Clinical Trials

Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 16
  • Start Date: March 2021
An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human β-Galactosidase cDNA for Treatment of GM1 Gangliosidosis
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Clinical Trial
  • Status: Recruiting
  • Participants: 40
  • Start Date: June 12, 2020
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Clinical Trial
  • Status: Recruiting
  • Participants: 40
  • Start Date: June 8, 2020
Eight At One Stroke: Attention Gangliosidoses A Registry Study for Patients With Gangliosidoses
Clinical Trial
  • Status: Not yet recruiting
  • Participants: 30
  • Start Date: June 1, 2020
Natural History of Morquio B and Late-Onset GM1 Gangliosidosis
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 30
  • Start Date: April 20, 2020
Natural History Study Using Interview and Video Capture of Infantile and Juvenile GM1 Gangliosidosis (GM1)
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Other, Procedure, Drug, Diagnostic Test, Biological, Behavioral
  • Participants: 45
  • Start Date: August 19, 2019
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 39
  • Start Date: June 7, 2019
Effects of N-Acetyl-L-Leucine on GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
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