Hope for rare disease: phase 3 trial of N-Acetyl-L-Leucine for Niemann-Pick type c

NCT ID NCT05163288

Recruiting now Disease control Sponsor: IntraBio Inc Source: ClinicalTrials.gov ↗

First seen Apr 08, 2026 · Last updated May 12, 2026 · Updated 3 times

Summary

This study tests an investigational drug, N-acetyl-L-leucine, in people aged 4 and older with Niemann-Pick disease type C, a rare genetic disorder that affects movement and thinking. Participants receive either the drug or a placebo for a set period, then may continue on the drug for up to 3 years. The goal is to see if the treatment improves coordination and daily function.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Amsterdam UMC
RECRUITING

Amsterdam, 1105, Netherlands

Contact Email: •••••@•••••

Contact
Comenius University in Bratislva
ACTIVE_NOT_RECRUITING

Bratislava, 833 40, Slovakia
First Faculty of Medicine, Charles University Hospital Prague
ACTIVE_NOT_RECRUITING

Prague, 128 08, Czechia
Great Ormond Street Hospital
ACTIVE_NOT_RECRUITING

London, WC1N 3JH, United Kingdom
Ludwig Maximilian University of Munich
NOT_YET_RECRUITING

München, 80539, Germany

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
Mayo Clinic
COMPLETED

Rochester, Minnesota, 55905, United States
Royal Free London NHS Foundation Trust
ACTIVE_NOT_RECRUITING

London, United Kingdom
Royal Manchester Children's Hospital
ACTIVE_NOT_RECRUITING

Manchester, M13 9WL, United Kingdom
SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders
ACTIVE_NOT_RECRUITING

Höchheim, 65239, Germany
The Royal Melbourne Hospital
ACTIVE_NOT_RECRUITING

Parkville, Victoria, 3050, Australia
University Hospital Bern Inselspital
ACTIVE_NOT_RECRUITING

Bern, 3010, Switzerland
University Hospital Münster
ACTIVE_NOT_RECRUITING

Münster, 48149, Germany
University of Giessen
ACTIVE_NOT_RECRUITING

Giessen, 35389, Germany
University of Hamburg
COMPLETED

Hamburg, 20246, Germany

Conditions

Explore the condition pages connected to this study.

NIEMANN-PICK DISEASE, TYPE C NIEMANN-PICK DISEASE, TYPE C