Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
NCT05163288 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- OTHER Placebo
- DRUG N-Acetyl-L-Leucine
Study Locations (14)
Other
- 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
- Ludwig Maximilian University of Munich — München
- University Hospital Münster — Münster
- Amsterdam UMC — Amsterdam
- Comenius University in Bratislva — Bratislava
- University Hospital Bern Inselspital — Bern
- Great Ormond Street Hospital — London
- Royal Free London NHS Foundation Trust — London
- Royal Manchester Children's Hospital — Manchester
Minnesota
- Mayo Clinic — Rochester
Victoria
- The Royal Melbourne Hospital — Parkville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 53 participants |
| Start Date | 2022-06-30 |
| Est. Completion | 2028-12 |
| Phase | Phase 3 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05163288
The ClinicalTrials.gov registry entry for NCT05163288 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 53 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is IntraBio, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Niemann-Pick Disease, Type C appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05163288 reports 14 study locations spanning 3 distinct geographic areas — top geographies include Other, Minnesota, Victoria. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05163288 about?
NCT05163288 is a clinical study titled "A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C". 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) compar...
What is the current status of trial NCT05163288?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 53 participants. The study started on 2022-06-30. Estimated completion is 2028-12.
What conditions does trial NCT05163288 study?
This clinical trial studies the following conditions: Niemann-Pick Disease, Type C. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05163288?
The interventions under investigation include: Placebo (OTHER), N-Acetyl-L-Leucine (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05163288?
This trial is sponsored by IntraBio, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05163288 being conducted?
This trial has 14 study locations across Minnesota, Victoria. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.