Niemann-Pick Disease Type C
Niemann-Pick disease type C (NPC) is a rare genetic disorder that affects the body's ability to transport cholesterol and other lipids within cells, leading to progressive neurological symptoms and organ dysfunction.
Niemann-Pick Disease Type C: N-Acetyl-L-Leucine Study
We are investigating whether N-Acetyl-L-Leucine can improve symptoms and quality of life for individuals with Niemann-Pick disease type C. The study also looks at the safety of this treatment for both older and younger patients.
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 at least 4 years old?
- Do you have a confirmed diagnosis of Niemann-Pick disease type C (NPC)?
- Do you weigh at least 15 kg?
- Do you have any known allergies to Acetyl leucine or its ingredients?
- Do you have any physical or mental health issues that might affect your ability to participate in a study?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Granules for Oral Suspension
N-Acetyl-L-Leucine
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.
Loading map...
Vseobecna Fakultni Nemocnice V Praze
Klinika pediatrie a dědičných poruch metabolismu
Prague, Czechia
Justus-Liebig-Universitaet Giessen
Neurology
Gießen, Germany
SphinCS GmbH
Clinical Science in LDS
Hochheim am Main, Germany
Sponsor: Intrabio Limited
Last updated: Nov 27, 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.