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
NCT ID: NCT07054515
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
147 participants
INTERVENTIONAL
2025-06-30
2027-11-04
Brief Summary
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Detailed Description
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Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.
For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Subprotocol NCT07082725: Intervention Group NPC
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
AZ-3102
Oral dispersible tablets
Placebo
A matching placebo will be administered in the same regimen as the intervention
Subprotocol NCT07082543: Intervention Group GM1/GM2
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
AZ-3102
Oral dispersible tablets
Placebo
A matching placebo will be administered in the same regimen as the intervention
Interventions
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AZ-3102
Oral dispersible tablets
Placebo
A matching placebo will be administered in the same regimen as the intervention
Other Intervention Names
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Eligibility Criteria
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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
4 Years
ALL
No
Sponsors
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Azafaros A.G.
INDUSTRY
Responsible Party
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Locations
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Children's Hospital and Research Center at Oakland
Oakland, California, United States
University of Minnesota Medical School
Minneapolis, Minnesota, United States
Mayo Clinic Childrens Center - PIN
Rochester, Minnesota, United States
Children's Medical Center Dallas
Dallas, Texas, United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, Argentina
Women's and Children's Hospital
North Adelaide, South Australia, Australia
Royal Melbourne Hospital
Parkville, Victoria, Australia
Royal Children's Hospital Melbourne - PIN
Parkville, Victoria, Australia
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, Brazil
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
Hospital Pequeno Príncipe
Curitiba, , Brazil
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, Canada
University of Alberta Medical Genetics Clinic
Edmonton, Alberta, Canada
Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
Montreal, Quebec, Canada
AP-HP - Hôpital Armand Trousseau
Paris, , France
SphinCS GmbH
Höchheim, , Germany
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, India
Seth G S Medical College and K E M Hospital
Mumbai, Maharashtra, India
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, India
JK Lone Hospital
Jaipur, Rajasthan, India
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, Portugal
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
Lisbon, , Portugal
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, Spain
Sahlgrenska universitetssjukhuset Östra
Gothenburg, Västra Götaland County, Sweden
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, Turkey (Türkiye)
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Bornova, İzmir, Turkey (Türkiye)
Great Ormond Street Hospital
London, London, United Kingdom
University College London Hospitals (UCLH)
London, Middlesex, United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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Central Contacts
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Patient Advocacy Representative
Role: CONTACT
Phone: Please reach out by email
Email: [email protected]
Contact for Healthcare Professionals
Role: CONTACT
Phone: Please reach out by email
Email: [email protected]
Other Identifiers
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2024-515778-28-00
Identifier Type: CTIS
Identifier Source: secondary_id
AZA-001-301
Identifier Type: -
Identifier Source: org_study_id