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Sandhoff Disease - 14 Studies Found

Status Study
Completed Study Name: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Condition:
  • Gangliosidoses, GM2
  • Sandhoff Disease
  • Drug: Pyrimethamine Pyrimetham
Completed Study Name: Gene Therapy for Tay-Sachs Disease
Condition:
  • Tay Sachs Disease
  • Sandhoff Disease
  • 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: 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
Recruiting Study Name: A Natural History Study of the Gangliosidoses
Condition:
  • Tay-Sachs Disease
  • Sandhoff Disease
  • Gangliosidosis
  • GM1-Gangliosidosis
  • GM2-Gangliosidosis

    • Date: 2014-10-23
Completed Study Name: HSCT for High Risk Inherited Inborn Errors
Condition:
  • Adrenoleukodystrophy
  • Metachromatic Leukodystrophy
  • Globoid
    Date: 2006-09-29
    Interventions:
    • Drug: Clofarabine days -7 thro
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
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
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