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Niemann-Pick Disease clinical trials at UCSF

3 in progress, 1 open to eligible people

Niemann-Pick disease is a rare, inherited disorder. UCSF is conducting a trial on a medicine called Trappsol Cyclo, given through an IV, to both kids and adults with a type of this disease. The trial is randomized, double-blind, and placebo-controlled.

Showing trials for
  • Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

    open to eligible people ages 3 years and up

    A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.

    Oakland, California and other locations

  • Early Access Program With Arimoclomol in US Patients With NPC

    Sorry, not accepting new patients

    NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.

    Oakland, California and other locations

  • Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

    Sorry, in progress, not accepting new patients

    A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick 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 participant's current prescribed best routine clinical care; participant's routine clinical 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 participants aged 6 to <24 months at study enrolment.

    Oakland, California and other locations

Our lead scientists for Niemann-Pick Disease research studies include .

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