Clinical Trials: Active, Not Recruiting

Disclaimer: Information regarding clinical trials for NPC disease is obtained from the https://clinicaltrials.gov website and may not capture every trial. Please see links for additional information about specific clinical trials.

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

ClinicalTrials.gov ID
NCT05163288
Last Update Posted
September 17, 2023
Sponsor
IntraBio Inc
Information provided by
IntraBio Inc (Responsible Party)
Study Description:

The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

ClinicalTrials.gov ID
NCT02612129
Last Update Posted
June 9, 2023
Sponsor
ZevraDenmark
Information provided by
ZevraDenmark (Responsible Party)
Study Description:

The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the participant's current prescribed best routine clinical care; participant's routine clinical care may, or may not, include miglustat.

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease (RAINBOW)

ClinicalTrials.gov ID
NCT05758922
Last Update Posted
December 15, 2023
Sponsor
Azafaros A.G.
Information provided by
Azafaros A.G. (Responsible Party)
Study Description:

This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients.

More information can be found on https://clinicaltrials.gov

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