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Oral nizubaglustat for late‑infantile and juvenile GM1 and 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

Phase 3 Interventional Azafaros A.G. · NCT07082543

This 18‑month randomized, double‑blind, placebo‑controlled trial will test whether daily oral nizubaglustat helps children and young people (age ≥4) with late‑infantile or juvenile GM1 or GM2 gangliosidosis reduce ataxia and other neurological symptoms.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment75 (estimated)
Ages4 Years and up
SexAll
SponsorAzafaros A.G. Industry-sponsored
Locations36 sites (Oakland, California and 35 other locations)
Trial IDNCT07082543 on ClinicalTrials.gov

What this trial studies

Participants with genetically confirmed late‑infantile or juvenile GM1 or GM2 gangliosidosis and measurable ataxia are randomly assigned to once‑daily oral nizubaglustat or matching placebo for 18 months at multiple US centers. The trial is double‑blind and includes regular clinical visits to measure ataxic manifestations (using standardized scales), other neurologic outcomes, pharmacokinetics, pharmacodynamics, and safety. Key eligibility includes symptom onset between ages 1–10 and a SARA score between 3 and 30 at baseline. Safety monitoring includes laboratory tests and adverse event surveillance throughout treatment.

Who should consider this trial

Good fit: Children and adolescents aged 4 years and older with genetically confirmed late‑infantile or juvenile GM1 or GM2 gangliosidosis, symptom onset between 1 and 10 years, and measurable ataxia (SARA score 3–30) are the best candidates.

Not a fit: Patients with adult‑onset disease or minimal/no ataxia, those with moderate to severe hepatic or renal impairment, other neurologic diseases, very low platelet counts, or body weight under 10 kg are unlikely to benefit or may be ineligible.

Why it matters

Potential benefit: If successful, nizubaglustat could slow or reduce ataxia and improve motor function and daily activities in affected children and adolescents.

How similar studies have performed: Related substrate‑reduction and other therapies for lysosomal storage disorders have shown promise in early studies, but large definitive benefits specifically in GM1/GM2 gangliosidosis remain limited.

Eligibility criteria

Show full inclusion / exclusion 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 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 Criteria:

* 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)

Where this trial is running

Oakland, California and 35 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Gangliosidoses, GM2Gangliosidosis, GM1Nizubaglustat
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.