NCT05163288 - A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 30, 2022

Primary Completion Date

June 12, 2023

Study Completion Date

December 31, 2028

Conditions

Niemann-Pick Disease, Type C

Interventions

DRUG

N-Acetyl-L-Leucine

N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)

OTHER

Placebo

Matching Placebo Sachet

Trial Locations (14)

1105

RECRUITING

Amsterdam UMC, Amsterdam

3010

ACTIVE_NOT_RECRUITING

University Hospital Bern Inselspital, Bern

3050

ACTIVE_NOT_RECRUITING

The Royal Melbourne Hospital, Parkville

20246

COMPLETED

University of Hamburg, Hamburg

35389

ACTIVE_NOT_RECRUITING

University of Giessen, Giessen

48149

ACTIVE_NOT_RECRUITING

University Hospital Münster, Münster

55905

COMPLETED

Mayo Clinic, Rochester

65239

ACTIVE_NOT_RECRUITING

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

80539

NOT_YET_RECRUITING

Ludwig Maximilian University of Munich, München

128 08

ACTIVE_NOT_RECRUITING

First Faculty of Medicine, Charles University Hospital Prague, Prague

833 40

ACTIVE_NOT_RECRUITING

Comenius University in Bratislva, Bratislava

WC1N 3JH

ACTIVE_NOT_RECRUITING

Great Ormond Street Hospital, London

Unknown

ACTIVE_NOT_RECRUITING

Royal Free London NHS Foundation Trust, London

M13 9WL

ACTIVE_NOT_RECRUITING

Royal Manchester Children's Hospital, Manchester