A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Recruiting

Phase 3 — Large-scale testing across thousands of people, comparing the new treatment against current standard treatments.

Trial locations(14 sites)

United States

Mayo Clinic, Rochester, Minnesota

Australia

The Royal Melbourne Hospital, Parkville, Victoria

Czechia

First Faculty of Medicine, Charles University Hospital Prague, Prague

Germany

University of Giessen, Giessen

University of Hamburg, Hamburg

SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders, Höchheim

Ludwig Maximilian University of Munich, München

University Hospital Münster, Münster

Netherlands

Amsterdam UMC, Amsterdam

Slovakia

Comenius University in Bratislva, Bratislava

Switzerland

University Hospital Bern Inselspital, Bern

United Kingdom

Great Ormond Street Hospital, London

Royal Free London NHS Foundation Trust, London

Royal Manchester Children's Hospital, Manchester