New hope for rare childhood brain disease: major trial tests first oral treatment
NCT ID NCT07082725
Summary
This study is testing whether a daily oral medication called nizubaglustat can safely slow the progression of Niemann-Pick Type C (NPC), a rare and severe genetic disorder that affects the brain. It will involve about 72 children and young people (ages 4+) with NPC over 18 months. The main goal is to see if the drug is better than a placebo at improving problems with movement and coordination (ataxia).
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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All India Institute of Medical Sciences (AIIMS) - New Delhi
RECRUITINGNew Delhi, National Capital Territory of Delhi, 110029, India
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Amrita Institute of Medical Sciences and Research Centre
RECRUITINGErnākulam, Kerala, 682041, India
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Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
NOT_YET_RECRUITINGAdana, Adana, 1250, Turkey (Türkiye)
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Centenario Hospital Miguel Hidalgo
NOT_YET_RECRUITINGAguascalientes, 20000, Mexico
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Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
NOT_YET_RECRUITINGMontreal, Quebec, H2X 0C1, Canada
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Children's Medical Center Dallas
NOT_YET_RECRUITINGDallas, Texas, 75235, United States
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Christian Medical College and Hospital
NOT_YET_RECRUITINGVellore, Tamil Nadu, 632004, India
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Ege Universitesi Tip Fakultesi
NOT_YET_RECRUITINGBornova, İzmir, 35100, Turkey (Türkiye)
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Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITINGMilan, 20133, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
NOT_YET_RECRUITINGÇankaya, Ankara, 6500, Turkey (Türkiye)
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Great Ormond Street Hospital
NOT_YET_RECRUITINGLondon, London, WC1N 3JH, United Kingdom
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Hospital Infantil Universitario Niño Jesus - PIN
RECRUITINGMadrid, Madrid, 28009, Spain
Contact Email: •••••@•••••
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Hospital Pequeno Principe
NOT_YET_RECRUITINGCuritiba, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Universitario Austral
NOT_YET_RECRUITINGCiudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina
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Hospital Universitario Dr. Jose Eleuterio González
NOT_YET_RECRUITINGMonterrey, Nuevo León, 64460, Mexico
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Hospital Universitario Vall d'Hebron - PPDS
RECRUITINGBarcelona, Barcelona, 8035, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITINGPorto Alegre, Rio Grande do Sul, 90560-030, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Hospital de Niños de La Santisima Trinidad
RECRUITINGCórdoba, Córdoba Province, X5004 ASL, Argentina
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Inselspital - Universitätsspital Bern
NOT_YET_RECRUITINGBern, Canton of Bern, 3010, Switzerland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Instituto Fernandes Figueira
NOT_YET_RECRUITINGRio de Janeiro, Rio de Janeiro, 22250-020, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
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JK Lone Hospital
RECRUITINGJaipur, Rajasthan, 302004, India
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Lysosomal Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030-7404, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
NOT_YET_RECRUITINGCalgary, Alberta, T3B 6A8, Canada
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Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Manchester Children's Hospital
NOT_YET_RECRUITINGManchester, M13 9WL, United Kingdom
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Royal Melbourne Hospital
NOT_YET_RECRUITINGParkville, Victoria, 3050, Australia
Contact Email: •••••@•••••
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SphinCS GmbH
NOT_YET_RECRUITINGHöchheim, 65239, Germany
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UCSF Children's Hospital and Research Center at Oakland
RECRUITINGOakland, California, 94609, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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ULS de Santo António, EPE - Centro Materno Infantil Norte
RECRUITINGPorto, Porto District, 4050-651, Portugal
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University College London Hospitals (UCLH)
NOT_YET_RECRUITINGLondon, Middlesex, WC1N 3BG, United Kingdom
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University of Alberta Medical Genetics Clinic
NOT_YET_RECRUITINGEdmonton, Alberta, T6G 2B7, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Minnesota Medical School
NOT_YET_RECRUITINGMinneapolis, Minnesota, 55455, United States
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Women's and Children's Hospital
RECRUITINGNorth Adelaide, South Australia, 5006, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.