Gangliosidoses - 9 Studies Found

Completed : Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
: Gangliosidoses GM2
: 2007-01-04
: Drug: miglustat Target dose of 320 mg/m^2/day (divided in 3 doses) will be based on the Body Surface Are
Completed : Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
:
  • Gangliosidoses, GM2
  • Sandhoff Disease
  • Drug: Pyrimethamine Pyrimetham
Completed : Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
:
  • GM2 Gangliosidoses
  • Tay-Sachs

  • : 2008-05-02
    : Drug: Zavesca (Miglustat)
Recruiting : A Natural History of Late Onset Tay-Sachs Disease
: GM2 Gangliosidosis
: 2016-07-19
Withdrawn : A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
:
  • G(M2) Ganglioside
  • Tay-Sachs Disease Ganglioside
  • Sandhoff D
    : 2008-05-15
    : Drug: Pyrimethamine Pyrimethamine will be given PO once daily for 8 consecutive weeks.
Completed : Gene Therapy for Tay-Sachs Disease
:
  • Tay Sachs Disease
  • Sandhoff Disease
  • Late Onset Tay Sachs Di
    : 2013-05-22
Completed : Diagnostic and Screening Study of Genetic Disorders
:
  • Tay-Sachs Disease
  • Porphyria, Erythropoietic
  • Leukodystrophy
    : 2000-07-05
Active, not recruiting : Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
:
  • Hurler Syndrome (MPS I)
  • Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sens
    : 2011-06-10
    : Biological: FCRx infusion Enriched hematopoetic stem cell infusion
Withdrawn : Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
:
  • Lysosomal Storage Diseases
  • Inborn Errors of Metabolism

: 2009-10-20
: Biological: ALD-601 ALD-601 is manufactured by Aldagen from the 20% compartment of the selected umbilica