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Arimoclomol double-blind randomized placebo-controlled study in Niemann-Pick disease type C
2015-004438-93Phase 2Completed
- Sponsor
- Orphazyme A/S
- Start
- Mar 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial in Niemann-Pick disease type C (NPC) comparing Arimoclomol with Placebo (2 arms). Primary endpoint: Change in NPC disease severity based on the 5-domain NPCCSS. Started Mar 2016; status: completed.
02
Drugs
arimoclomol
03
Conditions
Niemann-Pick disease type C (NPC)
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