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Phase 3, Double-blind, Randomized, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of 2000mg/kg of Trappsol Cyclo (Hydroxypropyl-B-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients With Niemann-Pick Disease Type C1 (TransportNPC)

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Phase 3, Double-blind, Randomized, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of 2000mg/kg of Trappsol Cyclo (Hydroxypropyl-B-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients With Niemann-Pick Disease Type C1 (TransportNPC)

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

Latest Information Update: 11 Feb 2025

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At a glance

  • Drugs Hydroxypropyl betadex (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Registrational; Therapeutic Use
  • Acronyms TransportNPC
  • Sponsors Cyclo Therapeutics

    • Most Recent Events

    • 07 Feb 2025 According to Cyclo Therapeutics media release, preliminary data from TransportNPC open label sub study presented at the 21st Annual WORLDSymposium
    • 07 Feb 2025 Preliminary results from TransportNPC open label sub study presented in the Cyclo Therapeutics Media Release
    • 14 Jan 2025 According to Cyclo Therapeutics media release, data from this trial will be presented at the 21st Annual WORLDSymposium

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