Journal
JOURNAL OF CHILD NEUROLOGY
Volume 25, Issue 3, Pages 300-305Publisher
SAGE PUBLICATIONS INC
DOI: 10.1177/0883073809344222
Keywords
miglustat; Niemann-Pick disease type C; efficacy; safety
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Funding
- Actelion Pharmaceuticals Ltd, Allschwil, Switzerland
- Actelion Pharmaceuticals Ltd
- Manchester Academic Health Sciences Centre (MAHSC)
- NIHR Manchester Biomedical Research Centre
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Niemann-Pick disease type C is a rare, genetic disease associated with impaired intracellular lipid trafficking and progressive neurological symptoms. Miglustat slowed disease progression in a 12-month randomized trial in juveniles and adults with Niemann-Pick disease type C, and in a Parallel, noncontrolled study in affected children. Here, the authors report the open-label extension to the pediatric study. Patients aged 4 to 12 years received open-label miglustat (dose adjusted for body surface area) for an initial 12 months, during a further 12-month extension, and a long-term, continued extension Phase. Efficacy assessments included horizontal saccadic eye movement, swallowing, and ambulation. Ten children completed 24 months treatment. Horizontal saccadic eye movement, ambulation, and swallowing were stabilized at 24 months. Analysis of key parameters of disease progression showed disease stability in 8 of 10 patients (80%). Miglustat stabilized neurological disease progression in pediatric patients with Niemann-Pick disease type C, with comparable safety and tolerability to that observed in adults and juveniles.
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