Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Status:
Terminated
Trial end date:
2019-07-31
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 4
Details
Lead Sponsor:
University of Minnesota University of Minnesota - Clinical and Translational Science Institute
Collaborators:
Lysosomal Disease Network National Center for Advancing Translational Science (NCATS) National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) National Institute of Neurological Disorders and Stroke (NINDS) Rare Diseases Clinical Research Network