Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)

Status: Terminated
Location: See location...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 4

The investigators hypothesize that a combination therapy using miglustat and the ketogenic diet for infantile and juvenile patients with gangliosidoses will create a synergy that 1) improves overall survival for patients with infantile or juvenile gangliosidoses, and 2) improves neurodevelopmental clinical outcomes of therapy, compared to data reported in previous natural history studies. The ketogenic diet is indicated for management of seizures in patients with seizure disorders. In this study, the ketogenic diet will be used to minimize or prevent gastrointestinal side-effects of miglustat. A Sandhoff disease mouse study has shown that the ketogenic diet may also improve central nervous system response to miglustat therapy (see Denny in Citations list below). Patients with infantile and juvenile gangliosidoses commonly suffer from seizure disorders, and use of the ketogenic diet in these patients may therefore also improve seizure management.

Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: No

• Subjects must have a documented infantile or juvenile gangliosidosis disease.

• Age: 17 years or less at time of enrollment

• Subjects and their caregivers must be willing to work with a ketogenic diet team for management of the subject's ketogenic diet.

United States
University of Minnesota
Time Frame
Start Date: December 22, 2015
Completion Date: July 31, 2019
Target number of participants: 16
Experimental: Syner-G Therapy Regimen
The Syner-G therapy regimen includes switching the research subject to a full-time ketogenic diet, and daily treatment with orally-administered miglustat, for the duration of the 60-month study.
Chester B. Whitley, Jeanine R. Jarnes, Jeanine R. Utz
Collaborators: National Center for Advancing Translational Sciences (NCATS), Lysosomal Disease Network, National Institute of Neurological Disorders and Stroke (NINDS), Rare Diseases Clinical Research Network, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Leads: University of Minnesota

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