New pill hopes to tame rare childhood brain diseases
NCT ID NCT07054515
First seen Jun 27, 2026 · Last updated Aug 14, 2026 · Updated 14 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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AP-HP - Hôpital Armand Trousseau
RECRUITINGParis, 75012, France
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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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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
RECRUITINGVellore, Tamil Nadu, 632004, India
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Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
RECRUITINGAdana, Adana, 1250, Turkey (Türkiye)
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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
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Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
RECRUITINGÇankaya, Ankara, 6500, Turkey (Türkiye)
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Great Ormond Street Hospital
RECRUITINGLondon, London, WC1N 3JH, United Kingdom
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Hospital Infantil Universitario Niño Jesus - PIN
RECRUITINGMadrid, Madrid, 28009, Spain
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Hospital Pequeno Príncipe
NOT_YET_RECRUITINGCuritiba, 80250-060, Brazil
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Hospital Universitario Austral
ACTIVE_NOT_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
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Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
RECRUITINGPorto Alegre, Rio Grande do Sul, 90560-030, Brazil
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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
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Instituto Fernandes Figueira
RECRUITINGRio de Janeiro, Rio de Janeiro, 22250-020, Brazil
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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
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M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
RECRUITINGCalgary, Alberta, T3B 6A8, Canada
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Mayo Clinic Childrens Center - PIN
RECRUITINGRochester, Minnesota, 55905, United States
Contact Email: •••••@•••••
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Royal Children's Hospital Melbourne - PIN
NOT_YET_RECRUITINGParkville, Victoria, 3052, Australia
Contact 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
ACTIVE_NOT_RECRUITINGGothenburg, Västra Götaland County, 416 50, Sweden
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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
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ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
RECRUITINGLisbon, 1649-035, Portugal
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ULS de Santo António, EPE - Centro Materno Infantil Norte
RECRUITINGPorto, Porto District, 4050-651, Portugal
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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
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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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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