Race against time: new pill tested to halt rare brain disease
NCT ID NCT04221451
Summary
This study tested whether a daily oral pill called venglustat could slow the progression of late-onset Tay-Sachs and Sandhoff disease, rare and severe inherited disorders that damage the brain and nerves. It involved about 75 adults and some younger participants over a two-year treatment period. The main goals were to see if the drug reduced harmful substances in the body and improved or stabilized physical abilities like hand coordination and walking.
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Contacts and locations
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Locations
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APHP - Centre Hospitalier la Pitié Salpetrière, Service de Neurologie, 47-83 Boulevard De l'Hôpital_Investigational site number 2500001
Paris, 75013, France
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Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004
Los Angeles, California, 90095, United States
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Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital Hills Road_investigational site number 8260001
Cambridge, Cambridgeshire, CB2 OQQ, United Kingdom
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Centro Hospitalar Universitário Lisboa Norte- Hospital Santa Maria Avenida Professor Egas Moniz_investigational site number 6200002
Lisbon, 1649-035, Portugal
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Clínica Universitaria Reina Fabiola Córdoba_investigational site number 0320001
Córdoba, X5004FHP, Argentina
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Emory Genetics - Investigational site number 8400006
Atlanta, Georgia, 30322, United States
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Gazi Universitesi Tip Fakultesi Emniyet Street Mevlana Blv Besevler Yenimahalle_investigational site number 7920001
Ankara, 06500, Turkey (Türkiye)
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Hospital Clínico Universitario de Santiago-CHUS. Travesia de Chopuana, s/n_investigational site number 7240002
Santiago de Compostela, Galicia [Galicia], 15706, Spain
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Hospital Maternoinfantil Sant Joan de Déu Paseo de Sant Joan de Déu, 2_investigational site number 7240001
Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain
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Hospital Universitari de Bellvitge. Feixa Llarga, S / N_investigational site number 7240004
L'Hospitalet de Llobregat, Barcelona [Barcelona], 08907, Spain
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Hospital Universitario Austral Pilar, Buenos Aires_Unidad de Investigación Clínica_investigational site number 0320002
Pilar, Buenos Aires, B1629ODT, Argentina
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Hospital de Clinicas de Porto Alegre _investigational site number 0760001
Porto Alegre, Rio Grande do Sul, 90035 003, Brazil
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Investigational Site Number : 3800001
Milan, 20133, Italy
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Investigational Site Number : 8260003
Manchester, M13 9WL, United Kingdom
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Japanese Red Cross Akita Hospital 222-1 Nawashirosawa, Kamikitatesaruta_investigational site number 3920001
Akita, Akita, 010-1495, Japan
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Massachusetts General Hospital - Charles River Plaza 175 Cambridge St. Ste 340 - Investigational site number 8400002
Boston, Massachusetts, 02114, United States
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NIH National Human Genome Research Institute - 10 Center Dr - Bldg. 10 CRC3-2551 MSC 1205 - Investigational site number 8400005
Bethesda, Maryland, 20892, United States
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NYU Langone - 550 First Avenue-Investigational site number 8400001
New York, New York, 10016, United States
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Research Center Of Neurology 80, Volokolamskoye shosse_investigational site number 6430001
Moscow, 125367, Russia
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Salford Royal NHS Foundation Trust Salford Royal Hospital Stott Lane_investigational site number 8260002
Salford, M6 8HD, United Kingdom
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Tohoku Medical and Pharmaceutical University Hospital_investigation site number 3920002
Sendai, Miyagi, 983-8512, Japan
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Universitätsklinikum der Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Feulgenstraße 10-12_investigational site number 2760001
Giessen, 35390, Germany
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Vseobecna Fakultni Nemocnice V Praze Metabolicke centrum U Nemocnice 2_investigational site number 2030001
Prague, 12808, Czechia
Conditions
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