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Recruiting NCT07082543

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

Conditions: Gangliosidoses, GM2, Gangliosidosis, GM1

Sex: All
Ages: 4 Years – N/A
Healthy volunteers: No
Phase: PHASE3
Enrollment: 75
Sponsor: Azafaros B.V.

Location: UCSF Children's Hospital and Research Center at Oakland Oakland California

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 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 \

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View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07082543). StuddyBuddy aggregates publicly available trial information.