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

Status: Recruiting

The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.

I'm interested

Age: Up to 18 years old
Healthy Volunteers:
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• no more than 17 years old
• documented infantile or juvenile gangliosidosis disease
Exclusion Criteria:

• severe kidney disease
• females who are pregnant or breast feeding
• females who are post puberty who are unwilling to use highly effective birth control
Interventions:

Other: Ketogenic Diet, Drug: miglustat

Conditions:

Rare Diseases

Keywords:

GM1 Gangliosidoses, GM2 Gangliosidoses, Sandhoff Disease, Tay-Sachs Disease

Contact(s): Jeanine Jarnes - utzx0002@umn.edu
Principal Investigator: Jeanine Jarnes
Phase: PHASE4
IRB Number: 1311M46101
System ID: 20890
See this study on ClinicalTrials.gov

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