-
A Study to Establish a Rare Diseases Registry for Niemann-Pick Disease Types A, B and C
Rochester, MN
The purposes of this study are to establish the natural history of the 3 diseases including their characteristics, management and outcomes; to assess clinical effectiveness of management and quality of care; to provide an inventory of patients for recruitment to interventional studies; to establish genotype-phenotype correlations; and to support post marketing obligations for approved therapies.
-
A Study To Evaluate N-Acetyl-L-Leucine For Niemann-Pick Disease, Type C (NPC)
Rochester, MN
The purpose of this study is to assess the safety and effectiveness of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
-
Nizubaglusta In Late-Infantile And Juvenile Forms Of Niemann-Pick Type C Disease And GM1 Gangliosidosis Or GM2 Gangliosidosis
Rochester, MN
The purpose of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of NPC disease or late-infantile/juvenile-onset forms of GM1 or GM2 gangliosidosis.
-
Arimoclomol Prospective Study In Patients Diagnosed With NiemannPick Disease Type C
Rochester, MN
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). 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 patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.