City

Minneapolis (1)

Washington (1)

Condition

GM2 Gangliosidoses (2)

Sandhoff Disease (2)

GM1 Gangliosidoses (1)

Tay-Sachs (1)

Tay-Sachs Disease (1)

Overall Status

Completed (1)

Terminated (1)

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Phase

phase 3 (1)

phase 4 (1)

Sponsor

Children's National Research Institute (1)

University of Minnesota (1)

Collaborator

Actelion (1)

Lysosomal Disease Network (1)

National Center for Advancing Translational Sciences (NCATS) (1)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (1)

National Institute of Neurological Disorders and Stroke (NINDS) (1)

Rare Diseases Clinical Research Network (1)

Intervention

ketogenic diet (1)

miglustat (1)

zavesca (miglustat) (1)

Study Documents

Intervention Type

drug (2)

other (1)

Funder Type

Other (2)

Study Type

Interventional (2)

gm2 gangliosidoses

Showing 1 - 2 of 2

Trials per page:

STUDY

CONDITIONS

INTERVENTIONS

LOCATIONS

LAST UPDATED

GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease Trial in Minneapolis (miglustat, Ketogenic Diet)

Terminated

Has Results

GM1 Gangliosidoses
+3 more

miglustat
Ketogenic Diet

Minneapolis, Minnesota

University of Minnesota

Mar 18, 2021

GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease Trial in Washington (Zavesca (Miglustat))

Completed

GM2 Gangliosidoses
+2 more

Zavesca (Miglustat)

Washington, District of Columbia

Children's National Medical Center

May 5, 2008