Early Access Program with Arimoclomol for the Treatment of Niemann-Pick disease Type C in the US

Brief description of study

Niemann-Pick disease type C (NPC) is a rare, progressive and debilitating neurodegenerative disease arising from mutations in the NPC1 or NPC2 genes, encoding lysosomal proteins that are essential in intracellular transport and metabolism of lipids. The purpose of this Early Access Program (EAP) is to allow patients with limited treatment options the possibility to access medicine in development. This treatment uses an investigational drug named Arimoclomol. Investigational means that the Food and Drug Administration (FDA) has not yet approved the drug for this use. How well this investigational drug works or even if it is safe has not yet been proven through clinical trials.


Clinical Study Identifier: s20-01202
ClinicalTrials.gov Identifier: NCT04316637
Principal Investigator: Claire Miller.
Other Investigator: John T Wells.


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