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Clinical Trials in the UK / NCT07054515
Recruiting Phase 3

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, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515 · tracked via the Priya Life Science UK tracker
Sponsor
Azafaros B.V.
Phase
Phase 3
Started
2025-06-30
Last updated
2026-09-16

Condition(s) studied

Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis

Investigational drug(s) / intervention(s)

AZ-3102Placebo

AZ-3102: Oral dispersible tablets

Placebo: A matching placebo will be administered in the same regimen as the intervention

Study summary

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Eligibility

Sex
ALL
Min age
4 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725). * Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543). Exclusion Criteria: * Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC * Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Primary outcome measure(s)

Trial sites (38)

FacilityCityRegionStatus
UCSF Children's Hospital and Research Center at Oakland Oakland California Recruiting
University of Minnesota Medical School Minneapolis Minnesota Not Yet Recruiting
Mayo Clinic Childrens Center - PIN Rochester Minnesota Recruiting
Children's Medical Center Dallas Dallas Texas Recruiting
Lysosomal Rare Disorders Research and Treatment Center Fairfax Virginia Recruiting
Hospital Universitario Austral Ciudad Autónoma Buenos Aires Buenos Aires Active Not Recruiting
Hospital de Niños de La Santisima Trinidad Córdoba Córdoba Province Recruiting
Women's and Children's Hospital North Adelaide South Australia Recruiting
Royal Melbourne Hospital Parkville Victoria Recruiting
Royal Children's Hospital Melbourne - PIN Parkville Victoria Recruiting
Instituto Fernandes Figueira Rio de Janeiro Rio de Janeiro Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS Porto Alegre Rio Grande do Sul Recruiting
Hospital Pequeno Príncipe Curitiba Brazil Not Yet Recruiting
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary Calgary Alberta Recruiting
University of Alberta Medical Genetics Clinic Edmonton Alberta Not Yet Recruiting
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis Montreal Quebec Not Yet Recruiting
AP-HP - Hôpital Armand Trousseau Paris France Recruiting
SphinCS GmbH Höchheim Germany Recruiting
Amrita Institute of Medical Sciences and Research Centre Ernākulam Kerala Recruiting
All India Institute of Medical Sciences (AIIMS) - New Delhi New Delhi National Capital Territory of Delhi Recruiting
JK Lone Hospital Jaipur Rajasthan Recruiting
Christian Medical College and Hospital Vellore Tamil Nadu Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy Recruiting
Hospital Universitario Dr. Jose Eleuterio González Monterrey Nuevo León Not Yet Recruiting
Centenario Hospital Miguel Hidalgo Aguascalientes Mexico Not Yet Recruiting
The Children's Hospital & the University of Child Health Lahore Lahore Punjab Province Not Yet Recruiting
ULS de Santo António, EPE - Centro Materno Infantil Norte Porto Porto District Recruiting
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS Lisbon Portugal Recruiting
Hospital Universitario Vall d'Hebron - PPDS Barcelona Barcelona Recruiting
Hospital Infantil Universitario Niño Jesus - PIN Madrid Madrid Recruiting
Sahlgrenska universitetssjukhuset Östra Gothenburg Västra Götaland County Active Not Recruiting
Inselspital - Universitätsspital Bern Bern Canton of Bern Recruiting
Cukurova Universitesi Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi Adana Adana Recruiting
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Çankaya Ankara Recruiting
Ege Universitesi Tip Fakultesi Bornova İzmir Recruiting
Great Ormond Street Hospital London London Recruiting
University College London Hospitals (UCLH) London Middlesex Not Yet Recruiting
Royal Manchester Children's Hospital Manchester United Kingdom Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07054515 on ClinicalTrials.gov ↗ ← All trials in the UK