Hope for rare brain disease: new drug trial targets movement and daily life

NCT ID NCT05163288

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called N-acetyl-L-leucine (IB1001) in people aged 4 and older with Niemann-Pick disease type C, a rare genetic disorder that affects movement and thinking. The trial compares the drug to a placebo to see if it improves coordination and quality of life. After the main study, participants can receive the drug for up to 3 more years.

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Conditions

The condition(s) this trial relates to.

Niemann-Pick disease type C

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NIEMANN-PICK DISEASE, TYPE C

Contacts and locations

Show contact details

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

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