Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Stopped early · Phase 4

Conditions studied: GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease

In brief

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.

Key facts

Study ID

NCT02030015

Run by

University of Minnesota

People needed

16

Starts

2015-12-22

Expected to finish

2019-07-31

Last updated by the study team

2021-04-14

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• A desire to not participate
• Patients who are older than 17 years will not be enrolled in this study.
• Children with severe renal impairment will not be enrolled in this study.
• Post-pubertal females who are pregnant, or who are unwilling to use highly-effective methods to prevent pregnancy, will be excluded from this study.
• Breast-feeding females will be excluded from this study.
• Subjects who have an allergy to miglustat or any of the components within the drug product will be excluded from this study.

Where it is running

• University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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