CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 33 enrolled
Drug / intervention
IB1001drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03759639
NCT03759639Phase 2Completed
CompareCT.gov

Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.

IntraBio Inc·interventional·Posted Nov 30, 2018·Updated Nov 28, 2023

In Brief

A Phase 2 clinical trial evaluating IB1001 for Niemann-Pick Disease, Type C. Completed, enrolled 33 participants across 9 sites in 5 countries.

Detailed Summary

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of NPC. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsNiemann-Pick Disease, Type C
CountriesGermany, Slovakia, Spain, United Kingdom, United States
Collaborators--

Timeline

Phase 2CompletedFinished
20192020202120222023202420252026
First PostedNov 30, 2018
Enrollment StartSep 4, 2019
Primary CompletionNov 7, 2022
TodayJul 2, 2026
Enrollment to primary: 3.2 yearsPosted 7.6 years ago

Interventions

IB1001drug

IB1001 (N-Acetyl-L-Leucine) is a modified amino-acid ester that is orally administered.