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Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C

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Trial Profile

Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C

Status: Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 22 Apr 2024

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At a glance

  • Drugs Arimoclomol (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Registrational; Therapeutic Use
  • Sponsors Orphazyme; Zevra Therapeutics

    • Most Recent Events

    • 19 Apr 2024 This study has been Completed in Denmark, According to European Clinical Trials Database record.
    • 15 Apr 2024 According to a Zevra Therapeutics Media release, data from the study were presented the data at the 2024 American Academy of Neurology Annual Meeting (AANAM)
    • 28 Mar 2024 According to a Zevra Therapeutics Media release,Research and development (R&D) expenses were $11.4 million for Q4 2023, compared to $6.5 million in Q4 2022. The increase in R&D expenses was primarily driven by the ongoing work supporting to support the arimoclomol NDA which was resubmitted to the FDA in December 2023.
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