Sandhoff Disease Clinical Trial
Official title:
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases. In this study no therapy is being offered.
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
Recruiting |
NCT04624789 -
Registry Gangliosidoses
|
||
Completed |
NCT03759665 -
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
|
Phase 2 | |
Active, not recruiting |
NCT04221451 -
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
|
Phase 3 | |
Completed |
NCT00672022 -
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
|
Phase 3 | |
Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
Active, not recruiting |
NCT04669535 -
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
|
Phase 1 | |
Recruiting |
NCT03333200 -
Longitudinal Study of Neurodegenerative Disorders
|
||
Active, not recruiting |
NCT05109793 -
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
|
||
Terminated |
NCT02030015 -
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
|
Phase 4 | |
Recruiting |
NCT00668187 -
A Natural History Study of the Gangliosidoses
|
||
Completed |
NCT01869270 -
Gene Therapy for Tay-Sachs Disease
|
N/A | |
Completed |
NCT01102686 -
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
|
Phase 1/Phase 2 | |
Terminated |
NCT01372228 -
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
|
Phase 1/Phase 2 |