A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Recruiting
Phase 3
Interventional Study
Gangliosidoses, GM2
Gangliosidosis, GM1
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 4 and older
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 75 (estimated)
- Sponsor
- Azafaros B.V. · Industry
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About This Trial
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
* Male and female participants aged 4 years and older at the time of informed consent
* Onset of neurological symptoms from 1 to 10 years
* Disability level at Baseline: Ataxic disturbances with a total SARA…
Contacts