title: Multi-centric Observational Study to Analyse the Diagnostic Molecular Features in the Clinical Setting of Kidney Allograft Biopsies

summary: The current standard for biopsy-based diagnoses of dysfunction of kidney transplants is the Banff Classification which represents arbitrary international consensus. Recent data-driven approaches using molecular and conventional technologies indicate that mere consensus produces frequently incorrect diagnoses with potential harm to patients due to inappropriate treatment. To address this unmet need and improve diagnostics in the area of organ transplantation, the Alberta Transplant Applied Genomics Centre (ATAGC) has developed a new diagnostic system that combines the molecular and histopathological features of transplant biopsies, plus clinical and laboratory parameters, to create the first Integrated Diagnostic System. The present study will validate and refine this system in 500 prospectively unselected biopsies for clinical indications from American, Canadian and European centres in addition to 300 biopsies already collected. Due to a considerable interest and support from participating Centers, the study is further extended to 1500 prospective biopsies. Thus this is the extension of the INTERCOM study (INTERCOMEX). In addition to demonstrating the feasibility and value of this System in routine patient care and clinical trials, the study will develop and optimize a transparent and user-friendly reporting format to communicate this information to clinicians and obtain detailed feedback on how this system can best improve patient care.

ReferenceCitation: Halloran PF, Famulski KS, Reeve J. Molecular assessment of disease states in kidney transplant biopsy samples. Nat Rev Nephrol. 2016 Sep;12(9):534-48. doi: 10.1038/nrneph.2016.85. Epub 2016 Jun 27.

ReferenceCitation: Einecke G, Reeve J, Gupta G, Bohmig GA, Eskandary F, Bromberg JS, Budde K, Halloran PF; INTERCOMEX investigators. Factors associated with kidney graft survival in pure antibody-mediated rejection at the time of indication biopsy: Importance of parenchymal injury but not disease activity. Am J Transplant. 2021 Apr;21(4):1391-1401. doi: 10.1111/ajt.16161. Epub 2020 Jul 31.

ReferenceCitation: Halloran PF, Pereira AB, Chang J, Matas A, Picton M, De Freitas D, Bromberg J, Seron D, Sellares J, Einecke G, Reeve J. Potential impact of microarray diagnosis of T cell-mediated rejection in kidney transplants: The INTERCOM study. Am J Transplant. 2013 Sep;13(9):2352-63. doi: 10.1111/ajt.12387. Epub 2013 Aug 5.

ReferenceCitation: Halloran PF, Pereira AB, Chang J, Matas A, Picton M, De Freitas D, Bromberg J, Seron D, Sellares J, Einecke G, Reeve J. Microarray diagnosis of antibody-mediated rejection in kidney transplant biopsies: an international prospective study (INTERCOM). Am J Transplant. 2013 Nov;13(11):2865-74. doi: 10.1111/ajt.12465. Epub 2013 Oct 3.

ReferenceCitation: Loupy A, Lefaucheur C, Vernerey D, Chang J, Hidalgo LG, Beuscart T, Verine J, Aubert O, Dubleumortier S, Duong van Huyen JP, Jouven X, Glotz D, Legendre C, Halloran PF. Molecular microscope strategy to improve risk stratification in early antibody-mediated kidney allograft rejection. J Am Soc Nephrol. 2014 Oct;25(10):2267-77. doi: 10.1681/ASN.2013111149. Epub 2014 Apr 3.

ReferenceCitation: Halloran PF, Chang J, Famulski K, Hidalgo LG, Salazar ID, Merino Lopez M, Matas A, Picton M, de Freitas D, Bromberg J, Seron D, Sellares J, Einecke G, Reeve J. Disappearance of T Cell-Mediated Rejection Despite Continued Antibody-Mediated Rejection in Late Kidney Transplant Recipients. J Am Soc Nephrol. 2015 Jul;26(7):1711-20. doi: 10.1681/ASN.2014060588. Epub 2014 Nov 6.

ReferenceCitation: Halloran PF, Merino Lopez M, Barreto Pereira A. Identifying Subphenotypes of Antibody-Mediated Rejection in Kidney Transplants. Am J Transplant. 2016 Mar;16(3):908-20. doi: 10.1111/ajt.13551. Epub 2016 Jan 6.

ReferenceCitation: Reeve J, Chang J, Salazar ID, Lopez MM, Halloran PF. Using Molecular Phenotyping to Guide Improvements in the Histologic Diagnosis of T Cell-Mediated Rejection. Am J Transplant. 2016 Apr;16(4):1183-92. doi: 10.1111/ajt.13572. Epub 2016 Jan 5.

ReferenceCitation: Eskandary F, Bond G, Kozakowski N, Regele H, Marinova L, Wahrmann M, Kikic Z, Haslacher H, Rasoul-Rockenschaub S, Kaltenecker CC, Konig F, Hidalgo LG, Oberbauer R, Halloran PF, Bohmig GA. Diagnostic Contribution of Donor-Specific Antibody Characteristics to Uncover Late Silent Antibody-Mediated Rejection-Results of a Cross-Sectional Screening Study. Transplantation. 2017 Mar;101(3):631-641. doi: 10.1097/TP.0000000000001195.

ReferenceCitation: Aubert O, Loupy A, Hidalgo L, Duong van Huyen JP, Higgins S, Viglietti D, Jouven X, Glotz D, Legendre C, Lefaucheur C, Halloran PF. Antibody-Mediated Rejection Due to Preexisting versus De Novo Donor-Specific Antibodies in Kidney Allograft Recipients. J Am Soc Nephrol. 2017 Jun;28(6):1912-1923. doi: 10.1681/ASN.2016070797. Epub 2017 Mar 2.

ReferenceCitation: Halloran PF, Reeve J, Akalin E, Aubert O, Bohmig GA, Brennan D, Bromberg J, Einecke G, Eskandary F, Gosset C, Duong Van Huyen JP, Gupta G, Lefaucheur C, Malone A, Mannon RB, Seron D, Sellares J, Weir M, Loupy A. Real Time Central Assessment of Kidney Transplant Indication Biopsies by Microarrays: The INTERCOMEX Study. Am J Transplant. 2017 Nov;17(11):2851-2862. doi: 10.1111/ajt.14329. Epub 2017 May 30.

ReferenceCitation: Reeve J, Bohmig GA, Eskandary F, Einecke G, Lefaucheur C, Loupy A, Halloran PF; MMDx-Kidney study group. Assessing rejection-related disease in kidney transplant biopsies based on archetypal analysis of molecular phenotypes. JCI Insight. 2017 Jun 15;2(12):e94197. doi: 10.1172/jci.insight.94197. eCollection 2017 Jun 15.

publisher_dissemination: American Society for Clinical Investigation

ReferenceCitation: Madill-Thomsen KS, Wiggins RC, Eskandary F, Bohmig GA, Halloran PF. The Effect of Cortex/Medulla Proportions on Molecular Diagnoses in Kidney Transplant Biopsies: Rejection and Injury Can Be Assessed in Medulla. Am J Transplant. 2017 Aug;17(8):2117-2128. doi: 10.1111/ajt.14233. Epub 2017 Mar 23.

repositories_institution:
PubMed Central - Europe PMC
University of Michigan - Deep Blue

ReferenceCitation: Halloran PF, Venner JM, Famulski KS. Comprehensive Analysis of Transcript Changes Associated With Allograft Rejection: Combining Universal and Selective Features. Am J Transplant. 2017 Jul;17(7):1754-1769. doi: 10.1111/ajt.14200. Epub 2017 Feb 25.

ReferenceCitation: Halloran PF, Matas A, Kasiske BL, Madill-Thomsen KS, Mackova M, Famulski KS. Molecular phenotype of kidney transplant indication biopsies with inflammation in scarred areas. Am J Transplant. 2019 May;19(5):1356-1370. doi: 10.1111/ajt.15178. Epub 2018 Dec 13.

ReferenceCitation: Reeve J, Bohmig GA, Eskandary F, Einecke G, Gupta G, Madill-Thomsen K, Mackova M, Halloran PF; INTERCOMEX MMDx-Kidney Study Group. Generating automated kidney transplant biopsy reports combining molecular measurements with ensembles of machine learning classifiers. Am J Transplant. 2019 Oct;19(10):2719-2731. doi: 10.1111/ajt.15351. Epub 2019 Apr 10. Erratum In: Am J Transplant. 2020 Feb;20(2):620-621.

ReferenceCitation: Madill-Thomsen K, Perkowska-Ptasinska A, Bohmig GA, Eskandary F, Einecke G, Gupta G, Halloran PF; MMDx-Kidney Study Group. Discrepancy analysis comparing molecular and histology diagnoses in kidney transplant biopsies. Am J Transplant. 2020 May;20(5):1341-1350. doi: 10.1111/ajt.15752. Epub 2020 Jan 23.

ReferenceCitation: Madill-Thomsen KS, Bohmig GA, Bromberg J, Einecke G, Eskandary F, Gupta G, Myslak M, Viklicky O, Perkowska-Ptasinska A, Solez K, Halloran PF; the INTERCOMEX Investigators. Relating Molecular T Cell-mediated Rejection Activity in Kidney Transplant Biopsies to Time and to Histologic Tubulitis and Atrophy-fibrosis. Transplantation. 2023 May 1;107(5):1102-1114. doi: 10.1097/TP.0000000000004396. Epub 2023 Apr 22.

ReferenceCitation: Halloran PF, Bohmig GA, Bromberg J, Einecke G, Eskandary FA, Gupta G, Myslak M, Viklicky O, Perkowska-Ptasinska A, Madill-Thomsen KS; INTERCOMEX Investigators. Archetypal Analysis of Injury in Kidney Transplant Biopsies Identifies Two Classes of Early AKI. Front Med (Lausanne). 2022 Apr 7;9:817324. doi: 10.3389/fmed.2022.817324. eCollection 2022.

ReferenceCitation: Sikosana MLN, Reeve J, Madill-Thomsen KS, Halloran PF; INTERCOMEX Investigators. Using Regression Equations to Enhance Interpretation of Histology Lesions of Kidney Transplant Rejection. Transplantation. 2023 Sep 20. doi: 10.1097/TP.0000000000004783. Online ahead of print.

ReferenceCitation: Halloran PF, Madill-Thomsen KS, Bohmig GA, Myslak M, Gupta G, Kumar D, Viklicky O, Perkowska-Ptasinska A, Famulski KS; INTERCOMEX Investigators. A 2-fold Approach to Polyoma Virus (BK) Nephropathy in Kidney Transplants: Distinguishing Direct Virus Effects From Cognate T Cell-mediated Inflammation. Transplantation. 2021 Nov 1;105(11):2374-2384. doi: 10.1097/TP.0000000000003884.

ReferenceCitation: Madill-Thomsen KS, Bohmig GA, Bromberg J, Einecke G, Eskandary F, Gupta G, Hidalgo LG, Myslak M, Viklicky O, Perkowska-Ptasinska A, Halloran PF; INTERCOMEX Investigators. Donor-Specific Antibody Is Associated with Increased Expression of Rejection Transcripts in Renal Transplant Biopsies Classified as No Rejection. J Am Soc Nephrol. 2021 Nov;32(11):2743-2758. doi: 10.1681/ASN.2021040433. Epub 2021 Jul 12.

ReferenceCitation: Halloran PF. Integrating molecular and histologic interpretation of transplant biopsies. Clin Transplant. 2021 Apr;35(4):e14244. doi: 10.1111/ctr.14244. Epub 2021 Feb 17. No abstract available.

ReferenceCitation: Halloran PF, Bohmig GA, Bromberg JS, Budde K, Gupta G, Einecke G, Eskandary F, Madill-Thomsen K, Reeve J; INTERCOMEX investigators. Discovering novel injury features in kidney transplant biopsies associated with TCMR and donor aging. Am J Transplant. 2021 May;21(5):1725-1739. doi: 10.1111/ajt.16374. Epub 2020 Nov 30.

ReferenceCitation: Venner JM, Famulski KS, Reeve J, Chang J, Halloran PF. Relationships among injury, fibrosis, and time in human kidney transplants. JCI Insight. 2016 Jan 21;1(1):e85323. doi: 10.1172/jci.insight.85323.

location_country:
Germany
Ireland
Poland
Slovenia
United Kingdom
Switzerland
Austria
United States
Korea, Republic of
Spain
Croatia
Czechia
Canada
France

location_facility: University of British Columbia, St. Paul's Hospital

location_facility: University of Wisconsin School of Medicine and Public Health

location_facility: Institute for Experimental and Clinical Medicine (IKEM)

location_facility: Department of Surgery, University of Usan, College of Medicine

location_facility: Department of Medicine, University of Alberta

location_facility: Pomeranian Medical University in Szczecin

location_facility: Texas Transplant Institute - Methodist Healthcare System

location_facility: University of Maryland School of Medicine

location_facility: Virginia Commonwealth University School of Medicine


type: EudraCT Number

title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Reslizumab (3.0 mg/kg) as Treatment for Patients With Eosinophilic Asthma Who Completed a Prior Teva-Sponsored Study in Eosinophilic Asthma

summary: The primary objective of the study is to evaluate the long-term safety of reslizumab at a dosage of 3.0 mg/kg every 4 weeks for approximately 24 months in pediatric and adult patients with eosinophilic asthma as assessed by adverse events, physical examination findings, vital sign measurements, and concomitant medication usage throughout the study (every 4 weeks), clinical laboratory test results, and measurement of antidrug antibodies.

status: Terminated

study_completion_date_type: Actual

study_type: Interventional

primary_purpose: Treatment

enrollment_type: Actual

lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

location_country:
Germany
Poland
New Zealand
Argentina
Australia
Brazil
Thailand
Chile
Canada
France
Czech Republic
Colombia
Slovakia
Hungary
Denmark
United States
Ukraine
Romania
Belgium
Malaysia
Peru
Philippines
Sweden
Netherlands
Mexico
Korea, Republic of
Greece
South Africa
Taiwan
Russian Federation
Israel

location_facility:
Teva Investigational Site 585
Teva Investigational Site 53
Teva Investigational Site 9
Teva Investigational Site 625
Teva Investigational Site 63
Teva Investigational Site 229
Teva Investigational Site 587
Teva Investigational Site 103
Teva Investigational Site 147
Teva Investigational Site 263
Teva Investigational Site 12
Teva Investigational Site 621
Teva Investigational Site 8
Teva Investigational Site 140
Teva Investigational Site 366
Teva Investigational Site 182
Teva Investigational Site 682
Teva Investigational Site 226
Teva Investigational Site 540
Teva Investigational Site 21
Teva Investigational Site 363
Teva Investigational Site 203
Teva Investigational Site 433
Teva Investigational Site 784
Teva Investigational Site 422
Teva Investigational Site 582
Teva Investigational Site 143
Teva Investigational Site 30
Teva Investigational Site 209
Teva Investigational Site 41
Teva Investigational Site 641
Teva Investigational Site 45
Teva Investigational Site 602
Teva Investigational Site 47
Teva Investigational Site 683
Teva Investigational Site 33
Teva Investigational Site 16
Teva Investigational Site 64
Teva Investigational Site 420
Teva Investigational Site 380
Teva Investigational Site 407
Teva Investigational Site 580
Teva Investigational Site 686
Teva Investigational Site 685
Teva Investigational Site 24
Teva Investigational Site 504
Teva Investigational Site 164
Teva Investigational Site 704
Teva Investigational Site 562
Teva Investigational Site 161
Teva Investigational Site 262
Teva Investigational Site 584
Teva Investigational Site 15
Teva Investigational Site 700
Teva Investigational Site 123
Teva Investigational Site 46
Teva Investigational Site 260
Teva Investigational Site 44
Teva Investigational Site 629
Teva Investigational Site 400
Teva Investigational Site 724
Teva Investigational Site 2
Teva Investigational Site 144
Teva Investigational Site 342
Teva Investigational Site 500
Teva Investigational Site 370
Teva Investigational Site 521
Teva Investigational Site 545
Teva Investigational Site 620
Teva Investigational Site 1
Teva Investigational Site 286
Teva Investigational Site 121
Teva Investigational Site 705
Teva Investigational Site 633
Teva Investigational Site 425
Teva Investigational Site 432
Teva Investigational Site 207
Teva Investigational Site 741
Teva Investigational Site 367
Teva Investigational Site 782
Teva Investigational Site 281
Teva Investigational Site 25
Teva Investigational Site 26
Teva Investigational Site 559
Teva Investigational Site 563
Teva Investigational Site 589
Teva Investigational Site 127
Teva Investigational Site 225
Teva Investigational Site 371
Teva Investigational Site 557
Teva Investigational Site 520
Teva Investigational Site 626
Teva Investigational Site 645
Teva Investigational Site 763
Teva Investigational Site 74
Teva Investigational Site 622
Teva Investigational Site 511
Teva Investigational Site 160
Teva Investigational Site 552
Teva Investigational Site 426
Teva Investigational Site 421
Teva Investigational Site 264
Teva Investigational Site 32
Teva Investigational Site 372
Teva Investigational Site 524
Teva Investigational Site 105
Teva Investigational Site 583
Teva Investigational Site 120
Teva Investigational Site 221
Teva Investigational Site 601
Teva Investigational Site 403
Teva Investigational Site 28
Teva Investigational Site 50
Teva Investigational Site 630
Teva Investigational Site 727
Teva Investigational Site 5
Teva Investigational Site 428
Teva Investigational Site 287
Teva Investigational Site 284
Teva Investigational Site 18
Teva Investigational Site 142
Teva Investigational Site 404
Teva Investigational Site 546
Teva Investigational Site 128
Teva Investigational Site 513
Teva Investigational Site 512
Teva Investigational Site 740
Teva Investigational Site 104
Teva Investigational Site 402
Teva Investigational Site 723
Teva Investigational Site 720
Teva Investigational Site 200
Teva Investigational Site 222
Teva Investigational Site 523
Teva Investigational Site 58
Teva Investigational Site 205
Teva Investigational Site 543
Teva Investigational Site 34
Teva Investigational Site 554
Teva Investigational Site 721
Teva Investigational Site 181
Teva Investigational Site 588
Teva Investigational Site 162
Teva Investigational Site 369
Teva Investigational Site 423
Teva Investigational Site 681
Teva Investigational Site 202
Teva Investigational Site 505
Teva Investigational Site 561
Teva Investigational Site 220
Teva Investigational Site 761
Teva Investigational Site 14
Teva Investigational Site 361
Teva Investigational Site 405
Teva Investigational Site 460
Teva Investigational Site 509
Teva Investigational Site 3
Teva Investigational Site 600
Teva Investigational Site 52
Teva Investigational Site 542
Teva Investigational Site 632
Teva Investigational Site 31
Teva Investigational Site 40
Teva Investigational Site 185
Teva Investigational Site 27
Teva Investigational Site 20
Teva Investigational Site 69
Teva Investigational Site 204
Teva Investigational Site 603
Teva Investigational Site 401
Teva Investigational Site 285
Teva Investigational Site 743
Teva Investigational Site 541
Teva Investigational Site 560
Teva Investigational Site 59
Teva Investigational Site 300
Teva Investigational Site 122
Teva Investigational Site 544
Teva Investigational Site 11
Teva Investigational Site 680
Teva Investigational Site 745
Teva Investigational Site 19
Teva Investigational Site 180
Teva Investigational Site 631
Teva Investigational Site 17
Teva Investigational Site 66
Teva Investigational Site 702
Teva Investigational Site 604
Teva Investigational Site 522
Teva Investigational Site 701
Teva Investigational Site 558
Teva Investigational Site 223
Teva Investigational Site 55
Teva Investigational Site 643
Teva Investigational Site 43
Teva Investigational Site 586
Teva Investigational Site 549
Teva Investigational Site 623
Teva Investigational Site 166
Teva Investigational Site 126
Teva Investigational Site 742
Teva Investigational Site 125
Teva Investigational Site 507
Teva Investigational Site 6
Teva Investigational Site 146
Teva Investigational Site 101
Teva Investigational Site 280
Teva Investigational Site 555
Teva Investigational Site 227
Teva Investigational Site 49
Teva Investigational Site 283
Teva Investigational Site 4
Teva Investigational Site 73
Teva Investigational Site 150
Teva Investigational Site 341
Teva Investigational Site 642
Teva Investigational Site 261
Teva Investigational Site 624
Teva Investigational Site 780

intervention_type: Drug

lead_sponsor_type: industriel

status_simplified: Unknown


title: The Multi-National Gilenya Pregnancy Exposure Registry in Multiple Sclerosis

summary: The purpose of the Multi-National Gilenya Pregnancy Exposure Registry in Multiple Sclerosis (MS) is to continuously monitor, evaluate, and assess for major and minor teratogenic effects in the offspring of women exposed to fingolimod before (up to 8 weeks before last menstrual period (LMP)) and during pregnancy in routine clinical practice. The overall aim is to collect and evaluate data on maternal, fetal, and infant outcomes and compare it with reference populations.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Prospective

enrollment_type: Anticipated

ipd_sharing: No

lead_sponsor: Novartis Pharmaceuticals

location_country:
Finland
Germany
Ireland
Poland
Argentina
Australia
Brazil
Czechia
Canada
France
Slovenia
Colombia
Portugal
Slovakia
Hungary
Denmark
Switzerland
United States
Romania
Lebanon
Belgium
Italy
Cyprus
Norway
United Kingdom
Sweden
Netherlands
Austria
Mexico
United Arab Emirates
Saudi Arabia
Spain
Greece
Russian Federation
Israel

status_simplified: Ongoing


lead_sponsor: BioMarin Pharmaceutical

summary:
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).

There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes.

This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.

ReferenceType: derived

ReferenceCitation: Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Elsevier

status: Completed

title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

type: Other Identifier

source: euctr

design_allocation: Randomized

location_country:
Germany
Italy
Colombia
Portugal
United Kingdom
Argentina
Denmark
Japan
Brazil
Netherlands
Qatar
United States
Korea, Republic of
Saudi Arabia
Taiwan
Canada
France

status_simplified: Completed


title: A Multicenter Longitudinal Study for Disease Profiling of Asthma and Chronic Obstructive Pulmonary Disease

summary: The purpose of this study is to characterize the clinical, physiologic, and molecular profiles of healthy participants, participants with mild, moderate, and severe asthma; and participants with moderate to severe Chronic Obstructive Pulmonary Disease (COPD).

primary_purpose: Basic Science

ReferenceCitation: Cass SP, Yang Y, Xiao J, McGrath JJC, Fantauzzi MF, Thayaparan D, Wang F, Liang Z, Long F, Stevenson CS, Chen R, Stampfli MR. Current smoking status is associated with reduced sputum immunoglobulin M and G expression in COPD. Eur Respir J. 2021 Feb 4;57(2):1902338. doi: 10.1183/13993003.02338-2019. Print 2021 Feb. No abstract available.

oa_host_type: publisher

evidence: open (via free pdf)

host_type: publisher

license_normalized: no license

version: publishedVersion

unpaywall_oa_status: bronze

publisher_dissemination: European Respiratory Society

ReferenceCitation: Loza MJ, Djukanovic R, Chung KF, Horowitz D, Ma K, Branigan P, Barnathan ES, Susulic VS, Silkoff PE, Sterk PJ, Baribaud F; ADEPT (Airways Disease Endotyping for Personalized Therapeutics) and U-BIOPRED (Unbiased Biomarkers for the Prediction of Respiratory Disease Outcome Consortium) investigators. Validated and longitudinally stable asthma phenotypes based on cluster analysis of the ADEPT study. Respir Res. 2016 Dec 15;17(1):165. doi: 10.1186/s12931-016-0482-9.

oa_colors: green gold

oa_host_type: publisher;repository

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

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

host_type: repository

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

repository_institution: Imperial College London - Spiral

repository_institution: Ghent University - Ghent University Academic Bibliography

repository_institution: University Library Bern - BORIS - Bern Open Repository and Information System

repository_institution: University of Zurich - Zurich Open Repository and Archive

evidence: oa repository (via pmcid lookup)

repository_institution: University of Southampton - ePrints Soton

version: acceptedVersion

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

lead_sponsor: Janssen Research & Development, LLC

location_country:
Germany
United Kingdom
Denmark
Romania
Netherlands
United States
Canada
France

intervention_type: Other


title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

summary: This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

ReferenceCitation: Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

publisher_dissemination: American Association for the Advancement of Science

location_country:
Germany
Italy
United Kingdom
Denmark
Australia
Switzerland
United States
Spain
Canada
France


title: A Phase 3, Open-label Study of the Safety and Effectiveness of AA4500 Administered Twice Per Treatment Cycle for up to Four Treatment Cycles (2 x 4) in Men With Peyronie's Disease

summary:
This study is a Phase 3, open-label study of the safety and efficacy of AA4500 0.58 mg in subjects with Peyronie's disease. Subjects will be screened for study eligibility within 21 days before the initial injection of study drug in the first treatment cycle.

After up to four treatment cycles, each subject will be followed for additional safety and efficacy assessments on Days 168 (± 7 days) and 252 (± 7 days) (nominal weeks 24 and 36).

After the final injection of each treatment cycle, the investigator will model the plaque in an attempt to stretch or elongate the plaque. If the subject's penile curvature is reduced to <15° after the first, second, or third cycle of injections or if the investigator determines further treatment is not clinically indicated (eg, adverse events; allergic reaction), subsequent treatment cycles will not be administered.

Approximately 300 subjects will be enrolled.

ReferenceCitation: Levine LA, Cuzin B, Mark S, Gelbard MK, Jones NA, Liu G, Kaufman GJ, Tursi JP, Ralph DJ. Clinical safety and effectiveness of collagenase clostridium histolyticum injection in patients with Peyronie's disease: a phase 3 open-label study. J Sex Med. 2015 Jan;12(1):248-58. doi: 10.1111/jsm.12731. Epub 2014 Nov 12.

publisher_dissemination: Oxford University Press

lead_sponsor: Endo Pharmaceuticals

location_country:
Germany
Italy
New Zealand
United Kingdom
Denmark
Sweden
United States
Spain
France

location_facility: Waitemata Urology Research North Shore Hospital

location_facility: Tristate Urologic Services PSC, Inc./ dba TUG Research

location_facility: Urology of Virginia-Sentara Medical Group

location_facility: Universitatsklinikum Freiburg, Abteilung Urologie

location_facility: Connecticut Clinical Research Center, LLC

location_facility: Aarhus Universitiy Hospital, Aalborg Sygehus

location_facility: Maimonides Medical Center, Division of Urology

intervention_type: Biological


title: An Open-label, Multicenter, Randomized Phase 2 Study Evaluating the Safety and Efficacy of Cisplatin and Pemetrexed With or Without Cixutumumab as First-Line Therapy in Patients With Advanced Nonsquamous Non-Small Cell Lung Carcinoma

summary: The primary purpose of this study is to evaluate the hypothesis that cixutumumab given in combination with cisplatin and pemetrexed is superior to cisplatin and pemetrexed as first-line therapy for patients with advanced nonsquamous non-small cell lung carcinoma (NSCLC).

ipd_sharing: Yes

ipd_sharing_description: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

lead_sponsor: Eli Lilly and Company

location_country:
Germany
Italy
Israel
Argentina
Brazil
Netherlands
Belgium
United States
Spain
Turkey
Canada
France

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


title: A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

summary: The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.

ReferenceCitation: Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

oa_host_type: repository

repositories_concat: PubMed Central

unpaywall_oa_status: green

publisher_dissemination: Wiley

ReferenceCitation: Rowe SM, McColley SA, Rietschel E, Li X, Bell SC, Konstan MW, Marigowda G, Waltz D, Boyle MP; VX09-809-102 Study Group. Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR. Ann Am Thorac Soc. 2017 Feb;14(2):213-219. doi: 10.1513/AnnalsATS.201609-689OC.

publisher_dissemination: American Thoracic Society

ReferenceCitation: Boyle MP, Bell SC, Konstan MW, McColley SA, Rowe SM, Rietschel E, Huang X, Waltz D, Patel NR, Rodman D; VX09-809-102 study group. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med. 2014 Jul;2(7):527-38. doi: 10.1016/S2213-2600(14)70132-8. Epub 2014 Jun 24.

lead_sponsor: Vertex Pharmaceuticals Incorporated

location_country:
Germany
New Zealand
United Kingdom
Australia
Belgium
United States
France


acronym: FUTURE 3

lead_sponsor: Actelion

summary: The primary objective of AC-052-373 was to assess the pharmacokinetic (PK) profile of two dosing regimens of the pediatric formulation of bosentan in children with pulmonary arterial hypertension (PAH) <12 years of age.

location_facility: CARE Hospitals, Cardiology Dep. Hyderabad - Site 5302

location_facility: Children's National Medical Center - Site 9104

location_facility: Cardiovascular Institute and Fuwai Hospital

location_facility: Universitätsklinikum Bonn Abteilung für Kinderkardiologie - Site 1404

location_facility: Justus-Liebig-Universität Giessen, Kinderherzzentrum - Site 1403

location_facility: RAMS Institution, Research Institute for complex issues of cardiovascular diseases, Siberian branch of the Russian Academy of Medical Sciences - Site 3805

location_facility: Wojewódzki Szpital Specjalistyczny we Wrocławiu Oddział Kardiologii Dziecięcej z pododdziałem Intensywnego Nadzoru Kardiologicznego - Site 3605

location_facility: Columbia University Medical Center Children's Hospital of New York Presbyterian - Site 9101

location_facility: Università Degli Studi di Padova - Dipartimento di Pediatria - Servizio di Cardiologia Pediatrica - Site 1501

location_facility: Szegedi Tudományegyetem ÁOK Szent-Györgyi Albert Klinikai Központ, Gyermekgyógyászati Klinika és Gyermekegészségügyi Központ - Site 3402

location_facility: Paediatric Cardiology Albert Luthuli Central Hospital - Site 6003

location_facility: Division of Paediatric Cardiology, Steve Biko Academic Hospital - Site 6002

location_facility: Moscow Scientific Research Institute for Pediatrics and Childrens Surgery of Rosmedtechnologies - Site 3804

location_facility: Clinical Diagnostic Center - Pediatric Cardiovascular and ANES and Intensive Care Department - Site 4103

location_facility: Universitair Medish Centrum Groningen, Kindercardiologie - Site 1601

location_facility: Gusak Ins Urgent and Recovery SUR AMS - Cardiovascular Rehabilitation Pediatric Department - Site 4101

location_facility: Uniwersyteckie Centrum Kliniczne Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca - Site 3604

location_facility: Deutsches Herzzentrum Kinderkardiologie - Site 1401

location_facility: Instituto Nacional de Cardiologia (INC) Ignacio Chavez - Site 8401

location_facility: State Educational Institution of Higher Professional Education "Saint Petersburg State Pediatric Medical Academy of Roszdrav" - Site 3801

location_facility: Shanghai Children's Medical Center - Site 5102

location_facility: Instytut Centrum Zdrowia Matki Polki Klinika Kardiologii ICMP w Lodz - Site 3602

location_facility: Hopital Necker-Enfants Malades, Service de Cardiologie Pédiatrique - Site 2201

location_facility: Institut za zdravstvenu zaštitu majke i deteta Srbije "Dr Vukan Čupić", Služba za ispitivanje i lečenje bolesti srca i krvnih sudova - Site 3902

location_facility: Schneider Children's Medical Center- Institute of pediatric cardiology - Site 7101

location_facility: Hospital Universitatario Vall d'Hebron, Neumologia - Site 1907

location_facility: Beijing Anzhen Hospital, Capital Medical University- Department of Pediatric Cardiology - Site 5103

location_facility: CHU de Toulouse - Hôpital des Enfants, Service de Cardiologie Pédiatrique - Site 2202

location_facility: Fakultní nemocnice v Motole, dětské kardiocentrum - Site 3301

location_facility: Univerzitetska dečja klinika, Služba za kardiologiju - Site 3901

location_facility: The Republican Scientific-Practical Center "Cardiology" - Site 3001

location_facility: Scientific Center of Cardiovascular Surgery named after A.N.Bakulev of the RAMS - Site 3803

location_facility: Texas Children's Hospital - Department of Cardiology - Site 9107

location_facility: Royal Children's Hospital Melbourne, Cardiology - Site 5001

location_facility: Indraprashta Apollo Hospitals, Pediatric Cardiology - Site 5303

location_facility: Gottsegen György Országos Kardiológiai Intézet, Gyermekszív Központ, Gyermek Kardiológiai osztály - Site 3401

location_facility: Federal State Institution "Federal center of Heart, Blood and Endocrinology named after V.A.Almazov Rosmedtekhnologies" - Site 3802

location_facility: Instytut Pomnik - Centrum Zdrowia Dziecka Klinika Kardiologii Dziecięcej - Site 3601

location_facility: Department of Paediatric Cardiology University of the Free State - Site 6001

location_facility: West China 2nd university Hospital-Center of interventional diagnosis and therapy for Children's cardiovascular disease - Site 5104

location_facility: Gover INS - Scientific Practical Cardiovascular Pediatric Center - MOH Ukraine - Site 4102

location_facility: Hospital Universitario La Paz - Paediatric Cardiology Department - Site 1906

location_facility: Shanghai Pulmonary Hospital, Department of Pulmonary Circulation - Site 5101

location_facility: Unidad de Investigacion Clinica en Medicina, SC (UDICEM) - Site 8402

location_facility: Ospedale Pediatrico "Bambino Gesù" - Dipartimento Medico Chirurgico di Cardiologia Pediatrica - Site 1502

title: An Open-label, Prospective Multicenter Study to Assess the Pharmacokinetics, Tolerability, Safety and Efficacy of the Pediatric Formulation of Bosentan Two Versus Three Times a Day in Children With Pulmonary Arterial Hypertension

location_country:
Germany
Poland
Australia
Czechia
Serbia
France
Belarus
Hungary
United States
Ukraine
India
China
Italy
Netherlands
Mexico
Spain
South Africa
Russian Federation
Israel


title: STA®-Liatest®D-Di-Exclusion of Venous Thromboembolism

acronym: DiET

summary: The purpose of this study is to demonstrate the ability of STA® Liatest® D-Di combined with a clinical pretest probability (PTP) to safely exclude pulmonary embolism (PE) or Deep Venous Thrombosis (DVT) in a 3 month follow-up.

ReferenceType: background

ReferenceCitation: Keeling DM, Mackie IJ, Moody A, Watson HG; Haemostasis and Thrombosis Task Force of the British Committee for Standards in Haematology. The diagnosis of deep vein thrombosis in symptomatic outpatients and the potential for clinical assessment and D-dimer assays to reduce the need for diagnostic imaging. Br J Haematol. 2004 Jan;124(1):15-25. doi: 10.1046/j.1365-2141.2003.04723.x. No abstract available.

ReferenceCitation: Goekoop RJ, Steeghs N, Niessen RW, Jonkers GJ, Dik H, Castel A, Werker-van Gelder L, Vlasveld LT, van Klink RC, Planken EV, Huisman MV. Simple and safe exclusion of pulmonary embolism in outpatients using quantitative D-dimer and Wells' simplified decision rule. Thromb Haemost. 2007 Jan;97(1):146-50.

ReferenceCitation: Kelly J, Hunt BJ. A clinical probability assessment and D-dimer measurement should be the initial step in the investigation of suspected venous thromboembolism. Chest. 2003 Sep;124(3):1116-9. No abstract available.

ReferenceCitation: Pasha SM, Klok FA, Snoep JD, Mos IC, Goekoop RJ, Rodger MA, Huisman MV. Safety of excluding acute pulmonary embolism based on an unlikely clinical probability by the Wells rule and normal D-dimer concentration: a meta-analysis. Thromb Res. 2010 Apr;125(4):e123-7. doi: 10.1016/j.thromres.2009.11.009. Epub 2009 Nov 26.

ReferenceCitation: Perrier A, Desmarais S, Goehring C, de Moerloose P, Morabia A, Unger PF, Slosman D, Junod A, Bounameaux H. D-dimer testing for suspected pulmonary embolism in outpatients. Am J Respir Crit Care Med. 1997 Aug;156(2 Pt 1):492-6. doi: 10.1164/ajrccm.156.2.9702032.

ReferenceCitation: Wells PS, Anderson DR, Rodger M, Forgie M, Kearon C, Dreyer J, Kovacs G, Mitchell M, Lewandowski B, Kovacs MJ. Evaluation of D-dimer in the diagnosis of suspected deep-vein thrombosis. N Engl J Med. 2003 Sep 25;349(13):1227-35. doi: 10.1056/NEJMoa023153.

ReferenceCitation: Pernod G, Caterino J, Maignan M, Tissier C, Kassis J, Lazarchick J; DIET study group. D-Dimer Use and Pulmonary Embolism Diagnosis in Emergency Units: Why Is There Such a Difference in Pulmonary Embolism Prevalence between the United States of America and Countries Outside USA? PLoS One. 2017 Jan 13;12(1):e0169268. doi: 10.1371/journal.pone.0169268. eCollection 2017.

evidence: oa repository (semantic scholar lookup)

version: submittedVersion

repositories_concat: PubMed Central;pdfs.semanticscholar.org

publisher_dissemination: Public Library of Science

lead_sponsor: Diagnostica Stago

sponsor_collaborators:
Diagnostica Stago
University Hospital, Grenoble

location_country:
Italy
United States
Spain
Canada
France

location_facility:
Northwestern Medical Center
Massachusetts General Hospital
Moses Cone Memorial Hospital
Maisonneuve Rosemont Hospital
University of Iowa
Oklahoma Health Sciences Center
Univeristy Medical Center
Medical Center of South Carolina
General Hospital
Brigham and Woman's Hosp
Indiana University Health
Texas Health Fort Worth Hospital
Lehigh Valley Hospital Health Network
IRCCS H San Raffaele
University Hospital


title: Study of GSK2302024A Antigen-Specific Cancer Immunotherapeutic Combined With Standard Neoadjuvant Treatment in Patients With WT1-positive Primary Invasive Breast Cancer

acronym: INDUCT

summary: The purpose of this study is to evaluate the safety, immunogenicity and clinical activity of a new WT1 anti-cancer immunotherapy in patients with WT1-positive Stage II or III breast cancer. The treatment will be given before surgery in combination with standard therapy.

ReferenceCitation: Higgins M, Curigliano G, Dieras V, Kuemmel S, Kunz G, Fasching PA, Campone M, Bachelot T, Krivorotko P, Chan S, Ferro A, Schwartzberg L, Gillet M, De Sousa Alves PM, Wascotte V, Lehmann FF, Goss P. Safety and immunogenicity of neoadjuvant treatment using WT1-immunotherapeutic in combination with standard therapy in patients with WT1-positive Stage II/III breast cancer: a randomized Phase I study. Breast Cancer Res Treat. 2017 Apr;162(3):479-488. doi: 10.1007/s10549-017-4130-y. Epub 2017 Feb 7.

oa_colors: green hybrid

evidence: open (via page says license)

unpaywall_oa_status: hybrid

ipd_sharing_description: IPD for this study is available via the Clinical Study Data Request site (click on the link provided below).

lead_sponsor: GlaxoSmithKline

location_country:
Germany
Italy
United Kingdom
Belgium
United States
Russian Federation
France


title: A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

summary: Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

ReferenceCitation: Feldt-Rasmussen U, Hughes D, Sunder-Plassmann G, Shankar S, Nedd K, Olivotto I, Ortiz D, Ohashi T, Hamazaki T, Skuban N, Yu J, Barth JA, Nicholls K. Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study. Mol Genet Metab. 2020 Sep-Oct;131(1-2):219-228. doi: 10.1016/j.ymgme.2020.07.007. Epub 2020 Aug 15.

evidence: open (via crossref license)

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

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

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

repositories_concat: UCL Discovery

ReferenceCitation: Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb3 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;:

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

ReferenceCitation: Haninger-Vacariu N, El-Hadi S, Pauler U, Foretnik M, Kain R, Prohaszka Z, Schmidt A, Skuban N, Barth JA, Sunder-Plassmann G. Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report. Case Rep Obstet Gynecol. 2019 Dec 21;2019:1030259. doi: 10.1155/2019/1030259. eCollection 2019.

repository_institution: Hindawi - Hindawi

repositories_concat: PubMed Central;hindawi.com

repositories_institution:
Hindawi - Hindawi
PubMed Central - Europe PMC

publisher_dissemination: Hindawi

ReferenceCitation: Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30.

ReferenceCitation: Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10. Erratum In: J Med Genet. 2018 Apr 16;:

repository_institution: University of Oxford - Oxford Academic Notes & Queries

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

repository_institution: University of Florence - Florence Research

repository_institution: University of California - eScholarship University of California

repositories_concat: PubMed Central;Pure (Denmark);UCL Discovery;University of Melbourne - Minerva Access;escholarship.org;flore.unifi.it;open-archive.highwire.org

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.

location_country:
Italy
United Kingdom
Denmark
Australia
Brazil
Japan
Austria
Belgium
United States
France


title: Dasatinib (BMS-354825) Combined With SMO Inhibitor (BMS-833923; XL139) in CML With Resistance or Suboptimal Response to a Prior TKI

summary: The purpose of the study is to determine the safety and tolerability of the combination of BMS-833923 plus dasatinib in patients with chronic myeloid leukemia.

design_allocation: Non-Randomized

lead_sponsor: Bristol-Myers Squibb

location_country:
Germany
Finland
Italy
United Kingdom
United States
Canada
France

location_facility: Sidney Kimmel Cancer Center At Johns Hopkins


title: International Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of KIACTA in Preventing Renal Function Decline in Patients With AA Amyloidosis

summary: The primary purpose of this study is to assess the efficacy and safety of treatment with Kiacta in adult patients with AA Amyloidosis.

primary_purpose: Prevention

lead_sponsor: C.T. Development America, Inc.

location_country:
Finland
Germany
Poland
Turkey
France
Lithuania
United States
Latvia
Ukraine
India
Tunisia
Belgium
Peru
Estonia
Egypt
Italy
United Kingdom
Sweden
Netherlands
Spain
Georgia
Russian Federation
Israel

location_facility: Sverdlovsk Regional Clinical Hospital #1

location_facility: Kemerovo State Medical Academy of Roszdrav

location_facility: Akademicki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

location_facility: Karolinska Universitetssjukhuset i Huddinge

location_facility: Municipal Medical & Preventive Institution Donetsk Regional Clinical Territorial Medical Association

location_facility: State Institution "Institute of Nephrology of AMS of Ukraine"

location_facility: Research Institute of Clinical and Experimental Lymphology

location_facility: Helsingin yliopistollinen keskussairaala / Meilahti

location_facility: Hospital of Lithuanian University of Health Sciences Kaunas Clinics

location_facility: National Scientific Center "Institute of cardiology n.a. academician M.D Strazhesko"

location_facility: Eskisehir Osmangazi University Medical Faculty

location_facility: Vilnius University Hospital Santariskiu Klinikos

location_facility: Cukurova University Medical Faculty Balcali Hospital

location_facility: Pauls Stradins Clinical University Hospital


title: Post-Myocardial Infarction Remodeling Prevention Therapy

acronym: PRomPT

summary: The purpose of this study is to demonstrate the feasibility of pacing as a therapy to prevent adverse remodeling of the myocardium following an acute myocardial infarction (MI) in patients at highest risk for adverse myocardial remodeling.

ReferenceCitation: Stone GW, Chung ES, Stancak B, Svendsen JH, Fischer TM, Kueffer F, Ryan T, Bax J, Leon A; PRomPT Trial Investigators. Peri-infarct zone pacing to prevent adverse left ventricular remodelling in patients with large myocardial infarction. Eur Heart J. 2016 Feb 1;37(5):484-93. doi: 10.1093/eurheartj/ehv436. Epub 2015 Aug 30.

lead_sponsor: Medtronic Cardiac Rhythm and Heart Failure

location_country:
Germany
Slovakia
Hungary
Denmark
United States
Saudi Arabia
France

location_facility: Baylor Jack and Jane Hamilton Heart and Vascular Hospital

location_facility: Centre Hospitalier Régional Universitaire de Lille

location_facility: Hospital of the University of Pennsylvania

location_facility: Vychodoslovensky ustav srdcovych a cievnych chorob, a.s.

intervention_type: Device


title: A Phase 2 Multicenter, Randomized, Double Blind, Placebo Controlled Study Of The Safety And Efficacy Of Pf-03049423 In Subjects With Ischemic Stroke

summary: The purpose of this study is to evaluate the safety and tolerability of PF-03049423 following multiple dose administration to subjects with ischemic stroke. The study will also evaluate the efficacy of PF-03049423, relative to placebo, in subjects with ischemic stroke following 90 days of therapy. The study will also explore the relationship between PF-03049423 concentration and blood pressure.

ReferenceCitation: Di Cesare F, Mancuso J, Woodward P, Bednar MM, Loudon PT; A9541004 Stroke Study Group. Phosphodiesterase-5 Inhibitor PF-03049423 Effect on Stroke Recovery: A Double-Blind, Placebo-Controlled Randomized Clinical Trial. J Stroke Cerebrovasc Dis. 2016 Mar;25(3):642-9. doi: 10.1016/j.jstrokecerebrovasdis.2015.11.026. Epub 2015 Dec 28.

lead_sponsor: Pfizer

location_country:
Germany
India
Hungary
United States
Korea, Republic of
Taiwan
Bulgaria
Canada
Czech Republic
France

location_facility: Oklahoma University Health Sciences Center

location_facility: Universitaetsklinikum Essen, Neurologische Klinik

location_facility: Samsung Medical Center, Department of Neurology

location_facility: MBALNP "Sveti Naum" EAD, Klinika za Intenzivno Lechenie na Nervni Bolesti

location_facility: Dr. Kennessey Albert Korhaz-Rendelointezet, Neurologiai Osztaly

location_facility: The Methodist Hospital Neurological Institute

location_facility: UMBAL Tsaritsa Yoanna, Klinika po nevrologia

location_facility: Universitetska mnogoprofilna bolnitsa za aktivno lechenie Aleksandrovska, Klinika po Nevrologia

location_facility: Orszagos Idegtudomanyi Intezet, Stroke-ambulancia

location_facility: Investigational Drug Services at OU Medical Center

location_facility: Massachusetts General Hospital/Department of Neurology

location_facility: Seoul National University Bundang Hospital, Department of Neurology

location_facility: Petz Aladar Megyei Oktato Korhaz, Neurologiai Osztaly

location_facility: Jose Canedo, M.D., West Georgia Neurology

location_facility: Hallym University Sacred Heart Hospital, Department of Neurology

location_facility: Chonnam National University Hospital, Department of Neurology

location_facility: UNC Department of Neurology Stroke Division

location_facility: Inha University Hospital, Department of Neurology

location_facility: Voennomeditsinska Akademia - MBAL- Pleven, Otdelenie po Nervni bolesti

location_facility: Penn State Milton South Hershey Medical Center / Penn State College of Medicine

location_facility: Fovarosi Onkormanyzat Peterfy Sandor utcai Korhaz-Rendelointezet es Baleseti Kozpont/Neurologia

location_facility: Vtora Mnogoprofilna Bolnitsa za Aktivno Lechenie, Otdelenie po Nevrologia

location_facility: Honvedkorhaz-Allami Egeszsegugyi Kozpont, Ideggyogyaszati Osztaly

location_facility: Penn State Hershey Rehabilitation Hospital

location_facility: Severance Hospital, Yonsei University College of Medicine, Department of Neurology

location_facility: Asan Medical Center, Department of Neurology

location_facility: Chang Gung Medical Foundation-Kaohsiung Chang Gung Memorial Hospital

location_facility: Klinikum Rechts der Isar, Neurologische Klinik

location_facility: MBAL "Tokuda Bolnitsa", Otdelenie po nevrologiya

location_facility: Semmelweis Egyetem AOK / Neurologiai Klinika

location_facility: Chang Gung Medical Foundation-Linkou Branch

location_facility: Medical Research & Health Education Foundation, Inc.


title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous LY2127399 in Patients With Systemic Lupus Erythematosus (SLE)

summary: The purpose of this SLE study is to evaluate the efficacy, safety and tolerability of two different doses of LY2127399 administered in addition to standard of care therapy in participants with active SLE.

ReferenceCitation: Toro-Dominguez D, Martorell-Marugan J, Martinez-Bueno M, Lopez-Dominguez R, Carnero-Montoro E, Barturen G, Goldman D, Petri M, Carmona-Saez P, Alarcon-Riquelme ME. Scoring personalized molecular portraits identify Systemic Lupus Erythematosus subtypes and predict individualized drug responses, symptomatology and disease progression. Brief Bioinform. 2022 Sep 20;23(5):bbac332. doi: 10.1093/bib/bbac332.

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: University of Granada - Institutional Repository of the University of Granada

repositories_concat: PubMed Central;digibug.ugr.es

ReferenceCitation: Hannon CW, McCourt C, Lima HC, Chen S, Bennett C. Interventions for cutaneous disease in systemic lupus erythematosus. Cochrane Database Syst Rev. 2021 Mar 9;3(3):CD007478. doi: 10.1002/14651858.CD007478.pub2.

ReferenceCitation: Li Y, Higgs RE, Hoffman RW, Dow ER, Liu X, Petri M, Wallace DJ, Dorner T, Eastwood BJ, Miller BB, Liu Y. A Bayesian gene network reveals insight into the JAK-STAT pathway in systemic lupus erythematosus. PLoS One. 2019 Dec 2;14(12):e0225651. doi: 10.1371/journal.pone.0225651. eCollection 2019.

ReferenceCitation: Kalunian KC, Urowitz MB, Isenberg D, Merrill JT, Petri M, Furie RA, Morgan-Cox MA, Taha R, Watts S, Silk M, Linnik MD. Clinical trial parameters that influence outcomes in lupus trials that use the systemic lupus erythematosus responder index. Rheumatology (Oxford). 2018 Jan 1;57(1):125-133. doi: 10.1093/rheumatology/kex368.

oa_colors: green other

repositories_institution: University College London - UCL Discovery

ReferenceCitation: Rovin BH, Dooley MA, Radhakrishnan J, Ginzler EM, Forrester TD, Anderson PW. The impact of tabalumab on the kidney in systemic lupus erythematosus: results from two phase 3 randomized, clinical trials. Lupus. 2016 Dec;25(14):1597-1601. doi: 10.1177/0961203316650734. Epub 2016 May 24.

publisher_dissemination: SAGE Publications

ReferenceCitation: Hoffman RW, Merrill JT, Alarcon-Riquelme MM, Petri M, Dow ER, Nantz E, Nisenbaum LK, Schroeder KM, Komocsar WJ, Perumal NB, Linnik MD, Airey DC, Liu Y, Rocha GV, Higgs RE. Gene Expression and Pharmacodynamic Changes in 1,760 Systemic Lupus Erythematosus Patients From Two Phase III Trials of BAFF Blockade With Tabalumab. Arthritis Rheumatol. 2017 Mar;69(3):643-654. doi: 10.1002/art.39950.

ReferenceCitation: Merrill JT, van Vollenhoven RF, Buyon JP, Furie RA, Stohl W, Morgan-Cox M, Dickson C, Anderson PW, Lee C, Berclaz PY, Dorner T. Efficacy and safety of subcutaneous tabalumab, a monoclonal antibody to B-cell activating factor, in patients with systemic lupus erythematosus: results from ILLUMINATE-2, a 52-week, phase III, multicentre, randomised, double-blind, placebo-controlled study. Ann Rheum Dis. 2016 Feb;75(2):332-40. doi: 10.1136/annrheumdis-2015-207654. Epub 2015 Aug 20.

repositories_institution: University of Oxford - Oxford Academic Notes & Queries

location_country:
New Zealand
Australia
Brazil
Canada
Serbia
France
Hungary
United States
Latvia
India
Romania
Tunisia
Malaysia
Ecuador
United Kingdom
Mexico
Spain
South Africa
Taiwan
Russian Federation
Israel

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.


acronym: OSKIRA - 1

lead_sponsor: AstraZeneca

summary: The purpose of the study is to evaluate the effectiveness of two dosing regimens of fostamatinib compared to placebo, in patients with rheumatoid arthritis (RA) who are taking methotrexate but not responding. The study will last for 1 year.

ReferenceCitation: Kjelgaard-Petersen CF, Platt A, Braddock M, Jenkins MA, Musa K, Graham E, Gantzel T, Slynn G, Weinblatt ME, Karsdal MA, Thudium CS, Bay-Jensen AC. Translational Biomarkers and Ex Vivo Models of Joint Tissues as a Tool for Drug Development in Rheumatoid Arthritis. Arthritis Rheumatol. 2018 Sep;70(9):1419-1428. doi: 10.1002/art.40527. Epub 2018 Jul 24.

repository_institution: Technical University of Denmark, DTU - Technical University of Denmark, DTU Orbit

repository_normalized: Pure (Denmark)

repositories:
PubMed Central
Pure (Denmark)

repositories_concat: PubMed Central;Pure (Denmark)

title: (OSKIRA-1): A Phase III, Multi-centre, Randomised, Double-Blind, Placebo-Controlled, Parallel Group Study of Two Dosing Regimens of Fostamatinib Disodium in Rheumatoid Arthritis Patients With an Inadequate Response to Methotrexate

location_country:
Poland
India
Slovakia
Hungary
Argentina
United Kingdom
Australia
Brazil
Mexico
Belgium
United States
Chile
Peru
Ukraine
Bulgaria
Estonia
France


lead_sponsor: Debiopharm International SA

summary: The study is to investigate whether participants with hepatitis C virus (HCV) genotype 1 who have a history of non-response/relapse to peginterferon alfa-2a (PEG) and ribavirin (RBV) may benefit from treatment with triple therapy alisporivir (ALV; DEB025) with PEG and RBV versus placebo with PEG and RBV.

title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase II Study on Efficacy and Safety of DEB025 Combined With Peg-IFN Alfa-2a and Ribavirin in Chronic Hepatitis C Genotype 1 Relapsers and Non-responders to Previous Peg-IFN Alfa-2 Plus Ribavirin Treatment

location_country:
Germany
Italy
Poland
Hungary
United Kingdom
Romania
Australia
Belgium
United States
Spain
Turkey
Taiwan
Puerto Rico
France


title: A Phase 1/2, Multi-Center, Open-Label, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of the mTOR Kinase Inhibitor CC-223 Administered Orally to Subjects With Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Multiple Myeloma

summary: The main purpose of this first human study with CC-223 is to assess the safety and action of a new class of experimental drug (dual mTOR inhibitors) in patients with advanced tumors unresponsive to standard therapies and to determine the appropriate dose and tumor type for later-stage clinical trials.

lead_sponsor: Celgene

location_country:
United States
Spain
United Kingdom
France

location_facility: Mayo Clinic Cancer Clinical Studies Unit

location_facility: Institut Gustave Roussy Faculte de Medecine Paris Sud Service de pneumologie

location_facility: NYU Cancer Institute - Bellevue Hospital

location_facility: University of California, San Francisco Hellen Diller Family Comprehensive Cancer Center

location_facility: Sarah Cannon Research Institute Drug Development Unit


title: A Phase II, Multicentre, Randomised, Double-Blind, Parallel Group, Placebo Controlled Study to Evaluate Safety, Tolerability and Clinical Efficacy of MT-1303 in Subjects With Moderate to Severe Active Crohn's Disease

summary:
The primary objectives of the study are:

To evaluate the safety and tolerability of MT-1303 in subjects with moderate to severe active Crohn's Disease（CD）
To evaluate the clinical efficacy of MT-1303 in subjects with moderate to severe active CD.

lead_sponsor: Mitsubishi Tanabe Pharma Corporation

location_country:
Germany
Italy
Poland
Slovakia
Hungary
Israel
Japan
Netherlands
Ukraine
Czech Republic
France

location_facility:
Investigational Site
Investigational site


title: A Phase Ib/II Study of the ALK Inhibitor Ceritinib in Combination With the CDK4/6 Inhibitor LEE011 in Patients With ALK-positive Non-Small Cell Lung Cancer

summary:
This was a Phase Ib/II study of the ALK inhibitor ceritinib in combination with the CDK4/6 inhibitor LEE011 in patients with ALK-positive non-small cell lung cancer.

The purpose of the study was to determine the MTD/RP2D of the LEE011 and ceritinib combination and evaluate whether the combination was safe and had beneficial effects in ALK-positive advanced non-small cell lung cancer patients.

This trial did not progress to Phase II. Trial population terminated before reaching Phase II

ipd_sharing: Undecided

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

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

lead_sponsor: Novartis Pharmaceuticals

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


title: Resect and Discard Extension to Fuji Intelligent Color Enhancement (FICE) of Narrow Band Imaging International Colorectal Endoscopic (NicE) Classification

acronym: FICE;NICE

summary: The purpose of this prospective, non-interventional study is to validate the Narrow Band Imaging International Colorectal Endoscopic (NICE) classification for differentiating subcentimetric hyperplastic and adenomatous polyps by using Fuji Intelligent Color Enhancement (FICE) technology with high-definition without optical magnification.

status: Unknown status

ReferenceCitation: Hewett DG, Kaltenbach T, Sano Y, Tanaka S, Saunders BP, Ponchon T, Soetikno R, Rex DK. Validation of a simple classification system for endoscopic diagnosis of small colorectal polyps using narrow-band imaging. Gastroenterology. 2012 Sep;143(3):599-607.e1. doi: 10.1053/j.gastro.2012.05.006. Epub 2012 May 15.

lead_sponsor: Istituto Clinico Humanitas

collaborators: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

location_country:
Germany
Italy
United Kingdom
Belgium
France

location_facility: Endoscopy Unit, Humanitas Research Hospital

location_facility: Solent Centre for Digestive Diseases , Royal Alexandra Hospital

location_facility: Dept of Gastroenterology and Endoscopy, University Hospital

location_facility: Department of Medicine I, University of Erlangen-Nürnberg Medical School

location_facility: Dept of Gastroenterology, Edouard Herriot Hospital, Hospices Civils of Lyon

location_facility: Digestive Endoscopy Unit, Nuovo Regina Margherita Hospital

location_facility: Dept of Gastroenterology and interventional Endoscopy, Krankenhaus Barmherzige Brüder

intervention_type: Procedure


lead_sponsor: Amgen

summary: The purpose of this study is to determine whether apremilast is safe and effective in the treatment of patients with psoriatic arthritis, specifically in improving signs and symptoms of psoriatic arthritis (tender and swollen joints, pain, physical function) in treated patients.

ReferenceType: result

ReferenceCitation: Kavanaugh A, Mease PJ, Gomez-Reino JJ, Adebajo AO, Wollenhaupt J, Gladman DD, Hochfeld M, Teng LL, Schett G, Lespessailles E, Hall S. Longterm (52-week) results of a phase III randomized, controlled trial of apremilast in patients with psoriatic arthritis. J Rheumatol. 2015 Mar;42(3):479-88. doi: 10.3899/jrheum.140647. Epub 2015 Jan 15.

publisher_dissemination: The Journal of Rheumatology

ReferenceCitation: Mease PJ, Gladman DD, Kavanaugh A, McGonagle D, Nash P, Guerette B, Nakasato P, Brunori M, Teng L, McInnes IB. Articular and Extra-Articular Benefits in ACR20 Non-responders at Week 104 Treated With Apremilast: Pooled Analysis of Three Randomized Controlled Trials. Rheumatol Ther. 2021 Dec;8(4):1677-1691. doi: 10.1007/s40744-021-00369-x. Epub 2021 Sep 18.

ReferenceCitation: Mease PJ, Gladman DD, Ogdie A, Coates LC, Behrens F, Kavanaugh A, McInnes I, Queiro R, Guerette B, Brunori M, Teng L, Smolen JS. Treatment-to-Target With Apremilast in Psoriatic Arthritis: The Probability of Achieving Targets and Comprehensive Control of Disease Manifestations. Arthritis Care Res (Hoboken). 2020 Jun;72(6):814-821. doi: 10.1002/acr.24134. Epub 2020 May 8.

repository_institution: The University of Glasgow - Enlighten: Publications

repositories_concat: PubMed Central;eprints.gla.ac.uk

ReferenceCitation: Kavanaugh A, Gladman DD, Edwards CJ, Schett G, Guerette B, Delev N, Teng L, Paris M, Mease PJ. Long-term experience with apremilast in patients with psoriatic arthritis: 5-year results from a PALACE 1-3 pooled analysis. Arthritis Res Ther. 2019 May 10;21(1):118. doi: 10.1186/s13075-019-1901-3.

repositories_concat: PubMed Central;aleph.bib-bvb.de

ReferenceCitation: Gladman DD, Kavanaugh A, Gomez-Reino JJ, Wollenhaupt J, Cutolo M, Schett G, Lespessailles E, Guerette B, Delev N, Teng L, Edwards CJ, Birbara CA, Mease PJ. Therapeutic benefit of apremilast on enthesitis and dactylitis in patients with psoriatic arthritis: a pooled analysis of the PALACE 1-3 studies. RMD Open. 2018 Jun 27;4(1):e000669. doi: 10.1136/rmdopen-2018-000669. eCollection 2018.

repository_institution: University of Massachusetts Medical School - eScholarship@UMMS

repository_institution: American Medical Association - The Journal of the American Medical Association (JAMA) Network

ReferenceCitation: Kavanaugh A, Mease PJ, Gomez-Reino JJ, Adebajo AO, Wollenhaupt J, Gladman DD, Lespessailles E, Hall S, Hochfeld M, Hu C, Hough D, Stevens RM, Schett G. Treatment of psoriatic arthritis in a phase 3 randomised, placebo-controlled trial with apremilast, an oral phosphodiesterase 4 inhibitor. Ann Rheum Dis. 2014 Jun;73(6):1020-6. doi: 10.1136/annrheumdis-2013-205056. Epub 2014 Mar 4.

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

location_facility: Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

location_facility: Hospital Universitario Marques de Valdecilla

location_facility: Veszprém Megyei Önkormányzat Csolnoky Ferenc Kórház-Rendelöintézet

location_facility: Departmental Hospital at Smolensk Station RZhD JSC

location_facility: Ryazan I.P. Pavlov State Medical University

location_facility: Hospital General Universitario Gregorio Maranon

location_facility: Arthritis and Rheumatic Disease Specialties

location_facility: Clinical Research Center of Reading, LLP

location_facility: Dermatology Treatment and Research Center

location_facility: Hospital Clinico Universitario de Santiago

location_facility: Centre Hospitalier Universitaire de Sherbrooke-Hospital Fleurimont

location_facility: Arthritis and Osteoporosis Associates LLP

location_facility: Honvéd Kórház - Állami Egészségügyi Központ

location_facility: The Center for Rheumatology and Bone Research

location_facility: Piedmont Medical Research Associates Inc

location_facility: Voronezh Regional Clinical Hopsital #1, Voronezh State Medical Academy

location_facility: St. Clare's Health Care Corporation of St. John's

location_facility: Queen Elizabeth Hospital for Rheumatic Disease

location_facility: The Arthritis Program Research Group Inc.

title: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group, Efficacy and Safety Study of Two Doses of Apremilast (CC-10004) in Subjects With Active Psoriatic Arthritis

ipd_sharing_description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

location_country:
Germany
Poland
New Zealand
Hungary
United Kingdom
Australia
Austria
United States
Spain
South Africa
Canada
Russian Federation
France


title: The VENUS Clinical Study (Verifying the Effectiveness of the NUsurface® System) A Multi-center, Prospective, Randomized, Interventional, Superiority Clinical Study

acronym: VENUS

summary: The NUsurface® Meniscus Implant Randomized Study is a multi-center, prospective randomized, interventional clinical trial to test the hypothesis that the NUsurface implant is superior to the non-surgical standard of care in treating the target population.The rationale for performing this clinical study is to gather clinical data to evaluate the safety and effectiveness of the NUsurface device compared to the Standard of Care.

ReferenceCitation: Shemesh M, Asher R, Zylberberg E, Guilak F, Linder-Ganz E, Elsner JJ. Viscoelastic properties of a synthetic meniscus implant. J Mech Behav Biomed Mater. 2014 Jan;29:42-55. doi: 10.1016/j.jmbbm.2013.08.021. Epub 2013 Sep 3.

ReferenceCitation: Zur G, Linder-Ganz E, Elsner JJ, Shani J, Brenner O, Agar G, Hershman EB, Arnoczky SP, Guilak F, Shterling A. Chondroprotective effects of a polycarbonate-urethane meniscal implant: histopathological results in a sheep model. Knee Surg Sports Traumatol Arthrosc. 2011 Feb;19(2):255-63. doi: 10.1007/s00167-010-1210-5. Epub 2010 Jul 16.

ReferenceCitation: Elsner JJ, Portnoy S, Guilak F, Shterling A, Linder-Ganz E. MRI-based characterization of bone anatomy in the human knee for size matching of a medial meniscal implant. J Biomech Eng. 2010 Oct;132(10):101008. doi: 10.1115/1.4002490.

ReferenceCitation: Elsner JJ, Portnoy S, Zur G, Guilak F, Shterling A, Linder-Ganz E. Design of a free-floating polycarbonate-urethane meniscal implant using finite element modeling and experimental validation. J Biomech Eng. 2010 Sep;132(9):095001. doi: 10.1115/1.4001892.

ReferenceCitation: Linder-Ganz E, Elsner JJ, Danino A, Guilak F, Shterling A. A novel quantitative approach for evaluating contact mechanics of meniscal replacements. J Biomech Eng. 2010 Feb;132(2):024501. doi: 10.1115/1.4000407.

ReferenceCitation: Elsner JJ, Shemesh M, Shefy-Peleg A, Gabet Y, Zylberberg E, Linder-Ganz E. Quantification of in vitro wear of a synthetic meniscus implant using gravimetric and micro-CT measurements. J Mech Behav Biomed Mater. 2015 Sep;49:310-20. doi: 10.1016/j.jmbbm.2015.05.017. Epub 2015 May 28.

ReferenceCitation: De Coninck T, Elsner JJ, Linder-Ganz E, Cromheecke M, Shemesh M, Huysse W, Verdonk R, Verstraete K, Verdonk P. In-vivo evaluation of the kinematic behavior of an artificial medial meniscus implant: A pilot study using open-MRI. Clin Biomech (Bristol, Avon). 2014 Sep;29(8):898-905. doi: 10.1016/j.clinbiomech.2014.07.001. Epub 2014 Jul 17.

lead_sponsor: Active Implants

location_country: Germany Italy France Netherlands Belgium Israel

location_facility:
University Hospital of Munich
Ichilov Hospital
Centre Hospitalier de Versailles
AZ Monica
Academisch Ziekenhuis Maastricht
Assaf Harofeh Medical Center
Ospedale Sacro Cuore - Don Calabria


acronym: PERSIST

summary: The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.

ReferenceCitation: McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.

sponsor_collaborators:
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation

title: An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis

location_country:
Germany
Ireland
United Kingdom
Australia
United States
Canada
Czech Republic
France


acronym: ACES 263

lead_sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

summary: This will be a multicenter, randomized, double-blind study designed to assess the efficacy, safety and tolerability of an oral Aripiprazole/Escitalopram combination therapy in participants with MDD who have demonstrated an incomplete response to a prospective trial of Escitalopram, and report a treatment history for the current MDD episode of an inadequate response to at least one and no more than three adequate trials of an approved antidepressant other than Escitalopram. An inadequate response is defined as less than a 50% reduction in depressive symptom severity as assessed by the participant's self-report on the Massachusetts General Hospital Antidepressant Treatment Response Questionnaire (ATRQ) and evaluated by the investigator as part of the participant's medical and psychiatric history. An adequate trial is defined as an antidepressant treatment for at least 6 weeks duration (or at least 3 weeks for combination treatments) at an approved dose as specified in the ATRQ.

location_facility:
Study Site 1
Study Site 2

title: A Multicenter, Randomized, Double-blind Study to Evaluate the Efficacy, Safety and Tolerability of an Oral Aripiprazole/Escitalopram Combination Therapy in Patients With Major Depressive Disorder

ipd_sharing_description: Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.

location_country:
Poland
India
Hungary
Sweden
United States
Spain
Malaysia
Croatia
South Africa
Canada
France


acronym: SHRINK

lead_sponsor: Astellas Pharma Inc

summary: The purpose is to see if solifenacin has any effect on bladder wall thickness and urine inflammatory marker measurements after 12 weeks of treatment.

title: A Study to Evaluate the Overall Effect of Solifenacin 5mg and 10mg on Bladder Wall Thickness and Urinary Nerve Growth Factor in Female Subjects With Overactive Bladder and a Diagnosis of Detrusor Overactivity - A Double-blind, Randomised, Placebo-controlled, Parallel Group, Multi-centre Study.

ipd_sharing_description: Details of the IPD sharing plan for this study can be found at www.clinicalstudydatarequest.com.

location_country:
Germany
Poland
Turkey
Canada
France
Czech Republic
Slovakia
Hungary
United States
Romania
Belgium
Italy
Norway
United Kingdom
Sweden
Austria
Spain
Bulgaria
Russian Federation
Israel


type: Registry Identifier

title: A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]

summary: The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.

location_country:
Italy
United Kingdom
United States
Canada
France

location_facility: University of South Alabama College of Medicine

location_facility: University of Colorado Health Science Center

location_facility: Children's Hospital of the King's Daughters

location_facility: Children's Hospital & Research Center at Oakland

location_facility: Grady Hospital, Georgia Comprehensive Sickle Cell Center


title: A Phase II, Multicenter, Randomized, Controlled, Open-label Study of the Safety, Efficacy and Pharmacokinetics of ABT-263 in Combination With Dose-intensive Rituximab, or Dose-intensive Rituximab Alone, in Previously Untreated Patients With B-Cell, Chronic Lymphocytic Leukemia (CLL)

summary: This Phase II, randomized, open-label, international, multicenter trial is designed to evaluate the safety and efficacy of rituximab monotherapy when given according to a dose intense regimen and to assess the safety, efficacy, and pharmacokinetics of ABT-263 when combined with dose-intense rituximab in previously untreated patients with B-cell CLL.

lead_sponsor: Genentech, Inc.

sponsor_collaborators:
Genentech, Inc.
AbbVie (prior sponsor, Abbott)

location_country:
Italy
Poland
Israel
United Kingdom
Australia
Brazil
United States
Ukraine
Czech Republic
Puerto Rico
Russian Federation
France


lead_sponsor: Teva Branded Pharmaceutical Products R&D, Inc.

summary: The study aims to evaluate the safety and clinical effect of daily oral treatment with laquinimod capsules in active lupus nephritis participants. This study will assess Laquinimod doses of 0.5 milligrams (mg)/day and 1 mg/day in combination with standard of care treatment (mycophenolate mofetil [MMF] and corticosteroids). Laquinimod is a novel immunomodulating drug which is currently in advanced stages of development by Teva Pharmaceuticals Ltd. for Multiple Sclerosis.

location_facility:
Teva Investigational Site 3509
Teva Investigational Site 1113
Teva Investigational Site 1115
Teva Investigational Site 1019
Teva Investigational Site 5007
Teva Investigational Site 3411
Teva Investigational Site 3413
Teva Investigational Site 5003
Teva Investigational Site 1024
Teva Investigational Site 1114
Teva Investigational Site 1017
Teva Investigational Site 1032
Teva Investigational Site 1016
Teva Investigational Site 5006
Teva Investigational Site 1026
Teva Investigational Site 3409
Teva Investigational Site 1025
Teva Investigational Site 3410
Teva Investigational Site 1018
Teva Investigational Site 1030
Teva Investigational Site 1028
Teva Investigational Site 1021
Teva Investigational Site 1022
Teva Investigational Site 5002
Teva Investigational Site 3412
Teva Investigational Site 5001
Teva Investigational Site 1031
Teva Investigational Site 5005
Teva Investigational Site 3510
Teva Investigational Site 1020

title: A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability and Clinical Effect of Laquinimod in Active Lupus Nephritis Patients, in Combination With Standard of Care (Mycophenolate Mofetil and Steroids)

location_country:
United Kingdom
United States
Canada
Russian Federation
France


title: A Phase 1/2 Dose-Escalation Study of XL147 (SAR245408) or XL765 (SAR245409) in Combination With Letrozole in Subjects With Hormone Receptor-Positive and HER2-Negative Breast Cancer Refractory to a Nonsteroidal Aromatase Inhibitor

summary:
Phase 1 of this study will evaluate the maximum tolerated dose (MTD) of XL147 when given in combination with letrozole (Femara) and of XL765 when given in combination with letrozole. After the MTD is established for each combination (Phase 2), subjects will be enrolled to evaluate the preliminary efficacy and safety of these combinations in subjects with breast cancer refractory to a non-steroidal aromatase inhibitor that is ER+/PGR+ and HER2-. Letrozole is used in the treatment of different types of breast cancer, but patients can develop resistance.

Upregulation of PI3K activity is one of the most common characteristics of human cancer cells, including breast tumor cells. Activation of PI3K results in stimulation of AKT and mTOR kinases, resulting in the promotion of tumor cell proliferation and survival. Preclinical and retrospective clinical data suggest that aberrant activation of the PI3K pathway may play a role in aromatase inhibitor resistance in patients with ER+, HER2- breast cancer. XL147 is a potent inhibitor of PI3K, and XL765 is a potent dual inhibitor of PI3K and mTOR; therefore either of these compounds in combination with letrozole warrants clinical investigation.

lead_sponsor: Sanofi

location_country:
United States
Spain
France

location_facility:
Investigational Site Number 1441
Investigational Site Number 5201
Investigational Site Number 1331
Investigational Site Number 1252
Investigational Site Number 1330
Investigational Site Number 1537
Investigational Site Number 3415
Investigational Site Number 5246
Investigational Site Number 3420
Investigational Site Number 3321
Investigational Site Number 3324
Investigational Site Number 3413
Investigational Site Number 1138
Investigational Site Number 1214
Investigational Site Number 3419
Investigational Site Number 1238
Investigational Site Number 1601


title: A Multicenter, Randomized, Addition to Baseline Treatment, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Satralizumab (SA237) in Patients With Neuromyelitis Optica (NMO) and NMO Spectrum Disorder (NMOSD)

summary: The objective of this study is to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic, and immunogenic profiles of satralizumab, compared with placebo, in addition to baseline immunosuppressive treatment in participants with NMO and NMOSD.

ReferenceCitation: Yamamura T, Weinshenker B, Yeaman MR, De Seze J, Patti F, Lobo P, von Budingen HC, Kou X, Weber K, Greenberg B. Long-term safety of satralizumab in neuromyelitis optica spectrum disorder (NMOSD) from SAkuraSky and SAkuraStar. Mult Scler Relat Disord. 2022 Oct;66:104025. doi: 10.1016/j.msard.2022.104025. Epub 2022 Jul 5.

ReferenceCitation: Yamamura T, Kleiter I, Fujihara K, Palace J, Greenberg B, Zakrzewska-Pniewska B, Patti F, Tsai CP, Saiz A, Yamazaki H, Kawata Y, Wright P, De Seze J. Trial of Satralizumab in Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019 Nov 28;381(22):2114-2124. doi: 10.1056/NEJMoa1901747.

evidence: open (via free article)

publisher_dissemination: Massachusetts Medical Society

lead_sponsor: Hoffmann-La Roche

sponsor_collaborators:
Hoffmann-La Roche
Chugai Pharmaceutical

location_country:
Germany
Italy
Poland
Hungary
United Kingdom
Japan
United States
Spain
Taiwan
Ukraine
France

location_facility: Hospital Clinico San Carlos; Servicio de Nefrologia

location_facility: PO G.Rodolico, AOU Policlinico-Vittorio Emanuele Catania

location_facility: M.A. - LEK A. M. Maciejowscy SC. Centrum Terapii SM

location_facility: Azienda Ospedaliera Sant'Andrea-Universitr di Roma La Sapien

location_facility: National Center of Neurology and Psychiatry

location_facility: St. Josef-Hospital, Klinik für Neurologie

location_facility: Niigata University Medical and Dental Hospital; Neurology

location_facility: Hopital de Hautepierre CHRU de Strasbourg

location_facility: Samodzielny Publiczny Szpital Kliniczny nr 4 w Lublinie

location_facility: National Cheng Kung University Hospital; Neurology

location_facility: Great Ormond Street Hospital For Children; Neurology

location_facility: Chang Gung Medical Foundation - LinKou Chang Gung Memorial Hospital - Neurology

location_facility: National Taiwan University Hospital; Neurology

location_facility: Szpital Kliniczny im. H.Swiecickiego UM w Poznaniu

location_facility: University Hospital of Wales; Dept of Neurology

location_facility: Kagoshima University Medical And Dental Hospital; Neurology and Geriatorics

location_facility: China Medical University Hospital; Neurology - Taichung

location_facility: Samodzielny Publiczny Centralny Szpital Klinicznyi

location_facility: Taipei Veterans General Hospital-Neurology

location_facility: NeuroCure Clinical Research Center (NCRC)

location_facility: Fukushima Medical University Hospital; Neurology

location_facility: The National Hospital for Neurology & Neurosurgery

location_facility: NZOZ Wielospecjalistyczna Poradnia Lekarska SYNAPSIS

location_facility: KZ "Dnipropetrovska oblasna dytiacha klinichna likarnia" DOR

location_facility: Tokyo Women's Medical University Hospital; Neurology


title: A Retrospective Analysis of Hematological Relapse Free Survival and Overall Survival in Adult Patients With Philadelphia-Negative B-Precursor Acute Lymphoblastic Leukemia in Complete Hematological Remission With Minimal Residual Disease

summary: A retrospective analysis of historical data looking at relapse free survival and overall survival rates in adult philadelphia negative B-pre-cursor acute lymphoblastic leukemia patients.

time_perspective: Retrospective

ReferenceCitation: Gokbuget N, Dombret H, Giebel S, Bruggemann M, Doubek M, Foa R, Hoelzer D, Kim C, Martinelli G, Parovichnikova E, Maria Ribera J, Schoonen M, Tuglus C, Zugmaier G, Bassan R. Blinatumomab vs historic standard-of-care treatment for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukaemia. Eur J Haematol. 2020 Apr;104(4):299-309. doi: 10.1111/ejh.13375. Epub 2020 Jan 24. Erratum In: Eur J Haematol. 2021 Apr;106(4):593.

sponsor_collaborators:
Amgen
European EWALL study groups

location_country:
Germany
Italy
Poland
United Kingdom
Spain
Czechia
Russian Federation
France


title: Multicenter Phase 2 Trial of ARQ 197 for Subjects With Relapsed or Refractory Germ Cell Tumors

summary: This is a multicenter, single-arm study for safety and efficacy.

lead_sponsor: Daiichi Sankyo, Inc.

location_country:
United States
United Kingdom
France


title: Retrospective Registry Providing Baseline Data on the Outcome of Left Atrial (LA) or LA Appendage (LAA) Thrombus in Patients With Nonvalvular Atrial Fibrillation (AF) or Atrial Flutter After Standard of Care (SoC) Anticoagulant Therapy

acronym: CLOT-AF

summary:
Thrombus outcome data will be collected retrospectively during 2011-2012 as a historical baseline of SoC with oral VKA (Vitamin K Antagonist(s) for the treatment of patients with nonvalvular AF or atrial flutter documented with LA/ LAA thrombi on transesophag-eal echocardiography (TEE).

The study is a company-sponsored, global, multi-center, retrospective, non-interventional study. Patients who suffered from hemodynamically stable nonvalvular AF or atrial flutter and had a diagnosed LA/ LAA thrombus between January 1st, 2011 and December 31st, 2012 will be identified through screening and review of medical records and included in the registry. Retrospective patient data will be collected from May 2nd, 2013 to May 2nd, 2014. The observation of each patient will cover the period from the diagnosis of an LA/ LAA thrombus until the end-of-treatment TEE following the 3-12 week SoC anticoagulation (AC) therapy. If no end-of-treatment TEE has been performed during 3-12 weeks of AC therapy, the observational period will end at 12 weeks after diagnosis at the latest. If more than one TEE was performed during treatment, the thrombus outcome will be collected from the last TEE performed within 12 weeks of treatment start.

ReferenceCitation: Lip GY, Hammerstingl C, Marin F, Cappato R, Meng IL, Kirsch B, Morandi E, van Eickels M, Cohen A. Rationale and design of a study exploring the efficacy of once-daily oral rivaroxaban (X-TRA) on the outcome of left atrial/left atrial appendage thrombus in nonvalvular atrial fibrillation or atrial flutter and a retrospective observational registry providing baseline data (CLOT-AF). Am Heart J. 2015 Apr;169(4):464-71.e2. doi: 10.1016/j.ahj.2014.12.020. Epub 2015 Jan 6.

repository_institution: University of Birmingham - University of Birmingham Research Portal

repositories_concat: Pure (Denmark)

lead_sponsor: Bayer

location_country:
Germany
Poland
Turkey
Bulgaria
Ukraine
Russian Federation
France


acronym: RESTORE

lead_sponsor: Edmond Pharma

summary: The aim of the study is to evaluate the effect of erdosteine, compared to placebo, on exacerbation rate over a 12-month treatment period in patients with moderate-to-severe COPD. Moreover, the effects of erdosteine on pulmonary function parameters, clinical symptoms and quality of life, and the long-term safety of the drug will be assessed.

ReferenceCitation: Dal Negro RW, Wedzicha JA, Iversen M, Fontana G, Page C, Cicero AF, Pozzi E, Calverley PMA; RESTORE group; RESTORE study. Effect of erdosteine on the rate and duration of COPD exacerbations: the RESTORE study. Eur Respir J. 2017 Oct 12;50(4):1700711. doi: 10.1183/13993003.00711-2017. Print 2017 Oct.

repository_institution: PubMed Central - Europe PMC

repository_institution: Imperial College London - Spiral

repository_institution: KU Leuven - Lirias

repository_normalized: LIRIAS (KU Leuven)

repositories_concat: LIRIAS (KU Leuven);PubMed Central;flore.unifi.it;spiral.imperial.ac.uk

repositories_institution: University of Florence - Florence Research

location_facility: Regional Respiratory Centre, Belfast City Hospital

location_facility: Clinic for emergency Medicine and Intensive care, Pneumology and Physiatry

location_facility: Katedra Pulmunologii i Alergologii, Klinika Gruźlicy Uniwersytetu Medycznego

location_facility: Nemocnica s Poliklinicou "Svateho Jakuba"n.o.

location_facility: Department of Respiratory Diseases, Copenhagen University Hospital

location_facility: Katedra i klinika Pulmonologii, Uniwersytet Medyczny

location_facility: UOC di Pneumologia-Ospedale Mater Salutis

location_facility: Ospedale Careggi, Malattie dell'Apparato Respiratorio

location_facility: Campus Gasthuisberg Dep.t Longfunctiemetingen

location_facility: Service de Pneumologie, Hopital Bois-Guillaume

location_facility: Clinica Pneumologie I, Spitalul Clinic Judetean Mureş

location_facility: UOC di Pneumologia- Ospedale Civile di Massa e Carrara

location_facility: Institutul de Pneumoftiziologie "Marius Nasta"

location_facility: Klinika Pulmonologii II Katedry, Collegium Medicum Uniwersytetu

location_facility: Cardiovascular and Respiratory Studies Department

location_facility: Spitalul de Pneumoftiziologie "Leon Daniello"

location_facility: Ospedale San Carlo Borromeo, Dipartimento Broncocardiopneumologico

location_facility: Specializovana NemocnicaSvorada Zobor n.o.

location_facility: Spitalul de Pneumoftiziologie "Victor Babes"

location_facility: Department of Respiratory Medicine, Faculty Hospital

location_facility: Katedra i Klinika Pneumonologii, Warszawski Uniwersytet Medyczny

location_facility: Aintree Chest Centre, University Hospital Aintree

location_facility: Clinic of Pulmonary Disease-Military Medical Academy

title: The Efficacy and Safety of Erdosteine in the Long-term Therapy of Chronic Obstructive Pulmonary Disease (COPD). A 12-month, Randomised, Double-blind, Placebo-controlled, Parallel Group, Multicenter Study

location_country:
Italy
Poland
Slovakia
United Kingdom
Denmark
Romania
Belgium
Bulgaria
Czech Republic
France


title: A Phase 3, Randomized, Controlled, Multi-Center, Open-Label Study to Compare Tivozanib (AV-951) to Sorafenib in Subjects With Advanced Renal Cell Carcinoma (TIVO-1)

summary: This is an open-label, randomized, controlled, multi-national, multi-center, parallel-arm trial comparing tivozanib to sorafenib in subjects with advanced RCC. The study is designed to compare the PFS, OS, ORR, DR, safety and tolerability, and kidney specific symptoms/health outcome measurements of tivozanib and sorafenib.

lead_sponsor: AVEO Pharmaceuticals, Inc.

location_country:
Italy
Poland
India
Hungary
Argentina
Romania
United Kingdom
United States
Chile
Czechia
Ukraine
Bulgaria
Canada
Serbia
Russian Federation
France

location_facility:
Site 492
Site 453
Site 451
Site 153
Site 490
Site 452
Site 497
Site 493
Site 483
Site 494
Site 457
Site 159
Site 133
Site 462
Site 122
Site 162
Site 491
Site 187
Site 464
Site 403
Site 466
Site 152
Site 170
Site 185
Site 121
Site 190
Site 154
Site 484
Site 480
Site 157
Site 461
Site 151
Site 158
Site 430
Site 444
Site 102
Site 458
Site 431
Site 435
Site 172
Site 180
Site 463
Site 498
Site 441
Site 443
Site 411
Site 110
Site 401
Site 467
Site 481
Site 130
Site 442
Site 434
Site 191
Site 123
Site 424
Site 456
Site 423
Site 482
Site 436
Site 432
Site 400
Site 184
Site 404
Site 422
Site 421
Site 433
Site 182
Site 459
Site 160
Site 173
Site 468
Site 402
Site 150
Site 440
Site 455
Site 450
Site 496
Site 186
Site 465
Site 156
Site 155
Site 454
Site 495
Site 161
Site 460


acronym: B-Long

lead_sponsor: Bioverativ Therapeutics Inc.

summary: The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy by comparing the annualized number of bleeding episodes between participants receiving rFIXFc on each prevention (prophylaxis) regimen and participants receiving rFIXFc on an episodic regimen. The secondary objectives of the study were: to evaluate and assess the pharmacokinetic (PK) parameter estimates of rFIXFc and rFIX (BeneFIX®) at baseline in the Sequential PK subgroup as well as rFIXFc at Week 26 (±1 week); to evaluate participants' response to treatment; to evaluate rFIXFc consumption.

ReferenceCitation: Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4.

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

sponsor_collaborators:
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

location_facility:
Ressearch Site
Research site
Research Site

title: B-LONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant, Long-acting Coagulation Factor IX Fc Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia B

location_country:
Germany
Italy
Poland
India
United Kingdom
Hong Kong
Australia
Brazil
Japan
Sweden
Belgium
United States
China
South Africa
Canada
Russian Federation
France


acronym: Safety and efficacy of positron emission tomography imaging with BAY 94-9172 (ZK 6013443)

lead_sponsor: Life Molecular Imaging SA

summary: To determine the sensitivity and specificity of the visual assessment of tracer uptake in the Florbetaben PET images compared to histological verification of the presence or absence of cerebral β-amyloid in the respective histopathologic post mortem specimens as the standard of truth

ReferenceCitation: Seibyl J, Catafau AM, Barthel H, Ishii K, Rowe CC, Leverenz JB, Ghetti B, Ironside JW, Takao M, Akatsu H, Murayama S, Bullich S, Mueller A, Koglin N, Schulz-Schaeffer WJ, Hoffmann A, Sabbagh MN, Stephens AW, Sabri O. Impact of Training Method on the Robustness of the Visual Assessment of 18F-Florbetaben PET Scans: Results from a Phase-3 Study. J Nucl Med. 2016 Jun;57(6):900-6. doi: 10.2967/jnumed.115.161927. Epub 2016 Jan 28.

repository_institution: University of Edinburgh - Edinburgh Research Explorer

repository_institution: Indiana University - Purdue University Indianapolis - IUPUI ScholarWorks

repository_institution: Indiana University - IUScholarWorks

publisher_dissemination: Society of Nuclear Medicine

ReferenceCitation: Sabri O, Sabbagh MN, Seibyl J, Barthel H, Akatsu H, Ouchi Y, Senda K, Murayama S, Ishii K, Takao M, Beach TG, Rowe CC, Leverenz JB, Ghetti B, Ironside JW, Catafau AM, Stephens AW, Mueller A, Koglin N, Hoffmann A, Roth K, Reininger C, Schulz-Schaeffer WJ; Florbetaben Phase 3 Study Group. Florbetaben PET imaging to detect amyloid beta plaques in Alzheimer's disease: phase 3 study. Alzheimers Dement. 2015 Aug;11(8):964-74. doi: 10.1016/j.jalz.2015.02.004. Epub 2015 Mar 28.

title: An Open-label, Non-randomized Study to Evaluate the Efficacy and Safety of BAY94-9172 (ZK 6013443) Positron Emission Tomography (PET) Imaging for Detection/Exclusion of Cerebral Beta-amyloid When Compared to Postmortem Histopathology

primary_purpose: Diagnostic

location_country:
Germany
Australia
Japan
United States
France


acronym: EMERGE

lead_sponsor: ZymoGenetics

summary: Interleukin 29 (IL-29) is a substance that is produced in the body to help fight viral infections. The purpose of this study is to evaluate the safety and antiviral effects of several different doses of PEG-rIL-29 (a man-made form of IL-29) when it is given in combination with daily oral doses of ribavirin (an antiviral drug) to subjects with hepatitis C infection who have received no prior treatment for this disease.

ReferenceCitation: Wang X, Hruska M, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 1: Modeling optimal treatment duration and sustained virologic response rates. J Clin Pharmacol. 2015 Jan;55(1):63-72. doi: 10.1002/jcph.363. Epub 2014 Jul 24.

ReferenceCitation: Hruska M, Wang X, Chan P, Ahmad A, Freeman J, Horga MA, Hillson J, Kansra V, Lopez-Talavera JC. Derivation of Phase 3 dosing for peginterferon lambda-1a in chronic hepatitis C, Part 2: Exposure-response analyses for efficacy and safety variables. J Clin Pharmacol. 2015 Jan;55(1):73-80. doi: 10.1002/jcph.361. Epub 2014 Jul 24.

sponsor_collaborators:
ZymoGenetics
Bristol-Myers Squibb

title: Randomized, Controlled Phase 2a/b Study of the Efficacy and Safety of PEG-rIL-29 Administered in Combination With Ribavirin to Treatment-Naive Subjects With Chronic Hepatitis C Virus Infection

location_country:
Germany
Italy
Poland
Romania
Australia
Austria
United States
Spain
Canada
Puerto Rico
France


acronym: TULIPIA

lead_sponsor: Ipsen

summary: The purpose of this study is to assess the efficacy of repeated subcutaneous (under the skin) injections at different doses of BIM 23A760 on growth hormone and insulin-like growth factor-1 levels in patients with acromegaly after 6 months of treatment.

location_facility: Massachusetts General Hospital / Neuroendocrine Unit

location_facility: Insituto Nacional de Neurologia y Neurocirugia

location_facility: Vilnius University Hospital "Santariskiu Klinikos"

location_facility: UIM Endocrinología Experimental, Hospital de Especialidades

location_facility: University Hospital Olomouc, Clinic of Internal Medicine

location_facility: National Medical University n.a. M.I.Pirogov

location_facility: Administration of Medical Services and Rehabilitation of "ARTEM"

location_facility: Centre Hospitalier Universitaire de Liège

location_facility: General University Hospital, Clinic of Internal Medicine,

location_facility: Oregon University, Dept. of Endocrinology and Neurosurgery

location_facility: Academy of Medical Science of Ukraine, Department of Clinical Endocrinology

location_facility: "C.I. Parhon" National Institute of Endocrinology

location_facility: Hospital Universitário Clementino Fraga Filho

location_facility: P. Stradins Clinical University Hospital

title: Phase II, Open, Randomised, Parallel Group, Noncomparative Multicentre Study to Assess the Efficacy and Safety of Repeated Subcutaneous (S.C.) Administration of Different Doses of BIM 23A760 in Acromegalic Patients

location_country:
Lithuania
Poland
Romania
Brazil
Netherlands
Mexico
Belgium
Sweden
United States
Latvia
Czechia
Ukraine
France