Pill vs. infusion: new hope for rare neurological disease
NCT ID NCT05222906
Summary
This study is testing if a daily oral pill called venglustat can work as well as the current standard of care—biweekly intravenous infusions—for maintaining stability in people with Gaucher disease type 3. It involves about 43 adults and teenagers (12+) who are already stable on their current infusion therapy. The main goal is to see if the pill can keep their neurological and physical symptoms from getting worse over a year.
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Contacts and locations
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Locations
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47-87, boulevard de l'hôpital - Investigational Site Number: 2500003
Paris, 75013, France
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Azienda Ospedaliera Universitaria (AOU) "Federico II" - Investigational Site Number: 3800002
Naples, 80131, Italy
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Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001
Winnipeg, Manitoba, R3E 3P4, Canada
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Cukurova University Medical School Hospital-Investigational Site Number : 7920001
Adana, 01790, Turkey (Türkiye)
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Debreceni Egyetem, Klinikai Központ, Reumatológiai Klinika - Investigational Site Number: 3480001
Debrecen, H-4032, Hungary
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Investigational Site Number : 3800003
Milan, Lombardy, 20122, Italy
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Gazi University Medical Hospital-Investigational Site Number : 7920002
Ankara, 06500, Turkey (Türkiye)
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Hopital Necker - Investigational Site Number: 2500001
Paris, 75015, France
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Hospital de Ninos - Investigational Site Number: 320001
Buenos Aires, 1426, Argentina
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Investigational Site Number : 8260001
London, NW3-2PF, United Kingdom
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Istanbul University Medical Faculty Hospital-Investigational Site Number : 7920004
Istanbul, 34093, Turkey (Türkiye)
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Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001
Fairfax, Virginia, 22030, United States
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National Taiwan University Hospital-Investigational Site Number: 1580001
Taipei, Taiwan, 10041, China
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Odawara Municipal Hospital-Investigational Site Number : 3920002
Odawara, Kanagawa, 250-8558, Japan
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Peking Union Medical College Hospital - Investigational Site Number: 1560001
Beijing, 100005, China
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SphinCS GmbH - Investigational Site Number: 2760001
Hochheim am Main, 65239, Germany
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Texas Oncology - Medical City Dallas Site Number : 8400008
Dallas, Texas, 75230, United States
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The First Affiliated Hospital - Investigational Site Number: 1560002
Guangzhou, 510080, China
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Tohoku University School of Medicine - Investigational Site Number: 3920001
Sendai, 980-8574, Japan
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University of Iowa - Investigational Site Number: 8400002
Iowa City, Iowa, 52242, United States
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Xinhua Hospital - Investigational Site Number: 1560004
Shanghai, 200092, China
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Yale University School of Medicine - Investigational Site Number: 8400003
New Haven, Connecticut, 06511, United States
Conditions
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