Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Status:
Active, not recruiting
Trial end date:
2022-05-08
Target enrollment:
Participant gender:
Summary
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.
The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study
including patients aged 6 to <24 months at study enrolment.