Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT05163288 · ClinicalTrials.gov registry record · Phase 3
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
A Phase 3 study of Niemann-Pick Disease, Type C, sponsored by IntraBio.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 53
- Enrollment target
- 14
- Study locations
NCT05163288 is a Phase 3 study of Niemann-Pick Disease, Type C that is actively recruiting participants, run by IntraBio. The registered enrollment target is 53 participants, below the 345-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (85% lower). The trial reports 14 study locations across 3 states.
The verdict
NCT05163288, a Phase 3 study of Niemann-Pick Disease, Type C, is actively recruiting participants, sponsored by IntraBio.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 53 participants
- Enrollment target
- 14
- Study locations
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?
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, categorized as Phase 3. The registered enrollment target is 53 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 345-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (85% lower). The listed sponsor is IntraBio, which has 6 total studies on file at ClinicalTrials.gov.
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.
NCT05163288 reports 14 study locations spanning 3 distinct geographic areas - top geographies include Other, Minnesota, Victoria.
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.
What interventions are being tested in trial NCT05163288?
The interventions under investigation include: Placebo (OTHER), N-Acetyl-L-Leucine (DRUG).
Who is sponsoring clinical trial NCT05163288?
This trial is sponsored by IntraBio, which has 6 total clinical trials registered on ClinicalTrials.gov.
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
Similar trials for Niemann-Pick Disease, Type C
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
RECRUITING
-
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
RECRUITING
-
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
COMPLETED · Phase 1
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.