New hope for rare brain disease as Final-Stage drug trial begins
NCT ID NCT05163288
First seen Apr 08, 2026 · Last updated Apr 28, 2026 · Updated 2 times
Summary
This study is testing an investigational drug called N-Acetyl-L-Leucine (IB1001) to see if it can help control symptoms and improve daily function for people with Niemann-Pick disease type C (NPC), a rare and serious genetic disorder. It is a Phase 3 trial, meaning it's in the final stage of testing before potential approval. Participants, who must be at least 4 years old, will take either the study drug or a placebo for periods of time to compare effects on movement, coordination, and quality of life.
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the original study
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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.
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
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Amsterdam UMC
RECRUITINGAmsterdam, 1105, Netherlands
Contact Email: •••••@•••••
Contact
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Comenius University in Bratislva
ACTIVE_NOT_RECRUITINGBratislava, 833 40, Slovakia
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First Faculty of Medicine, Charles University Hospital Prague
ACTIVE_NOT_RECRUITINGPrague, 128 08, Czechia
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Great Ormond Street Hospital
ACTIVE_NOT_RECRUITINGLondon, WC1N 3JH, United Kingdom
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Ludwig Maximilian University of Munich
NOT_YET_RECRUITINGMünchen, 80539, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Mayo Clinic
COMPLETEDRochester, Minnesota, 55905, United States
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Royal Free London NHS Foundation Trust
ACTIVE_NOT_RECRUITINGLondon, United Kingdom
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Royal Manchester Children's Hospital
ACTIVE_NOT_RECRUITINGManchester, M13 9WL, United Kingdom
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SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders
ACTIVE_NOT_RECRUITINGHöchheim, 65239, Germany
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The Royal Melbourne Hospital
ACTIVE_NOT_RECRUITINGParkville, Victoria, 3050, Australia
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University Hospital Bern Inselspital
ACTIVE_NOT_RECRUITINGBern, 3010, Switzerland
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University Hospital Münster
ACTIVE_NOT_RECRUITINGMünster, 48149, Germany
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University of Giessen
ACTIVE_NOT_RECRUITINGGiessen, 35389, Germany
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University of Hamburg
COMPLETEDHamburg, 20246, Germany
Conditions
Explore the condition pages connected to this study.