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

Launched by AZAFAROS A.G. · Jul 16, 2025

Keywords

ClinConnect Summary

This clinical trial is testing an oral medicine called nizubaglustat (AZ-3102) to see if it is safe and helpful for children and young people who have certain rare genetic conditions called GM1 or GM2 gangliosidosis. These conditions affect the brain and nervous system, usually starting between 1 and 10 years of age, and can cause problems with movement and coordination. The study will last about 18 months and will compare nizubaglustat to a placebo (a pill without medicine) to find out if the drug can improve symptoms or slow down the disease.

Children and young people aged 4 years and older who have a confirmed diagnosis of GM1 gangliosidosis or related GM2 conditions like Tay-Sachs or Sandhoff disease might be able to join. Participants should have some difficulties with movement but still be within a certain range of disability, and they need to have stable health aside from their gangliosidosis. Both boys and girls can take part, but those who can have children will need to follow specific birth control guidance during the study. If eligible, participants will be randomly assigned to receive either the medicine or placebo without knowing which one, and they will be closely monitored throughout the study to check for safety and any benefits from the treatment.

Gender

ALL

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 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)