Niemann-Pick Type C Disease and GM1/GM2 Gangliosidoses
Niemann-Pick type C disease and GM1/GM2 gangliosidoses are rare genetic disorders that affect the body's ability to metabolize fats and sugars, leading to neurological and physical symptoms.
Niemann-Pick Type C and GM1/GM2 Gangliosidosis: Nizubaglustat Study
We are investigating how well nizubaglustat works and its safety for children with Niemann-Pick type C disease and GM1 or GM2 gangliosidosis. This study aims to see if it can improve movement and coordination compared to a placebo.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you between 2 and 15 years old?
- Do you have a confirmed diagnosis of NPC disease?
- Have you had any neurological symptoms for 2 years or more?
- Are you currently taking any anti-epileptic medications that have changed in the last 3 months?
- Do you have severe liver problems, like high liver enzyme levels?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Microcrystalline cellulose
Nizubaglustat
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Centre Hospitalier Regional De Marseille
Departement of Pediatric Neurometabolism
Marseille, France
Assistance Publique Hopitaux De Paris
Department of child neurolgy
Saint-Mandé, France
SphinCS GmbH
Hochheim am Main, Germany
Sponsor: Azafaros B.V.
Last updated: Nov 26, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.