Clinical Trial

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Recruiting Phase 3
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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.
Protocol Amendment History 12 amendments
This ClinicalTrials.gov record has been amended 12 times since 2021-12-06; most recent amendment 2026-03-30.
Status change: Active, Not Recruiting → Recruiting 2025-06-10
Status change: Recruiting → Active, Not Recruiting 2022-12-30
Status change: Not Yet Recruiting → Recruiting 2022-07-04
Trial Details
NCT Number NCT05163288
Lead Sponsor IntraBio Inc
Conditions Niemann-Pick Disease, Type C
Enrollment 53 participants
Start Date 2022-06-30
Primary Completion 2030-06-12 (estimated)
Study Completion 2030-12 (estimated)
Updated on ClinicalTrials.gov 2026-04-03