title: Automated Pupillometry for Coma Prognostication After Cardiac Arrest

summary:
Background: Sedation and therapeutic hypothermia (TH) delay neurological responses and might reduce the accuracy of clinical examination to predict outcome after cardiac arrest (CA). Electroencephalography (EEG) and somato-sensory evoked potentials (SSEP) might significantly improve prognostication of post-CA coma, however, EEG and SSEP are not always available and require specific expertise for their interpretation. Automated video pupillometry is a novel electronic device that contains an infrared light camera which enables to measure quantitatively the percentage of pupillary reaction to a calibrated light stimulation. In a recent study of a cohort of comatose CA survivors (n=50 patients) it was found that quantitative PLR was more accurate than standard PLR (manual pen light) in predicting 3-month outcome, irrespective of temperature and sedation, and had comparable prognostic accuracy than electrophysiological exams, including electroencephalography (EEG) and somato-sensory evoked potentials (SSEP).

Aim of the study: In light of these promising results, the investigators would like to confirm the prognostic value of quantitative PLR in a large multicenter cohort of comatose post-CA patients.

Design of the study: Prospective, multicenter, observational outcome trial.

lead_sponsor: Centre Hospitalier Universitaire Vaudois

collaborators: Azienda Ospedaliera San Gerardo di Monza

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

location_country: Germany Italy Sweden Netherlands Switzerland Belgium Luxembourg France


type: Registry Identifier

title: Deep Brain Stimulation (DBS) of the Globus Pallidus (GP) in Huntington's Disease (HD): A Prospective, Randomised, Controlled, International, Multi-centre Study

acronym: HD-DBS

summary: The aim of the study is to prove the efficacy and safety of pallidal DBS in HD patients and to show superiority of DBS on motor function in the stimulation group compared to stimulation-off group

study_start_date_type: Actual

status: Completed

study_completion_date_type: Actual

study_type: Interventional

design_allocation: Randomized

primary_purpose: Treatment

enrollment_type: Actual

ReferenceType: background

ReferenceCitation: Moro E, Lang AE, Strafella AP, Poon YY, Arango PM, Dagher A, Hutchison WD, Lozano AM. Bilateral globus pallidus stimulation for Huntington's disease. Ann Neurol. 2004 Aug;56(2):290-4. doi: 10.1002/ana.20183.

ReferenceCitation: Groiss SJ WL, Suedmeyer, M, Ploner M, Reck C, Voges J, SturmV, Timmermann L, Schnitzler A. Effect of bilateral pallidal deep brain stimulation in Huntington's disease: A case report. Mov Disord, Volume 21, Issue S15. Tenth International Congress of Parkinson's Disease and Movement Disorders. Kyoto 2006.

ReferenceCitation: Fasano A, Cadeddu F, Guidubaldi A, Piano C, Soleti F, Zinzi P, Bentivoglio AR. The long-term effect of tetrabenazine in the management of Huntington disease. Clin Neuropharmacol. 2008 Nov-Dec;31(6):313-8. doi: 10.1097/WNF.0b013e318166da60.

ReferenceCitation: Wojtecki L, Groiss SJ, Ferrea S, Elben S, Hartmann CJ, Dunnett SB, Rosser A, Saft C, Sudmeyer M, Ohmann C, Schnitzler A, Vesper J; Surgical Approaches Working Group of the European Huntington's Disease Network (EHDN). A Prospective Pilot Trial for Pallidal Deep Brain Stimulation in Huntington's Disease. Front Neurol. 2015 Aug 18;6:177. doi: 10.3389/fneur.2015.00177. eCollection 2015.

ReferenceCitation: Wojtecki L, Groiss SJ, Hartmann CJ, Elben S, Omlor S, Schnitzler A, Vesper J. Deep Brain Stimulation in Huntington's Disease-Preliminary Evidence on Pathophysiology, Efficacy and Safety. Brain Sci. 2016 Aug 30;6(3):38. doi: 10.3390/brainsci6030038.

ReferenceType: derived

ReferenceCitation: Rodrigues FB, Ferreira JJ, Wild EJ. Huntington's Disease Clinical Trials Corner: June 2019. J Huntingtons Dis. 2019;8(3):363-371. doi: 10.3233/JHD-199003.

oa_colors: green hybrid

oa_host_type: publisher;repository

evidence: open (via page says Open Access)

host_type: publisher

license: unspecified-oa

license_normalized: unspecified-oa

version: publishedVersion

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

host_type: repository

license: cc-by-nc

license_normalized: cc-by-nc

repository_institution: PubMed Central - Europe PMC

repository_normalized: PubMed Central

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

repository_institution: University College London - UCL Discovery

repository_normalized: UCL Discovery

evidence: oa repository (via pmcid lookup)

license_normalized: no license

repositories:
PubMed Central
UCL Discovery

repositories_concat: PubMed Central;UCL Discovery

unpaywall_oa_status: hybrid

publisher_dissemination: IOS Press

ReferenceCitation: Hartmann CJ, Groiss SJ, Vesper J, Schnitzler A, Wojtecki L. Brain stimulation in Huntington's disease. Neurodegener Dis Manag. 2016 Jun;6(3):223-36. doi: 10.2217/nmt-2016-0007. Epub 2016 May 27.

oa_host_type: closed

repositories_concat: closed

publisher_dissemination: Future Science Ltd

ipd_sharing: Yes

ipd_sharing_description: After the study the data will be provided to the CHDI. The Foundation may use, and make available for use by the Foundation Collaborators, the Study Data for the following purposes: (A) to design and guide future research studies and clinical trials and (B) to support and enable the following scientific discussion and research: (1) to better understand HD or other diseases being studied, (2) that furthers the development of treatments for HD or other diseases or (3) that furthers biomedical research.

lead_sponsor: Heinrich-Heine University, Duesseldorf

collaborators: Egyptian Society of Neurological Surgeons

location_country: Germany Switzerland Austria France

location_facility: University hospital Heinrich Heine University Düsseldorf

location_facility: CHU Amiens Hôpital nord, Department of neurosurgery and Department of neurology

location_facility: Hôpital Roger Salengro, Service de Neurologie et Pathologie du mouvement

intervention_type: Device

lead_sponsor_type: academique

status_simplified: Completed


title: Type 3 Von Willebrand International Registries Inhibitor Prospective Study

summary: International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand Factor (VWF) concentrates used to treat VWD patients.

status: Active, not recruiting

study_completion_date_type: Anticipated

study_type: Observational

time_perspective: Other

ReferenceCitation: Eikenboom JC. Congenital von Willebrand disease type 3: clinical manifestations, pathophysiology and molecular biology. Best Pract Res Clin Haematol. 2001 Jun;14(2):365-79. doi: 10.1053/beha.2001.0139.

ReferenceCitation: Sadler JE, Budde U, Eikenboom JC, Favaloro EJ, Hill FG, Holmberg L, Ingerslev J, Lee CA, Lillicrap D, Mannucci PM, Mazurier C, Meyer D, Nichols WL, Nishino M, Peake IR, Rodeghiero F, Schneppenheim R, Ruggeri ZM, Srivastava A, Montgomery RR, Federici AB; Working Party on von Willebrand Disease Classification. Update on the pathophysiology and classification of von Willebrand disease: a report of the Subcommittee on von Willebrand Factor. J Thromb Haemost. 2006 Oct;4(10):2103-14. doi: 10.1111/j.1538-7836.2006.02146.x. Epub 2006 Aug 2.

ReferenceCitation: Nilsson IM. Commentary to Erik von Willebrand's original paper from 1926 'Hereditar pseudohemofili'. Haemophilia. 1999 May;5(3):220-1. doi: 10.1046/j.1365-2516.1999.0320a.x. No abstract available.

ReferenceCitation: NILSSON IM, BLOMBACK M, BLOMBACK B. v. Willebrand's disease in Sweden; its pathogenesis and treatment. Acta Med Scand. 1959 Jun 30;164:263-78. No abstract available.

ReferenceCitation: Mannucci PM, Bloom AL, Larrieu MJ, Nilsson IM, West RR. Atherosclerosis and von Willebrand factor. I. Prevalence of severe von Willebrand's disease in western Europe and Israel. Br J Haematol. 1984 May;57(1):163-9. doi: 10.1111/j.1365-2141.1984.tb02876.x.

ReferenceCitation: Weiss HJ, Ball AP, Mannucci PM. Incidence of severe von Willebrand's disease. N Engl J Med. 1982 Jul 8;307(2):127. doi: 10.1056/NEJM198207083070222. No abstract available.

ReferenceCitation: Berliner SA, Seligsohn U, Zivelin A, Zwang E, Sofferman G. A relatively high frequency of severe (type III) von Willebrand's disease in Israel. Br J Haematol. 1986 Mar;62(3):535-43. doi: 10.1111/j.1365-2141.1986.tb02966.x.

ReferenceCitation: Iorio A, Oliovecchio E, Morfini M, Mannucci PM; Association of Italian Hemophilia Centres Directors. Italian Registry of Haemophilia and Allied Disorders. Objectives, methodology and data analysis. Haemophilia. 2008 May;14(3):444-53. doi: 10.1111/j.1365-2516.2008.01679.x. Epub 2008 Mar 18. Erratum In: Haemophilia. 2009 Mar;15(2):637.

ReferenceCitation: Federici AB, Mannucci PM. Management of inherited von Willebrand disease in 2007. Ann Med. 2007;39(5):346-58. doi: 10.1080/07853890701513738.

ReferenceCitation: Silwer J. von Willebrand's disease in Sweden. Acta Paediatr Scand Suppl. 1973;238:1-159. No abstract available.

ReferenceCitation: Lak M, Peyvandi F, Mannucci PM. Clinical manifestations and complications of childbirth and replacement therapy in 385 Iranian patients with type 3 von Willebrand disease. Br J Haematol. 2000 Dec;111(4):1236-9. doi: 10.1046/j.1365-2141.2000.02507.x.

ReferenceCitation: Federici AB, Castaman G, Mannucci PM; Italian Association of Hemophilia Centers (AICE). Guidelines for the diagnosis and management of von Willebrand disease in Italy. Haemophilia. 2002 Sep;8(5):607-21. doi: 10.1046/j.1365-2516.2002.00672.x.

ReferenceCitation: Rodeghiero F, Castaman G, Tosetto A, Batlle J, Baudo F, Cappelletti A, Casana P, De Bosch N, Eikenboom JC, Federici AB, Lethagen S, Linari S, Srivastava A. The discriminant power of bleeding history for the diagnosis of type 1 von Willebrand disease: an international, multicenter study. J Thromb Haemost. 2005 Dec;3(12):2619-26. doi: 10.1111/j.1538-7836.2005.01663.x. Erratum In: J Thromb Haemost. 2006 Apr;4(4):925.

ReferenceCitation: Tosetto A, Rodeghiero F, Castaman G, Goodeve A, Federici AB, Batlle J, Meyer D, Fressinaud E, Mazurier C, Goudemand J, Eikenboom J, Schneppenheim R, Budde U, Ingerslev J, Vorlova Z, Habart D, Holmberg L, Lethagen S, Pasi J, Hill F, Peake I. A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD). J Thromb Haemost. 2006 Apr;4(4):766-73. doi: 10.1111/j.1538-7836.2006.01847.x.

ReferenceCitation: Cattaneo M, Federici AB, Lecchi A, Agati B, Lombardi R, Stabile F, Bucciarelli P. Evaluation of the PFA-100 system in the diagnosis and therapeutic monitoring of patients with von Willebrand disease. Thromb Haemost. 1999 Jul;82(1):35-9.

ReferenceCitation: Mannucci PM, Lattuada A, Castaman G, Lombardi R, Colibretti ML, Ciavarella N, Rodeghiero F. Heterogeneous phenotypes of platelet and plasma von Willebrand factor in obligatory heterozygotes for severe von Willebrand disease. Blood. 1989 Nov 15;74(7):2433-6.

ReferenceCitation: Mannucci PM, Federici AB. Antibodies to von Willebrand factor in von Willebrand disease. Adv Exp Med Biol. 1995;386:87-92. doi: 10.1007/978-1-4613-0331-2_7. No abstract available.

ReferenceCitation: Federici AB. Clinical and molecular markers of inherited von Willebrand disease type 3: are deletions of the VWF gene associated with alloantibodies to VWF? J Thromb Haemost. 2008 Oct;6(10):1726-8. doi: 10.1111/j.1538-7836.2008.03146.x. No abstract available.

ReferenceCitation: Veltkamp JJ, van Tilburg NH. Detection of heterozygotes for recessive von Willebrand's disease by the assay of antihemophilic-factor-like antigen. N Engl J Med. 1973 Oct 25;289(17):882-5. doi: 10.1056/NEJM197310252891703. No abstract available.

ReferenceCitation: Shelton-Inloes BB, Chehab FF, Mannucci PM, Federici AB, Sadler JE. Gene deletions correlate with the development of alloantibodies in von Willebrand disease. J Clin Invest. 1987 May;79(5):1459-65. doi: 10.1172/JCI112974.

ReferenceCitation: Ngo KY, Glotz VT, Koziol JA, Lynch DC, Gitschier J, Ranieri P, Ciavarella N, Ruggeri ZM, Zimmerman TS. Homozygous and heterozygous deletions of the von Willebrand factor gene in patients and carriers of severe von Willebrand disease. Proc Natl Acad Sci U S A. 1988 Apr;85(8):2753-7. doi: 10.1073/pnas.85.8.2753.

ReferenceCitation: Schneppenheim R, Krey S, Bergmann F, Bock D, Budde U, Lange M, Linde R, Mittler U, Meili E, Mertes G, et al. Genetic heterogeneity of severe von Willebrand disease type III in the German population. Hum Genet. 1994 Dec;94(6):640-52. doi: 10.1007/BF00206958.

ReferenceCitation: Eikenboom JC, Castaman G, Vos HL, Bertina RM, Rodeghiero F. Characterization of the genetic defects in recessive type 1 and type 3 von Willebrand disease patients of Italian origin. Thromb Haemost. 1998 Apr;79(4):709-17.

ReferenceCitation: Schneppenheim R, Castaman G, Federici AB, Kreuz W, Marschalek R, Oldenburg J, Oyen F, Budde U. A common 253-kb deletion involving VWF and TMEM16B in German and Italian patients with severe von Willebrand disease type 3. J Thromb Haemost. 2007 Apr;5(4):722-8. doi: 10.1111/j.1538-7836.2007.02460.x. Epub 2007 Feb 26.

ReferenceCitation: Peake IR, Liddell MB, Moodie P, Standen G, Mancuso DJ, Tuley EA, Westfield LA, Sorace JM, Sadler JE, Verweij CL, et al. Severe type III von Willebrand's disease caused by deletion of exon 42 of the von Willebrand factor gene: family studies that identify carriers of the condition and a compound heterozygous individual. Blood. 1990 Feb 1;75(3):654-61.

ReferenceCitation: Mancuso DJ, Tuley EA, Castillo R, de Bosch N, Mannucci PM, Sadler JE. Characterization of partial gene deletions in type III von Willebrand disease with alloantibody inhibitors. Thromb Haemost. 1994 Aug;72(2):180-5.

ReferenceCitation: Baronciani L, Cozzi G, Canciani MT, Peyvandi F, Srivastava A, Federici AB, Mannucci PM. Molecular characterization of a multiethnic group of 21 patients with type 3 von Willebrand disease. Thromb Haemost. 2000 Oct;84(4):536-40.

ReferenceCitation: Mohl A, Marschalek R, Masszi T, Nagy E, Obser T, Oyen F, Sallai K, Bodo I, Schneppenheim R. An Alu-mediated novel large deletion is the most frequent cause of type 3 von Willebrand disease in Hungary. J Thromb Haemost. 2008 Oct;6(10):1729-35. doi: 10.1111/j.1538-7836.2008.03107.x. Epub 2008 Jul 28.

ReferenceCitation: Xie F, Wang X, Cooper DN, Chuzhanova N, Fang Y, Cai X, Wang Z, Wang H. A novel Alu-mediated 61-kb deletion of the von Willebrand factor (VWF) gene whose breakpoints co-locate with putative matrix attachment regions. Blood Cells Mol Dis. 2006 May-Jun;36(3):385-91. doi: 10.1016/j.bcmd.2006.03.003. Epub 2006 May 11.

ReferenceCitation: Cooper DN, Krawczak M. The mutational spectrum of single base-pair substitutions causing human genetic disease: patterns and predictions. Hum Genet. 1990 Jun;85(1):55-74. doi: 10.1007/BF00276326.

ReferenceCitation: Zhang ZP, Lindstedt M, Falk G, Blomback M, Egberg N, Anvret M. Nonsense mutations of the von Willebrand factor gene in patients with von Willebrand disease type III and type I. Am J Hum Genet. 1992 Oct;51(4):850-8.

ReferenceCitation: Eikenboom JC, Ploos van Amstel HK, Reitsma PH, Briet E. Mutations in severe, type III von Willebrand's disease in the Dutch population: candidate missense and nonsense mutations associated with reduced levels of von Willebrand factor messenger RNA. Thromb Haemost. 1992 Oct 5;68(4):448-54.

ReferenceCitation: Bahnak BR, Lavergne JM, Rothschild C, Meyer D. A stop codon in a patient with severe type III von Willebrand disease. Blood. 1991 Aug 15;78(4):1148-9. No abstract available.

ReferenceCitation: Zhang ZP, Falk G, Blomback M, Egberg N, Anvret M. Identification of a new nonsense mutation in the von Willebrand factor gene in patients with von Willebrand disease type III. Hum Mol Genet. 1992 Apr;1(1):61-2. doi: 10.1093/hmg/1.1.61. No abstract available.

ReferenceCitation: Zhang ZP, Blomback M, Egberg N, Falk G, Anvret M. Characterization of the von Willebrand factor gene (VWF) in von Willebrand disease type III patients from 24 families of Swedish and Finnish origin. Genomics. 1994 May 1;21(1):188-93. doi: 10.1006/geno.1994.1241.

ReferenceCitation: Hagiwara T, Inaba H, Yoshida S, Nagaizumi K, Arai M, Hanabusa H, Fukutake K. A novel mutation Gly 1672-->Arg in type 2A and a homozygous mutation in type 2B von Willebrand disease. Thromb Haemost. 1996 Aug;76(2):253-7.

ReferenceCitation: Gupta PK, Saxena R, Adamtziki E, Budde U, Oyen F, Obser T, Schneppenheim R. Genetic defects in von Willebrand disease type 3 in Indian and Greek patients. Blood Cells Mol Dis. 2008 Sep-Oct;41(2):219-22. doi: 10.1016/j.bcmd.2008.03.004. Epub 2008 May 16.

ReferenceCitation: Casana P, Martinez F, Haya S, Lorenzo JI, Espinos C, Aznar JA. Q1311X: a novel nonsense mutation of putative ancient origin in the von Willebrand factor gene. Br J Haematol. 2000 Nov;111(2):552-5. doi: 10.1046/j.1365-2141.2000.02410.x.

ReferenceCitation: Surdhar GK, Enayat MS, Lawson S, Williams MD, Hill FG. Homozygous gene conversion in von Willebrand factor gene as a cause of type 3 von Willebrand disease and predisposition to inhibitor development. Blood. 2001 Jul 1;98(1):248-50. doi: 10.1182/blood.v98.1.248. No abstract available.

ReferenceCitation: Baronciani L, Cozzi G, Canciani MT, Peyvandi F, Srivastava A, Federici AB, Mannucci PM. Molecular defects in type 3 von Willebrand disease: updated results from 40 multiethnic patients. Blood Cells Mol Dis. 2003 May-Jun;30(3):264-70. doi: 10.1016/s1079-9796(03)00033-0.

ReferenceCitation: Gupta PK, Adamtziki E, Budde U, Jaiprakash M, Kumar H, Harbeck-Seu A, Kannan M, Oyen F, Obser T, Wedekind I, Saxena R, Schneppenheim R. Gene conversions are a common cause of von Willebrand disease. Br J Haematol. 2005 Sep;130(5):752-8. doi: 10.1111/j.1365-2141.2005.05660.x.

ReferenceCitation: Eikenboom JC, Vink T, Briet E, Sixma JJ, Reitsma PH. Multiple substitutions in the von Willebrand factor gene that mimic the pseudogene sequence. Proc Natl Acad Sci U S A. 1994 Mar 15;91(6):2221-4. doi: 10.1073/pnas.91.6.2221.

ReferenceCitation: Zhang ZP, Falk G, Blomback M, Egberg N, Anvret M. A single cytosine deletion in exon 18 of the von Willebrand factor gene is the most common mutation in Swedish vWD type III patients. Hum Mol Genet. 1992 Dec;1(9):767-8. doi: 10.1093/hmg/1.9.767. No abstract available.

ReferenceCitation: Xie F, Wang X, Cooper DN, Lan F, Fang Y, Cai X, Wang Z, Wang H. Compound heterozygosity for two novel mutations (1203insG/Y1456X) in the von Willebrand factor gene causing type 3 von Willebrand disease. Haemophilia. 2007 Sep;13(5):645-8. doi: 10.1111/j.1365-2516.2007.01514.x.

ReferenceCitation: Wetzstein V, Budde U, Oyen F, Ding X, Herrmann J, Liebig B, Schneppenheim R. Intracranial hemorrhage in a term newborn with severe von Willebrand disease type 3 associated with sinus venous thrombosis. Haematologica. 2006 Dec;91(12 Suppl):ECR60.

ReferenceCitation: Krawczak M, Cooper DN. Gene deletions causing human genetic disease: mechanisms of mutagenesis and the role of the local DNA sequence environment. Hum Genet. 1991 Mar;86(5):425-41. doi: 10.1007/BF00194629.

ReferenceCitation: Efstratiadis A, Posakony JW, Maniatis T, Lawn RM, O'Connell C, Spritz RA, DeRiel JK, Forget BG, Weissman SM, Slightom JL, Blechl AE, Smithies O, Baralle FE, Shoulders CC, Proudfoot NJ. The structure and evolution of the human beta-globin gene family. Cell. 1980 Oct;21(3):653-68. doi: 10.1016/0092-8674(80)90429-8.

ReferenceCitation: Zhang ZP, Blomback M, Nyman D, Anvret M. Mutations of von Willebrand factor gene in families with von Willebrand disease in the Aland Islands. Proc Natl Acad Sci U S A. 1993 Sep 1;90(17):7937-40. doi: 10.1073/pnas.90.17.7937.

ReferenceCitation: Mohlke KL, Nichols WC, Rehemtulla A, Kaufman RJ, Fagerstrom HM, Ritvanen KL, Kekomaki R, Ginsburg D. A common frameshift mutation in von Willebrand factor does not alter mRNA stability but interferes with normal propeptide processing. Br J Haematol. 1996 Oct;95(1):184-91. doi: 10.1046/j.1365-2141.1996.7572377.x.

ReferenceCitation: Eikenboom JC, Reitsma PH, van der Velden PA, Briet E. Instability of repeats of the von Willebrand factor gene variable number tandem repeat sequence in intron 40. Br J Haematol. 1993 Jul;84(3):533-5. doi: 10.1111/j.1365-2141.1993.tb03114.x.

ReferenceCitation: Antonarakis SE. Recommendations for a nomenclature system for human gene mutations. Nomenclature Working Group. Hum Mutat. 1998;11(1):1-3. doi: 10.1002/(SICI)1098-1004(1998)11:13.0.CO;2-O. No abstract available.

ReferenceCitation: Mertes G, Ludwig M, Finkelnburg B, Krawczak M, Schwaab R, Brackmann HH, Olek K. A G+3-to-T donor splice site mutation leads to skipping of exon 50 in von Willebrand factor mRNA. Genomics. 1994 Nov 1;24(1):190-1. doi: 10.1006/geno.1994.1602. No abstract available.

ReferenceCitation: Zhang Z, Lindstedt M, Blomback M, Anvret M. Effects of the mutant von Willebrand factor gene in von Willebrand disease. Hum Genet. 1995 Oct;96(4):388-94. doi: 10.1007/BF00191794.

ReferenceCitation: Allen S, Abuzenadah AM, Hinks J, Blagg JL, Gursel T, Ingerslev J, Goodeve AC, Peake IR, Daly ME. A novel von Willebrand disease-causing mutation (Arg273Trp) in the von Willebrand factor propeptide that results in defective multimerization and secretion. Blood. 2000 Jul 15;96(2):560-8.

ReferenceCitation: Castaman G, Lattuada A, Mannucci PM, Rodeghiero F. Factor VIII:C increases after desmopressin in a subgroup of patients with autosomal recessive severe von Willebrand disease. Br J Haematol. 1995 Jan;89(1):147-51. doi: 10.1111/j.1365-2141.1995.tb08921.x.

ReferenceCitation: Tjernberg P, Castaman G, Vos HL, Bertina RM, Eikenboom JC. Homozygous C2362F von Willebrand factor induces intracellular retention of mutant von Willebrand factor resulting in autosomal recessive severe von Willebrand disease. Br J Haematol. 2006 May;133(4):409-18. doi: 10.1111/j.1365-2141.2006.06055.x.

ReferenceCitation: Castaman G, Eikenboom JC, Lattuada A, Mannucci PM, Rodeghiero F. Heightened proteolysis of the von Willebrand factor subunit in patients with von Willebrand disease hemizygous or homozygous for the C2362F mutation. Br J Haematol. 2000 Jan;108(1):188-90. doi: 10.1046/j.1365-2141.2000.01807.x.

ReferenceCitation: Baronciani L, Federici AB, Cozzi G, La Marca S, Punzo M, Rubini V, Canciani MT, Mannucci PM. Expression studies of missense mutations p.D141Y, p.C275S located in the propeptide of von Willebrand factor in patients with type 3 von Willebrand disease. Haemophilia. 2008 May;14(3):549-55. doi: 10.1111/j.1365-2516.2008.01682.x. Epub 2008 Mar 4.

ReferenceCitation: Voorberg J, Fontijn R, Calafat J, Janssen H, van Mourik JA, Pannekoek H. Assembly and routing of von Willebrand factor variants: the requirements for disulfide-linked dimerization reside within the carboxy-terminal 151 amino acids. J Cell Biol. 1991 Apr;113(1):195-205. doi: 10.1083/jcb.113.1.195.

ReferenceCitation: Peake IR, Bowen D, Bignell P, Liddell MB, Sadler JE, Standen G, Bloom AL. Family studies and prenatal diagnosis in severe von Willebrand disease by polymerase chain reaction amplification of a variable number tandem repeat region of the von Willebrand factor gene. Blood. 1990 Aug 1;76(3):555-61.

ReferenceCitation: Federici AB, Mazurier C, Berntorp E, Lee CA, Scharrer I, Goudemand J, Lethagen S, Nitu I, Ludwig G, Hilbert L, Mannucci PM. Biologic response to desmopressin in patients with severe type 1 and type 2 von Willebrand disease: results of a multicenter European study. Blood. 2004 Mar 15;103(6):2032-8. doi: 10.1182/blood-2003-06-2072. Epub 2003 Nov 20.

ReferenceCitation: Castaman G, Lethagen S, Federici AB, Tosetto A, Goodeve A, Budde U, Batlle J, Meyer D, Mazurier C, Fressinaud E, Goudemand J, Eikenboom J, Schneppenheim R, Ingerslev J, Vorlova Z, Habart D, Holmberg L, Pasi J, Hill F, Peake I, Rodeghiero F. Response to desmopressin is influenced by the genotype and phenotype in type 1 von Willebrand disease (VWD): results from the European Study MCMDM-1VWD. Blood. 2008 Apr 1;111(7):3531-9. doi: 10.1182/blood-2007-08-109231. Epub 2008 Jan 29.

ReferenceCitation: Cattaneo M, Moia M, Delle Valle P, Castellana P, Mannucci PM. DDAVP shortens the prolonged bleeding times of patients with severe von Willebrand disease treated with cryoprecipitate. Evidence for a mechanism of action independent of released von Willebrand factor. Blood. 1989 Nov 1;74(6):1972-5.

ReferenceCitation: Mannucci PM, Chediak J, Hanna W, Byrnes J, Ledford M, Ewenstein BM, Retzios AD, Kapelan BA, Schwartz RS, Kessler C; Alphanate Study Group. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood. 2002 Jan 15;99(2):450-6. doi: 10.1182/blood.v99.2.450.

ReferenceCitation: Federici AB, Baudo F, Caracciolo C, Mancuso G, Mazzucconi MG, Musso R, Schinco PC, Targhetta R, Mannuccio Mannucci P. Clinical efficacy of highly purified, doubly virus-inactivated factor VIII/von Willebrand factor concentrate (Fanhdi) in the treatment of von Willebrand disease: a retrospective clinical study. Haemophilia. 2002 Nov;8(6):761-7. doi: 10.1046/j.1365-2516.2002.00688.x.

ReferenceCitation: Bello IF, Yuste VJ, Molina MQ, Navarro FH. Fanhdi, efficacy and safety in von Willebrand's disease: prospective international study results. Haemophilia. 2007 Dec;13 Suppl 5:25-32. doi: 10.1111/j.1365-2516.2007.01570.x.

ReferenceCitation: Federici AB, Barillari G, Zanon E, Mazzucconi MG, Musso R, Targhetta R, Mannucci PM. Efficacy and safety of highly purified, doubly virus-inactivated VWF/FVIII concentrates in inherited von Willebrand's disease: results of an Italian cohort study on 120 patients characterized by bleeding severity score. Haemophilia. 2010 Jan;16(1):101-10. doi: 10.1111/j.1365-2516.2009.02088.x. Epub 2009 Oct 6.

ReferenceCitation: Dobrkovska A, Krzensk U, Chediak JR. Pharmacokinetics, efficacy and safety of Humate-P in von Willebrand disease. Haemophilia. 1998;4 Suppl 3:33-9. doi: 10.1046/j.1365-2516.1998.0040s3033.x.

ReferenceCitation: Lillicrap D, Poon MC, Walker I, Xie F, Schwartz BA; Association of Hemophilia Clinic Directors of Canada. Efficacy and safety of the factor VIII/von Willebrand factor concentrate, haemate-P/humate-P: ristocetin cofactor unit dosing in patients with von Willebrand disease. Thromb Haemost. 2002 Feb;87(2):224-30.

ReferenceCitation: Franchini M, Rossetti G, Tagliaferri A, Pattacini C, Pozzoli D, Lippi G, Manzato F, Bertuzzo D, Gandini G. Efficacy and safety of factor VIII/von Willebrand's factor concentrate (Haemate-P) in preventing bleeding during surgery or invasive procedures in patients with von Willebrand disease. Haematologica. 2003 Nov;88(11):1279-83.

ReferenceCitation: Federici AB, Castaman G, Franchini M, Morfini M, Zanon E, Coppola A, Tagliaferri A, Boeri E, Mazzucconi MG, Rossetti G, Mannucci PM. Clinical use of Haemate P in inherited von Willebrand's disease: a cohort study on 100 Italian patients. Haematologica. 2007 Jul;92(7):944-51. doi: 10.3324/haematol.11124.

ReferenceCitation: Gill JC, Ewenstein BM, Thompson AR, Mueller-Velten G, Schwartz BA; Humate-P Study Group. Successful treatment of urgent bleeding in von Willebrand disease with factor VIII/VWF concentrate (Humate-P): use of the ristocetin cofactor assay (VWF:RCo) to measure potency and to guide therapy. Haemophilia. 2003 Nov;9(6):688-95. doi: 10.1046/j.1351-8216.2003.00816.x.

ReferenceCitation: Thompson AR, Gill JC, Ewenstein BM, Mueller-Velten G, Schwartz BA; Humate-P Study Group. Successful treatment for patients with von Willebrand disease undergoing urgent surgery using factor VIII/VWF concentrate (Humate-P). Haemophilia. 2004 Jan;10(1):42-51. doi: 10.1046/j.1351-8216.2003.00809.x.

ReferenceCitation: Lethagen S, Kyrle PA, Castaman G, Haertel S, Mannucci PM; HAEMATE P Surgical Study Group. von Willebrand factor/factor VIII concentrate (Haemate P) dosing based on pharmacokinetics: a prospective multicenter trial in elective surgery. J Thromb Haemost. 2007 Jul;5(7):1420-30. doi: 10.1111/j.1538-7836.2007.02588.x. Epub 2007 Apr 16.

ReferenceCitation: Stadler M, Gruber G, Kannicht C, Biesert L, Radomski KU, Suhartono H, Pock K, Neisser-Svae A, Weinberger J, Romisch J, Svae TE. Characterisation of a novel high-purity, double virus inactivated von Willebrand Factor and Factor VIII concentrate (Wilate). Biologicals. 2006 Dec;34(4):281-8. doi: 10.1016/j.biologicals.2005.11.010. Epub 2006 Feb 23.

ReferenceCitation: Favaloro EJ, Lloyd J, Rowell J, Baker R, Rickard K, Kershaw G, Street A, Scarff K, Barrese G, Maher D, McLachlan AJ. Comparison of the pharmacokinetics of two von Willebrand factor concentrates [Biostate and AHF (High Purity)] in people with von Willebrand disorder. A randomised cross-over, multi-centre study. Thromb Haemost. 2007 Jun;97(6):922-30.

ReferenceCitation: Shortt J, Dunkley S, Rickard K, Baker R, Street A. Efficacy and safety of a high purity, double virus inactivated factor VIII/von Willebrand factor concentrate (Biostate) in patients with von Willebrand disorder requiring invasive or surgical procedures. Haemophilia. 2007 Mar;13(2):144-8. doi: 10.1111/j.1365-2516.2006.01430.x.

ReferenceCitation: Menache D, Aronson DL, Darr F, Montgomery RR, Gill JC, Kessler CM, Lusher JM, Phatak PD, Shapiro AD, Thompson AR, White GC 2nd. Pharmacokinetics of von Willebrand factor and factor VIIIC in patients with severe von Willebrand disease (type 3 VWD): estimation of the rate of factor VIIIC synthesis. Cooperative Study Groups. Br J Haematol. 1996 Sep;94(4):740-5. doi: 10.1046/j.1365-2141.1996.d01-1860.x.

ReferenceCitation: Goudemand J, Scharrer I, Berntorp E, Lee CA, Borel-Derlon A, Stieltjes N, Caron C, Scherrmann JM, Bridey F, Tellier Z, Federici AB, Mannucci PM. Pharmacokinetic studies on Wilfactin, a von Willebrand factor concentrate with a low factor VIII content treated with three virus-inactivation/removal methods. J Thromb Haemost. 2005 Oct;3(10):2219-27. doi: 10.1111/j.1538-7836.2005.01435.x.

ReferenceCitation: Borel-Derlon A, Federici AB, Roussel-Robert V, Goudemand J, Lee CA, Scharrer I, Rothschild C, Berntorp E, Henriet C, Tellier Z, Bridey F, Mannucci PM. Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients. J Thromb Haemost. 2007 Jun;5(6):1115-24. doi: 10.1111/j.1538-7836.2007.02562.x.

ReferenceCitation: Bergamaschini L, Mannucci PM, Federici AB, Coppola R, Guzzoni S, Agostoni A. Posttransfusion anaphylactic reactions in a patient with severe von Willebrand disease: role of complement and alloantibodies to von Willebrand factor. J Lab Clin Med. 1995 Mar;125(3):348-55.

ReferenceCitation: Ciavarella N, Schiavoni M, Valenzano E, Mangini F, Inchingolo F. Use of recombinant factor VIIa (NovoSeven) in the treatment of two patients with type III von Willebrand's disease and an inhibitor against von Willebrand factor. Haemostasis. 1996;26 Suppl 1:150-4. doi: 10.1159/000217258.

ReferenceCitation: Boyer-Neumann C, Dreyfus M, Wolf M, Veyradier A, Meyer D. Multi-therapeutic approach to manage delivery in an alloimmunized patient with type 3 von Willebrand disease. J Thromb Haemost. 2003 Jan;1(1):190-2. doi: 10.1046/j.1538-7836.2003.00009.x. No abstract available.

ReferenceCitation: Berntorp E, Petrini P. Long-term prophylaxis in von Willebrand disease. Blood Coagul Fibrinolysis. 2005 Apr;16 Suppl 1:S23-6. doi: 10.1097/01.mbc.0000167659.23262.18.

ReferenceCitation: De Meyer SF, Vanhoorelbeke K, Chuah MK, Pareyn I, Gillijns V, Hebbel RP, Collen D, Deckmyn H, VandenDriessche T. Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor. Blood. 2006 Jun 15;107(12):4728-36. doi: 10.1182/blood-2005-09-3605. Epub 2006 Feb 14.

ReferenceCitation: von Willebrand EA. (1926) Hereditär Pseudohemofili. Finska Läkaresällskapets Handlingar 68, 87-112.

ReferenceCitation: Federici AB, Bucciarelli P, Castaman G et al. (2007) Incidence and determinants of bleeding in different types of von Willebrand disease:results of the first prospective multicenter study on 814 Italian patients Blood 110, 713

ReferenceCitation: Abuzenadah AM, Gursel T, Ingerslev J et al. (1999) Mutational analysis of the von Willebrand factor gene in 27 families from Turkey with von Willebrand disease Thromb Haemost 82 (Suppl), 283

ReferenceCitation: Castaman G, Giacomelli SH, Coppola A et al. (2008) Molecular bases of type 3 von Willebrand disease in Italy: report on 12 families Blood Transfus Suppl 3, 44

ReferenceCitation: Gazda H, Budde U, Krey S et al. (1997) Delta C in exon 18 of the von Willebrand factor gene is the most common mutation in patients with severe von Willebrand disease type 3 in Poland Blood 90 (Suppl 1), 94b

ReferenceCitation: Titapiwatanakun R, Guenther JC, Asmann YW et al. (2007) Novel Mutations in Types 2 & 3 von Willebrand Disease and Correlation with von Willebrand Factor Multimer Patterns. Blood 110, 2136

ReferenceCitation: Montgomery RR, Jozwiak MA, Hutter JJ et al. (1999) A homozygous variant of the von Willebrand factor (VWF) that fails to c-terminal dimerize resulting in loss of VWF multimers larger than dimer Blood 94 (Suppl 1), 443a

ReferenceCitation: Schneppenheim R, Budde U, Drewke E et al. (1999) Cysteine mutations of von Willebrand factor correlate with different types of von Willebrand disease Thromb Haemost 82 (Suppl), 283

ReferenceCitation: Enayat MS, Guilliatt AM, Surdhar GK et al. (2001) Identification of five novel mutations in families with type 3 von Willebrand's disease Thromb Haemost 86 (Suppl), P1810

ReferenceCitation: Mannucci PM. Treatment of von Willebrand's Disease. N Engl J Med. 2004 Aug 12;351(7):683-94. doi: 10.1056/NEJMra040403. No abstract available.

ReferenceCitation: Federici AB, Gianniello F, Canciani MT et al. (2005) Secondary long-term prophylaxis in severe patients with von Willebrand disease: an Italian cohort study Blood 106, 507a

ReferenceCitation: Marchese M, De Cristofaro R, Federici AB, Biondi A, Petruzziello L, Tringali A, Spada C, Mutignani M, Ronconi P, Costamagna G. Duodenal and gastric Dieulafoy's lesions in a patient with type 2A von Willebrand's disease. Gastrointest Endosc. 2005 Feb;61(2):322-5. doi: 10.1016/s0016-5107(04)02636-7. No abstract available.

ReferenceCitation: Tosetto A, Rodeghiero F, Castaman G, Bernardi M, Bertoncello K, Goodeve A, Federici AB, Batlle J, Meyer D, Mazurier C, Goudemand J, Eikenboom J, Schneppenheim R, Budde U, Ingerslev J, Vorlova Z, Habart D, Holmberg L, Lethagen S, Pasi J, Hill F, Peake I. Impact of plasma von Willebrand factor levels in the diagnosis of type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1VWD). J Thromb Haemost. 2007 Apr;5(4):715-21. doi: 10.1111/j.1538-7836.2007.02444.x.

ReferenceCitation: Federici AB. Highly purified VWF/FVIII concentrates in the treatment and prophylaxis of von Willebrand disease: the PRO. WILL Study. Haemophilia. 2007 Dec;13 Suppl 5:15-24. doi: 10.1111/j.1365-2516.2007.01573.x.

ReferenceCitation: Federici AB, Mannucci PM, Castaman G, Baronciani L, Bucciarelli P, Canciani MT, Pecci A, Lenting PJ, De Groot PG. Clinical and molecular predictors of thrombocytopenia and risk of bleeding in patients with von Willebrand disease type 2B: a cohort study of 67 patients. Blood. 2009 Jan 15;113(3):526-34. doi: 10.1182/blood-2008-04-152280. Epub 2008 Sep 19.

ReferenceCitation: Gritti G, Cortelezzi A, Bucciarelli P, Rezzonico F, Lonati S, La Marca S, Silvestris I, Federici AB. Circulating and progenitor endothelial cells are abnormal in patients with different types of von Willebrand disease and correlate with markers of angiogenesis. Am J Hematol. 2011 Aug;86(8):650-6. doi: 10.1002/ajh.22070. Epub 2011 May 31.

ReferenceCitation: Abshire TC, Federici AB, Alvarez MT, Bowen J, Carcao MD, Cox Gill J, Key NS, Kouides PA, Kurnik K, Lail AE, Leebeek FW, Makris M, Mannucci PM, Winikoff R, Berntorp E; VWD PN. Prophylaxis in severe forms of von Willebrand's disease: results from the von Willebrand Disease Prophylaxis Network (VWD PN). Haemophilia. 2013 Jan;19(1):76-81. doi: 10.1111/j.1365-2516.2012.02916.x. Epub 2012 Jul 23.

ReferenceCitation: Castaman G, Goodeve A, Eikenboom J; European Group on von Willebrand Disease. Principles of care for the diagnosis and treatment of von Willebrand disease. Haematologica. 2013 May;98(5):667-74. doi: 10.3324/haematol.2012.077263.

ReferenceCitation: Federici AB, Bucciarelli P, Castaman G, Mazzucconi MG, Morfini M, Rocino A, Schiavoni M, Peyvandi F, Rodeghiero F, Mannucci PM. The bleeding score predicts clinical outcomes and replacement therapy in adults with von Willebrand disease. Blood. 2014 Jun 26;123(26):4037-44. doi: 10.1182/blood-2014-02-557264. Epub 2014 Apr 30.

ReferenceCitation: Abdul-Kadir R, McLintock C, Ducloy AS, El-Refaey H, England A, Federici AB, Grotegut CA, Halimeh S, Herman JH, Hofer S, James AH, Kouides PA, Paidas MJ, Peyvandi F, Winikoff R. Evaluation and management of postpartum hemorrhage: consensus from an international expert panel. Transfusion. 2014 Jul;54(7):1756-68. doi: 10.1111/trf.12550. Epub 2014 Mar 12. Erratum In: Transfusion. 2015 Mar;55(3):691.

ReferenceCitation: Makris M, Federici AB, Mannucci PM, Bolton-Maggs PHB, Yee TT, Abshire T, Berntorp E. The natural history of occult or angiodysplastic gastrointestinal bleeding in von Willebrand disease. Haemophilia. 2015 May;21(3):338-342. doi: 10.1111/hae.12571. Epub 2014 Nov 7.

ReferenceCitation: Federici AB. Clinical and laboratory diagnosis of VWD. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):524-30. doi: 10.1182/asheducation-2014.1.524. Epub 2014 Nov 18.

ReferenceCitation: Holm E, Abshire TC, Bowen J, Alvarez MT, Bolton-Maggs P, Carcao M, Federici AB, Gill JC, Halimeh S, Kempton C, Key NS, Kouides P, Lail A, Landorph A, Leebeek F, Makris M, Mannucci P, Mauser-Bunschoten EP, Nugent D, Valentino LA, Winikoff R, Berntorp E. Changes in bleeding patterns in von Willebrand disease after institution of long-term replacement therapy: results from the von Willebrand Disease Prophylaxis Network. Blood Coagul Fibrinolysis. 2015 Jun;26(4):383-8. doi: 10.1097/MBC.0000000000000257.

ReferenceCitation: Randi AM, Laffan MA. Von Willebrand factor and angiogenesis: basic and applied issues. J Thromb Haemost. 2017 Jan;15(1):13-20. doi: 10.1111/jth.13551.

ReferenceCitation: Federici AB. Current and emerging approaches for assessing von Willebrand disease in 2016. Int J Lab Hematol. 2016 May;38 Suppl 1:41-9. doi: 10.1111/ijlh.12540.

ReferenceCitation: De Jong A, Eikenboom J. Developments in the diagnostic procedures for von Willebrand disease. J Thromb Haemost. 2016 Mar;14(3):449-60. doi: 10.1111/jth.13243. Epub 2016 Feb 12.

ReferenceCitation: Abshire T, Cox-Gill J, Kempton CL, Leebeek FW, Carcao M, Kouides P, Donfield S, Berntorp E. Prophylaxis escalation in severe von Willebrand disease: a prospective study from the von Willebrand Disease Prophylaxis Network. J Thromb Haemost. 2015 Sep;13(9):1585-9. doi: 10.1111/jth.12995. Epub 2015 Jul 14.

ReferenceCitation: Engelen ET, van Galen KP, Schutgens RE. Thalidomide for treatment of gastrointestinal bleedings due to angiodysplasia: a case report in acquired von Willebrand syndrome and review of the literature. Haemophilia. 2015 Jul;21(4):419-29. doi: 10.1111/hae.12701. Epub 2015 Apr 30.

ReferenceCitation: Franchini M, Mannucci PM. Gastrointestinal angiodysplasia and bleeding in von Willebrand disease. Thromb Haemost. 2014 Sep 2;112(3):427-31. doi: 10.1160/TH13-11-0952. Epub 2014 Jun 5.

ReferenceCitation: Randi AM, Laffan MA, Starke RD. Von Willebrand factor, angiodysplasia and angiogenesis. Mediterr J Hematol Infect Dis. 2013 Sep 2;5(1):e2013060. doi: 10.4084/MJHID.2013.060.

ReferenceCitation: Thachil J, Hay CR, Campbell S. Tamoxifen for recurrent bleeds due to angiodysplasia in von Willebrand's disease. Haemophilia. 2013 Sep;19(5):e313-5. doi: 10.1111/hae.12196. Epub 2013 Jun 4. No abstract available.

ReferenceCitation: Castaman G, Federici AB, Tosetto A, La Marca S, Stufano F, Mannucci PM, Rodeghiero F. Different bleeding risk in type 2A and 2M von Willebrand disease: a 2-year prospective study in 107 patients. J Thromb Haemost. 2012 Apr;10(4):632-8. doi: 10.1111/j.1538-7836.2012.04661.x.

ReferenceCitation: Starke RD, Ferraro F, Paschalaki KE, Dryden NH, McKinnon TA, Sutton RE, Payne EM, Haskard DO, Hughes AD, Cutler DF, Laffan MA, Randi AM. Endothelial von Willebrand factor regulates angiogenesis. Blood. 2011 Jan 20;117(3):1071-80. doi: 10.1182/blood-2010-01-264507. Epub 2010 Nov 3.

ReferenceCitation: Veyradier A, Balian A, Wolf M, Giraud V, Montembault S, Obert B, Dagher I, Chaput JC, Meyer D, Naveau S. Abnormal von Willebrand factor in bleeding angiodysplasias of the digestive tract. Gastroenterology. 2001 Feb;120(2):346-53. doi: 10.1053/gast.2001.21204.

ReferenceCitation: Bowers M, McNulty O, Mayne E. Octreotide in the treatment of gastrointestinal bleeding caused by angiodysplasia in two patients with von Willebrand's disease. Br J Haematol. 2000 Mar;108(3):524-7. doi: 10.1046/j.1365-2141.2000.01897.x.

ReferenceCitation: Van Belle E, Rauch A, Vincent F, Robin E, Kibler M, Labreuche J, Jeanpierre E, Levade M, Hurt C, Rousse N, Dally JB, Debry N, Dallongeville J, Vincentelli A, Delhaye C, Auffray JL, Juthier F, Schurtz G, Lemesle G, Caspar T, Morel O, Dumonteil N, Duhamel A, Paris C, Dupont-Prado A, Legendre P, Mouquet F, Marchant B, Hermoire S, Corseaux D, Moussa K, Manchuelle A, Bauchart JJ, Loobuyck V, Caron C, Zawadzki C, Leroy F, Bodart JC, Staels B, Goudemand J, Lenting PJ, Susen S. Von Willebrand Factor Multimers during Transcatheter Aortic-Valve Replacement. N Engl J Med. 2016 Jul 28;375(4):335-44. doi: 10.1056/NEJMoa1505643.

ReferenceCitation: Susen S, Rauch A, Van Belle E, Vincentelli A, Lenting PJ. Circulatory support devices: fundamental aspects and clinical management of bleeding and thrombosis. J Thromb Haemost. 2015 Oct;13(10):1757-67. doi: 10.1111/jth.13120. Epub 2015 Sep 25.

ReferenceCitation: Blatchford O, Murray WR, Blatchford M. A risk score to predict need for treatment for upper-gastrointestinal haemorrhage. Lancet. 2000 Oct 14;356(9238):1318-21. doi: 10.1016/S0140-6736(00)02816-6.

ReferenceCitation: Gerson LB, Fidler JL, Cave DR, Leighton JA. ACG Clinical Guideline: Diagnosis and Management of Small Bowel Bleeding. Am J Gastroenterol. 2015 Sep;110(9):1265-87; quiz 1288. doi: 10.1038/ajg.2015.246. Epub 2015 Aug 25.

ReferenceCitation: Strate LL, Gralnek IM. ACG Clinical Guideline: Management of Patients With Acute Lower Gastrointestinal Bleeding. Am J Gastroenterol. 2016 Apr;111(4):459-74. doi: 10.1038/ajg.2016.41. Epub 2016 Mar 1. Erratum In: Am J Gastroenterol. 2016 May;111(5):755.

ReferenceCitation: Jackson CS, Gerson LB. Management of gastrointestinal angiodysplastic lesions (GIADs): a systematic review and meta-analysis. Am J Gastroenterol. 2014 Apr;109(4):474-83; quiz 484. doi: 10.1038/ajg.2014.19. Epub 2014 Mar 18.

ReferenceCitation: Martin-Grace J, Tamagno G. Somatostatin analogs in the medical management of occult bleeding of the lower digestive tract. Gastroenterol Res Pract. 2015;2015:702921. doi: 10.1155/2015/702921. Epub 2015 Mar 9.

ReferenceCitation: Holleran G, Hall B, Breslin N, McNamara D. Long-acting somatostatin analogues provide significant beneficial effect in patients with refractory small bowel angiodysplasia: Results from a proof of concept open label mono-centre trial. United European Gastroenterol J. 2016 Feb;4(1):70-6. doi: 10.1177/2050640614559121. Epub 2015 Nov 5.

sponsor_collaborators:
Fondazione Angelo Bianchi Bonomi
Sintesi Research Srl

location_country:
Finland
Germany
Italy
Hungary
United Kingdom
Iran, Islamic Republic of
Sweden
Netherlands
Spain
France

location_facility: University Clinic Bonn - Institute of Experimental Haematology & Transfusion Medicine

location_facility: Central Manchester University Hospital NHS Foundation Trust - Manchester Royal Infirmary - Manchester Royal Eye Hospital

location_facility: St. Istvan & St. Laszlo Hospital of Budapest - Hematology and Stem Cell Transplantation

location_facility: Thrombosis Hemostasis Research Center - Vali-Asr Hospital - Emam Khmeini Complex Hospital - Tehran University of Medical Science

location_facility: Centro Emofilia e Trombosi - Fondazione Angelo Bianchi Bonomi - IRCCS Ospedale Ca' Granda - Dip. di Medicina Interna - Università degli Studi di Milano

location_facility: Hemophilia- Thalassaemia Center of Mashhad (Sarvar Clinic) - Mashad University of Medical Science

location_facility: Azienda Ospedaliera Universitaria Careggi - Agenzia per l'Emofilia - Centro di Riferimento Coagulopatie Congenite

location_facility: Helsinki University Central Hospital, Department Internal Medicine, Coagulation Disorders, at Haematology and Laboratory Services

location_facility: University Children's Hospital - Department of Pediatric Hematology and Oncology

location_facility: Erasmus Medical Center - Department of Hematology

location_facility: Lund University - Centre for Thrombosis and Haemostasis - Skane University Hospital

location_facility: Ahvaz Jundishpur University of Medical Sciences - Research Center for Thalassemia & Hemoglobinopathy - Division of Hematology & Oncology

location_facility: Complejo Hospitalario Universitario de A Coruña - Servicio de Hematología y Hemoterapia

location_facility: Centre Régional de Traitement de l'Hémophilie - Laboratoire d'Hématologie

location_facility: Department of Haematology, Haemostasis, Oncology and Stem Cell Transplantation - Hannover Medical School - Haemophilia Care Centre

location_facility: Azienda Ospedaliera Policlinico Consorziale di Bari - Unità Operativa Semplice di Emostasi e Trombosi

location_facility: Hospital Universitari General Vall d'Hebron - Unidad de Hemofilia

location_facility: Seid-ol-Shohada Hospital - Hemophilia Center - Esfahan University of Medical Science

location_facility: Dipartimento di Terapie Cellulari ed Ematologia - Centro Malattie Emorragiche e Trombotiche - Ospedale San Bortolo

location_facility: Iranian Hemophilia Comprehensive Treatment Centre - Iranian Hemophilia Society

location_facility: Mofid Comprehensive Care Centre for Children with Hemophilia - Shahid Beheshti University of Medical Science

location_facility: Leiden University Medical Center - Department of Hematology - Hemostasis and Thrombosis Center

location_facility: Dipartimento di Biotecnologie Cellulari ed Ematologia - Università "Sapienza" di Roma - Policlinico Umberto I

location_facility: Nemazee Hospital Hemophilia Center - Shiraz University of Medical Science

location_facility: Institut d'Hématologie - Hôpital Cardiologique - University of Lille - Haematology Department


type: EudraCT Number

title: Open Label Study to Evaluate the Pharmacokinetics of Fidaxomicin in Inflammatory Bowel Disease (IBD) Subjects With Clostridium Difficile Infection (CDI)

acronym: PROFILE

summary:
The purpose of this study is to investigate the plasma pharmacokinetics (PK) of fidaxomicin (FDX) and primary metabolite OP-1118 in Subjects with Inflammatory Bowel Disease (IBD) and C. difficile Infection (CDI).

This study will also compare CDI clinical response to the microbiological response in terms of magnitude of reduction of C. difficile total viable count and spore count during treatment with FDX and if achieved; the time to microbial eradication; determine time to negative CDI toxin assay in stool specimens during treatment with FDX; assess the stool concentrations of FDX and metabolite OP-1118 throughout therapy; assess the length of hospital stay, readmissions and resource utilization for IBD patients receiving FDX; record the incidence and severity of Adverse Events (AEs) and document the impact of treatment on Quality of Life as measured by the changes in Short Inflammatory Bowel Disease Questionnaire (IBDQ) score.

ReferenceCitation: Hogenauer C, Mahida Y, Stallmach A, Marteau P, Rydzewska G, Ivashkin V, Gargalianos-Kakolyris P, Michon I, Adomakoh N, Georgopali A, Tretter R, Karas A, Reinisch W. Pharmacokinetics and safety of fidaxomicin in patients with inflammatory bowel disease and Clostridium difficile infection: an open-label Phase IIIb/IV study (PROFILE). J Antimicrob Chemother. 2018 Dec 1;73(12):3430-3441. doi: 10.1093/jac/dky368.

evidence: open (via crossref license)

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

repository_normalized: HAL

repository_institution: University of Nottingham - Repository@Nottingham

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Archive ouverte HAL

publisher_dissemination: Oxford University Press

ipd_sharing: No

ipd_sharing_description: Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."

lead_sponsor: Astellas Pharma Europe Ltd.

location_country:
Italy
Poland
United Kingdom
Austria
Greece
Russian Federation
France

location_facility:
Site PL48003
Site AT43001
Site FR33001
Site PL48002
Site FR33002
Site RU70001
Site RU70003
Site GR30004
Site GB44002
Site RU70002
Site IT39003
Site IT39001

intervention_type: Drug

lead_sponsor_type: industriel


title: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C in Order to Characterise the Individual Patient Disease Profile and Historic Signo-symptomatology Progression Pattern

summary:
This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical, imaging, biological(biomarkers) and quality of life data.

Patients will be offered enrollment into a Phase II/III study on arimoclomol at the end of the study.

time_perspective: Prospective

ReferenceCitation: Mengel E, Bembi B, Del Toro M, Deodato F, Gautschi M, Grunewald S, Gronborg S, Heron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Symonds T, Hudgens S, Patterson MC, Guldberg C, Ingemann L, Petersen NHT, Kirkegaard T, I Dali C. Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study. Orphanet J Rare Dis. 2020 Nov 23;15(1):328. doi: 10.1186/s13023-020-01616-0. Erratum In: Orphanet J Rare Dis. 2021 Jun 1;16(1):246.

oa_colors: green gold

evidence: oa journal (via doaj)

license: cc-by

license_normalized: cc-by

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

evidence: oa repository (via page says license)

repository_normalized: Research Square

version: submittedVersion

repository_institution: Research Square - Research Square

repositories:
PubMed Central
Research Square
boris.unibe.ch

unpaywall_oa_status: gold

publisher_dissemination: Springer-Nature

lead_sponsor: ZevraDenmark

location_country:
Germany
Italy
Poland
United Kingdom
Denmark
Switzerland
Spain
France

location_facility: University Hospital Copenhagen (Rigshospitalet)

location_facility: Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia"


lead_sponsor: Novartis Pharmaceuticals

summary: To determine the MTD/RP2D of the HDM201 and LEE011 combination and evaluate whether the combination is safe and has beneficial effects in patients with liposarcoma.

title: A Phase Ib/II, Open-label, Multicenter Study of Oral HDM201 in Combination With Oral LEE011 in Adult Patients With Liposarcoma

source: euctr

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

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

location_country: Germany Singapore Spain Taiwan Canada France

ipd_sharing: Undecided


type: Other Identifier

title: Assessing the Severity of Metabolic-related Liver Injuries in Aging HIV-monoinfected Patients: a European Multicentre Study

acronym: ANRS ECHAM

summary: This study will allow to assess liver related injuries in HIV patients.

primary_purpose: Diagnostic

lead_sponsor: ANRS, Emerging Infectious Diseases

location_country: Germany Belgium France

location_facility: Center for HIV and Hepato-Gastroenterology

intervention_type: Procedure


title: A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia Care

summary: To describe the current treatment practice during 12 months for previously treated patients diagnosed with haemophilia A or haemophilia B.

time_perspective: Retrospective

lead_sponsor: Swedish Orphan Biovitrum

location_country:
Germany
Italy
United Kingdom
Sweden
Belgium
Spain
France

location_facility: Swedish Orphan Biovitrum Investigational Site


acronym: GadaCAD 1

lead_sponsor: Bayer

summary:
Participants being evaluated for suspected or known Coronary artery Disease (CAD) based on signs and/or symptoms, will be invited to participate in the study. The duration for a participant in the study may range from 2 days to 4-6 weeks. One to four visits to the study doctor will be required.

This study will investigate the diagnostic results of gadobutrol-enhanced Cardiac Magnetic Resonance Imaging (CMRI) images regarding the detection (sensitivity) and exclusion (specificity) of coronary artery disease utilizing a uniform image acquisition software. The CMR images will be tested either against the results from routine clinical Coronary Angiography (CA) or those from Computed Tomography Angiography (CTA), which is used as the standard of reference. The CA/CTA may have been performed up to 4 weeks prior to enrollment or be scheduled up to 4/6 weeks after the study.

CMRI and CA/CTA images will be collected for an independent image review (blinded read).

title: Multicenter Open-label Study to Evaluate Efficacy of Gadobutrol-enhanced Cardiac Magnetic Resonance Imaging (CMRI) for Detection of Significant Coronary Artery Disease (CAD) in Subjects With Known or Suspected CAD by a Blinded Image Analysis

location_country:
Germany
New Zealand
United Kingdom
Switzerland
United States
Korea, Republic of
France


title: SD01 Master Study (Safety and Efficacy Study)

summary: The study is designed to confirm safety and efficacy of the SD01 ICD (implantable cardioverter-defibrillator) lead.

lead_sponsor: Biotronik SE & Co. KG

location_country: Germany Hungary Latvia Czechia France

location_facility: Bovas P. Stradina KUS Latvian Center of Cardiology

location_facility: Städtisches Klinikum München Bogenhausen

status_simplified: Ongoing


title: A Phase II Randomized Study of TTField Therapy (150 kHz) Versus Supportive Care in Non-small Cell Lung Cancer Patients With 1-5 Brain Metastases Following Optimal Standard Local Treatment

summary: The study is a prospective, randomly controlled phase II trial, designed to test the efficacy, safety and neurocognitive outcomes of a medical device, the NovoTTF-100A, in the treatment of NSCLC patients with controlled systemic disease, following optimal standard local treatment for 1-5 brain metastases (BM). The device is an experimental, portable, battery operated device for chronic administration of alternating electric fields (termed TTFields or TTF) to the region of the malignant tumor, by means of surface, insulated electrode arrays.

status: Unknown status

ReferenceCitation: Kirson ED, Gurvich Z, Schneiderman R, Dekel E, Itzhaki A, Wasserman Y, Schatzberger R, Palti Y. Disruption of cancer cell replication by alternating electric fields. Cancer Res. 2004 May 1;64(9):3288-95. doi: 10.1158/0008-5472.can-04-0083.

ReferenceCitation: Kirson ED, Dbaly V, Tovarys F, Vymazal J, Soustiel JF, Itzhaki A, Mordechovich D, Steinberg-Shapira S, Gurvich Z, Schneiderman R, Wasserman Y, Salzberg M, Ryffel B, Goldsher D, Dekel E, Palti Y. Alternating electric fields arrest cell proliferation in animal tumor models and human brain tumors. Proc Natl Acad Sci U S A. 2007 Jun 12;104(24):10152-7. doi: 10.1073/pnas.0702916104. Epub 2007 Jun 5.

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

ReferenceCitation: Pless M, Weinberg U. Tumor treating fields: concept, evidence and future. Expert Opin Investig Drugs. 2011 Aug;20(8):1099-106. doi: 10.1517/13543784.2011.583236. Epub 2011 May 9.

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

ReferenceCitation: Pless M, Droege C, von Moos R, Salzberg M, Betticher D. A phase I/II trial of Tumor Treating Fields (TTFields) therapy in combination with pemetrexed for advanced non-small cell lung cancer. Lung Cancer. 2013 Sep;81(3):445-450. doi: 10.1016/j.lungcan.2013.06.025. Epub 2013 Jul 23.

ReferenceCitation: Weinberg, U., et al. An Open Label Pilot Study of Tumor Treating Fields (TTFields) in Combination with Pemetrexed for Advanced Non-small Cell Lung Cancer (NSCLC). in ERS Annual Congress. 2010. Publisher: Abstract 363.

lead_sponsor: NovoCure Ltd.

location_country:
Italy
Poland
Spain
Czech Republic
France

location_facility: Szpital Kliniczny Przemienienia Pańskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

location_facility: ICO L'Hospitalet - Hospital Duran i Reynals

status_simplified: Unknown


title: A Phase I/II Dose Schedule Finding Study of ch14.18/CHO Continuous Infusion Combined With Subcutaneous Aldesleukin (IL-2) in Patients With Primary Refractory or Relapsed Neuroblastoma

acronym: LTI

summary: The main aim of this clinical trial is to find a way of giving ch14.18/CHO, in combination with subcutaneous aldesleukin (IL-2) and oral isotretinoin (13-cis-RA), to children and young people with primary refractory or relapsed neuroblastoma without intravenous morphine.

enrollment_type: Anticipated

lead_sponsor: St. Anna Kinderkrebsforschung

collaborators: Gustave Roussy, Cancer Campus, Grand Paris

collaborators: Schneider Children's Medical Center, Israel

collaborators: Great Ormond Street Hospital for Children NHS Foundation Trust

collaborators: Johann Wolfgang Goethe University Hospital

collaborators: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

collaborators: Alder Hey Children's NHS Foundation Trust

collaborators: University Hospitals Bristol and Weston NHS Foundation Trust

collaborators: Hospital Infantil Universitario Niño Jesús, Madrid, Spain

collaborators: University Hospital Southampton NHS Foundation Trust

collaborators: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

location_country:
Germany
Italy
France
United Kingdom
Austria
Spain
Israel

location_facility: Schneider Children's Medical Centre of Israel

location_facility: University Hospitals Bristol NHS Foundation Trust

location_facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust

location_facility: Birmingham Children's Hospital NHS Foundation Trust

location_facility: University College Hospitals NHS Foundation Trust


title: Sidus(TM) Stem-Free Shoulder - A Multi-center, Prospective, Non-controlled Post Market Clinical Follow-up Study.

summary:
This post market clinical follow-up study is designed to confirm safety and performance of the Sidus Stem-Free Shoulder when used in hemi or total shoulder arthroplasty.

The safety of the implant will be evaluated by monitoring the frequency and incidence of all kinds of adverse events.

The performance will be determined by analyzing the implant survival, overall pain and functional performances (based on Constant & Murley score and ASES score), subject quality of life (EuroQol EQ5D) and radiographic parameters (e.g. radiolucencies, osteolysis, component migration) of study subjects who received the Sidus Stem-Free Shoulder.

The Sidus Stem-Free Shoulder is not approved for use in the US.

lead_sponsor: Zimmer Biomet

sponsor_collaborators:
Zimmer Biomet
Zimmer, GmbH

location_country:
Germany
Italy
United Kingdom
Austria
France

location_facility: Centre Hospitalier Universitaire Toulouse

location_facility: Azienda Ospedaliera Universitaria Careggi


acronym: Not applicable

summary:
The purpose of this study is to compare how the body absorbs and processes two different formulations of the anti-rejection medication tacrolimus (Advagraf® or Prograf®) in children receiving an organ transplant, and how safe and effective they are over a longer period of time.

This study is for children less than 16 years old. No minimum age has been set, however, to be included in this study participants must able to swallow the medication capsules intact.

location_facility:
Site GB42
Site GB45
Site PL71
Site FR33
Site GB43
Site IT52
Site FR32
Site GB46
Site GB44
Site CZ61

title: A Phase II, Parallel Group, Randomized, Multicentre, Open Label Study to Compare the Pharmacokinetics of Tacrolimus in De Novo Pediatric Allograft Recipients Treated With an Advagraf® or Prograf® Based Immunosuppressive Regimen, Including a Long-Term Follow-Up

primary_purpose: Other

location_country:
Italy
Poland
United Kingdom
Czechia
France


title: ALFApump® System Versus Large Volume Paracentesis in the Treatment of Refractory Ascites. A Multicentre Randomised Controlled Study.

summary: This is a multicentre, open, randomised, and controlled trial conducted in sixty (60) patients diagnosed with refractory or recurrent ascites. Patients will be randomised (enrolled) to either treatment arm A- implanted with the ALFApump System or treatment arm B-standard of care with evacuation large volume paracentesis. The main aims of the study are to determine the paracentesis free survival, defined as the time to the first large volume therapeutic paracentesis > 5 litres. The secondary aims are to assess non-inferiority of cirrhosis-related complications in the group of patients randomized to the ALFApump system group, as well as nutritional effects, resource utilisation, patient quality of life and survival non-inferiority.

ReferenceCitation: Stepanova M, Nader F, Bureau C, Adebayo D, Elkrief L, Valla D, Peck-Radosavljevic M, McCune A, Vargas V, Simon-Talero M, Cordoba J, Angeli P, Rossi S, MacDonald S, Capel J, Jalan R, Younossi ZM. Patients with refractory ascites treated with alfapump(R) system have better health-related quality of life as compared to those treated with large volume paracentesis: the results of a multicenter randomized controlled study. Qual Life Res. 2018 Jun;27(6):1513-1520. doi: 10.1007/s11136-018-1813-8. Epub 2018 Feb 19.

oa_host_type: repository

repositories_concat: UCL Discovery

repositories_institution: University College London - UCL Discovery

unpaywall_oa_status: green

ReferenceCitation: Bureau C, Adebayo D, Chalret de Rieu M, Elkrief L, Valla D, Peck-Radosavljevic M, McCune A, Vargas V, Simon-Talero M, Cordoba J, Angeli P, Rosi S, MacDonald S, Malago M, Stepanova M, Younossi ZM, Trepte C, Watson R, Borisenko O, Sun S, Inhaber N, Jalan R. Alfapump(R) system vs. large volume paracentesis for refractory ascites: A multicenter randomized controlled study. J Hepatol. 2017 Nov;67(5):940-949. doi: 10.1016/j.jhep.2017.06.010. Epub 2017 Jun 21. Erratum In: J Hepatol. 2018 Jan 29;: J Hepatol. 2020 Mar;72(3):595-596.

version: acceptedVersion

publisher_dissemination: Elsevier

lead_sponsor: Sequana Medical N.V.

location_country:
Italy
United Kingdom
Austria
Spain
France

location_facility: Vienna General Hospital and Medical School, AKH

location_facility: Centre Hospitalier Universitaire de Toulouse


title: A Multi-center, Parallel-group, Double-blind, Placebo-controlled Single and Multiple Dose Escalation Study to Assess the Safety and Tolerability and the Pharmacokinetic Properties of SAR228810 Given as IV Infusion or as SC Injection in Patients With Mild to Moderate Alzheimer's Disease.

summary:
Primary Objective:

- To assess the safety and tolerability of escalating single and multiple doses of SAR228810 in patients with Alzheimer's disease (AD)

Secondary Objective:

- To evaluate the pharmacokinetic properties of SAR228810 after escalating single and multiple doses of SAR228810 in AD patients

lead_sponsor: Sanofi

location_country:
France
Netherlands
South Africa
Bulgaria
Sweden

location_facility:
Investigational Site Number 710001
Investigational Site Number 752003
Investigational Site Number 752002
Investigational Site Number 250002
Investigational Site Number 752001
Investigational Site Number 100001
Investigational Site Number 250001
Investigational Site Number 528001


title: Randomized Controlled Study on the Effect of the Hemodiafiltration Plasma Sodium Biofeedback System on Cardiovascular Stability in Hemodialysis Patients

acronym: AIMS

summary: Aim of the present randomized controlled multinational trial is the comparison of a novel biofeedback system on sodium profiling applied to a endogenous hemodiafiltration therapy, with the standard (no sodium profiling) hemodiafiltration technique on the intradialytic overall and cardiovascular stability.

lead_sponsor: Azienda Ospedaliera di Lecco

collaborators: IRCCS Azienda Ospedaliero-Universitaria di Bologna

location_country: Germany Italy Belgium Spain France

location_facility: Dept. of Nephrology and Dialysis Centre Pasteur Vallery Radat

location_facility: UO Nefrologia Dialisi e Trapianto Policlinico S. Orsola Malpighi

location_facility: Nephrology and Dialysis Unit Ospedali Riuniti


title: Dose Escalation, Safety, Pharmacokinetic and Pharmacodynamic, First in Man Study, of SAR125844 Single Agent Administered as Slow Intravenous Infusion in Adult Patients With Advanced Malignant Solid Tumors

acronym: SARMET

summary:
Primary Objectives:

To determine the maximum tolerated dose (MTD) of SAR125844. To confirm safety profile of SAR125844 when administered as single agent at the MTD.

To evaluate the preliminary anti-tumoral effect of SAR125844 in patients with MET-gene amplified solid tumors (including sub-group of MET-amplified non-small cell lung cancer [NSCLC] patients) and in patients with Phospho-MET positive tumors without MET-gene amplification.

Secondary Objectives:

To characterize the global safety profile including cumulative toxicities. To evaluate the pharmacokinetic profile of SAR125844 in the proposed dosing schedule(s).

To assess preliminary antitumor activity in patients with measurable/evaluable disease, according to RECIST 1.1 criteria.

To explore the pharmacodynamic effects (PD) of SAR125844. To explore MET gene amplification status in Circulating Tumoral Cells (CTCs) and on tumor biopsies collected during the study, in the escalation part only.

To evaluate other pharmacodynamic biomarkers and help selection of patients who could benefit from SAR125844.

To explore MET-gene amplification status in circulating DNA.

design_allocation: Non-Randomized

ReferenceCitation: Angevin E, Spitaleri G, Rodon J, Dotti K, Isambert N, Salvagni S, Moreno V, Assadourian S, Gomez C, Harnois M, Hollebecque A, Azaro A, Hervieu A, Rihawi K, De Marinis F. A first-in-human phase I study of SAR125844, a selective MET tyrosine kinase inhibitor, in patients with advanced solid tumours with MET amplification. Eur J Cancer. 2017 Dec;87:131-139. doi: 10.1016/j.ejca.2017.10.016. Epub 2017 Nov 14.

oa_host_type: publisher

license: cc-by-nc-nd

license_normalized: cc-by-nc-nd

location_country:
United States
Italy
Spain
France

location_facility:
Investigational Site Number 380004
Investigational Site Number 724001
Investigational Site Number 724003
Investigational Site Number 250002
Investigational Site Number 380002
Investigational Site Number 250001
Investigational Site Number 380001
Investigational Site Number 840001


acronym: PROGRESSION

summary: The purpose of this study, a follow up to study FG506-CL-0403, is to see how safe and effective Modigraf® is (Part A) and to see how safe and effective it is to change your child's medication from Modigraf® to Prograf® (Part B).

location_facility: Site GB13 Cent. Manchester Uni. Hospital

location_facility: Site DE31 Kliniken der Medizinischen Hoc

status: Terminated

title: A Long-term, Open-label, Non-comparative Study to Evaluate the Safety and Efficacy of a Modigraf® Based Immunosuppression Regimen in Paediatric Solid Allograft Recipients

primary_purpose: Prevention

location_country:
Germany
Poland
United Kingdom
Belgium
Spain
France


summary:
The phase Ib part of the trial will assess the MTD of AUY922 in combination with Trastuzumab in patients with Trastuzumab-refractory locally advanced or metastatic HER2+. The MTD is defined as the highest drug dosage not causing in the first cycle of treatment (28 days) medically unacceptable dose limiting toxicity (DLT).

The phase II part of the trial will assess any potential effect on efficacy of adding AUY922 to Trastuzumab in patients with Trastuzumab-refractory locally advanced or metastatic HER2+ breast cancer.

Both AUY922 and Trastuzumab will be administered as a weekly IV infusion. Treatment should be continued as long as the patient does not have disease progression and tolerates the treatment. The following reasons are examples of acceptable reasons for discontinuing the study; tumor progression (by RECIST, as assessed by the investigator), unacceptable toxicity, death, or discontinuation from the study for any other reason, such as patient refusal, withdrawn consent, lost to follow-up or investigator decision.

ReferenceType: result

ReferenceCitation: Kong A, Rea D, Ahmed S, Beck JT, Lopez Lopez R, Biganzoli L, Armstrong AC, Aglietta M, Alba E, Campone M, Hsu Schmitz SF, Lefebvre C, Akimov M, Lee SC. Phase 1B/2 study of the HSP90 inhibitor AUY922 plus trastuzumab in metastatic HER2-positive breast cancer patients who have progressed on trastuzumab-based regimen. Oncotarget. 2016 Jun 21;7(25):37680-37692. doi: 10.18632/oncotarget.8974.

evidence: open (via page says license)

repository_institution: University of Turin - Institutional Research Information System University of Turin

repository_institution: La Trobe University - OPAL (Open@LaTrobe)

repository_institution: University of Leicester - Leicester Research Archive

publisher_dissemination: Impact Journals, LLC

location_facility: Highlands Oncology Group Dept of Highlands Oncology Grp

title: A Phase Ib/II, Multi-center, Open-label Study to Evaluate the Efficacy of AUY922 in Combination With Trastuzumab in Patients With Locally Advanced or Metastatic HER2-positive Breast Cancer, That Has Progressed After or During at Least One Trastuzumab-containing Regimen

location_country:
Germany
Italy
United Kingdom
Singapore
Sweden
United States
Spain
France


title: SPIRIT V: A Clinical Evaluation of the XIENCE V® Everolimus Eluting Coronary Stent System in the Treatment of Patients With de Novo Coronary Artery Lesions

summary: The purpose of this Clinical Evaluation is a continuation in the assessment of the performance of the XIENCE V® Everolimus Eluting Coronary Stent System (XIENCE V® EECSS) in the treatment of patients with de novo coronary artery lesions in patients (Diabetic sub-study).

lead_sponsor: Abbott Medical Devices

location_country:
Germany
Italy
France
Poland
United Kingdom
Netherlands
Austria
Spain
Thailand
Malaysia
Israel

location_facility:
La Paz
Herzzentrum
Wessex Cardiac Unit
Hospital Puerta de Hierro
Hospital Belvigte de Barcelona
Maasstad Ziekenhuis
C.H.U. - Hopital Michallon
General De Alicante
Azienda Ospedaliera di Padova
King Chulalongkorn Memorial Hospital
Herzzentrum Siegburg GmbH
Medisch Centrum Alkmaar
Hospital Virgen de la Arrixaca
CHU Lille - Hôpital Cardiologique
Clinico San Carlos
Lukas Krankenhaus Neuss
Sheba Medical Center
Hospital General de Valencia
Medical University of Bydgoszcz
Azienda Ospedaliera Riuniti
Azienda Ospedaliera S. Gdi Dio Salerno
King's College Hospital
Institute Jantung Negara
Hospital Santa Creu I Sant Pau
Ospedale Civile
Universitätsklinikum
IRCCS Policlinico San Matteo
Salzburger Landeskliniken


lead_sponsor: PTC Therapeutics

summary: Methylmalonic acidemia (MMA) is a rare genetic disorder caused by mutations in the gene for mitochondrial enzyme methylmalonyl-CoA mutase (MCM) or in one of the genes for adenosylcobalamin (AdoCbl). Lack of these proteins causes toxic elevations of methylmalonic acid (MMacid) in blood, urine, and other tissues. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of the disease in approximately 5% to 20% of participants with mutations in the MCM gene, and approximately 20% to >50% of participants with mutations in one of the AdoCbl genes. Ataluren is an orally delivered, investigational drug that acts to overcome the effects of the premature stop codon, potentially enabling the production of functional MCM/AdoCbl. This study is a Phase 2a trial evaluating the safety and activity of ataluren in participants with MMA due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely decrease MMacid levels.

ReferenceCitation: Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL. PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. doi: 10.1038/nature05756. Epub 2007 Apr 22.

ReferenceCitation: Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140.

sponsor_collaborators:
PTC Therapeutics
Genzyme, a Sanofi Company

location_facility: University Hospital, Department of Pediatrics

location_facility: Istituti Clinici di Perfezionamento, Milano

location_facility: ZNA Queen Paola Child Hospital and Provincial Centre for Metabolic Disorders

title: A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia

location_country:
Germany
Italy
United Kingdom
Switzerland
Belgium
France


title: Evaluation of PillCam™ Colon 2 in Visualization of the Colon

summary: Evaluate accuracy of PCCE-2 in detecting patients with colonic polyps as compared to conventional colonoscopy.

lead_sponsor: Medtronic - MITG

location_country: Germany Italy Sweden Netherlands Belgium Spain France

location_facility: Chef de Service dHepato-Gastro-Enterologie et dAssistance Nutritionnelle CHU Nantes

location_facility: Academic Medical Center University of Amsterdam


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

summary:
RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Temsirolimus may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. It is not yet known whether radiation therapy is more effective when given together with temsirolimus or temozolomide in treating patients with glioblastoma.

PURPOSE: This randomized phase II trial is studying giving radiation therapy together with temsirolimus to see how well it works compared with giving radiation therapy together with temozolomide in treating patients with newly diagnosed glioblastoma.

location_facility: Erasmus MC - Daniel den Hoed Cancer Center

location_facility: Institut de Cancerologie de l'Ouest (ICO) - Centre Rene Gauducheau

location_facility: ICO Badalona - Hospital Germans Trias i Pujol

location_facility: Clatterbridge Cancer Centre NHS Foundation Trust

title: Radiation Therapy and Concurrent Plus Adjuvant Temsirolimus (CCI-779) Versus Chemo-Irradiation With Temozolomide in Newly Diagnosed Glioblastoma Without Methylation of the MGMT Gene Promoter - A Randomized Multicenter, Open-Label, Phase II Study.

location_country:
Germany
Italy
United Kingdom
Netherlands
Switzerland
Belgium
Spain
France


acronym: Clinical phase III trial of Treosulfan-based conditioning vs. RIC

lead_sponsor: medac GmbH

summary: This randomized allogeneic transplantation protocol compares i.v. Treosulfan-based conditioning therapy with reduced intensity i.v. Busulfan-based conditioning in adult AML and MDS patients at increased risk for standard conditioning therapies. The protocol is based on results of previous phase I/II trials evaluating Treosulfan/Fludarabine conditioning prior to allogeneic haematopoietic stem cell transplantation. The reference arm (reduced intensity i.v. Busulfan/Fludarabine) is considered to be accepted medical practice for the study patient population.

ReferenceCitation: Beelen DW, Trenschel R, Stelljes M, Groth C, Masszi T, Remenyi P, Wagner-Drouet EM, Hauptrock B, Dreger P, Luft T, Bethge W, Vogel W, Ciceri F, Peccatori J, Stolzel F, Schetelig J, Junghanss C, Grosse-Thie C, Michallet M, Labussiere-Wallet H, Schaefer-Eckart K, Dressler S, Grigoleit GU, Mielke S, Scheid C, Holtick U, Patriarca F, Medeot M, Rambaldi A, Mico MC, Niederwieser D, Franke GN, Hilgendorf I, Winkelmann NR, Russo D, Socie G, Peffault de Latour R, Holler E, Wolff D, Glass B, Casper J, Wulf G, Menzel H, Basara N, Bieniaszewska M, Stuhler G, Verbeek M, Grass S, Iori AP, Finke J, Benedetti F, Pichlmeier U, Hemmelmann C, Tribanek M, Klein A, Mylius HA, Baumgart J, Dzierzak-Mietla M, Markiewicz M. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): a randomised, non-inferiority, phase 3 trial. Lancet Haematol. 2020 Jan;7(1):e28-e39. doi: 10.1016/S2352-3026(19)30157-7. Epub 2019 Oct 9.

location_facility: Malteser Krankenhaus St. Franziskus-Hospital

location_facility: Universitätsmedizin Goettingen, Haematolgie und Onkologie

location_facility: Universitätsklinikum Carl Gustav Carus Dresden, Med. Klinik I

location_facility: Johannes-Gutenberg-Universität Mainz, III. Medizinische Klinik

location_facility: Policlinico GB Rossi (Borgo Roma), Div. di Ematologia

location_facility: Helsinki University Central Hospital, Dept. of Medicine

location_facility: Klinikum Nürnberg, 5. Medizinische Klinik

location_facility: Clinica Ematologica ed Unita di Terapie Cellulari 'Carlo Melzi'

location_facility: Klinikum Rechts der Isar der TU München, III. Med. Klinik

location_facility: Universitätsklinikum Leipzig, Haematologie, internistische Onkologie

location_facility: St. Istvan and St. Laszlo Hospital of Budapest

title: Clinical Phase III Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Reduced-intensity Conditioning (RIC) Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With AML or MDS Considered Ineligible to Standard Conditioning Regimens

location_country: Finland Italy Germany Poland Hungary France


title: Efficacy And Safety Of Eraxis/Ecalta (Anidulafungin) Compared To Cancidas (Caspofungin) In Neutropenic Patients With Invasive Candida Infection

summary: The purpose of this study is to gather information on the use of anidulafungin for the treatment of Candida infection in patients with an abnormal immune system. It is expected that anidulafungin will be at least as safe and as effective as the comparator drug, caspofungin.

ReferenceCitation: De Rosa FG, Busca A, Capparella MR, Yan JL, Aram JA. Invasive Candidiasis in Patients with Solid Tumors Treated with Anidulafungin: A Post Hoc Analysis of Efficacy and Safety of Six Pooled Studies. Clin Drug Investig. 2021 Jun;41(6):539-548. doi: 10.1007/s40261-021-01024-7. Epub 2021 Apr 23.

repositories_concat: PubMed Central;iris.unito.it

repositories_institution: University of Turin - Institutional Research Information System University of Turin

ReferenceCitation: Kontoyiannis DP, Bassetti M, Nucci M, Capparella MR, Yan JL, Aram J, Hogan PA. Anidulafungin for the treatment of candidaemia caused by Candida parapsilosis: Analysis of pooled data from six prospective clinical studies. Mycoses. 2017 Oct;60(10):663-667. doi: 10.1111/myc.12641. Epub 2017 Jun 9.

evidence: open (via free pdf)

unpaywall_oa_status: bronze

publisher_dissemination: Wiley

ReferenceCitation: Kullberg BJ, Vasquez J, Mootsikapun P, Nucci M, Paiva JA, Garbino J, Yan JL, Aram J, Capparella MR, Conte U, Schlamm H, Swanson R, Herbrecht R. Efficacy of anidulafungin in 539 patients with invasive candidiasis: a patient-level pooled analysis of six clinical trials. J Antimicrob Chemother. 2017 Aug 1;72(8):2368-2377. doi: 10.1093/jac/dkx116.

repository_institution: Radboud University - Radboud Repository

repositories_concat: PubMed Central;repository.ubn.ru.nl

repositories_institution:
PubMed Central - Europe PMC
Radboud University - Radboud Repository

lead_sponsor: Pfizer

location_country:
Italy
Poland
Slovakia
Bosnia and Herzegovina
Russian Federation
France


title: Randomized Phase III Intergroup Trial in Resected Stage 2 (Dukes B) Colon Cancer: 6-Month Infusional 5FU-CPT11 (+/- Folinic Acid) Versus Observation - Determination of Biologic Predictive and Response Factors

summary:
RATIONALE: Drugs used in chemotherapy, such as irinotecan, leucovorin, and fluorouracil, work in different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug and giving them after surgery may kill any remaining tumor cells. It is not yet known whether combination chemotherapy is more effective than observation alone in treating patients who have undergone surgery for colon cancer.

PURPOSE: This randomized phase III trial is studying irinotecan and fluorouracil with or without leucovorin to see how well they work compared to observation alone in treating patients who have undergone surgery for stage II colon cancer.

lead_sponsor: Federation Francophone de Cancerologie Digestive

location_country: Germany Italy Portugal Spain Egypt France

location_facility: Instituto Portugues de Oncologia Centro do Porto, S. A.

location_facility: Centre Hospitalier Universitaire Ambroise Pare - Boulogne

location_facility: Universita Degli Studi di Florence - Policlinico di Careggi


title: An International Field Study of the Reliability and Validity of the EORTC-QLQ-C30 and a Disease Specific Questionnaire Module (the EORTC OES-24) in Assessing the Quality of Life of Patients With Oesophageal Cancer

summary:
RATIONALE: Questionnaires that measure quality of life may improve the ability to plan treatment for patients who have cancer of the esophagus.

PURPOSE: This clinical trial studies quality-of-life assessments of patients with cancer of the esophagus who are receiving treatment.

ReferenceCitation: Conroy T, Etienne PL, Adenis A, Ducreux M, Paillot B, Oliveira J, Seitz JF, Francois E, Van Cutsem E, Wagener DJ, Kohser F, Daamen S, Praet M, Gorlia T, Baron B, Wils J; European Organisation for Research and Treatment of Cancer Gastrointestinal Tract Cancer Cooperative Group. Vinorelbine and cisplatin in metastatic squamous cell carcinoma of the oesophagus: response, toxicity, quality of life and survival. Ann Oncol. 2002 May;13(5):721-9. doi: 10.1093/annonc/mdf063.

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

location_country:
Germany
Norway
United Kingdom
Australia
Sweden
Switzerland
Spain
France

location_facility: Hopital Cantonal Universitaire de Geneva


title: Liver Investigation: Testing Marker Utility in Steatohepatitis (LITMUS): Assessment & Validation of Imaging Modality Performance Across the NAFLD Spectrum in a Prospectively Recruited Cohort

summary: The LITMUS Imaging Study is a prospectively recruited, observational study of patients with histologically characterised non-alcoholic fatty liver disease (NAFLD). It aims to evaluate the diagnostic performance of imaging biomarkers (ultrasound elastography and magnetic resonance biomarkers) against NAFLD histological scores in a cross-sectional analysis and the natural history of NAFLD in a longitudinal study.

status: Recruiting

lead_sponsor: University of Oxford

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

location_country:
Germany
Finland
Italy
United Kingdom
Sweden
Switzerland
United States
Spain
Greece
France

location_facility: Institut ICAN - Institute of Cardiometabolism And Nutrition Hôpital de la Pitié Salpêtrière

location_facility: Institute of Biomedicine of Sevilla (IBiS), Virgen del Rocío University Hospital

location_facility: Department of Medical Sciences University of Torino

location_facility: Le Centre de Recherche Clinique (CRC) du CHU d'Angers

location_facility: The Newcastle Upon Tyne Hospitals Nhs Foundation Trust

location_facility: UNIVERSITÄTSMEDIZIN der Johannes Gutenberg Universität Mainz

location_facility: Oxford University Hospitals NHS Foundation Trust


title: A Randomized, Participant and Investigator Blinded, Placebo-Controlled Study to Evaluate the Ability of a Single Intrathecally Administered Dose of NIO752 to Lower Cerebrospinal Fluid Total Tau Levels in Participants With Early Alzheimer's Disease

summary: Phase 1b study to assess the pharmacodynamics, safety, tolerability, and pharmacokinetics of NIO752 in patients with early Alzheimer's disease (AD)

lead_sponsor: Novartis Pharmaceuticals

location_country: Finland Spain Sweden France


title: Open-label Study Evaluating the Effect of Tusamitamab Ravtansine on the QTc Interval in Participants With Metastatic Solid Tumors

acronym: TusaRav-QT

summary: This is a Phase1, single-arm study for treatment. This is a prospective multicenter, multinational, open-label study to assess the effect of tusamitamab ravtansine on the QT interval in participants with metastatic colorectal cancer (CRC), nonsquamous non small cell lung cancer (NSQ NSCLC), or gastric/ gastroesophageal junction (GEJ) adenocarcinoma for which in the judgement of the Investigator, no standard alternative therapy is available.

ipd_sharing_description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

location_country:
Belgium
United States
Spain
Turkey
France

location_facility: Mary Crowley Cancer Research Center Site Number : 8400002


title: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life

acronym: HYDRO-PROTECT

summary:
Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive formation of renal cysts which ultimately lead to a loss of renal function.

Tolvaptan (a V2R antagonist) is currently the only effective treatment for preserving renal function in ADPKD. However, side-effects such as polyuria limit its tolerability and thereby the therapeutic potential. This study will test whether co-administration with hydochlorothiazide can improve V2RA efficacy (slowing kidney function decline) and tolerability (quality of life) in ADPKD. Approximately 300 patients will be enrolled.

study_start_date_type: Anticipated

status: Not yet recruiting

lead_sponsor: University Medical Center Groningen

location_country:
Germany
United Kingdom
Netherlands
Belgium
Spain
France

location_facility: Med. Klinik und Poliklinik III, Universitätsklinikum Dresden.


title: A Randomised, Controlled, Assessor-blind, Parallel Groups, Multicentre, Multinational Trial Comparing the Ovarian Response of a Starting Dose of 15 μg Follitropin Delta (REKOVELLE) to a Starting Dose of 225 IU Follitropin Alfa (GONAL-F) in Conventional Regimens in Controlled Ovarian Stimulation in Women Undergoing an Assisted Reproductive Technology Programme

summary:
Hormone preparations like REKOVELLE and GONAL-F are used to make the body produce multiple egg sacs, allowing more eggs to be collected from women undergoing infertility treatment.

The main purpose of this trial is to see how many eggs are produced with a starting dose of 15 µg REKOVELLE compared to a starting dose of 225 IU GONAL-F. This research is intended to provide more knowledge about REKOVELLE, including the doses given in the trial.

lead_sponsor: Ferring Pharmaceuticals

location_country:
Italy
United Kingdom
Austria
Spain
France


title: A Phase 1b, Open-label, Single-arm, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Treatment With JNJ-73763989, JNJ-64300535, and Nucleos(t)Ide Analogs in Virologically Suppressed, HBeAg-negative Participants With Chronic Hepatitis B Virus Infection

acronym: OSPREY

summary: The purpose of this study is to evaluate the efficacy of the study intervention based on hepatitis B surface antigen (HBsAg) levels.

ipd_sharing_description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson and Johnson is available at www.janssen.com/clinical trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

lead_sponsor: Janssen Research & Development, LLC

location_country:
Italy
Poland
New Zealand
United Kingdom
Belgium
Spain
Taiwan
France

location_facility: Chang Gung Memorial Hospital Linkou Branch

location_facility: Azienda Ospedaliero Universitaria Pisana


title: A Phase Ib Dose Finding Study Assessing Safety and Activity of [177Lu]Lu-DOTA-TATE in Newly Diagnosed Glioblastoma in Combination With Radiotherapy With or Without Temozolomide and in Recurrent Glioblastoma as Single Agent

summary: A Dose Finding Study of [177Lu]Lu-DOTA-TATE in Newly Diagnosed Glioblastoma in Combination with Standard of Care and in Recurrent Glioblastoma as a Single Agent

location_country:
Portugal
Switzerland
United States
Spain
France

location_facility: MD Anderson Cancer Center/University of Texas MDACC

location_facility: University of Pittsburgh University of Pittsburgh (2)


title: Prospective, Observational, Open-label, Clinical Investigation of Intra-articular Arthrosamid® Injection in Subjects With Knee Osteoarthritis

acronym: LUNA

summary: This is a prospective, long-term, observational, all-comers, open-label, multi-centre clinical investigation enrolling subjects with knee osteoarthritis who is eligible for treatment with Arthrosamid® according to the Instruction for Use.

lead_sponsor: Contura

location_country:
Germany
Italy
United Kingdom
Denmark
France

location_facility: University Medical Center of Johannes Gutenberg-University Mainz (UMC-Mainz)


title: A Phase 1/1b Open-label, First-in-human, Single Agent, Dose Escalation and Expansion Study for the Evaluation of Safety, Pharmacokinetics, Pharmacodynamics, and Anti-tumor Activity of SAR443216 in Participants With Relapsed/Refractory HER2 Expressing Solid Tumors.

summary:
Primary Objectives:

Part 1 (Dose Escalation)

To determine the MTD/maximum administered dose (MAD) of SAR443216 administered as a single agent in participants with HER2 expressing solid tumors and determine the RD(s) for intravenous (IV) and subcutaneous (SC) administration in the dose escalation part.
To determine the safety of SAR443216 after intravenous (IV) and subcutaneous (SC) administration.

Part 2 (Dose expansion)

• To assess preliminary clinical activity of single agent SAR443216 at the RD(s) in participants with HER2 expressing solid tumors, with various levels of HER2 expression.

Secondary Objectives:

Part 1 • To assess preliminary clinical activity of single agent SAR443216 after IV and SC administration at the RD(s) in participants with HER2 expressing solid tumors, with various levels of HER2 expression.

Part 2

• To determine the safety of SAR443216.

Part 1 and 2

To characterize the pharmacokinetic (PK) profile of SAR443216 when administered as a single agent after IV and SC (Part 1 only) administration.
To evaluate the immunogenicity of SAR443216 after IV and SC administration.
To assess preliminary clinical activity of single agent SAR443216 at the RD(s) in participants with HER2 expressing solid tumors, with various levels of HER2 expression.

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

location_facility: ~University of Texas - MD Anderson Cancer Center Site Number : 8400002


title: A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe

summary: This is a retrospective, observational, multicenter study to collect Real-World Evidence (RWE) data on systemic AL-AMY patients in Europe. Data from paper/electronic medical records and/or electronic databases from key reference centers in Europe will be used. Data will either be entered by the site staff in the electronic Case Report Form (eCRF) or, where feasible, transferred directly, always in accordance to local regulations.

lead_sponsor: European Myeloma Network

location_country:
Germany
Italy
Portugal
United Kingdom
Netherlands
Austria
Spain
Greece
Czechia
France

location_facility: Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers

location_facility: Servicio de Hematología, Hospital Clínic de Barcelona

location_facility: Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology

location_facility: Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics

location_facility: Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University

location_facility: Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy

location_facility: University Hospital Ostrava, Clinic of Hematooncology

location_facility: Centro Hospitalar E Universitário São João

location_facility: University College London Medical School, Uk Royal Free Hospital

location_facility: Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien

location_facility: Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini

location_facility: University Medical Center Utrecht, Umc Utrecht, Department of Hematology


title: An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

acronym: APeX-A

summary: This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

lead_sponsor: BioCryst Pharmaceuticals

location_country:
Poland
North Macedonia
Slovakia
Korea, Republic of
South Africa
Czechia
France


title: A Prospective, Open, Non-comparative, Post-market Clinical Follow-up Investigation to Confirm Safety and Performance of Avance Solo NPWT System in Low to Moderate Exuding Acute (Traumatic Wounds and Flaps and Grafts), as Well as Subacute (e.g., Dehisced Wounds) Wounds

acronym: ASOLO-SW

summary: The primary objective of this post market clinical follow-up (PMCF) investigation is to confirm safety and performance of Avance® Solo NPWT System in low to moderate exuding acute (traumatic wounds and flaps and grafts), as well as subacute (e.g., dehisced wounds) wounds when used in accordance with the Instructions for Use, for up to 28 days.

lead_sponsor: Molnlycke Health Care AB

sponsor_collaborators:
Molnlycke Health Care AB
Syntactx

location_country: Germany Italy Belgium France

location_facility: UNIVERSITÄTSKLINIKUM Schleswig-Holstein Campus Lübeck

location_facility: A.O.U. Citta della Salute e della Scienza di Torino - A.O.U. Molinette San Giovanni Battista