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评价RG6042在亨廷顿舞蹈病患者中的临床研究

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研究单位: [1]四川大学华西医院 [2]eparment of Neurology [3]epartment of Neurology [4]niversitatskliniik für Neurologie [5]epartment fuer Neurologie [6]ivision of Neurology [7]eurologisk Afdeling F,Neurogenetisk Afsnit [8]ervice de Neurologie [9]ERVICE DE NEUROLOGIE [10]épartement de Génétique et Cytogénétique [11]eurologie [12]epartement de Neurologie [13]linik für Neurologie [14]entrum für Seltene Erkrankungen [15]bt. Neuropsychiatrie [16]echnologiepark Münster [17]untington-Center NRW,Abt. Neurodegeneration [18]OC Clinica Neurologica [19]INOGMI [20].O.C. Genetica Medica-Neurogenetica [21]OC Neurologia [22]iaprtimento Scienze Neurologiche e Psichiatriche [23]nità Ricerca e Cura Huntington e Malattie Rare [24]eurology Department [25]ddzial Neurologiczny [26]ervicio de Neurologia [27]ervicio de Neurología [28]anchester Centre for Genomic Medicine [29]edical Genectics [30]esearch Division,Psychiatry [31]cGovern Medical School [32]浙江大学医学院附属第二医院 [33]中山大学附属第一医院 [34]首都医科大学宣武医院 [35]福建医科大学附属第一医院 [36]复旦大学附属华山医院 [37]Hospital Ramos Mejía [38]Hospital Britanico de Buenos Aires [39]WESTMEAD HOSPITAL [40]Monash Medical Centre [41]Royal Melbourne Hospital [42]Christian-Doppler-Klinik - Universitatsklinikum [43]Uniklinik fuer Neurologie,Medizinische Universitaet Innsbruck [44]Centre Hospitalier de l’Université de Montréal (CHUM) [45]Centre for Movement Disorders [46]University of British Columbia Hospital [47]Ottawa Hospital Research Institute [48]True North Clinical Research-Halifax [49]University of Alberta Hospital [50]Centro de Trastornos del Movimiento [51]Rigshospitalet,Hukommelsesklinikken [52]Aarhus Universitetshospital [53]CHU de la Timone - Hopital d Adultes [54]HOPITAL HENRI MONDOR [55]Hospital Pitié Salpêtrère [56]Hopital Gui de Chauliac [57]CHU toulouse - Hospital Purpan [58]Hopital Roger Salengro [59]CHU Amiens Hopital Sud [60]CHU Angers,Batiement Larrey 2 [61]Universitatsklinikum Ulm [62]Universitatsklinikum Schleswig-Holstein / Campus Lübeck [63]Charité - Universitatsmed. Berlin,Klinik für Psychiatrie und Psychotherapie [64]Uniklinik RWTH Aachen [65]George-Huntington- Institut GmbH [66]St. Josef-Hospital,Neurologische Klinik der Ruhr-Uni [67]German Center for Neurodegenerative Diseases (DZNE) [68]University tsklinikum Erlangen,Abteilung Molekulare Neurologie [69]IRCCS Istituto delle Scienze Neurologiche [70]IRCCS A.O.U.SAN MARTINO IST [71]Fondazione IRCCS Istituto Neurologico Carlo Besta [72]Azienda Ospedaliera Sant'Andrea [73]A.O.U. Careggi [74]IRCCS Casa Sollievo Della Sofferenza [75]Okayama University Graduate School of Medicine,Densitry and Pharmaceutical Sciences. [76]National Hospital Organization Sagamihara National Hospital [77]Obihiro Kosei General Hospital [78]National Hospital Organization Niigata National Hospital [79]Osaka General Medical Center [80]Kuwana City Medical Center [81]National Center of Neurology and Psychiatry [82]LUMC [83]Universitair Medisch Centrum Groningen [84]New Zealand Brain Research Institute [85]Wellington Hospital [86]Auckland DHB - Neurlogy Department [87]Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K [88]Szpital Sw. Wojciecha [89]Instytut Psychiatrii i Neurologii [90]Research Center of Neurology [91]FSBHI Siberian Clinical Center of the Federal Medical and Biological Agency [92]Сlinical hospital at Kazan station,Republican Center for Movement Disorders and Botulinum Therapy [93]Hospital de Cruces [94]Hospital Universitario de Burgos. Servicio de Neurología [95]Hospital Universitario Virgen Macarena [96]Fundacion Jimenez Diaz [97]Hospital de la Santa Creu i Sant Pau [98]Hospital Ramon y Cajal [99]Hospital Clinic Servicio de Neurologia [100]Hospital Universitario la Fe [101]Hospital Universitario de Badajoz [102]Neurozentrum Siloah [103]Universitatsspital Basel [104]Cambridge Centre for Brain Repair [105]Leeds General Infirmary [106]Central Manchester University Hospitals NHS Foundation Trust [107]University Hospital Southampton NHS Foundation Trust [108]Aberdeen Royal Infirmary [109]John Radcliffe Hospital [110]Royal Hallamshire Hospital [111]QUEEN ELIZABETH HOSPITAL [112]Queen Elizabeth University Hospital Glasgow [113]University Hospital of Wales [114]National Hospital For Neurology and Neurosurgery [115]Evergreen Health Care Center [116]Georgetown University [117]John Hopkins University School of Medicine [118]Northwestern University [119]Stanford Univ Medical Center [120]Vanderbilt University Medical Center [121]University of California San Diego [122]Washington University School of Medicine [123]Beth Israel Deaconess Medical Center [124]The University of Texas Health Science Center at Houston [125]Rocky Mountain Movement Disorders Center [126]Columbia University [127]University of Utah Clinical Neurosciences Center [128]Dent Neurological Institute [129]Barrow Neurological Institute [130]University of South Florida [131]SC3 Research Group,Inc [132]UAB MEDICINE [133]University of Pittsburgh [134]Barrow Neurological Institute [135]University of California Davis Medical System [136]INEBA

研究目的:
本研究将评价RO7234292 与安慰剂相比在显性亨廷顿舞蹈病(HD)患者中的有效性、安全性、药代动力学(PK)和生物标志物效应。主要有效性目的是基于以下终点评价RO7234292 相较于安慰剂的有效性:第101 周时复合统一亨廷顿舞蹈病评定量表(cUHDRS)评分相对基线变化

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