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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: Recruiting Phase 3 study of Niemann-Pick Disease, Type C, sponsored by IntraBio.

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. According to ClinicalTrials.gov, the official US trial registry.

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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

Niemann-Pick Disease, Type C

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

FieldValue
Enrollment Target 53 participants
Start Date 2022-06-30
Est. Completion 2028-12
Phase Phase 3
IntraBio

6 total trials

What NCT05163288 shows while recruiting

NCT05163288 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 53 participants, a relatively small participant target, below the 345-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (85% lower).

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 lists 14 locations in 3 states (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.

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More recruiting context for NCT05163288

Source: ClinicalTrials.gov NCT05163288, the US trial registry maintained by the National Library of Medicine. NCT05163288 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.