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Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
Sponsor: Azafaros B.V.
Summary
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to continue nizubaglustat treatment.
Official title: An Open-label, Roll-over Trial to Evaluate the Long-term Safety and Tolerability of Nizubaglustat (AZ-3102) in Participants With Rare Lysosomal Disorders Who Have Participated in Previous Nizubaglustat Trials and Wish to Continue Nizubaglustat Treatment
Key Details
Gender
All
Age Range
4 Years - Any
Study Type
INTERVENTIONAL
Enrollment
218
Start Date
2026-12-10
Completion Date
2030-04-01
Last Updated
2026-10-21
Healthy Volunteers
No
Interventions
AZ-3102
Daily oral intake of nizubaglustat (AZ-3102) dispersible tablets