Status | Study |
Recruiting |
Study Name: Biomarker for GM1/GM2 - Gangliosidoses Condition: Gangliosidosis GM1-Gangliosidosis GM2-Gangliosidosis Date: 2014-10-23 |
Recruiting |
Study Name: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Condition: Adrenoleukodystrophy Batten Disease Mucopolysaccharidosis Date: 2014-09-23 Interventions: Biological: DUOC-01 Intrathecal administration of DUOC-01 |
Recruiting |
Study Name: Synergistic Enteral Regimen for Treatment of the Gangliosidoses Condition: GM1 Gangliosidoses GM2 Gangliosidoses Tay-Sachs Disease Date: 2013-12-17 Interventions: Drug: miglustat The Syner-G th |
Completed |
Study Name: Gene Therapy for Tay-Sachs Disease Condition: Tay Sachs Disease Sandhoff Disease Date: 2013-05-22 |
Completed |
Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Condition: Lysosomal Storage Disease Peroxisomal Disorder Date: 2012-06-20 Interventions: Drug: Campath-1H A daily dose |
Active, not recruiting |
Study Name: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Condition: Hurler Syndrome (MPS I) Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sens Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
Completed |
Study Name: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) Condition: Gangliosidoses, GM2 Sandhoff Disease Date: 2009-10-16 Interventions: Drug: Pyrimethamine Pyrimetham |
Withdrawn |
Study Name: A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis Condition: G(M2) Ganglioside Tay-Sachs Disease Ganglioside Date: 2008-05-15 Interventions: Drug: Pyrimethamine Pyrimethamine will be given PO once daily for 8 consecutive weeks. |
Completed |
Study Name: Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses Condition: GM2 Gangliosidoses Tay-Sachs Sandhof Date: 2008-05-02 Interventions: Drug: Zavesca (Miglustat) |
Recruiting |
Study Name: A Natural History Study of the Gangliosidoses Condition: Tay-Sachs Disease Sandhoff Disease Date: 2008-04-25 |