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
IntraBio Inc
IntraBio Inc (Responsible Party)
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
ZevraDenmark
ZevraDenmark (Responsible Party)
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)
Azafaros A.G.
Azafaros A.G. (Responsible Party)
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
Share This Content