Sandhoff Disease Clinical Trial
Official title:
Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the symptomatic treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of GM2 Gangliosidosis. The Extension Phase was considered exploratory.
In the Parent Study, Patients will be assessed during three study phases: a baseline period, a 6-week treatment period, and a 6-week post-treatment washout period. If within 6 weeks prior to the initial screening visit, a patient has received any of the prohibited medications defined in the eligibility criteria (irrespective of the preceding treatment duration) a wash-out study-run-in of 6 weeks is required prior to the first baseline assessment. All patients will receive the study drug during the treatment period. For each individual patient, the Parent Study lasts for approximately 3.5 - 4 months during which there are 6 visits to the study site. This Extension Phase allows patients who have completed the Parent Study to, at the discretion of the Principal Investigator (PI), continue treatment with N-Acetyl-L-Leucine (IB1001). Patients will receive treatment with IB1001 for two one-year treatment periods, separated by a 6-week washout. All patients will receive the study drug during these two one-year treatment periods. For each individual patient, the Extension Phase lasts for approximately 25.5 months, during which there are 6 visits to the study site. ;
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
|
||
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 |
NCT04470713 -
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
|
||
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 |