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Niemann-Pick Disease clinical trials at UC Health

3 in progress, 1 open to eligible people

Showing trials for
  • Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

    open to eligible people ages 18 years and up

    The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.

    at UCSF

  • Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C

    Sorry, in progress, not accepting new patients

    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.

    at UCSF

  • VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

    Sorry, in progress, not accepting new patients

    This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Part A and B, two out of every three patients will receive the study drug. The doctor will give the third patient an injection with nothing in it (sham control). In Part C, all participants will receive study drug.

    at UCSF

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