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Clinical trial

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

NCT07399704 is registered in ClinicalTrials.gov with Azafaros B.V. as lead sponsor.

NCT NCT07399704
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Registry record

As filed in ClinicalTrials.gov.

Title
Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat
Lead sponsor
Azafaros B.V.
Phase
Phase 2
Overall status
Active, not recruiting
Start date
February 4, 2026
Completion date
August 7, 2030
Enrollment
21 (estimated)
Sites
3

Conditions

As the registry lists them; a condition with its own page is linked.

gm2 gangliosidosis, niemann-pick type c disease