City

Minneapolis (1)

Pittsburgh (1)

Overall Status

Recruiting (1)

Terminated (1)

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Phase

phase 4 (1)

Sponsor

University of Minnesota (1)

University of Pittsburgh (1)

Collaborator

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

hematopoetic stem cell transplantation (1)

ketogenic diet (1)

miglustat (1)

palliative care (1)

Study Documents

Intervention Type

other (2)

biological (1)

drug (1)

Funder Type

Other (2)

Study Type

Interventional (1)

Observational (1)

gm1 gangliosidoses

Showing 1 - 2 of 2

Trials per page:

STUDY

CONDITIONS

INTERVENTIONS

LOCATIONS

LAST UPDATED

The Natural History of Metachromatic Leukodystrophy

Recruiting

MLD
+26 more

Palliative Care
Hematopoetic Stem Cell Transplantation

Pittsburgh, Pennsylvania

The Program for the Study of Neurodevelopment in Rare Disorders,

Nov 2, 2021

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