{"id":108,"date":"2023-08-10T15:20:33","date_gmt":"2023-08-10T15:20:33","guid":{"rendered":"https:\/\/pediatricbrainfoundation.org\/archive\/new-clinical-trial-niemann-pick-c\/"},"modified":"2023-08-10T15:20:33","modified_gmt":"2023-08-10T15:20:33","slug":"new-clinical-trial-niemann-pick-c","status":"publish","type":"page","link":"https:\/\/pediatricbrainfoundation.org\/archive\/new-clinical-trial-niemann-pick-c\/","title":{"rendered":"New Clinical Trial for Niemann Pick C"},"content":{"rendered":"
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New Clinical Trial for Niemann Pick C<\/h1>\n<\/section><\/div>\n
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\n Monday, November 23, 2015<\/span>  \u00b7  Posted by Kristin Pence <\/div>\n

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Vtesse, Inc. Initiates Phase 2b\/3 Clinical Trial of
\nVTS-270 for Treatment of Niemann-Pick Type C1 (NPC) Disease<\/span><\/b><\/p>\n

NPC is a progressive,
\nirreversible, chronically debilitating \u2013 and ultimately lethal \u2013 genetic
\ndisease. It is caused by a defect in lipid transportation within the cell,
\nwhich leads to excessive accumulation of lipids in the brain, liver and spleen.
\nVTS-270, has shown promise in pre-clinical and clinical studies as a potential
\ntreatment for NPC. It is a well-characterized mixture of
\n(2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in
\npre-clinical and clinical studies at the National Institutes of Health, as well
\nas under individual compassionate-use Investigational New Drug applications
\n(iINDs) and in other academic labs. Most recently, Vtesse announced positive
\nclinical results from the on-going Phase 1 study that demonstrated VTS-270
\nstabilizes the overall measures of NPC while improving certain disease
\nmeasures. NPC is a genetic disease affecting an estimated one in 100,000 to
\n150,000 children and is often misdiagnosed and\/or under-diagnosed. Affected
\npatients are usually identified in early childhood with ataxia, exhibit
\nprogressive impairment of motor and intellectual function, and often die before
\nadulthood.Vtesse has worked extensively with regulators in the United States
\nand Europe with the goal of conducting its pivotal study under one global
\nprotocol that will evaluate safety and efficacy of VTS-270 to support approval
\nof the drug by regulatory agencies in both regions.<\/p>\n

Vtesse\u2019s Phase 2b\/3
\nprospective, randomized, double-blind, sham-controlled trial of VTS-270 will be
\nconducted in patients affected by NPC disease. The trial will take place in up
\nto 20 centers in the United States and Europe. It is a three-part, efficacy and
\nsafety trial of VTS-270, administered by the lumbar intrathecal route (IT)
\nevery two weeks, with a planned enrollment of approximately 51 patients (in
\nParts A and B). In Part A of the study, researchers will evaluate three
\ndifferent dose levels of VTS-270 versus sham-control to determine the dose
\nlevel for Parts B and C. All participants in the pivotal trial will be eligible
\nto receive treatment with VTS-270 in Part C, the open-label extension, until
\nthe time of regulatory decisions.<\/p>\n

Preliminary analyses of the
\nPhase 1 trial, conducted post-hoc, suggest that the rate of disease progression
\nhad slowed down (based on a standardized measure) in children treated with
\nVTS-270 as compared to the rate in an age- and disease severity-matched cohort
\nobtained from a separate natural history study of NPC patients.<\/p>\n

More information on this
\nPhase 2b\/3 study can be found at Vtesse\u2019s clinical trial website:\u00a0www.theNPCstudy.com<\/span><\/a>. Vtesse launched the website this summer as a resource to
\npatients, parents, caregivers and physicians. \u00a0If you would like to
\ncontact Vtesse directly, please reach out to Carrie Burke at carrie@vtessepharma.com<\/span><\/a> or 240-801-9268. \u00a0<\/p>\n


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