title: Influencia Del Estrés Agudo en el Aprendizaje Motor de Una Tarea Manual Precisa, en la Memoria a Corto y Largo Plazo y en la Capacidad de Imaginación Motora

summary:
In motor learning, it is essential to consider that movements are produced by the cooperation and combination of many brain structures and are influenced by the emotions to which individuals are subjected. Several neural circuits have been identified that closely link the emotional system and the motion control system.

Stress is a physiological or psychological response to internal or external stressors. In principle, it has an adaptive role. However, the neuroendocrine and autonomic response generated by stress can affect cognitive processes such as memory. In addition, it has been shown to influence motor learning, especially the execution of skills in the early stages of learning.

Understanding how movement, emotions and interactions are regulated is significant because of the large number of movements humans perform. Of these, manual tasks represent precise movements that require the integration of many elements by the nervous system to perform these tasks successfully. It is still unknown how acute stress influence the way manual tasks are learned.

On the other hand, motor imagery (MI) is a cognitive process that is an important contributor to how movements are planned and executed. Its use has been recommended to improve movement learning and task execution. For an MI program to be effective and individualized, it is imperative to know this capacity. However, it is also still unknown how acute stress can affect our motor imagery ability.

The main objective of this study is to determine and quantify the effects of acute stress in the learning of a precise manual task not previously trained on four parameters of fine motor control: time, error, speed, and accuracy. On the other hand, the aim is to determine if the ability of internal visual, external visual, and kinaesthetic imagery varies when we are subjected to acute stress.

It is expected that non-anxious, non-stressed participants who are not induced with acute stress will show better motor performance on the fine motor task and better motor imagery ability. In contrast, it is expected that participants without anxiety and stress who are induced with acute stress will show poorer motor performance on the fine motor task and poorer motor imagery ability.

ReferenceCitation: Ahn SN, Lee J. Effects of sensory awareness, imagery and observation on electroencephalography in adult with psychological stress. J Phys Ther Sci. 2019 Jan;31(1):17-19. doi: 10.1589/jpts.31.17. Epub 2019 Jan 10.

ReferenceCitation: Smeets T, Cornelisse S, Quaedflieg CW, Meyer T, Jelicic M, Merckelbach H. Introducing the Maastricht Acute Stress Test (MAST): a quick and non-invasive approach to elicit robust autonomic and glucocorticoid stress responses. Psychoneuroendocrinology. 2012 Dec;37(12):1998-2008. doi: 10.1016/j.psyneuen.2012.04.012. Epub 2012 May 18.

ReferenceCitation: Bali A, Jaggi AS. Clinical experimental stress studies: methods and assessment. Rev Neurosci. 2015;26(5):555-79. doi: 10.1515/revneuro-2015-0004. Review.

location_facility:
European University of Madrid
Claude Bernard University Lyon 1
Complutense University of Madrid
University of Alcala


title: Influencia de Los Niveles de Alerta en el Aprendizaje Motor de Una Tarea Manual Precisa, en la Memoria a Corto y Largo Plazo y en la Capacidad de Imaginación Motora

summary:
In motor learning is essential to consider that movements are produced by the cooperation and combination of many brain structures and are influenced by the emotions to which individuals are subjected. Several neural circuits have been identified that closely link the emotional system and the motion control system.

Arousal is associated with many emotional responses and has effects on the nervous and motor system. In line with the "Inverted 'U' Hypothesis", all levels of both high and low arousal do not allow optimal task performance, yet moderate levels lead to excellent performance. Arousal also plays a vital role in movement learning, where a critical element is memory. There is evidence that a minimum level of arousal is required to encode or record information and that that moderate levels of arousal improve memory.

Understanding how movement, emotions and interactions are regulated is significant because of the large number of movements humans perform. Of these, manual tasks represent precise movements that require the integration of many elements by the nervous system to perform these tasks successfully. How different levels of arousal influence the way manual tasks are learned is still unknown.

On the other hand, motor imagery (MI) is a cognitive process that is an important contributor to how movements are planned and executed. The use of MI has been recommended to improve movement learning and task execution. For an effective and individualize MI program is imperative to know this capacity. However, how different levels of arousal can affect our motor imagery ability is also still unknown.

The main objective of this study is to determine and quantify the effects of arousal levels in the learning of a precise manual task not previously trained on four parameters of fine motor control: time, error, speed, and accuracy. On the other hand, the aim is to determine if the ability of internal visual, external visual, and kinaesthetic imagery varies when participants are subjected to different levels of arousal.

Researches expect that non-anxious, non-stressed participants who are shown images that elicit an optimal level of arousal will show better motor performance on the fine motor task and better motor imagery ability. In contrast, researches expect that participants without anxiety and stress who are shown pictures that elicit a sub-optimal level of arousal will show poorer motor performance on the fine motor task and poorer motor imagery ability.

study_start_date_type: Actual

status: Recruiting

study_completion_date_type: Anticipated

study_type: Interventional

design_allocation: Randomized

primary_purpose: Basic Science

enrollment_type: Anticipated

ReferenceType: background

ReferenceCitation: Stins JF, Beek PJ. Effects of affective picture viewing on postural control. BMC Neurosci. 2007 Oct 4;8:83.

ReferenceCitation: Coombes SA, Cauraugh JH, Janelle CM. Emotion and movement: activation of defensive circuitry alters the magnitude of a sustained muscle contraction. Neurosci Lett. 2006 Apr 3;396(3):192-6. Epub 2005 Dec 20.

ReferenceCitation: MacIntyre TE, Madan CR, Moran AP, Collet C, Guillot A. Motor imagery, performance and motor rehabilitation. Prog Brain Res. 2018;240:141-159. doi: 10.1016/bs.pbr.2018.09.010. Epub 2018 Oct 24. Review.

ReferenceCitation: Schuster C, Hilfiker R, Amft O, Scheidhauer A, Andrews B, Butler J, Kischka U, Ettlin T. Best practice for motor imagery: a systematic literature review on motor imagery training elements in five different disciplines. BMC Med. 2011 Jun 17;9:75. doi: 10.1186/1741-7015-9-75. Review.

ReferenceCitation: Trapero-Asenjo S, Gallego-Izquierdo T, Pecos-Martín D, Nunez-Nagy S. Translation, cultural adaptation, and validation of the Spanish version of the Movement Imagery Questionnaire-3 (MIQ-3). Musculoskelet Sci Pract. 2021 Feb;51:102313. doi: 10.1016/j.msksp.2020.102313. Epub 2020 Dec 9.

ReferenceCitation: Oldfield RC. The assessment and analysis of handedness: the Edinburgh inventory. Neuropsychologia. 1971 Mar;9(1):97-113.

lead_sponsor: Susana Nunez Nagy

location_country: France Spain

intervention_type: Other

lead_sponsor_type: industriel

status_simplified: Ongoing


type: Other Grant/Funding Number

title: A Multimodal Approach to Personalized Tracking of Evolving State-Of-Consciousness in Brain- Injured Patients

acronym: PerBrain

summary: Improved treatment of severe brain injuries has resulted in increased survival rates. While some of these patients regain consciousness after a transient state of coma, others may develop a disorder of consciousness (DoC). Diagnosis of DoC currently relies on standardized behavioral assessment. The importance of accuracy in such diagnosis cannot be overstated, as it guides critical decisions on treatment (including pain management), and could underlie end-of-life decisions. Despite this importance, current behavioral diagnosis often fails, if because of the major sensory and motor deficits associated with DoC, or because of the heterogeneous etiology and pathophysiology associated with the condition. Finally, the need for accurate diagnosis and prognosis transcends the needs of the patients alone: caregiving of these patients is very stressful, principally for the large uncertainty associated with them. Thus, more accurate diagnosis and prognosis provide major relief for caregivers, and paradoxically, even if the news is not "good". For all these reasons it is critical to developing personalized diagnosis and prognosis prediction tools that permit a stratified analysis at the single-patient level. The PerBrain Project will benefit from the multidisciplinary partners' expertise, and the unique opportunity to perform longitudinal assessments in four clinical sites through both established and novel electrophysiological, neuroimaging, and physiological techniques. Based on the collected data, the investigators will develop a multimodal personalized diagnostic tool for DoC patients using state-of-the-art computational tools, such as machine learning, in order to better determine the current state (diagnosis) and future outcome (prognosis). The overall aim of this project will provide for a better understanding of the pathophysiological mechanisms in DoC, which will, in turn, allow personalized rehabilitation strategies, and improved single-patient predictions of state and prognosis.

study_type: Observational

time_perspective: Prospective

ReferenceCitation: Giacino JT, Kalmar K. Diagnostic and prognostic guidelines for the vegetative and minimally conscious states. Neuropsychol Rehabil. 2005 Jul-Sep;15(3-4):166-74.

ReferenceCitation: Raimondo F, Rohaut B, Demertzi A, Valente M, Engemann DA, Salti M, Fernandez Slezak D, Naccache L, Sitt JD. Brain-heart interactions reveal consciousness in noncommunicating patients. Ann Neurol. 2017 Oct;82(4):578-591. doi: 10.1002/ana.25045. Epub 2017 Oct 11.

ReferenceCitation: Arzi A, Rozenkrantz L, Gorodisky L, Rozenkrantz D, Holtzman Y, Ravia A, Bekinschtein TA, Galperin T, Krimchansky BZ, Cohen G, Oksamitni A, Aidinoff E, Sacher Y, Sobel N. Olfactory sniffing signals consciousness in unresponsive patients with brain injuries. Nature. 2020 May;581(7809):428-433. doi: 10.1038/s41586-020-2245-5. Epub 2020 Apr 29.

ReferenceCitation: Casali AG, Gosseries O, Rosanova M, Boly M, Sarasso S, Casali KR, Casarotto S, Bruno MA, Laureys S, Tononi G, Massimini M. A theoretically based index of consciousness independent of sensory processing and behavior. Sci Transl Med. 2013 Aug 14;5(198):198ra105. doi: 10.1126/scitranslmed.3006294.

ReferenceCitation: Engemann DA, Raimondo F, King JR, Rohaut B, Louppe G, Faugeras F, Annen J, Cassol H, Gosseries O, Fernandez-Slezak D, Laureys S, Naccache L, Dehaene S, Sitt JD. Robust EEG-based cross-site and cross-protocol classification of states of consciousness. Brain. 2018 Nov 1;141(11):3179-3192. doi: 10.1093/brain/awy251.

ReferenceCitation: Vogler J, Klein AM, Bender A. Long-term health-related quality-of-life in patients with acquired brain injury and their caregivers. Brain Inj. 2014;28(11):1381-8. doi: 10.3109/02699052.2014.919536. Epub 2014 Jun 19.

ipd_sharing: No

lead_sponsor: Brain & Spine Institute (ICM)

location_country: Israel Germany Italy France

location_facility: University Hospital of the Ludwig-Maximilians-University of Munich

location_facility: Dipartimento di Cura e Riabilitazione delle Gravi Cerebrolesioni Acquisite (GCA)

intervention_type: Behavioral


title: Exhaled Breath Analysis Using eNose Technology as a Biomarker for Diagnosis and Disease Progression in Fibrotic Interstitial Lung Disease

acronym: ILDnose

summary: The ILDnose study a multinational, multicenter, prospective, longitudinal study in outpatients with pulmonary fibrosis. The aim is to assess the accuracy of eNose technology as diagnostic tool for diagnosis and differentiation between the most prevalent fibrotic interstitial lung diseases. The value of eNose as biomarker for disease progression and response to treatment is also assessed. Besides, validity of several questionnaires for pulmonary fibrosis is investigated.

primary_purpose: Diagnostic

lead_sponsor: Erasmus Medical Center

location_country:
Germany
United Kingdom
France
Netherlands

location_facility: University Lyon 1, Louis Pradel hospital, Lyon. FranceService de pneumologie, hôpital Louis Pradel

intervention_type: Diagnostic Test


title: An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

acronym: NEXUS

summary: An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)

primary_purpose: Treatment

lead_sponsor: Minoryx Therapeutics, S.L.

location_country: Germany France Spain

location_facility: Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

intervention_type: Drug


title: An Integrated European Platform to Conduct Translational Studies in Myelodysplastic Syndromes Based on the EuroBloodNet Infrastructure

summary:
Rationale Myelodysplastic syndromes (MDS) are rare cancers with unmet medical needs. Study of MDS has been rapidly transformed by genome characterization.

The investigators hypothesize that comprehensive analyses of large patient population will allow to correctly estimate the effect of each mutation on clinical outcomes, and that niche factors and immune dysfunctions may influence the development of MDS, clonal evolution and response to treatments

Aims

1- Investigate gene mutations, niche factors and immune dysfunctions influencing the development of MDS, and define biomarkers for early identification of individuals at risk; 2- Develop prognostic models for MDS patients through integration of comprehensive genomic/clinical information; 3- Define biomarkers to better stratify the individual probability of response to specific treatments

Methods EuroBloodNet, the European Reference Network in rare hematological diseases, will provide a basis for research activities. Study of genomic features of clonal dominance in elderly subjects enrolled in large population-based studies and description of the dynamics of clonal establishment and evolution; study of bone marrow microenvironment to identify immune dysfunctions influencing MDS development. Development of inclusive statistical models to accurately predict clinical outcome at individual level, based on large MDS populations with comprehensive genomic/clinical data. Finally, analysis of mutational screening and immune profiles from patients enrolled in prospective trials, to provide evidence on genetic/immunologic profiles associated with probability of response to specific compounds

Expected results To characterize how clonal hematopoiesis relates to the induction of MDS clinical phenotype, and to test the utility of gene sequencing to detect subjects at risk of developing MDS. To define effective prognostic systems and biomarkers to stratify the individual probability of response to treatment

time_perspective: Other

ReferenceCitation: Adès L, Itzykson R, Fenaux P. Myelodysplastic syndromes. Lancet. 2014 Jun 28;383(9936):2239-52. doi: 10.1016/S0140-6736(13)61901-7. Epub 2014 Mar 21. Review.

ReferenceCitation: Malcovati L, Hellström-Lindberg E, Bowen D, Adès L, Cermak J, Del Cañizo C, Della Porta MG, Fenaux P, Gattermann N, Germing U, Jansen JH, Mittelman M, Mufti G, Platzbecker U, Sanz GF, Selleslag D, Skov-Holm M, Stauder R, Symeonidis A, van de Loosdrecht AA, de Witte T, Cazzola M; European Leukemia Net. Diagnosis and treatment of primary myelodysplastic syndromes in adults: recommendations from the European LeukemiaNet. Blood. 2013 Oct 24;122(17):2943-64. doi: 10.1182/blood-2013-03-492884. Epub 2013 Aug 26.

ReferenceCitation: Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Solé F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstöcker M, Sekeres M, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20;120(12):2454-65. Epub 2012 Jun 27.

ReferenceCitation: Schanz J, Tüchler H, Solé F, Mallo M, Luño E, Cervera J, Granada I, Hildebrandt B, Slovak ML, Ohyashiki K, Steidl C, Fonatsch C, Pfeilstöcker M, Nösslinger T, Valent P, Giagounidis A, Aul C, Lübbert M, Stauder R, Krieger O, Garcia-Manero G, Faderl S, Pierce S, Le Beau MM, Bennett JM, Greenberg P, Germing U, Haase D. New comprehensive cytogenetic scoring system for primary myelodysplastic syndromes (MDS) and oligoblastic acute myeloid leukemia after MDS derived from an international database merge. J Clin Oncol. 2012 Mar 10;30(8):820-9. doi: 10.1200/JCO.2011.35.6394. Epub 2012 Feb 13.

ReferenceCitation: Nomdedeu M, Calvo X, Pereira A, Carrió A, Solé F, Luño E, Cervera J, Vallespí T, Muñoz C, Gómez C, Arias A, Such E, Sanz G, Grau J, Insunza A, Calasanz MJ, Ardanaz MT, Hernández-Rivas JM, Azaceta G, Álvarez S, Sánchez J, Martín ML, Bargay J, Gómez V, Cervero CJ, Allegue MJ, Collado R, Campo E, Esteve J, Nomdedeu B, Costa D; Spanish Group of Myelodysplastic Syndromes. Prognostic impact of chromosomal translocations in myelodysplastic syndromes and chronic myelomonocytic leukemia patients. A study by the spanish group of myelodysplastic syndromes. Genes Chromosomes Cancer. 2016 Apr;55(4):322-7. doi: 10.1002/gcc.22333. Epub 2015 Dec 22.

ReferenceCitation: Schanz J, Tüchler H, Solé F, Mallo M, Luño E, Cervera J, Grau J, Hildebrandt B, Slovak ML, Ohyashiki K, Steidl C, Fonatsch C, Pfeilstöcker M, Nösslinger T, Valent P, Giagounidis A, Aul C, Lübbert M, Stauder R, Krieger O, Le Beau MM, Bennett JM, Greenberg P, Germing U, Haase D. Monosomal karyotype in MDS: explaining the poor prognosis? Leukemia. 2013 Oct;27(10):1988-95. doi: 10.1038/leu.2013.187. Epub 2013 Jun 21.

ReferenceCitation: Valcárcel D, Ademà V, Solé F, Ortega M, Nomdedeu B, Sanz G, Luño E, Cañizo C, de la Serna J, Ardanaz M, Marco V, Collado R, Grau J, Montoro J, Mallo M, Vallespí T. Complex, not monosomal, karyotype is the cytogenetic marker of poorest prognosis in patients with primary myelodysplastic syndrome. J Clin Oncol. 2013 Mar 1;31(7):916-22. doi: 10.1200/JCO.2012.41.6073. Epub 2013 Jan 14.

ReferenceCitation: Della Porta MG, Alessandrino EP, Bacigalupo A, van Lint MT, Malcovati L, Pascutto C, Falda M, Bernardi M, Onida F, Guidi S, Iori AP, Cerretti R, Marenco P, Pioltelli P, Angelucci E, Oneto R, Ripamonti F, Bernasconi P, Bosi A, Cazzola M, Rambaldi A; Gruppo Italiano Trapianto di Midollo Osseo. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014 Apr 10;123(15):2333-42. doi: 10.1182/blood-2013-12-542720. Epub 2014 Feb 20.

ReferenceCitation: Papaemmanuil E, Gerstung M, Malcovati L, Tauro S, Gundem G, Van Loo P, Yoon CJ, Ellis P, Wedge DC, Pellagatti A, Shlien A, Groves MJ, Forbes SA, Raine K, Hinton J, Mudie LJ, McLaren S, Hardy C, Latimer C, Della Porta MG, O'Meara S, Ambaglio I, Galli A, Butler AP, Walldin G, Teague JW, Quek L, Sternberg A, Gambacorti-Passerini C, Cross NC, Green AR, Boultwood J, Vyas P, Hellstrom-Lindberg E, Bowen D, Cazzola M, Stratton MR, Campbell PJ; Chronic Myeloid Disorders Working Group of the International Cancer Genome Consortium. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013 Nov 21;122(22):3616-27; quiz 3699. doi: 10.1182/blood-2013-08-518886. Epub 2013 Sep 12.

ReferenceCitation: Papaemmanuil E, Cazzola M, Boultwood J, Malcovati L, Vyas P, Bowen D, Pellagatti A, Wainscoat JS, Hellstrom-Lindberg E, Gambacorti-Passerini C, Godfrey AL, Rapado I, Cvejic A, Rance R, McGee C, Ellis P, Mudie LJ, Stephens PJ, McLaren S, Massie CE, Tarpey PS, Varela I, Nik-Zainal S, Davies HR, Shlien A, Jones D, Raine K, Hinton J, Butler AP, Teague JW, Baxter EJ, Score J, Galli A, Della Porta MG, Travaglino E, Groves M, Tauro S, Munshi NC, Anderson KC, El-Naggar A, Fischer A, Mustonen V, Warren AJ, Cross NC, Green AR, Futreal PA, Stratton MR, Campbell PJ; Chronic Myeloid Disorders Working Group of the International Cancer Genome Consortium. Somatic SF3B1 mutation in myelodysplasia with ring sideroblasts. N Engl J Med. 2011 Oct 13;365(15):1384-95. doi: 10.1056/NEJMoa1103283. Epub 2011 Sep 26.

ReferenceCitation: Della Porta MG, Tuechler H, Malcovati L, Schanz J, Sanz G, Garcia-Manero G, Solé F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstöcker M, Sekeres MA, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D, Greenberg PL, Cazzola M. Validation of WHO classification-based Prognostic Scoring System (WPSS) for myelodysplastic syndromes and comparison with the revised International Prognostic Scoring System (IPSS-R). A study of the International Working Group for Prognosis in Myelodysplasia (IWG-PM). Leukemia. 2015 Jul;29(7):1502-13. doi: 10.1038/leu.2015.55. Epub 2015 Feb 27.

ReferenceCitation: Della Porta MG, Travaglino E, Boveri E, Ponzoni M, Malcovati L, Papaemmanuil E, Rigolin GM, Pascutto C, Croci G, Gianelli U, Milani R, Ambaglio I, Elena C, Ubezio M, Da Via' MC, Bono E, Pietra D, Quaglia F, Bastia R, Ferretti V, Cuneo A, Morra E, Campbell PJ, Orazi A, Invernizzi R, Cazzola M; Rete Ematologica Lombarda (REL) Clinical Network. Minimal morphological criteria for defining bone marrow dysplasia: a basis for clinical implementation of WHO classification of myelodysplastic syndromes. Leukemia. 2015 Jan;29(1):66-75. doi: 10.1038/leu.2014.161. Epub 2014 May 20.

ReferenceCitation: Della Porta MG, Jackson CH, Alessandrino EP, Rossi M, Bacigalupo A, van Lint MT, Bernardi M, Allione B, Bosi A, Guidi S, Santini V, Malcovati L, Ubezio M, Milanesi C, Todisco E, Voso MT, Musto P, Onida F, Iori AP, Cerretti R, Grillo G, Molteni A, Pioltelli P, Borin L, Angelucci E, Oldani E, Sica S, Pascutto C, Ferretti V, Santoro A, Bonifazi F, Cazzola M, Rambaldi A. Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System. Leukemia. 2017 Nov;31(11):2449-2457. doi: 10.1038/leu.2017.88. Epub 2017 Mar 21.

ReferenceCitation: Jaiswal S, Fontanillas P, Flannick J, Manning A, Grauman PV, Mar BG, Lindsley RC, Mermel CH, Burtt N, Chavez A, Higgins JM, Moltchanov V, Kuo FC, Kluk MJ, Henderson B, Kinnunen L, Koistinen HA, Ladenvall C, Getz G, Correa A, Banahan BF, Gabriel S, Kathiresan S, Stringham HM, McCarthy MI, Boehnke M, Tuomilehto J, Haiman C, Groop L, Atzmon G, Wilson JG, Neuberg D, Altshuler D, Ebert BL. Age-related clonal hematopoiesis associated with adverse outcomes. N Engl J Med. 2014 Dec 25;371(26):2488-98. doi: 10.1056/NEJMoa1408617. Epub 2014 Nov 26.

ReferenceCitation: Weidner H, Rauner M, Trautmann F, Schmitt J, Balaian E, Mies A, Helas S, Baschant U, Khandanpour C, Bornhäuser M, Hofbauer LC, Platzbecker U. Myelodysplastic syndromes and bone loss in mice and men. Leukemia. 2017 Apr;31(4):1003-1007. doi: 10.1038/leu.2017.7. Epub 2017 Jan 11.

ReferenceCitation: Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.

family: Fenaux

full_name: Pierre Fenaux

given: Pierre

sequence: first

family: Mufti

full_name: Ghulam J Mufti

given: Ghulam J

sequence: additional

family: Hellstrom-Lindberg

full_name: Eva Hellstrom-Lindberg

given: Eva

family: Santini

full_name: Valeria Santini

given: Valeria

family: Finelli

full_name: Carlo Finelli

given: Carlo

family: Giagounidis

full_name: Aristoteles Giagounidis

given: Aristoteles

family: Schoch

full_name: Robert Schoch

given: Robert

family: Gattermann

full_name: Norbert Gattermann

given: Norbert

family: Sanz

full_name: Guillermo Sanz

given: Guillermo

family: List

full_name: Alan List

given: Alan

family: Gore

full_name: Steven D Gore

given: Steven D

family: Seymour

full_name: John F Seymour

given: John F

family: Bennett

full_name: John M Bennett

given: John M

family: Byrd

full_name: John Byrd

given: John

family: Backstrom

full_name: Jay Backstrom

given: Jay

family: Zimmerman

full_name: Linda Zimmerman

given: Linda

family: McKenzie

full_name: David McKenzie

given: David

family: Beach

full_name: CL Beach

given: CL

family: Silverman

full_name: Lewis R Silverman

given: Lewis R

bso_classification: Medical research

datasource: user

ReferenceCitation: Fenaux P, Giagounidis A, Selleslag D, Beyne-Rauzy O, Mufti G, Mittelman M, Muus P, Te Boekhorst P, Sanz G, Del Cañizo C, Guerci-Bresler A, Nilsson L, Platzbecker U, Lübbert M, Quesnel B, Cazzola M, Ganser A, Bowen D, Schlegelberger B, Aul C, Knight R, Francis J, Fu T, Hellström-Lindberg E; MDS-004 Lenalidomide del5q Study Group. A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q. Blood. 2011 Oct 6;118(14):3765-76. doi: 10.1182/blood-2011-01-330126. Epub 2011 Jul 13.

name: Hopital Avicenne, Assistance Publique–Hôpitaux de Paris, Université Paris XIII, Bobigny, France;

name: St Johannes Hospital, Duisburg, Germany;

name: Centre Hospitalier Universitaire Purpan Pavillion de Médecines, Toulouse, France;

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

name: Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands;

name: Erasmus Medical Center, Rotterdam, The Netherlands;

name: Hospital Universitario de Salamanca, Salamanca, Spain;

name: Centre Hospitalier Universitaire Nancy Hématologie et Médecine Interne, Vandoeuvre, France;

name: Skåne University Hospital, Lund, Sweden;

name: Universitätsklinikum Carl Gustav Carus, Dresden, Germany;

name: University of Freiburg Medical Center, Freiburg, Germany;

name: Centre Hospitalier Régional Universitaire Claude Huriez Service des Maladies du Sang, Lille, France;

name: Department of Hematology Oncology, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, University of Pavia, Pavia, Italy;

name: Hannover Medical School, Hannover, Germany;

name: St James's Institute of Oncology, Leeds, United Kingdom;

name: Karolinska Institutet, Karolinska University Hospital, Stockholm, Sweden

name: for the MDS-004 Lenalidomide del5q Study Group

ReferenceCitation: Mallo M, Luño E, Sanzo C, Cervera J, Haase D, Schanz J, García-Manero G, del Cañizo C, Sanz GF, Solé F. Clinical impact of the clone size in MDS cases with monosomy 7 or 7q deletion, trisomy 8, 20q deletion and loss of Y chromosome. Leuk Res. 2011 Jun;35(6):834-6. doi: 10.1016/j.leukres.2011.01.003. Epub 2011 Jan 26.

ReferenceCitation: Della Porta MG, Gallì A, Bacigalupo A, Zibellini S, Bernardi M, Rizzo E, Allione B, van Lint MT, Pioltelli P, Marenco P, Bosi A, Voso MT, Sica S, Cuzzola M, Angelucci E, Rossi M, Ubezio M, Malovini A, Limongelli I, Ferretti VV, Spinelli O, Tresoldi C, Pozzi S, Luchetti S, Pezzetti L, Catricalà S, Milanesi C, Riva A, Bruno B, Ciceri F, Bonifazi F, Bellazzi R, Papaemmanuil E, Santoro A, Alessandrino EP, Rambaldi A, Cazzola M. Clinical Effects of Driver Somatic Mutations on the Outcomes of Patients With Myelodysplastic Syndromes Treated With Allogeneic Hematopoietic Stem-Cell Transplantation. J Clin Oncol. 2016 Oct 20;34(30):3627-3637. doi: 10.1200/JCO.2016.67.3616.

ReferenceCitation: Gerstung M, Pellagatti A, Malcovati L, Giagounidis A, Porta MG, Jädersten M, Dolatshad H, Verma A, Cross NC, Vyas P, Killick S, Hellström-Lindberg E, Cazzola M, Papaemmanuil E, Campbell PJ, Boultwood J. Combining gene mutation with gene expression data improves outcome prediction in myelodysplastic syndromes. Nat Commun. 2015 Jan 9;6:5901. doi: 10.1038/ncomms6901.

ReferenceCitation: Gerstung M, Papaemmanuil E, Martincorena I, Bullinger L, Gaidzik VI, Paschka P, Heuser M, Thol F, Bolli N, Ganly P, Ganser A, McDermott U, Döhner K, Schlenk RF, Döhner H, Campbell PJ. Precision oncology for acute myeloid leukemia using a knowledge bank approach. Nat Genet. 2017 Mar;49(3):332-340. doi: 10.1038/ng.3756. Epub 2017 Jan 16.

ReferenceCitation: Schneider RK, Ademà V, Heckl D, Järås M, Mallo M, Lord AM, Chu LP, McConkey ME, Kramann R, Mullally A, Bejar R, Solé F, Ebert BL. Role of casein kinase 1A1 in the biology and targeted therapy of del(5q) MDS. Cancer Cell. 2014 Oct 13;26(4):509-20. doi: 10.1016/j.ccr.2014.08.001. Epub 2014 Sep 18.

ReferenceCitation: Platzbecker U, Germing U, Götze KS, Kiewe P, Mayer K, Chromik J, Radsak M, Wolff T, Zhang X, Laadem A, Sherman ML, Attie KM, Giagounidis A. Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study. Lancet Oncol. 2017 Oct;18(10):1338-1347. doi: 10.1016/S1470-2045(17)30615-0. Epub 2017 Sep 1. Erratum in: Lancet Oncol. 2017 Oct;18(10):e562.

ReferenceCitation: Platzbecker U, Hofbauer LC, Ehninger G, Hölig K. The clinical, quality of life, and economic consequences of chronic anemia and transfusion support in patients with myelodysplastic syndromes. Leuk Res. 2012 May;36(5):525-36. doi: 10.1016/j.leukres.2012.01.006. Epub 2012 Feb 1. Review.

ReferenceCitation: Platzbecker U, Schetelig J, Finke J, Trenschel R, Scott BL, Kobbe G, Schaefer-Eckart K, Bornhäuser M, Itzykson R, Germing U, Beelen D, Ehninger G, Fenaux P, Deeg HJ, Adès L; German MDS Study; Cooperative Transplant Study Group; Fred Hutchinson Cancer Research Center; Groupe Francophone des Myelodysplasies. Allogeneic hematopoietic cell transplantation in patients age 60-70 years with de novo high-risk myelodysplastic syndrome or secondary acute myelogenous leukemia: comparison with patients lacking donors who received azacitidine. Biol Blood Marrow Transplant. 2012 Sep;18(9):1415-21. doi: 10.1016/j.bbmt.2012.05.003. Epub 2012 May 11.

ReferenceCitation: Park S, Hamel JF, Toma A, Kelaidi C, Thépot S, Campelo MD, Santini V, Sekeres MA, Balleari E, Kaivers J, Sapena R, Götze K, Müller-Thomas C, Beyne-Rauzy O, Stamatoullas A, Kotsianidis I, Komrokji R, Steensma DP, Fensterl J, Roboz GJ, Bernal T, Ramos F, Calabuig M, Guerci-Bresler A, Bordessoule D, Cony-Makhoul P, Cheze S, Wattel E, Rose C, Vey N, Gioia D, Ferrero D, Gaidano G, Cametti G, Pane F, Sanna A, Germing U, Sanz GF, Dreyfus F, Fenaux P. Outcome of Lower-Risk Patients With Myelodysplastic Syndromes Without 5q Deletion After Failure of Erythropoiesis-Stimulating Agents. J Clin Oncol. 2017 May 10;35(14):1591-1597. doi: 10.1200/JCO.2016.71.3271. Epub 2017 Mar 28.

ReferenceCitation: Lamarque M, Raynaud S, Itzykson R, Thepot S, Quesnel B, Dreyfus F, Rauzy OB, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Visanica S, Stamatoullas A, Fenaux P, Adès L. The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience. Blood. 2012 Dec 13;120(25):5084-5. doi: 10.1182/blood-2012-09-453555. Erratum in: Blood. 2014 Jun 26;123(26):4152.

ReferenceCitation: Itzykson R, Thépot S, Quesnel B, Dreyfus F, Beyne-Rauzy O, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Salanoubat C, Visanica S, Stamatoullas A, Isnard F, Marfaing-Koka A, de Botton S, Chelghoum Y, Taksin AL, Plantier I, Ame S, Boehrer S, Gardin C, Beach CL, Adès L, Fenaux P; Groupe Francophone des Myelodysplasies(GFM). Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine. Blood. 2011 Jan 13;117(2):403-11. doi: 10.1182/blood-2010-06-289280. Epub 2010 Oct 12.

ReferenceCitation: Fenaux P, Mufti GJ, Hellström-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.

name: From the Avicenne, Assistance Publique-Hôpitaux de Paris (AP-HP), Université Paris XIII, Bobigny; Hôpital Saint-Louis, AP-HP, Université Paris 7, Paris, France; King's College Hospital, London, United Kingdom; Karolinska University Hospital (Huddinge), Stockholm, Sweden; Azienda Ospedaliera Universitaria Careggi, Florence, Italy; Universitätsklinikum Düsseldorf, Düsseldorf, Germany; Hospital Universitario La Fe, Valencia, Spain; Peter MacCallum Cancer Centre and University of Melbourne, Melbourne,...

publisher: American Society of Clinical Oncology (ASCO)

title: Azacitidine Prolongs Overall Survival Compared With Conventional Care Regimens in Elderly Patients With Low Bone Marrow Blast Count Acute Myeloid Leukemia

ReferenceCitation: Mallo M, Del Rey M, Ibáñez M, Calasanz MJ, Arenillas L, Larráyoz MJ, Pedro C, Jerez A, Maciejewski J, Costa D, Nomdedeu M, Diez-Campelo M, Lumbreras E, González-Martínez T, Marugán I, Such E, Cervera J, Cigudosa JC, Alvarez S, Florensa L, Hernández JM, Solé F. Response to lenalidomide in myelodysplastic syndromes with del(5q): influence of cytogenetics and mutations. Br J Haematol. 2013 Jul;162(1):74-86. doi: 10.1111/bjh.12354. Epub 2013 Apr 25.

ReferenceCitation: Mekinian A, Grignano E, Braun T, Decaux O, Liozon E, Costedoat-Chalumeau N, Kahn JE, Hamidou M, Park S, Puéchal X, Toussirot E, Falgarone G, Launay D, Morel N, Trouiller S, Mathian A, Gombert B, Schoindre Y, Lioger B, De Wazieres B, Amoura Z, Buchdaul AL, Georgin-Lavialle S, Dion J, Madaule S, Raffray L, Cathebras P, Piette JC, Rose C, Ziza JM, Lortholary O, Montestruc F, Omouri M, Denis G, Rossignol J, Nimubona S, Adès L, Gardin C, Fenaux P, Fain O. Systemic inflammatory and autoimmune manifestations associated with myelodysplastic syndromes and chronic myelomonocytic leukaemia: a French multicentre retrospective study. Rheumatology (Oxford). 2016 Feb;55(2):291-300. doi: 10.1093/rheumatology/kev294. Epub 2015 Sep 8.

ReferenceCitation: Fraison JB, Mekinian A, Grignano E, Kahn JE, Arlet JB, Decaux O, Denis G, Buchdahl AL, Omouri M, Maigne G, Aouba A, Leon N, Berthier S, Liozon E, Park S, Gardin C, Lortholary O, Rossignol J, Fenaux P, Fain O, Braun T. Efficacy of Azacitidine in autoimmune and inflammatory disorders associated with myelodysplastic syndromes and chronic myelomonocytic leukemia. Leuk Res. 2016 Apr;43:13-7. doi: 10.1016/j.leukres.2016.02.005. Epub 2016 Feb 20.

ReferenceCitation: Khaznadar Z, Boissel N, Agaugué S, Henry G, Cheok M, Vignon M, Geromin D, Cayuela JM, Castaigne S, Pautas C, Raffoux E, Lachuer J, Sigaux F, Preudhomme C, Dombret H, Dulphy N, Toubert A. Defective NK Cells in Acute Myeloid Leukemia Patients at Diagnosis Are Associated with Blast Transcriptional Signatures of Immune Evasion. J Immunol. 2015 Sep 15;195(6):2580-90. doi: 10.4049/jimmunol.1500262. Epub 2015 Aug 5.

location_facility: Service d'hématologie séniors - Hôpital St Louis / Université Paris 7

location_facility: Josep Carreras Leukaemia Research Institute (IJC)


type: EudraCT Number

title: A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study in Parkinson's Disease Patients With Moderate to Severe Dyskinesia to Assess the Efficacy and Safety/Tolerability of Two Dose Combinations of JM-010

acronym: ASTORIA

summary: The current study will explore the efficacy, safety and tolerability of 2 dose combinations of JM-010 to determine the optimal doses of each component to be studied in confirmatory clinical trials.

ReferenceType: derived

ReferenceCitation: McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS - DYSKINESIA. J Parkinsons Dis. 2019;9(3):449-465. doi: 10.3233/JPD-199002. Review.

name: Parkinson’s advocate, Innovation Fixer Ltd, Oxford, UK

family: McFarthing

full_name: Kevin McFarthing

given: Kevin

name: Parkinson’s Disease and Movement Disorders Center, Northwestern University Feinberg School of Medicine, Chicago, IL, USA

family: Prakash

full_name: Neha Prakash

given: Neha

family: Simuni

full_name: Tanya Simuni

given: Tanya

genre: journal-article

genre_raw: journal-article

journal_name: Journal of Parkinson's Disease

oa_colors: green other

oa_host_type: publisher;repository

evidence: open (via page says Open Access)

host_type: publisher

license: implied-oa

license_normalized: implied-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 pmcid lookup)

license_normalized: no license

publisher: IOS Press

publisher_dissemination: IOS Press

publisher_group: IOS Press

publisher_normalized: IOS Press

title: CLINICAL TRIAL HIGHLIGHTS – DYSKINESIA

lead_sponsor: Contera Pharma

location_country: Italy Germany France Spain

location_facility:
Contera Investigational site_DE
Contera Investigational site_ES
Contera Investigational site_IT
Contera Investigational site_FR


title: A Multicenter, Non-controlled and Open-label Trial Following Subjects Implanted Bilaterally With the Toric Extended-depth-of-focus Mini WELL Toric Ready Intraocular Lens for 6 Months After the Second Eye Implant

acronym: FUSION

summary:
This is a multicenter, non-comparative trial following participants implanted bilaterally with the Mini WELL Toric Ready intraocular lens (IOL) for 6 months after the second eye implant.

The objective of the study is to evaluate the clinical performance of the Mini WELL Toric Ready intraocular lens (IOL), in particular, visual performance and rotational stability at 6 months following the second eye implantation in participants suffering from bilateral cataract with corneal astigmatism.

There are 2 hypotheses:

Rotational Stability: the rotation of the Mini WELL Toric Ready IOL compared between Visit 0a (the day of surgery) and Visit 4 (120 180 days post-operative follow-up) shall be less than ±10° in 90% of the cases.
Severe Visual Distortions: the rate of severe visual distortions for the Mini WELL Toric Ready IOL reported at 6 months postoperative will be less than 10 percents.

lead_sponsor: SIFI SpA

location_facility: Service ophtalmologie Segment Antérieur- Hopital Pellegrin - CHU de Bordeaux

location_facility: Hospital Universitario Quirónsalud Madrid

location_facility: OFTALVIST CIO JEREZ Clínica Jerez (Cadiz)

location_facility: Universitäts-Augenklinik Heidelberg International Vision Correction Research Centre (IVCRC)

intervention_type: Device


intervention_type: Biological

status: Enrolling by invitation

lead_sponsor: BioMarin Pharmaceutical

location_country:
South Africa
United Kingdom
France

location_facility: University Hospital Southampton NHS Foundation Trust

location_facility: Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center

location_facility: Nantes University Hospital Center - Hotel Dieu Hospital

source: euctr

title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5

summary: This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.

status_simplified: Unknown


title: A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

acronym: CareCN

summary:
This is a Phase 1/2, multinational, open-label, escalating-dose study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):

a follow-up of approximately 12 months (48 weeks)
a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.

primary_purpose: Other

lead_sponsor: Genethon

location_country: Italy France Netherlands

location_facility: Azienda Ospedaliera Universitaria Federico II

intervention_type: Genetic


ReferenceCitation: Risitano AM, Röth A, Soret J, Frieri C, de Fontbrune FS, Marano L, Alashkar F, Benajiba L, Marotta S, Rozenberg I, Milojevic J, End P, Nidamarthy PK, Junge G, Peffault de Latour R. Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. Lancet Haematol. 2021 May;8(5):e344-e354. doi: 10.1016/S2352-3026(21)00028-4. Epub 2021 Mar 23.

family: Risitano

full_name: Antonio M Risitano

given: Antonio M

family: Röth

full_name: Alexander Röth

given: Alexander

family: Soret

full_name: Juliette Soret

given: Juliette

family: Frieri

full_name: Camilla Frieri

given: Camilla

family: de Fontbrune

full_name: Flore Sicre de Fontbrune

given: Flore Sicre

family: Marano

full_name: Luana Marano

given: Luana

family: Alashkar

full_name: Ferras Alashkar

given: Ferras

family: Benajiba

full_name: Lina Benajiba

given: Lina

family: Marotta

full_name: Serena Marotta

given: Serena

family: Rozenberg

full_name: Izabela Rozenberg

given: Izabela

family: Milojevic

full_name: Julie Milojevic

given: Julie

family: End

full_name: Peter End

given: Peter

family: Nidamarthy

full_name: Prasanna K Nidamarthy

given: Prasanna K

family: Junge

full_name: Guido Junge

given: Guido

family: Peffault de Latour

full_name: Régis Peffault de Latour

given: Régis

journal_name: The Lancet Haematology

publisher: Elsevier BV

publisher_dissemination: Elsevier

publisher_group: Elsevier

publisher_normalized: Elsevier

design_allocation: Non-Randomized

status: Active, not recruiting

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

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

lead_sponsor: Novartis Pharmaceuticals

enrollment_type: Actual

location_country: Italy Germany France

ipd_sharing: Yes

title: An Open Label, Single Arm, Multiple Dose Study to Assess Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of LNP023 When Administered in Addition to Standard of Care (SoC) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis

summary: The main purpose of this study is to evaluate the efficacy of LNP023 in patients with PNH, showing signs of active hemolysis despite treatment with SoC (defined as an antibody with anti C5 activity).


status: Completed

lead_sponsor: Inventiva Pharma

location_country:
Germany
United Kingdom
France
Portugal

acronym: iMProveS

location_facility:
Villa Metabolica
Hôpital Femme-Mère-Enfant
Royal Free Hospital
Centro Hospitalar S. João

study_completion_date_type: Actual

title: A Phase IIa Study to Investigate Safety, Pharmacokinetics, and Efficacy of Odiparcil in Patients 16 Years and Above With Mucopolysaccharidosis (MPS) Type VI

summary:
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs). Medical need for treatment of MPS is still very high due to the poor penetration of the recombinant enzymes into the blood brain barrier as well as the ocular barriers and into tissues that are poorly vascularized, such as cartilages and bones. Odiparcil is an orally active compound that allows the synthesis of soluble glycosaminoglycans (GAGs), mainly chondroitin sulfate (CS) and dermatane sulfate (DS). The neosynthesized solubles GAGs are then excreted in urine. By diverting endogenous GAG synthesis to the synthesis of soluble odiparcil linked GAGs, odiparcil should decrease the intracellular pool of GAGs and consequently decrease the lysosomal GAG accumulation.

The primary objective of the study is to assess the safety and efficacy of two doses of odiparcil in MPS VI patients and to provide evidence to enable the selection of the relevant dose of odiparcil for phase III study. The secondary objective of this study is to characterize the dose response, PK and PD of odiparcil.

status_simplified: Completed


title: Procalcitonin REveals Good Recovery After Acute Diverticulitis: the PREGRAD Study

acronym: PREGRAD

summary:
Diverticular disease is a common disease in developed countries, affecting 2.5 million individuals in the United States (US). Prevalence of diverticula increases with age and goes up to 50 to 66% in patients older than age 80 years. Approximately 10 to 25% of patients with diverticulosis will develop diverticulitis. Acute diverticulitis (AD) accounts for 312,000 admissions and 1.5 million days of inpatient care in the US, where its annual treatment costs exceed 2.6 billion dollars. With the ageing of global population these numbers are expected to rise.

Procalcitonin (PCT) is a biomarker widely used to monitor bacterial infections and guide antibiotic therapy in Intensive Care Units and has been shown to be useful in different surgical fields such as acute appendicitis. Recently, has been demonstrated that PCT and CPR have good predictive value of anastomotic leak (AL) after colorectal surgery.

A multicentric study has been designed to test if PCT, CRP and WBC values might be able to predict the outcomes of patients admitted in emergency setting for acute diverticulitis. In particular if they might distinguish between patients needing only conservative (nothing per os, iv fluids and antibiotics) or interventional therapy such as radiological drain or even surgery, in the aim to optimize and individualize each patients therapy and speed patients discharge.

status: Unknown status

lead_sponsor: University of Roma La Sapienza

location_country: Italy France

location_facility: General and Emergency Surgery, Niguarda Hospital

location_facility: Department of Digestive Surgery, University Hospital

location_facility: Department of Surgical and Medical Sciences and Translational Medicine, General Surgery and Emergency Surgery Units, Sant'Andrea Hospital, 'Sapienza' University of Rome

location_facility: Department of Surgery, General Surgery Unit, Azienda Ospedaliero- Universitaria 'Ospedali Riuniti di Trieste'

intervention_type: Procedure

lead_sponsor_type: academique


title: Investigation of the Effect of Daily Consumption of Bioactive Enriched Foods (BEF) on Biochemical and Anthropometric Markers of Metabolic Syndrome (MS)

summary:
This is a multi-centre, randomised, double-blind, placebo-controlled, parallel-arm dietary intervention study. In total, 800 men and women at risk for Metabolic Syndrome (MS) will be recruited. Subjects will be eligible to the study if they present with two to four of the MS diagnostic criteria, at least one of them being:

fasting triglycerides ≥150 mg/dL but ≤400 mg/dL OR
HDL-cholesterol ≤50 mg/mL in women, ≤ 40mg/mL in men (with fasting triglycerides ≥110 mg/dL).

Each of the four recruiting centres will recruit 200 volunteers. Participants will be randomly assigned to one of four groups to receive either:

Dairy BEF + egg placebo + bakery placebo
Egg BEF + dairy placebo + bakery placebo
Bakery BEF + dairy placebo + egg placebo
Dairy, egg and bakery placebo

Participants will be required to consume all three of the allocated products each day for 12 weeks.

Eligible volunteers will be included and randomly allocated to one of the four groups. At baseline, 6 weeks and 12 weeks after inclusion, each participant will visit the recruiting centre for clinical and biochemical investigations. At 3 weeks and 9 weeks participants will complete questionnaires relating to their satisfaction with the food products, compliance to consumption of the study food products, and any gastrointestinal side effects or health-related adverse events that have occurred in the previous 3 weeks.

At each recruiting centre 40 participants will be required to take part in additional activities, these are: stool sample collection, adipose tissue aspiration, body composition analysis by dual energy x-ray absorptiometry (DEXA) and assessment of physical activity.

status: Terminated

primary_purpose: Prevention

ipd_sharing_description: Individual case report forms are available to collect study data into electronic form from all the recruiting centres. A central electronic database has been elaborated to capture, validate and manage the entered data by means of a variety of edit checks (e.g., subjecting the data to range checks, valid value checks, cross-checks, and manual review) that provide feedback to those entering/providing the data.

lead_sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna

location_country:
Italy
Germany
United Kingdom
France

location_facility: University of Bologna, Department of Medical and Surgical Sciences and Azienda Ospedaliero Universitaria di Bologna, Policlinico S.Orsola-Malpighi

location_facility: Centre de Récherche en Nutrition Humaine d'Auvergne

intervention_type: Dietary Supplement


title: Anti-Thrombotic Strategy to Lower All Cardiovascular and Neurologic Ischemic and Hemorrhagic Events After Trans-Aortic Valve Implantation for Aortic Stenosis

acronym: ATLANTIS

summary:
ATLANTIS is a multicenter, phase IIIb, prospective, open-label, randomized trial.

The objective of this study is to demonstrate superiority of a strategy of anticoagulation with apixaban (Anti-Xa Group) as compared to the current standard of care in patients who have undergone a successful TAVI procedure.

The randomization is stratified according to the presence or not of a mandatory indication for anticoagulation for a reason other than the TAVI procedure (e.g. atrial fibrillation or DVT/PE).

ReferenceCitation: Collet JP, Berti S, Cequier A, Van Belle E, Lefevre T, Leprince P, Neumann FJ, Vicaut E, Montalescot G. Oral anti-Xa anticoagulation after trans-aortic valve implantation for aortic stenosis: The randomized ATLANTIS trial. Am Heart J. 2018 Jun;200:44-50. doi: 10.1016/j.ahj.2018.03.008. Epub 2018 Mar 10.

family: Collet

full_name: Jean-Philippe Collet

given: Jean-Philippe

family: Berti

full_name: Sergio Berti

given: Sergio

family: Cequier

full_name: Angel Cequier

given: Angel

family: Van Belle

full_name: Eric Van Belle

given: Eric

family: Lefevre

full_name: Thierry Lefevre

given: Thierry

family: Leprince

full_name: Pascal Leprince

given: Pascal

family: Neumann

full_name: Franz-Josef Neumann

given: Franz-Josef

family: Vicaut

full_name: Eric Vicaut

family: Montalescot

full_name: Gilles Montalescot

given: Gilles

journal_name: American Heart Journal

ReferenceCitation: Piayda K, Zeus T, Sievert H, Kelm M, Polzin A. Subclinical leaflet thrombosis. Lancet. 2018 Mar 10;391(10124):937-938. doi: 10.1016/S0140-6736(18)30534-8.

family: Piayda

full_name: Kerstin Piayda

given: Kerstin

family: Zeus

full_name: Tobias Zeus

given: Tobias

family: Sievert

full_name: Horst Sievert

given: Horst

family: Kelm

full_name: Malte Kelm

given: Malte

family: Polzin

full_name: Amin Polzin

given: Amin

journal_name: The Lancet

oa_host_type: publisher

evidence: open (via free pdf)

title: Subclinical leaflet thrombosis

lead_sponsor: Assistance Publique - Hôpitaux de Paris

location_facility: Division of Cardiology and Angiology II, University Heart Center Freiburg

location_facility: Hospitalet de Llobregat-Hospital Universitari de Bellvitge

location_facility: Unità Operativa di Cardiologia Fondazione Gabriele Monasterio C.N.R.

location_facility: ACTION Study Group, Institut de Cardiologie, Centre Hospitalier Universitaire Pitié Salpêtrière (APHP), UPMC


title: Advanced Bradycardia Device Feature Utilization and Clinical Outcomes II

acronym: BRADYCARE II

summary: The purpose of this study is to characterize the utilization of diagnostics capabilities in St. Jude Medical pacemakers to manage patients with a standard bradycardia pacing indication.

lead_sponsor: Abbott Medical Devices

location_country: Italy Germany France Japan


title: A Randomized Controlled Multicenter Trial of a Five Day Course of Oral Colistin and Neomycin Followed by Restoration of the Gut Microbiota Using Fecal Transplantation to Eradicate Intestinal Carriage of Extended Spectrum Beta-lactamase or Carbapenemase-producing Enterobacteriaceae in High-risk Patients

acronym: R-GNOSIS WP3

summary: This investigator initiated,international, multicenter open-label, randomized controlled trial aims to assess whether a 5 day course of oral nonabsorbable antibiotics (colistin sulfate 2 million IU per os 4x/day and neomycin sulfate 500 mg (salt) per os 4x/day ) followed by fecal microbiota transplantation (administered either via nasogastric administration or via capsules) is effective at eradicating intestinal carriage of beta-lactamase producing Enterobacteriaceae (ESBL-E) and carbapenemase producing Enterobacteriaceae (CPE). compared to no intervention (current standard of care) in adult non-immunosuppressed patients .

ReferenceCitation: Huttner B, Haustein T, Uçkay I, Renzi G, Stewardson A, Schaerrer D, Agostinho A, Andremont A, Schrenzel J, Pittet D, Harbarth S. Decolonization of intestinal carriage of extended-spectrum β-lactamase-producing Enterobacteriaceae with oral colistin and neomycin: a randomized, double-blind, placebo-controlled trial. J Antimicrob Chemother. 2013 Oct;68(10):2375-82. doi: 10.1093/jac/dkt174. Epub 2013 May 29.

ReferenceCitation: Youngster I, Russell GH, Pindar C, Ziv-Baran T, Sauk J, Hohmann EL. Oral, capsulized, frozen fecal microbiota transplantation for relapsing Clostridium difficile infection. JAMA. 2014 Nov 5;312(17):1772-8. doi: 10.1001/jama.2014.13875. Erratum in: JAMA. 2015 Feb 17;313(7):729.

ReferenceCitation: de Lastours V, Poirel L, Huttner B, Harbarth S, Denamur E, Nordmann P. Emergence of colistin-resistant Gram-negative Enterobacterales in the gut of patients receiving oral colistin and neomycin decontamination. J Infect. 2020 May;80(5):578-606. doi: 10.1016/j.jinf.2020.01.003. Epub 2020 Jan 15.

family: de Lastours

full_name: Victoire de Lastours

given: Victoire

family: Poirel

full_name: Laurent Poirel

given: Laurent

family: Huttner

full_name: Benedikt Huttner

given: Benedikt

family: Harbarth

full_name: Stephan Harbarth

given: Stephan

family: Denamur

full_name: Erick Denamur

given: Erick

family: Nordmann

full_name: Patrice Nordmann

given: Patrice

journal_name: Journal of Infection

oa_host_type: repository

repository_institution: University of Geneva - Archive ouverte UNIGE

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

repository_institution: Universität Bern - RERO DOC

ReferenceCitation: Huttner BD, de Lastours V, Wassenberg M, Maharshak N, Mauris A, Galperine T, Zanichelli V, Kapel N, Bellanger A, Olearo F, Duval X, Armand-Lefevre L, Carmeli Y, Bonten M, Fantin B, Harbarth S; R-Gnosis WP3 study group. A 5-day course of oral antibiotics followed by faecal transplantation to eradicate carriage of multidrug-resistant Enterobacteriaceae: a randomized clinical trial. Clin Microbiol Infect. 2019 Jul;25(7):830-838. doi: 10.1016/j.cmi.2018.12.009. Epub 2019 Jan 4.

full_name: B.D. Huttner

full_name: V. de Lastours

given: V.

family: Wassenberg

full_name: M. Wassenberg

given: M.

family: Maharshak

full_name: N. Maharshak

given: N.

family: Mauris

full_name: A. Mauris

given: A.

family: Galperine

full_name: T. Galperine

given: T.

family: Zanichelli

full_name: V. Zanichelli

family: Kapel

full_name: N. Kapel

family: Bellanger

full_name: A. Bellanger

family: Olearo

full_name: F. Olearo

given: F.

family: Duval

full_name: X. Duval

given: X.

family: Armand-Lefevre

full_name: L. Armand-Lefevre

given: L.

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

repository_institution: Le Centre pour la Communication Scientifique Directe - HAL - Université de Lille

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

location_facility: Assistance Publique-Hôpitaux de Paris, Hôpital Beaujon


title: A Non-interventional Prospective Study on Allogeneic Stem Cell Transplantation in Patients With AL Amyloidosis

summary:
Light-chain (AL-) amyloidosis is a very rare monoclonal plasma cell disorder with poor prognosis. Rarity of disease has precluded performance of randomized controlled trials comparing various possible treatment modalities. In general, treatment of AL amyloidosis has been adapted from myeloma (MM) therapy. There is large experience with allo SCT in MM. Based on small series of patients and case reports allogeneic transplant has emerged as potentially effective. However, more formal proof of concept of using allogeneic hematopoietic transplantation for treatment of AL Amyloidosis is lacking.

Therefore, given the limitations of conventionally collected registry data (dubious follow-up information and extreme heterogeneity), we developed the: "EBMT non-interventional prospective study on allogeneic transplantation in AL Amyloidosis" which means that transplant centers that already do perform allogeneic transplants for AL Amyloidosis will be encouraged to register their patients with AL Amyloidosis very timely with the EBMT, followed by mandatory submission of EBMT MedB and follow-up forms. The diagnosis of AL Amyloidosis would be based on uniform criteria. All EBMT centres performing allogeneic transplants for Amyloidosis will be invited to participate in this study and centres will be asked to report all AL Amyloidosis cases referred for transplantation using a simple registration form and then to submit Amyloidosis MED B forms for each transplanted patient and follow-up forms as necessary. In conclusion, it should be possible to largely improve the usual quality of registry-based data and to generate scientifically sound knowledge on HSCT in an orphan disease such as AL Amyloidosis.

ReferenceCitation: Schönland et al. EBMT retrospective data, Blood 2005.

ReferenceCitation: Schönland et al. DLI data, Haematologica, 2008.

lead_sponsor: European Society for Blood and Marrow Transplantation

location_country: Germany France Switzerland

location_facility:
University Hospital
University of Heidelberg
CHRU Limoges
University Hospital Eppendorf


lead_sponsor: Laboratoire français de Fractionnement et de Biotechnologies

location_country: Turkey France Morocco Lebanon

location_facility: Faculty Ihsan Dogramaci Children's Hospital

title: Clinical Pharmacology, Efficacy and Safety Study of FGTW in Paediatric Patients With Severe Congenital Fibrinogen Deficiency

summary: The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.


type: Other Identifier

title: A Phase 1 Study of Combination Therapy With SAR405838 and Pimasertib in Patients With Advanced Cancer

summary:
Primary Objectives:

To determine the recommended Phase 2 dose of SAR405838 / pimasertib combination therapy in patients with solid tumors.

To assess the anti-tumor activities of SAR405838 / pimasertib in patients with solid tumors.

Secondary Objectives:

To characterize the pharmacokinetic profile of SAR405838 and pimasertib.

To evaluate the pharmacodynamic effect of the SAR405838 and pimasertib.

To characterize genetic status in tumor tissue and circulating tumor DNA.

ReferenceCitation: de Weger VA, de Jonge M, Langenberg MHG, Schellens JHM, Lolkema M, Varga A, Demers B, Thomas K, Hsu K, Tuffal G, Goodstal S, Macé S, Deutsch E. A phase I study of the HDM2 antagonist SAR405838 combined with the MEK inhibitor pimasertib in patients with advanced solid tumours. Br J Cancer. 2019 Feb;120(3):286-293. doi: 10.1038/s41416-018-0355-8. Epub 2018 Dec 26.

family: de Weger

full_name: Vincent A. de Weger

given: Vincent A.

family: de Jonge

full_name: Maja de Jonge

given: Maja

family: Langenberg

full_name: Marlies H. G. Langenberg

given: Marlies H. G.

family: Schellens

full_name: Jan H. M. Schellens

given: Jan H. M.

family: Lolkema

full_name: Martijn Lolkema

given: Martijn

family: Varga

full_name: Andrea Varga

given: Andrea

family: Demers

full_name: Brigitte Demers

given: Brigitte

family: Thomas

full_name: Koruth Thomas

given: Koruth

family: Hsu

full_name: Karl Hsu

given: Karl

family: Tuffal

full_name: Gilles Tuffal

given: Gilles

family: Goodstal

full_name: Samantha Goodstal

given: Samantha

family: Macé

full_name: Sandrine Macé

given: Sandrine

family: Deutsch

full_name: Eric Deutsch

given: Eric

journal_name: British Journal of Cancer

oa_colors: green hybrid

evidence: open (via page says license)

license: cc-by

license_normalized: cc-by

version: submittedVersion

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

publisher: Springer Science and Business Media LLC

publisher_dissemination: Springer-Nature

publisher_group: Springer-Nature

publisher_normalized: Springer

lead_sponsor: Sanofi

location_country: France Netherlands

location_facility:
Investigational Site Number 528002
Investigational Site Number 250001
Investigational Site Number 528003
Investigational Site Number 528001


title: Lower Urinary Tract Symptoms in Patients Suffering From Systemic Sclerosis: a Multi-centric Longitudinal Study

summary:
Urinary symptoms must be frequent in Scleroderma. In one hand, mobility limitation by joint stiffness and skin sclerosis, forced diuresis due to heart involvement (cardiomyopathy or pulmonary hypertension), diuretics use and corticoid-induced hyperglycaemia, as well as narcotic medication use, puts patients at higher risk of secondary bladder filling and voiding dysfunction. In another hand, few case report and small sample observational studies have identified a specific sclerosis of the urinary tract. Those two mechanisms must be more frequent in the diffuse cutaneous form of scleroderma (dcSSc) compare to the limited one (lcSSc). But prevalence or incidence is unknown.

Urinary symptoms are seldom reported by those suffering from them and are rarely part of a systemic evaluation. In a threatening disease, urinary symptoms assessment might seem to be of no priority. But LUTS have a real impact on many aspect of everyday living. Furthermore urinary tract involvement might predispose to urinary tract infection due to flow limitation and stagnation. Since it is an inner fibrosis it might be associated with a more aggressive form of disease conferring a greater loss of physical function, higher risk for hospital admission and death.

Thus, identifying urinary symptoms would permit to address specific rehabilitation or medication therapy, in order to minimize the consequences of the bothersome symptoms and identify those subjects at higher risk of urinary infection, aggressive disease/loss of function or death.

This study will also give basement to build an interventional study directed toward LUTS treatment in this population.

In this prospective cohort we would like to:

Compare the prevalence of lower urinary tract symptoms (LUTS) in diffuse and limited forms of systemic sclerosis.
Determine the prevalence (at inclusion) and incidence (in a two years period) of LUTS among patients suffering from systemic sclerosis.
Evaluate the impact of LUTS symptoms on Quality of life.
Compare the discrimination ability of Cochin-hand score and HAQ score to predict incontinence in this population.
Evaluate the association between LUTS symptoms, hospital admission rate, urinary tract infection, mortality and loss of autonomy.

ReferenceCitation: John G, Avouac J, Piantoni S, Polito P, Fredi M, Cozzi F, Airò P, Truchetet ME, Franceschini F, Allanore Y, Chizzolini C. Prevalence and Disease-Specific Risk Factors for Lower Urinary Tract Symptoms in Systemic Sclerosis: An International Multicenter Study. Arthritis Care Res (Hoboken). 2018 Aug;70(8):1218-1227. doi: 10.1002/acr.23454. Epub 2018 Jun 19.

name: Hôpital Neuchâtelois, La Chaux-de-Fonds, Switzerland, and Geneva University Hospitals; Geneva Switzerland

family: John

full_name: Gregor John

given: Gregor

family: Avouac

full_name: Jérôme Avouac

given: Jérôme

family: Piantoni

full_name: Silvia Piantoni

given: Silvia

name: University Hospital of Padua; Padua Italy

family: Polito

full_name: Pamela Polito

given: Pamela

family: Fredi

full_name: Micaela Fredi

given: Micaela

family: Cozzi

full_name: Franco Cozzi

given: Franco

family: Airò

full_name: Paolo Airò

given: Paolo

name: CNRS-UMR 5164 Immuno Concept, Bordeaux University and Bordeaux Hospital; Bordeaux France

family: Truchetet

full_name: Marie-Elise Truchetet

given: Marie-Elise

family: Franceschini

full_name: Franco Franceschini

family: Allanore

full_name: Yannick Allanore

given: Yannick

name: University Hospital and School of Medicine, University of Geneva; Geneva Switzerland

family: Chizzolini

full_name: Carlo Chizzolini

given: Carlo

journal_name: Arthritis Care & Research

publisher: Wiley

publisher_dissemination: Wiley

publisher_group: Wiley

publisher_normalized: Wiley

ReferenceCitation: John G, Allanore Y, Polito P, Piantoni S, Fredi M, Avouac J, Franceschini F, Truchetet ME, Cozzi F, Airo P, Chizzolini C. The limited cutaneous form of systemic sclerosis is associated with urinary incontinence: an international multicentre study. Rheumatology (Oxford). 2017 Nov 1;56(11):1874-1883. doi: 10.1093/rheumatology/kex230.

family: Airo

full_name: Paolo Airo

location_facility: Azienda Ospedaliera Spedali Civili di Brescia


title: A Phase 1b, Multi-center, Open-label, Dose Escalation Study of GSK2256098 (FAK Inhibitor) in Combination With Trametinib (MEK Inhibitor) in Subjects With Advanced Solid Tumors

summary: The purpose of this study is to assess the safety of combination treatment of GSK2256098 and trametinib in mesothelioma subjects and subjects with other selected tumor types. Also, the study will identify a maximum tolerated combination dose of GSK2256098 and trametinib. This study is a Phase I, open-label, dose-escalation study to determine maximal tolerated dose (MTD) and the recommended Phase 2 dose (RP2D) and regimens for oral MEK inhibitor trametinib (once daily [OD]dosing) and the oral FAK inhibitor GSK2256098 (twice daily [BID] dosing). The synergy of the combination was observed over a wide range of concentrations and results in several-fold reduction in compound concentration to achieve equivalent biological responses compared to either single agent. The dose and schedule of dosing may be modified based on emerging safety, pharmacokinetic (PK), and pharmacodynamic (PD) data. The study will be conducted in two parts; Part 1 Dose Escalation to determine the MTD and RP2D and Part 2 Expansion Cohort to further evaluate the safety and tolerability of trametinib and GSK2256098 at the RP2D and determine clinical activity. Additionally, in Part 1 Dose Escalation, additional subjects with malignant pleural mesothelioma (MPM) will be recruited at doses that are considered tolerable in order to assess PD in MPM subjects at each dose (the Pharmacodynamic Cohort). The Expansion Cohort will be limited to subjects with MPM who have progressed or are intolerant to first-line therapy.

ReferenceCitation: Mak G, Soria JC, Blagden SP, Plummer R, Fleming RA, Nebot N, Zhang J, Mazumdar J, Rogan D, Gazzah A, Rizzuto I, Greystoke A, Yan L, Tolson J, Auger KR, Arkenau HT. A phase Ib dose-finding, pharmacokinetic study of the focal adhesion kinase inhibitor GSK2256098 and trametinib in patients with advanced solid tumours. Br J Cancer. 2019 May;120(10):975-981. doi: 10.1038/s41416-019-0452-3. Epub 2019 Apr 17.

ipd_sharing_description: IPD for this study will be made available via the Clinical Study Data Request site.

lead_sponsor: GlaxoSmithKline

location_country:
United Kingdom
France


title: Xarelto (Rivaroxaban) Risk Minimisation Plan Evaluation: Patient and Physician Knowledge of Key Safety Messages

summary: This cross-sectional epidemiologic study measured physician and patient awareness and understanding of the key messages in the Xarelto prescriber guide and Xarelto patient alert card.

time_perspective: Cross-Sectional

lead_sponsor: Bayer

location_country:
Germany
United Kingdom
France
Spain


title: Retrospective Collection of Effectiveness and Safety Data From Patients Treated With Liraglutide or DPP-4 Inhibitor in Primary Care in Europe

summary: This study is conducted in Europe. The aim of this study is to demonstrate the clinical effectiveness and safety of liraglutide and dipeptidyl peptidase-4 (DPP-4) inhibitor therapy in routine primary care in Europe.

time_perspective: Retrospective

lead_sponsor: Novo Nordisk A/S


title: A Non-interventional Study to Evaluate the Use of Pliaglis in Daily Practice

summary: A multe-center, non-interventional study in Europe to evaluate Pliaglis in daily practice

ReferenceCitation: Jain R, Arias GM, Naranjo P, Murison M, López Estebaranz JL, Fratila A, Prager W, Barona CG, Weidmann M, Dahan S, Cartier H, Sattler G, Podda M. The Use of a Self-Occluding Topical Anasthetic in Daily Practice: A Non-Interventional Study. J Drugs Dermatol. 2018 Apr 1;17(4):413-418.

lead_sponsor: Q-Med AB

location_facility:
Clínica Dermatológica Dr. Guillén
Klinikum Darmstadt Hautklinik
Centre Médical Saint-Jean
Swansea Laser Clinic


title: A Phase 1, Multi-center, Non-randomized, Open Label, Dose Escalation Design Study of Sorafenib (BAY43-9006) in Combination With Eribulin in Subjects With Advanced, Metastatic or Refractory Solid Tumors

summary:
This Phase 1 study will be conducted in an open-label, non-randomized, dose-escalation design in subjects with advanced, metastatic or refractory solid malignancy who are not candidates for standard therapy. The study drugs are sorafenib and eribulin mesylate.

Up to 24 subjects with solid tumors will participate in the dose escalation part of the study, and once the maximum tolerated dose is defined, up to 30 subjects with advanced, metastatic or refractory solid tumors will participate in the expansion phase of the study.

Eribulin (mesylate) will be administered intravenously at a fixed dose of 1.4 mg/m2 on Days 1 and 8 of 21-day Cycles.

The starting sorafenib dose (Dose Level 1) is 200 mg twice daily. Sorafenib is given orally, continuously on days 11 to 21 of Cycle 1, and from Day 1 to Day 21 of all subsequent cycles. If 200 mg sorafenib twice daily is tolerated with eribulin, the sorafenib dose will be escalated sequentially to 200 mg morning dose and 400 mg evening dose (Dose Level 2) in a new cohort. If Dose Level 2 is tolerated, a second dose escalation to 400 mg twice daily (Dose Level 3) will be studied in a new cohort. If the starting dose of sorafenib is not tolerated with eribulin, the sorafenib dose will be de-escalated to 200 mg once daily in a new cohort. Subjects will need to receive two cycles of eribulin plus sorafenib therapy and safety data for the first and second cycle needs to be available before the start of the next cohort.

location_country: Germany France


summary: This study is a multicenter, prospective, observational study to evaluate the long-term safety of OZURDEX® in patients with macular oedema following central retinal vein occlusion (CRVO) or branch retinal vein occlusion (BRVO) or patients with non-infectious posterior segment uveitis in real-world clinical practice.

lead_sponsor: Allergan


title: An Open-label, Non-randomized, Dose Escalation, Safety and Pharmacokinetic Phase I Study of Ombrabulin (AVE8062) in Combination With Bevacizumab Administered by Intravenous Infusion Every 3 Weeks in Patients With Advanced Solid Tumors.

summary:
Primary Objective:

- To determine the maximum administered dose (MAD) and the maximum tolerated dose (MTD) of ombrabulin in combination with best tolerated dose of bevacizumab based on the incidence of related Dose Limiting Toxicities (DLTs).

Secondary Objectives:

To assess the overall safety profile of the combination
To characterize the pharmacokinetic (PK) profile of both ombrabulin and bevacizumab when given in combination
To evaluate preliminary evidence of anti-tumor activity
To assess the pharmacodynamic effect using (Dynamic Contrast Enhanced Ultra-Sound) DCE-US, measuring biomarkers

location_country:
Italy
United Kingdom
France

location_facility:
Investigational Site Number 250001
Investigational Site Number 380002
Investigational Site Number 380001
Investigational Site Number 826001


title: A Multiple-Dose Study To Evaluate Safety, Tolerability, Pharmacokinetics and Antiviral Activity of Ritonavir-Boosted RO5190591 in Combination With Peginterferon Alfa-2a Plus Ribavirin in Patients With Chronic Hepatitis C Genotype 1

summary: This study will evaluate the efficacy, safety and tolerability of danoprevir (RO5190591) plus ritonavir as compared to danoprevir alone or placebo plus ritonavir in patients with chronic hepatitis C genotype 1 receiving Pegasys (peginterferon alfa-2a) and ribavirin. Patients in cohorts will be randomized to receive either oral doses of danoprevir, or danoprevir plus ritonavir, or placebo plus ritonavir. All patients will receive Pegasys (180mcg sc once weekly) plus ribavirin (1000-1200mg/day po), with the option to continue this treatment after completion of study drug treatment. Anticipated time on study treatment is up to 12 weeks.

ReferenceCitation: Gane EJ, Rouzier R, Wiercinska-Drapalo A, Larrey DG, Morcos PN, Brennan BJ, Le Pogam S, Nájera I, Petric R, Tran JQ, Kulkarni R, Zhang Y, Smith P, Yetzer ES, Shulman NS. Efficacy and safety of danoprevir-ritonavir plus peginterferon alfa-2a-ribavirin in hepatitis C virus genotype 1 prior null responders. Antimicrob Agents Chemother. 2014;58(2):1136-45. doi: 10.1128/AAC.01515-13. Epub 2013 Dec 2.

family: Gane

full_name: Edward J. Gane

given: Edward J.

family: Rouzier

full_name: Régine Rouzier

given: Régine

family: Wiercinska-Drapalo

full_name: Alicja Wiercinska-Drapalo

given: Alicja

family: Larrey

full_name: Dominique G. Larrey

given: Dominique G.

family: Morcos

full_name: Peter N. Morcos

given: Peter N.

family: Brennan

full_name: Barbara J. Brennan

given: Barbara J.

family: Le Pogam

full_name: Sophie Le Pogam

given: Sophie

family: Nájera

full_name: Isabel Nájera

given: Isabel

family: Petric

full_name: Rosemary Petric

given: Rosemary

family: Tran

full_name: Jonathan Q. Tran

given: Jonathan Q.

family: Kulkarni

full_name: Rohit Kulkarni

given: Rohit

family: Zhang

full_name: Ying Zhang

given: Ying

family: Smith

full_name: Patrick Smith

given: Patrick

family: Yetzer

full_name: Ellen S. Yetzer

given: Ellen S.

family: Shulman

full_name: Nancy S. Shulman

given: Nancy S.

journal_name: Antimicrobial Agents and Chemotherapy

publisher: American Society for Microbiology

publisher_dissemination: American Society for Microbiology

publisher_group: American Society for Microbiology

publisher_normalized: American Society for Microbiology

title: Efficacy and Safety of Danoprevir-Ritonavir plus Peginterferon Alfa-2a–Ribavirin in Hepatitis C Virus Genotype 1 Prior Null Responders

ReferenceCitation: Morcos PN, Chang L, Kulkarni R, Giraudon M, Shulman N, Brennan BJ, Smith PF, Tran JQ. A randomised study of the effect of danoprevir/ritonavir or ritonavir on substrates of cytochrome P450 (CYP) 3A and 2C9 in chronic hepatitis C patients using a drug cocktail. Eur J Clin Pharmacol. 2013 Nov;69(11):1939-49. doi: 10.1007/s00228-013-1556-y. Epub 2013 Jul 20.

family: Chang

full_name: Linda Chang

journal_name: European Journal of Clinical Pharmacology

ReferenceCitation: Gane EJ, Rouzier R, Stedman C, Wiercinska-Drapalo A, Horban A, Chang L, Zhang Y, Sampeur P, Nájera I, Smith P, Shulman NS, Tran JQ. Antiviral activity, safety, and pharmacokinetics of danoprevir/ritonavir plus PEG-IFN α-2a/RBV in hepatitis C patients. J Hepatol. 2011 Nov;55(5):972-9. doi: 10.1016/j.jhep.2011.01.046. Epub 2011 Feb 24.


title: A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis

summary: The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

ReferenceCitation: Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.

family: Coelho

full_name: Teresa Coelho

given: Teresa

family: Adams

full_name: David Adams

given: David

family: Silva

full_name: Ana Silva

given: Ana

family: Lozeron

full_name: Pierre Lozeron

given: Pierre

family: Hawkins

full_name: Philip N. Hawkins

given: Philip N.

family: Mant

full_name: Timothy Mant

given: Timothy

family: Perez

full_name: Javier Perez

given: Javier

family: Chiesa

full_name: Joseph Chiesa

given: Joseph

family: Warrington

full_name: Steve Warrington

given: Steve

family: Tranter

full_name: Elizabeth Tranter

given: Elizabeth

family: Munisamy

full_name: Malathy Munisamy

given: Malathy

family: Falzone

full_name: Rick Falzone

given: Rick

family: Harrop

full_name: Jamie Harrop

given: Jamie

family: Cehelsky

full_name: Jeffrey Cehelsky

given: Jeffrey

family: Bettencourt

full_name: Brian R. Bettencourt

given: Brian R.

family: Geissler

full_name: Mary Geissler

given: Mary

family: Butler

full_name: James S. Butler

given: James S.

family: Sehgal

full_name: Alfica Sehgal

given: Alfica

family: Meyers

full_name: Rachel E. Meyers

given: Rachel E.

family: Chen

full_name: Qingmin Chen

given: Qingmin

family: Borland

full_name: Todd Borland

given: Todd

family: Hutabarat

full_name: Renta M. Hutabarat

given: Renta M.

family: Clausen

full_name: Valerie A. Clausen

given: Valerie A.

title: Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis


title: Prospective, International Multi-centre, Uncontrolled, Post Marketing Surveillance Study to Monitor the Long-term Performance of the DePuy PROXIMA™ Hip in Subjects With Indications Suitable for a Primary Total Hip Arthroplasty

summary: The purpose of this study is to monitor the performance of the DePuy PROXIMA™ hip in the treatment of patients with hip joint disease requiring a total hip replacement. Patients who enter the study will be evaluated at regular intervals following hip surgery using patient, clinical and x-ray assessments.

lead_sponsor: DePuy International

location_country:
Spain
United Kingdom
France
Portugal

location_facility:
Hospital de Santo Antonio
Hospital Belle Isle
Pilgrim Hospital
Hospital Clinic Barcelona


title: A Clinical Study to Evaluate Safety of the ExAblate 2100 UF V2 System in the Treatment of Symptomatic Uterine Fibroids

summary:
The first magnetic resonance-guided focused ultrasound (MRgFUS) treatment of uterine fibroid using the ExAblate 2000 system was performed in 2001. Since then, more than 5000 treatments were done in more than 60 different hospitals around the world. The experience accumulated in this novel treatment was collected by InSightec and implemented into software and hardware updates, clinical tips and guidelines, all aimed to improve the clinical results and their durability, while maintaining a high level of safety.

Based on extensive clinical experience and our internal research and development effort goals toward continuous improvement in ExAblate treatment safety and performance, limited changes have been made to the current ExAblate system. This modified ExAblate system version is designated as the ExAblate 2100 UF V2 system.

The modifications are believed to improve system's friendliness to the user, without introducing new risks or other issues of safety of the device, and should not have any negative impact on the safety or technical efficacy of the treatments for patients.

lead_sponsor: InSightec

location_country:
Israel
United Kingdom
Russian Federation
France

location_facility: Research Centre of Obstetric / Gynaecology & Perinatology


title: A Randomized, Placebo-Controlled, Double-blind, Parallel Group, Multi-centre Phase II Dose-Finding Study of M516102 in the Treatment of Pruritus Associated With Atopic Dermatitis

summary: This study is to investigate the efficacy and safety of M516102 and to evaluate the dose response relationship of M516102 and placebo, in the treatment of pruritus associated with atopic dermatitis.

lead_sponsor: Maruho Co., Ltd.

location_country:
Germany
United Kingdom
France
Poland

location_facility: Samodzielny Publiczny Szpital Kliniczny Nr 1


title: An Open-label Extension to a 52-week, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study in Patients With Alzheimer's Disease to Investigate the Safety and Tolerability of Repeated Subcutaneous Injections of CAD106

summary: This study will evaluate the safety and tolerability of repeated subcutaneous injections of CAD106 in patients with Alzheimer's disease.

ReferenceCitation: Farlow MR, Andreasen N, Riviere ME, Vostiar I, Vitaliti A, Sovago J, Caputo A, Winblad B, Graf A. Long-term treatment with active Aβ immunotherapy with CAD106 in mild Alzheimer's disease. Alzheimers Res Ther. 2015 Apr 27;7(1):23. doi: 10.1186/s13195-015-0108-3. eCollection 2015.

family: Farlow

full_name: Martin R Farlow

given: Martin R

family: Andreasen

full_name: Niels Andreasen

given: Niels

family: Riviere

full_name: Marie-Emmanuelle Riviere

given: Marie-Emmanuelle

family: Vostiar

full_name: Igor Vostiar

given: Igor

family: Vitaliti

full_name: Alessandra Vitaliti

given: Alessandra

family: Sovago

full_name: Judit Sovago

given: Judit

family: Caputo

full_name: Angelika Caputo

given: Angelika

family: Winblad

full_name: Bengt Winblad

given: Bengt

family: Graf

full_name: Ana Graf

given: Ana

currency_apc_doaj: GBP

journal_name: Alzheimer's Research & Therapy

oa_colors: green gold

evidence: oa journal (via doaj)

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

repository_institution: Indiana University - IUScholarWorks

repositories_institution:
Indiana University - IUScholarWorks
PubMed Central - Europe PMC

title: Long-term treatment with active Aβ immunotherapy with CAD106 in mild Alzheimer’s disease

lead_sponsor: Novartis

location_country:
Sweden
United Kingdom
France
Switzerland


title: Acceptability of Long-term Progestin-only Contraception in Europe

summary: The study examines the use of Mirena or Implanon for long-term contraception in women. The duration of therapy use is the key focus of the study. Also, any reasons for discontinuation and the safety profile will be examined. In addition, patients are asked to fill out a short questionnaire about their menstrual bleeding before and during therapy.

location_country:
Slovakia
Ireland
France
United Kingdom


ReferenceType: result

ReferenceCitation: Barnes N, van Noord JA, Brindicci C, Lindemann L, Varoli G, Perpiña M, Guastalla D, Casula D, Patel S, Chanez P; FACTO (Foster® As Complete Treatment Option) Study Group. Stepping-across controlled asthmatic patients to extrafine beclometasone/formoterol combination. Pulm Pharmacol Ther. 2013 Oct;26(5):555-61. doi: 10.1016/j.pupt.2013.01.011. Epub 2013 Mar 22.

family: Barnes

full_name: Neil Barnes

given: Neil

family: van Noord

full_name: Jan Arie van Noord

given: Jan Arie

family: Brindicci

full_name: Caterina Brindicci

given: Caterina

family: Lindemann

full_name: Ludger Lindemann

given: Ludger

family: Varoli

full_name: Guido Varoli

given: Guido

family: Perpiña

full_name: Miguel Perpiña

given: Miguel

family: Guastalla

full_name: Daniele Guastalla

given: Daniele

family: Casula

full_name: Daniela Casula

given: Daniela

family: Patel

full_name: Shishir Patel

given: Shishir

family: Chanez

full_name: Pascal Chanez

given: Pascal

journal_name: Pulmonary Pharmacology & Therapeutics

lead_sponsor: Chiesi Farmaceutici S.p.A.

location_country: Spain Germany France Netherlands

acronym: FACTO

location_facility: Allergologie imUmkreis der Praxis Pneumologie

title: A PHASE 4, MULTINATIONAL, MULTICENTRE, DOUBLE BLIND, DOUBLE DUMMY, RANDOMIZED, PARALLEL GROUP, CONTROLLED CLINICAL STUDY OF FIXED COMBINATION BECLOMETHASONE DIPROPIONATE 100 µg PLUS FORMOTEROL FUMARATE 6 µg pMDI WITH HFA-134A PROPELLANT (CHF1535, FOSTER®) VERSUS FLUTICASONE 250 µg PLUS SALMETEROL 50 µg DPI (SERETIDE® DISKUS®) AS MAINTENANCE TREATMENT IN CONTROLLED ASTHMATIC PATIENTS.

summary: Double blind, multinational, multicentre, randomised, 2 arm parallel group study


title: Two-arm, Randomized, Open-label, Phase IIIb Study Investigating the Safety of a 3 Hour i.p. Infusion of Catumaxomab With and Without Prednisolone Premedication in Patients With Malignant Ascites Due to Epithelial Cancer

acronym: CASIMAS

summary:
This is a randomized phase IIIb study investigating the treatment of malignant ascites due to epithelial cancer (carcinomas) with the trifunctional antibody catumaxomab. In order to make the catumaxomab treatment more convenient for the patient and the hospital praxis the tolerability of 3 hour infusions of catumaxomab with and without premedication of prednisolone is evaluated.

A total of 208 patients with malignant ascites due to epithelial cancer will be allocated to two treatment groups in a 1:1 ratio.

ReferenceCitation: Lordick F, Ott K, Weitz J, Jäger D. The evolving role of catumaxomab in gastric cancer. Expert Opin Biol Ther. 2008 Sep;8(9):1407-15. doi: 10.1517/14712598.8.9.1407 . Review.

ReferenceCitation: Burges A, Wimberger P, Kümper C, Gorbounova V, Sommer H, Schmalfeldt B, Pfisterer J, Lichinitser M, Makhson A, Moiseyenko V, Lahr A, Schulze E, Jäger M, Ströhlein MA, Heiss MM, Gottwald T, Lindhofer H, Kimmig R. Effective relief of malignant ascites in patients with advanced ovarian cancer by a trifunctional anti-EpCAM x anti-CD3 antibody: a phase I/II study. Clin Cancer Res. 2007 Jul 1;13(13):3899-905.

ReferenceCitation: Shen J, Zhu Z. Catumaxomab, a rat/murine hybrid trifunctional bispecific monoclonal antibody for the treatment of cancer. Curr Opin Mol Ther. 2008 Jun;10(3):273-84.

ReferenceCitation: Fossati M, Buzzonetti A, Monego G, Catzola V, Scambia G, Fattorossi A, Battaglia A. Immunological changes in the ascites of cancer patients after intraperitoneal administration of the bispecific antibody catumaxomab (anti-EpCAM×anti-CD3). Gynecol Oncol. 2015 Aug;138(2):343-51. doi: 10.1016/j.ygyno.2015.06.003. Epub 2015 Jun 3.

family: Fossati

full_name: Marco Fossati

given: Marco

family: Buzzonetti

full_name: Alexia Buzzonetti

given: Alexia

family: Monego

full_name: Giovanni Monego

given: Giovanni

family: Catzola

full_name: Valentina Catzola

given: Valentina

family: Scambia

full_name: Giovanni Scambia

family: Fattorossi

full_name: Andrea Fattorossi

given: Andrea

family: Battaglia

full_name: Alessandra Battaglia

journal_name: Gynecologic Oncology

lead_sponsor: Neovii Biotech

location_country: Italy Germany France Spain

location_facility:
Study site
Study Site


title: A Randomised, Double-Blind, Multicentre Study to Compare the Efficacy and Tolerability of Oral Ivermectin to Malathion 0.5% Lotion in the Treatment of Head Lice Infestation

summary: The purpose of this study is to compare 2 single doses of ivermectin as tablets with 2 single applications of malathion 0.5% lotion (Days 1 and 8) in clearing head lice, in patients who have recently used standard head lice treatments without success.

ReferenceCitation: Chosidow O, Giraudeau B, Cottrell J, Izri A, Hofmann R, Mann SG, Burgess I. Oral ivermectin versus malathion lotion for difficult-to-treat head lice. N Engl J Med. 2010 Mar 11;362(10):896-905. doi: 10.1056/NEJMoa0905471. Erratum in: N Engl J Med. 2010 Apr 29;362(17):1647.

family: Chosidow

full_name: Olivier Chosidow

given: Olivier

family: Giraudeau

full_name: Bruno Giraudeau

given: Bruno

family: Cottrell

full_name: Jeremy Cottrell

given: Jeremy

family: Izri

full_name: Arezki Izri

given: Arezki

family: Hofmann

full_name: Robert Hofmann

given: Robert

family: Mann

full_name: Stephen G. Mann

given: Stephen G.

family: Burgess

full_name: Ian Burgess

given: Ian

journal_name: New England Journal of Medicine

publisher: Massachusetts Medical Society

publisher_dissemination: Massachusetts Medical Society

publisher_group: Massachusetts Medical Society

publisher_normalized: Massachusetts Medical Society

title: Oral Ivermectin versus Malathion Lotion for Difficult-to-Treat Head Lice

lead_sponsor: Johnson & Johnson Consumer and Personal Products Worldwide

location_country:
Israel
Ireland
France
United Kingdom

location_facility:
Synexus Clinical Research Centre
Chaim Sheba Medical Centre
Hopital Avicenne
Shandon Clinic


title: A Twenty-four Week, Open-label, Non-comparative, Multi-center Study to Assess the Efficacy and Tolerability of an Aliskiren-based Treatment Algorithm in Patients With Mild to Moderate Hypertension.

summary: This study will assess the efficacy of an aliskiren based treatment regimen in reaching blood pressure (BP) target in patients with mild to moderate hypertension. (defined as mean sitting Systolic Blood Pressure [msSBP] ≥ 140 mmHg and < 180 mmHg and/or mean sitting Diastolic Blood Pressure [msDBP] ≥ 90 and <110 mmHg).

location_country: Slovakia Romania Hungary France


title: Vanguard™ Deep Dish Rotating Platform Knee Clinical Evaluation

summary: This study is being conducted on the Vanguard™ Deep Dish Rotating Platform (DD RP) to evaluate the clinical efficacy of the DDRP components.

lead_sponsor: Zimmer Biomet

location_country:
United Kingdom
France
Netherlands

location_facility: Zuyderland Medisch Centrum Sittard-Geleen


title: A 52-week,Multi-center,Randomized,Double-blind,Placebo-controlled,Parallel Group Study in Patients With Mild Alzheimer's Disease to Investigate the Safety and Tolerability of Repeated Subcutaneous Injections of CAD106

summary: This study will evaluate the safety and tolerability of repeated subcutaneous injections of CAD106 in patients with mild Alzheimer's disease.

family: Farlow

full_name: Martin R Farlow

given: Martin R

family: Andreasen

full_name: Niels Andreasen

given: Niels

family: Riviere

full_name: Marie-Emmanuelle Riviere

given: Marie-Emmanuelle

family: Vostiar

full_name: Igor Vostiar

given: Igor

family: Vitaliti

full_name: Alessandra Vitaliti

given: Alessandra

family: Sovago

full_name: Judit Sovago

given: Judit

family: Caputo

full_name: Angelika Caputo

given: Angelika

family: Winblad

full_name: Bengt Winblad

given: Bengt

family: Graf

full_name: Ana Graf

given: Ana

location_facility: Centre Hospitalier Universitaire Pellegrin


title: An Observational, Prospective Evaluation of the Trifecta™ Valve

summary: This trial is designed to confirm the clinical safety and effectiveness of the Trifecta valve by establishing associated adverse event rates, clinical status as indicated by New York Heart Association (NYHA) functional classification, hemodynamic performance, and hematology analysis.

lead_sponsor: Abbott Medical Devices

location_country: Germany France Netherlands

location_facility:
Deutsches Herzzentrum München
Hôpital de Pontchaillou
Universitat Herzzentrum Leipzig GmbH
Onze Lieve Vrouwe Gasthuis


title: A Prospective, Single-arm, Multicenter 2-phase Feasibility Study to Assess the TAXUS Petal Paclitaxel-Eluting Bifurcation Coronary Stent System (TAXUS Petal)for the Treatment of de Novo Atherosclerotic Bifurcation Lesions

summary: The purpose of this study is to determine the safety and feasibility of use in humans of the TAXUS Petal Paclitaxel-Eluting Bifurcation Coronary Stent System in the treatment of de novo lesions in native coronary arteries involving a major side branch. The TAXUS® Petal™ is an investigational device with an indication of improving coronary artery luminal diameter while maintaining side branch access in subjects with symptomatic ischemic disease due to discrete atherosclerotic bifurcation lesions

ReferenceCitation: Ormiston JA, Lefèvre T, Grube E, Allocco DJ, Dawkins KD. First human use of the TAXUS Petal paclitaxel-eluting bifurcation stent. EuroIntervention. 2010 May;6(1):46-53. doi: 10.4244/.

lead_sponsor: Boston Scientific Corporation

location_country:
Germany
France
New Zealand

location_facility: Institut Hospitalier Jacques Cartier-ICPS, 6, Avenue du Noyer Lambert

location_facility: Auckland City Hospital, Cardiac Investigations Unit, Park Road

location_facility: Mercy Angiography Unit, 98 Mountain Road, First Floor

location_facility: HELIOS Klinikum Siegburg, Ringstrasse 49


title: A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major.

summary: In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.

lead_sponsor: Novartis Pharmaceuticals

location_country: Italy France


summary:
The primary objective of this trial is to demonstrate the equivalence, in terms of immunogenicity, of three different industrial lots of the investigational vaccine thereby supporting consistency of the manufacturing process.

Secondary Objectives:

Immunogenicity

To demonstrate that the investigational vaccine induces an immune response at least as good as the one induced by the reference vaccine, in terms of antibody titers.

To assess the immunogenicity of the investigational vaccine using parameters defined in the European Medicines Agency (EMEA) Note for Guidance (CPMP/BWP/214/96).

Safety:

To demonstrate that the investigational vaccine is at least as well tolerated as the reference vaccine, in terms of defined safety profile.

To describe the safety profile after vaccination.

Comfort of the vaccination assessment:

To assess the pain immediately after the injection using a Verbal Rating Scale. To describe the vaccination comfort after the injection using a -Patient-Reported Outcome questionnaire: the Vaccination Comfort Questionnaire.

ReferenceCitation: Arnou R, Eavis P, Pardo JR, Ambrozaitis A, Kazek MP, Weber F. Immunogenicity, large scale safety and lot consistency of an intradermal influenza vaccine in adults aged 18-60 years: Randomized, controlled, phase III trial. Hum Vaccin. 2010 Apr;6(4):346-54. Epub 2010 Apr 15.

ReferenceCitation: Chevat C, Viala-Danten M, Dias-Barbosa C, Nguyen VH. Development and psychometric validation of a self-administered questionnaire assessing the acceptance of influenza vaccination: the Vaccinees' Perception of Injection (VAPI) questionnaire. Health Qual Life Outcomes. 2009 Mar 4;7:21. doi: 10.1186/1477-7525-7-21.

family: Chevat

full_name: Catherine Chevat

given: Catherine

family: Viala-Danten

full_name: Muriel Viala-Danten

given: Muriel

family: Dias-Barbosa

full_name: Carla Dias-Barbosa

given: Carla

family: Nguyen

full_name: Van Hung Nguyen

given: Van Hung

journal_name: Health and Quality of Life Outcomes

publisher_dissemination: Springer

publisher_group: Springer

title: Development and psychometric validation of a self-administered questionnaire assessing the acceptance of influenza vaccination: the Vaccinees' Perception of Injection (VAPI©) questionnaire

lead_sponsor: Sanofi Pasteur, a Sanofi Company

location_country:
Spain
United Kingdom
France
Lithuania