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.
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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

Patient Advocacy Representative

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info@azafaros.com

CONTACT

Contact for Healthcare Professionals

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medinfo@azafaros.com

CONTACT