family: Khairy

full_name: Paul Khairy

given: Paul

family: Andrade

full_name: Jason G. Andrade

given: Jason G.

family: Hoffmann

full_name: Boris A. Hoffmann

given: Boris A.

family: Levesque

full_name: Sylvie Levesque

given: Sylvie

family: Verma

full_name: Atul Verma

given: Atul

family: Weerasooriya

full_name: Rukshen Weerasooriya

given: Rukshen

family: Novak

full_name: Paul Novak

family: Arentz

full_name: Thomas Arentz

given: Thomas

family: Deisenhofer

full_name: Isabel Deisenhofer

given: Isabel

family: Rostock

full_name: Thomas Rostock

family: Steven

full_name: Daniel Steven

given: Daniel

family: Rivard

full_name: Lena Rivard

given: Lena

family: Guerra

full_name: Peter G. Guerra

given: Peter G.

family: Dyrda

full_name: Katia Dyrda

given: Katia

family: Mondesert

full_name: Blandine Mondesert

given: Blandine

family: Dubuc

full_name: Marc Dubuc

given: Marc

family: Thibault

full_name: Bernard Thibault

given: Bernard

family: Talajic

full_name: Mario Talajic

given: Mario

family: Roy

full_name: Denis Roy

given: Denis

family: Nattel

full_name: Stanley Nattel

given: Stanley

family: Macle

full_name: Laurent Macle

given: Laurent

journal_name: Circulation: Arrhythmia and Electrophysiology

publisher: Ovid Technologies (Wolters Kluwer Health)

ReferenceCitation: Macle L, Khairy P, Weerasooriya R, Novak P, Verma A, Willems S, Arentz T, Deisenhofer I, Veenhuyzen G, Scavée C, Jaïs P, Puererfellner H, Levesque S, Andrade JG, Rivard L, Guerra PG, Dubuc M, Thibault B, Talajic M, Roy D, Nattel S; ADVICE trial investigators. Adenosine-guided pulmonary vein isolation for the treatment of paroxysmal atrial fibrillation: an international, multicentre, randomised superiority trial. Lancet. 2015 Aug 15;386(9994):672-9. doi: 10.1016/S0140-6736(15)60026-5. Epub 2015 Jul 23.

ReferenceCitation: Andrade JG, Pollak SJ, Monir G, Khairy P, Dubuc M, Roy D, Talajic M, Deyell M, Rivard L, Thibault B, Guerra PG, Nattel S, Macle L. Pulmonary vein isolation using a pace-capture-guided versus an adenosine-guided approach: effect on dormant conduction and long-term freedom from recurrent atrial fibrillation--a prospective study. Circ Arrhythm Electrophysiol. 2013 Dec;6(6):1103-8. doi: 10.1161/CIRCEP.113.000454. Epub 2013 Oct 4. Erratum in: Circ Arrhythm Electrophysiol. 2015 Feb;8(1):247.

name: From the Electrophysiology Service at the Montreal Heart Institute, Department of Medicine, Université de Montréal, Montreal, Québec, Canada (J.G.A., P.K., M.D., D.R., M.T., L.R., B.T., P.G.G., S.N., L.M.); Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada (J.G.A., M.D.); and Arrhythmia and Ablation Center, Department of Cardiology, Florida Hospital, Orlando, FL (S.J.P., G.M.).

title: Pulmonary Vein Isolation Using a Pace-Capture–Guided Versus an Adenosine-Guided Approach

ReferenceCitation: Macle L, Khairy P, Verma A, Weerasooriya R, Willems S, Arentz T, Novak P, Veenhuyzen G, Scavée C, Skanes A, Puererfellner H, Jaïs P, Khaykin Y, Rivard L, Guerra PG, Dubuc M, Thibault B, Talajic M, Roy D, Nattel S; ADVICE Study Investigators. Adenosine following pulmonary vein isolation to target dormant conduction elimination (ADVICE): methods and rationale. Can J Cardiol. 2012 Mar-Apr;28(2):184-90. doi: 10.1016/j.cjca.2011.10.008. Epub 2012 Jan 2.

title: Adenosine Following Pulmonary Vein Isolation to Target Dormant Conduction Elimination (ADVICE): Methods and Rationale

location_country: Austria Germany France Australia Belgium Canada

location_facility: McMaster University and Hamilton Health Sciences

location_facility: Institut universitaire de cardiologie et de pneumologie de Québec

location_facility: Centre hospitalier universitaire de Sherbrooke


ReferenceType: result

ReferenceCitation: Scheid C, Reece D, Beksac M, Spencer A, Callander N, Sonneveld P, Kalimi G, Cai C, Shi M, Scott JW, Stewart AK. Phase 2 study of dovitinib in patients with relapsed or refractory multiple myeloma with or without t(4;14) translocation. Eur J Haematol. 2015 Oct;95(4):316-24. doi: 10.1111/ejh.12491. Epub 2015 Jan 22.

name: University Hospital of Cologne Cologne Germany

family: Scheid

full_name: Christof Scheid

given: Christof

name: Princess Margaret Cancer Centre Toronto ON Canada

family: Reece

full_name: Donna Reece

given: Donna

name: Ankara University School of Medicine Ankara Turkey

family: Beksac

full_name: Meral Beksac

given: Meral

name: Alfred Health‐Monash University Melbourne Vic. Australia

family: Spencer

full_name: Andrew Spencer

given: Andrew

family: Callander

full_name: Natalie Callander

given: Natalie

name: Erasmus Medical Center Rotterdam the Netherlands

family: Sonneveld

full_name: Pieter Sonneveld

given: Pieter

name: Novartis Pharmaceuticals East Hanover NJ USA

family: Kalimi

full_name: Ghulam Kalimi

given: Ghulam

family: Cai

full_name: Can Cai

given: Can

family: Shi

full_name: Michael Shi

given: Michael

family: Scott

full_name: Jeffrey W. Scott

given: Jeffrey W.

family: Stewart

full_name: A. Keith Stewart

given: A. Keith

journal_name: European Journal of Haematology

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

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

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

lead_sponsor: Novartis Pharmaceuticals

location_country:
United States
Netherlands
Germany
France
Australia
Turkey
Canada

location_facility: Duke University Medical Center Dept. of DUMC (4)

location_facility: University of South Alabama / Mitchell Cancer Institute Dept. of Mitchell Cancer Inst.

location_facility: Hematology and Oncology Specialists Dept of Hem&Onc Specialist - 2

location_facility: Medical College of Wisconsin Med College of Wisconsin

location_facility: Central Coast Medical Oncology Corporation

location_facility: Central Hematology Oncology Medical Group

location_facility: Cancer Centers of the Carolinas Dept. of CC of the Carolinas

location_facility: University of Texas Southwestern Medical Center UTSW Medical Center

location_facility: Kootenai Medical Center Kootenai Medical Center

location_facility: St. Jude Heritage Medical Group Virginia Crosson Cancer Center

location_facility: University of Tennessee Cancer Institute SC-2

location_facility: Memorial Sloan Kettering Cancer Center MSKCC

type: EudraCT Number

source: euctr

title: A Phase II, Multi-center, Non-randomized, Open-label Study of TKI258 in Patients With Relapsed or Refractory Multiple Myeloma, Who Are With or Without t(4;14) Translocation

summary: This study will evaluate safety and efficacy of TKI258 in patients with relapsed or refractory multiple myeloma


title: A Multicenter, Randomized, Open-Label, Parallel-Group, Active-Controlled Study to Evaluate the Benefits of Switching Therapy (Glatiramer Acetate or Interferon Beta-1a) to Natalizumab in Subjects With Relapsing Remitting Multiple Sclerosis

acronym: SURPASS

summary: This was a multicenter, randomized, open-label, parallel-group, active-controlled study. Prior to randomization, participants were to have been treated with glatiramer acetate or interferon β-1a (44 μg). Participants were to be randomized to receive natalizumab, interferon β-1a 44 μg, or glatiramer acetate.

status: Terminated

lead_sponsor: Biogen

location_country:
Italy
United States
Sweden
Finland
France
Poland
Slovenia
Hungary
Australia
Latvia
Spain
Canada
Czech Republic

status_simplified: Unknown


title: Retrospective Observational Study Of Subjects With Cytokine-Refractory Metastatic Renal Cancer Treated With Axitinib (AG-013736) To Estimate 5-Yr Survival

summary: The primary objective of this observational study is to retrospectively collect current survival data for patients originally included in axitinib A4061012 [NCT00076011] study to estimate the 5-year survival rate in subjects with metastatic renal cancer cell treated with axitinib.

study_type: Observational

time_perspective: Retrospective

lead_sponsor: Pfizer

location_country:
Germany
United States
France


title: Spinal Cord Stimulation With Precision® Spinal Cord Stimulation System Versus Reoperation for Failed Back Surgery Syndrome

acronym: Evidence

summary: The EVIDENCE trial is a randomized controlled trial comparing the therapeutic effectiveness and cost-effectiveness of the Precision® Spinal Cord Stimulation with that of reoperation as a treatment of pain in patients with Failed Back Surgery Syndrome (FBSS). FBSS is defined as persistent or recurrent pain following one or more lumbosacral spine surgical procedures. Surgical procedures that can result in FBSS can be categorized as either decompression or decompression followed by fusion with or without instrumentation. The pain of FBSS is categorized as neuropathic, which involves pathological nerve activity and is commonly characterized by patients as shooting or burning and/or nociceptive, which signals actual or impending tissue damage or inflammation.

lead_sponsor: Boston Scientific Corporation

location_country:
United States
United Kingdom
Canada
France

location_facility: The University of Illinois Medical Center

intervention_type: Device


type: Other Identifier

title: Open-Label Extension for Alzheimer's Disease Patients Who Complete One of Two Semagacestat Phase 3 Double-Blind Studies (H6L-MC-LFAN or H6L-MC-LFBC)

acronym: Identity XT

summary:
The primary objective of the original study was to assess the safety of semagacestat in Alzheimer's disease (AD) patients during 24 months of open-label treatment. Baseline for the efficacy measures is defined as the baseline for feeder studies LFAN (NCT00594568) and LFBC (NCT00762411). For all safety analyses (adverse events), baseline for patients will be week 0 of this study (LFBF).

Preliminary results from LFAN and LFBC showed semagacestat did not slow disease progression and was associated with worsening of clinical measures of cognition and the ability to perform activities of daily living. Study drug was stopped in all studies. Studies LFAN, LFBC and LFBF have been amended to continue collecting safety data, including cognitive scores, for at least seven months. The CT-Registry will reflect results of analyses from the original protocol in addition to those from the amended protocol. Very few participants from LFBC rolled over into LFBF (N = 9). Due to insufficient sample size, the data for LFBC participants who rolled into LFBF were not analyzed.

lead_sponsor: Eli Lilly and Company

location_country:
Italy
United States
Chile
South Africa
Sweden
Germany
Finland
France
Japan
Poland
Israel
Australia
Spain
Canada

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


title: A Multi-Center, Prospective Study of the WallFlex Biliary RX Fully Covered Stent for the Treatment of Benign Biliary Strictures

summary: The purpose of this study is to evaluate the safety and performance of the WallFlex® Biliary RX Fully Covered Stent as a treatment of biliary obstruction resulting from benign bile duct strictures.

study_start_date_type: Actual

design_allocation: Non-Randomized

ReferenceCitation: Devière J, Nageshwar Reddy D, Püspök A, Ponchon T, Bruno MJ, Bourke MJ, Neuhaus H, Roy A, González-Huix Lladó F, Barkun AN, Kortan PP, Navarrete C, Peetermans J, Blero D, Lakhtakia S, Dolak W, Lepilliez V, Poley JW, Tringali A, Costamagna G; Benign Biliary Stenoses Working Group. Successful management of benign biliary strictures with fully covered self-expanding metal stents. Gastroenterology. 2014 Aug;147(2):385-95; quiz e15. doi: 10.1053/j.gastro.2014.04.043. Epub 2014 May 4.

family: Devière

full_name: Jacques Devière

given: Jacques

family: Reddy

full_name: D. Nageshwar Reddy

given: D. Nageshwar

family: Püspök

full_name: Andreas Püspök

given: Andreas

family: Ponchon

full_name: Thierry Ponchon

given: Thierry

family: Bruno

full_name: Marco J. Bruno

given: Marco J.

family: Bourke

full_name: Michael J. Bourke

given: Michael J.

family: Neuhaus

full_name: Horst Neuhaus

given: Horst

full_name: André Roy

given: André

family: González-Huix Lladó

full_name: Ferrán González-Huix Lladó

given: Ferrán

family: Barkun

full_name: Alan N. Barkun

given: Alan N.

family: Kortan

full_name: Paul P. Kortan

given: Paul P.

family: Navarrete

full_name: Claudio Navarrete

given: Claudio

family: Peetermans

full_name: Joyce Peetermans

given: Joyce

family: Blero

full_name: Daniel Blero

given: Daniel

family: Lakhtakia

full_name: Sundeep Lakhtakia

given: Sundeep

family: Dolak

full_name: Werner Dolak

given: Werner

family: Lepilliez

full_name: Vincent Lepilliez

given: Vincent

family: Poley

full_name: Jan W. Poley

given: Jan W.

family: Tringali

full_name: Andrea Tringali

given: Andrea

family: Costamagna

full_name: Guido Costamagna

given: Guido

journal_name: Gastroenterology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

title: Successful Management of Benign Biliary Strictures With Fully Covered Self-Expanding Metal Stents

ReferenceCitation: Poley JW, Ponchon T, Puespoek A, Bruno M, Roy A, Peetermans J, Rousseau M, Lépilliez V, Dolak W, Tringali A, Blero D, Carr-Locke D, Costamagna G, Devière J; Benign Biliary Stenoses Working Group. Fully covered self-expanding metal stents for benign biliary stricture after orthotopic liver transplant: 5-year outcomes. Gastrointest Endosc. 2020 Dec;92(6):1216-1224. doi: 10.1016/j.gie.2020.04.078. Epub 2020 May 15.

full_name: Jan-Werner Poley

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

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

ReferenceCitation: Tringali A, Reddy DN, Ponchon T, Neuhaus H, Lladó FG, Navarrete C, Bruno MJ, Kortan PP, Lakhtakia S, Peetermans J, Rousseau M, Carr-Locke D, Devière J, Costamagna G; Benign Biliary Stenoses Working Group. Treatment of post-cholecystectomy biliary strictures with fully-covered self-expanding metal stents - results after 5 years of follow-up. BMC Gastroenterol. 2019 Dec 12;19(1):214. doi: 10.1186/s12876-019-1129-3.

name: for the Benign Biliary Stenoses Working Group

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

title: Treatment of post-cholecystectomy biliary strictures with fully-covered self-expanding metal stents – results after 5 years of follow-up

ReferenceCitation: Lakhtakia S, Reddy N, Dolak W, Ponchon T, Bruno MJ, Bourke MJ, Neuhaus H, Roy A, González-Huix Lladó F, Kortan PP, Peetermans J, Rousseau M, Costamagna G, Devière J; Benign Biliary Stenoses Working Group. Long-term outcomes after temporary placement of a self-expanding fully covered metal stent for benign biliary strictures secondary to chronic pancreatitis. Gastrointest Endosc. 2020 Feb;91(2):361-369.e3. doi: 10.1016/j.gie.2019.08.037. Epub 2019 Sep 5.

location_country: Italy Austria Chile India Netherlands Germany France Australia Belgium Spain Canada

location_facility: Hospital Universitari Doctor Josep Trueta

location_facility: Università Cattolica del Sacro Cuore Policlinico A. Gemelli

location_facility: Centre Hospitalier de l'Université de Montréal-Saint-Luc Hospital


title: Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Dose-finding Study to Evaluate the Efficacy, Safety, and Tolerability of Three Doses of ACT-128800, an Oral S1P1 Receptor Agonist, Administered for Twenty-four Weeks in Patients With Relapsing-remitting Multiple Sclerosis

summary: This study will assess the efficacy, safety, and tolerability of ACT-128800 in patients with relapsing-remitting multiple sclerosis.

ReferenceCitation: Olsson T, Boster A, Fernández Ó, Freedman MS, Pozzilli C, Bach D, Berkani O, Mueller MS, Sidorenko T, Radue EW, Melanson M. Oral ponesimod in relapsing-remitting multiple sclerosis: a randomised phase II trial. J Neurol Neurosurg Psychiatry. 2014 Nov;85(11):1198-208. doi: 10.1136/jnnp-2013-307282. Epub 2014 Mar 21.

family: Olsson

full_name: T. Olsson

given: T.

family: Boster

full_name: A. Boster

given: A.

family: Fernandez

full_name: O. Fernandez

given: O.

family: Freedman

full_name: M. S. Freedman

given: M. S.

family: Pozzilli

full_name: C. Pozzilli

given: C.

family: Bach

full_name: D. Bach

given: D.

family: Berkani

full_name: O. Berkani

family: Mueller

full_name: M. S. Mueller

family: Sidorenko

full_name: T. Sidorenko

family: Radue

full_name: E.-W. Radue

family: Melanson

full_name: M. Melanson

given: M.

journal_name: Journal of Neurology, Neurosurgery & Psychiatry

evidence: open (via page says license)

license: cc-by-nc

license_normalized: cc-by-nc

publisher: BMJ

publisher_dissemination: British Medical Journal

publisher_group: British Medical Journal

publisher_normalized: British Medical Journal

ReferenceCitation: Gisleskog PO, Valenzuela B, Scherz T, Burcklen M, Pérez-Ruixo JJ, Poggesi I. An Exposure-Response Analysis of the Clinical Efficacy of Ponesimod in a Randomized Phase II Study in Patients with Multiple Sclerosis. Clin Pharmacokinet. 2021 Sep;60(9):1227-1237. doi: 10.1007/s40262-021-01020-2. Epub 2021 Apr 29.

family: Gisleskog

full_name: Per Olsson Gisleskog

given: Per Olsson

family: Valenzuela

full_name: Belén Valenzuela

given: Belén

family: Scherz

full_name: Tatiana Scherz

given: Tatiana

family: Burcklen

full_name: Michel Burcklen

given: Michel

family: Pérez-Ruixo

full_name: Juan Jose Pérez-Ruixo

given: Juan Jose

family: Poggesi

location_country:
Finland
Romania
Australia
Belgium
Russian Federation
Ukraine
France
Poland
Canada
Sweden
Switzerland
Netherlands
Bulgaria
Hungary
Germany
United States
United Kingdom
Italy
Austria
Serbia
Czechia
Israel
Spain

location_facility:
Clinical Investigative Site # 2106
Clinical Investigative Site # 3128
Clinical Investigative Site 1302
Clinical Investigative Site 3130
Clinical Investigative Site # 3135
Clinical Investigative Site 2704
Clinical Investigative Site 2800
Clinical Investigative Site 1600
Clinical Investigative Site # 1806
Clinical Investigative Site 2202
Clinical Investigative Site 1805
Clinical Investigative Site 1804
Clinical Investigative Site 2802
Clinical Investigative Site 1500
Clinical Investigative Site 1802
Clinical Investigative Site 1400
Clinical Investigative Site 3127
Clinical Investigative Site 3105
Clinical Investigative Site 2402
Clinical Investigative Site 2502
Clinical Investigative Site 1504
Clinical Investigative Site 1401
Clinical Investigative Site 3304
Clinical Investigative Site 1602
Clinical Investigative Site # 3202
Clinical Investigative Site 1203
Clinical Investigative Site 2702
Clinical Investigative Site 3101
Clinical Investigative Site 2701
Clinical Investigative Site 1100
Clinical Investigative Site 3133
Clinical Investigative Site 3200
Clinical Investigative Site 2003
Clinical Investigative 1807
Clinical Investigative Site 3201
Clinical Investigative Site 1501
Clinical Investigative Site 2105
Clinical Investigative Site 2302
Clinical Investigative Site 3209
Clinical Investigative Site # 3203
Clinical Investigative Site 3119
Clinical Investigative Site 2900
Clinical Investigative Site # 3204
Clinical Investigative Site 3113
Clinical Investigative Site # 1205
Clinical Investigative Site 2400
Clinical Investigative Site 3100
Clinical Investigative Site 3117
Clinical Investigative Site # 1904
Clinical Investigative Site 2201
Clinical Investigative Site 3303
Clinical Investigative Site 2705
Clinical Investigative Site 1503
Clinical Investigative Site 1900
Clinical Investigative Site 3129
Clinical Investigative Site 1201
Clinical Investigative Site 2706
Clinical Investigative Site 3125
Clinical Investigative Site # 1401
Clinical Investigative Site 2501
Clinical Investigative Site 1502
Clinical Investigative Site 1908
Clinical Investigative Site 2304
Clinical Investigative Site 1601
Clinical Investigative Site 1905
Clinical Investigative Site 2401
Clinical Investigative Site 1803
Clinical Investigative Site 3115
Clinical Investigative Site 1204
Clinical Investigative Site 2700
Clinical Investigative Site 2104
Clinical Investigative Site 1303
Clinical Investigative Site 2002
Clinical Investigative Site 2101
Clinical Investigative Site # 3206
Clinical Investigative Site 1603
Clinical Investigator 3104
Clinical Investigative Site 3002
Clinical Investigative Site 2001
Clinical Investigative Site 1506
Clinical Investigative Site 3132
Clinical Investigative Site 3118
Clinical Investigative Site 3003
Clinical Investigative Site 3004
Clinical Investigative Site 1301
Clinical Investigative Site 1304
Clinical Investigative site 1800
Clinical Investigative Site 1101
Clinical Investigative Site 3120
Clinical Investigative Site 3300
Clinical Investigative Site 2102
Clinical Investigative Site 1102
Clinical Investigative Site 3112
Clinical Investigative Site 3208
Clinical Investigative Site 2103
Clinical Investigative Site 2305
Clinical Investigator 3136
Clinical Investigative Site 2303
Clinical Investigator 3126
Clinical Investigative Site 2000
Clinical Investigative Site 3102
Clinical Investigative Site 2801
Clinical Investigative Site 3111
Clinical Investigative Site 1902
Clinical Invesigative Site 3116
Clinical Investigative Site # 3201
Clinical Investigative Site # 1001
Clinical Investigative Site 2203
Clinical Investigative Site 1901
Clinical Investigative Site 1000
Clinical Investigative Site 2901
Clinical Investigative Site 1701
Clinical Investigative Site 2503
Clinical Investigative Site 3302
Clinical Investigative Site 3107


title: RECHARGE Sub-Study to the Implantable Systems Performance Registry (ISPR)

acronym: RECHARGE

summary: The purpose of this study is to assess the recharge feature of the Activa RC System in patients who are receiving Deep Brain Stimulation (DBS) for Parkinson's Disease (PD), Essential Tremor (ET), or dystonia.

lead_sponsor: MedtronicNeuro

location_country:
Austria
Germany
France
Spain
United States
United Kingdom


title: Patterns of Non-Adherence to Dual Anti-Platelet Regimen In Stented Patients: An Observational Single-Arm Study (The PARIS Registry)

acronym: PARIS

summary: The purpose of this observational research study is to determine when and why patients discontinue, interrupt, or disrupt the regimen of anti-platelet medications prescribed following stent implantation, and to examine the relationship between specific patterns of non-adherence and patient outcomes.

time_perspective: Prospective

ReferenceCitation: Baber U, Leisman DE, Cohen DJ, Gibson CM, Henry TD, Dangas G, Moliterno D, Kini A, Krucoff M, Colombo A, Chieffo A, Sartori S, Witzenbichler B, Steg PG, Pocock SJ, Mehran R. Tailoring Antiplatelet Therapy Intensity to Ischemic and Bleeding Risk. Circ Cardiovasc Qual Outcomes. 2019 Jan;12(1):e004945. doi: 10.1161/CIRCOUTCOMES.118.004945.

name: Cardiovascular Institute, Icahn School of Medicine at Mount Sinai, New York, NY (U.B., D.E.L., G.D., A.K., S.S., R.M.).

family: Baber

full_name: Usman Baber

given: Usman

family: Leisman

full_name: Daniel E. Leisman

given: Daniel E.

name: Saint Luke’s Mid America Heart Institute, University of Missouri–Kansas City (D.J.C.).

family: Cohen

full_name: David J. Cohen

given: David J.

name: Division of Cardiology, Beth Israel Deaconess Medical Center, Boston, MA (C.M.G.).

family: Gibson

full_name: C. Michael Gibson

given: C. Michael

name: The Heart Institute, Cedars-Sinai Medical Center, Los Angeles, CA (T.D.H.).

family: Henry

full_name: Timothy D. Henry

given: Timothy D.

family: Dangas

full_name: George Dangas

given: George

name: Gill Heart Institute and Division of Cardiovascular Medicine, University of Kentucky, Lexington KY (D.M.).

family: Moliterno

full_name: David Moliterno

given: David

family: Kini

full_name: Annapoorna Kini

given: Annapoorna

name: Duke University Medical Center, Duke Clinical Research Institute, Durham, NC (M.K.).

family: Krucoff

full_name: Mitchell Krucoff

given: Mitchell

name: Cardio-Thoracic Department, San Raffaele Scientific Institute, Milan, Italy (A. Colombo, A. Chieffo).

family: Colombo

full_name: Antonio Colombo

given: Antonio

family: Chieffo

full_name: Alaide Chieffo

given: Alaide

family: Sartori

full_name: Samantha Sartori

given: Samantha

name: Helios Amper-Klinikum, Dachau, Germany (B.W.).

family: Witzenbichler

full_name: Bernhard Witzenbichler

given: Bernhard

name: Université Paris-Diderot, Sorbonne Paris-Cité, France (P.G.S.).

family: Steg

full_name: Philippe Gabriel Steg

given: Philippe Gabriel

name: London School of Hygiene and Tropical Medicine, United Kingdom (S.J.P.).

family: Pocock

full_name: Stuart J. Pocock

given: Stuart J.

family: Mehran

full_name: Roxana Mehran

given: Roxana

journal_name: Circulation: Cardiovascular Quality and Outcomes

oa_host_type: publisher

publisher_dissemination: Wolters Kluwer Health

publisher_group: Wolters Kluwer Health

publisher_normalized: Wolters Kluwer Health

ReferenceCitation: Shah B, Baber U, Pocock SJ, Krucoff MW, Ariti C, Gibson CM, Steg PG, Weisz G, Witzenbichler B, Henry TD, Kini AS, Stuckey T, Cohen DJ, Iakovou I, Dangas G, Aquino MB, Sartori S, Chieffo A, Moliterno DJ, Colombo A, Mehran R. White Blood Cell Count and Major Adverse Cardiovascular Events After Percutaneous Coronary Intervention in the Contemporary Era: Insights From the PARIS Study (Patterns of Non-Adherence to Anti-Platelet Regimens in Stented Patients Registry). Circ Cardiovasc Interv. 2017 Sep;10(9). pii: e004981. doi: 10.1161/CIRCINTERVENTIONS.117.004981.

name: From the Department of Medicine (Cardiology), New York Harbor Health Care System, Manhattan VA Hospital (B.S.); Department of Medicine (Cardiology), New York University School of Medicine (B.S.); Department of Medicine (Cardiology), Icahn School of Medicine at Mount Sinai, New York, NY (U.B., A.S.K., G.D., M.B.A., S.S., R.M.); Medical Statistics, London School of Hygiene and Tropical Medicine, United Kingdom (S.J.P., C.A.); Department of Medicine (Cardiology), Duke University School of Medicine,...

family: Shah

full_name: Binita Shah

given: Binita

full_name: Mitchell W. Krucoff

given: Mitchell W.

repository_institution: London School of Hygiene and Tropical Medicine - LSHTM Research Online

ReferenceCitation: Baber U, Mehran R, Giustino G, Cohen DJ, Henry TD, Sartori S, Ariti C, Litherland C, Dangas G, Gibson CM, Krucoff MW, Moliterno DJ, Kirtane AJ, Stone GW, Colombo A, Chieffo A, Kini AS, Witzenbichler B, Weisz G, Steg PG, Pocock S. Coronary Thrombosis and Major Bleeding After PCI With Drug-Eluting Stents: Risk Scores From PARIS. J Am Coll Cardiol. 2016 May 17;67(19):2224-2234. doi: 10.1016/j.jacc.2016.02.064. Epub 2016 Apr 11.

journal_name: Journal of the American College of Cardiology

ReferenceCitation: Mehran R, Baber U, Steg PG, Ariti C, Weisz G, Witzenbichler B, Henry TD, Kini AS, Stuckey T, Cohen DJ, Berger PB, Iakovou I, Dangas G, Waksman R, Antoniucci D, Sartori S, Krucoff MW, Hermiller JB, Shawl F, Gibson CM, Chieffo A, Alu M, Moliterno DJ, Colombo A, Pocock S. Cessation of dual antiplatelet treatment and cardiac events after percutaneous coronary intervention (PARIS): 2 year results from a prospective observational study. Lancet. 2013 Nov 23;382(9906):1714-22. doi: 10.1016/S0140-6736(13)61720-1. Epub 2013 Sep 1.

location_country:
Italy
United States
France
Greece
Germany

location_facility: LeBauer Cardiovascular Research Foundation

location_facility: Saint Luke's/ Mid-America Heart Institute


ReferenceCitation: Roger SD, Gaillard CA, Bock AH, Carrera F, Eckardt KU, Van Wyck DB, Cronin M, Meier Y, Larroque S, Macdougall IC; FIND-CKD Study Investigators. Safety of intravenous ferric carboxymaltose versus oral iron in patients with nondialysis-dependent CKD: an analysis of the 1-year FIND-CKD trial. Nephrol Dial Transplant. 2017 Sep 1;32(9):1530-1539. doi: 10.1093/ndt/gfw264.

family: Roger

full_name: Simon D. Roger

given: Simon D.

name: Department of Nephrology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands

family: Gaillard

full_name: Carlo A. Gaillard

given: Carlo A.

name: Department of Nephrology, Kantonsspital, Aarau, Switzerland

family: Bock

full_name: Andreas H. Bock

given: Andreas H.

name: Department of Dialysis, Eurodial, DaVita, Leiria, Portugal

family: Carrera

full_name: Fernando Carrera

given: Fernando

name: Department of Nephrology and Hypertension, University of Erlangen-Nürnberg, Erlangen, Germany

family: Eckardt

full_name: Kai-Uwe Eckardt

given: Kai-Uwe

name: Clinical Support Services, Davita Healthcare Partners, Denver, CO, USA

family: Van Wyck

full_name: David B. Van Wyck

given: David B.

name: Vifor Pharma Ltd, Glattbrugg, Switzerland

family: Cronin

full_name: Maureen Cronin

given: Maureen

family: Meier

full_name: Yvonne Meier

given: Yvonne

family: Larroque

full_name: Sylvain Larroque

given: Sylvain

name: Department of Renal Medicine, King's College Hospital, London, UK

family: Macdougall

full_name: Iain C. Macdougall

given: Iain C.

family: Gilles

full_name: Alastair Gilles

given: Alastair

family: Faull

full_name: Randall Faull

given: Randall

family: Toussaint

full_name: Nigel D. Toussaint

given: Nigel D.

family: McMahon

full_name: Lawrence McMahon

given: Lawrence

family: Suranyi

full_name: Michael Suranyi

given: Michael

family: Mudge

full_name: David Mudge

family: Hutchison

full_name: Brian Hutchison

given: Brian

family: Irish

full_name: Ashley Irish

given: Ashley

family: Kerr

full_name: Peter Kerr

given: Peter

family: Kulkarni

full_name: Hemant Kulkarni

given: Hemant

family: Elder

full_name: Grahame Elder

given: Grahame

family: Jardine

full_name: Margaret Jardine

given: Margaret

family: Lhotta

full_name: Karl Lhotta

given: Karl

family: Mayer

full_name: Gert Mayer

given: Gert

name: on behalf of the FIND-CKD Study Investigators

repository_institution: St George's University of London - St George's Online Research Archive

ReferenceCitation: Macdougall IC, Bock AH, Carrera F, Eckardt KU, Gaillard C, Van Wyck D, Meier Y, Larroque S, Roger SD; FIND-CKD Study investigators. Renal function in patients with non-dialysis chronic kidney disease receiving intravenous ferric carboxymaltose: an analysis of the randomized FIND-CKD trial. BMC Nephrol. 2017 Jan 17;18(1):24. doi: 10.1186/s12882-017-0444-6.

name: on behalf of the FIND-CKD Study investigators

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

repository_institution: Kooperativer Bibliotheksverbund Berlin-Brandenburg (KOBV), on behalf of the Universitätsbibliothek Erlangen-Nürnberg - OPUS FAU - Online-Publikationssystem der Friedrich-Alexander-Universität Erlangen-Nürnberg

ReferenceCitation: Macdougall IC, Bock AH, Carrera F, Eckardt KU, Gaillard C, Van Wyck D, Roubert B, Nolen JG, Roger SD; FIND-CKD Study Investigators. FIND-CKD: a randomized trial of intravenous ferric carboxymaltose versus oral iron in patients with chronic kidney disease and iron deficiency anaemia. Nephrol Dial Transplant. 2014 Nov;29(11):2075-84. doi: 10.1093/ndt/gfu201. Epub 2014 Jun 2.

location_country:
Italy
Austria
Sweden
United Kingdom
Netherlands
Spain
Germany
France
Poland
Greece
Norway
Romania
Australia
Belgium
Portugal
Turkey
Denmark
United States
Czech Republic

location_facility: Medizinische Universität Innsbruck Univ.-Klinik für Innere Medizin IV

location_facility: Nemocnice s poliklinikou v Novem Jicine, p.o. p.o. Interni oddeleni - nefrologie a dialyza

location_facility: RHMS Baudour - Department of Nephrology and Dialysis

location_facility: Miedzyleski Szpital Spec. Oddzial I Wewnetrzny I Nefrologii

location_facility: Cukurova University Medical Faculty Balcali Hospital - Department of Nephrology

location_facility: Hospital Universitario Marqués de Valdecilla - Servicio de Nefrología

location_facility: General Hospital of Arta - Nephrology Department

location_facility: Spitalul Clinic de Nefrologie"Dr Carol Davila"

location_facility: Meander Medisch Centrum - Locatie Amersfoort Lichtenberg

location_facility: Lillebalt Frederica Sygehus Department of Nephrology

location_facility: Ospedali Riuniti Anzio-Nettuno ASL ROMA H U.O. Nefrologia e Dialisi

title: An Open-label, Multicentre, Randomised, 3-arm Study to Investigate the Comparative Efficacy and Safety of Intravenous Ferric Carboxymaltose (Ferinject High and Low Dosage Regimens) Versus Oral Iron for the Treatment of Iron Deficiency Anaemia in Subjects With Non-dialysis-dependent Chronic Kidney Disease

summary: Phase IIIb study to evaluate the long-term efficacy of ferric carboxymaltose (FCM) (using targeted ferritin levels to determine dosing) or oral iron in non-dialysis-dependent chronic kidney disease (NDD-CKD) subjects with iron deficiency anaemia (IDA).


ReferenceCitation: Steg PG, Mehta SR, Jukema JW, Lip GY, Gibson CM, Kovar F, Kala P, Garcia-Hernandez A, Renfurm RW, Granger CB; RUBY-1 Investigators. RUBY-1: a randomized, double-blind, placebo-controlled trial of the safety and tolerability of the novel oral factor Xa inhibitor darexaban (YM150) following acute coronary syndrome. Eur Heart J. 2011 Oct;32(20):2541-54. doi: 10.1093/eurheartj/ehr334. Epub 2011 Aug 30.

family: Steg

full_name: Ph. Gabriel Steg

given: Ph. Gabriel

family: Mehta

full_name: Shamir R. Mehta

given: Shamir R.

family: Jukema

full_name: J. Wouter Jukema

given: J. Wouter

family: Lip

full_name: Gregory Y.H. Lip

given: Gregory Y.H.

family: Gibson

full_name: C. Michael Gibson

given: C. Michael

family: Kovar

full_name: Frantisek Kovar

given: Frantisek

family: Kala

full_name: Petr Kala

given: Petr

family: Garcia-Hernandez

full_name: Alberto Garcia-Hernandez

given: Alberto

family: Renfurm

full_name: Ronny W. Renfurm

given: Ronny W.

family: Granger

full_name: Christopher B. Granger

given: Christopher B.

journal_name: European Heart Journal

evidence: open (via page says Open Access)

license: implied-oa

license_normalized: implied-oa

repository_institution: Queen Mary University of London - Queen Mary Research Online

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

lead_sponsor: Astellas Pharma Europe B.V.

primary_purpose: Prevention

location_country:
Romania
Belgium
Australia
Russian Federation
Denmark
Czech Republic
Ukraine
India
France
Poland
Slovakia
Argentina
Canada
Korea, Republic of
Colombia
South Africa
Netherlands
Hungary
Germany
Brazil
Mexico

title: A Randomized, Double-Blind, Placebo Controlled Multi-Center and Parallel Group Study of the Safety, Tolerability and Efficacy of YM150 in Combination With Standard Treatment in Secondary Prevention of Ischemic Vascular Events in Subjects With Acute Coronary Syndromes

summary: The purpose of this study is to evaluate the safety and efficacy of various doses of YM150 (the experimental drug) in the prevention of ischemic vascular events in subjects with recent acute coronary syndromes.


title: A Confirmatory, Placebo-controlled, Randomised, Double-blind, Single-dummy, Parallel Group, Ratio-finding Study in Constipated Pain Patients to Establish an Optimal Hydromorphone - Naloxone Ratio With an Improved Bowel Function and a Comparable Analgesic Efficacy Compared to Hydromorphone Alone

summary: The purpose of this study is to establish an optimal hydromorphone - naloxone ratio with an improved bowel function for constipated pain patients.

enrollment_type: Anticipated

lead_sponsor: Mundipharma Research GmbH & Co KG

location_country:
Austria
Switzerland
Netherlands
United Kingdom
Finland
France
Poland
Israel
Romania
Australia
Belgium
Denmark
Germany
Czech Republic

location_facility: Szpital Uniwersytecki w Krakowie Zakład Badania

location_facility: Spitalul Clinic Judetean de Urgenta Cluj


title: A Phase 2a, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of Telaprevir in Combination With Peginterferon Alfa-2a (Pegasys®) and Ribavirin (Copegus®) in Subjects Who Have Chronic HCV-1/HIV-1 Co-Infection and Are Treatment-Naïve for Hepatitis C

summary: The purpose of this study is to determine whether the combination of telaprevir, peginterferon alfa-2a, and ribavirin is safe and effective in treating hepatitis C virus (HCV) infection in subjects who are infected with both HCV and human immunodeficiency virus (HIV).

ReferenceCitation: Sulkowski MS, Sherman KE, Dieterich DT, Bsharat M, Mahnke L, Rockstroh JK, Gharakhanian S, McCallister S, Henshaw J, Girard PM, Adiwijaya B, Garg V, Rubin RA, Adda N, Soriano V. Combination therapy with telaprevir for chronic hepatitis C virus genotype 1 infection in patients with HIV: a randomized trial. Ann Intern Med. 2013 Jul 16;159(2):86-96. doi: 10.7326/0003-4819-159-2-201307160-00654.

family: Sulkowski

full_name: Mark S. Sulkowski

given: Mark S.

family: Sherman

full_name: Kenneth E. Sherman

given: Kenneth E.

family: Dieterich

full_name: Douglas T. Dieterich

given: Douglas T.

family: Bsharat

full_name: Mohammad Bsharat

given: Mohammad

family: Mahnke

full_name: Lisa Mahnke

given: Lisa

family: Rockstroh

full_name: Jürgen K. Rockstroh

given: Jürgen K.

family: Gharakhanian

full_name: Shahin Gharakhanian

given: Shahin

family: McCallister

full_name: Scott McCallister

given: Scott

family: Henshaw

full_name: Joshua Henshaw

given: Joshua

family: Girard

full_name: Pierre-Marie Girard

given: Pierre-Marie

family: Adiwijaya

full_name: Bambang Adiwijaya

given: Bambang

family: Garg

full_name: Varun Garg

given: Varun

family: Rubin

full_name: Raymond A. Rubin

given: Raymond A.

family: Adda

full_name: Nathalie Adda

given: Nathalie

family: Soriano

full_name: Vincent Soriano

given: Vincent

journal_name: Annals of Internal Medicine

publisher: American College of Physicians

publisher_dissemination: American College of Physicians

publisher_group: American College of Physicians

publisher_normalized: American College of Physicians

title: Combination Therapy With Telaprevir for Chronic Hepatitis C Virus Genotype 1 Infection in Patients With HIV

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Germany
United States
France
Spain


title: A Phase I Trial of NECTAR (Nelarabine, Etoposide and Cyclophosphamide in T-ALL Relapse): A Joint Study of TACL and POETIC

summary: Nelarabine has shown significant activity in patients with T-cell malignancies. This study will determine the safety and maximum tolerated dose of the combination of nelarabine, cyclophosphamide and etoposide in patients with first bone marrow relapse of T-ALL, or first relapse of T-LL.

lead_sponsor: Therapeutic Advances in Childhood Leukemia Consortium

location_country:
Italy
Austria
United States
Netherlands
France
Australia
Canada

location_facility: Childrens Hospital & Clinics of Minnesota

location_facility: Levine Children's Hospital at Carolinas Medical Center

location_facility: Children's Hospital New York-Presbyterian

location_facility: Children's Healthcare of Atlanta, Emory University

location_facility: The Children's Hospital, University of Colorado


title: Assessment of Efficacy and Safety of Thioctic Acid in the Oral Treatment of Diabetic Polyneuropathy (Stage 1 or 2) NATHAN1 A Randomized, Placebo-controlled, Double-blind Multi-centre Trial With 2 Parallel Groups

summary: To assess clinical efficacy and safety of long-term orally administered thioctic acid in the treatment of diabetic polyneuropathy.

ReferenceCitation: Ziegler D, Low PA, Litchy WJ, Boulton AJ, Vinik AI, Freeman R, Samigullin R, Tritschler H, Munzel U, Maus J, Schütte K, Dyck PJ. Efficacy and safety of antioxidant treatment with α-lipoic acid over 4 years in diabetic polyneuropathy: the NATHAN 1 trial. Diabetes Care. 2011 Sep;34(9):2054-60. doi: 10.2337/dc11-0503. Epub 2011 Jul 20.

family: Ziegler

full_name: Dan Ziegler

given: Dan

family: Low

full_name: Phillip A. Low

given: Phillip A.

family: Litchy

full_name: William J. Litchy

given: William J.

family: Boulton

full_name: Andrew J.M. Boulton

given: Andrew J.M.

family: Vinik

full_name: Aaron I. Vinik

given: Aaron I.

family: Freeman

full_name: Roy Freeman

given: Roy

family: Samigullin

full_name: Rustem Samigullin

given: Rustem

family: Tritschler

full_name: Hans Tritschler

given: Hans

family: Munzel

full_name: Ullrich Munzel

given: Ullrich

family: Maus

full_name: Joachim Maus

given: Joachim

family: Schütte

full_name: Klemens Schütte

given: Klemens

family: Dyck

full_name: Peter J. Dyck

given: Peter J.

journal_name: Diabetes Care

oa_colors: green other

repository_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

repository_institution: The University of Manchester - Research Explorer

repository_normalized: Pure (Denmark)

repositories:
PubMed Central
Pure (Denmark)
dash.harvard.edu

publisher: American Diabetes Association

publisher_dissemination: American Diabetes Association

publisher_group: American Diabetes Association

publisher_normalized: American Diabetes Association

title: Efficacy and Safety of Antioxidant Treatment With α-Lipoic Acid Over 4 Years in Diabetic Polyneuropathy

lead_sponsor: MEDA Pharma GmbH & Co. KG

location_country:
Italy
Sweden
Netherlands
Croatia
France
Spain
Denmark
United States
United Kingdom

location_facility: Hospital del Mar Department Neurophysiology

location_facility: East Carolina University, School of Medicine

location_facility: University of Missouri Dept. of Neurology

location_facility: Manchester Royal Infirmary Department of Medicine

location_facility: University of Texas South Western Medical Center

location_facility: Hospital Geriatrico Diabetological Service

location_facility: University Clinic for Diabetes, Endocrinology and Metabolic

location_facility: University Hospital Utrecht Department of Internal Medicine

location_facility: Health Partners Riverside Neurology Clinic


title: REVERSEII: Validation of the "Men and HERDOO2"- A Clinical Decision Rule to Identify Patients With "Unprovoked" Venous Thromboembolism Who Can Discontinue Anticoagulants After 6 Months of Treatment.

acronym: REVERSEII

summary: The main objective of this study is to verify whether a new clinical decision rule identifying patients diagnosed with unprovoked blood clots who have a low risk of recurrence can safely stop oral anticoagulant therapy after 5-7 months of treatment.

primary_purpose: Diagnostic

ReferenceType: background

ReferenceCitation: Rodger MA, Kahn SR, Wells PS, Anderson DA, Chagnon I, Le Gal G, Solymoss S, Crowther M, Perrier A, White R, Vickars L, Ramsay T, Betancourt MT, Kovacs MJ. Identifying unprovoked thromboembolism patients at low risk for recurrence who can discontinue anticoagulant therapy. CMAJ. 2008 Aug 26;179(5):417-26. doi: 10.1503/cmaj.080493.

apc_source: doaj

family: Rodger

full_name: M. A. Rodger

given: M. A.

family: Kahn

full_name: S. R. Kahn

given: S. R.

family: Wells

full_name: P. S. Wells

given: P. S.

family: Anderson

full_name: D. A. Anderson

given: D. A.

family: Chagnon

full_name: I. Chagnon

given: I.

family: Le Gal

full_name: G. Le Gal

given: G.

family: Solymoss

full_name: S. Solymoss

given: S.

family: Crowther

full_name: M. Crowther

given: M.

family: Perrier

full_name: A. Perrier

given: A.

family: White

full_name: R. White

given: R.

family: Vickars

full_name: L. Vickars

given: L.

family: Ramsay

full_name: T. Ramsay

given: T.

family: Betancourt

full_name: M. T. Betancourt

given: M. T.

family: Kovacs

full_name: M. J. Kovacs

given: M. J.

currency_apc_doaj: CAD

journal_name: Canadian Medical Association Journal

oa_colors: green gold

publisher: CMA Joule Inc.

publisher_dissemination: CMA Joule Inc.

publisher_group: CMA Joule Inc.

publisher_normalized: CMA Joule Inc.

ReferenceCitation: Rodger MA, Le Gal G, Anderson DR, Schmidt J, Pernod G, Kahn SR, Righini M, Mismetti P, Kearon C, Meyer G, Elias A, Ramsay T, Ortel TL, Huisman MV, Kovacs MJ; REVERSE II Study Investigators. Validating the HERDOO2 rule to guide treatment duration for women with unprovoked venous thrombosis: multinational prospective cohort management study. BMJ. 2017 Mar 17;356:j1065. doi: 10.1136/bmj.j1065.

full_name: Marc A Rodger

given: Marc A

full_name: Gregoire Le Gal

given: Gregoire

repository_institution: University of Geneva - Archive ouverte UNIGE

repository_institution: University of Oxford - Oxford Academic Notes & Queries

location_country:
United States
India
Switzerland
France
Belgium
Australia
Canada

location_facility: CDHA-Queen Elizabeth II Health Science Centre

location_facility: Centre Hospitalier Regional Universitaire de Brest


acronym: ISPR

summary:
The Implantable Systems Performance Registry (ISPR) was developed to evaluate the long-term reliability and performance of market-released Medtronic Neuromodulation infusion and stimulation products.

Completion Notice: The ISPR was created by Medtronic to monitor the performance of commercially available products. This registry was initially designed to track performance of Medtronic's implantable infusion and spinal cord stimulation systems beginning in August 2003 and June 2004, respectively. Medtronic DBS Systems were added to the ISPR in June 2009, and Sacral Neuromodulation Systems in April 2010. Medtronic retired the ISPR in April 2016 and merged the data collected from this registry into the Product Surveillance Registry (PSR). Please refer to that record, Clinical Trials identifier: NCT01524276, for further information.

location_country:
Italy
Austria
Germany
France
Spain
United States
United Kingdom

location_facility: Site recruiting for sacral nerve stimulation

location_facility: Site is recruiting for Deep Brain Stimulation


ReferenceCitation: Bartlett NL, Chen R, Fanale MA, Brice P, Gopal A, Smith SE, Advani R, Matous JV, Ramchandren R, Rosenblatt JD, Huebner D, Levine P, Grove L, Forero-Torres A. Retreatment with brentuximab vedotin in patients with CD30-positive hematologic malignancies. J Hematol Oncol. 2014 Mar 19;7:24. doi: 10.1186/1756-8722-7-24.

family: Bartlett

full_name: Nancy L Bartlett

given: Nancy L

family: Chen

full_name: Robert Chen

given: Robert

family: Fanale

full_name: Michelle A Fanale

given: Michelle A

family: Brice

full_name: Pauline Brice

given: Pauline

family: Gopal

full_name: Ajay Gopal

given: Ajay

family: Smith

full_name: Scott E Smith

given: Scott E

family: Advani

full_name: Ranjana Advani

given: Ranjana

family: Matous

full_name: Jeffrey V Matous

given: Jeffrey V

family: Ramchandren

full_name: Radhakrishnan Ramchandren

given: Radhakrishnan

family: Rosenblatt

full_name: Joseph D Rosenblatt

given: Joseph D

family: Huebner

full_name: Dirk Huebner

given: Dirk

family: Levine

full_name: Pamela Levine

given: Pamela

family: Grove

full_name: Laurie Grove

given: Laurie

family: Forero-Torres

full_name: Andres Forero-Torres

given: Andres

currency_apc_doaj: GBP

journal_name: Journal of Hematology & Oncology

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer

publisher_group: Springer

publisher_normalized: Springer

ReferenceCitation: Gopal AK, Ramchandren R, O'Connor OA, Berryman RB, Advani RH, Chen R, Smith SE, Cooper M, Rothe A, Matous JV, Grove LE, Zain J. Safety and efficacy of brentuximab vedotin for Hodgkin lymphoma recurring after allogeneic stem cell transplantation. Blood. 2012 Jul 19;120(3):560-8. doi: 10.1182/blood-2011-12-397893. Epub 2012 Apr 17.

full_name: Ajay K. Gopal

given: Ajay K.

name: Center for Lymphoid Malignancies, Columbia University Medical Center, New York, NY;

family: O'Connor

name: Baylor University Medical Center, Dallas, TX;

name: Stanford University Medical Center, Palo Alto, CA;

name: City of Hope National Medical Center, Duarte, CA;

name: St Francis Hospital and Medical Center, Beech Grove, IN;

name: University Hospital of Cologne, Cologne, Germany;

name: Colorado Blood Cancer Institute, Denver, CO;

name: New York University Langone Medical Center, New York, NY

location_facility: The John Theurer Cancer Center, Hackensack University Medical Center

location_facility: Seattle Cancer Care Alliance / University of Washington Medical Center

location_facility: MD Anderson Cancer Center /The University of Texas

location_facility: Washington University School of Medicine

location_facility: Loyola University Medical Center - Cardinal Bernadin Cancer Center

location_facility: University of Miami Miller School of Medicine / Sylvester Comprehensive Cancer Center

location_facility: Karmanos Cancer Institute / Wayne State University

location_facility: St. Francis Medical Group Oncology & Hematology Specialists

title: Treatment With SGN-35 in Patients With CD30-positive Hematologic Malignancies Who Have Previously Participated in an SGN-35 Study

summary: This is a multicenter, open-label study to evaluate the safety and efficacy of treatment with brentuximab vedotin (SGN-35) in patients who have previously participated in an brentuximab vedotin study.


title: A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat (LBH589) Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML).

summary: The purpose of this randomized, two-arm, open-label expansion phase study was to collect preliminary efficacy data of panobinostat at the recommended phase II dose (RPIID) level in combination with azacytidine (5-Aza) versus an active control arm 5-Aza alone. This randomized phase II part also allowed collecting safety data of panobinostat in combination with 5-Aza in comparison to single-agent 5-aza.

ReferenceCitation: Garcia-Manero G, Sekeres MA, Egyed M, Breccia M, Graux C, Cavenagh JD, Salman H, Illes A, Fenaux P, DeAngelo DJ, Stauder R, Yee K, Zhu N, Lee JH, Valcarcel D, MacWhannell A, Borbenyi Z, Gazi L, Acharyya S, Ide S, Marker M, Ottmann OG. A phase 1b/2b multicenter study of oral panobinostat plus azacitidine in adults with MDS, CMML or AML with ⩽30% blasts. Leukemia. 2017 Dec;31(12):2799-2806. doi: 10.1038/leu.2017.159. Epub 2017 May 26.

family: Garcia-Manero

full_name: G Garcia-Manero

given: G

family: Sekeres

full_name: M A Sekeres

given: M A

family: Egyed

full_name: M Egyed

given: M

family: Breccia

full_name: M Breccia

family: Graux

full_name: C Graux

given: C

family: Cavenagh

full_name: J D Cavenagh

given: J D

family: Salman

full_name: H Salman

given: H

family: Illes

full_name: A Illes

given: A

family: Fenaux

full_name: P Fenaux

given: P

family: DeAngelo

full_name: D J DeAngelo

given: D J

family: Stauder

full_name: R Stauder

given: R

family: Yee

full_name: K Yee

given: K

family: Zhu

full_name: N Zhu

given: N

family: Lee

full_name: J-H Lee

given: J-H

family: Valcarcel

full_name: D Valcarcel

given: D

family: MacWhannell

full_name: A MacWhannell

family: Borbenyi

full_name: Z Borbenyi

given: Z

family: Gazi

full_name: L Gazi

given: L

family: Acharyya

full_name: S Acharyya

given: S

family: Ide

full_name: S Ide

family: Marker

full_name: M Marker

family: Ottmann

full_name: O G Ottmann

given: O G

repository_institution: University of Szeged - SZTE Publicatio Repozitórium - SZTE - Repository of Publications

repository_institution: Cardiff University - ORCA Online Research @Cardiff

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

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

location_country:
Italy
Austria
United States
Sweden
Switzerland
Germany
France
Thailand
Hungary
Belgium
Spain
Canada
United Kingdom
Korea, Republic of

location_facility: Dana Farber Cancer Institute Beth Israel Deaconess Med Ctr

location_facility: University of Texas MD Anderson Cancer Center Dept of MD Anderson (16)

location_facility: Cleveland Clinic Foundation Cleve Clinic

location_facility: University of Kansas Hospital and Medical Center SC - Univ KS

location_facility: Medical University of South Carolina -Hollings Cancer Center MUSC

location_facility: Georgia Health Sciences University Dept. of MCG

location_facility: Goshen Center for Cancer Care IU Cancer Center

location_facility: Memorial Sloan Kettering Sloan Kettering 2


ReferenceCitation: Lukina E, Watman N, Arreguin EA, Banikazemi M, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Rosenthal DI, Kaper M, Singh T, Puga AC, Bonate PL, Peterschmitt MJ. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood. 2010 Aug 12;116(6):893-9. doi: 10.1182/blood-2010-03-273151. Epub 2010 May 3.

ReferenceCitation: Lukina E, Watman N, Arreguin EA, Dragosky M, Iastrebner M, Rosenbaum H, Phillips M, Pastores GM, Kamath RS, Rosenthal DI, Kaper M, Singh T, Puga AC, Peterschmitt MJ. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood. 2010 Nov 18;116(20):4095-8. doi: 10.1182/blood-2010-06-293902. Epub 2010 Aug 16. Erratum in: Blood. 2011 May 19;117(20):5551.

ReferenceCitation: McEachern KA, Fung J, Komarnitsky S, Siegel CS, Chuang WL, Hutto E, Shayman JA, Grabowski GA, Aerts JM, Cheng SH, Copeland DP, Marshall J. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol Genet Metab. 2007 Jul;91(3):259-67. Epub 2007 May 16.

ReferenceCitation: Kamath RS, Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Aguzzi R, Puga AC, Norfleet AM, Peterschmitt MJ, Rosenthal DI. Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. Skeletal Radiol. 2014 Oct;43(10):1353-60. doi: 10.1007/s00256-014-1891-9. Epub 2014 May 10.

ReferenceCitation: Lukina E, Watman N, Dragosky M, Pastores GM, Arreguin EA, Rosenbaum H, Zimran A, Angell J, Ross L, Puga AC, Peterschmitt JM. Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment. Blood Cells Mol Dis. 2014 Dec;53(4):274-6. doi: 10.1016/j.bcmd.2014.04.002. Epub 2014 May 15.

ReferenceCitation: Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Goker-Alpan O, Watman N, El-Beshlawy A, Kishnani PS, Pedroso ML, Gaemers SJM, Tayag R, Peterschmitt MJ. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017 Apr 27;129(17):2375-2383. doi: 10.1182/blood-2016-12-758409. Epub 2017 Feb 6.

name: Department of Medicine, University of Cambridge, Addenbrooke’s Hospital, Cambridge, United Kingdom;

family: Cox

full_name: Timothy M. Cox

given: Timothy M.

name: Hospital de Niños Ricardo Gutiérrez, Buenos Aires, Argentina;

family: Drelichman

full_name: Guillermo Drelichman

given: Guillermo

name: State Institute of Haematology “Arthur de Siqueira Cavalcanti,” Rio de Janeiro, Brazil;

family: Cravo

full_name: Renata Cravo

given: Renata

family: Balwani

full_name: Manisha Balwani

given: Manisha

name: Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;

family: Burrow

full_name: Thomas Andrew Burrow

given: Thomas Andrew

name: Universidade Federal de São Paulo, São Paulo, Brazil;

family: Martins

full_name: Ana Maria Martins

given: Ana Maria

name: National Research Center for Hematology, Moscow, Russia;

family: Lukina

full_name: Elena Lukina

given: Elena

name: Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, CA;

family: Rosenbloom

full_name: Barry Rosenbloom

given: Barry

name: Lysosomal Disorders Unit, O&O Alpan, LLC, Fairfax, VA;

family: Goker-Alpan

full_name: Ozlem Goker-Alpan

given: Ozlem

name: Hospital Ramos Mejia, Buenos Aires, Argentina;

family: Watman

full_name: Nora Watman

given: Nora

name: Pediatric Hematology, Cairo University, Cairo, Egypt;

family: El-Beshlawy

full_name: Amal El-Beshlawy

given: Amal

name: Department of Pediatrics, Duke University School of Medicine, Durham, NC;

family: Kishnani

full_name: Priya S. Kishnani

given: Priya S.

name: Gastroenterology Department, Universidade Federal do Paraná, Curitiba, Brazil;

family: Pedroso

full_name: Maria Lucia Pedroso

given: Maria Lucia

family: Gaemers

full_name: Sebastiaan J. M. Gaemers

given: Sebastiaan J. M.

family: Tayag

full_name: Regina Tayag

given: Regina

family: Peterschmitt

full_name: M. Judith Peterschmitt

given: M. Judith

evidence: open (via free article)

repository_institution: University of Cambridge - Apollo

repositories_institution:
PubMed Central - Europe PMC
University of Cambridge - Apollo

ReferenceCitation: Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Ross L, Angell J, Puga AC. Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial. Lancet. 2015 Jun 13;385(9985):2355-62. doi: 10.1016/S0140-6736(14)61841-9. Epub 2015 Mar 26. Erratum in: Lancet. 2015 Jun 13;385(9985):2354.

location_country:
Italy
Brazil
United States
Spain
Germany
France
Argentina
Australia
Egypt
Russian Federation
Canada
United Kingdom

location_facility: Hematology Research Center of Ministry of Healthcare of the Russian Federation

location_facility: Hospital de Clinicas da Universidade Federal do Parana

location_facility: Northwest Oncology Hematology Associates PA

location_facility: Katholische Kliniken Oberhausen gem. GmbH

location_facility: Instituto de Estadual de Hematologia Arthur de Siqueria Cavalcanti

location_facility: Hospital General de Agudos J.M Ramos Mejia

location_facility: Azienda Ospedaliero Universitaria Careggi

location_facility: Cincinnati Children's Hospital Medical Center

location_facility: Mount Sinai Hospital and the Samuel Lunenfeld Research Institute

location_facility: University of Iowa Hospitals and Clinics

location_facility: Cambridge University Hosptials, Addenbrookes Hospital

location_facility: University of Colorado Health Science Center - Aurora

location_facility: Hospital General de Ninos Dr. Ricardo Gutierrez

location_facility: Abou El Reesh Children's University Hospital (El Mounira), Faculty of Medicine (Kasr Al-Aini), Cairo University Hospitals, El Mounira, Cairo, Egypt

location_facility: Azienda Ospedialiero-Universitaria S. Maria Della Misericordia

title: A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

summary: This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had reached therapeutic goals with enzyme replacement therapy (ERT).


lead_sponsor: Astellas Pharma Inc

location_country:
Malaysia
Australia
Russian Federation
Czech Republic
Ukraine
India
Thailand
France
Japan
Poland
Slovakia
Korea, Republic of
South Africa
Netherlands
Philippines
Bulgaria
Hungary
Germany
United Kingdom
Austria
Israel
Spain
Estonia

title: A Phase 2, Double-Blind, Double-Dummy, Randomized, Parallel Group Dose Finding Study To Investigate the Safety and Tolerability of YM150 in Subjects With Non-Valvular Atrial Fibrillation and to Compare the Safety and Tolerability With Warfarin

summary: The purpose of this study is to investigate the optimal daily dose and dose regimen of YM150 in subjects with non-valvular atrial fibrillation (NVAF), primarily based on safety and tolerability data.


title: A Study Assessing the REG1 Anticoagulation System Compared Heparin in Subjects With Acute Coronary Syndrome

acronym: RADAR

summary: The purpose of this study is to evaluate the safety and efficacy of the REG1 anticoagulation System in Acute Coronary Syndrome (ACS) patients undergoing cardiac catheterization.

ReferenceCitation: TCT-176: Access and non-Access Site Bleeding in Acute Coronary Syndrome Patients Treated with a Novel Actively Reversible Factor IXa Inhibitor. Results from the RADAR Trial J Am Coll Cardiol 2011 58: B47

ReferenceCitation: Park EJ, Choi J, Lee KC, Na DH. Emerging PEGylated non-biologic drugs. Expert Opin Emerg Drugs. 2019 Jun;24(2):107-119. doi: 10.1080/14728214.2019.1604684. Epub 2019 Apr 19. Review.

name: College of Pharmacy, Chung-Ang University, Seoul, Republic of Korea

name: D&D Pharmatech, Seongnam, Republic of Korea

family: Park

full_name: Eun Ji Park

given: Eun Ji

family: Choi

full_name: Jiyoung Choi

given: Jiyoung

name: College of Pharmacy, SungKyunKwan University, Suwon, Republic of Korea

family: Lee

full_name: Kang Choon Lee

given: Kang Choon

full_name: Dong Hee Na

given: Dong Hee

journal_name: Expert Opinion on Emerging Drugs

publisher: Informa UK Limited

publisher_dissemination: Informa

publisher_group: Informa

publisher_normalized: Informa

title: Emerging PEGylated non-biologic drugs

ReferenceCitation: Povsic TJ, Vavalle JP, Alexander JH, Aberle LH, Zelenkofske SL, Becker RC, Buller CE, Cohen MG, Cornel JH, Kasprzak JD, Montalescot G, Fail PS, Sarembock IJ, Mehran R; RADAR Investigators. Use of the REG1 anticoagulation system in patients with acute coronary syndromes undergoing percutaneous coronary intervention: results from the phase II RADAR-PCI study. EuroIntervention. 2014 Aug;10(4):431-8. doi: 10.4244/EIJY14M06_01.

family: Povsic

full_name: Thomas J. Povsic

given: Thomas J.

family: Vavalle

full_name: John P. Vavalle

given: John P.

family: Alexander

full_name: John H. Alexander

given: John H.

family: Aberle

full_name: Laura H. Aberle

given: Laura H.

family: Zelenkofske

full_name: Steven L. Zelenkofske

given: Steven L.

family: Becker

full_name: Richard C. Becker

given: Richard C.

family: Buller

full_name: Christopher E. Buller

given: Christopher E.

family: Cohen

full_name: Mauricio G. Cohen

given: Mauricio G.

family: Cornel

full_name: Jan H. Cornel

given: Jan H.

family: Kasprzak

full_name: Jaroslaw D. Kasprzak

given: Jaroslaw D.

family: Montalescot

full_name: Gilles Montalescot

given: Gilles

family: Fail

full_name: Peter S. Fail

given: Peter S.

family: Sarembock

full_name: Ian J. Sarembock

given: Ian J.

family: Mehran

full_name: Roxana Mehran

given: Roxana

journal_name: EuroIntervention

publisher: Europa Digital & Publishing

publisher_dissemination: Europa Digital & Publishing

publisher_group: Europa Digital & Publishing

publisher_normalized: Europa Digital & Publishing

ReferenceCitation: Povsic TJ, Cohen MG, Chan MY, Zelenkofske SL, Wargin WA, Harrington RA, Alexander JH, Rusconi CP, Becker RC. Dose selection for a direct and selective factor IXa inhibitor and its complementary reversal agent: translating pharmacokinetic and pharmacodynamic properties of the REG1 system to clinical trial design. J Thromb Thrombolysis. 2011 Jul;32(1):21-31. doi: 10.1007/s11239-011-0588-3.

title: Dose Selection for a Direct and Selective Factor IXa Inhibitor and its Complementary Reversal Agent: Translating Pharmacokinetic and Pharmacodynamic Properties of the REG1 System to Clinical Trial Design

ReferenceCitation: Povsic TJ, Cohen MG, Mehran R, Buller CE, Bode C, Cornel JH, Kasprzak JD, Montalescot G, Joseph D, Wargin WA, Rusconi CP, Zelenkofske SL, Becker RC, Alexander JH. A randomized, partially blinded, multicenter, active-controlled, dose-ranging study assessing the safety, efficacy, and pharmacodynamics of the REG1 anticoagulation system in patients with acute coronary syndromes: design and rationale of the RADAR Phase IIb trial. Am Heart J. 2011 Feb;161(2):261-268.e1-2. doi: 10.1016/j.ahj.2010.10.022.

title: A randomized, partially blinded, multicenter, active-controlled, dose-ranging study assessing the safety, efficacy, and pharmacodynamics of the REG1 anticoagulation system in patients with acute coronary syndromes: Design and rationale of the RADAR Phase IIb trial

location_facility: University of Florida-Medicine Cardiology Research

location_facility: Universitaetsklinikum Halle/Saale Klinik fÃ¼r Innere Medizin III

location_facility: Klinikum Fulda gAG Medizinische Klinik I

location_facility: Klinikum Quedlinburg GmbH Klinik fuer Innere Medizin und Kardiologie

location_facility: University of North Carolina - School of Medicine

location_facility: Klinikum der Universitaet Muenchen GroÃŸhadern Medizinische Klinik und Poliklinik I der LMU

location_facility: MVZ am Kuechwald GmbH Ambulantes Herzzentrum

location_facility: Klinikum der Stadt Ludwigshafen am Rhein gGmbH

location_facility: Jim Moran Heart & Vascular Research Institute

location_facility: Universitatsklinikum Freiburg Abt. Innere Medizin III

location_facility: Universitaetsklinik Magdeburg Klinik fÃ¼r Kardiologie Angiologie und Pneumologie

location_facility: Cardiovascular Institute of the South - Houma

location_facility: University of Pennsylvania Health System

location_facility: Montreal Heart Institute Research Center

location_facility: Klinikum Coburg GmbH 2. Med. Klinik Ebene 3 Studienbuero


title: A Randomised Open-label Study Comparing the Safety and Efficacy of Ritonavir Boosted Lopinavir and 2-3N(t)RTI Backbone Versus Ritonavir Boosted Lopinavir and Raltegravir in Participants Virologically Failing First-line NNRTI/2N(t)RTI Therapy

acronym: SECOND-LINE

summary:
The investigators hypothesize that following virological failure of a standard NNRTI+2N(T)RTI regimen second-line antiretroviral therapy consisting of ritonavir-boosted lopinavir and 2N(T)RTIs will offer comparable efficacy to that provided by ritonavir-boosted lopinavir and raltegravir.

The study will be conducted for 96-weeks with the primary endpoint analyzed after 48-weeks.

The primary endpoint is virological: a comparison of virological suppression in plasma < 200 copies/mL between the randomized arms after 48 weeks.

Secondary and exploratory endpoints include virological, immunological, safety, clinical, metabolic, drug adherence, drug resistance and quality of life.

ReferenceCitation: Henry RT, Jiamsakul A, Law M, Losso M, Kamarulzaman A, Phanuphak P, Kumarasamy N, Foulkes S, Mohapi L, Nwizu C, Wood R, Kelleher A, Polizzotto M; SECOND-LINE Study Group. Factors Associated With and Characteristic of HIV/Tuberculosis Co-Infection: A Retrospective Analysis of SECOND-LINE Clinical Trial Participants. J Acquir Immune Defic Syndr. 2021 May 1;87(1):720-729. doi: 10.1097/QAI.0000000000002619.

family: Henry

full_name: Rebecca T. Henry

given: Rebecca T.

family: Jiamsakul

full_name: Awachana Jiamsakul

given: Awachana

family: Law

full_name: Matthew Law

given: Matthew

family: Losso

full_name: Marcelo Losso

given: Marcelo

family: Kamarulzaman

full_name: Adeeba Kamarulzaman

given: Adeeba

family: Phanuphak

full_name: Praphan Phanuphak

given: Praphan

family: Kumarasamy

full_name: Nagalingeswaran Kumarasamy

given: Nagalingeswaran

family: Foulkes

full_name: Sharne Foulkes

given: Sharne

family: Mohapi

full_name: Lerato Mohapi

given: Lerato

family: Nwizu

full_name: Chidi Nwizu

given: Chidi

family: Wood

full_name: Robin Wood

given: Robin

family: Kelleher

full_name: Anthony Kelleher

given: Anthony

family: Polizzotto

full_name: Mark Polizzotto

given: Mark

journal_name: JAIDS Journal of Acquired Immune Deficiency Syndromes

ReferenceCitation: Amin J, Boyd MA, Kumarasamy N, Moore CL, Losso MH, Nwizu CA, Mohapi L, Kerr SJ, Sohn AH, Teppler H, Renjifo B, Molina JM, Emery S, Cooper DA. Raltegravir non-inferior to nucleoside based regimens in second-line therapy with lopinavir/ritonavir over 96 weeks: a randomised open label study for the treatment of HIV-1 infection. PLoS One. 2015 Feb 27;10(2):e0118228. doi: 10.1371/journal.pone.0118228. eCollection 2015. Erratum in: PLoS One. 2015;10(10):e0140623.

family: Amin

full_name: Janaki Amin

given: Janaki

family: Boyd

full_name: Mark A. Boyd

given: Mark A.

family: Moore

full_name: Cecilia L. Moore

given: Cecilia L.

full_name: Marcello H. Losso

given: Marcello H.

full_name: Chidi A. Nwizu

given: Chidi A.

family: Kerr

full_name: Stephen J. Kerr

given: Stephen J.

repository_institution: The University of Queensland - Queensland's institutional digital repository

title: Raltegravir Non-Inferior to Nucleoside Based Regimens in SECOND-LINE Therapy with Lopinavir/Ritonavir over 96 Weeks: A Randomised Open Label Study for the Treatment Of HIV-1 Infection

ReferenceCitation: Martin A, Moore CL, Mallon PW, Hoy JF, Emery S, Belloso WH, Phanuphak P, Ferret S, Cooper DA, Boyd MA; Second-Line Study Team. HIV lipodystrophy in participants randomised to lopinavir/ritonavir (LPV/r) +2-3 nucleoside/nucleotide reverse transcriptase inhibitors (N(t)RTI) or LPV/r + raltegravir as second-line antiretroviral therapy. PLoS One. 2013 Oct 30;8(10):e77138. doi: 10.1371/journal.pone.0077138. eCollection 2013.

repository_institution: University of Adelaide - Adelaide Research & Scholarship (AR&S)

repository_institution: University of Wollongong - Research Online

repository_institution: Monash University - Monash University Research Portal

title: HIV Lipodystrophy in Participants Randomised to Lopinavir/Ritonavir (LPV/r) +2–3 Nucleoside/Nucleotide Reverse Transcriptase Inhibitors (N(t)RTI) or LPV/r + Raltegravir as Second-Line Antiretroviral Therapy

ReferenceCitation: SECOND-LINE Study Group, Boyd MA, Kumarasamy N, Moore CL, Nwizu C, Losso MH, Mohapi L, Martin A, Kerr S, Sohn AH, Teppler H, Van de Steen O, Molina JM, Emery S, Cooper DA. Ritonavir-boosted lopinavir plus nucleoside or nucleotide reverse transcriptase inhibitors versus ritonavir-boosted lopinavir plus raltegravir for treatment of HIV-1 infection in adults with virological failure of a standard first-line ART regimen (SECOND-LINE): a randomised, open-label, non-inferiority study. Lancet. 2013 Jun 15;381(9883):2091-9. doi: 10.1016/S0140-6736(13)61164-2.

location_country:
South Africa
Chile
Peru
India
Hong Kong
Mexico
France
Malaysia
Singapore
Ireland
Argentina
Australia
New Zealand
Nigeria
Germany
United Kingdom
Taiwan

location_facility: Hospital de la Universidad Catolica Pontificia

location_facility: Hospital Interzonal General de Agudos, Oscar Alende

location_facility: Instituto de Medicina Tropical Alexander von Humboldt, Universidad Peruana Cayetano Heredia

location_facility: Hospital General de Agudos 'Teodoro Alvarez'

location_facility: Instituto Nacional de Ciencias Medicas y Nutricion "Salvado Zubiran"


title: Attain Ability® Model 4196 Left Ventricular Lead Chronic Performance Post Approval Study

summary: The purpose of this study is to evaluate long-term performance of the 4196 LV Lead. This evaluation is based on the number of lead-related complications occurring during the study compared to the number of leads enrolled in the study. The leads will be followed for 5 years after implant. This study is required by FDA as a condition of approval of the Model 4196 LV Lead and is integrated within the Product Surveillance Registry (PAN Registry).

lead_sponsor: Medtronic

location_country:
Italy
Austria
United States
Netherlands
France
Belgium
Spain
Canada


title: A Prospective Multicenter Double Blind Randomized Controlled Trial to Explore the Tolerability, Safety and Efficacy of the H-Coil Deep Transcranial Magnetic Stimulation (TMS) in Subjects With Major Depression Disorder (MDD)

summary: The purpose of the study is to evaluate the efficacy and safety of deep brain rTMS, (Transcranial Magnetic Stimulation), a new experimental procedure using the H-Coil, in subjects with Major Depressive Disorder that have been previously unsuccessfully treated with antidepressant medications.

lead_sponsor: Brainsway

location_country:
United States
France
Israel
Germany
Canada

location_facility: Columbia University / New York State Psychiatric Institute

location_facility: Advanced Mental Health Care Inc. - Juno Beach

location_facility: Duke Medical Center Department of Psychiatry & Behavioral Sciences

location_facility: UT Southwestern Medical Center at Dallas

location_facility: Klinik für Psychiatrie und Psychotherapie, Ludwig-Maximilians-Universität

location_facility: Universitätsklinikum Bonn, Klinik und Poliklinik für Psychiatrie und Psychotherapie

location_facility: Advanced Mental Health Care Inc. - Royal Palm Beach

location_facility: Center for Addiction and Mental Health (CAMH)


ReferenceCitation: Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease. Genet Med. 2021 Jan;23(1):192-201. doi: 10.1038/s41436-020-00968-z. Epub 2020 Sep 30. Erratum in: Genet Med. 2020 Nov 20;:.

family: Bichet

full_name: Daniel G. Bichet

given: Daniel G.

family: Aerts

full_name: Johannes M. Aerts

given: Johannes M.

family: Auray-Blais

full_name: Christiane Auray-Blais

given: Christiane

family: Maruyama

full_name: Hiroki Maruyama

given: Hiroki

family: Mehta

full_name: Atul B. Mehta

given: Atul B.

family: Skuban

full_name: Nina Skuban

given: Nina

family: Krusinska

full_name: Eva Krusinska

given: Eva

family: Schiffmann

full_name: Raphael Schiffmann

given: Raphael

journal_name: Genetics in Medicine

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

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

title: Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

ReferenceCitation: Germain DP, Nicholls K, Giugliani R, Bichet DG, Hughes DA, Barisoni LM, Colvin RB, Jennette JC, Skuban N, Castelli JP, Benjamin E, Barth JA, Viereck C. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study. Genet Med. 2019 Sep;21(9):1987-1997. doi: 10.1038/s41436-019-0451-z. Epub 2019 Feb 6.

family: Germain

full_name: Dominique P. Germain

given: Dominique P.

family: Nicholls

full_name: Kathy Nicholls

given: Kathy

family: Giugliani

full_name: Roberto Giugliani

given: Roberto

family: Hughes

full_name: Derralynn A. Hughes

given: Derralynn A.

family: Barisoni

full_name: Laura M. Barisoni

given: Laura M.

family: Colvin

full_name: Robert B. Colvin

given: Robert B.

family: Jennette

full_name: J. Charles Jennette

given: J. Charles

family: Castelli

full_name: Jeffrey P. Castelli

given: Jeffrey P.

repository_institution: University of Melbourne - Minerva Access

repository_institution: University College London - UCL Discovery

repository_institution: Universidade Federal do Rio Grande do Sul - Lume

ReferenceCitation: Schiffmann R, Bichet DG, Jovanovic A, Hughes DA, Giugliani R, Feldt-Rasmussen U, Shankar SP, Barisoni L, Colvin RB, Jennette JC, Holdbrook F, Mulberg A, Castelli JP, Skuban N, Barth JA, Nicholls K. Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial. Orphanet J Rare Dis. 2018 Apr 27;13(1):68. doi: 10.1186/s13023-018-0813-7.

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

repository_institution: University of Copenhagen - Research at the University of Copenhagen

repository_institution: California Digital Library - eScholarship

repository_institution: Red Federada de Repositorios Institucionales de Publicaciones Científicas - LA Referencia

repositories_institution: Harvard University - Digital Access to Scholarship at Harvard (DASH)

ReferenceCitation: Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22.

ReferenceCitation: Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.

title: Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

location_country:
Italy
Brazil
United States
France
Poland
Argentina
Australia
Egypt
Spain
Turkey
Denmark
Canada
United Kingdom

acronym: Study of the Effects of Oral AT1001 in Patients with Fabry Disease

title: A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

summary: The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).


intervention_type: Biological

location_country:
Italy
Saudi Arabia
Lebanon
France
Egypt
United States
United Kingdom

title: A Phase 3, Open-Label, Single-Arm Trial Evaluating the Safety, Tolerability, and Immunogenicity of 13-valent Pneumococcal Conjugate Vaccine in Children With Sickle Cell Disease Previously Immunized With 23vPS Vaccine

summary: This study is to evaluate the safety, tolerability and immunogenicity of 13-valent pneumococcal Conjugate Vaccine in children with Sickle Cell Disease who have already been vaccinated with 23-valent polysaccharide vaccine. The study will measure the amount of antibodies (the proteins that fight off germs) produced by children with Sickle Cell Disease after they have been given the 13-valent pneumococcal vaccine between 6 and less than 18 years of age. They will be given the vaccination twice, each vaccination separated by approximately 6 months.


ReferenceCitation: Davies JC, Wainwright CE, Canny GJ, Chilvers MA, Howenstine MS, Munck A, Mainz JG, Rodriguez S, Li H, Yen K, Ordoñez CL, Ahrens R; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med. 2013 Jun 1;187(11):1219-25. doi: 10.1164/rccm.201301-0153OC.

family: Davies

full_name: Jane C. Davies

given: Jane C.

family: Wainwright

full_name: Claire E. Wainwright

given: Claire E.

family: Canny

full_name: Gerard J. Canny

given: Gerard J.

family: Chilvers

full_name: Mark A. Chilvers

given: Mark A.

family: Howenstine

full_name: Michelle S. Howenstine

given: Michelle S.

family: Munck

full_name: Anne Munck

given: Anne

family: Mainz

full_name: Jochen G. Mainz

given: Jochen G.

family: Rodriguez

full_name: Sally Rodriguez

given: Sally

family: Li

full_name: Haihong Li

given: Haihong

family: Yen

full_name: Karl Yen

given: Karl

family: Ordoñez

full_name: Claudia L. Ordoñez

given: Claudia L.

family: Ahrens

full_name: Richard Ahrens

given: Richard

journal_name: American Journal of Respiratory and Critical Care Medicine

oa_host_type: repository

repository_institution: PubMed Central - Europe PMC

publisher: American Thoracic Society

publisher_dissemination: American Thoracic Society

publisher_group: American Thoracic Society

publisher_normalized: American Thoracic Society

title: Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

location_country:
United States
Germany
France
Ireland
Australia
Canada
United Kingdom

acronym: ENVISION

location_facility: Hôpital Robert Debré - Service de gastro-entérologiemucoviscidose et nutrition

location_facility: East Tennessee Children's Hospital Pediatric Pulmonary and Respiratory Care

location_facility: Dept of Gene Therapy, Imperial College London

location_facility: Kinder- und Jugendklinik Universitätsklinikum Erlangen

location_facility: University of Virginia Pediatric Respiratory Medicine

location_facility: University of Nebraska Medical Center Pediatric Pulmonary/ CF

location_facility: University of Utah Pediatric Pulmonology

location_facility: Helen DeVos Children's Hospital Spectrum Health Hospitals

location_facility: University of Iowa Department of Pediatrics

location_facility: Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin

title: A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 to 11 Years With Cystic Fibrosis and the G551D Mutation

summary: The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.