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
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 Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Azafaros A.G.
75 participants
Jun 30, 2025
INTERVENTIONAL
Conditions
Summary
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
Eligibility
Inclusion Criteria6
- 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 score of ≥3 and ≤30 at Baseline
- Females of childbearing potential who are sexually active willing to follow the contraceptive guidance
- Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance
Exclusion Criteria9
- A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results
- Body weight of \<10 kg
- The presence of another neurologic disease
- The presence of moderate or severe hepatic impairment
- The presence of moderate or severe renal impairment
- Platelet count of \<100x10\^9/L
- The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and/or a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline
- Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening
- A positive serum pregnancy test (for women of childbearing potential)
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Interventions
Nizubaglustat
Placebo
Locations(36)
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NCT07082543