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N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

NCT03759639 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2023-11-28

Study results available
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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.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

IB1001

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

Sponsors & Collaborators

  • IntraBio Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-04
Primary Completion
2022-11-07
Completion
2022-11-07
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Slovakia
  • Spain
  • 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 NCT03759639 on ClinicalTrials.gov