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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-28NCT07082725

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OrganizationsLinked trials
Azafaros B.V.5

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Research sitesCityCountryLinked trials
All India Institute of Medical Sciences (AIIMS) - New DelhiNew DelhiIndia5
Amrita Institute of Medical Sciences and Research CentreErnākulamIndia3
Centenario Hospital Miguel HidalgoAguascalientesMexico2
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-DenisMontrealCanada2
Children's Medical Center DallasDallasUnited States18
Christian Medical College and HospitalVelloreIndia2
Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma MerkeziAdanaTurkey (Türkiye)2
Ege Universitesi Tip FakultesiBornovaTurkey (Türkiye)3
Fondazione IRCCS Istituto Neurologico Carlo BestaMilanItaly35
Gazi Universitesi Saglik Arastirma ve Uygulama MerkeziÇankayaTurkey (Türkiye)3
Great Ormond Street HospitalLondonUnited Kingdom2
Hospital de Clinicas de Porto Alegre (HCPA) - PPDSPorto AlegreBrazil5
Hospital de Niños de La Santisima TrinidadCórdobaArgentina3
Hospital Infantil Universitario Niño Jesus - PINMadridSpain3
Hospital Pequeno PrincipeCuritibaBrazil1
Hospital Universitario Dr. Jose Eleuterio GonzálezMonterreyMexico3
Hospital Universitario Vall d'Hebron - PPDSBarcelonaSpain4
Inselspital - Universitätsspital BernBernSwitzerland4
Instituto Fernandes FigueiraRio de JaneiroBrazil3
JK Lone HospitalJaipurIndia3
Lysosomal Rare Disorders Research and Treatment CenterFairfaxUnited States3
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in CalgaryCalgaryCanada3
Mayo Clinic Children's Center - PINRochesterUnited States2
Royal Children's Hospital Melbourne - PINParkvilleAustralia7
Royal Manchester Children's HospitalManchesterUnited Kingdom32