This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick …

Niemann-Pick Disease, Type C

~Estimated

~$10 – $675

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT00344331

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…

Niemann-Pick Disease, Type C

Documented

Up to $190

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT05588167

Phase 3

Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick…

Niemann-Pick Disease, Type C

~Estimated

~$10 – $8,750

IntraBio IncNCT05163288