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

NCT05163288 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 53

Last updated 2026-04-03

No results posted yet for this study

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.

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

Sponsors & Collaborators

  • IntraBio Inc

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-30
Primary Completion
2030-06-12
Completion
2030-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Czechia
  • Germany
  • Netherlands
  • Slovakia
  • Switzerland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05163288 on ClinicalTrials.gov