Hope for rare brain disease: new drug trial targets movement and daily life
NCT ID NCT05163288
First seen Apr 08, 2026 · Last updated May 25, 2026 · Updated 4 times
Summary
This study tests an experimental drug called N-acetyl-L-leucine (IB1001) for people aged 4 and older with Niemann-Pick disease type C (NPC), a rare genetic disorder that affects movement and thinking. About 53 participants will receive either the drug or a placebo for a period, then switch to the other. The goal is to see if the drug safely improves coordination, daily function, and quality of life. After the main study, all participants can receive the drug for up to 3 more years.
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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
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