Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

IntraBio Inc

Summary

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.

Description

This is a multinational, randomized, placebo-controlled, double-blinded, cross-over Phase III study that will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) versus Placebo for the treatment of Niemann-Pick type C disease (NPC). Patients will be assessed during three study periods: a baseline period (approximately 2-weeks), after which they will be randomized (1:1) to receive treatment with IB1001 or Placebo for approximately 12-weeks during the first intervention period ("Period I"). Following Period I, patients will crossover to receive the opposite treatment (IB1001 or Placeb…

Eligibility

Age range: 4+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: 1. Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness 2. Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of signing informed consent. 3. Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose and confirm to continue through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in \…

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

Locations (14)

Mayo Clinic
Rochester, Minnesota
The Royal Melbourne Hospital
Parkville, Victoria
First Faculty of Medicine, Charles University Hospital Prague
Prague
University of Giessen
Giessen
University of Hamburg
Hamburg
SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders
Höchheim