6 studies found for:    "GM2-gangliosidosis, AB variant" OR "Gangliosidoses GM2" OR "Tay-Sachs Disease, AB Variant"
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Rank Status Study
1 Unknown  Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
2 Completed Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Conditions: GM2 Gangliosidoses;   Tay-Sachs;   Sandhoff Disease
Intervention: Drug: Zavesca (Miglustat)
3 Recruiting Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Conditions: GM1 Gangliosidoses;   GM2 Gangliosidoses;   Tay-Sachs Disease;   Sandhoff Disease
Interventions: Drug: miglustat;   Behavioral: Ketogenic Diet
4 Withdrawn A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
Conditions: G(M2) Ganglioside;   Tay-Sachs Disease Ganglioside;   Sandhoff Disease Ganglioside
Intervention: Drug: Pyrimethamine
5 Completed Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Conditions: Gangliosidoses, GM2;   Sandhoff Disease;   Tay-Sachs Disease
Interventions: Drug: Pyrimethamine;   Drug: Leucovorin
6 Active, not recruiting Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Conditions: Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention: Biological: ALD-601

Indicates status has not been verified in more than two years