Condition: Niemann-Pick Disease, Type C · Sponsor: IntraBio Inc
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care. Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.
This description comes directly from the study's public registry record.
Michael Strupp, MD · +44 8081 641283 · mstrupp@intrabio.com
Taylor Fields · +44 8081 641283 · tfields@intrabio.com
Always discuss trial participation with your own doctor first.
| Mayo Clinic | Rochester, Minnesota, United States | Completed |
| The Royal Melbourne Hospital | Parkville, Victoria, Australia | Active Not Recruiting |
| First Faculty of Medicine, Charles University Hospital Prague | Prague, Czechia | Active Not Recruiting |
| University of Giessen | Giessen, Germany | Active Not Recruiting |
| University of Hamburg | Hamburg, Germany | Completed |
| SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders | Höchheim, Germany | Active Not Recruiting |
| Ludwig Maximilian University of Munich | München, Germany | Not Yet Recruiting |
| University Hospital Münster | Münster, Germany | Active Not Recruiting |
| Amsterdam UMC | Amsterdam, Netherlands | Recruiting |
| Comenius University in Bratislva | Bratislava, Slovakia | Active Not Recruiting |
| University Hospital Bern Inselspital | Bern, Switzerland | Active Not Recruiting |
| Great Ormond Street Hospital | London, United Kingdom | Active Not Recruiting |
+ 2 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05163288