Experimental drug for rare brain diseases shows promise but trial ends early
NCT ID NCT04221451
First seen Jan 30, 2026 · Last updated May 14, 2026 · Updated 12 times
Summary
This study tested an oral drug called venglustat in adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that damage the nervous system. The goal was to see if the drug could reduce harmful substances in the brain and slow disease progression. The trial was terminated early, but data collected may help understand how to treat these conditions.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
Explore the condition pages connected to this study.