New pill hopes to tame rare childhood brain diseases
NCT ID NCT07054515
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests an oral drug called nizubaglustat (AZ-3102) in people aged 4 and older with Niemann-Pick type C disease, GM1 gangliosidosis, or GM2 gangliosidosis. The study lasts 18 months and compares the drug to a placebo to see if it is safe and can slow disease progression. About 147 participants will be enrolled across multiple centers.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
nizubaglustat (AZ-3102), an oral drug
What this could lead to
If successful, this could provide a treatment option to slow or manage these rare, progressive neurological diseases.
What could go wrong
This is an early-stage Phase 3 trial with only 147 participants, so results may not apply to all patients. The drug may cause side effects or fail to show benefit.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
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
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
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 Príncipe
NOT_YET_RECRUITINGCuritiba, 80250-060, 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
RECRUITINGBern, Canton of Bern, 3010, Switzerland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Instituto Fernandes Figueira
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 Childrens 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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Royal Melbourne Hospital
RECRUITINGParkville, Victoria, 3050, Australia
Contact Email: •••••@•••••
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Sahlgrenska universitetssjukhuset Östra
RECRUITINGGothenburg, Västra Götaland County, 416 50, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
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SphinCS GmbH
RECRUITINGHöchheim, 65239, Germany
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The Children's Hospital & the University of Child Health Lahore
NOT_YET_RECRUITINGLahore, Punjab Province, Pakistan
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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: •••••@•••••