About
Small Molecules
Areas of Focus
Categories
Small Molecule Pharmaceutical
Clinical Trials (5)
View allTotal Trials
5
As Lead Sponsor
5
As Collaborator
0
Total Enrollment
338
By Phase
PHASE3: 3PHASE2: 1
By Status
RECRUITING: 3ACTIVE_NOT_RECRUITING: 1COMPLETED: 1
Clinical Trials Pipeline5 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2021
2022
2023
2024
2025
2026
2027
2028
Today
Hover over trial bars to see details
Recent Trials
NCT07054515Lead Sponsor
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
PHASE3Recruiting
147 participants
Started: Jun 30, 2025 · Completed: Nov 4, 2027
NCT07082543Lead Sponsor
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
PHASE3Recruiting
75 participants
Started: Jun 30, 2025 · Completed: Nov 4, 2027
NCT07082725Lead Sponsor
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
PHASE3Recruiting
72 participants
Started: Jun 30, 2025 · Completed: Nov 4, 2027
NCT05758922Lead Sponsor
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease
PHASE2Active, not recruiting
13 participants
Started: Apr 24, 2023 · Completed: Mar 31, 2026
NCT05109793Lead Sponsor
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
N/ACompleted
31 participants
Started: Feb 22, 2022 · Completed: Sep 16, 2025
Locations (1)
Headquarters
DC Naarden, Netherlands
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