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
Launched by AZAFAROS A.G. · Jun 27, 2025
Trial Information
Current as of November 12, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is testing a new oral medicine called nizubaglustat (AZ-3102) to see if it is safe and helpful for people with certain rare genetic diseases that affect the brain and nervous system. These diseases include late-infantile and juvenile forms of Niemann-Pick type C disease, GM1 gangliosidosis, and GM2 gangliosidosis (which includes Tay-Sachs and Sandhoff diseases). The study will last about 18 months and will compare the effects of the medicine to a placebo (a pill without medicine) without participants or doctors knowing who is receiving which treatment until the study ends.
The trial is open to both boys and girls, as well as adults, who are 4 years old or older and have been diagnosed with one of these specific forms of the diseases mentioned. Participants can expect to take the medicine by mouth and will have regular check-ups at multiple study centers to monitor their health and how well the medicine works. This study is not yet recruiting, but if you or a family member fits these criteria and are interested, it’s important to speak with a healthcare provider or the study team for more details.
Gender
ALL
Eligibility criteria
- 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
About Azafaros A.G.
Azafaros A.G. is an innovative biopharmaceutical company dedicated to advancing the development of novel therapeutics for rare diseases, with a strong emphasis on precision medicine. Leveraging cutting-edge research and a robust scientific foundation, Azafaros focuses on creating targeted treatments that address unmet medical needs, particularly in the fields of metabolic and neurological disorders. Committed to improving patient outcomes, the company collaborates with leading academic institutions and industry partners to drive clinical trials that ensure the safety and efficacy of its drug candidates. Through its strategic approach and commitment to scientific excellence, Azafaros A.G. aims to make a meaningful impact on the lives of patients worldwide.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
North Adelaide, South Australia, Australia
Parkville, Victoria, Australia
Minneapolis, Minnesota, United States
Oakland, California, United States
Dallas, Texas, United States
London, United Kingdom
Rio De Janeiro, Brazil
Bern, Switzerland
Milan, Italy
Manchester, United Kingdom
Bornova, Izmir, Turkey
Milano, Italy
Barcelona, Spain
Parkville, Victoria, Australia
Paris, France
Madrid, Spain
Jaipur, Rajasthan, India
Montreal, Quebec, Canada
Ernakulam, Kerala, India
New Delhi, Delhi, India
Curitiba, Brazil
Gothenburg, Sweden
Rochester, Minnesota, United States
Fairfax, Virginia, United States
Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
Córdoba, Argentina
Porto Alegre, Rio Grande Do Sul, Brazil
Calgary, Alberta, Canada
Edmonton, Alberta, Canada
Montréal, Quebec, Canada
Marseille, France
Mumbai, Maharashtra, India
Lisbon, Portugal
Porto, Portugal
Göteborg, Vastra Gotalands Lan, Sweden
çankaya, Ankara, Turkey
Adana, Turkey
London, Middlesex, United Kingdom
Rio De Janeiro, Rio De Janeiro, Brazil
Ernākulam, Kerala, India
New Delhi, National Capital Territory Of Delhi, India
Porto, Porto District, Portugal
Barcelona, Barcelona, Spain
Madrid, Madrid, Spain
Bern, Canton Of Bern, Switzerland
Adana, Adana, Turkey (Türkiye)
çankaya, Ankara, Turkey (Türkiye)
Bornova, Turkey (Türkiye)
London, London, United Kingdom
Höchheim, Germany
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported