Hope for kids with rare brain diseases: new pill trial launches
NCT ID NCT07082543
First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 25 times
Summary
This study tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with GM1 or GM2 gangliosidosis—rare genetic disorders that damage the brain and nerves. Over 18 months, half the participants will receive the drug and half a placebo to see if it slows disease progression and improves movement and speech. The goal is to find a treatment that helps control these devastating 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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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AP-HP - Hôpital Armand Trousseau
RECRUITINGParis, 75012, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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All India Institute of Medical Sciences (AIIMS) - New Delhi
RECRUITINGNew Delhi, National Capital Territory of Delhi, 110029, India
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Amrita Institute of Medical Sciences and Research Centre
RECRUITINGErnākulam, Kerala, 682041, India
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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 NHSFT
NOT_YET_RECRUITINGLondon, 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
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
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
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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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Mayo Clinic Children's Center - PIN
RECRUITINGRochester, Minnesota, 55905, United States
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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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Sahlgrenska universitetssjukhuset Östra
RECRUITINGGothenburg, Västra Götaland County, 416 50, Sweden
Contact Phone: •••-•••-•••• 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 Santa Maria,EPE - Hospital de Santa Maria - PPDS
RECRUITINGLisbon, 1649-035, Portugal
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.